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Oyster Point Pharma Announces Enrollment of First Subject in the OLYMPIA Phase 2 Clinical Trial of OC-01 (varenicline) Nasal Spray for Patients with…

Posted: June 22, 2021 at 1:52 am


PRINCETON, N.J., June 21, 2021 (GLOBE NEWSWIRE) -- Oyster Point Pharma, Inc. (Nasdaq: OYST), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class therapies to treat ophthalmic diseases, today announced enrollment of the first subject in the OLYMPIA Phase 2 clinical trial of OC-01 (varenicline) nasal spray for the treatment of Stage 1 Neurotrophic Keratopathy (NK).

This is an exciting milestone as we continue to develop this potentially new treatment option for patients with Stage 1 Neurotrophic Keratopathy, said Jeffrey Nau, Ph.D., MMS, president and chief executive officer of Oyster Point Pharma. We believe that NK affects more patients than are currently diagnosed as the disease has the potential to be undiagnosed or misdiagnosed. Stage 1 NK patients may present with additional ocular surface issues, including dry eye disease, which affects 38 million1 patients.

The OLYMPIA Phase 2 study is a multicenter, randomized, double-masked, placebo-controlled clinical trial to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray in subjects with Mackies Classification Stage 1 Neurotrophic Keratopathy. The study is expected to enroll approximately 100 subjects at approximately 18 U.S. sites. In this clinical trial, OC-01 (varenicline) nasal spray will be administered three times a day, as compared to placebo (vehicle) nasal spray. The pre-specified primary endpoint of the trial will be the percentage of subjects who achieve complete resolution of fluorescein corneal staining at Day 56.

NK is characterized as a degenerative disease of the cornea due to impairment of trigeminal innervation that results in corneal epithelial damage, said Marian Macsai, M.D., chief medical officer of Oyster Point Pharma. We believe that OC-01 nasal spray may activate natural tear production through the trigeminal parasympathetic pathway, bypassing the impaired corneal nerves to stimulate the production of natural tear film and potentially improve corneal sensitivity and healing.

About Oyster Point PharmaOyster Point Pharma is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class therapies to treat ophthalmic diseases.

About OC-01 (varenicline) Nasal SprayOC-01 (varenicline) nasal spray is a highly selective cholinergic agonist being developed as a multidose preservative-free nasal spray to treat the signs and symptoms of dry eye disease and neurotrophic keratopathy. The parasympathetic nervous system, the rest and digest system of the body, controls tear film homeostasis partially via the trigeminal nerve, which is accessible within the nose. Administered as a preservative-free, aqueous nasal spray, in pre-clinical and clinical studies, OC-01 (varenicline) nasal spray was shown to have a novel mechanism of action with activation of the trigeminal parasympathetic pathway in the nasal cavity to activate natural tear film production. The human tear film is a complex mixture of more than 1,500 different proteins, including growth factors and antibodies, as well as numerous classes of lipids and mucins. This complex tear film is responsible for forming the primary refracting surface of the eye, as well as protecting and moisturizing the cornea. In December 2020, Oyster Point submitted to the U.S. Food and Drug Administration (FDA) a New Drug Application (NDA) for OC-01 (varenicline) nasal spray for the treatment of signs and symptoms of dry eye disease. The Prescription Drug User Fee Act (PDUFA) target action date is October 17, 2021, with a planned U.S. launch of OC-01 (varenicline) nasal spray in this indication in the fourth quarter of 2021, if approved by the FDA. OC-01 (varenicline) nasal spray is an investigational new drug and has not been approved for any use in any country. The safety and efficacy of OC-01 (varenicline) nasal spray have not been established.

About Neurotrophic KeratopathyNeurotrophic Keratopathy (NK) is a rare disease characterized by decreased corneal sensitivity and poor corneal healing. The most common causes of corneal sensation loss are viral infection (herpes simplex virus and herpes zoster keratoconjunctivitis), followed by chemical burns, physical injuries, and ocular surface surgery. In addition, systemic diseases such as diabetes and multiple sclerosis may decrease sensory nerve function or damage sensory fibers. NK can be classified broadly into three stages: Stage 1 (mild) consists of ocular surface irregularities and reduced vision, Stage 2 (moderate) exhibits a non-healing persistent defect of the corneal epithelium, and Stage 3 (severe) exhibits corneal ulceration, which may progress to corneal melting and perforation. If not adequately addressed, NK can lead to vision loss and a breakdown of corneal integrity, potentially leading to cornea transplantation.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that reflect the current beliefs, expectations and assumptions of the Company regarding the future of the Companys business, our future plans and strategies, regulatory approvals, clinical results, future financial condition and other future conditions. All statements other than statements of historical facts contained in this press release, including express or implied statements regarding product candidates, regulatory approvals, planned pre-clinical studies and clinical trials, expected results of pre-clinical studies or clinical trials, and their timing and likelihood of success, expected research and development costs, as well as plans and objectives of management for future operations, are forward-looking statements. The words if approved, may, will, should, would, expect, plan, anticipate, could, intend, target, project, contemplate, believe, estimate, predict, potential or continue or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, among other things: the uncertainties inherent in pharmaceutical research and development, including pre-clinical study and clinical trial results and additional analysis of existing data, and the likelihood of our pre-clinical studies and clinical trials demonstrating the safety and efficacy of our product candidates, and other positive results; the timing of initiation of our future clinical trials, and the reporting of data from our current and future trials; the timing or likelihood of regulatory filings and approvals for our product candidates; our ability to obtain and maintain regulatory approvals of our product candidates; our plans relating to commercializing our product candidates, if approved; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of our product candidates; our plans relating to the further pre-clinical and clinical development and manufacturing of our product candidates, including additional indications which we may pursue; the prevalence of dry eye disease and neurotrophic keratopathy and the size of the market opportunity for our product candidates; the expected potential benefits of strategic collaborations with third parties and our ability to attract collaborators with development, regulatory and commercialization expertise; our ability to recruit and retain key personnel needed to develop and commercialize our product candidates, if approved, and to grow our company; existing regulations and regulatory developments in the United States and other jurisdictions; our plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available; our continued reliance on third parties to conduct additional clinical trials of our product candidates, and for the manufacture of our product candidates for pre-clinical studies and clinical trials; the accuracy of our estimates regarding expenses, future revenue, capital requirements and needs for additional financing; our financial performance; market conditions; the sufficiency of our existing capital resources to fund our future operating expenses and capital expenditure requirements; and other risks described in the Risk Factors section included in our public filings that we have made and will make with the Securities and Exchange Commission. The Company is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

1- Market Scope 2020 Dry Eye Products Report: A Global Market Analysis for 2019 to 2025

Investor Contact:Tim McCarthyLifeSci Advisors, LLC(212) 915-2564investors@oysterpointrx.com

Media Contact:Sheryl Seapy, Real Chemistry(213) 262-9390sseapy@realchemistry.com

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Oyster Point Pharma Announces Enrollment of First Subject in the OLYMPIA Phase 2 Clinical Trial of OC-01 (varenicline) Nasal Spray for Patients with...

Spruce Biosciences Announces Publication of Phase 2 Results for Tildacerfont in Journal of Clinical Endocrinology and Metabolism – Business Wire

Posted: at 1:52 am


SAN FRANCISCO--(BUSINESS WIRE)--Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today announced the publication of the results from two Phase 2 clinical studies investigating tildacerfont in adult patients with classic CAH in the Journal of Clinical Endocrinology and Metabolism.

The data published in the Journal of Clinical Endocrinology and Metabolism demonstrates the potential of tildacerfont to reduce androgen excess without increasing the total daily glucocorticoid dose in patients with classic CAH, said Kyriakie Sarafoglou, MD, Associate Professor, Department of Pediatrics Divisions of Endocrinology and Genetics & Metabolism at the University of Minnesota Medical School.

SPR001-201 was an open-label, multi-dose, Phase 2a dose-escalation study which evaluated the ability of tildacerfont to lower adrenocorticotropic hormone (ACTH), 17-hydroxyprogesterone (17-OHP), and androstenedione (A4) at doses ranging from 200mg daily to 1,000mg daily in patients with congenital adrenal hyperplasia due to 21-hydroxylase deficiency. SPR001-202 was an open-label, 12-week Phase 2a clinical trial, which assessed the ability of a daily dose of 400mg of tildacerfont to lower ACTH, 17-OHP, and A4 over a 12-week dosing period. SPR001-201 and SPR001-202 comprised the entire Phase 2a clinical development program for tildacerfont in adult classic CAH. In the studies, efficacy was evaluated by changes from baseline in ACTH, 17-OHP, and A4 according to baseline A4 2x upper limit of normal (ULN), denoted as baseline good disease control, or A4 >2x ULN, denoted as baseline poor disease control. Safety was evaluated using adverse events and laboratory assessments.

The results of the studies showed that tildacerfont reduced key hormone biomarkers towards normal levels in the baseline poor disease control group, including normalization of ACTH and A4 in 60% and 40% of patients, respectively. In patients with baseline good disease control, these hormones were maintained near or below normal levels. Tildacerfont was generally safe and well-tolerated. The findings from these studies support the ongoing global late-stage studies of tildacerfont in adults with classic CAH CAHmelia-203 (assessing the ability of tildacerfont to reduce excessive adrenal androgens in patients with poor disease control) and CAHmelia-204 (assessing the ability of tildacerfont to reduce glucocorticoid usage in patients with good disease control while maintaining control of androgens).

Classic CAH is a serious and potentially life-threatening condition that has not benefited from new effective treatment options in approximately 50 years, said Rosh Dias, M.D., MRCP, Chief Medical Officer of Spruce Biosciences. People living with classic CAH must grapple with the difficult balance of managing their adrenal androgen excess and supraphysiologic dosing of glucocorticoids, the existing standard of care therapy. In our Phase 2a studies, I was pleased to see that tildacerfont was able to produce meaningful reductions in highly elevated hormones in classic CAH patients, including in some cases normalization of these hormones, over a 12-week period without increases to daily steroid doses. This has not been reported to date with any other investigational product candidate.

About Tildacerfont

Tildacerfont is a potent and highly selective, non-steroidal, oral antagonist of the CRF1 receptor, which is the receptor for corticotropin-releasing factor (CRF), a hormone that is secreted by the hypothalamus. The CRF1 receptor is abundantly expressed in the pituitary gland where it is the primary regulator of the HPA axis. By blocking the CRF1 receptor, tildacerfont has the potential to address the uncontrolled cortisol feedback regulatory pathway in CAH, and in turn reduce the production of ACTH in the pituitary, limiting the amount of androgen produced downstream from the adrenal gland. Tildacerfont has been evaluated in 171 patients across seven clinical trials in which it has been generally well tolerated. No drug-related serious adverse events have been reported related to tildacerfont treatment.

About Spruce Biosciences

Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet need. Spruce is initially developing its wholly owned product candidate, tildacerfont, as the potential first non-steroidal therapy for patients suffering from classic congenital adrenal hyperplasia (CAH). Classic CAH is a serious and life-threatening disease with no known novel therapies approved in approximately 50 years. Spruce is also developing tildacerfont for women suffering from a rare form of polycystic ovary syndrome (PCOS) with primary adrenal androgen excess. To learn more, visit http://www.sprucebiosciences.com and follow us on Twitter @Spruce_Bio, LinkedIn, Facebook and YouTube.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the results, conduct, progress and timing of Spruces clinical trials. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as potential, demonstrates, may and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Spruces current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Spruces business in general, the impact of the COVID-19 pandemic, and the other risks described in Spruces filings with the U.S. Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on managements assumptions and estimates as of such date. Spruce undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Spruce Biosciences Announces Publication of Phase 2 Results for Tildacerfont in Journal of Clinical Endocrinology and Metabolism - Business Wire

GSK and Vir Biotechnology Announce Continuing Progress of the COMET Clinical Development Program for Sotrovimab – BioSpace

Posted: at 1:52 am


LONDON and SAN FRANCISCO, June 21, 2021 (GLOBE NEWSWIRE) -- GlaxoSmithKline plc (LSE/NYSE: GSK) and Vir Biotechnology, Inc. (Nasdaq: VIR) today announced final, confirmatory results from the Phase 3 COMET-ICE (COVID-19 Monoclonal antibody Efficacy Trial Intent to Care Early) trial demonstrating that sotrovimab, an investigational SARS-CoV-2 monoclonal antibody, significantly reduced the risk of hospitalization or death among high-risk adult outpatients with mild-to-moderate COVID-19. Additionally, the U.S. National Institutes of Health (NIH) updated its COVID-19 treatment guidelines to recommend sotrovimab for non-hospitalized patients with mild-to-moderate COVID-19 who are at high risk of clinical progression and noted that sotrovimab appears to retain activity against current variants of concern and interest.

The primary efficacy analysis of all 1,057 patients in the COMET-ICE trial demonstrated a 79% reduction (adjusted relative risk reduction) (p<0.001) in hospitalization for more than 24 hours or death due to any cause, by Day 29 compared to placebo, meeting the primary endpoint of the trial.

The number of patients in the trial who were hospitalized for >24 hours for acute management of any illness or death from any cause at Day 29 was six patients in the sotrovimab arm (1%), versus 30 patients in the placebo arm (6%). In the sotrovimab arm, it is possible that half of those patients who were hospitalized were for reasons other than progression of COVID-19 (e.g., small bowel obstruction, lung cancer and diabetic foot ulcer); this was not the case for patients in the placebo arm. In the safety analysis, 1,037 participants were followed through at least 29 days. The most common adverse events observed in the sotrovimab treatment group in COMET-ICE were rash (1%) and diarrhea (2%), all of which were Grade 1 (mild) or Grade 2 (moderate). No other treatment-emergent adverse events were reported at a higher rate with sotrovimab compared to placebo. The companies plan to submit the full COMET-ICE data set to a peer-reviewed journal for publication.

Christopher Corsico, senior vice president of development, GSK, said: Effective medicines to treat those who become infected with SARS-CoV-2 remain a critical part of the solution to this pandemic. We are working diligently to increase access to sotrovimab in the U.S. and across the globe, including evaluating the potential to simplify administration with an intramuscular formulation.

George Scangos, Ph.D., chief executive officer of Vir, said: We are pleased that the profound interim efficacy from the COMET-ICE trial has now been validated by the full study population. These results, combined with the growing number of pending global authorizations, as well as the recent recommendation by the NIH COVID-19 Treatment Guidelines Panel, support our confidence in the potential role of sotrovimab in the fight against this pandemic.

Updated NIH Guidelines Recommend SotrovimabThe NIH recently updated its guidelines regarding the emergency use authorizations of anti-SARS-CoV-2 monoclonal antibodies for the treatment of COVID-19 in the U.S. to recommend the use of sotrovimab for non-hospitalized patients with mild-to-moderate COVID-19 who are at high risk of clinical progression. The guidelines note that the target binding site of sotrovimab is in a region of the virus that does not overlap with the binding site location of key mutations in current variants of concern and interest. These guidelines were based upon an interim analysis of 583 patients in the COMET-ICE trial, which was stopped early in March 2020 by an independent data monitoring committee because interim results demonstrated evidence of sotrovimabs clinical efficacy. The interim study results demonstrated an 85% (p=0.002) reduction in hospitalization for more than 24 hours or death in those receiving sotrovimab compared to placebo, the primary endpoint of the trial.

These data have informed global regulatory reviews to date, including the positive scientific opinion issued by the European Medicines Agencys (EMA) Committee for Human Medicinal Products (CHMP) under Article 5(3) of Regulation 726/2004, as well as the Emergency Use Authorization (EUA) granted by the U.S. Food and Drug Administration (FDA).

The companies are actively working with government agencies around the world to make sotrovimab available to patients in need of treatment.

Continued Progress with the COMET Clinical Development ProgramThe companies are also pleased to announce continued progress with the robust COMET clinical development program, which aims to provide clinical evidence from several studies over the course of the next year.

