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Clinical Trials Are Moving Out of the Lab and Into Peoples Homes – The New York Times

Posted: February 25, 2021 at 6:45 am


When the pandemic hit last year, clinical trials took a hit. Universities closed, and hospitals turned their attention to battling the new disease. Many studies that required repeated, in-person visits with volunteers were delayed or scrapped.

But some scientists found creative ways to continue their research even when face-to-face interaction was inherently risky. They mailed medications, performed exams over video chat and asked patients to monitor their own vitals at home.

Many scientists say this shift toward virtual studies is long overdue. If these practices persist, they could make clinical trials cheaper, more efficient and more equitable offering state-of-the-art research opportunities to people who otherwise wouldnt have the time or resources to take advantage of them.

Weve discovered that we can do things differently, and I dont think well go back to life as we used to know it, said Dr. Mustafa Khasraw, a medical oncologist and clinical trial specialist at Duke University.

According to one analysis, nearly 6,000 trials registered on ClinicalTrials.gov were stopped between Jan. 1 and May 31, roughly twice as many compared with non-pandemic times.

At Johns Hopkins University, for instance, researchers delayed their investigation into how adults aged 65 to 80 metabolized tenofovir, a drug used to prevent and treat H.I.V.

The idea of recruiting older people who we know are particularly vulnerable recruiting them to answer a fundamental question that is not going to immediately change care or impact their health just seemed like not what we should be doing, said Dr. Namandje Bumpus, the pharmacologist leading the study, which remains on hold.

In Flint, Mich., researchers had to stop enrolling emergency-room patients for a hypertension trial. Other volunteers quit the study or became difficult to reach.

Their phone service has dropped or they have very different schedules or theyre harder to reach because theyre caring for someone, said Dr. Lesli Skolarus, a stroke neurologist at the University of Michigan who is leading the study.

Dr. Skolarus and her colleagues kept the trial going, albeit with some modifications. Most notably, they scrapped their in-person follow-up visits, instead asking participants to use take-home blood pressure cuffs and to send photos of the readings via text message.

Other research teams made similar adjustments. Neurologists at Massachusetts General Hospital in Boston revamped a pilot study of methylphenidate, the active ingredient in Ritalin, in seniors with mild dementia or cognitive impairment. Instead of going to the hospital every two weeks, study participants are now receiving their medication by mail, taking cognitive assessments over video conference, playing brain games on their computers, and completing daily surveys at home.

Essentially, this is now a totally virtual trial, said Dr. Steven Arnold, the neurologist leading the trial.

Even when scientists cant eliminate in-person visits, theyre finding ways to reduce them. When Lorraine Wilner, a 78-year-old retiree with metastatic breast cancer, first began a clinical trial at Duke University last summer, she had to make the three-hour drive to the Durham, N.C., campus every four weeks, for blood work and occasional other tests. She said she would always leave with a full gas tank, so I dont have to stop at a gas station or touch things or go into places where half the people dont have a mask on, she said.

But she can now have her blood drawn at a lab near her home in Lancaster, S.C. Researchers then review the results with her over a video call. She still has to drive up to Duke for periodic scans, but the reduced traveling has been a great relief. It makes it a lot more convenient, she said.

Remote trials are likely to persist in a post-pandemic era, researchers say. Cutting back on in-person visits could make recruiting patients easier and reduce dropout rates, leading to quicker, cheaper clinical trials, said Dr. Ray Dorsey, a neurologist at the University of Rochester who conducted remote research for years.

In fact, he noted, enrollment in one of his current virtual studies, which is tracking people with a genetic predisposition to Parkinsons, actually surged last spring. While most clinical studies were paused or delayed, ours accelerated in the midst of the pandemic, he said.

The shift to virtual trials could also help diversify clinical research, encouraging more low-income and rural patients to enroll, said Dr. Hala Borno, an oncologist at the University of California, San Francisco. The pandemic, she said, does really allow us to step back and reflect on the burdens that weve been placing on patients for a really long time.

Virtual trials are not a panacea. Researchers will have to ensure that they can thoroughly monitor volunteers health without in-person visits, and be mindful of the fact that not all patients have access to, or are comfortable with, technology.

And in some cases, scientists still need to demonstrate that remote testing is reliable. While Dr. Arnold is optimistic that in-home cognitive tests could provide a better window into his patients everyday functioning, he noted that homes are uncontrolled environments. Maybe theres a cat crawling on them or grandchildren in the next room, he said.

There is also the unpredictable nature of human behavior. Dr. Brennan Spiegel, a gastroenterologist and the director of health services research at the Cedars-Sinai Health System, frequently uses Fitbits to monitor trial subjects remotely. But a participant once put the device on a dog. Several others sent their Fitbits through the wash. You get a lot of steps all of a sudden thousands and thousands of steps, he said.

And some treatments simply may not work as well at a distance. Last January, Clay Coleman Jr., a 61-year-old Chicago resident, enrolled in a clinical trial to treat his peripheral artery disease, which caused intense pain whenever he tried to walk. It was very hard, said Mr. Coleman, who doesnt drive. My legs are very important to me because thats how I get around.

He hoped that the trial which involved taking a blood pressure medication and participating in a supervised exercise program could get him back into walking shape. Three times a week, he traveled to a local gym for a structured treadmill workout with a coach. I had been there maybe six weeks or so before this virus thing came around, he said.

Suddenly, the gym was out. Instead, Mr. Colemans coach called him regularly on the phone and encouraged him to keep moving.

Dr. Mary McDermott, a general internist at Northwestern University who is running the trial, isnt sure how effective this kind of remote coaching will be. We cannot assume that remote interventions are going to be the same, she said. Or that remote measurements are going to replace everything that we have done in person.

Still, the pandemic has demonstrated that there is room for reform. Dr. Deepak Bhatt, a cardiologist at Brigham and Womens Hospital in Boston, is part of a team starting a trial of an injectable blood thinner later this year. After the first, in-person medical visit, appointments will be virtual.

Im quite sure if Covid had not occurred, we would have done things the usual way, he said. Sometimes, he added, it takes a crisis to provoke change.

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Clinical Trials Are Moving Out of the Lab and Into Peoples Homes - The New York Times

EMVision Medical Devices to expand clinical studies of portable brain scanner on stroke patients – Small Caps

Posted: at 6:45 am


Medical imaging technology developerEMVision Medical Devices (ASX: EMV)has elected to expand on an initial clinical study involving its portable brain scanner in stroke patients at Brisbanes Princess Alexandra Hospital.

The company has developed a portable and non-invasive brain scanner to assist with diagnosing and monitoring time-sensitive neurological disorders including stroke and traumatic brain injury.

A pilot trial was carried out last yearusing a prototype of the portable brain scanner on patients with ischaemic or haemorrhagic stroke.

This study was undertaken at Princess Alexandra Hospital and was the first of its kind using EMVisions technology and was designed to improve understanding of stroke on electromagnetic scattering effects in the brain.

Its focus was to produce datasets that enable EMVision to further refine its algorithm and compare against computerised tomography and magnetic resonance imaging.

In the initial trial, 39 patients participated with most over the age of 60. The study revealed the device could classify stroke type with accuracy ranging between 93.3-96%.

EMVision noted the initial clinical trial had been successful and supported by investigators and stroke experts Australia-wide.

As a result, the company has elected to expand the trial to include a further 20 patients.

The rationale behind the enlarged study was to increase the diversity of stroke types and investigate potential in extended monitoring applications by the bedside.

Further patient datasets for our algorithms will allow continued improvements to localisation and classification of strokes, EMVision explained.

EMVision is in discussions with a number of hospital sites for wider increased clinical study.

The potential of our technology continues to see strong support from stroke experts in these hospitals, the company added.

In parallel with the trials, EMVision has been engaging with the FDA regarding securing approvals for the portable brain scanner.

As part of this, the FDA has recommended the De Novo process as the most appropriate regulatory pathway for the device. This pathway has been designed for low-to-moderate risk, first-of-a-kind products.

EMVision is also pursuing an application for the Breakthrough Device Designation program which, if granted, will enable development and assessment of the brain scanner to be fast-tracked.

To further ensure EMVision secures FDA approval for its technology, the company has continued making improvements to deliver the next generation device.

EMVision aims to make the next generation device smaller, more robust and easier to use.

Significant advances have been made in the fundamental design and engineering based on our trial, the company noted.

This has included substantial progress in algorithms based on data derived from the research.

To shore up the companys ongoing research and refinements, key staff appointments have been made. A global leader in medical imaging innovation Prof Stuart Crozier joined the company as chief scientific officer during the six months ending December 2020.

EMVision also stated it had added a number of accomplished medtech experts to its product development team.

According to EMVision, it has conducted extensive market research regarding the potential for its technology in hospitals.

The company concluded there was substantial opportunity to deliver savings to hospitals and healthcare system well beyond the cost of the product over its lifetime therefore boosting its appeal.

Additionally, EMVision is engaging with global medical imaging and device manufacturers, which it says have shown significant interest in the companys technology and its potential.

Our interactions have been with some of the most senior executives and decision-makers at these companies, with our international visibility increasing, the company added.

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EMVision Medical Devices to expand clinical studies of portable brain scanner on stroke patients - Small Caps

Lengthy clinical trials to test vaccines modified for variants won’t be necessary, FDA says – Livescience.com

Posted: at 6:45 am


If the novel coronavirus mutates such that current COVID-19 vaccines are no longer effective at fighting the virus, researchers will have to modify the vaccines.

But new guidelines issued by the Food and Drug Administration (FDA) indicate that long, extensive clinical trials may not be necessary to test the modified vaccines. Rather, the modified vaccines can be tested in small-scale trials like those conducted to develop the flu vaccine every year, The New York Times reported.

"We know the country is eager to return to a new normal, and the emergence of the virus variants raises new concerns about the performance of these products," Dr. Janet Woodcock, acting FDA commissioner, said in a statement. "By issuing these guidances, we want the American public to know that we are using every tool in our toolbox to fight this pandemic, including pivoting as the virus adapts."

Related: Quick guide: COVID-19 vaccines in use

Current evidence suggests that the Pfizer and Moderna coronavirus vaccines, the only two that are authorized for emergency use in the U.S., remain effective against the circulating variants, according to the statement. (Studies have indicated, however, that they may be less effective against the variant first detected in South Africa, known as the B.1.351 variant, than they are against other variants.)

But if the virus evolves to be "moderately or fully resistant" to the current COVID-19 vaccines, then it may be necessary to modify them, according to the statement. The mRNA technology that was used to make both vaccines allows for quick modifications; the companies have said that they could create modified versions in a period of six weeks, according to the Times.

Because the companies would be modifying the same vaccines only slightly in order to make them better against the new variants, extensive clinical trials involving testing the vaccine against a placebo in thousands of people may not be needed.

Rather, the new guidelines suggest that testing the modified vaccine on a small group of volunteers may be enough. After being vaccinated, these volunteers would give blood samples to be tested in the lab, the Times reported.

