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Joint Statement of the Integrity of Vaccine Trials and the Inclusion of Black, Indigenous and People of Color – Howard Newsroom

Posted: September 20, 2020 at 5:58 am


By Drs. Wayne A. I. Frederick, David M. Carlisle, Valerie Montgomery Rice and James Hildreth

We, as representatives of the four historically Black medical schools in our nation, are committed to the inclusion of Black, Indigenous and people of color (BIPOC) as we engage in research initiatives focused on the novel coronavirus, SARS CoV-2. The virus, COVID-19, disproportionately impacts the number of infections, complications, and deaths in our communities. Our research efforts will be governed by the basic principles of respect of persons, beneficence, and justice.

Respect for persons demands that our communities enter into research voluntarily and with adequate information. Beneficence ensures that our communities will recognize the benefits and risks that may result from the improvement of knowledge through their participation in research. And finally, justice will be achieved by ensuring that no person is denied participation in research without good reason, nor will anyone be unduly burdened by their participation.

Our decisions to recommend participation in clinical studies, including vaccine trials, will always be informed by rigorous science carried out under international rules governing safe and ethical conduct of research. Our approach will be unbiased nor influenced by financial or non-financial conflicts. We will rely on peer-reviewed, transparent science is an important component in protecting the welfare of persons who volunteer to participate in clinical studies.

Specifically, we stand together to:

These fundamental principles are inherent to each of us as individual medical schools, and collectively we pledge to use our unified voice to advocate for all who consider and those who participate in COVID-related clinical and translational research.

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Joint Statement of the Integrity of Vaccine Trials and the Inclusion of Black, Indigenous and People of Color - Howard Newsroom

MorphoSys and I-Mab Announce FDA Clearance of IND Application for MOR210/TJ210 in Patients with Advanced Cancer – BioSpace

Posted: September 18, 2020 at 9:59 am


PLANEGG/MUNICH, Germany and SHANGHAI, China, Sept. 17, 2020 /PRNewswire/ -- MorphoSys AG(FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) and I-Mab (Nasdaq: IMAB)today jointly announced that the U.S. Food and Drug Administration (FDA) has clearedthe Investigational New Drug application (IND) for MorphoSys' investigational human anti-C5aR1 antibody MOR210/TJ210 for the treatment of relapsed or refractory advanced solid tumors. The phase 1 clinical trial,designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of MOR210/TJ210, may proceedand is expected to commence subsequently.

MOR210/TJ210 is a highly differentiated monoclonal antibody that is directed against complement factor C5a receptor 1 (C5aR1). Tumor and stromal cells produce C5a that attracts immunosuppressive cell types such as myeloid-derived suppressor cells (MDSCs), M2 macrophages and neutrophils through C5aR1 expressed on their surface, contributing to a hostile tumor microenvironment towards T cells. MOR210/TJ210 is thought to blockthe interaction between C5a and C5aR1 by binding to C5aR1 and retardthe migration of suppressor cells. It has been shown to exert strong anti-tumor activity in combination with immune checkpoint inhibitors in preclinical studies.

"MOR210/TJ210 has demonstrated encouraging results in preclinical studies. We look forward to progressing MOR210/TJ210 into clinical studies which will enable us to characterize the safety and tolerability of MOR210/TJ210, as well as its potential clinical benefits in patients with cancers",said Dr Joan Shen, Chief Executive Officer of I-Mab.

"The FDA clearance of the IND application to initiate a Phase 1 clinical trial of MOR210/TJ210 is an important step forward in developing a new treatment for patients with advanced cancer", said Dr Malte Peters, Chief Research & Development Officer of MorphoSys. "We look forward to joining forces with I-Mab in developing highly innovative treatments in oncology and are pleased to support our partner during this significant phase."

MorphoSys and I-Mab entered into an exclusive strategic collaboration and licensing agreement to develop and commercialize MOR210/TJ210 in November 2018. Under the terms of agreement, I-Mab receives exclusive rights to develop and commercialize MOR210/TJ210 in Greater China and South Korea, while MorphoSys retains rights in other parts of the world. With support from MorphoSys, I-Mab will also fund and conduct all global development activities of MOR210/TJ210, including clinical trials in China and the U.S., towards clinical proof-of-concept (PoC) in oncology.

The two companies are also collaborating on MorphoSys' investigational human CD38 antibody MOR202/TJ202. I-Mab owns theexclusive rightsfor development and commercializationin mainland China, Taiwan, Hong Kong and Macao and started two registrational trials to evaluate MOR202/TJ202 in patients with relapsed or refractory multiple myeloma in 2019.

About MOR210/TJ210

MOR210 is a novel humanantibody directed againstC5aR derived from MorphoSys's HuCAL Platinumtechnology. C5aR, the receptor of the complement factor C5a, is investigated as a potential new drug target in the field of immuno-oncology and autoimmune diseases. Tumors have been shown to produce high amounts of C5a,which, by recruiting and activating myeloid-derived suppressor cells (MDSCs), M2 macrophages and neutrophils, is assumed to contribute to an immune-suppressive pro-tumorigenic microenvironment. MOR210/TJ210 is intended to block the interaction between C5a and its receptor, thereby potentially neutralizingthe immune suppressive function and enablingimmune cells to attack the tumor.