GSK and Vir are committed to ongoing evaluation of sotrovimab as the COVID-19 landscape continues to evolve at different rates across the globe and new variants of concern and interest emerge. Data from in vitro studies, published in bioRxiv, have demonstrated that sotrovimab maintains activity against circulating variants of concern and interest, including the Gamma (P.1), Epsilon (B.1.427/B.1.429), Delta (B.1.617.1), Iota (B.1.526), Beta (B.1.351) and Alpha (B.1.1.7) variants. GSK and Vir are continuing to evaluate the ability of sotrovimab to maintain activity against new and emerging variants through in vitro studies. The clinical impact of this in vitro variants data is not yet known.

About Sotrovimab (previously VIR-7831)Sotrovimab is an investigational SARS-CoV-2 monoclonal antibody. Preclinical data suggest it has the potential to both block viral entry into healthy cells and clear infected cells. The antibody binds to an epitope on SARS-CoV-2 that is shared with SARS-CoV-1 (the virus that causes SARS), indicating that the epitope is highly conserved, which may make it more difficult for resistance to develop. Sotrovimab, which incorporates Xencors Xtend technology, also has been designed to achieve high concentration in the lungs to ensure optimal penetration into airway tissues affected by SARS-CoV-2 and to have an extended half-life.

The following is a summary of information for sotrovimab. Healthcare providers in the U.S. should review the Fact Sheets for information on the authorized use of sotrovimab and mandatory requirements of the EUA. Please see the FDA Letter of Authorization, Fact Sheet for Healthcare Providers, and Fact Sheet for Patients, Parents, and Caregivers. For more information on the EMA positive scientific opinion, please review the EU Conditions of Use.

Important Information about SotrovimabSotrovimab has been authorized by the FDA for the emergency use described below. Sotrovimab is not FDA-approved for this use.

Sotrovimab is authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use of sotrovimab under section 564(b)(1) of the Act, 21 U.S.C. 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner.

Authorized Use The U.S. Food and Drug Administration (FDA) has issued an Emergency Use Authorization (EUA) to permit the emergency use of the unapproved product sotrovimab for the treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death.

Limitations of Authorized Use Sotrovimab is not authorized for use in patients:

Benefit of treatment with sotrovimab has not been observed in patients hospitalized due to COVID-19. SARS-CoV-2 monoclonal antibodies may be associated with worse clinical outcomes when administered to hospitalized patients with COVID-19 requiring high flow oxygen or mechanical ventilation.

Important Safety Information for Sotrovimab

Warnings

There are limited clinical data available for sotrovimab. Serious and unexpected adverse events may occur that have not been previously reported with sotrovimab use.

Hypersensitivity Including Anaphylaxis and Infusion-Related Reactions

Serious hypersensitivity reactions, including anaphylaxis have been observed with administration of sotrovimab. If signs and symptoms of a clinically significant hypersensitivity reaction or anaphylaxis occur, immediately discontinue administration and initiate appropriate medications and/or supportive care.

Infusion-related reactions, occurring during the infusion and up to 24 hours after the infusion, have been observed with administration of sotrovimab. These reactions may be severe or life threatening.

Signs and symptoms of infusion-related reactions may include: fever, difficulty breathing, reduced oxygen saturation, chills, fatigue, arrhythmia (eg, atrial fibrillation, sinus tachycardia, bradycardia), chest pain or discomfort, weakness, altered mental status, nausea, headache, bronchospasm, hypotension, hypertension, angioedema, throat irritation, rash including urticaria, pruritus, myalgia, vaso-vagal reactions (eg, pre-syncope, syncope), dizziness and diaphoresis.

Consider slowing or stopping the infusion and administer appropriate medications and/or supportive care if an infusion-related reaction occurs.

Hypersensitivity reactions occurring more than 24 hours after the infusion have also been reported with the use of SARS-CoV-2 monoclonal antibodies under Emergency Use Authorization.

Clinical Worsening After SARS-CoV-2 Monoclonal Antibody Administration

Clinical worsening of COVID-19 after administration of SARS-CoV-2 monoclonal antibody treatment has been reported and may include signs or symptoms of fever, hypoxia or increased respiratory difficulty, arrhythmia (eg, atrial fibrillation, tachycardia, bradycardia), fatigue, and altered mental status. Some of these events required hospitalization. It is not known if these events were related to SARS-CoV-2 monoclonal antibody use or were due to progression of COVID-19.

Limitations of Benefit and Potential for Risk in Patients with Severe COVID-19

Benefit of treatment with sotrovimab has not been observed in patients hospitalized due to COVID-19. SARS-CoV-2 monoclonal antibodies may be associated with worse clinical outcomes when administered to hospitalized patients with COVID-19 requiring high flow oxygen or mechanical ventilation. Therefore, sotrovimab is not authorized for use in patients: who are hospitalized due to COVID-19, OR who require oxygen therapy due to COVID-19, OR who require an increase in baseline oxygen flow rate due to COVID-19 in those on chronic oxygen therapy due to underlying non-COVID-19 related comorbidity.

ADVERSE EVENTS

The most common treatment-emergent adverse events observed in the sotrovimab treatment group in COMET-ICE were rash (2%) and diarrhea (1%), all of which were Grade 1 (mild) or Grade 2 (moderate). No other treatment-emergent adverse events were reported at a higher rate with sotrovimab compared to placebo.

USE IN SPECIFIC POPULATIONS

Pregnancy

There are insufficient data to evaluate a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcome. Sotrovimab should be used during pregnancy only if the potential benefit justifies the potential risk for the mother and the fetus.

Lactation

There are no available data on the presence of sotrovimab in human milk, the effects on the breastfed infant, or the effects on milk production. Individuals with COVID-19 who are breastfeeding should follow practices according to clinical guidelines to avoid exposing the infant to COVID-19.

About the Vir and GSK CollaborationIn April 2020, Vir and GSK entered into a collaboration to research and develop solutions for coronaviruses, including SARS-CoV-2, the virus that causes COVID-19. The collaboration uses Virs proprietary monoclonal antibody platform technology to accelerate existing and identify new anti-viral antibodies that could be used as therapeutic or preventive options to help address the current COVID-19 pandemic and future outbreaks. The companies will leverage GSKs expertise in functional genomics and combine their capabilities in CRISPR screening and artificial intelligence to identify anti-coronavirus compounds that target cellular host genes. They will also apply their combined expertise to research SARS-CoV-2 and other coronavirus vaccines.

GSK Commitment to Tackling COVID-19GSKs response to COVID-19 has been one of the broadest in the industry, with three potential treatments in addition to our vaccine candidates in development with partner organizations.

GSK is collaborating with several organizations on COVID-19 vaccines by providing access to our adjuvant technology. In addition to our work with Medicago, we recently announced positive Phase 2 data from our collaboration with Sanofi to develop an adjuvanted, protein-based vaccine candidate and expect to begin a Phase 3 trial in Q2. An earlier stage collaboration with SK Bioscience is also ongoing. SK Bioscience receives funding from CEPI and the Bill and Melinda Gates Foundation to develop differentiated, affordable COVID-19 vaccines for supply globally through the COVAX facility. The use of an adjuvant can be of particular importance in a pandemic since it may reduce the amount of vaccine protein required per dose, allowing more vaccine doses to be produced and contributing to protecting more people.

GSK is also working with mRNA specialist, CureVac, to jointly develop next generation, multi-valent mRNA vaccines for COVID-19 with the potential to address multiple emerging variants in one vaccine. GSK will also support manufacturing of up to 100m doses of CureVacs first generation COVID-19 vaccine. GSK is also providing manufacturing support for up to 60m doses of Novavax COVID-19 vaccine in the UK.

GSK is also exploring potential therapeutic or treatment options for COVID-19 patients. We are collaborating with Vir Biotechnology to develop existing and identify new anti-viral antibodies that could be used as therapeutic or preventive options for COVID-19. We reported that an Independent Data Monitoring Committee recommended that the Phase 3 COMET-ICE trial evaluating sotrovimab as monotherapy for the early treatment of COVID-19 in adults at high risk of hospitalization be stopped for enrollment due to evidence of profound efficacy, based on an interim analysis of data from the trial. An analysis of data through Day 29 of the COMET-ICE trial was consistent with interim results. We have received Emergency Use Authorization in the U.S. and are seeking authorizations in other countries. We are also assessing whether an investigational monoclonal antibody, otilimab, can help severely ill COVID-19 patients aged over 70 who experience an overreaction of their immune system.

Virs Commitment to COVID-19Vir was founded with the mission of addressing the worlds most serious infectious diseases. In 2020, Vir responded rapidly to the COVID-19 pandemic by leveraging our unique scientific insights and industry-leading antibody platform to explore multiple monoclonal antibodies as potential therapeutic or preventive options for COVID-19. Sotrovimab is the first SARS-CoV-2-targeting antibody Vir advanced into the clinic. It was carefully selected for its demonstrated promise in preclinical research, including an anticipated high barrier to resistance and potential ability to both block the virus from entering healthy cells and clear infected cells. Vir is continuing to pursue novel therapeutic and prophylactic solutions to combat SARS-CoV-2 and future coronavirus pandemics, both independently and in collaboration with its partners.

About GSK GSK is a science-led global healthcare company with a special purpose: to help people do more, feel better, live longer. For further information please visit http://www.gsk.com/about-us.

About Vir Biotechnology

Vir Biotechnology is a clinical-stage immunology company focused on combining immunologic insights with cutting-edge technologies to treat and prevent serious infectious diseases. Vir has assembled four technology platforms that are designed to stimulate and enhance the immune system by exploiting critical observations of natural immune processes. Its current development pipeline consists of product candidates targeting COVID-19, hepatitis B virus, influenza A and human immunodeficiency virus. For more information, please visit http://www.vir.bio.

GSK Cautionary Statement Regarding Forward-Looking StatementsGSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the Company's Annual Report on Form 20-F for 2020 and any impacts of the COVID-19 pandemic.

Vir Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as may, will, plan, potential, aim, promising and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Virs expectations and assumptions as of the date of this press release. Forward-looking statements contained in this press release include, but are not limited to, statements regarding the final data from the COMET-ICE trial, NIH guidelines recommending the use of sotrovimab in the treatment of COVID-19, the initiation of Virs COMET-TAIL clinical trial, the clinical development program for sotrovimab, Virs capacity to manufacture and supply sotrovimab, the ability of sotrovimab to treat and/or prevent COVID-19, the ability of sotrovimab to maintain activity against circulating variants of concern, and statements related to regulatory authorizations and approvals, including plans and discussions with the FDA, EMA and other global regulators. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical or clinical studies, challenges in the treatment of hospitalized patients, difficulties in collaborating with other companies or government agencies, challenges in accessing manufacturing capacity, successful development and/or commercialization of alternative product candidates by Virs competitors, changes in expected or existing competition, delays in or disruptions to Virs business or clinical trials due to the COVID-19 pandemic, geopolitical changes or other external factors, and unexpected litigation or other disputes. Other factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Virs filings with the U.S. Securities and Exchange Commission, including the section titled Risk Factors contained therein. Except as required by law, Vir assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

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GSK and Vir Biotechnology Announce Continuing Progress of the COMET Clinical Development Program for Sotrovimab - BioSpace

CytomX Therapeutics Announces Publication of First-in-Human Data for CX-2029 in Clinical Cancer Research – GlobeNewswire

Posted: at 1:52 am


SOUTH SAN FRANCISCO, Calif., June 21, 2021 (GLOBE NEWSWIRE) -- CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational conditionally activated therapeutics based on its Probody technology platform, today announced that results from its Phase 1 first-in-human study of CX-2029 in patients with advanced solid tumors were published online in the peer-reviewed journal Clinical Cancer Research. This study showed that CX-2029, currently being co-developed by CytomX and AbbVie, was generally well-tolerated and can elicit anti-tumor responses in certain patients.

These results highlight that our industry-leading Probody platform can be successfully leveraged to create conditionally activated ADCs against previously undruggable targets. For the first time, CD71 has been shown to be a viable therapeutic cancer target, said Alison L. Hannah, M.D., senior vice president and chief medical officer of CytomX Therapeutics.

CD71 is a cell surface protein essential for iron uptake in dividing cells and is highly expressed in a number of solid and hematologic cancers. However, given its central role in iron metabolism, CD71 is present on most healthy cells and has been, until now, undruggable with conventional ADCs. CX-2029 is designed to be activated in the tumor microenvironment by tumor-associated proteases, thereby limiting off-tumor toxicity and creating a therapeutic window for CD71.

The goal of this Phase 1 dose-escalation, multicenter study was to evaluate the safety, pharmacokinetics, pharmacodynamics, and antitumor activity of CX-2029. A total of 45 patients were enrolled to receive CX-2029 intravenously every three weeks at dose levels ranging from 0.1 mg/kg to 5 mg/kg.

Encouraging preliminary clinical activity was observed at doses of 2 mg/kg and higher. Notably, three of four patients with squamous non-small cell lung carcinoma (NSCLC) had stable disease (SD) or better, including two confirmed partial responses (PRs) (at doses of 3 and 5 mg/kg); and seven of eight patients with head and neck squamous cell carcinoma (HNSCC) had SD or better, including one confirmed PR at 3 mg/kg and one prolonged SD ongoing at approximately 25 weeks, as of the reported August 2020 data cutoff.

Despite having received a median of three prior lines of cancer regimens (range, 116), these heavily-pretreated patients generally tolerated CX-2029 well. The most common dose-dependent adverse events were anemia and neutropenia, toxicities commonly associated with the payload of this ADC (monomethyl auristatin E). Based on several safety parameters, including no cycle 1 DLTs, no discontinuations due to toxicity, and a long-term tolerability that appeared to be acceptable for chronic administration with supportive care for anemia, 3 mg/kg every 3 weeks was declared the recommended Phase 2 dose.

The ongoing Phase 2 expansion study is evaluating CX-2029 as monotherapy in four cohorts: squamous NSCLC, HNSCC, esophageal and gastroesophageal junction cancers (both adenocarcinoma and squamous histologies), and diffuse large B-cell lymphoma. Initial results are expected in the fourth quarter of 2021.

AboutCytomX TherapeuticsCytomX is a clinical-stage, oncology-focused biopharmaceutical company with a vision of transforming lives with safer, more effective therapies. We are developing a novel class of investigational conditionally activated therapeutics, based on our Probody technology platform, for the treatment of cancer. CytomX has strategic drug discovery and development collaborations with AbbVie, Amgen, Astellas, and Bristol Myers Squibb.

Probody therapeutics are conditionally activated biologics designed to remain inactive until they are activated by proteases in the tumor microenvironment. As a result, Probody therapeutics are intended to bind selectively to tumors and decrease binding to healthy tissue, to minimize toxicity and potentially create safer, more effective therapies. As leaders in the field, our innovative technology is designed to turn previously undruggable targets into druggable targets and to enable more effective combination therapies. CytomX and its partners, comprised of leading biotechnology and pharmaceutical companies, have developed a robust pipeline of potential first-in-class therapeutic candidates against novel, difficult to drug targets and potential best-in-class immunotherapeutic candidates against clinically validated targets. The CytomX clinical-stage pipeline comprises five assets, four of which are in Phase 2 clinical studies. First-in-class product candidates against previously undruggable targets include a CD166-targeting conditionally activated antibody-drug conjugate wholly owned by CytomX (praluzatamab ravtansine, CX-2009) and a CD71-targeting conditionally activated antibody-drug conjugate partnered with AbbVie (CX-2029). CD166 and CD71 are among cancer targets that are considered to be inaccessible to conventional antibody-drug conjugates due to their presence on many healthy tissues. The CytomX clinical-stage pipeline also includes cancer immunotherapeutic candidates against validated targets such as the CTLA-4-targeting Probodies, BMS-986249 and BMS-986288, partnered with Bristol Myers Squibb, and our wholly-owned conditionally activated anti-PD-L1 antibody, pacmilimab (CX-072). For additional information about CytomX Therapeutics, visit http://www.cytomx.com and follow us on LinkedIn and Twitter.