The immune response generated in response to the modified vaccine will be compared to the immune response generated by the authorized vaccine, according to the statement. The guidelines encourage testing of the modified vaccine in an animal model and in both people who have previously been vaccinated with a coronavirus vaccine and those who have not been vaccinated at all.

Also, researchers will need to conduct safety assessments of the modified vaccines, such as assessing them for serious or adverse reactions after vaccination, according to the guidelines.

But further discussions will be needed for a final decision on whether such modified COVID-19 vaccines can be authorized in the future without clinical studies, according to the statement.

The new guidelines, issued for companies that make COVID-19 vaccines, tests and therapeutics, were added to the end of a longer guidance document for companies seeking emergency approval for coronavirus vaccines.

Originally published on Live Science.

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Lengthy clinical trials to test vaccines modified for variants won't be necessary, FDA says - Livescience.com

Greenwich LifeSciences Announces Acceptance of Two Abstracts at Upcoming Major Cancer Conference – Business Wire

Posted: at 6:45 am


STAFFORD, Texas--(BUSINESS WIRE)--Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the Company), a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery, today announced that two abstracts have been accepted for presentation at the upcoming American Association for Cancer Research (AACR) Annual Meeting 2021, including two corresponding posters. The AACR 2021 conference will be held in a virtual format from April 10-15, 2021. The AACR plans to publish the titles on March 10, 2021 at 4:30 pm EST, the abstracts on April 9, 2021 at 12:01 am EST, and the posters on April 10, 2021.

Snehal Patel, CEO of Greenwich LifeSciences, commented, We are pleased that both of our abstracts were accepted by the AACR. On December 9, 2020, we presented the Phase IIb clinical trial Kaplan Meier analysis of disease free survival for HER2/neu 3+ patients treated with GP2 immunotherapy, which showed 100% survival over 5 years of follow-up (0% breast cancer recurrences, p = 0.0338) if the patients received their primary GP2 treatments following surgery and Herceptin treatment. In the first abstract and poster, we are excited to present the final 5 year analysis of the immune response over time for all patients in the Phase IIb clinical trial.

Patel added, As the immune response is the primary mechanism of action, this final analysis assessing GP2s effectiveness in creating peak immunity is important in further validating the clinical outcome, where we observed the 0% recurrence rate, and will provide more insight into the design of the Phase III clinical trial, which is our second abstract and poster. Immune response data is critical in developing dosing and booster treatment strategies designed to achieve and sustain peak immunity, providing protection against metastatic breast cancer recurrence for as long as possible.

The first abstract and poster will present the final 5 year immune response data across all patient populations from the completed prospective, randomized, placebo-controlled, single-blinded, multicenter, Phase IIb clinical trial. The presentation will include analysis of the various methods used to measure immune responses for both HER2/neu 3+ and HER2/neu 1-2+ patient populations, including comparison of peak immune response versus baseline immune response at multiple time points.

The second abstract and poster, jointly sponsored with Baylor College of Medicine, will present the design of the planned Phase III clinical trial. The clinical trial is designed as a single registration trial that will include an interim analysis seeking conditional marketing approval from the FDA upon the interim analysis data read out followed by the submission of a Biologics Licensing Application (BLA). Additional features of the clinical trial design will be presented to breast cancer key opinion leaders as we recruit clinicians and clinical sites for participation in the Phase III clinical trial.

About the AACR Annual Meeting 2021

The AACR is the first and largest cancer research organization dedicated to accelerating the conquest of cancer and has more than 48,000 members residing in 127 countries and territories. The AACR Annual Meeting program covers the latest discoveries across the spectrum of cancer research from population science and prevention; to cancer biology, translational, and clinical studies; to survivorship and advocacy and highlights the work of the best minds in research and medicine from institutions all over the world.

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. HER2/neu (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

About Greenwich LifeSciences, Inc.

Greenwich LifeSciences is a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery. GP2 is a 9 amino acid transmembrane peptide of the HER2/neu protein. In a randomized, single-blinded, placebo-controlled, multi-center (16 sites led by MD Anderson Cancer Center) Phase IIb clinical trial, no recurrences were observed in the HER2/neu 3+ adjuvant setting after median 5 years of follow-up, if the patient received the 6 primary intradermal injections over the first 6 months (p = 0.0338). Of the 138 patients that have been treated with GP2 to date over 4 clinical trials, GP2 treatment was well tolerated and no serious adverse events were observed related to GP2 immunotherapy. Greenwich LifeSciences is planning to commence a Phase III clinical trial using a similar treatment regime as the Phase IIb clinical trial. For more information on Greenwich LifeSciences, please visit the Companys website: http://www.greenwichlifesciences.com

Forward-Looking Statement Disclaimer

Statements in this press release contain forward-looking statements that are subject to substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as anticipate, believe, contemplate, could, estimate, expect, intend, seek, may, might, plan, potential, predict, project, target, aim, should, "will, would, or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on Greenwich LifeSciences Inc.s current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict, including statements regarding the intended use of net proceeds from the public offering; consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. These and other risks and uncertainties are described more fully in the section titled Risk Factors in the final prospectus related to the public offering filed with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Greenwich LifeSciences, Inc. undertakes no duty to update such information except as required under applicable law.

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Greenwich LifeSciences Announces Acceptance of Two Abstracts at Upcoming Major Cancer Conference - Business Wire

Selecta Biosciences Announces Science Advances Publication Highlighting Potential Potency and Durability Benefits of ImmTOR in Gene Therapy – BioSpace

Posted: at 6:45 am


WATERTOWN, Mass., Feb. 24, 2021 (GLOBE NEWSWIRE) -- Selecta Biosciences Inc. (NASDAQ: SELB, Selecta), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, today announced the peer-reviewed publication of a study investigating the effects of the co-administration of ImmTOR nanoparticles to adeno-associated viral (AAV) vectors on transgene expression in mice. The data, published in a Science Advances paper titled Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles, demonstrate that the addition of ImmTOR nanoparticles to AAV vectors has the potential enhance the efficacy, safety and durability of gene therapies by mediating more efficient transgene expression at the first dose and by enabling vector redosing by preventing the formation of capsid-specific antibodies. These findings underscore the promise of Selectas ImmTOR platform to address current limitations, notably immunogenicity, safety and durability, of gene therapy.

The results in this publication continue to expand our knowledge of and validate the benefits of co-administering ImmTOR with AAV gene therapies, said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. Along with our previous research, this study demonstrates the multipronged mechanism of ImmTOR that makes it a particularly attractive candidate in clinical indications where repeat vector dosing may be necessary, most notably in children. Importantly, this study also shows the enhanced transgene expression induced by admixing ImmTOR and AAV vectors, which may enable lower doses of AAV to be used. We look forward to continuing to investigate the benefits of adding ImmTOR to gene therapy in clinical studies this year.

In the study, researchers evaluated the effects of ImmTOR on repeat administration of the same AAV vector expressing secreted embryonic alkaline phosphatase (SEAP), a widely used reporter gene transgene, in mice. Co-administration of ImmTOR and AAV8-SEAP showed a beneficial effect on transgene expression after the first dose and reached levels approximately two-to-three-fold higher than that observed in mice treated with the AAV vector alone. The first dose benefit was immediate, dose dependent and not mouse strain or capsid specific. The rapid and enhanced transgene expression may enable therapeutic benefit at lower doses of AAV and faster onset of transgene- directed therapeutic effects.

The study also investigated the extent to which the addition of ImmTOR nanoparticles to AAV vectors, known as admixing, has on expression and its potential mechanism. Admixing of ImmTOR and AAV showed even higher levels of vector genome copies in the liver and mRNA and protein expression of the transgene SEAP, compared to sequential administration of AAV-SEAP and ImmTOR or dosing with AAV-SEAP alone. The cumulative benefit of enhancing first dose transgene expression and enabling repeat dosing can provide up to a four-fold increase in transgene expression compared to gene therapy with AAV vector alone. Admixing ImmTOR and AAV prior to injection was important for enhanced transgene expression after the first dose, but not required for inhibition of AAV-specific antibodies or for repeated administration of AAV vectors. Despite this finding in mice, a recent study performed in non-human primates (NHP) has demonstrated that admixing of ImmTOR with AAV was not necessary to observe the increased levels and durability of transgene expression at the first dose.

AboutSelecta Biosciences, Inc.Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the bodys natural self-tolerance in autoimmune diseases. The companys first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visitwww.selectabio.com.

Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects ofSelecta Biosciences, Inc.(the company), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the timing of any clinical trials in the field of gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy, the ability of the Company and AskBio to develop gene therapy products using ImmTOR and AskBios technology, the novelty of treatment paradigms that the Company is able to develop, whether the observations made in non-human primate study subjects will translate to studies performed with human beings, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the companys intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human primates, the unproven approach of the companys ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with theSecurities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

For Investors:Bruce MackleLifeSci Advisors, LLC+1-929-469-3859bmackle@lifesciadvisors.com

For Media: Meredith Sosulski, Ph.D.LifeSci Communications, LLC+1-929-469-3851msosulski@lifescicomms.com

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Selecta Biosciences Announces Science Advances Publication Highlighting Potential Potency and Durability Benefits of ImmTOR in Gene Therapy - BioSpace

Somru BioScience and Veeda Clinical Research Announce Joint Venture Global Biotherapeutic Contract Research Organization (CRO) in India – Business…

Posted: at 6:45 am


CHARLOTTETOWN, Prince Edward Island & AHMEDABAD, India--(BUSINESS WIRE)--Ingenuity BioSciences Pvt. Ltd Veeda Clinical Research, one of Indias leading and largest independent CRO companies, and Somru BioScience Inc., a leading Canadian-based biotechnology company based in Charlottetown, Prince Edward Island, are proud to announce the establishment of an innovation-centric bioanalytical laboratory in Ahmedabad, India.

Veeda and Somru announced the launch of their premier global centre of excellence lab under the flagship of Ingenuity BioSciences Pvt. Ltd. The Board of Directors of both companies have made a strategic alliance to expand their specialized service offerings, complementing their well-established portfolio in clinical services and bioassay kit development. This collaborative joint venture aims to capture the growing market needs of the global biosimilars market projected to grow from USD 28 billion in 2020 to USD 103 billion in 2026, with a CAGR of 24.2%. Somru BioScience and Veeda Clinical Research will have an equal partnership in the new joint venture.

I am delighted with the fruition of our partnership with Somru BioScience which combines the strengths of our organizations to reinforce our vision of becoming the preferred research partners for innovative biopharmaceutical companies globally. The joint venture will bring Somrus global standard, integrated bioassay capabilities to service the rapidly growing biosimilars industry in the region and, combined with Veedas strong clinical research credentials, will provide complete clinical development solutions to our clients," said Ajay Tandon, Managing Director, Veeda Clinical Research.

Ingenuity BioSciences will be established as a premier and agile bioanalytical laboratory. Somru BioSciences proprietary automation platform and cloud based Aegryris bioanalytical and productivity software suite will be a potential game-changer in the innovation-based drug discovery industry.