About MorphoSys

MorphoSys (FSE & NASDAQ: MOR) is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of exceptional, innovative therapies for patients suffering from serious diseases. The focus is on cancer. Based on its leading expertise in antibody, protein and peptide technologies, MorphoSys, together with its partners, has developed and contributed to the development of more than 100 product candidates, of which 27 are currently in clinical development. In 2017, Tremfya, marketed by Janssen for the treatment of plaque psoriasis, became the first drug based on MorphoSys' antibody technology to receive regulatory approval. In July 2020,the U.S. Food and Drug Administration (FDA) granted accelerated approval ofthe company's proprietary product Monjuvi (tafasitamab-cxix) in combination with lenalidomide inpatients with a certain type oflymphoma.Headquartered near Munich, Germany, the MorphoSys group, including the fully owned U.S. subsidiary MorphoSys US Inc., has ~500 employees.

More information at http://www.morphosys.com orMorphoSys-US.com.

Monjuvi is a registered trademark of MorphoSys AG.

Tremfya is a registered trademark of Janssen Biotech.

About I-Mab

I-Mab (Nasdaq: IMAB) is a dynamic, global biotech company exclusively focused on discovery, development and soon commercialization of novel or highly differentiated biologics in the therapeutic areas of immuno-oncology and autoimmune diseases. The Company's mission is to bring transformational medicines to patients around the world through innovation. I-Mab's innovative pipeline of more than 10 clinical and pre-clinical stage drug candidates is driven by the Company's Fast-to-PoC (Proof-of-Concept) and Fast-to-Market development strategies through internal R&D and global partnerships. The Company is on track to transitioning from a clinical stage biotech company toward a fully integrated global biopharmaceutical company with cutting-edge R&D capabilities, world-class GMP manufacturing facility and commercial capability. I-Mab has offices in Beijing, Shanghai, Hong Kong and Maryland, United States. For more information, please visit http://ir.i-mabbiopharma.com

MorphoSys Forward-Looking Statements

This communication contains certain forward-looking statements concerning the MorphoSys group of companies, including the expectations regarding the further clinical development of MOR210/TJ210,interactions with regulatory authorities and expectations regarding regulatory filings and possible approvals for MOR210/TJ210as well as the potential future commercializationof MOR210/TJ210. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "would," "could," "potential," "possible," "hope" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The forward-looking statements contained herein represent the judgment of MorphoSys as of the date of this release and involve known and unknown risks and uncertainties, which might cause the actual results, financial condition and liquidity, performance or achievements of MorphoSys, or industry results, to be materially different from any historic or future results, financial conditions and liquidity, performance or achievements expressed or implied by such forward-looking statements. In addition, even if MorphoSys' results, performance, financial condition and liquidity, and the development of the industry in which it operates are consistent with such forward-looking statements, they may not be predictive of results or developments in future periods. Among the factors that may result in differences are MorphoSys' expectations regarding risks and uncertainties related to the impact of the COVID-19 pandemic to MorphoSys' business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products, the global collaboration and license agreement for MOR210/TJ210, the further clinical development of MOR210/TJ210, and MorphoSys' ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials, additional interactions with regulatory authorities and expectations regarding future regulatory filings , MorphoSys' reliance on collaborations with third parties, estimating the commercial potential of its development programs and other risks indicated in the risk factors included in MorphoSys' Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. MorphoSys expressly disclaims any obligation to update any such forward-looking statements in this document to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements, unless specifically required by law or regulation.

I-Mab Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding data from the TJ210/MOR210 Phase 1 trial of advanced cancers, the potential implications of clinical data for patients, and I-Mab's advancement of, and anticipated clinical development, regulatory milestones and commercialization of TJ210/MOR210. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to I-Mab's ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or NDA/BLA approval; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of I-Mab's drug candidates; I-Mab's ability to achieve commercial success for its drug candidates, if approved; I-Mab's ability to obtain and maintain protection of intellectual property for its technology and drugs; I-Mab's reliance on third parties to conduct drug development, manufacturing and other services; I-Mab's limited operating history and I-Mab's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates; and the impact of the COVID-19 pandemic on the Company's clinical development, commercial and other operations, as well as those risks more fully discussed in the "Risk Factors" section in I-Mab's most recent annual report on Form 20-F, as well as discussions of potential risks, uncertainties, and other important factors in I-Mab's subsequent filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to I-Mab, and I-Mab undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

For more information, please contact:

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SOURCE I-Mab

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MorphoSys and I-Mab Announce FDA Clearance of IND Application for MOR210/TJ210 in Patients with Advanced Cancer - BioSpace

Codiak Initiates Subject Dosing in a Phase 1 Clinical Trial of exoIL-12 for the Treatment of Cancer – Yahoo Finance

Posted: September 16, 2020 at 1:51 am


- exoIL-12, engineered with Codiaks proprietary engEx Platform, is Codiaks first program to enter clinical development

- exoIL-12 is designed to allow optimal dosing of IL-12 and overcome tolerability challenges by localizing IL-12 in the tumor microenvironment

Codiak BioSciences, Inc., a clinical-stage company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, today announced the initiation of subject dosing in its Phase 1 clinical trial of its novel exosome therapeutic candidate, exoIL-12. Engineered using Codiaks proprietary engEx Platform and designed to display IL-12 on the exosome surface, exoIL-12 is designed to enhance dose control of IL-12 and limit systemic exposure and associated toxicity by localizing IL-12 in the tumor microenvironment (TME). The trial will evaluate single ascending doses (SAD) of exoIL-12 in healthy volunteers and then transition to patients with early stage cutaneous T cell lymphoma (CTCL) with repeat dosing of pharmacologically active doses identified in the healthy volunteer SAD study. The trial is Codiaks first human clinical trial and the first of two clinical development programs Codiak expects to initiate in 2020.