CytomX Therapeutics Forward-Looking StatementsThis press release includes forward-looking statements. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that are difficult to predict, may be beyond our control, and may cause the actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied in such statements. Accordingly, you should not rely on any of these forward-looking statements, including those relating to the potential benefits, safety and efficacy or progress of CytomXs or any of its collaborative partners product candidates, including praluzatamab ravtansine (CX-2009), CX-2029, BMS-986249, BMS-986288, and pacmilimab (CX-072), the potential benefits or applications of CytomXs Probody platform technology, CytomXs ability to develop and advance product candidates into and successfully complete clinical trials, including the ongoing and planned clinical trials of praluzatamab ravtansine, CX-2029, BMS-986249, BMS-986288, and pacmilimab (CX-072), and the timing of the commencement of clinical trials and other development milestones. Risks and uncertainties that contribute to the uncertain nature of the forward-looking statements include: the unproven nature of CytomXs novel Probody platform technology; CytomXs clinical trial product candidates are in the initial stages of clinical development and its other product candidates are currently in preclinical development, and the process by which preclinical and clinical development could potentially lead to an approved product is long and subject to significant risks and uncertainties, including the risk that the COVID-19 worldwide pandemic may continue to negatively impact the business, research and clinical operations of CytomX or its partners, including the development of preclinical drug candidates due to delays in and disruption of research activities and the development of clinical drug candidates due to delays in or disruption of clinical trials, including impacts on the enrollment of patients in clinical trials or other clinical trial disruptions; the possibility that the results of early clinical trials may not be predictive of future results, including clinical trials for CX-2029; the possibility that CytomXs clinical trials will not be successful; the possibility that current preclinical research may not result in additional product candidates; CytomXs dependence on the success of praluzatamab ravtansine, CX-2029, BMS-986249, BMS-986288, and pacmilimab (CX-072); CytomXs reliance on third parties for the manufacture of the companys product candidates; and possible regulatory developments inthe United Statesand foreign countries. Additional applicable risks and uncertainties include those relating to our preclinical research and development, clinical development, and other risks identified under the heading "Risk Factors" included in CytomXs Quarterly Report on Form 10-Q filed with theSEConMay 6, 2021. The forward-looking statements contained in this press release are based on information currently available to CytomX and speak only as of the date on which they are made. CytomX does not undertake and specifically disclaims any obligation to update any forward-looking statements, whether as a result of any new information, future events, changed circumstances or otherwise.

Probody is a U.S. registered trademark of CytomX Therapeutics, Inc.

CytomX Contact:Chau Cheng, PhD MBAVP, Investor Relations & Corp. Communicationsccheng@cytomx.comDirect: (650) 273-4999

Investor and Media Contact:Stern Investor RelationsStephanie Ascherstephanie.ascher@sternir.com212-362-1200

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CytomX Therapeutics Announces Publication of First-in-Human Data for CX-2029 in Clinical Cancer Research - GlobeNewswire

COVID-19: India Has a Habit of Bad Clinical Trials – The Wire Science

Posted: at 1:52 am


Representative: A health official draws a dose of Covishield in Colombo, Sri Lanka, January 29, 2021. Photo: Reuters/Dinuka Liyanawatte/File Photo

Mumbai: On June 15, All India Institute of Medical Sciences (AIIMS), Delhi, started inoculating children with Covaxin as part of Bharat Biotechs paediatric clinical trial to evaluate the safety, reactogenicity and immunogenicity of Covaxin in 2- to 18-year-old children.

The trial plans to enrol around 525 children across hospitals. This is a very small cohort. For example, Modernas paediatric trial aims to recruit 7,050 children aged 6 months to 12 years. It had conducted another trial with 3,732 teenagers aged 12-18 years. Similarly, Pfizer is conducting a paediatric trial with 4,644 children aged 6 months to 12 years; and its trial with adolescents enrolled 2,260 participants aged 12-15 years.

Experts have raised concerns about Bharat Biotechs trial over its small size, and because it doesnt have a placebo arm.

And while the criticism is significant, it may not be limited to Bharat Biotech.

The Hyderabad-based pharmaceutical companys small paediatric trial is one of many clinical studies conducted in India that have, or intend to have, a small number of participants.

Such studies are said to be underpowered. According to one 2007 paper, a studys power is the probability of saying there is a difference when a difference actually exists. An underpowered study does not have a sufficiently large sample size to answer the research question of interest.

It is possible to calculate the ideal sample size before the trial begins. This means while trial investigators can write off an inconclusive result as the product of the small sample size, theyre expected to have good reasons for why they didnt include more participants.

However, Indias drug regulator, the Drug Controller General, has complicated this picture, especially in the last couple years. It has passively condoned underpowering by awarding the fruits of such trials with emergency use approvals. Itolizumab was okayed after a trial with 30 participants, favipiravir with 150 participants, Virafin with 290 participants (40 and 250 in phase 2 and 3 trials) and 2-deoxy-d-glucose with 330 participants (110 and 220 in phase 2 and 3 trials).

Also read: Is Biocons Itolizumab Good News? Hard to Tell, Thanks to the Bad Science.

* * *

Randomised control trials are difficult to conduct more so with a crumbling health infrastructure in the middle of an epidemic but this isnt an acceptable excuse to not have one.

Conducting multiple small trials with a few hundred participants is not the same as conducting one large trial with thousands. When the former are summed up, their underpoweredness is magnified, rendering the total findings less than useful.

In India, researchers who plan to conduct clinical studies involving human participants need to register each study on the Clinical Trial Registry of India (CTRI) before it begins. Since the pandemic began, more than 1,300 such trials have been registered on CTRI.

Not all trials are completed; many never kick off and many others stop midway, for multiple reasons. This said, Indias COVID-19 research output has still been piddling especially in quality, because most studies are underpowered and often methodologically flawed as well.

For example, there are at least 15 trials registered on CTRI involving hydroxychloroquine, the drug that India pushed in a big way in 2020. These trials together include thousands of patients but they produced little usable evidence last year.

Instead, all the evidence we have on the efficacy of different drugs has come from large adaptive trials, especially the WHOs SOLIDARITY, the UKs RECOVERY and the USs ACTT trials.

(Researchers sometimes unearth important patterns by studying multiple small trials together. However, this isnt possible when the trials measure different parameters, thus arriving at incomparable outcomes.

For example, trials for hydroxychloroquine at AIIMS Delhi, with 116 participants, and at AIIMS Raipur, with 50 measured progression to severe disease and virological clearance at day six, with no overlap.)

No country for negative trials

Another problem is publication bias when researchers dont publish their results because the latter are deemed to be insignificant.

For example, Mumbai-based Wockhardt conducted a trial for convalescent plasma in May 2020. In a meeting with experts at the Central Drug Standards Control Organisation on February 11, 2021, Wockhardt submitted that its trial failed to show any benefit with plasma but it hasnt published a paper detailing the trials results. The PLATINA trial in Maharashtra met a similar fate.

Emails to researchers involved in both trials hadnt elicited a response at the time of publishing this article.

Not publishing the results of a trial is a breach of the ethical obligations that researchers have towards study participants, and is a grave injustice to our people, Dr Aju Mathew, an epidemiologist and a cancer specialist in Kochi, told The Wire Science.

In a study published in December 2020, Dr Mathew and his peers found that only 55% of cancer trials registered in India were eventually published. They also reported that trials conducted by international pharmaceutical companies were more likely to be published than Indian ones.

Publication bias can have a serious impact on patient care. COVID-19 is a new disease: before drug trials got underway, healthcare workers drew on their experience and education to make informed guesses about treatments to administer. Negative results in this regard can help workers improve their protocols by subtracting bad options.

Indeed, institutions around the world dropped drugs like hydroxychloroquine, lopinavir and ritonavir from their protocols thanks to negative results from the RECOVERY and SOLIDARITY trials.

Also read: A Good Registry Means Accountable Clinical Trials. But Does India Have One?

Poor output

Smaller trials in India have also been less accountable and have operated with little oversight.

The UKs RECOVERY trial recruited around 40,000 participants at 180 sites. The University of Oxford, which is in charge, centrally determined the protocol to follow and the outcomes to watch out for, and used new results from their own study to update their trial1. Both positive and negative results were published.

No such effort has emerged out of India for reasons including bureaucratic lethargy, little collaboration between institutions, lack of autonomy and of incentives, Dr Mathew said. Other experts also mentioned deficient institutional funding and subpar expertise.

We are a nation of band-aid action. We dont plan ahead of time.

We cant wake up in the middle of the pandemic and expect world-class research, said Dr Soumyadeep Bhaumik, co-head of the meta-research and evidence synthesis unit at the George Institute, New Delhi. A research ecosystem develops after years of systematic investment in institutions, expertise and a regulatory framework.

It was only on June 10, 2021, that the Indian Council for Medical Research issued a tender to develop a large, multicentre, adaptive trial platform like RECOVERY.

According to a researcher who worked on a prominent Centre-funded COVID-19 trial in 2020, a serious lack of expertise among participating clinicians also compounded the chronic under-investment. It took a lot of hand-holding and training to get them to appropriately complete the trial, the researcher said.

It can feel both good and bad to know that India is in fact capable of conducting well-designed trials. For example, the PLACID trial, an ICMR-funded enterprise, enrolled 464 patients (calculated to be sufficient power) from April 22 to July 14, 2020, to study the efficacy of convalescent plasma. The results were published as a preprint paper on September 10 and in BMJ a month later.

In another example, as of October 15, 2020, Indian doctors had enrolled 937 Indian participants at several hospitals in the country for the SOLIDARITY trial.

Even when large, centralised trials arent feasible, Dr Bhaumik said a set of core outcome measures could ensure small trials measuring the same things also watch out for the same results.

Here, experts deliberate on and recommend some common patient-centric outcomes for clinicians to use when designing their trials. Adopting these outcomes wouldnt render all trials the same.

They could measure other outcomes as well but using a core outcome set means the results of these small trials can be pooled to inform policy and practice, Dr Bhaumik said. We need to start developing core outcome sets for diseases of significance to our country.

In the time of COVID-19, this duty fell on the ICMRs shoulders, but which is yet to issue any such directions to the many COVID-19 trials happening in the country.

In fact, in the loudest directive the organisation sent forth (and quickly withdrew), its chief Dr Balram Bhargava demanded in July 2020 that hospitals involved in Covaxins clinical trial complete it in just two months, or face the governments music. The results of this trial are yet to be published.

Ronak Borana is a science communicator based in Mumbai. He tweets at @ronaklmno.

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COVID-19: India Has a Habit of Bad Clinical Trials - The Wire Science

Jasper Therapeutics and Aruvant Announce Research Collaboration to Study JSP191, an Antibody-Based Conditioning Agent, with ARU-1801, a Novel Gene…

Posted: at 1:52 am


REDWOOD CITY, Calif. and NEW YORK and BASEL, Switzerland, June 21, 2021 /PRNewswire/ --Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, andAruvant Sciences, a private company focused on developing gene therapies for rare diseases, today announced that they have entered a non-exclusive research collaboration to evaluate the use of JSP191, Jasper's anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning agent with ARU-1801, Aruvant's investigational lentiviral gene therapy for sickle cell disease (SCD). The objective of the collaboration is to evaluate the use of JSP191 as an effective and more tolerable conditioning agent that can expand the number of patients who can receive ARU-1801, a potentially curative treatment for SCD.

"This research collaboration with Aruvant is the first to use a clinical-stage antibody-based conditioning agent and a novel clinical-stage gene therapy, giving this combination a clear advantage by moving beyond the harsh conditioning agents currently used for gene therapy and establishing this next-generation potentially curative treatment as a leader in sickle cell disease," said Kevin N. Heller, M.D., executive vice president, research and development of Jasper. "Our goal is to establish JSP191 as a potential new standard of care conditioning agent, broadly in autologous gene therapy and allogeneic hematopoietic stem cell transplantation."

Gene therapies and gene editing technologies generally require that a patient's own hematopoietic stem cells first be depleted from the bone marrow to facilitate the engraftment of the new, gene-modified stem cells through a process called conditioning. Other investigational gene therapies and gene editing approaches in SCD use a high-dose chemotherapy such as busulfan for the conditioning regimen, which can place patients at prolonged risk for infection and bleeding, secondary malignancy and infertility. ARU-1801 is currently the only gene therapy that has demonstrated durable efficacy using both a lower dose of chemotherapy and a different agent than busulfan with a more limited side effect profile. The Aruvant-Jasper partnership is focused on evaluating the potential of using JSP191, a highly targeted anti-CD117 (stem cell factor receptor) monoclonal antibody agent, as the foundationof a novel conditioning regimen for use in combination with ARU-1801 to further reduce the negative side effects while maintaining efficacy.

"The unique attributes of ARU-1801 enable us to bring a potentially curative one-time therapy to individuals with sickle cell disease that can be delivered in the safest way possible," said Will Chou, M.D., Aruvant chief executive officer. "By partnering with Jasper to evaluate the use of JSP191 with ARU-1801, we are one step closer to developing a next-generation definitive therapy with an even more patient-friendly conditioning regimen. We believe that this combination may be able to further expand the number of patients who can benefit from ARU-1801 in the future, including potentially those with more moderate disease."

About JSP191JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. While hematopoietic cell transplantation can be curative for patients, its use is limited because standard high dose myeloablative conditioning is associated with severe toxicities and standard low dose conditioning has limited efficacy. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients. It is currently enrolling in two clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML) and severe combined immunodeficiency (SCID) and expects to begin enrollment in four additional studies in 2021 for severe autoimmune disease, sickle cell disease, chronic granulomatous disease and Fanconi anemia patients undergoing hematopoietic cell transplantation.

About ARU-1801ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of graft-versus-host disease (GvHD) seen with allogeneic stem cell transplants. Unlike investigational gene therapies and gene editing approaches which require fully myeloablative conditioning, the unique characteristics of ARU-1801 allow it to be given with reduced intensity conditioning ("RIC"). Compared to myeloablative approaches, the lower dose chemotherapy regimen underlying RIC has the potential to reduce not only hospital length of stay, but also the risk of short- and long-term adverse events such as infection and infertility. Preliminary clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM StudyAruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD. This Phase 1/2 study is currently enrolling participants, and information may be found at momentumtrials.comwhich includes a patient brochure, an eligibility questionnaireand information for healthcare providers.

About Jasper TherapeuticsJasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, a first-in-class anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic and autologous hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in development for individuals suffering from sickle cell disease (SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in a Phase 1/2 clinical trial, the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. The company's second product candidate, ARU-2801, is in development to cure hypophosphatasia, a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high mortality when not treated. Data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival. For more information on the ongoing ARU-1801 clinical study, please visit http://www.momentumtrials.comand for more on the company, please visit http://www.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

About RoivantRoivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants nimble and focused biopharmaceutical and health technology companies. For more information, please visit http://www.roivant.com.

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Antengene Announces Fifteen Clinical Studies and Results of Selinexor to be Presented at ASCO 2021 – PRNewswire

Posted: June 6, 2021 at 1:51 am


SHANGHAI and HONG KONG, June 1, 2021 /PRNewswire/ -- Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, announced that fifteen studies and results of selinexor, the world's first approved oral selective inhibitor of nuclear export (SINE), will be presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place in a virtual format on June 4 to 8.

Selected Abstracts:

Effects of weekly selinexor, bortezomib, dexamethasone (XVd) versus standard twice weekly bortezomib and dexamethasone (Vd) on RAS-mutated previously treated multiple myeloma (MM).

Abstract #: 8027

Effects of refractory status to lenalidomide on safety and efficacy of selinexor, bortezomib, and dexamethasone (XVd) versus bortezomib and dexamethasone (Vd) in patients with previously treated multiple myeloma.

Abstract #: 8024

Survival among older patients with previously treated multiple myeloma treated with selinexor, bortezomib, and dexamethasone (XVd) in the BOSTON study.

Abstract #: 8019

Updated overall survival of eltanexor for the treatment of patients with hypomethylating agent refractory myelodysplastic syndrome.