This relationship will allow us to leverage our extensive experience as a biosimilar solution provider in this fast-emerging Asian market, said Mohammed Moin, CEO of Somru BioScience Inc. We are very impressed with Veedas track record as an excellent clinical service provider in India and are excited to be working with them.

Ingenuity BioSciences aims to offer end-to-end and modular technical services, encompassing both pre-clinical and clinical domains. The Ingenuity BioSciences scientific team is comprised of talented scientists with in-depth and extensive knowledge of bioassay method development and sample analysis, clinical trial management, and global regulatory requirements. Business development and technical teams are enthusiastic and excited to collaborate and serve Indian and International clients across pan-Asia and European markets. Ingenuity BioSciences website will be soon available and provide detailed information on service offerings.

Ingenuity BioSciences will be led by Mohammed Moin, CEO of Somru BioScience and Dr. Ravi Krovidi, CEO of Ingenuity BioSciences Pvt Ltd. The location of Ingenuity BioSciences in Ahmedabad, India, gives its clients a unique advantage of sharing the years of trust, regulatory expertise and reputation established by the Veeda Clinical Research services ecosystem.

We look forward to closely working with biosimilar and biotherapeutics companies. Our vision is to become a preferred CRO clinical partner in product development's success journey from early inception to product launch. The Biosimilarization wave is gaining significant traction, with several important monoclonal antibodies going off patent. We are very excited to partner with Somru team, said Dr. Ravi Krovidi, CEO of Ingenuity Biosciences Pvt Ltd.

About Somru BioScience Inc.

Somru Bioscience Inc. is an innovative biotechnology company based out of Prince Edward Island, Canada. Somru has developed a portfolio of reagents, ELISA kits, innovative biosimilar comparability tools and a cloud based Aegryris software suite to enable companies to significantly reduce the cost and timeline for biosimilar development. Somru technologies reduce early development risk by identifying clinically meaningful differences prior to running expensive clinical trials. The company has customers throughout North and South America, Europe, Australia, and Asia.

somrubioscience.com

About Veeda Clinical Research

Veeda is a leading independent Clinical Research Organization in India conducting a diverse range of clinical studies including bioavailability and bioequivalence (BA/BE) studies, pharmacokinetics, pharmacodynamics, early to late phase clinical development and clinical endpoint studies for generic molecules, new chemical entities (NCE), and biopharmaceuticals. Veeda strives for excellence in quality and has been the partner of choice for many global pharmaceutical companies over the last 16 years. Veeda is reputed for its best-in-class scientific expertise, quality, and ethics. Veeda has an exemplary regulatory record of successfully completing USFDA, ANVISA, WHO, MHRA, AGES, ANSM, MCC, DCGI and NPRA audits to date.

veedacr.com

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Somru BioScience and Veeda Clinical Research Announce Joint Venture Global Biotherapeutic Contract Research Organization (CRO) in India - Business...

Diffusion Pharmaceuticals Completes Phase 1b Study of TSC in Hospitalized COVID-19 PatientsNo Dose Limiting Toxicities or Serious Adverse Events…

Posted: February 17, 2021 at 12:51 am


CHARLOTTESVILLE, Va., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Diffusion PharmaceuticalsInc. (NASDAQ: DFFN)(Diffusion or the Company), an innovative biopharmaceutical company developing novel therapies to deliver oxygen to areas of the body where it is needed most, today announced completion and topline data from the open-label, Phase 1b clinical trial of its novel, diffusion-enhancing therapeutic, trans sodium crocetinate (TSC), in hospitalized COVID-19 patients with confirmed hypoxemia, the most common cause of tissue hypoxia.

The primary objective of the trial was to evaluate the safety and tolerability of TSC administered on a more frequent dosing regimen not previously tested in a clinical trial setting. Secondary endpoints included pharmacokinetic measurement of TSC levels after dosing, relative improvements in blood oxygen levels, and certain other clinical parameters related to COVID-19.

In this trial, patients were divided into four sequential cohorts of six patients, with each patient in a dose cohort receiving the same intravenous doses of 0.25 mg/kg, 0.5 mg/kg, 1.0 mg/kg, or 1.5 mg/kg, depending on the patients cohort. All patients in the study were administered intravenous doses of TSC every six hours for a minimum of five days and up to 15 days. On Friday, February 12, 2021, the external safety monitoring committee, established as part of the trial protocol, met to review safety data from the final, 1.5 mg/kg dose, cohort and determined that no dose-limiting toxicities or serious adverse events were observed. This result is consistent with the committees determinations following their analyses of the safety data from each of the other dose cohorts in the trial. Evaluation of secondary endpoint data is ongoing and will be available early in the second quarter of 2021.

We are grateful to the patients and the dedicated healthcare providers who volunteered to participate in this critically important study, said Chris Galloway, M.D., Chief Medical Officer. We believe TSCs diffusion-enhancing mechanism of action has the potential to provide benefits in numerous medical conditions complicated by hypoxia, including COVID-19. The safety, tolerability, and repeat escalating-dose pharmacokinetic data obtained from this study will support our broader TSC development program.

As previously announced, the company plans two additional clinical studies designed to evaluate the efficacy of TSC on both ends of oxygens complete journey through the body, from improving uptake in the lungs to enhancing delivery from the microcirculation into tissue.

The first study (the TCOM Study) will measure the effects of TSC on peripheral tissue oxygen delivery using a device called a transcutaneous oximeter, or TCOM. This will be a randomized, blinded and placebo-controlled trial in healthy volunteers to determine TSCs ability to enhance oxygen delivery versus placebo. Diffusion expects to initiate the TCOM Study by the end of the first quarter 2021 and complete it in the second quarter of 2021. Topline data should be available within one to two months following study completion.

The second study (the DLCO Study) will measure the effects of TSC on the ability of the lungs to transfer gas from inspired air to the bloodstream, using carbon monoxide as a surrogate for oxygen. The DLCO Study will be conducted in patients with previously diagnosed interstitial lung disease. Diffusion plans to commence the DLCO Study in the second quarter of 2021 and complete it in the third quarter of 2021; topline results should be available within one to two months of study completion and will inform the companys ongoing TSC development plan.

The Company anticipates that these studies will provide important dose-response data that will guide its product and commercial development strategies, which will focus on treatment of conditions related to the hypoxia continuum. Diffusion intends to announce certain additional information regarding these strategies later in the first quarter of 2021.

About TSC

TSC was designed to enhance the level of organization among water molecules by increasing the amount of hydrogen bonding and is being developed to enhance the diffusion of oxygen to tissues with low oxygen levels, also known as hypoxia, a serious complication of many of medicines most intractable and difficult-to-treat conditions. In clinical studies, when dosed up to four times daily, TSC has been demonstrated safe and tolerable in more than 180 patients included in trials across a variety of indications including patients with glioblastoma multiforme brain cancer (GBM), peripheral artery disease with intermittent claudication, stroke, and COVID-19. In pre-clinical data, TSC has been observed to affect oxygen diffusion to hypoxic tissues and provide a functional benefit in animal models of GBM, acute respiratory distress syndrome/acute lung injury, hemorrhagic shock, and ischemic stroke.

About Diffusion Pharmaceuticals Inc.Diffusion Pharmaceuticals Inc. is an innovative biopharmaceutical company developing novel therapies to deliver oxygen to areas of the body where it is needed most. Diffusions lead product candidate, TSC, is being developed to enhance the diffusion of oxygen to tissues with low oxygen levels, also known as hypoxia, a serious complication of many of medicines most intractable and difficult-to-treat conditions. In addition to TSC, Diffusions product candidate DFN-529, a novel PI3K/Akt/mTOR pathway inhibitor, is in early-stage development. For more information, please visit us atwww.diffusionpharma.com.

Forward-Looking StatementsThis press release includes express and implied forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding (i) the anticipated timing of further announcements regarding the Companys Phase 1b clinical trial and (ii) the Companys future development plans for TSC and the timing of certain milestones related thereto. The Company may, in some cases, use terms such as believes, estimates, anticipates, expects, plans, intends, may, could, might, will, should, approximately, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Although the Company believes that it has a reasonable basis for each forward-looking statement contained herein, forward-looking statements by their nature involve risks and uncertainties, known and unknown, many of which are beyond the Companys control, and as a result the Companys actual results could differ materially from those expressed or implied in any forward-looking statement. Particular risks and uncertainties include, among other things, those related to: the Companys ability to design, initiate, execute, and complete its ongoing and planned studies evaluating TSC; the Companys ability to obtain additional financing; the Companys ability to develop, obtain regulatory approval for, and commercialize TSC or any other product candidate; the ongoing COVID-19 pandemic; general economic, political, business, industry, and market conditions; and the other factors discussed under the heading Risk Factors in the Companys most recent Annual Report on Form 10-K and its other filings with the U.S. Securities and Exchange Commission. Any forward-looking statements in this press release speak only as of the date hereof (or such earlier date as may be identified) and, except as required by applicable law, rule, or regulation, the Company undertakes no obligation to update any such statements after the date hereof.

Contacts

Investors:Tiberend Strategic Advisors, Inc.Maureen McEnroe, CFA/Miriam Weber Miller(212)375-2664/ (212) 375-2694mmcenroe@tiberend.com/mmiller@tiberend.com

Media:Jeffrey FreedmanRooneyPartners(646) 432-0191jfreedman@rooneyco.com

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Diffusion Pharmaceuticals Completes Phase 1b Study of TSC in Hospitalized COVID-19 PatientsNo Dose Limiting Toxicities or Serious Adverse Events...

A Meta-Analysis of Randomized Intervention Trials Confirms: MEMORY HEALTH Patented Carotenoid Formula Improves Cognitive Performance and Protects…

Posted: at 12:51 am


BIRMINGHAM, Mich., Feb. 16, 2021 /PRNewswire/ --The analysis, by Davinelli et al., represents the first systematic review combining multiple randomized, double-blind, placebo-controlled studies known as the 'gold standard' of clinical studies. This meta-analysis specifically evaluated studies of the effects of carotenoids on outcomes associated with cognitive performance, and included three separate randomized studies of the MEMORY HEALTH carotenoid formula.

The results indicate clearly that carotenoid interventions are associated with better cognitive performance. Thus, supplementing with these carotenoids via MEMORY HEALTHmay help to reduce the risk of cognitive impairment and dementia.

Accumulating evidence has indicated that many of the underlying mechanisms associated with dementia begin a decade or more before the onset of symptoms. Therefore, the identification of specific interventions that may prevent the decline in cognitive function during this preclinical phase is crucial in terms of public health policy.

Non-pharmacological approaches focused on lifestyle factors represent a cost-effective and practical strategy to reducing or slowing age-related cognitive decline. Nutrition is a modifiable lifestyle factors that has been consistently associated with cognition at various levels, including greater adherence to healthy dietary patterns, the intake of specific nutrients, or the consumption of specific dietary antioxidants such as carotenoids.