"To our knowledge, exoIL-12 is the first engineered exosome to enter clinical development, which makes the initiation of this trial a true milestone not only for Codiak but for the entire exosome therapeutics field," said Douglas E. Williams, Ph.D., CEO, Codiak. "Our engEx platform allows us to engineer exosomes to selectively deliver potent therapeutic payloads, such as IL-12, to potentially enhance the therapeutic index. We believe that exoIL-12 may unlock the well-documented therapeutic potential of this cytokine by retaining its activity within the tumor and reducing systemic exposure and the adverse events seen in the past with other formulations of IL-12."

Codiak is initially focusing development of exoIL-12 on tumors that have previously shown clinical responses to IL-12 used as a monotherapy, such as CTCL. While the biological rationale for IL-12 as a cancer treatment has been validated in previous human clinical studies, its utility has been severely limited due to serious adverse events caused by systemic exposure.

Codiak has engineered exoIL-12 to display fully active IL-12 on the surface of the exosome, which is designed to facilitate potent local pharmacology at the tumor injection site with precisely quantified doses. Exosomal delivery has demonstrated limited systemic exposure to IL-12 in preclinical models and resulted in significant and prolonged pharmacodynamic activity and both local and systemic anti-tumor immune responses.

The Phase 1 clinical trial, which is being conducted at the Phase 1 unit Richmond Pharmacology LTD, London, UK, is designed to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of exoIL-12 following single ascending subcutaneous doses in healthy volunteers, followed by repeat dose exoIL-12 into the lesions of stage IA-IIB CTCL patients. Patients with CTCL will be monitored for safety, pharmacokinetics, pharmacodynamic effects in blood and tumor biopsies, and local and systemic anti-tumor efficacy using validated CTCL assessment criteria. Preliminary results from healthy volunteers are anticipated by the end of 2020 and safety, biomarker and preliminary efficacy results from CTCL patients are anticipated in mid-2021.

About exoIL-12

exoIL-12 is Codiaks exosome therapeutic candidate engineered to display fully active IL-12 on the surface of the exosome, using the exosomal protein, PTGFRN, as a scaffold protein, and designed to facilitate potent local pharmacology at the injection site with precisely quantified doses. By limiting systemic exposure of IL-12 and associated toxicity, Codiak hopes to enhance the therapeutic index with exoIL-12, delivering a more robust tumor response, dose control and an improved safety profile.

Codiak intends to focus development of exoIL-12 on tumors that have, in previous clinical testing, shown clinical responses to IL-12 used as a monotherapy. This includes cutaneous T cell lymphoma (CTCL), melanoma, Merkel cell carcinoma, Kaposi sarcoma, glioblastoma multiforme and triple negative breast cancer.

Story continues

About the engEx Platform

Codiaks proprietary engEx Platform is designed to enable the development of engineered exosome therapeutics for a wide spectrum of diseases and to manufacture them reproducibly and at scale to pharmaceutical standards. By leveraging the inherent biology, function and tolerability profile of exosomes, Codiak is developing engEx exosomes designed to carry and protect potent drug molecules, provide selective delivery and elicit the desired pharmacology at the desired tissue and cellular sites. Through its engEx Platform, Codiak seeks to direct tropism and distribution by engineering exosomes to carry on their surface specific targeting drug moieties, such as proteins, antibodies/fragments, and peptides, individually or in combination. Codiak scientists have identified two exosomal proteins that serve as surface and luminal scaffolds. By engineering the exosome surface or lumen and optimizing the route of administration, Codiak aims to deliver engEx exosomes to the desired cell and tissue to more selectively engage the drug target, potentially enhancing the therapeutic index by improving potency and reducing toxicity.

About Codiak BioSciences

Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of diseases, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease. For more information, visit http://www.codiakbio.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200915005378/en/

Contacts

Lindy DevereuxScient PRT: 646-515-5730E: media@codiakbio.com

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Codiak Initiates Subject Dosing in a Phase 1 Clinical Trial of exoIL-12 for the Treatment of Cancer - Yahoo Finance

Apellis Announces Submission of Pegcetacoplan Marketing Applications to FDA and EMA for Patients with PNH – BioSpace

Posted: at 1:51 am


WALTHAM, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States and European Union, respectively.

Pegcetacoplan has demonstrated its potential to elevate the standard of care in PNH and the submissions of the U.S. and EU marketing applications represent an important step in our efforts to bring the first targeted C3 therapy to people with PNH as quickly as possible, said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. Building on our progress with the FDA and EMA, we are also proud that the TGA has granted pegcetacoplan orphan drug designation for the treatment of patients with PNH in Australia.