Abstract #: e19037

Results of the phase 2 MARCH Study: Oral ATG-010 (Selinexor) plus low dosedexamethasone in Chinese patients with relapsed/refractory multiple myeloma (RRMM) previously treated with an immunomodulatory agent (IMiD) and a proteasome inhibitor (PI).

Abstract #: e20002

Oral selinexor, pomalidomide, and dexamethasone (XPd) at recommended phase 2 dose in relapsed refractory multiple myeloma (MM).

Abstract #: 8018

SIENDO/ENGOT-EN5/GOG-3055: A randomized phase 3 trial of maintenance selinexor versus placebo after combination platinum-based chemotherapy in advanced or recurrent endometrial cancer.

Abstract #: TPS5610

Open-label phase 1 study evaluating the tolerability and anti-tumor activity of selinexor and pembrolizumab in colorectal cancer.

Abstract #: e15579

A phase 1/2 study of selinexor in combination with standard of care therapy for newly diagnosed or recurrent glioblastoma.

Abstract #: TPS2071

A phase Ib/II study of selinexor in combination with imatinib in patients with advanced gastrointestinal stromal tumor (GIST): SeliGIST/GEIS-41 trial.

Abstract #: 11534

Selinexor in combination with weekly paclitaxel in patients with advanced or metastatic solid tumors: Results of an open label, single-center, multiarm phase 1b study.

Abstract #: 5565

Once weekly selinexor, carfilzomib, and dexamethasone (XKd) in carfilzomib nonrefractory multiple myeloma (MM) patients.

Abstract #: 8038

A randomized, open-label, phase 3 study of low-dose selinexor and lenalidomide (Len) versus len maintenance post autologous stem cell transplant (ASCT) for newly diagnosed multiple myeloma (NDMM): ALLG MM23, Sealand.

Abstract #: TPS8055

Molecular predictors of response to selinexor in advanced unresectable de-differentiated liposarcoma (DDLS).

Abstract #: 11509

Selinexor containing regimens in patients with multiple myeloma (MM) previously treated with anti-CD38 monoclonal antibodies (CD38 mAbs).

Abstract #: e20020

About Antengene

Antengene Corporation Limited ("Antengene", SEHK: 6996.HK) is a leading clinical-stage R&D driven biopharmaceutical company focused on innovative medicines for oncology and other life-threatening diseases. Antengene aims to provide the most advanced anti-cancer drugs to patients in the Asia Pacific Region and around the world. Since its establishment in 2017, Antengene has built a broad and expanding pipeline of clinical and pre-clinical stage assets through partnerships as well as in-house drug discovery, and obtained 15 investigational new drug (IND) approvals and submitted 5 new drug applications (NDAs) in multiple markets in Asia Pacific. Antengene's vision is to "Treat Patients Beyond Borders". Antengene is focused on and committed to addressing significant unmet medical needs by discovering, developing and commercializing first-in-class/best-in-class therapeutics.

SOURCE Antengene Corporation Limited

http://www.antengene.com

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Antengene Announces Fifteen Clinical Studies and Results of Selinexor to be Presented at ASCO 2021 - PRNewswire

Magenta Therapeutics Announces Additional Preliminary Positive Results from Ongoing Phase 2 Clinical Trial of MGTA-145 and Plerixafor in Patients with…

Posted: at 1:51 am


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplants to more patients, today announced additional positive results from a Phase 2 clinical trial of MGTA-145 and plerixafor in patients with multiple myeloma at the American Society of Clinical Oncology (ASCO) Annual Meeting, being held virtually June 4-8, 2021.

We are very pleased to see continued favorable results for MGTA-145 and plerixafor for stem cell mobilization and collection in patients with multiple myeloma, said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics. These results build on those previously disclosed from this study and the Phase 1 trials to further demonstrate MGTA-145 and plerixafors potential as a rapid, reliable, well-tolerated approach to stem cell mobilization and collection, which has positive implications for patients and donors.

Additional Results MGTA-145 Multiple Myeloma Phase 2 Clinical Trial

The investigator-initiated, 25-patient Phase 2 clinical trial is designed to evaluate the ability of MGTA-145, in combination with plerixafor, to mobilize and collect hematopoietic stem cells for autologous stem cell transplant in patients with multiple myeloma. This study is led by Surbhi Sidana, M.D., Assistant Professor of Medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford University School of Medicine.

Previously reported results from this trial were announced on May 12, 2021, in a published abstract for the European Hematology Association (EHA) Congress and provided preliminary data from the initial cohort of 10 patients.

Summary of cumulative results through data cut-off date:

As indicated previously, this trial has broad and clinically representative inclusion criteria and includes patients that represent the general transplant-eligible population of patients with multiple myeloma. Patients enrolled in this trial included those patients with risk factors that could impact stem cell mobilization and collection, such as myeloma-directed therapies that are known to impact stem cell collection, previous malignancy treated with chemotherapy and/or radiation, and other co-morbid conditions. Mobilization agents may be less effective in patients with multiple risk factors. Final clinical data from this trial are anticipated by the end of 2021. MGTA-145 is also being evaluated for its ability to mobilize and collect stem cells from donors for allogenic transplant in patients with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) in a Phase 2 trial, and an additional Phase 2 study is planned to initiate in patients with sickle cell disease in the second half of 2021.

ASCO Poster Presentation

Title: Phase 2 Study of MGTA-145 + Plerixafor for Rapid and Reliable Hematopoietic Stem Cell (HSC) Mobilization for Autologous Stem Cell Transplant in Multiple Myeloma (Abstract #8023)

Author: Surbhi Sidana, M.D., Assistant Professor of Medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University School of MedicinePoster Session: Hematologic Malignancies Plasma Cell DyscrasiaDate/Time: All e-posters are now available in the ASCO Annual Meeting virtual platform

These results will also be presented as an encore at the EHA Virtual Congress, available via the conferences virtual platform on Friday, June 11 at 3:00am EDT / 9:00am CEST.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of stem cell transplant to more patients with blood cancers, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant community to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Magentas future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting pre-clinical and clinical data, the anticipated timing of clinical trials and regulatory filings, the development of product candidates and advancement of preclinical programs, the potential benefits of our product candidates, the timing, progress and success of collaborations, as well as other statements containing the words anticipate, believe, continue, could, endeavor, estimate, expect, anticipate, intend, may, might, plan, potential, predict, project, seek, should, target, will or would and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether preliminary results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials or to market products; whether Magenta's cash resources will be sufficient to fund Magenta's foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, uncertainties and assumptions regarding the impact of the continuing COVID-19 pandemic on Magentas business, operations, strategy, goals and anticipated timelines, Magentas ongoing and planned preclinical activities, Magentas ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, Magentas timelines for regulatory submissions and Magentas financial position; and other risks concerning Magenta's programs and operations are described in additional detail in its Annual Report on Form 10-K filed on March 3, 2021, its Quarterly Reports on Form 10-Q and its other filings made with the Securities and Exchange Commission from time to time. Although Magenta's forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Magenta. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Magenta undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.

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Magenta Therapeutics Announces Additional Preliminary Positive Results from Ongoing Phase 2 Clinical Trial of MGTA-145 and Plerixafor in Patients with...

Antengene Announces Abstracts on Twelve Clinical Studies of Selinexor Selected by EHA 2021 – PRNewswire

Posted: at 1:51 am


SHANGHAI and HONG KONG, June 1, 2021 /PRNewswire/ -- Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, announced that results from twelve studies of selinexor, the world's first approved oral selective inhibitor of nuclear export (SINE), were selected for the European Hematology Association (EHA) Annual Congress, taking place in a virtual format on June 9 to 17.

Selected Abstracts:

Phase 2 MARCH study of oral ATG-010 plus low dose dexamethasone in Chinese patients withrelapsed/refractory multiple myeloma previously exposed to an immunomodulatory agent and a proteasome inhibitor.

Abstract #: PB1670

Once weekly selinexor, carfilzomib, and dexamethasone (XKd) in carfilzomib nonrefractory multiple myeloma (MM) patients.

Abstract #: S188

Selinexor containing regimens in patients with multiple myeloma previously treated with anti-CD38 monoclonal antibodies.

Abstract #: EP1002

Oral selinexor, pomalidomide, and dexamethasone (XPd) at recommended phase 2 dose in relapsed/refractory multiple myeloma (MM).

Abstract #: EP1008

Ciltacabtagene Autoleucel versus selinexor + dexamethasone in patients with relapsed/refractory multiple myeloma (RRMM) treated with 3 lines of prior therapy: A matching adjusted indirect comparison.

Abstract #: EP1049

Cost effectiveness comparison of belantamab mafodotin and selinexor inrelapsed refractory multiple myeloma.

Abstract #: EP1173

Lymphocyte count effect on efficacy and safety of single agent oral selinexor in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL): A post-hoc analysis from phase 2B SADAL study.

Abstract #: EP530

Genomic correlates of respones to selinexor inmultiple myeloma from the BOSTON study reveal a predictive signature.

Abstract #: EP936

Effects of selinexor on previouslytreated multiple myeloma (MM) with RAS-mutations.

Abstract #: EP966

Efficacy and safety of selinexor, bortezomib, and dexamethasone based on refractory status to lenalidomide in patients with previously treated multiple myeloma: A post-hoc analysis of the BOSTON study.

Abstract #: EP974

Survival among older patients with previously treatedmultiple myeloma treated with selinexor, bortezomib, and dexamethasone (XVd) in the BOSTON study.

Abstract #: EP976

Updated overall survival of eltanexor for the treatment of patients with hypomethylating agent refractory myelodysplastic syndrome.

Abstract #: EP924

About Antengene

Antengene Corporation Limited ("Antengene", SEHK: 6996.HK) is a leading clinical-stage R&D driven biopharmaceutical company focused on innovative medicines for oncology and other life threatening diseases. Antengene aims to provide the most advanced anti-cancer drugs to patients in the Asia Pacific Region and around the world. Since its establishment in 2017, Antengene has built a broad and expanding pipeline of clinical and pre-clinical stage assets through partnerships as well as in-house drug discovery, and obtained 15 investigational new drug (IND) approvals and submitted 5 new drug applications (NDA) in multiple markets in Asia Pacific. Antengene's vision is to "Treat Patients Beyond Borders". Antengene is focused on and committed to addressing significant unmet medical needs by discovering, developing and commercializing first-in-class/best-in-class therapeutics.

SOURCE Antengene Corporation Limited

http://www.antengene.com

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Antengene Announces Abstracts on Twelve Clinical Studies of Selinexor Selected by EHA 2021 - PRNewswire

Clinical Trial Results of the BCMA CAR-T Co-developed by Innovent and IASO Biotherapeutics Commented in American Society of Hematology’s Medical…

Posted: at 1:51 am


SAN FRANCISCO, U.S. and SUZHOU, China, June 2, 2021 /PRNewswire/ --Innovent Biologics, Inc. ("Innovent", HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, together with IASO Biotherapeutics (IASO Bio), today jointly announced that the results of initial clinical studies on treatment of relapsed or refractory multiple myeloma (R/R MMin subjects using fully human BCMA-targeting CAR, an investigational chimeric antigen receptor (CAR) T-cell therapy co-developed by IASO Bio and Innovent (Innovent: IBI326, IASO Bio: CT103A) was published in "Blood", a peer-reviewed medical journal specializing in hematology. The name of the article is "A phase 1 study of a novel fully human BCMA-targeting CAR (CT103A) in patients with relapsed/refractory multiple myeloma." The editors of "Blood" were so impressed by the unique persistence of IBI326 and the authors' exposition on the re-treatment prospects of the disease during the studies that they invited experts from University College London Cancer Institute to write a review entitled "BCMA CARs in multiple myeloma: room for more" (DOI 10.1182/blood.2021010833).

Background and results of IBI326 clinical studies:

Despite recent advances in multiple myeloma (MM) treatment strategies, particularly the emergence and clinical application of immunomodulatory drugs, proteasome inhibitors, and monoclonal antibodies that have improved the survival of MM, it remains an incurable plasma cell cancer, and relapse is almost inevitable in all patients. Results from previously published clinical trials showed that; 33% to 88% of patients with relapsed/ refractory MM (R/R MM) had objective antimyeloma responses after treatment with antiB-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells 1-7. However, it remains a great challenge to achieve durable responses, and relapse or disease progression is observed in 28% to 88% of patients at a median follow-up time of 2 to 15 months 1-7.

The clinical study published in "Blood"was conducted in Department of Hematology, Tongji Medical College of HUST in China (ChiCTR1800018137). The results showed IBI326 has favorable safety profile with mild and controllable cytokine release syndrome (CRS). IBI326 shows excellent expansion and longer persistence during transfusion. In addition, subjects relapsed from a prior murine BCMA CAR-T treatment were also benefited from IBI326.

Efficacy:

Safety: No Immune effector cell-associated neurotoxicity syndrome (ICANS) was observed in any of the dose groups. CRS was observed from 94% of the subjects, and there was a comparatively high rate of severe CRS (28% grade 3; 13% would have had grade 3 if the highest dose had been excluded). In dose expansion study, investigator recommended 1X106/kg CAR+ T cells as recommended Phase II dose.

CAR T-cell expansion: According to ddPCR-based CAR transgene quantification, the median time to reach peak concentration after infusion was 12 days (range, 7-26 days), a strong indication of rapid CAR expansion. No significant difference was observed among 3 dosing groups. For the 4 patients who had participated in prior murine BCMA CAR T-cell trials, low levels of expansions of previously infused murine CAR T cells were observed with short durations compared with IBI326, indicating that the therapeutic response should mainly be attributed to IBI326 expansion.

Immunogenicity: ADA was tested in all 18 subjects and two samples of one subject were validated as positive for ADA.

Comments on the "Blood" article :

In the article, 3 experts from University College London (UCL) Cancer Institute, Lydia Sarah, Hui Lee and Kwee L. Yong commented on the IBI326 studies (as "CT103" in the article). "Wang et al describe clinical outcomes that we have come to expect in the field today and include sufficient details on their trial protocol and subject cohort to allow considered comparison with other trials globally. The authors are candid in their assessment of the trial, and they discuss the possible impact of their comparatively treatment-naive cohort and their lymphodepletion regimen (combining fludarabine with high-dose cyclophosphamide) on response, toxicity, and CAR persistence. Even so, the study is noteworthy for the duration of CAR persistence and its inclusion of subjects previously treated with a murine BCMA CAR."

They also mentioned that both points are significant because it is well known that CARs targeting CD19 in acute lymphocytic leukemia (ALL) can maintain durable responses in 40% of subjects despite therapy being given for refractory disease and multiple relapses8. However, even with initial disease regression, durable responses with CAR T cells have not been achieved in MM9-11. In the first 2 BCMA CAR trials reported, subjects had a median PFS of 1 year. Although the outcomes reported in the CARTITUDE-1 trial were better, it is still not clear whether prolonged remissions can be obtained with existing CAR T-cell products in MM.

The experts subsequently noted and affirmed the in vivo persistence of IBI326. They compared the clinical trial data for CT103 and 2 other CAR-T drugs launched in the market. "In trials assessing the earliest BCMA CARs, bb2121 and LCAR-B38M/JNJ-4528, persistence was typically 6 months. Given this landscape, the reported persistence of CT103 is notable. The maximum concentration of circulating CT103 is lower than other CARs, but it has a median persistence of 308 days, which will likely increase with follow-up. Robust expansion was seen in all subjects, and at last follow-up (median, 13 months), CAR T cells had fallen below 1 X 102 copies per mg of DNA in only 5 subjects."

In addition to relatively high persistence, the experts also noted relative low binding affinity (10 nM) of CT103. "Another unusual feature of CT103 is its relatively low binding affinity (10 nM), which the authors have usefully compared with that of bb2121. CT103 has a slower on-rate, and both binders have similarly fast off-rates compared with the CD19 binder in tisagenlecleucel and axicabtagene ciloleucel (ie, FMC63). The association between binding affinity and clinical outcomes is still uncertain and is likely to be target and context dependent. In CD19, a lower-affinity CAR has been associated with improved persistence in pediatric ALL in which the authors postulated that a faster off-rate may facilitate serial triggering. More work is needed to define the binding kinetics that may influence the efficacy of BCMA CARs."