This is the first cardinal meta-analysis providing evidence on the quantitative measure of the association between carotenoids and cognitive function. "This represents a significant milestone," says Professor James Stringham, PhD. "It demonstrates support akin to global acceptance of the effects."

The meta-analysis identified 9 studies with a total of 4402 nondemented subjects, whose age ranged from 45 to 78 years. Results of the pooled meta-analysis found a significant positive effect of carotenoid intervention on cognitive outcomes. The positive effects of carotenoids on cognitive functions were proven statistically significant.

"MEMORY HEALTHis unique as it is based on the largest, growing independent scientific evidence in the industry," says Edward Shehab, Managing Partner. "No other formula currently on the market can provide such a strong body of scientific studies that reached their primary outcome, (i.e., showed positive results targeted), hence our motto REAL SCIENCE FOR REAL RESULTS," he added.

"We have a patented formula containing the three dietary carotenoids Lutein, Zeaxanthin, and Meso-Zeaxanthin. The meta-analysis indicates that the administration of the carotenoids improves cognitive effects in both older and younger subjects, as well as demented and nondemented subjects. The meta-analysis findings were in agreement with previous narrative reviews indicating that higher dietary intake and blood concentrations of carotenoids are associated with lower risk of age-related cognitive decline, cognitive impairment, and dementia and Alzheimer's disease.

MEMORY HEALTHis the only brain heath supplement with the exclusive patent on all three dietary carotenoids Lutein, Zeaxanthin, and Meso-Zeaxanthin [in combination with Omega-3s DHA and EPA].

On December 17, 2020, MEMORY HEALTHbecame the first brain health supplement to receive a United States patent for the prevention and treatment of neurodegenerative disease [a method for the prevention and treatment of dementia in a human subject]. A similar patent was awarded to MEMORY HEALTHin the United Kingdom.

The MEMORY HEALTH formula was scientifically developed and tested in double-blind, placebo-controlled clinical trials on both demented and nondemented subjects over an 18-month period. The results of these trials were published in the scientific Journal of Alzheimer's disease and showed statistically significant improvements in cognitive function across both demented and nondemented subjects.

The meta-analysis concludes that these results highlight the potential role of carotenoid supplementation in the protection of mental functions even in subjects without cognitive impairment. This is particularly important because the population is aging, and preservation of cognitive function is crucial for individual autonomy and quality of life, even in non-demented subjects.

Media Contact: Edward Shehab [emailprotected]2482021374

SOURCE Memory Health

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A Meta-Analysis of Randomized Intervention Trials Confirms: MEMORY HEALTH Patented Carotenoid Formula Improves Cognitive Performance and Protects...

Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -…

Posted: at 12:51 am


REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the launch of a Phase 1/2 clinical trial to evaluate JSP191, Jaspers first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen prior to allogeneic transplant for sickle cell disease (SCD). Jasper Therapeutics and the National Heart, Lung, and Blood Institute (NHLBI) have entered into a clinical trial agreement in which NHLBI will serve as the Investigational New Drug (IND) sponsor for this study.

SCD is a lifelong inherited blood disorder that affects hemoglobin, a protein in red blood cells that delivers oxygen to tissues and organs throughout the body. Approximately 300,000 infants are born with SCD annually worldwide, and the number of cases is expected to significantly increase. Currently, hematopoietic stem cell transplantation (HSCT) is the only cure available for SCD.

"This clinical trial agreement with the NHLBI expands the development of JSP191 for transplant conditioning and could bring curative transplants to more patients in need," said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. "We look forward to collaborating with the NHLBI and learning more about the potential for JSP191 in patients living with sickle cell disease."

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from the bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals in stem cells leading to cell death. This creates space in the bone marrow for engraftment of donor or gene-corrected transplanted stem cells.

Preclinical studies have shown that JSP191, as a single agent, safely depletes normal and diseased hematopoietic stem cells, including in animal models of severe combined immunodeficiency (SCID), myelodysplastic syndromes (MDS), and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate Jasper Therapeutics-sponsored clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID. Blood stem cell transplantation offers the only potentially curative therapy for SCID. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

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Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -...

Artios Pharma Announces the Start of First Clinical Study with the Dosing of its ATR Inhibitor, ART0380, to Patients – PRNewswire

Posted: February 11, 2021 at 6:54 am


CAMBRIDGE, England and NEW YORK, Feb. 11, 2021 /PRNewswire/ -- Artios Pharma Limited ("Artios"), a leading DNA Damage Response (DDR) company developing a broad pipeline of precision medicines for the treatment of cancer, today announced the start of a clinical trial of itssmall-molecule ATR inhibitor, ART0380, in patients with advanced or metastatic solid tumors. ART0380 was originally in-licensed from The University of Texas MD Anderson Cancer Center and ShangPharma Innovation in 2019. The molecule was jointly developed as part of a collaboration between ShangPharma and MD Anderson's Therapeutics Discovery Division.

The clinical trial of the ATR (Ataxia telangiectasia and Rad3-related kinase) inhibitor is an open-label, multi-center, Phase I/IIa study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of ART0380 as a monotherapy and in combination with gemcitabine in patients with advanced or metastatic solid cancers. The study will enroll up to 180patients and will be conducted at multiple oncology centers across the USA, UK, and Europe. Full details can be found at http://www.clinicaltrials.govunder the identifier NCT04657068.

Niall Martin, Chief Executive Officer at Artios Pharma, said:"Our vision is to build on the success of PARP inhibition to enable more patients to benefit from DDR targeting medicines. By using insights gained from our longstanding experience in this field, we believe that our approach will address the challenges of resistance to targeted therapy, identify patients with sensitive cancers, and optimize the therapeutic index. We have done extensive preclinical work to characterise and differentiate our lead candidates andwill have two programs, ART0380 and ART4215, a first in class Pol theta inhibitor, in the clinic by end of 2021. Following the recent strategic collaboration with Merck KGaA, Darmstadt, Germany, taking ART0380 into the clinic is another major milestone for Artios in the development of next generation DNA Damage Response treatments for hard-to-treat cancers."

Melissa Johnson, MD, Program Director, Lung Cancer Research, Sarah Cannon Research Institute at Tennessee Oncology, Principal Investigator for the trial, said:"We have a strong heritage in developing novel cancer therapies. We see DDR as an exciting and promising area of research and are excited to collaborate with Artios on this important clinical trial."

ART0380 is a new investigational medicinal product that is a potent and selective inhibitor of ATR. ATR is an important signalling protein in the cellular DNA damage response to replication stress and DNA double-strand breaks that occur as cells multiply.

ART0380 is being developed as an oral anti-cancer agent for the treatment of patients with cancers harbouring defects in DNA repair and in combination with agents including established and novel agents that cause DNA damage and/or suppress a cancer's ability to repair DNA damage.

About Artios Pharma Limited

Artios is a leading DNA Damage Response (DDR) company focused on developing first-in-class treatments for cancer. The Company is led by an experienced scientific and leadership team with proven expertise in DDR drug discovery, including the identification and development of the PARP inhibitor olaparib. It has a unique partnership with Cancer Research UK (CRUK), and collaborations with leading DNA repair researchers worldwide, such as The Institute of Cancer Research (ICR), London, the Netherlands Cancer Institute (NKI) and the Crick Institute, London. Artios is building a pipeline of next-generation DDR programmes to target hard to treat cancers, including Phase I/IIa clinical studies in 2021 for its ATR inhibitor ART0380 in treating DDR defective tumours, and the first-in-class Pol theta inhibitor ART4215 for mono therapy and combination treatments. In December 2020, Artios entered into a collaboration agreement with Merck KGaA, Darmstadt, Germany to identify and develop precision oncology medicines targeting nucleases.Merck KGaA, Darmstadt, Germanyhas the right to opt into exclusive development and commercialization of compounds on up to eight targets and Artios to receive up to US$860 million total milestones per target. It is backed by blue chip investors including: AbbVie Ventures, Andera Partners, Arix Bioscience plc, IP Group plc, Life Science Partners (LSP), M Ventures, Novartis Venture Fund (NVF), Pfizer Ventures and SV Health Investors. Artios is based at the Babraham Research Campus in Cambridge, UK, with an office in New York City, USA.www.artiospharma.com

SOURCE Artios

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Artios Pharma Announces the Start of First Clinical Study with the Dosing of its ATR Inhibitor, ART0380, to Patients - PRNewswire

Protalix BioTherapeutics and Chiesi Global Rare Diseases Present Key Clinical Data of Pegunigalsidase Alfa for the Treatment of Fabry Disease at the…

Posted: at 6:54 am


The BRIDGE clinical trial was a Phase III 12 month open label, single arm switch over study evaluating the safety and efficacy of pegunigalsidase alfa, 1 mg/kg infused every two weeks, in up to 22 Fabry patients previously treated with agalsidase alfa, marketed by Takeda Pharmaceutical Company Limited (formerly Shire Plc) as Replagal, for at least two years and on a stable dose for at least six months. Replagal is not approved in the United States.

The data to be presented by Prof. Linhart showed substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR slope) in both male and female patients who were switched from agalsidase alfa to pegunigalsidasealfa.

Twenty of twenty-two patients completed the 12-month treatment duration, 18 of which opted to roll over to a long-term extension study and continue to be treated with pegunigalsidasealfa.

In the BRIDGE study, the mean annualized eGFR slope of the study participants improved from -5.90 mL/min/1.73m2/year while on agalsidase alfa to -1.19 mL/min/1.73m2/year on PRX-102 in all patients. Male patients improved from -6.36mL/min/1.73m2/year to 1.73mL/min/1.73m2/year and female patients improved from -5.03mL/min/1.73m2/year to 0.21mL/min/1.73m2/year. Following the switch to pegunigalsidasealfa there was a decrease in patients with progressing or fast progressing kidney disease, and the majority of patients achieved a stable status post-switch.

An immunogenicity assessment indicated that four out of 20 patients (20%) developed persistent antidrug antibodies over the course of the study, of which two had neutralizing activity.

Baseline characteristics of the 20 patients that completed the study, ranging from ages 28 to 60 years, were as follows: mean eGFR 75.87mL/min/1.73m2 in males, and 86.14mL/min/1.73m2 in females and plasma lyso-Gb3 mean levels were 51.81nM and 13.81nM in males and females, respectively. While lyso-Gb3 levels remain slightly high, particularly within the male cohort, continuous reduction in lyso-Gb3 levels was observed of 19.55nM (32.35%) in males and 4.57nM (29.81%) in females.

"The final analysis of the BRIDGE Study in Fabry patients previously treated with agalsidase alfa demonstrates a potential benefit of pegunigalsidase alfa on renal function," said Prof. Linhart.

Pegunigalsidasealfa was well tolerated in the study, with all adverse events being transient in nature without sequelae. Among the 22 patients enrolled in the BRIDGE study, the majority of treatment emergent adverse events were mild or moderate in severity (96.9%), with two patients (9.1%) withdrawing from the therapy due to hypersensitivity reaction that was resolved. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea.