The NDA and MAA submissions are based on results from the Phase 3 PEGASUS study, which met its primary endpoint, demonstrating the superiority of pegcetacoplan to eculizumab with a statistically significant improvement in hemoglobin levels at 16 weeks, as well as higher normalization rates across key markers of hemolysis and clinically meaningful improvement in FACIT-fatigue score. The safety profile of pegcetacoplan was comparable to eculizumab in the study. The FDA and EMA decisions on acceptance of the NDA and MAA submissions are expected in Q4 2020.

In another regulatory milestone, the Australian Therapeutic Goods Administration (TGA) granted orphan drug designation to pegcetacoplan in PNH. This designation in Australia is granted to therapies for serious rare diseases that have the potential to provide a significant benefit in comparison to approved treatments. Apellis plans to submit a marketing application in Australia in the fourth quarter of 2020.

About Pegcetacoplan (APL-2)Pegcetacoplan is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea). Retrospective studies show that, even on eculizumab, approximately 70% of people with PNH have low hemoglobin levels,1,2 and 36% require one or more transfusions a year.2

About ApellisApellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

Apellis Forward-Looking StatementStatements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the implications of preliminary clinical data. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether the companys clinical trials will be fully enrolled and completed when anticipated; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the companys clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G or any other indication when expected or at all; whether, if Apellis products receive approval, they will be successfully distributed and marketed; and other factors discussed in the Risk Factors section of Apellis Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on July 30, 2020 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Media Contact:Mark Dolemedia@apellis.com617.420.4839

Investor Contact: Sam Martin / Maghan MeyersArgot Partnerssam@argotpartners.com / maghan@argotpartners.com 212.600.1902____________________________________1. Risitano AM, Notaro R, Marando L, et al. (2009) Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood. 2009 Apr 23;113(17):4094-100.2. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

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Apellis Announces Submission of Pegcetacoplan Marketing Applications to FDA and EMA for Patients with PNH - BioSpace

Innovent and Lilly Jointly Announce Results of Six Clinical Studies of TYVYT (sintilimab injection) to be Presented at the European Society for…

Posted: September 14, 2020 at 5:53 pm


SAN FRANCISCO and SUZHOU, China, Sept. 14, 2020 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for treatment of oncology, metabolic, autoimmune and other major diseases, and Eli Lilly and Company ("Lilly", NYSE: LLY) jointly announce that results of six clinical studies of TYVYT (sintilimab injection) will be presented during the upcoming European Society of Medical Oncology ("ESMO") Virtual Congress 2020 from September 19th to 21st. The annual ESMO conference is among the most prestigious and influential global oncology conferences, during which oncologists around the world will share the latest research progress in cancer treatments.

The six sintilimab studies to be presented at ESMO Virtual Congress include two LBAs (late-breaking abstracts, mini oral) and four e-posters (including two ongoing Phase 3 studies). The studies cover indications including lung cancer, gynecological cancer, hepatocellular carcinoma, gastric cancer, and other solid tumors. A brief summary of the studies is as follows:

Cancer Type: Lung Cancer

Presentation type: LBA (mini oral)

Biomarker Results from the ORIENT-11 Study (NCT 03607539): Finding biomarkers to accurately predict the efficacy of Immuno-combination therapy is still a hotspot and difficult issue in the study of PD-1 inhibitors. In the ORIENT-11 study, sequencing was conducted on baseline tumor biopsies to explore the association between immune related genes and clinical efficacy. The results could improve our understanding of the mechanism of action of immunotherapy-chemotherapy combination and provide a scientific rationale for future selection of suitable patients.

Researcher: Professor Yunpeng Yang, Sun Yat sen University cancer center

Results of the ORIENT-12 Study (NCT03629925): sintilimab plus gemcitabine and platinum chemotherapy as first-line treatment for locally advanced or metastatic squamous non-small-cell lung cancer (sqNSCLC). Clinical benefit from platinum-based chemotherapy for patients with advanced sqNSCLC is limited. Previous studies have shown the clinical benefits of the combination therapy of PD-1 inhibitor with paclitaxel/platinum chemotherapy as first-line treatment for sqNSCLC. In a Phase 1b cohort study, sintilimab in combination with gemcitabine/platinum chemotherapy has shown good efficacy and acceptable safety as first-line treatment for sqNSCLC. ORIENT-12 is a randomized, double-blind, Phase 3 study evaluating sintilimab or placebo in combination with gemcitabine and platinum chemotherapy as first-line treatment for locally advanced or metastatic sqNSCLC. ORIENT-12 has demonstrated for the first time survival benefit by treatment with PD-1 inhibitor in combination with gemcitabine and platinum chemotherapy in first-line sqNSCLC.

Researcher: Professor Caicun Zhou, Shanghai Pulmonary Hospital, Tongji University

Cancer Type: Hepatocellular carcinoma (HCC)

Presentation Type: e-poster

Sintilimab plus IBI305 (bevacizumab) as the first-line treatment for advanced HCC (NCT03794440). So far the treatment of first-line advanced HCC is limited with feasible choices such as sorafenib or lenvatinib. Immuno-oncology inhibitors have shown therapeutic value in HCC, with PD-L1 inhibitor (atezolizumab) in combination with a VEGF inhibitor reporting clinical benefits in unresectable or metastatic HCC patients before systemic treatment. This study will announce the safety and preliminary efficacy of combining PD-1 inhibitor and VEGF inhibitor in the first line treatment for patients with advanced unresectable or metastatic HCC. Currently sintilimab is undergoing Phase 2/3 study in combination with Byvasda (bevacizumab injection) in comparison with sorafenib in the first-line treatment of advanced HCC.