They also made special mention of the unusual practice of enrolling 4 subjects who had previously been treated with a murine BCMA CAR for CT103 clinical trials. "Unusually, this study enrolled 4 subjects who had previously been treated with a murine BCMA CAR. CT103 expanded in all 4 subjects, and in 3 of them, there was transient and low-level expansion of the murine BCMA CAR construct after lymphodepletion. This indicates that undetectable levels of CAR T cells can remain at disease relapse with antigen-positive disease, but they are likely to be exhausted or terminally differentiated. This further underlines the importance of defining the drivers of disease control in terms of CAR manufacture or in the tumor niche."

The experts from UCL Cancer Institute ended with positive remarks and optimism on the persistence of CT103 and its treatment of MM, "More widely, we are witnessing rapid developments in MM CAR T-cell therapy. We await maturity of promising but early data from an increasing number of MM CARs jostling for a spot on the global landscape, some of which may achieve persistence to rival CT10313. CT103 thus enters a rather crowded field but is a welcome addition for its report of increased persistence and contribution to the discussion surrounding the prospect of retreatment."

About IBI326 (BCMA CAR-T)

IBI326 is an innovative therapy co-developed by Innovent and IASO Bio. Previous studies indicate subjects with relapsed/refractory multiple myeloma (R/R MM) who received high-dose BCMA-targeting CAR-T cells may achieve better remission but have worse adverse events. Moreover, once the disease progresses again, the re-infusion of CAR-T cells will not be effective. To solve this dilemma, IBI326 has been developed, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3 activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, the construct of the BCMA CAR-T is potent and persistent. In February 2021, IBI326 was granted breakthrough therapy designation by the NMPA for the treatment of relapsed/refractory multiple myeloma.

About Innovent

Inspired by the spirit of "Start with Integrity, Succeed through Action," Innovent's mission is to develop, manufacture and commercialize high-quality biopharmaceutical products that are affordable to ordinary people. Established in 2011, Innovent is committed to developing, manufacturing and commercializing high quality innovative medicines for the treatment of cancer, autoimmune, metabolic diseases, and other major therapeutic areas. On October 31, 2018, Innovent was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 01801.HK.

Since its inception, Innovent has developed a fully integrated multi-functional platform which includes R&D, CMC (Chemistry, Manufacturing, and Controls), clinical development and commercialization capabilities. By leveraging this platform, the company has built a robust pipeline of 24 valuable assets in major therapeutic areas, with 4 products officially approved for marketing in China - TYVYT (sintilimab injection), BYVASDA (bevacizumab biosimilar injection), SULINNO (adalimumab biosimilar injection) and HALPRYZA (rituximab biosimilar injection), one Biologics License Application (BLA)submission for sintilimab accepted by the U.S. FDA, six assets in Phase 3 or pivotal clinical trials, and 14 more molecules in clinical trials. TYVYT (sintilimab injection) was included in the National Reimbursement Drug List (NRDL) in 2019 as the historically first PD-1 inhibitor entering in NRDL and the only PD-1 included in the list in that year.

Innovent has built an international team of advanced talented professionals in high-end biopharmaceutical development and commercialization, including many overseas experts. The company has also entered into strategic collaborations with Eli Lilly and Company, Adimab, Incyte, MD Anderson Cancer Center, Hanmi and other international partners. Innovent strives to work with all relevant parties to help advance China's biopharmaceutical industry, improve drug availability to ordinary people and enhance the quality of the patients' lives. For more information, please visit: http://www.innoventbio.com.

About IASO Bio

IASO Bio is a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and autoimmune diseases. Leveraging its proprietary fully human antibody discovery platform (IMARS), high-throughput CAR-T drug priority platform, and proprietary manufacturing processes, IASO Bio is developing a rich clinical-stage pipeline of multiple autologous and allogeneic CAR-T and biologics product candidates. Currently, the company is developing a diversified portfolio of over 10 novel pipeline products, IASO's leading asset, IBI326, an innovative anti-BCMA CAR-T cell therapy under pivotal study for relapsed/refractory (R/R) multiple myeloma (RRMM), was granted Breakthrough Therapeutic Designation by China's National Medical Products Administration (NMPA) in February 2021. For more information on IASO, please visit http://www.iasobio.com.

Forward-Looking Statements

This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words "anticipate", "believe", "estimate", "expect", "intend" and similar expressions, as they relate to the Company, are intended to identify certain of such forward-looking statements. The Company does not intend to update these forward-looking statements regularly.

These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of the Company with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond the Company's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, the Company's competitive environment and political, economic, legal and social conditions.

The Company, the Directors and the employees of the Company assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialise or turn out to be incorrect.

REFERENCES:

1. Ali SA, Shi V, Maric I, et al. T cells expressing an anti-B-cell maturation antigen chimeric antigen receptor cause remissions of multiple myeloma. Blood. 2016;128(13):1688-1700.

2. Xu J, Wang Q, Xu H, et al. Anti-BCMA CAR-T cells for treatment of plasma cell dyscrasia: case report on POEMS syndrome and multiple myeloma. J Hematol Oncol. 2018;11(1):128.

3.Zhao WH, Liu J, Wang BY, et al. A phase 1, open-label study of LCAR-B38M, a chimeric antigen receptor T cell therapy directed against B cell maturation antigen, in subjects with relapsed or refractory multiple myeloma. J Hematol Oncol. 2018;11(1):141.

4. Brudno JN, Maric I, Hartman SD, et al. T cells genetically modified to express an anti-B-cell maturation antigen chimeric antigen receptor cause remissions of poor-prognosis relapsed multiple myeloma. J Clin Oncol. 2018;36(22): 2267-2280.

5. Raje N, Berdeja J, Lin Y, et al. Anti-BCMA CAR T-cell therapy bb2121 in relapsed or refractory multiple myeloma. N Engl J Med. 2019; 380(18):1726-1737.

6. Xu J, Chen LJ, Yang SS, et al. Exploratory trial of a biepitopic CAR T-targeting B cell maturation antigen in relapsed/refractory multiple myeloma. Proc Natl Acad Sci U S A. 2019; 116(19):9543-9551.

7. Cohen AD, Garfall AL, Stadtmauer EA, et al. B cell maturation antigen-specific CAR T cells are clinically active in multiple myeloma. J Clin Invest. 2019;129(6):2210-2221.

8.Maude SL, Laetsch TW, Buechner J, et al. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N Engl J Med. 2018;378(5):439-448

9.Brudno JN, Maric I, Hartman SD, et al. T cells genetically modified to express an anti-B-cell maturation antigen chimeric antigen receptor cause remissions of poor-prognosis relapsed multiple myeloma. J Clin Oncol. 2018;36(22): 2267-2280.

10. Raje N, Berdeja J, Lin Y, et al. Anti-BCMA CAR T-cell therapy bb2121 in relapsed or refractory multiple myeloma. N Engl J Med. 2019; 380(18):1726-1737.

11. Madduri D, Berdeja JG, Usmani SZ, et al CARTITUDE-1: Phase 1b/2 study of ciltacabtagene autoleucel, a B-cell maturation antigendirected chimeric antigen receptor T cell therapy, in relapsed/refractory multiple myeloma [abstract]. Blood. 2020;136(suppl 1). Abstract 177.

12Ghorashian S, Kramer AM, Onuoha S, et al. Enhanced CAR T cell expansion and prolonged persistence in pediatric subjects with ALL treated with a low-affinity CD19 CAR. Nat Med. 2019;25(9):1408-1414.

13. Mailankody S, Jakubowiak AJ, Htut M, et alOrvacabtagene autoleucel (orva-cel), a B-cell maturation antigen (BCMA)-directed CAR T cell therapy for subjects (pts) with relapsed/ refractory multiple myeloma (RRMM): update of the phase 1/2 EVOLVE study (NCT03430011) [abstract]. J Clin Oncol. 2020; 38(suppl 15). Abstract 8504.

SOURCE Innovent Biologics, Inc.

http://www.innoventbio.com

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Clinical Trial Results of the BCMA CAR-T Co-developed by Innovent and IASO Biotherapeutics Commented in American Society of Hematology's Medical...

Medical Research and the COVID Effect – Avera Health

Posted: at 1:51 am


Worldwide, as cases and deaths escalated, it was a race for a cure or at least an effective treatment for COVID-19.

Closer to home, Avera researchers were on the lookout for opportunities to participate in clinical trials that could offer patients options and ultimately, hope.

This journey started with giving convalescent plasma to patients diagnosed with COVID-19. Individuals who recovered from COVID-19 were asked to donate plasma and these antibodies were transferred to hospitalized patients in the hopes this would help convey additional protections. This treatment seemed to help some patients, but the data was unclear as to how well it worked. As physicians and scientists continued to learn more about the virus, numerous other experimental therapeutic options became available.

One experimental therapeutic option is the Regeneron monoclonal antibody cocktail, known as REGEN-COV. This is a cocktail of two lab-developed synthetic antibodies that specifically target and neutralize the virus by attaching to the spike protein of COVID thus preventing the spikes from latching onto healthy cells.

As COVID cases began appearing in the region, Avera research experts began working with their contacts to find opportunities. Our physicians learned all that was available about the virus and reviewed many clinical trial protocols trying to target the ones with the most promise, said Amy Elliott, PhD, Chief Clinical Research Officer of Avera Research Institute. Regeneron emerged as being highly recommended. Avera underwent a competitive process and was selected by Regeneron for these studies due to its integrated hospital structure and strong background in clinical research, Elliott added.

In August, Avera started by enrolling patients in three Regeneron studies for hospitalized COVID patients, non-hospitalized COVID patients, and people who were exposed to COVID-19. Enrolling patients were made aware that they would either receive the study drug or a placebo.

Enrollment in these trials went exceptionally well and we were considered by Regeneron to be a high enrolling site, Elliott said.

Regeneron went on to receive emergency use authorization (EUA) in November at the height of the COVID surge in the Midwest. Patients could receive Regeneron only if they met certain criteria, so the ongoing clinical studies continued to give people options.

Bamlanivimab, another monoclonal antibody treatment, also received EUA in November.

At Avera, an analysis of patients from mid-November of 2020 to January 2021 followed 356 patients who received monoclonal antibody treatment. A 4.5% hospitalization rate was noted compared to a projection of 9-11% for this group of patients.

Regeneron recently announced results that indicate that the cocktail is effective in preventing the onset of symptoms and lessening the severity of symptoms in someone who tests positive for COVID.

Avera McKennan Hospital & University Health Center was also a clinical collaborator with Totient and Ginkgo Bioworks to identify antibodies against COVID-19 for further development.

And, early on, Avera treated hospitalized patients with the antiviral drug remdesivir which received EUA in May and FDA approval in October 2020. Avera participated in a clinical trial with Gilead to look at the efficacy of using remdesivir with patients not requiring hospitalization. This study recently ended patient recruitment with results coming.

Most often with research, it takes a long time to see effects reach a large patient population, Elliott said. It was very inspirational to know we played an important role in helping to bring Regeneron to EUA. Our physicians were ahead of the curve when it came to embracing that research, and they could see first-hand the impact it was making.

Even before the EUA was granted for Regeneron, it was motivating to caregivers to be able to offer their patients some hope through participating in the clinical studies, even though they knew that half the patients participating would receive a placebo, said Jyoti Angal, MPH, CIP, Director of Clinical Research at Avera Research Institute.

Clinical trials provided options to patients as physicians considered how to treat their disease based on the available science of the time. We were honored to take part in the Regeneron study and help bring that drug to our region earlier than anyone else did, Elliott said.

COVID has had its effect on how we work, play, travel and relate to other people. Its also had its effect on research.

One of the things the pandemic caused us to do was to look at non-traditional data collection methods. Researchers will continue to be motivated to reach out to people who cant come to us, Angal said.

People from many aspects of the organization came together to allow Avera to set up its research clinic very quickly. COVID also intensified physician interest in research and provided learning opportunities for medical residents from the University of South Dakota COVID research not only brought about effective treatments for the virus research brought about vaccines, all of which are having a worldwide impact for the greater good. Because of that, theres increasing interest for people going into the field of medical research, Angal said.

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Medical Research and the COVID Effect - Avera Health

Protalix Biotherapeutics and Chiesi Global Rare Diseases Provide Update Regarding Clinical Development of PRX-102 for Treatment of Fabry Disease -…

Posted: at 1:51 am


Protalix will host a conference call today, June 2, 2021, at 8:30 am Eastern Daylight Time, to review regulatory matters related to the development of PRX-102. To participate in the conference call, please dial the following numbers prior to the start of the call:

Conference Call Details:

Wednesday, June2, 2021, 8:30am Eastern Daylight Time (EDT)Domestic:1-877-423-9813International: 1-201-689-8573Conference ID: 13720271

The conference call will also be webcast livefrom the Protalix website and will be available via the following links:

Webcast Details:

Company Link: https://protalixbiotherapeutics.gcs-web.com/events0 Webcast Link: https://tinyurl.com/42857k4w Conference ID: 13720271

Please access the websites at least 15 minutes ahead of the conference to register, download and install any necessary audio software.

PRX-102Development Program Update

PRX102 is currently being studied in the pivotal PhaseIII BALANCE clinical trial and in two ongoing long-term extension studies, all of which are part of the overall clinical development of PRX102 for the proposed treatment of Fabry disease. The BALANCE study is a 24month, randomized, double-blind, active control study of PRX102 in Fabry patients with impaired renal function and is designed to evaluate the safety and efficacy of 1mg/kg of PRX102 dosed every two weeks compared to agalsidase beta (Fabrazyme). The study enrolled 78 patients who were randomized on a 2:1 scheme. The BALANCE study is ongoing and assignment to treatment arm remains blinded.

The primary endpoint of the interim analysis is the comparison of mean annualized changes (slope) of the eGFR (CKD-EPI) after completion of at least 12 months of treatment between the two treatment arms. The interim efficacy analysis was conducted on two pre-defined analysis sets: Intention to Treat (ITT), consisting of all randomized patients who received at least one dose (77patients), considered as the primary analysis for this interim review; and the Per Protocol (PP), consisting of all patients who completed at least 12 months of treatment with no major protocol violations (74 patients). The patient population (ITT analysis set) of the study is comprised of 47 males (61%) and 30 females (39%) with a mean age of 44.3 years.

The initial top-line results show that the lower boundary of the confidence interval for the mean difference between the two treatments was below the non-inferiority margin pre-specified for this interim analysis in the ITT analysis set and above such limit in the PP analysis set. At the time of this analysis, two patients discontinued participation due to treatment emergent adverse events (TEAEs). Of these two patients, one discontinued participation due to a related adverse event. No deaths were registered. Overall, safety data appears favorable and consistent with what was observed in previous clinical studies with PRX-102. Unblinded final data is anticipated to be released in the second quarter of 2022 after all remaining patients have completed the 24-month treatment period.

Based on the interim analysis of the 12-month data generated from the BALANCE study, and in combination with previously reported positive data from the PhaseIII BRIGHT and BRIDGE clinical trials of PRX102, Protalix and Chiesi intend to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the review of PRX102 for the proposed treatment of Fabry disease.

"We look forward to submission of the MAA to EMA for the European Union and to continuing to work with the FDA toward approval in the United States," said Dror Bashan, Protalix's President and Chief Executive Officer. "These regulatory milestones are currently our primary focus."

"The BALANCE study continues as planned through its 24-month treatment duration to support its final analysis. Based on the entire clinical development program, which includes the BRIGHT and BRIDGE studies, we believe that PRX102 has the potential to become an important treatment option for both male and female Fabry patients. The BRIGHT and BRIDGE studies have been completed and the studies met the defined endpoints," continued Mr. Bashan.