Additional details can be found on the WORLDSymposiumwebsite at https://worldsymposia.org/. Copies of the presentation materials will be made available on Protalix's website under the Presentations tab in the Investors section at the time of the poster session.

About Fabry Disease

Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal -Galactosidase-A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person's body.

Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the -Galactosidase-A enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with time and, accordingly, Gb3 accumulates, primarily in the blood and in the blood vessel walls. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure particularly of the kidneys, but also of the heart and the cerebrovascular system.

About Pegunigalsidase Alfa

Pegunigalsidase alfa (PRX-102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant -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. PRX102 has been designed to potentially address the continued unmet clinical need in Fabry patients.

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 visitwww.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.

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 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 for marketing by the U.S. Food and Drug Administration 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 version of the recombinant human GalactosidaseA protein for the proposed treatment of Fabry disease; OPRX106, an orally- delivered anti-inflammatory treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others. Protalix has partnered with Chiesi Global Rare Diseases, both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa.

Forward-Looking Statements

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," "plan," "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: failure or delay in the commencement or completion of our preclinical and clinical trials which may be caused by several factors, including: risks that the FDA will not accept an application for accelerated approval of PRX102 with the data generated to date or will request additional data or other conditions of our submission of any application for accelerated approval of PRX102; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to monitor patients adequately during or after treatment; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; and slower than expected rates of patient recruitment; risks associated with the novel coronavirus disease (COVID19) outbreak, which may adversely impact our business, preclinical studies and clinical trials; the risk that the results of the clinical trials of our product candidates will not support our claims of safety or efficacy, 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 Chiesi Farmaceutici and any other collaborator, distributor or partner; risks related to the amount of our future revenues and expenditures; the risk that despite the FDA's grant of fast track designation for PRX102, we may not experience a faster development process, review or approval compared to applications considered for approval under conventional FDA procedures; risks related to the FDA's ability to withdraw the fast track designation at any time; 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; delays in the approval or potential rejection of any applications we file with the FDA or other health regulatory authorities, and other risks relating to the review process; 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.

Investor ContactChuck Padala, Managing DirectorLifeSci Advisors646-627-8390[emailprotected]

SOURCE Protalix BioTherapeutics, Inc.; Chiesi

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Protalix BioTherapeutics and Chiesi Global Rare Diseases Present Key Clinical Data of Pegunigalsidase Alfa for the Treatment of Fabry Disease at the...

InspireMD Appoints Leading Interventional Cardiologist Chris Metzger, MD as Principal Investigator for CGuard Registration Trial in the United States…

Posted: February 9, 2021 at 6:53 pm


TEL AVIV, Israel, Feb. 09, 2021 (GLOBE NEWSWIRE) -- InspireMD, Inc. (NYSE American: NSPR), developer of the CGuard Embolic Prevention System (EPS) for the prevention of stroke caused by carotid artery disease, announced today the appointment of Chris Metzger, M.D., system chair of clinical research at Ballad Health System in Eastern Tennessee as the principal investigator for its planned FDA registration trial for CGuard EPS.

InspireMD is extremely fortunate to have Chris Metzger enthusiastically agree to shepherd the CGuard clinical trial. Chris is an extremely skilled, high volume cardiovascular and carotid stent operator. He is also a highly regarded mentor and educator. His vast clinical trial experience will bring strong leadership to this landmark FDA study," stated Gary Roubin, M.D., Ph.D., InspireMD Director and internationally renowned interventional cardiologist recognized for his pioneering work in carotid stenting and embolic and protection devices.

Dr. Metzger currently serves as medical director of the Interventional and Diagnostic Catheterization Labs at Holston Valley Medical Center and medical director of clinical research at Ballard Health Systems, both located in Eastern Tennessee and Southwestern Virginia. Dr. Metzger is widely published and has participated in more than 100 clinical studies, in most of which he served as principal investigator.

Following a close review of the extensive European experience and clinical outcomes, Im looking forward to participating in the upcoming U.S. pivotal trial for CGuard. There remains a significant need to address stroke prevention in the United States and based on CGuards protective MicroNet technology, Im intrigued with the potential for success here, commented Dr. Metzger.

The appointment of Chris Metzger as our principal investigator is an important step toward the initiation of our pivotal clinical trial for CGuard in the United States. We are confident that connecting the study to such a well-respected and experienced practitioner will help guide the continued development and execution of the study to a successful conclusion, reflective of the results we have seen internationally. We are grateful to Dr. Metzger for his collaboration and look forward to providing updates on the trials development, added Marvin Slosman, CEO of InspireMD.

About the CGuard EPS

The CGuard Embolic Protection System is an advanced platform solution designed to deliver the flexibility of the traditional open-cell stent with advanced protection from peri-procedural and post-procedural embolic events caused by plaque prolapse through the stent strut that can lead to stroke. CGuards unique MicroNet technology mitigates the prolapse and associated embolization and has shown superior clinical outcomes for patients against alternative carotid stent types, conventional and next-generation double-layer stents, as well as invasive procedures such as endarterectomy, a major surgical procedure. InspireMDs CGuard has created a new dimension in the protected treatment of carotid artery disease and has the potential to establish a new standard of care for the management of carotid artery disease and stroke prevention.

About InspireMD, Inc.InspireMD seeks to utilize its proprietary MicroNet technology to make its products the industry standard for carotid stenting by providing outstanding acute results and durable, stroke-free, long-term outcomes. For more information, visit http://www.inspiremd.com. InspireMD routinely posts information that may be important to investors in the Investors section of its website.

Forward-looking StatementsThis press release contains forward-looking statements. Such statements may be preceded by the words intends, may, will, plans, expects, anticipates, projects, predicts, estimates, aims, believes, hopes, potential or similar words. Forward-looking statements are not guarantees of future performance, are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Companys control, and cannot be predicted or quantified and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) market acceptance of our existing and new products, (ii) negative clinical trial results or lengthy product delays in key markets, (iii) an inability to secure regulatory approvals for the sale of our products, (iv) the impact of the COVID-19 pandemic on our manufacturing, sales, business plan and the global economy, (v) intense competition in the medical device industry from much larger, multinational companies, (vi) product liability claims, (vii) product malfunctions, (viii) our limited manufacturing capabilities and reliance on subcontractors for assistance, (ix) insufficient or inadequate reimbursement by governmental and other third party payers for our products, (x) our efforts to successfully obtain and maintain intellectual property protection covering our products, which may not be successful, (xi) legislative or regulatory reform of the healthcare system in both the U.S. and foreign jurisdictions, (xii) our reliance on single suppliers for certain product components, (xiii) the fact that we will need to raise additional capital to meet our business requirements in the future and that such capital raising may be costly, dilutive or difficult to obtain and (xiv) the fact that we conduct business in multiple foreign jurisdictions, exposing us to foreign currency exchange rate fluctuations, logistical and communications challenges, burdens and costs of compliance with foreign laws and political and economic instability in each jurisdiction. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Companys filings with the Securities and Exchange Commission (SEC), including the Companys Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SECs web site at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.

Investor Contacts:

Craig ShoreChief Financial OfficerInspireMD, Inc.888-776-6804craigs@inspiremd.com

Original post:
InspireMD Appoints Leading Interventional Cardiologist Chris Metzger, MD as Principal Investigator for CGuard Registration Trial in the United States...

Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with…

Posted: at 6:53 pm


REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced positive preliminary findings from its ongoing multicenter Phase 1 clinical trial of JSP191, a first-in-class anti-CD117 (stem cell factor receptor) monoclonal antibody, as a conditioning agent in older patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) undergoing hematopoietic (blood) cell transplantation.

Data from the first six patients who received a single dose of JSP191 prior to transplantation showed successful engraftment in all six patients. Complete donor myeloid chimerism (equal or greater than 95%) was observed in five of six evaluable patients at 28 days, and all three evaluable patients had total donor chimerism equal or greater than 95% observed at day 90. In addition, at 28 days, three of five evaluable patients showed complete eradication of measurable residual disease (MRD) as measured by next-generation sequencing. Two of the five evaluable patients showed substantial reductions in MRD. No treatment-related serious adverse events were reported.

The findings were presented by lead investigator Lori Muffly, M.D., M.S., Assistant Professor of Medicine (Blood and Bone Marrow Transplantation) at Stanford Medicine, as a late-breaking abstract at the 2021 Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).

These early clinical results are the first to demonstrate that JSP191 administered in combination with a standard non-myeloablative regimen of low-dose radiation and fludarabine is well tolerated and can clear measurable residual disease in older adults with MDS or AML undergoing hematopoietic cell transplantation a patient population with historically few options, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. These patients could be cured by hematopoietic cell transplantation, but the standard-of-care myeloablative conditioning regimens used today are highly toxic and associated with high rates of morbidity and mortality particularly in older adults. Traditional lower intensity transplant conditioning regimens are better tolerated in older adults, but are associated with higher rates of relapse in MDS/AML patients with measurable residual disease. JSP191, a well-tolerated biologic conditioning agent that targets and depletes both normal hematopoietic stem cells and those that initiate MDS and AML, has the potential to be a curative option for these patients.

The open-label, multicenter Phase 1 study (JSP-CP-003) is evaluating the safety, tolerability and efficacy of adding JSP191 to the standard conditioning regimen of low-dose radiation and fludarabine among patients age 65 to 74 years with MDS or AML undergoing hematopoietic cell transplantation. Patients were ineligible for full myeloablative conditioning. The primary outcome measure of the study is the safety and tolerability of JSP191 as a conditioning regimen up to one year following a donor cell transplant.

We designed JSP191 to be given as outpatient conditioning and to have both the efficacy and safety profile required for use in newborn patients and older patients for successful outcomes, said Wendy Pang, M.D., Ph.D. Executive Director, Research and Translational Medicine, of Jasper Therapeutics. We are enthusiastic about the reduction of measurable residual disease seen in these patients, especially given that it is associated with improved relapse-free survival. We are excited to continue our research in MDS/AML, with plans for an expanded study. We are evaluating JSP191, the only antibody of its kind, in two ongoing clinical studies and are encouraged by the positive clinical data seen to date.

About MDS and AML

Myelodysplastic syndromes (MDS) are a group of disorders in which immature blood-forming cells in the bone marrow become abnormal and do not make new blood cells or make defective blood cells, leading to low numbers of normal blood cells, especially red blood cells.1 In about one in three patients, MDS can progress to acute myeloid leukemia (AML), a rapidly progressing cancer of the bone marrow cells.1 Both are diseases of the elderly with high mortality. Each year, about 5,000 patients with MDS and 8,000 people with AML in the G7 countries receive hematopoietic cell transplants. These transplants are curative but are underused due to the toxicity of the current high-intensity conditioning regimen, which includes the chemotherapy agents busulfan and fludarabine.