Researcher: Academician Jia Fan, Zhongshan Hospital, Fudan University

Cancer type: Gastric Cancer

Report type: e-poster

ORIENT-106 Study: To date, systemic chemotherapy remains the main choice for unresectable locally advanced or metastatic gastric cancer / gastroesophageal junction adenocarcinoma (G/GEJ). The prognosis of these patients is poor with the median overall survival (mOS) only about one year. Preclinical studies have shown that an anti-VEGF receptor 2 (VEGFR-2) antibody can restart the tumor microenvironment to avoid immunosuppression of tumor cells. In clinical studies, it was also observed that blocking PD-1 and VEGFR-2 at the same time could achieve synergistic anti-tumor effect. The ORIENT-106 study based on this theory is a multicenter, randomized, open label Phase 3 clinical trial to verify the efficacy and safety of sintilimab (IgG4 PD-1 inhibitor) and ramucirumab (IgG1 VEGFR-2 antagonist) as the first-line treatment for locally advanced or metastatic G/GEJ.

Researcher: President Ruihua Xu, Sun Yat sen University Cancer Center

Cancer type: Gynecological Tumor

Report type: e-poster

There are limited effective treatment for advanced cervical cancer patients who have previously received platinum-based chemotherapy. PD-1 inhibitor monotherapy has shown promising efficacy in patients with cervical cancer with positive PD-L1 expression. The combination of PD-1/PD-L1 inhibitors plus anti-angiogenesis drugs has shown significant anti-tumor activity in certain cancers. Professor Qin Xu from Fujian Cancer Hospital conducted a phase II study of sintilimab plus anlotinib for the treatment of advanced cervical cancer with positive PD-L1 expression. The study may potentially further improve the clinical outcomes of patients with advanced cervical cancer who have previously received platinum-based chemotherapy.

Researcher: Professor Qin Xu, Fujian Cancer Hospital

Cancer type: Solid Tumors

Report type: e-poster

The antitumor effect of chemotherapy combined with either PARP inhibitors or PD-1 inhibitors have been demonstrated in several studies, and previous researches have shown a synergetic effect of PARP inhibitors combining with PD-1 inhibitors. However, little was known regarding the combination of the three regimens. This is a phase 1b clinical study initiated by Professor Hu Yi of the Chinese PLA General Hospital, exploring the combination of sintilimab, platinum and niraparib (a PARP1/2 inhibitor) in the treatment of previously treated advanced solid tumors. The novel triple combination could potentially overcome resistance and further improve clinical outcomes of patients with advanced solid tumors who failed standard therapy.

Researcher: Professor Yi Hu, Chinese people's Liberation Army General Hospital

About Innovent

Inspired by the spirit of "Start with Integrity, Succeed through Action," Innovent's mission is to develop 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, metabolic, autoimmune 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 3 products, TYVYT (sintilimab injection), BYVASDA (bevacizumab injection) and SULINNO (adalimumab injection), on market, 1 asset under NDA review with priority review status, 4 assets in Phase III or pivotal clinical trials, and additional 15 molecules in or close to clinical trials. TYVYT (sintilimab injection) has been the only PD-1 inhibitor included in the NRDL since 2019.

Innovent has built an international team with expertise in cutting-edge biological drug development and commercialization. The company has also entered into strategic collaborations with Eli Lilly, Adimab, Incyte, Alector, MD Anderson Cancer Center, Hanmi and other international partners. For more information, please visit: http://www.innoventbio.com.

About Eli Lilly and Company

Lilly is a global healthcare leader that unites caring with discovery to create medicines to make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us at http://www.lilly.com.

About Innovent Biologic's strategic cooperation with Eli Lilly and Company

Innovent entered into a strategic collaboration with Lilly focusing on biological medicine in March 2015 a groundbreaking partnership between a Chinese pharmaceutical company and a multinational pharmaceutical company. Under the agreement, Innovent and Lilly are co-developing and commercializing oncology medicines, including TYVYT (sintilimab injection) in China. In October 2015, the two companies announced the extension of their existing collaboration to include co-development of three additional antibodies targeting oncology indications. In August 2019, Innovent entered into an additional licensing agreement with Lilly to develop and commercialize a potentially global best-in-class diabetes medicine in China. In August 2020, Innovent and Lilly announced an expansion of their strategic alliance for TYVYT (sintilimab injection). Its collaboration with Lilly indicates that Innovent has established a comprehensive level of cooperation between China's innovative pharmaceuticals sector and the international pharmaceuticals sector in areas such as R&D, CMC, clinical development and commercialization.

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SOURCE Innovent Biologics, Inc.

Company Codes: HongKong:1801, OTC-PINK:IVBIY, NYSE:LLY

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Innovent and Lilly Jointly Announce Results of Six Clinical Studies of TYVYT (sintilimab injection) to be Presented at the European Society for...