In addition to the BALANCE study, the PRX-102 clinical program currently includes extension studies for patients who completed the BRIDGE, BRIGHT and BALANCE studies, as well as a PhaseI/II clinical trial of PRX102. Currently, more than 100 patients who participated in such studies continue to be treated in the extension studies, and additional patients completing the BALANCE study are expected to join the extension studies.

"We thank the patients and clinicians participating in our completed and ongoing clinical studies evaluating PRX-102. As we plan for MAA submission in the EU, we remain committed to advancing our development program for PRX-102 in the United States while also making access to therapy available to eligible patients through our U.S. expanded access program," said Giacomo Chiesi, head of Chiesi Global Rare Diseases.

Regarding the regulatory process in the United States, Protalix and Chiesi plan to submit a TypeA meeting request with the FDA to discuss the path for approval of PRX102.

About Pegunigalsidase Alfa (PRX102)

Pegunigalsidase alfa (PRX102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant a-Galactosidase-A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX102 has been observed to have a circulatory half-life of approximately 80 hours. Protalix designed PRX102 to potentially address the continued unmet clinical need in Fabry patients.

About Protalix BioTherapeutics, Inc.

Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. Protalix was the first company to gain U.S.Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix's unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.

Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved by the FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.

Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified stabilized version of the recombinant human a-GalactosidaseA protein for the treatment of Fabry disease; alidornase alfa or PRX110, for the treatment of various human respiratory diseases or conditions; PRX115, a plant cell-expressed recombinant PEGylated uricase for the treatment of refractory gout; PRX119, a plant cell-expressed long action DNase I for the treatment of NETs-related diseases; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa, and with SarcoMed USA, Inc. for the worldwide development and commercialization of PRX110 for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis, and other related diseases via inhaled delivery.

About Chiesi Global Rare Diseases

Chiesi Global Rare Diseases is a business unit of the Chiesi Group established in February 2020 and focused on research and development of treatments for rare and ultra-rare disorders. The Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments available. The unit is also a dedicated partner with global leaders in patient advocacy, research and patient care. For more information visit http://www.chiesiglobalrarediseases.com.

About Chiesi Group

Based in Parma, Italy, Chiesi Farmaceutici is an international research-focused healthcare group with 85 years of experience in the pharmaceutical industry and a global presence in 29 countries. Chiesi researches, develops, and markets innovative drugs in the respiratory therapeutics, specialist medicine, and rare disease areas. Its R&D organization is headquartered in Parma (Italy), and is integrated with R&D groups in France, the USA, the UK, and Sweden to advance Chiesi's pre-clinical, clinical, and registration programs. Chiesi employs nearly 6,000 people.

Chiesi Group is a certified Benefit corporation. For more information, please visit http://www.chiesi.com.

Protalix BioTherapeutics Forward-Looking Statements Disclaimer

To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "may," "plan," "will," "would," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: risks related to the timing and progress of the preparation of an updated BLA addressing the complete response letter; risks related to the timing, progress and likelihood of final approval by the FDA of a resubmitted BLA for PRX102 and, if approved, whether the use of PRX102 will be commercially successful; failure or delay in the commencement or completion of our preclinical studies and clinical trials, which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; and inability to monitor patients adequately during or after treatment; delays in the approval or potential rejection of any applications we file with the FDA, EMA or other health regulatory authorities, and other risks relating to the review process; risks associated with the novel coronavirus disease, or COVID19, outbreak, which may adversely impact our business, preclinical studies and clinical trials; risks related to any transactions we may effect in the public or private equity markets to raise capital to finance future research and development activities, general and administrative expenses and working capital; the risk that the results of the clinical trials of our product candidates will not support the applicable claims of safety or efficacy, or that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks related to our ability to maintain and manage our relationship with our collaborators, distributors or partners; risks related to the amount and sufficiency of our cash and cash equivalents; risks relating to our ability to make scheduled payments of the principal of, to pay interest on or to refinance our outstanding notes or any other indebtedness; our dependence on performance by third party providers of services and supplies, including without limitation, clinical trial services; delays in our preparation and filing of applications for regulatory approval; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies and institutions; potential product liability risks, and risks of securing adequate levels of product liability and other necessary insurance coverage; and other factors described in our filings with the U.S.Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law.

Protalix BioTherapeutics Investor Contact

Chuck Padala, Managing DirectorLifeSci Advisors+1-646-627-8390[emailprotected]

Chiesi Global Rare Diseases Media Contact

Jenna UrbanBerry & Company Public Relations +1-212-253-8881[emailprotected]

SOURCE Protalix BioTherapeutics, Inc. and Chiesi

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Protalix Biotherapeutics and Chiesi Global Rare Diseases Provide Update Regarding Clinical Development of PRX-102 for Treatment of Fabry Disease -...

Adaptive Phage Therapeutics Initiates Phase 1/2 Trial of PhageBank in Urinary Tract Infections – Business Wire

Posted: at 1:51 am


GAITHERSBURG, Md.--(BUSINESS WIRE)--Adaptive Phage Therapeutics (APT), a clinical-stage biotechnology company dedicated to providing therapies to treat infectious diseases, today announced that the first patient has been dosed in a Phase 1/2 study of bacteriophage therapy (PhageBank) to evaluate the safety, tolerability, and efficacy of targeted, personalized, bacteriophage (phage) treatments in patients with urinary tract infection (UTI).

This multi-center clinical trial represents the first study of an expanding phage library for intravenous administration and/or bladder instillation of phage therapy. PhageBank is APTs continually expanding phage library that functions as an integrated logistics platform to dispense phage on demand. The phage distributed are selected via a PhageBank Susceptibility Test (PST) that is being advanced and commercialized by APT in collaboration with Mayo Clinic Laboratories. The PST enables rapid, automated identification of individual phage to be included in patient-specific therapy to treat bacterial infections.

Im delighted to announce the initiation of APTs first PhageBank clinical trial, which is being funded in part by the U.S. Department of Defense through an advanced development contract intended to accelerate clinical progression of PhageBank therapy to more effectively treat multidrug-resistant infections, said Greg Merril, CEO and co-founder, Adaptive Phage Therapeutics. This Phase 1/2 UTI trial is the first of several initial indications in which APT plans to study PhageBank, following the success achieved in PhageBank treatment of more than 40 emergency INDs for individual patients. APT recently received IND clearance for PhageBank treatment of prosthetic joint infection (PJI) and diabetic foot osteomyelitis (DFO), and plans to initiate clinical studies for these two indications. Initial data readouts in PJI and DFO are expected in 2022.

The UTI Phase 1/2 study, evaluating the safety and efficacy of bacteriophage therapy, is being conducted at the James J. Peters VA Medical Center in Bronx, New York, as well as multiple other U.S. clinical study sites, and is supported in part by the U.S. Department of Defense (DoD) under a $14.2 million advanced development contract.

This interventional, randomized, placebo-controlled, open label study is designed to enroll approximately 156 patients. Patients enrolled have urinary tract infections due to either E. coli or K. pneumoniae. The trial will use pre-specified criteria to determine phage regimens for evaluation. Results from an initial cohort will be used to confirm or modify the phage dosing regimen and route, before progressing to patients with symptomatic infection at a higher risk of recurrence. Patients will be followed for bacterial clearance or recurrence of urinary tract infection. Clinical results from this trial are expected to lead to regulatory approval to advance directly into pivotal trials for chronic and recurrent urinary tract infections.

Adaptive Phage Therapeutics, Inc.

Adaptive Phage Therapeutics (APT) is a clinical-stage company advancing therapies to treat multi-drug resistant infections. Prior antimicrobial therapeutic approaches have been fixed, while pathogens continue to evolve resistance to each of those therapeutics, causing those drug products to become rapidly less effective in commercial use as antimicrobial resistance (AMR) increases over time.

APTs PhageBank approach leverages an ever-expanding library of bacteriophage (phage) that collectively provide evergreen broad spectrum and polymicrobial coverage. PhageBank phages are matched through a proprietary phage susceptibility assay that APT has teamed with Mayo Clinic Laboratories to commercialize on a global scale.

APTs technology was originally developed by the biodefense program of U.S. Department of Defense. APT acquired the world-wide exclusive commercial rights in 2017. Under FDA emergency Investigational New Drug allowance, APT has provided investigational PhageBank therapy to treat more than 40 critically ill patients in which standard-of-care antibiotics had failed.

For more information, visit http://www.aphage.com.

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Adaptive Phage Therapeutics Initiates Phase 1/2 Trial of PhageBank in Urinary Tract Infections - Business Wire

Kintara Presents Updates on Two Phase 2 Clinical Trials at the 2021 American Association for Cancer Research Annual Meeting – The Baytown Sun

Posted: April 16, 2021 at 1:47 am


SAN DIEGO, April 12, 2021 /PRNewswire/ -- Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company developing novel cancer therapies for patients who are failing, or resistant to, current treatment regimens, today announced interim data results from two Phase 2 clinical trials evaluating VAL-083, the Company's lead compound, for the treatment of glioblastoma multiforme (GBM). The data were presented in two posters at the 2021 American Association for Cancer Research (AACR) Annual Meeting, which is taking place virtually from April 10-15, 2021.

Poster CT238 provides an update from two patient groups receiving VAL-083 in an open-label, Phase 2 study in recurrent and adjuvant unmethylated GBM settings being conducted at the MD Anderson Cancer Center in Houston, Texas. The second poster, CT172, updates the open-label, Phase 2 study of VAL-083 as a first-line treatment in newly-diagnosed, unmethylated GBM patients being conducted at Sun Yat-sen University Cancer Center in China.

"These interim data updates at the AACR Annual Meeting continue to demonstrate VAL-083's potential as a game-changing treatment option for GBM patients," commented Saiid Zarrabian, Kintara's Chief Executive Officer. "Furthermore, it's important to note that both trials have provided valuable insights as we prepared to initiate the VAL-083 arm of the Global Coalition for Adaptive Research GBM AGILE registrational study which commenced patient enrollment in February 2021."

Poster CT238: "Phase 2 study of dianhydrogalactitol (VAL-083) in patients with MGMT-unmethylated, bevacizumab-nave glioblastoma in the adjuvant or recurrent setting"

In newly-diagnosed patients receiving VAL-083 as adjuvant therapy following treatment with radiation and temozolomide (TMZ), for the 33 efficacy evaluable patients (of a planned 36 patients) as of the data cut-off of March 12, 2021, median progression-free survival (PFS) is currently 10.0 months (95% confidence interval: CI 8.2-10.8). While not a head-to-head study, this PFS data compares favorably to historical TMZ control of 5.3 months* and 6.9 months**, respectively.

For patients in the fully enrolled recurrent group receiving second-line therapy with VAL-083 following first-line TMZ failure, 89 patients have been enrolled as of the data cut-off of March 12, 2021 with 35 patients (35 efficacy evaluable) initially receiving a dose of 40 mg/m2/day and 54 (48 efficacy evaluable) initially receiving the treatment dose that is being carried forward in the GBM AGILE study of 30 mg/ m2/day on days 1, 2 and 3 of a 21-day cycle. Median overall survival (mOS) for the 83 efficacy evaluable patients who have completed at least once cycle of treatment was 7.5 months (CI 6.0-9.0 months). Additionally, for the 48 efficacy evaluable patients initially receiving a dose of 30 mg/ m2/day, mOS is currently 7.9 months (CI 5.9-9.9 months). While this is not a head-to-head trial, historically lomustine, which is the most commonly used chemotherapy for these patients, has demonstrated mOS of 7.2 months***.

Consistent with prior studies, myelosuppression is the most common adverse event with VAL-083 in both the recurrent GBM and adjuvant treatment settings. In the 30 mg/m2/day starting dose cohort (the dose that is being carried forward in the GBM AGILE study) seven subjects have experienced a serious adverse event (SAE) possibly related to VAL-083 in the recurrent group and one patient has experienced a possibly drug-related SAE in the adjuvant group as of the relevant data cut-off dates.

Poster CT172: "Phase 2 clinical trial of dianhydrogalactitol (VAL-083) in patients with newly-diagnosed MGMT-unmethylated GBM"

In the open-label, Phase 2 study of VAL-083 as a first-line treatment in newly-diagnosed, unmethylated GBM patients, median PFS for the 29 patients, as of the March 11, 2021 cut-off date, is currently 9.3 months (CI 6.4-12.0 months). Additionally, for the 25 patients initially receiving the treatment dose that is being carried forward in the GBM AGILE study of 30 mg/m2/day on days 1, 2 and 3 of a 21-day cycle, median PFS was reported to be 8.7 months (CI 6.4-12.5 months). While not a head-to-head study, this PFS data compares favorably to historical TMZ control of 5.3 months* and 6.9 months**, respectively. Three subjects have experienced an SAE possibly related to VAL-083. Multiple treatment cycles of VAL-083 at the 30 mg/m2/day dose in combination with standard radiation treatment (2 Gy/day, 5 days/week) were shown to be generally safe and well-tolerated. This study has been fully enrolled, and all patients have completed treatment with VAL-083 and are currently in follow-up.

*Hegi et al N Eng J Med 352; 997-1003 (2005) **Tanguturi et al. NeuroOncol. 19(7): 908-917 (2017) *** Wick et al N.Eng.J.Med . 377:1954 1963 (2017)

About Kintara Located in San Diego, California, Kintara (Nasdaq: KTRA) is dedicated to the development of novel cancer therapies for patients with rare unmet medical needs. Kintara is currently developing two Phase 3-ready therapeutics, VAL-083 for GBM and REM-001 for cutaneous metastatic breast cancer (CMBC).

VAL-083 is a "first-in-class", small-molecule chemotherapeutic with a novel mechanism of action that has demonstrated clinical activity against a range of cancers, including central nervous system, ovarian and other solid tumors (e.g., NSCLC, bladder cancer, head and neck) in U.S. clinical trials sponsored by the National Cancer Institute (NCI). Based on Kintara's internal research programs and these prior NCI-sponsored clinical studies, Kintara is currently conducting clinical trials to support the development and commercialization of VAL-083 in GBM.

REM-001 is a proprietary, late-stage photodynamic therapy platform that holds promise as a localized cutaneous, or visceral, tumor treatment as well as in other potential indications. REM-001 therapy has been previously studied in four Phase 2/3 clinical trials in patients with CMBC who had previously received chemotherapy and/or failed radiation therapy. With clinical efficacy of 80% complete responses of CMBC evaluable lesions and an existing robust safety database of approximately 1,100 patients across multiple indications, Kintara is advancing the REM-001 CMBC program to late-stage pivotal testing.

For more information, please visit http://www.kintara.com or follow us on Twitter at @KintaraThera, Facebook and Linkedin.

Safe Harbor Statement Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including statements regarding the status of the Company's clinical trials and the GBM AGILE study. Any forward-looking statements contained herein are based on current expectations but are subject to a number of risks and uncertainties. The factors that could cause actual future results to differ materially from current expectations include, but are not limited to, risks and uncertainties relating to the impact of the COVID-19 pandemic on the Company's operations and clinical trials; the Company's ability to develop, market and sell products based on its technology; the expected benefits and efficacy of the Company's products and technology; the availability of substantial additional funding for the Company to continue its operations and to conduct research and development, clinical studies and future product commercialization; and the Company's business, research, product development, regulatory approval, marketing and distribution plans and strategies. These and other factors are identified and described in more detail in the Company's filings with the SEC, including the Company's Annual Report on Form 10-K for the year ended June 30, 2020, the Company's Quarterly Reports on Form 10-Q, and the Company's Current Reports on Form 8-K.