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplantation. A Phase 1/2 dose-escalation and expansion trial is evaluating JSP191 as a sole conditioning agent to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplantation for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. Data presented at the 62nd American Society of Hematology (ASH) Annual Meeting showed that a single dose of JSP191 administered prior to stem cell transplantation in a 6-month-old infant was effective in establishing sustained donor chimerism followed by development of B, T and NK immune cells. No treatment-related adverse events were reported. A Phase 1 clinical study is evaluating JSP191 in combination with another low-intensity conditioning regimen in patients with MDS or AML undergoing hematopoietic cell transplantation. For more information about the design of these two ongoing clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

1 https://www.cancer.org/cancer/myelodysplastic-syndrome/about/what-is-mds.html

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Jasper Therapeutics Announces Positive Data from Phase 1 Clinical Trial of JSP191 as Targeted Stem Cell Conditioning Agent in Patients with...

Sorrento Announces Subsidiary Company – ADNAB, Inc. – to Develop and Commercialize ADNAB Platform Products for Hematological Malignancies and Solid…

Posted: at 6:53 pm


SAN DIEGO, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, Sorrento) today announced the formation of ADNAB, Inc., a subsidiary Company, that will develop and commercialize a Mayo Clinic-developed technology platform for the manufacture of antibody-drug conjugates (ADC), each called an ADNAB.

An ADNAB is an immune complex of nanoparticle albumin-bound drug products e.g., nab-paclitaxel, which are non-covalently conjugated with tumor-targeting monoclonal antibodies (mAbs). The ADNAB platform was developed by Svetomir Markovic, M.D., Ph.D., and his research team at Mayo Clinic. To date, Dr. Markovics team have successfully formed nine (9) potential ADNAB candidates, including two (2) that are currently enrolling in an FDA supervised, investigator-initiated human trial.

Utilizing Sorrentos G-MAB library of fully humanized monoclonal antibodies, the ADNAB platform will generate a broad portfolio of product candidates targeting liquid and solid tumors. We believe this platform has broad potential. said Henry Ji, Ph.D., Chairman and CEO of the newly formed ADNAB, Inc. Our Vision, is to extend the reach of this platform to therapeutic areas beyond oncology; we have already begun work on an ADNAB for auto-immune diseases.

Mayo Clinic physician Tom Habermann, M.D., serves as the Principal Investigator for Study LS1681, which is evaluating a rituximab-ADNAB in relapsed/refractory B-cell lymphomas. Relapsed-Refractory Diffuse Large B Cell Lymphoma (RR DLBCL), which accounts for approximately one-third of patients with DLBCL, remains a major cause of morbidity and mortality.

I think this technology has the potential to be impactful said Bradley J. Monk, M.D., Professor of Gynecologic Oncology at the University of Arizona College of Medicine. Im especially excited by the flexibility of this platform and Im anxious to see what we might be able to do with a product that targets CA-125, added Dr. Monk.

The Mayo team has spent years fine-tuning this technology and now we have a collaborator that can provide the resources necessary to accelerate and scale this program, said Svetomir Markovic, M.D., Ph.D., who discovered and developed the ADNAB platform technology.

The published clinical studies testing this platform technology are encouraging and we are looking forward to working with the Mayo team on both the ongoing and future clinical studies. Unquestionably, Sorrento and Mayo share the common goal of utilizing this technology to develop multiple therapies for the possible benefit of cancer patients, noted Ji.

ADNAB, Inc. plans to file multiple INDs this year; and to request Breakthrough Therapy designation from the FDA in both ovarian and endometrial cancers.

About Sorrento Therapeutics, Inc.

Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to treat cancers and COVID-19. Sorrentos multimodal, multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as fully human antibodies (G-MAB library), clinical stage immuno-cellular therapies (CAR-T, DAR-T), antibody-drug conjugates (ADCs), and clinical stage oncolytic virus (Seprehvir). Sorrento is also developing potential antiviral therapies and vaccines against coronaviruses, including COVI-GUARD, COVI-AMG, COVI-SHIELD, Gene-MAb, COVI-MSC and COVI-DROPS; and diagnostic test solutions, including COVI-TRACK, COVI-STIX and COVI-TRACE.

Sorrentos commitment to life-enhancing therapies for patients is also demonstrated by our effort to advance a first-in-class (TRPV1 agonist) non-opioid pain management small molecule, resiniferatoxin (RTX), and SP-102 (10 mg, dexamethasone sodium phosphate viscous gel) (SEMDEXA), a novel, viscous gel formulation of a widely used corticosteroid for epidural injections to treat lumbosacral radicular pain, or sciatica, and to commercialize ZTlido (lidocaine topical system) 1.8% for the treatment of post-herpetic neuralgia. RTX has completed a phase IB trial for intractable pain associated with cancer and a phase 1B trial in osteoarthritis patients. SEMDEXA is in a pivotal Phase 3 trial for the treatment of lumbosacral radicular pain, or sciatica. ZTlido was approved by the FDA on February 28, 2018.

For more information visitwww.sorrentotherapeutics.com

Financial information

Mayo Clinic and Dr. Markovic have a financial interest in the technology referenced in this news release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.

Forward-Looking Statements

This press release and any statements made for and during any presentation or meeting contain forward-looking statements related toSorrento Therapeutics, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995 and subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding plans and timing for the development and commercialization of the ADNAB technology for multiple therapies, including endometrial cancer, ovarian cancer, angiosarcoma and B-Cell lymphomas; the preliminary proof-of-concept results of Investigator-Initiated Trials to date; the ability for the ADNAB technology to potentially generate a broad portfolio of product candidates targeting liquid and solid tumors; plans to expand the ADNAB platform to other therapeutic areas, including auto-immune diseases; the continuation of ongoing clinical trials and initiation of future trials at Mayo Clinic; the planned filing of multiple IND applications and plans to seek breakthrough therapy designation for STI-0201 in ovarian and endometrial cancers; and Sorrentos research, clinical and commercial plans with respect to the ADNAB technology. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements, include, but are not limited to: risks related to Sorrentos and its subsidiaries, affiliates and partners technologies and prospects and collaborations with partners, including, but not limited to risks related to seeking regulatory approval for any antibody product candidates utilizing the ADNAB technology; clinical development risks, including risks in the progress, timing, cost, and results of clinical trials and product development programs; risk of difficulties or delays in obtaining regulatory approvals; risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval; risks that prior test results may not be replicated in future studies and trials; risks of manufacturing and supplying drug product; risks related to leveraging the expertise of its employees, subsidiaries, affiliates and partners to assist Sorrento in the execution of its cancer, anti-tumor and G-MAB antibody strategies; risks related to the global impact of COVID-19; and other risks that are described in Sorrentos most recent periodic reports filed with theSecurities and Exchange Commission, including Sorrentos Annual Report on Form 10-K for the year endedDecember 31, 2019, and subsequent Quarterly Reports on Form 10-Q filed with theSecurities and Exchange Commission, including the risk factors set forth in those filings. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law.

Media and Investor RelationsContact: Alexis Nahama, DVM (SVP Corporate Development)Telephone: 1.858.203.4120Email: mediarelations@sorrentotherapeutics.com

Sorrentoand the Sorrento logo are registered trademarks ofSorrento Therapeutics, Inc.

G-MAB, DAR-T, ANDAB, COVI-GUARD, COVI-AMG, COVI-SHIELD, Gene-MAb, COVI-DROPS, COVI-MSC, COVI-TRACK, COVI-TRACE and COVI-STIX are trademarks ofSorrento Therapeutics, Inc.

SEMDEXA is a trademark of Semnur Pharmaceuticals, Inc.

ZTlidois a trademark owned byScilex Pharmaceuticals Inc.

All other trademarks are the property of their respective owners.

2021Sorrento Therapeutics, Inc.All Rights Reserved.

1 https://clinicaltrials.gov/ct2/show/NCT03003546?term=Ls1681&draw=2&rank=12 https://clinicaltrials.gov/ct2/show/NCT02020707?term=mc1371&draw=2&rank=13 DOI: 10.1200/JCO.2020.38.15_suppl.e18097Journal of Clinical Oncology38, no.15_suppl

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Sorrento Announces Subsidiary Company - ADNAB, Inc. - to Develop and Commercialize ADNAB Platform Products for Hematological Malignancies and Solid...

Innovent Announces First Patient Dosed in Phase III Pivotal Trial of IBI310 (CTLA-4) Combined with TYVYT (sintilimab injection) for the Treatment of…

Posted: at 6:53 pm


SAN FRANCISCO and SUZHOU, China, Feb. 8, 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 oncology, metabolic, autoimmune and other major diseases, announces that the first patient has been successfully enrolled and dosed in the randomized, open-label, phase III, multicenter clinical study (NCT04720716) evaluating its IBI310 (anti CTLA-4 monoclonal antibody) in combination with TYVYT (sintilimab injection) for the first-line treatment of patient with advanced hepatocellular carcinoma (HCC).

In recent years, immune checkpoint inhibitors have brought new hope to HCC patient population with acceptable safety and encouraging efficacy.NCT04720716 is a randomized, open-label, controlled, multicenter phase III study evaluating the efficacy and safety of IBI310 in combination with TYVYT (sintilimab injection) for advanced HCC patients. Primary endpoints include overall survival and objective response rate. The study was led by Professor Fan Jia from Zhongshan Hospital of Fudan University, and Professor Qin Shukuifrom Affiliated Jinling Hospital, Medical School of Nanjing University. This study involves more than 50 clinical centers.

Dr. Hui Zhou, Vice President and Head of Medical Sciences and Oncology Strategy of Innovent, stated: "CTLA-4 is an important immunosuppressive receptor. IBI310 in combination with TYVYT (sintilimab injection) has shown promising preliminary good safety and anti-tumor activity, suggesting the potential anti-tumor value for this combination. We will evaluate the efficacy of IBI310 combined with TYVYT (sintilimab injection) in this study and we hope to provide more effective treatment to benefit patients and their families."

About IBI310

IBI310 is a recombinant fully-human monoclonal antibody against cytotoxic T lymphocytic associated antigen 4 (CTLA-4). IBI310 can interfere with the binding of CTLA-4 and CD80/CD86 on antigen presenting cells, thereby blocking the inhibitory effect on T cell activation. IBI310 can promote the activation and amplification of T cells, and enhance the anti-tumor ability of the immune system.

CTLA-4 provides a new approach for immunotherapy in many diseases, including tumors. Innovent has announced the preliminary results of the Phase 1 clinical study about anti-CTLA-4 monoclonal antibody (NCT03545971) at the 56th Annual American Society of Clinical Oncology (ASCO) (Online Publication, Abstract No. 302489). Phase II/III clinical studies of IBI310 combined with TYVYT (sintilimab injection) for multiple tumors are ongoing.

About TYVYT (Sintilimab Injection)

TYVYT (sintilimab injection) is an innovative PD-1 inhibitor with global quality standards jointly developed by Innovent and Eli Lilly and Company. In December 2018, TYVYT (sintilimab injection) was first approved by the China NMPA for the treatment of relapsed or refractory classic Hodgkin's lymphoma after two lines or later of systemic chemotherapy. In February 2021, TYVYT (sintilimab injection) was approved by the China NMPA in combination with pemetrexed and platinum chemotherapy as first-line therapy for the treatment of nonsquamous non-small cell lung cancer. TYVYT (sintilimab injection)was included in the National Reimbursement Drug List (NRDL) in 2019 as the first PD-1 inhibitor and the only PD-1 included in the list in that year.