Vaxart Announces FDA Clearance of IND Application for Oral COVID-19 Vaccine and Provides Update on COVID-19 Program – GlobeNewswire

Posted: at 5:53 pm


Recruitment for Phase 1 clinical study expected to start this month

Data from ongoing hamster challenge study expected in October

SOUTH SAN FRANCISCO, Calif., Sept. 14, 2020 (GLOBE NEWSWIRE) -- Vaxart, Inc., a clinical-stage biotechnology company developing oral vaccines that are administered by tablet rather than by injection, today announced that the U.S. Food and Drug Administration (FDA) has completed its review of the Companys Investigational New Drug (IND) application for its Phase 1 clinical trial evaluating its oral COVID-19 vaccine candidate. The Company also provided an update on its COVID-19 program.

Our goal is to deliver the best, most elegant solution for conferring mass protection against COVID-19. Our oral tablet vaccine offers a much more attractive mode of administration than injectables and may confer superior protection against COVID-19 due to activation of mucosal immunity. Importantly, our room-temperature stable tablet is significantly easier and cheaper to store and distribute to the farthest corners of the US and the globe, as it does not require the very costly and complex refrigerated cold chain needed for injectable vaccines., said Andrei Floroiu, chief executive officer of Vaxart. The IND clearance and the initiation of our Phase 1 clinical trial moves us a step closer to proving the superiority of our convenient oral COVID-19 solution in the clinic. We are thus excited to start enrollment for our Phase 1 this month.

COVID-19 Program Updates:

In addition, and as previously disclosed, Vaxart is awaiting results from a non-human primate (NHP) challenge study that is testing its vaccine in a harmonized protocol as part of Operation Warp Speed. This preclinical program is being conducted in collaboration with the Biomedical Advanced Research and Development Authority (BARDA) and other entities working with Operation Warp Speed.

Sean Tucker Ph.D., chief scientific officer added, In addition to our clinical program progress, we have a hamster challenge study underway with our COVID-19 vaccine candidate to assess the potential contribution of inducing mucosal immunity to overall efficacy. We are also awaiting the results from a non-human primate study. Both hamsters and monkeys are susceptible to SARS-CoV-2 infection and these models may be capable of providing a deeper understanding of the immune responses and correlates of protection elicited by our oral vaccine candidate.

About VaxartVaxart is a clinical-stage biotechnology company developing a range of oral recombinant vaccines based on its proprietary delivery platform. Vaxart vaccines are administered using convenient room temperature-stable tablets that can be stored and shipped without refrigeration and eliminate the risk of needle-stick injury. Vaxart has demonstrated 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. Vaxart has filed broad domestic and international patents covering its proprietary technology and creations for oral vaccination using adenovirus and TLR3 agonists.

Note Regarding Forward-Looking StatementsThis 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 patients 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 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. Vaxart may 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 that Vaxart makes, 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 a Vaxart collaborator may not attain development and commercial milestones; that Vaxart or 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; that Vaxart may 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 to Vaxart, if at all; the impact of government healthcare proposals and policies; competitive factors; that if we fail to comply with the policies, rules and regulations governing Operation Warp Speed, or do not ultimately receive funding or complete our planned non-human primate challenge study for any other reason, our business and operations would be materially and adversely impacted; and other risks described in the Risk Factors sections of Vaxarts Quarterly and Annual Reports filed with the SEC. Vaxart does not assume any obligation to update any forward-looking statements, except as required by law.

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Vaxart Announces FDA Clearance of IND Application for Oral COVID-19 Vaccine and Provides Update on COVID-19 Program - GlobeNewswire

Datacubed Health Raises Capital to Create the Future of Patient Engagement – Business Wire

Posted: at 5:53 pm


NEW YORK--(BUSINESS WIRE)--Datacubed Health (Datacubed), a healthcare technology platform that provides electronic clinical outcomes assessments (eCOA) for patients participating in clinical trials, announced today an investment from Sterling Partners (Sterling), an investment firm focused on transformational growth. Datacubeds eCOA SaaS platform is driven by behavioral science and creates frictionless experiences for patients and seamless data collection throughout a clinical study.

As the company continues to grow and work with new partners, including leading pharmaceutical companies, chief research officers, health systems, and insurance companies, Sterlings capital will be deployed to invest in talent development, increase operational efficiencies, and expand to adjacent markets. Sterling has nearly four decades of experience building high-growth, tech-enabled businesses and healthcare organizations. Sterling currently manages over $1.5 billion and has invested over $500 million dollars in the healthcare and life sciences sectors.

With Sterling's investment, Datacubed has now raised nearly $30 million from its founders, angel investors, and Sterling.

COVID-19 Creates Critical Market Opportunity for eCOA

Clinical trials are incredibly complex, often involving thousands of participants, costing tens of millions of dollars, and lasting a decade or more. These critical studies still rely on outdated forms of patient communication and tracking that create friction and data gaps throughout the trial process, resulting in errors and delays in bringing potentially life-saving drugs to market.

Datacubeds disruptive technology platform increases compliance rates from an average of 60-70% to over 85%, which significantly decreases trial costs and brings products to market earlier. Datacubed allows clinical trial study teams to set up trials within minutes and greatly facilitates engagement with trial participants. As one of the industrys leading SaaS solutions coupled with its foundation in behavioral science, Datacubed accelerates the time-to-market for new trials while improving patient engagement, compliance, and retention.