CONTACTS Investors CORE IR 516-222-2560 ir@coreir.com

Media Jules Abraham Director of Public Relations CORE IR 917-885-7378 julesa@coreir.com

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Kintara Presents Updates on Two Phase 2 Clinical Trials at the 2021 American Association for Cancer Research Annual Meeting - The Baytown Sun

Ardelyx Collaboration Partner, Kyowa Kirin, Announces Initiation of Phase 3 Clinical Studies of Tenapanor for Hyperphosphatemia in Japan – PRNewswire

Posted: at 1:47 am


FREMONT, Calif.and WALTHAM, Mass., April 14, 2021 /PRNewswire/ --Ardelyx, Inc. (Nasdaq: ARDX), a biopharmaceutical company focused on developing innovative first-in-class medicines to improve treatment for people with kidney and cardiorenal diseases, today announced that its collaboration partner in Japan, Kyowa Kirin Co., Ltd. (TSE: 4151, Kyowa Kirin), has initiated four Phase 3 clinical studies in Japan evaluating tenapanor for hyperphosphatemia. The achievement of this development milestone triggers a $5 million payment to Ardelyx.

The Phase 3 clinical trials consist of a multi-center, randomized, double-blind, placebo-controlled, parallel-group comparative study; a phosphate binder-combination parallel-group comparative study; an open-label, single-arm study evaluating hyperphosphatemia patients on peritoneal dialysis; and a long-term study evaluating serum phosphorus in patients who switch from one or more phosphate binders to tenapanor for hyperphosphatemia in Japan.

"As we approach our April 29 PDUFA date for tenapanor for the control of serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis and prepare for potential commercialization in the U.S., we are pleased to see the significant progress made by our partner Kyowa Kirin,"said Mike Raab, president and chief executive officer of Ardelyx. "We are thrilled to have key strategic partners like Kyowa Kirin, along with our partners in Canada and China, to support development of, and once approved, patient access to, tenapanor globally."

Under the terms of the license agreement fortenapanorwith Kyowa Kirin, which was signed in 2017Ardelyx received a $30 million upfront payment and is eligible to receive up to $55.0 million in total development milestones and 8.5 billion yen in commercialization milestones. Ardelyx is also eligible to receive high-teen royalties on sales throughout the term of the agreement. Kyowa Kirin has been granted the exclusive rights to develop, market and commercialize tenapanor for cardiorenal diseases and conditions associated with them, including hyperphosphatemia, in Japan.

About Tenapanor for HyperphosphatemiaTenapanor, discovered and developed by Ardelyx, is a first-in-class, proprietary, oral medicine for which an NDA is under review by the FDA for the control of serum phosphorus in adult patients with CKD on dialysis. Tenapanor has a unique mechanism of action and acts locally in the gut to inhibit the sodium hydrogen exchanger 3 (NHE3). This results in a conformational change of the epithelial cell junctions, thereby significantly reducing paracellular uptake of phosphate at the primary pathway of phosphate absorption. Ardelyx is conducting NORMALIZE, an ongoing extension study of the PHREEDOM Phase 3 monotherapy study, which is designed to evaluate the ability of tenapanor, as monotherapy or in combination with sevelamer, to achieve serum phosphorus levels in the normal range (2.5 4.5 mg/dL) in patients with chronic kidney disease (CKD) on dialysis. Planned analyses have demonstrated that the use of tenapanor as a foundational approach, as monotherapy or in combination with sevelamer carbonate, produces a significant phosphorus-lowering effect. After ~ 20 months of treatment with tenapanor alone or with low doses of sevelamer, patients exhibited a mean serum phosphorus reduction of 2.33 mg/dL, from a mean baseline phosphorus of 7.27 mg/dL at the beginning of the PHREEDOM trial to a mean of 4.94 mg/dL.

About Ardelyx, Inc.Ardelyx is focused on discovering, developing, and commercializing innovative first-in-class medicines to enhance the lives of patients with kidney and cardiorenal diseases. Ardelyx is advancing tenapanor, a novel product candidate to control serum phosphorus in adult patients with CKD on dialysis, for which the company's NDA is currently under review by the FDA, with a PDUFA date of April 29, 2021. Ardelyx is also advancing RDX013, a potassium secretagogue, for the potential treatment of elevated serum potassium, or hyperkalemia, a problem among certain patients with kidney and/or heart disease and has an early-stage program in metabolic acidosis, a serious electrolyte disorder in patients with CKD. In addition, Ardelyx received FDA approval of IBSRELA (tenapanor) on September 12, 2019. Ardelyx has established agreements with Kyowa Kirin in Japan, Fosun Pharma in China and Knight Therapeutics in Canada for the development and commercialization of tenapanor in their respective territories.

Forward Looking StatementsTo the extent that statements contained in this press release are not descriptions of historical facts regarding Ardelyx, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor of the Private Securities Reform Act of 1995, including the potential for tenapanor in controlling serum phosphorus in chronic kidney disease patients on dialysis as monotherapy or in combination with Sevelamer carbonate. Such forward-looking statements involve substantial risks and uncertainties that could cause Ardelia's future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties associated with the drug development, regulatory approval and commercialization process. Ardelyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ardelia's business in general, please refer to Ardelia's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 8, 2021, and its future current and periodic reports to be filed with the Securities and Exchange Commission.

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Ardelyx Collaboration Partner, Kyowa Kirin, Announces Initiation of Phase 3 Clinical Studies of Tenapanor for Hyperphosphatemia in Japan - PRNewswire

Clinical Trials Critical for the Evaluation of Advanced Medical Imaging Tools – PRNewswire

Posted: at 1:47 am


NEW YORK, April 14, 2021 /PRNewswire/ -- Image-guided surgery offers surgeons greater control of the procedure and real-time feedback, thus providing better outcomes for patients. Accuracy in surgical procedures is critical for serious or life-threatening conditions, such as cancer, and medical technology companies are providing imaging tools to help surgeons deliver better results to their patients. Companies such as Perimeter Medical Imaging AI (TSXV:PINK), Butterfly Network (NYSE:BFLY), ENDRA Life Sciences Inc. (NASDAQ:NDRA), Asensus Surgical Inc. (NYSE:ASXC),and Avinger Inc. (NASDAQ:AVGR) have engaged in the research and development of advanced imaging tools for use in different procedures. In addition to the development of these tools, these companies are seeking regulatory approval and collaborating in clinical studies that provide data on the application of these imaging tools.

Perimeter Medical Imaging Announces Clinical Study Using OCT Imaging System

Perimeter Medical Imaging AI (TSXV:PINK),an early-stage medical device company seeking to transform cancer surgery with ultra-high resolution, real-time, advanced imaging tools, recentlyannounced the FDA 510(k) approval for the company's Optical Coherence Tomography (OCT) Imaging System, designed to examine tissue microstructures during surgery by providing cross-sectional, real-time margin visualization.

Since the approval of the imaging system, Perimeter Medical Imaging AI has announced acollaboration with Dr. Beth DuPree, a surgeon at Northern Arizona Healthcare Verde Valley Medical Center for a clinical study that will evaluate the use of Perimeter's OCT imaging system during breast-conserving surgery.

For most breast cancer patients, the complete removal of cancerous tissue while conserving as much healthy tissue as possible is often a challenge. Perimeter's OCT Imaging System allows surgeons to get clear margins, thus eliminating the need for re-operation and the associated complications and costs.

The goal of this study is to demonstrate the effectiveness of Perimeter's OCT imaging system during clinical procedures to improve precision when excising tissue during surgery.

Commenting about the study, Dr. Beth DuPree said, "I am excited to lead this study using Perimeter's OCT Imaging System, which provides ultra-high resolution visualization of a specimen in real-time. The use of this technology in my OR adds to my confidence in the final margin status, for patients, the need to re-operate can cause emotional stress, discomfort, and inconvenience, while potentially delaying other treatment and therapies. This is a non-invasive technology that aims to provide better 'peace of mind' for both the patient and surgeon while lowering the likelihood of needing to reschedule another surgery. It is my hope that the data generated from this study will help validate the use of this technology in breast conservation surgery and ultimately create better results for my patients."

The study is open to patients at the Northern Arizona Healthcare Verde Valley Medical Center.

Perimeteralso announced an update to its ATLAS AI Project, which is working on advancing the company's next-gen artificial intelligence and machine learning tools through clinical development. Perimeter's proprietary "ImgAssist" AI technology has been tested and trained using an extensive dataset of excised breast tissue images collected during the first stage of the ATLAS AI Project.

Collaborations and Regulatory Approvals Driving the Adoption of Advanced Medical Tools

The president and CEO of Butterfly Network (NYSE:BFLY) in a March 29 conference call with investors outlined the company's plans to expand in different directions, including creating new uses for its portable ultrasound technology. The company is aiming to forge new collaborations with clinical partners and healthcare organizations that will adopt Butterfly's technology. Butterfly has already secured partnerships with the American College of Cardiology and Atrium Health to expand the use of its iQ+ whole-body ultrasound device in cardiovascular care. Butterfly also received $584 million as part of its $1.5 billion deal with Longview Acquisition Corp.

Pioneer of enhanced ultrasound technologies, ENDRA Life Sciences Inc. (NASDAQ:NDRA) has been collaborating with key players conducting clinical trials for various conditions using its Thermo Acoustic Enhanced Ultrasound (TAEUS). ENDRAannounced a partnership with Inselspital University Hospital in Bern Switzerland for a clinical study of the TAEUS device for assessing Non-Alcoholic Fatty Liver Disease. ENDRA also signed acollaboration agreement with Hepion Pharmaceuticals, a clinical-stage biopharmaceutical company. In this agreement, Hepion will use ENDRA's TAEUS as an add-on technology in the Phase 2b clinical development of CRV431, which targets non-alcoholic steatohepatitis and other liver diseases.

Asensus Surgical, Inc. (NYSE:ASXC), formerly TransEnterix Inc., is a medical device company at the forefront of Performance Guided Surgery. It recentlyannounced an FDA clearance for its Senhance Surgical System, a laparoscopic platform that uses augmented intelligence to provide unmatched performance and patient outcomes through machine learning. This approval comes after an earlier announcement in January 2021, where the company received the CE mark of approval for its Intelligent Surgical Unit for the Senhance Surgical System use in Europe. "The expansion into general surgery for the Senhance Surgical System is a major milestone for the growth and clinical applicability of our technology," said Anthony Fernando, Asensus Surgical President and CEO. "General surgery is, by far, the largest area of manual laparoscopy which can benefit from the precision and insight of Performance-Guided Surgery including previous indications granted, the Senhance Surgical System can now be utilized in over 2.7 million general surgical procedures performed in the US annually."

Avinger Inc. (NASDAQ:AVGR) is a commercial-stage medical device company responsible for the marketing of the first and only intravascular image-guided, catheter-based system for the diagnosis and treatment of patients with peripheral artery disease. The company recentlyannounced that it received 510(k) clearance from the FDA for its Ocelaris, a next-generation image-guided chronic total occlusion crossing system, which will be marketed under the brand name Tigereye. Avinger received the clearance for Tigereye in 2020 and has since launched the CTO crossing system in 30 clinical sites with more than 160 cases successfully performed.

Medical device companies such as Perimeter Medical Imaging AI continue to advance the industry, leading not only to potential breakthroughs that could change how surgeries are performed, but also to better outcomes for the patients.

For more information on Perimeter Medical Imaging AI (TSXV:PINK), click here.

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Clinical Trials Critical for the Evaluation of Advanced Medical Imaging Tools - PRNewswire

Regulus Therapeutics Announces Completion of Dosing in the First Cohort of Phase 1b Clinical Trial of RGLS4326 for the Treatment of Patients with…

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LA JOLLA, Calif., April 15, 2021 /PRNewswire/ --Regulus Therapeutics Inc.(Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that it completed dosing in the first cohort of nine patients with ADPKD in its Phase 1b clinical trial of RGLS4326. The study is evaluating the safety, pharmacokinetics, and biomarkers (polycystin 1 (PC1) and polycystin 2 (PC2)) of multiple doses of RGLS4326 in patients with ADPKD.

"We expect to complete patient follow-up soon and report safety, pharmacokinetics, and biomarker data from this first cohort of patients within the next several weeks once we receive the analysis from our vendors," said Jay Hagan, CEO of Regulus. "Additionally, we are making good progress enrolling patients with ADPKD into the second cohort of this Phase 1b study."

About RGLS4326 Phase 1b

The Phase 1b is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD. The study is designed to evaluate the safety, pharmacokinetics, and changes in levels of polycystin 1 (PC1) and polycystin 2 (PC2) in patients with ADPKD administered RGLS4326 every other week for a total of four doses. The dose level for the first cohort is 1mg/kg of RGLS4326 and the dose level for the second cohort is 0.3mg/kg. The third and final cohort will be dosed at a level to be determined based on the results of the first two cohorts.

For more information about the clinical trial design, please visit http://www.clinicaltrials.gov (NCT04536688).

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD.The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by FDAuntil the second set of requirements outlined by the agency have been satisfactorily addressed. The Company will use information from the Phase 1 clinical studies, including the first cohort of the Phase 1b study together with information from the recently completed additional nonclinical studies generated in 2020, in its plan to address the second set of requirements outlined in the Partial Clinical Hold letter to support studies of extended duration. Regulus plans to discuss its approach to addressing the remaining Partial Clinical Hold requirements with FDA in mid-2021. RGLS4326 has received orphan drug designation from FDA in July 2020.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About Regulus

Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs.Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field.Regulus maintains its corporate headquarters in La Jolla, CA.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the completion of preclinical and clinical activities concerning the RGLS4326 program, the timing of data and the sufficiency of the data resulting from the ongoing or planned preclinical studies required to recommence clinical studies for extended duration dosing and the timing of preclinical and clinical activities. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor of building a business around such drugs, and feedback from the FDA.In addition, while Regulus expects the COVID-19 pandemic to adversely affect its business operations and financial results, the extent of the impact on Regulus' ability to achieve its preclinical and clinical development objectives and the value of and market for its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

SOURCE Regulus Therapeutics Inc.

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Specific Diagnostics Announces the Appointment of Scott Wehage as Director of US Clinical Trials – PR Web

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Specific Diagnostics Patented Sensor Panel

MOUNTAIN VIEW, Calif. (PRWEB) April 15, 2021

Specific Diagnostics today announced the appointment of Scott Wehage as Director of US Clinical Trials. Previously Mr. Wehage was a Clinical Project Manager for Becton Dickinsons Global Clinical Develop Department, where he oversaw clinical trials for device and IVD product development. Mr. Wehage will oversee the implementation of Specifics expanding clinical studies strategy in the US.

We look forward to bringing Specifics Reveal Rapid AST Instrument to the US, with Scotts large-scale clinical trial experience he is ideally suited for this role, said Dr. Paul A. Rhodes, Specifics CEO.

Im truly looking forward to working with all of the distinguished KOL sites we are contracting with for our US clinical trials, said Mr. Wehage. Once FDA approved, I believe the Reveal will truly change the way US hospital systems manage and treat blood stream infections.

Prior to joining Specific, Mr. Wehage has worked in clinical research for over 20 years where he managed clinical trials for Becton Dickinson including a large, multisite US trial that ran for more than 4 years. Previously he was a program manager at Johns Hopkins University. Mr. Wehage has also served as President of the Baltimore Chapter of SoCRA (Society of Clinical Research Associates). He holds a Masters of Science from Johns Hopkins University.

About Specific

Specific Diagnostics has developed in vitro diagnostic systems based upon a unique, patented metabolomic signature technology that enables rapid detection and identification of microorganisms as they grow in culture. Its first commercial application applies this fundamental new platform to the rapid determination of antimicrobial susceptibility directly from positive blood cultures, as well as isolate dilutions. Specific is based in Mountain View, CA.For press inquiries, please contact: press@specificdx.com

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Journal for ImmunoTherapy of Cancer Publishes Phase 1 Clinical Research Showing Belapectin, Galectin Therapeutics’ Galectin-3 Inhibitor, Enhances…

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NORCROSS, Ga., April 13, 2021 (GLOBE NEWSWIRE) -- Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin proteins, today announced that a paper published in the peer-reviewed Journal for ImmunoTherapy of Cancer (JITC), the highest ranked fully open access immunology journal, provides further clinical evidence that using belapectin, a potent galectin-3 inhibitor, in combination with pembrolizumab (KEYTRUDA), a PD-1 inhibitor, significantly enhances tumor response to immunotherapy in patients with advanced metastatic melanoma (MM) and head and neck squamous cell carcinoma (HNSCC).