Currently TYVYT (sintilimab injection) has three supplemental New Drug Applications ("sNDA") under review by the NMPA. In August 2020, the NMPA accepted sNDA for TYVYT (sintilimab injection)in combination with GEMZAR (gemcitabine for injection) and platinum chemotherapy as first-line therapy in squamous NSCLC. In January 2021, the NMPA accepted the sNDA for TYVYT (sintilimab injection)in combination with BYVASDA (bevacizumab injection)as first-line therapy in Hepatocellular Carcinoma (HCC) and the sNDA for TYVYT (sintilimab injection)as second-line therapy in squamous NSCLC. Besides, in May 2020, TYVYT (sintilimab injection) monotherapy met the primary endpoint of overall survival in the Phase 2 ORIENT-2 study as second-line therapy in patients with advanced or metastatic esophageal squamous cell carcinoma.

TYVYT (sintilimab injection), is a type of immunoglobulin G4 monoclonal antibody, which binds to PD-1 molecules on the surface of T-cells, block the PD-1 / PD-Ligand 1 (PD-L1) pathway and reactivate T-cells to kill cancer cells. Innovent is currently conducting more than 20 clinical studies with TYVYT (sintilimab injection) to evaluate its safety and efficacy in a wide variety of cancer indications, including more than 10 registrational or pivotal clinical studies. Meanwhile, Innovent is conducting clinical research studies on TYVYT (sintilimab injection) worldwide.

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 and other major diseases. 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. Leveraging the platform, the company has built a robust pipeline of 23 valuable assets in the fields of cancer, metabolic, autoimmune diseases and other major therapeutic areas, with 4 products - TYVYT (sintilimab injection), BYVASDA (bevacizumab biosimilar injection), SULINNO (adalimumab biosimilar injection) and HALPRYZA (rituximab biosimilar injection) - officially approved for marketing in China, four assets in Phase 3 or pivotal clinical studies, and additional 15 molecules in clinical studies. TYVYT 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 talents in high-end biological drug 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.

Note:

TYVYT (sintilimab injection) is not an approved product in the United States.

BYVASDA (bevacizumab biosimilar injection) is not an approved product in the United States.

SULINNO (adalimumab biosimilar injection) is not an approved product in the United States.

HALPRYZA (rituximab biosimilar injection) is not an approved product in the United States.

TYVYT (sintilimab injection, Innovent)

BYVASDA(bevacizumab biosimilar injection, Innovent)

SULINNO (adalimumab biosimilar injection, Innovent)

HALPRYZA (rituximab biosimilar injection, Innovent)

ALIMTAand GEMZARare trademarks owned by or licensed to Eli Lilly and Company, its subsidiaries, or affiliates.

Disclaimer:

1. This indication is still hasn't been approved in China.

2. Innovent does not recommend any off-label usage.

3. For medical and healthcare professionals only.

SOURCE Innovent Biologics, Inc.

http://www.innoventbio.com

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Innovent Announces First Patient Dosed in Phase III Pivotal Trial of IBI310 (CTLA-4) Combined with TYVYT (sintilimab injection) for the Treatment of...

Global Clinical Trials Management System (CTMS) Market was estimated to grow at a rate of 14.6% during the forecast period owing to rising number of…

Posted: February 7, 2021 at 2:51 am


Every year the life science industry witnesses a global increase in the number of clinical trials. The increase in the number of clinical trials carried out can be due to factors such as the rapidly rising geriatric numbers across the developed and emerging regions, growing incidence of chronic diseases, the expiry of breakthrough medications, the scarcity of government funding for clinical trials and fierce competition in the pharmaceutical industry amongst others. As of December, 2017, 260,848 clinical trials were registered around the globe.

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Market players have implemented a range of growth strategies, such as product launches, strategic acquisitions, alliances, partnerships, collaborations and expansions, to boost their product portfolios and maintain a competitive position in the clinical trials management system market. Among those business strategies, product improvements, agreements and alliances were the growth strategy most widely adopted by the industry participants. On the other hand, long approval time and high cost for clinical trials, difficulties in recruiting and enrolling the patients for clinical trial are certain areas of concern in the market. High costs involved in clinical studies can, however, can critically impact the overall clinical trials management system market.

Oracle (US) is major player in the CTMS business. Because of its diverse geographic reach and large customer base, strong brand identity, selective acquisition program and partnerships, the company is considered one of the top players in the clinical trials management system market. The company provides cloud based clinical trials management system. The business has a large regional reach over 175 countries. The company focuses primarily on expansions and acquisitions to improve its dominant market position. It opened its Oracle Cloud EU area in Germany in 2017, with the introduction of modern infrastructure as a service (IaaS) architecture and platform as a service (PaaS) architecture.

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The detailed research study provides qualitative and quantitative analysis of the global clinical trials management system market. The geographical analysis done emphasizes on each of the major countries across North America, Europe, Asia Pacific, Middle East & Africa, and Latin America.

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Clinical Trials Management Systems Market

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Exonate announces the initiation of Phase Ib/II clinical trial for Diabetic Macular Oedema, as part of its collaboration with Janssen – GlobeNewswire

Posted: at 2:51 am


CAMBRIDGE, United Kingdom, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Exonate, an mRNA therapy company focused on retinal diseases, today announced that in collaboration with Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, the first patient has been dosed with Exonate's lead compound, EXN 407, in a Phase Ib/II clinical trial of patient volunteers with centre-involved diabetic macular oedema (CI-DMO). The original agreement with Janssen was facilitated by Johnson & Johnson Innovation.

Dr Catherine Beech, Chief Executive Officer of Exonate, said: "The initiation of our first clinical trial is an important step in the validation of our eye drop approach. This is a unique opportunity to create a drug that may have the potential to improve the treatment of patients with retinal vascular diseases and transform the lives of those suffering from vision loss. The collaboration with Janssen has been incredibly positive and together, we have designed a study that we believe will deliver meaningful results."

By exploiting the alternative splicing of Vascular Endothelial Growth Factor (VEGF), Exonate has developed small-molecules for the treatment of retinal neovascular diseases. EXN 407 inhibits serine/arginine-protein kinase 1 (SRPK1). SRPK1 enables production of VEGF, which initiates or inhibits vessel formation depending on alternative splicing. CI-DMO is caused by an increase of vessel formation on the retina and current treatment options for CI-DMO and other retinal diseases require intravitreal injections directly into the eye. EXN 407 has been designed with sufficient ocular permeability to be given topically as eye drops and represents a shift in the potential treatment of retinal vascular eye disease compared to current treatments for VEGF-dependent retinal vascular diseases. Pre-clinical studies have demonstrated an effect on neovascularisation and retinal vascular permeability induced by diabetes, without any significant tolerability or safety issues.

The double-blind, randomised multicentre trial of 48 patients is being conducted at retinal centres across Australia. The trial consists of a dose escalation phase during which three doses of EXN 407 and a placebo are tested, followed by an expansion phase with a larger cohort of patient volunteers and a longer drug dosing period. The study aims to demonstrate safety and tolerability and an exploratory end point of efficacy through reduction in retinal thickness in a proportion of patients.

To date, Australia has managed the COVID-19 pandemic such that no major delays are expected in patient recruitment and we anticipate topline results in early 2022.

David Bates, Scientific Founder and CSO, added: "It is a credit to many people that this complex and potentially game-changing therapy has reached first-in-human studies. This has been an immense team-effort from the original lab discovery work at the Universities of Bristol, New South Wales and Nottingham to the development of the pre-clinical and clinical programmes by the Exonate team, our contractors and collaborators. We look forward to learning the study results in due course."

DMO is the most common cause of vision loss among people with diabetic retinopathy and affects approximately 21 million people worldwide[1]. DMO is a build-up of fluid in a region of the retina called the macula and is associated with an increase in retinal thickness due to leakage of fluid and plasma proteins from retinal vessels which leads to central vision loss. Although DMO is more likely to occur as diabetic retinopathy worsens, it can happen at any stage of the disease.

1. Yau, JWY for the META-EYE Disease Study Group. Global Prevalence and Major Risk Factors of Diabetic Retinopathy. Diabetes Care 2012, 35:556-564.

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Exonate announces the initiation of Phase Ib/II clinical trial for Diabetic Macular Oedema, as part of its collaboration with Janssen - GlobeNewswire

Medidata expands partnership with Horizon Therapeutics on clinical trials – Clinical Trials Arena

Posted: February 5, 2021 at 11:54 pm


American technology firm Medidata, a subsidiary of Dassault Systmes, has expanded its partnership with biopharmaceutical company Horizon Therapeutics to improve efficiencies and outcomes in clinical trials.

Under the expanded partnership, Medidata will provide a wide range of solutions for Horizons clinical research needs in various therapeutic areas, such as ophthalmology, rheumatology, and nephrology.

Medidata co-founder and co-CEO Tarek Sherif said: Were delighted to be a key partner to Horizon and its exceptional journey that benefits patients by advancing science and treatments.

Medidatas goal is to facilitate Horizons path forward using advanced analytics and technology. Were looking forward to further supporting the companys vision, especially as it relates to improving efficiencies and speeding the completion of clinical studies.

Using Medidatas most used platform, Medidata Rave Clinical Cloud, Horizon will access a broad range of clinical trial solutions.

The Medidata platform is used to accelerate clinical research with a set of applications such as data capture, data management, trial planning, as well as trial management.

Furthermore, Horizon will use MedidatasIntelligent Trialsproduct to bring cross-industry real-time performance metrics, predictive models, and forecasting capabilities for clinical trial planning as well as its execution.

Built on the industrys broadest clinical trial dataset, Intelligent Trials brings artificial intelligence (AI), partnered with advanced analytics.

It covers over six million patients in more than 22,000 clinical trials.

GlobalData's TMT Themes 2021 Report tells you everything you need to know about disruptive tech themes and which companies are best placed to help you digitally transform your business.

Horizon Therapeutics research and development executive vice-president Karin Rosn said: Through the use of the Medidata technologies, we have been able to create a more efficient, manageable and cost-effective clinical trial process, which led us to expand this productive relationship.

As our clinical needs have evolved, the Medidata platform gives us the flexibility and agility to scale and grow.

In July last year, Medidata said it would support clinical trials of Modernas Covid-19 vaccine candidate mRNA-1273 after the companies entered a partnership.

Software Solutions for Clinical Trial and Medical Research Project Management

28 Aug 2020

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Vaxart Announces Positive Preliminary Data from Phase 1 Clinical Trial Evaluating Its Oral COVID-19 Tablet Vaccine Candidate – GlobeNewswire

Posted: at 11:54 pm


SOUTH SAN FRANCISCO, Calif., Feb. 03, 2021 (GLOBE NEWSWIRE) -- Vaxart, Inc., (NASDAQ: VXRT), a clinical-stage biotechnology company developing oral vaccines administered by tablet, today announcedpreliminary data from its Phase 1 study of VXA-CoV2-1 showing that its oral COVID-19 tablet vaccine candidate was generally well-tolerated, and immunogenic as measured by multiple markers of immune response to SARS-CoV-2 antigens.