Most trial-related processes are inefficient and require extensive face-to-face time from patients, clinicians, and trial managers, said Brett Kleger, CEO of Datacubed. COVID-19 has completely uprooted the old way of conducting clinical trials. Datacubeds technology meets todays clinical research needs by making the entire process easier for patients and materially improving retention through behavioral science to better understand the human condition.

A Proven Platform Driven by Behavioral Science

Datacubed is led by CEO Kleger and Founder and Chairman of the Board Dr. Paul Glimcher, who also serves as Chief Scientific Officer. Kleger has over 20 years as an operational, commercial, and product executive building and growing both early-stage and established large-scale life sciences technology companies, including DrugDev, IQVIA, IMS Health, and Acurian. Dr. Paul Glimcher has been ranked a top 25 behavioral economist in the world. He is one of the foremost researchers focused on the study of human behavior and decision-making, and is known for his central role in founding and developing the field of neuroeconomics, which takes an interdisciplinary approach to understanding how humans make decisions. Together with their team, Kleger and Dr. Glimcher are focused on reducing the risk that poor data collection or lack of patient adherence to the trials protocols prevents or delays a drug coming to market.

Were excited to partner with Datacubed and work with its leadership team and employees to be the preeminent provider of patient engagement in the clinical trial market. In the health space, Datacubed is a game-changer and we look forward to helping broaden the platform to other parts of the drug commercialization process, said Steven Taslitz, Co-founder and Chairman of Sterling Partners. Broadly improving patient engagement and data collection plays a critical role in accelerating the discovery of new treatments.

About Datacubed: Datacubed Health (Datacubed) is a pioneering technology company making better science and healthier communities a reality. Datacubed applies individualized solutions for the capture of data, including smartphone apps, wearable, in-home, and environmental sensors, for remote engagement with patients and for virtual clinical studies. Focusing on healthcare and life sciences, they offer software and services driven by behavioral science to improve patient retention and compliance, resulting in better data and positive health outcomes. Datacubed believes that the best way to unlock the insights that have eluded scientists is understanding the holistic life of an individual. For more information, please visit https://www.datacubed.com/.

About Sterling Partners: Sterling Partners (Sterling) is a diversified investment management platform founded in 1983 and based in Chicago. The firm started with four young entrepreneurs, who went on to build one of Chicagos most prominent private equity firms. Today, the firm has expanded beyond its strong private equity practice into several other investment strategies. Complementing its institutional fund practice, Sterling invests in a wide variety of companies in various stages of growth - from early stage, high-growth businesses to mature, profitable companies on a deal-by-deal basis. These investments cross several industries, and the firm makes control, non-control, and preferred equity investments. Sterling adds value to its portfolio companies and the founders with whom it partners with its entrepreneurial roots, deep domain expertise, focus on transformational growth, and access to world-class executive talent. For more information, please visit http://www.sterlingpartners.com.

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Datacubed Health Raises Capital to Create the Future of Patient Engagement - Business Wire

Ascentage Pharma’s MDM2-p53 Inhibitor APG-115 Granted Orphan Drug Designation by the FDA for the Treatment of Gastric Cancer – BioSpace

Posted: at 5:53 pm


SUZHOU, China and ROCKVILLE, Md., Sept. 14, 2020 /PRNewswire/ -- Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the US Food and Drug Administration (FDA) has granted APG-115, a novel MDM2-p53 inhibitor being developed by the company, an Orphan Drug Designation (ODD) for the treatment of gastric cancer (GC). This is the first ODD granted for APG-115.

The term "orphan drugs" refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions. In the United States, an orphan disease is defined as a disease or condition with a prevalence of less than 200,000 patients in the country. Since the Orphan Drug Act was passed in 1983, the US government has provided incentives and policy support to encourage development of orphan drugs. This ODD from the FDA qualifies APG-115 for various development incentives, including a tax credit on expenditures incurred in clinical studies, a waiver of the New Drug Application (NDA) fee, research grant awarded by the FDA, and most importantly, 7 years of US market exclusivity upon approval for the treatment of GC.

The global incidence of GC is significantly different between eastern and western countries. According to the National Cancer Institute's Surveillance, Epidemiology, and End Results Program, in 2017, there were an estimated 116,525 people living with GC in the US1. GC is currently considered as a rare disease in US. Asian countries such as China and Japan have a much higher incidence. GC is the third leading cause of cancer deaths worldwide, followed only by lung and colorectal cancer in overall mortality. About 1 in 12 of all oncological deaths are attributable to GC2.

The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for GC recommends a multidisciplinary approach for treatment of unresectable, advanced, metastatic GC patients. For patients with disease progression receiving second-line therapy, there are not many treatment options and the prognosis remains poor. Thus, effective treatment options are urgently needed3 to improve the disease outcome and reduce the mortality.

APG-115 is an orally administered, selective, small-molecule inhibitor of the MDM2-p53 protein-protein interaction (PPI). APG-115 has strong binding affinity to MDM2 and is designed to activate tumor suppression activity of p53 by blocking the MDM2-p53 PPI. APG-115 is the first MDM2-p53 inhibitor entering clinical development in China, with multiple ongoing clinical studies in solid tumors and hematologic malignancies in China and the US. APG-115 has shown promising results in preclinical studies for the treatment of GC.