The paper, titled Enhancing Clinical and Immunological Effects of anti-PD-1 with Belapectin, a Galectin-3 Inhibitor (doi:10.1136/jitc-2021-002371) describes results from an ongoing Phase 1 clinical study, a collaboration between Galectin Therapeutics and Providence Cancer Institute in Portland, Oregon.

Following the recent publication of positive preclinical results that showed the inhibition of galectin-3 in combination with an agonist anti-OX40 monoclonal antibody reprograms the tumor microenvironment to favor anti-tumor activity, the current study tests the clinical hypothesis that galectin-3 blockade with belapectin in combination with pembrolizumab enhances tumor response for patients with advanced MM or HNSCC.

In the study, as previously disclosed, an objective response was observed in 50% of MM (7/14) and 33% of HNSCC (2/6) patients. This compares favorably to published response rates on pembrolizumab alone. The authors noted that the combination was associated with fewer immune-mediated adverse events than anticipated with pembrolizumab alone. In addition, the analysis of patients tumor tissue revealed reduced monocytic myeloid-derived suppressor cells and increased effector memory T-cell activation in responders compared with non-responders. Also, an increased baseline expression of galectin-3 positive tumor cells correlated with clinical response.

Immunotherapy is a significant breakthrough in the treatment of many cancers, but tumor-induced immune suppression contributes to treatment resistance. Galectin-3 is an important driver of this tumor-induced immunosuppression, and this clinical study constitutes proof-of-concept that the addition of belapectin, a galectin-3 inhibitor, to a PD-1 inhibitor can benefit cancer patients, said Dr. Brendan Curti, M.D., Earle A. Chiles Research Institute, a division of Providence, and the first author of the paper.

The analysis of patients tumor tissues is consistent with previously published pre-clinical data with belapectin and confirms the ability of belapectin to modulate the tumor microenvironment to favor anti-tumor activity. The possibility to improve the tolerance and safety of immunotherapy is also very exciting, commented Pol F. Boudes, M.D., Chief Medical Officer of Galectin Therapeutics. These proof-of-concept clinical data provide a strong rationale to initiate a randomized placebo-controlled phase 2 clinical trial to evaluate the efficacy and safety of belapectin in combination with a PD-1 inhibitor compared to a PD-1 inhibitor alone in this cancer patient population. We look forward to continuing our work with Providence Cancer Institute, and we anticipate the upcoming release of additional data from the expansion cohort in this study.

About Belapectin (GR-MD-02)

Belapectin (GR-MD-02) is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of NASH and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs including fibrotic disorders of the liver, lung, kidney, heart and vascular system. Belapectin binds to galectin-3 and disrupts its function. Preclinical data in animals have shown that belapectin has robust treatment effects in reversing liver fibrosis and cirrhosis. A Phase 2 study showed belapectin may prevent the development of esophageal varices in NASH cirrhosis; these results provide the basis for the conduct of the NAVIGATE trial. The NAVIGATE trial (NAVIGATEnash.com), entitled A Seamless Adaptive Phase 2b/3, Double-Blind, Randomized, Placebo-controlled Multicenter, International Study Evaluating the Efficacy and Safety of Belapectin (GR-MD-02) for the Prevention of Esophageal Varices in NASH Cirrhosis began enrolling patients in June 2020 and is posted on http://www.clinicaltrials.gov (NCT04365868). Galectin-3 also has a significant role in cancer, and the Company is supporting a Phase 1 study in combined immunotherapy of belapectin and KEYTRUDA in treatment of advanced melanoma and in head and neck cancer.

About Galectin Therapeutics

Galectin Therapeutics is dedicated to developing novel therapies to improve the lives of patients with chronic liver disease and cancer. Galectins lead drug belapectin (formerly known as GR-MD-02) is a carbohydrate-based drug that inhibits the galectin-3 protein which is directly involved in multiple inflammatory, fibrotic, and malignant diseases, for which it has Fast Track designation by the U.S. Food and Drug Administration. The lead development program is in non-alcoholic steatohepatitis (NASH) with cirrhosis, the most advanced form of NASH-related fibrosis. This is the most common liver disease and one of the largest drug development opportunities available today. Additional development programs are in treatment of combination immunotherapy for advanced melanoma and other malignancies. Advancement of these additional clinical programs is largely dependent on finding a suitable partner. Galectin seeks to leverage extensive scientific and development expertise as well as established relationships with external sources to achieve cost-effective and efficient development. Additional information is available at http://www.galectintherapeutics.com.

About Providence Cancer Institute

Providence Cancer Institute, a part of Providence St. Joseph Health, offers the latest in cancer services, including diagnostic, treatment, prevention, education, support and internationally renowned research. Providence Cancer Institute is home to the Earle A. Chiles Research Institute, a world-class research facility located within the Robert W. Franz Cancer Center in Portland, Oregon, and is a recognized leader in the field of cancer immunotherapy since 1993. Investigators lead more than 400 active clinical trials in key areas such as cancers of the: breast, colon/rectum, prostate, lung, esophagus, liver and pancreas, head and neck, ovary, skin and blood. Other studies are investigating treatments for COVID-19. Learn more at providenceoregon.org/cancer.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements relate to future events or future financial performance, and use words such as may, estimate, could, expect and others. They are based on managements current expectations and are subject to factors and uncertainties that could cause actual results to differ materially from those described in the statements. These statements include those regarding the hope that Galectins development program for belapectin will lead to the first therapy for the treatment of fatty liver disease with cirrhosis and those regarding the hope that our lead compounds will be successful in cancer immunotherapy and in other therapeutic indications. Factors that could cause actual performance to differ materially from those discussed in the forward-looking statements include, among others, that trial endpoints required by the FDA may not be achieved; Galectin may not be successful in developing effective treatments and/or obtaining the requisite approvals for the use of belapectin or any of its other drugs in development; the Company may not be successful in scaling up manufacturing and meeting requirements related to chemistry, manufacturing and control matters; the Companys current NAVIGATE clinical trial and any future clinical studies as modified to meet the requirements of the FDA may not produce positive results in a timely fashion, if at all, and could require larger and longer trials, which would be time consuming and costly; plans regarding development, approval and marketing of any of Galectins drugs are subject to change at any time based on the changing needs of the Company as determined by management and regulatory agencies; regardless of the results of any of its development programs, Galectin may be unsuccessful in developing partnerships with other companies or raising additional capital that would allow it to further develop and/or fund any studies or trials. Galectin has incurred operating losses since inception, and its ability to successfully develop and market drugs may be impacted by its ability to manage costs and finance continuing operations. Global factors such as COVID-19 may limit access to NASH patient populations around the globe and slow trial enrollment and prolong the duration of the trial and significantly impact associated costs as well as impact other trial related activities including, amongst others, manufacturing and regulatory reviews. For a discussion of additional factors impacting Galectins business, see the Companys Annual Report on Form 10-K for the year ended December 31, 2020, and subsequent filings with the SEC. You should not place undue reliance on forward-looking statements. Although subsequent events may cause its views to change, management disclaims any obligation to update forward-looking statements.

Company Contact:Jack Callicutt, Chief Financial Officer(678) 620-3186ir@galectintherapeutics.com

Galectin Therapeutics and its associated logo is a registered trademark of Galectin Therapeutics Inc. Belapectin is the USAN assigned name for Galectin Therapeutics galectin-3 inhibitor GR-MD-02.

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Journal for ImmunoTherapy of Cancer Publishes Phase 1 Clinical Research Showing Belapectin, Galectin Therapeutics' Galectin-3 Inhibitor, Enhances...

INOVIO’s COVID-19 Vaccine Candidate, INO-4800, Provides Broad Cross-reactive Immune Responses In Humans Against Variants of Concern – BioSpace

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PLYMOUTH MEETING, Pa., April 15, 2021 /PRNewswire/ --Inovio (NASDAQ:INO), a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases, and cancer, today announced the results of a study focusing on the human immune responses induced by INOVIO's DNA vaccine candidate for COVID-19, INO-4800, against variants of concern. The results showed that INO-4800 induced a robust T cell response against all spike protein variants tested, which the company believes will be key in providing protection against SARS-CoV-2 variants, in addition to providing similar levels of neutralizing activity against both the UK and Brazilian variants as those against the original strain. The study, entitled "INO-4800 DNA Vaccine Induces Neutralizing Antibodies and T cell Activity Against Global SARS-CoV-2 Variants," has been submitted for peer review and can be viewed at https://www.biorxiv.org/content/10.1101/2021.04.14.439719v1.

Dr. J. Joseph Kim, INOVIO's President & CEO, said, "These results are consistent with our expectation that INO-4800, which was found to be well-tolerated and able to produce a balanced immune response in our Phase 1 trial, is able to generate both neutralizing antibodies and robust T cell responses both of which will be essential to protect against the emerging variants of concern."

Regarding INO-4800 development Dr. Kim said, "We will report Phase 2 results in 2Q, and, with FDA concurrence, expect to move into Phase 3 in the second quarter of this year. Our COVID vaccine's projected safety profile, its stability at room temperature for more than a year, and likely ability to safely boost numerous times positions INOVIO's COVID-19 vaccine as an important factor in addressing the virus in its pandemic and endemic states."

In the study, clinical samples were collected at varying timepoints post-immunization from subjects in INOVIO's Phase 1 US-based INO-4800 clinical trial. Antibodies capable of neutralizing activity were measured against the spike protein variants tested, including B.1.1.7 (UK variant), B.1.351 (South African variant) and P.1. (Brazilian variant). The study showed the T cell responses induced by INO-4800 vaccination were fully maintained against the UK, South African and Brazilian variants when compared to the T cell responses to the original Wuhan strain. The neutralization levels of INO-4800 against South Africa and UK variants were reduced to the levels similar to the previous reports of mRNA or viral vector vaccines. Furthermore, despite recent reports showing a reduction in neutralizing activity against the Brazilian variant by the mRNA or viral vector vaccines, INO-4800 generated robust neutralizing antibodies at levels against Brazilian variant which were comparable to those against the Wuhan strain. Taken together with the data showing the maintenance of T cell activity, the results reported in this study provide a comprehensive overview of cross-reactive cellular and humoral immune responses against SARS-CoV-2 variants for INO-4800 vaccinated individuals that may be important for protection against variant strains of SARS-CoV-2.

Collectively, the studies showcase how the combination of robust T cell responses in conjunction with neutralizing antibody responses provide an important defense against both emerging and potential future viral variants, exhibiting broad protection against the virus and less impacted by the mutational changes a virus may undergo.

About INO-4800

INO-4800 is INOVIO's DNA vaccine candidate against SARS-CoV-2, the novel coronavirus that causes COVID-19. Composed of an optimized DNA plasmid, INO-4800 is delivered directly into cells in the body via a proprietary smart device to produce a robust and tolerable immune response. INO-4800 is the only nucleic-acid based vaccine that is stable at room temperature for more than a year, at 37o C for more than a month, has a five-year projected shelf life at normal refrigeration temperature and does not need to be frozen during transport or storage all of which are important considerations when preparing for mass immunizations.

About INOVIO's DNA Medicines Platform

INOVIO has 15 DNA medicine clinical programs currently in development focused on infectious diseases, cancer and HPV-associated diseases. DNA medicines are composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

INOVIO's DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using INOVIO's proprietary hand-held smart device called CELLECTRA. The CELLECTRA uses a brief electrical pulse to reversibly open small pores in the cell to allow the plasmids to enter, overcoming a key limitation of other DNA and nucleic acid approaches, such as mRNA. Once inside the cell, the DNA plasmids enable the cell to produce the targeted antigen, which is processed naturally in the cell and triggers the desired T cell and antibody-mediated immune responses. INOVIO's DNA medicines do not interfere with or change in any way an individual's own DNA. The advantages of INOVIO's DNA medicine platform are how fast DNA medicines can be designed and manufactured; the stability of the products, which do not require cold storage or transport; and the robust immune response, safety profile, and tolerability that have been observed in clinical trials.

With more than 3,000 patients receiving INOVIO investigational DNA medicines in more than 7,000 applications across a range of clinical trials, INOVIO has a strong track record of rapidly generating DNA medicine candidates with potential to meet urgent global health needs.

About INOVIO

INOVIO is a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases, cancer, and diseases associated with HPV. INOVIO is the first and only company to have clinically demonstrated that a DNA medicine can be delivered directly into cells in the body via a proprietary smart device to produce a robust and tolerable immune response. Specifically, INOVIO's lead candidate VGX-3100 met primary and secondary endpoints for all evaluable subjects in REVEAL 1, in the first of two Phase 3 trials for precancerous cervical dysplasia, demonstrating ability to destroy and clear both high-grade cervical lesions and the underlying high-risk HPV 16 and 18. INOVIO is also evaluating INO-4800, a DNA vaccine candidate against COVID-19, in a Phase 2 clinical trial in the U.S., as well as Phase 2 trials in China and South Korea. Partners and collaborators include Advaccine, ApolloBio Corporation, AstraZeneca, The Bill & Melinda Gates Foundation, Coalition for Epidemic Preparedness Innovations (CEPI), Defense Advanced Research Projects Agency (DARPA)/Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND)/Department of Defense (DOD), HIV Vaccines Trial Network, International Vaccine Institute (IVI), Kaneka Eurogentec, Medical CBRN Defense Consortium (MCDC), National Cancer Institute, National Institutes of Health, National Institute of Allergy and Infectious Diseases, Ology Bioservices, the Parker Institute for Cancer Immunotherapy, Plumbline Life Sciences, Regeneron, Richter-Helm BioLogics, Thermo Fisher Scientific, University of Pennsylvania, Walter Reed Army Institute of Research, and The Wistar Institute. For more information, visit http://www.inovio.com.

CONTACTS:

Media: Jeff Richardson, 267-440-4211, jrichardson@inovio.comInvestors: Ben Matone, 484-362-0076, ben.matone@inovio.com

This press release contains certain forward-looking statements relating to our business, including our plans to develop DNA medicines and in particular our clinical work relating to INO-4800 and its potential effectiveness against SARS-CoV-2 variants, our expectations regarding our research and development programs, including the planned initiation and conduct of preclinical studies and clinical trials and the availability and timing of data from those studies and trials, and our ability to successfully manufacture and produce large quantities of our product candidates if they receive regulatory approval. Actual events or results may differ from the expectations set forth herein as a result of a number of factors, including uncertainties inherent in pre-clinical studies, clinical trials, product development programs and commercialization activities and outcomes, our ability to secure sufficient manufacturing capacity to mass produce our product candidates, the availability of funding to support continuing research and studies in an effort to prove safety and efficacy of electroporation technology as a delivery mechanism or develop viable DNA medicines, our ability to support our pipeline of DNA medicine products, the ability of our collaborators to attain development and commercial milestones for products we license and product sales that will enable us to receive future payments and royalties, the adequacy of our capital resources, the availability or potential availability of alternative therapies or treatments for the conditions targeted by us or our collaborators, including alternatives that may be more efficacious or cost effective than any therapy or treatment that we and our collaborators hope to develop, issues involving product liability, issues involving patents and whether they or licenses to them will provide us with meaningful protection from others using the covered technologies, whether such proprietary rights are enforceable or defensible or infringe or allegedly infringe on rights of others or can withstand claims of invalidity and whether we can finance or devote other significant resources that may be necessary to prosecute, protect or defend them, the level of corporate expenditures, assessments of our technology by potential corporate or other partners or collaborators, capital market conditions, the impact of government healthcare proposals and other factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2020, and other filings we make from time to time with the Securities and Exchange Commission. There can be no assurance that any product candidate in our pipeline will be successfully developed, manufactured or commercialized, that final results of clinical trials will be supportive of regulatory approvals required to market products, or that any of the forward-looking information provided herein will be proven accurate. Forward-looking statements speak only as of the date of this release, and we undertake no obligation to update or revise these statements, except as may be required by law.

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SOURCE INOVIO Pharmaceuticals, Inc.

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