Our Phase I results highlight the importance of our differentiated vaccine design, as they suggest VXA-CoV2-1 could have broad activity against existing and future coronavirus strains. These results are timely, as we are seeing the emergence of new variants less responsive to first generation vaccines, thus making potential cross-reactivity another important advantage of next-generation vaccines, said Andrei Floroiu, Vaxarts Chief Executive Officer.

Vaxarts scientists recognized early the risk of variants of SARS-CoV-2 emerging and they designed a vaccine with the potential to be protective not only against the prevalent strain, but also against emerging mutations of the Spike (S) protein, by including both the S and N proteins. Virtually all other COVID-19 vaccines include just the S protein.

These results, together with recent data from our peers, further raise our confidence in the success of VXA-CoV2-1 and the broad potential of our platform, continued Floroiu.

We previously showed that our oral tablet vaccine technology worked to protect against flu another airborne virus as well as the leading injectable, but through a different mechanism, in a Phase II trial sponsored by BARDA. With COVID-19, we have now seen that many vaccine approaches mRNA, protein, and viral vector, including three adenovirus vaccines are protective, and that all available positive COVID-19 hamster challenge studies such as ours have translated into protection against COVID-19 in human trials, Floroiu said.

These clinical data further differentiate our COVID-19 vaccine and enable us to meaningfully advance discussions with healthcare officials in the U.S. and around the world about how Vaxart may be able to help them fight back against COVID-19 with a transformative solution - a room-temperature stable oral vaccine that is not only easier to distribute and administer, but may also be more broadly protective, Floroiu added.

Sean Tucker, Ph.D., Vaxarts Chief Scientific Officer, will present the Phase 1 data as part of a clinical trial update at the New York Academy of Sciences Symposium The Quest for a COVID-19 Vaccine today at 1:15 p.m. ET. Register here to attend the symposium.

Preliminary Phase 1 trial results from a pooled analysis of all cohorts include:

VXA-CoV2-1 was generally well-tolerated:

VXA-CoV2-1 triggered multiple immune responses against SARS-CoV-2 antigens, including:

Viral variants with altered S proteins are becoming established in the population before the majority of people can be vaccinated. To end the pandemic, the world needs a vaccine that can provide long-lasting protection from emerging strains, Dr. Tucker said.

T-cells can provide long-lasting cross-reactive protection against current and emerging strains of the virus. Our vaccine induced a high percentage of responding CD8+ T cells against both Spike (S) and Nucleoprotein (N) proteins, which may provide protection against variants with alterations in the faster-changing S protein. We expect that our vaccine will be less impacted by new variants than injectable vaccines, Dr. Tucker added.

Vaxart expects to broaden its COVID-19 vaccine development plans, with efforts that could include:

Clinical Trial design

The Phase I study (NCT04563702) was designed to evaluate the safety and immunogenicity of VXA-CoV2-1 vaccine with multiple dosing schedules. Subjects were divided into three cohorts. The first cohort (5 subjects) received two low doses of vaccine 29 days apart. The remaining cohorts (15 subjects each) received a single low or high dose of the vaccine. Safety and tolerability were monitored following vaccination as well as signs of immunogenicity, including general and SARs-CoV-2 specific immune responses.

AboutVaxart

Vaxartis a clinical-stage biotechnology company developing a range of oral recombinant vaccines based on its proprietary delivery platform.Vaxartvaccines are designed to be administered using tablets that can be stored and shipped without refrigeration and eliminate the risk of needle-stick injury.Vaxarthas believes that its proprietary tablet vaccine delivery platform is suitable to deliver recombinant vaccines, positioning the company to develop oral versions of currently marketed vaccines and to design recombinant vaccines for new indications. Its development programs currently include tablet vaccines designed to protect against coronavirus, Norovirus, seasonal influenza and respiratory syncytial virus (RSV), as well as a therapeutic vaccine for human papillomavirus (HPV), Vaxarts first immuno-oncology indication.Vaxarthas filed broad domestic and international patents covering its proprietary technology and creations for oral vaccination using adenovirus and TLR3 agonists.

Note Regarding Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Vaxarts strategy, prospects, plans and objectives, results from preclinical and clinical trials, commercialization agreements and licenses, beliefs and expectations of management are forward-looking statements. These forward-looking statements may be accompanied by such words as should, believe, could, potential, will, expected, plan and other words and terms of similar meaning. Examples of such statements include, but are not limited to, statements relating to Vaxarts ability to develop (including enrolling a sufficient number of subjects and manufacturing sufficient quantities of its product candidates) and commercialize its COVID-19 vaccine candidate and preclinical or clinical results and trial data (including plans with respect to the COVID-19 vaccine product candidates); expectations regarding the timing and nature of future announcements including, those related to clinical trials and results of preclinical studies; Vaxarts expectations with respect to the important advantages it believes its oral vaccine platform can offer over injectable alternatives, particularly for coronaviruses; the potential applicability of results seen in our preclinical studies or trials to those that may be seen in human studies or clinical trials; the expected role of mucosal immunity in blocking transmission of COVID-19; and Vaxarts expectations with respect to the effectiveness of its products or product candidates, including Vaxarts potential role in mitigating the impact of COVID-19 globally.Vaxartmay not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements thatVaxartmakes, including uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials or preclinical studies, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial and preclinical study data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical studies; decisions by regulatory authorities impacting labeling, manufacturing processes, and safety that could affect the availability or commercial potential of any product candidate, including the possibility that Vaxarts product candidates may not be approved by the FDA or non-U.S.regulatory authorities; that, even if approved by the FDA or non-U.S.regulatory authorities, Vaxarts product candidates may not achieve broad market acceptance; that aVaxartcollaborator may not attain development and commercial milestones; thatVaxartor its partners may experience manufacturing issues and delays due to events within, or outside of, Vaxarts or its partners control, including the recent outbreak of COVID-19; difficulties in production, particularly in scaling up initial production, including difficulties with production costs and yields, quality control, including stability of the product candidate and quality assurance testing, shortages of qualified personnel or key raw materials, and compliance with strictly enforced federal, state, and foreign regulations; thatVaxartmay not be able to obtain, maintain and enforce necessary patent and other intellectual property protection; that Vaxarts capital resources may be inadequate; Vaxarts ability to obtain sufficient capital to fund its operations on terms acceptable toVaxart, if at all; the impact of government healthcare proposals and policies; competitive factors; and other risks described in the Risk Factors sections of Vaxarts Quarterly and Annual Reports filed with theSEC.Vaxartdoes not assume any obligation to update any forward-looking statements, except as required by law.

Contacts:

Investor Relations

David R. HolmesLifeSci Advisors, LLCTel: (646) 970-4995dholmes@lifesciadvisors.com

Media Relations

Gloria GasaaturaLifeSci CommunicationsTel: (646) 970-4688ggasaatura@lifescicomms.com

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Vaxart Announces Positive Preliminary Data from Phase 1 Clinical Trial Evaluating Its Oral COVID-19 Tablet Vaccine Candidate - GlobeNewswire

Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)…

Posted: at 11:54 pm


- First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies

- Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2,an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease.

We are proud to bring the first potentially disease-modifying treatment for GM2 gangliosidosis to the clinic, which is a milestone for Sio, for patients, and for the field of gene therapy, said Gavin Corcoran, M.D., Chief R&D Officer of Sio. By restoring lysosomal enzyme activity where it is essential, AXO-AAV-GM2 has the potential to change the course of this disease and help affected children attain and retain important neuro-developmental milestones. The prior expanded access study of AXO-AAV-GM2 provided important proof-of-concept data and we look forward to the results of the first stage of our study as we strive to develop a treatment for children suffering from this rapidly progressive and fatal disease.

Florian Eichler, M.D., Director of the Leukodystrophy Service of the Center for Rare Neurological Diseases at Massachusetts General Hospital, and principal investigator, added, To date, the current GM2 treatment landscape is limited to supportive care, underscoring the significant need for new treatment options to address this devastating pediatric neurodegenerative disease. AXO-AAV-GM2 has significant potential to address the clinical manifestations of both Tay Sachs and Sandhoff diseases, and as a result, the dosing of this patient represents a major step forward for this therapy. We look forward to evaluating the results of this study and advancing the first potentially disease-modifying treatment option for patients with GM2.

The Phase 1/2 study (NCT04669535) is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal cord of pediatric participants with both infantile and juvenile GM2 gangliosidosis. AXO-AAV-GM2 has been granted Orphan Drug and Rare Pediatric Disease Designation by the FDA and is the first investigational gene therapy to enter clinical trials for GM2 gangliosidosis. In 2019, clinical evidence from two patients under an expanded access IND found that treatment with AXO-AAV-GM2 was generally well-tolerated and associated with improved bioactivity outcomes.

The families of children with Sandhoff and Tay-Sachs diseases show incredible bravery in choosing to participate in investigational studies of novel therapeutics like AXO-AAV-GM2. We share their hope that this treatment can halt or reverse the otherwise inexorable course of these tragic diseases, said Terence R. Flotte, MD, Professor of Pediatrics and Dean at the University of Massachusetts Medical School and principal investigator of the trial.

GM2 gangliosidosis is a set of rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode the enzyme -Hexosaminidase A. It can be categorized into two distinct diseases, Tay-Sachs disease, which results from a mutation in the gene encoding the alpha subunit of the -Hexosaminidase A enzyme (HEXA), and Sandhoff disease, which results from a mutation in the gene encoding the beta subunit of the -Hexosaminidase A enzyme (HEXB). Children affected by GM2 gangliosidosis suffer from a progressively debilitating disease course and reduced life expectancy.

Sue Kahn, Executive Director of National Tay-Sachs & Allied Diseases Association(NTSAD), added, This news represents the culmination of many years of work to advance this research and immense support from the GM2 community, and it underscores the dire need for new treatment options capable of providing meaningful benefits to patients and families. We are extremely excited by the progress Sio has made and the hope it brings to our community.

Sio aims to advance the program through strategic partnerships with leading research organizations. The Company has a partnership with Viralgen, an AskBio subsidiary, to support AAV-based vector manufacturing of clinical trial material for the registrational study. Additionally, through an existing genetic testing collaboration with Invitae, ongoing partnership with GM2 gangliosidosis patient groups, and collaboration with leading academic researchers at the University of Massachusetts Medical School and Massachusetts General Hospital, Sio has begun patient identification and screening activities for the ongoing clinical study.

About AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the -hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via delivery of two co-administered AAVrh8 vectors.

About Sio Gene TherapiesSio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry, and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

In 2018, Sio licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as expect potentially, and potential, and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 2020, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media

Josephine Belluardo, Ph.D.LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officerinvestors@siogtx.com

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Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)...

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