"At present, the treatment of GC represents an urgent unmet clinical need globally. This ODD by the US FDA for the treatment of GC marks an important milestone in the global development and commercialization of APG-115," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "All the policy support and incentives as a result of this ODD will help us accelerate the global clinical development of APG-115, which we hope will soon transform to benefit more patients."

References:

1. 2020 Cancer Incidence Data, Surveillance, Epidemiology, and End Results Program, National Cancer Institute

2. Rawla, P., & Barsouk, A. (2019). Epidemiology of gastric cancer: global trends, risk factors and prevention.

3. Gastric Cancer, NCCN Clinical Practice Guidelines in Oncology, National Comprehensive Cancer Nerwork 2020.

About APG-115

APG-115 is an orally administered, selective, small-molecule inhibitor of the MDM2-p53 PPI. APG-115 has strong binding affinity to MDM2 and is designed to activate p53 tumor suppression activity by blocking the MDM2-p53 PPI. Ascentage Pharma has previously commenced three clinical trials of APG-115 in the US, including a Phase I study as single agent, a Phase Ib/II study in combination with pembrolizumab for treatment of metastatic melanoma and other advanced solid tumors, and a Phase I/II study as a single agent or in combination with chemotherapy for treatment of salivary gland cancer. APG-115 is the first MDM2-p53 inhibitor to enter clinical studies in China. A Phase I study as a single agent, and a Phase Ib study as a single agent or in combination with chemotherapy for treatment of AML (acute myeloid leukemia) or MDS (myelodysplastic syndrome) are ongoing in China.

About Ascentage Pharma

Ascentage Pharma (6855.HK) is a globally, clinical-stage biotechnology company engaged in developing novel therapies for cancers, CHB, and senesce diseases. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 6855.HK.

Ascentage Pharma focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis, or programmed cell death. The company has built a pipeline of eight clinical drug candidates, including novel, highly potent Bcl-2, and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation tyrosine kinase inhibitors. Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company is conducting more than 30 Phase I/II clinical trials in the US, Australia, and China. The company's core drug candidate HQP1351 was recently granted orphan drug and fast-track designations by the US Food and Drug Administration (FDA), and a New Drug Application for HQP1351 has been submitted in China. APG-2575, another key drug candidate of the company, was recently granted orphan drug designation by the FDA.

Forward-Looking Statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events, or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

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SOURCE Ascentage Pharma

Company Codes: HongKong:6855

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Ascentage Pharma's MDM2-p53 Inhibitor APG-115 Granted Orphan Drug Designation by the FDA for the Treatment of Gastric Cancer - BioSpace

Baricitinib in Combination with Remdesivir Reduces Time to Recovery in Hospitalized Patients with COVID-19 in NIAID-Sponsored ACTT-2 Trial – BioSpace

Posted: at 5:53 pm


INDIANAPOLIS, Sept. 14, 2020 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ: INCY) announced today initial data emerging from the Adaptive COVID-19 Treatment Trial (ACTT-2) sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH). ACTT-2 included more than 1,000 patients and began on May 8 to assess the efficacy and safety of a 4-mg dose of baricitinib plus remdesivir versus remdesivir in hospitalized patients with COVID-19. Baricitinib in combination with remdesivir met the primary endpoint of reduction of time to recovery in comparison with remdesivir.

Study investigators noted an approximately one-day reduction in median recovery time for the overall patient population treated with baricitinib in combination with remdesivir versus those treated with remdesivir. This finding was statistically significant. Recovery was defined as the participant being well enough for hospital discharge, meaning the participant either no longer required supplemental oxygen or ongoing medical care in the hospital, or was no longer hospitalized at Day 29. The study also met a key secondary endpoint comparing patient outcomes at Day 15 using an ordinal 8-point scale ranging from fully recovered to death.

An independent data and safety monitoring board overseeing the double-blind, randomized controlled trial met regularly throughout the trial to review safety data. Additional analyses are ongoing to understand other clinical outcome data, including mortality and safety data. NIAID is expected to publish full details of the study in a peer-reviewed journal.

"We are pleased with these data from the ACTT-2 study," said Patrik Jonsson, Lilly senior vice president and president of Lilly Bio-Medicines. "There is an urgent need to identify COVID-19 treatments, and we will continue to work with NIAID to understand these data and next steps on baricitinib's role moving forward. We appreciate NIAID selecting baricitinib for inclusion in this important study and the participants, investigators and collaborators for the vital roles they played."

"These findings from ACTT-2 are another step as we improve the care of these patients," said Andre Kalil, M.D., professor at the University of Nebraska Medical Center and a principal investigator of the ACTT studies. "These data may help us to better understand baricitinib's potential role in the treatment of COVID-19."

Based on the ACTT-2 data, Lilly plans to discuss the potential for emergency use authorization (EUA) with the U.S. Food and Drug Administration (FDA) and to explore similar measures with other regulatory agencies for baricitinib as a treatment of hospitalized patients with COVID-19. If authorized for use, Lilly will propose that baricitinib be available through commercial channels and will work with hospitals and governments to ensure patient access. Lilly will continue to create adequate supply for rheumatoid arthritis (RA) patients and ensure baricitinib remains available in countries where it is approved. In the U.S., baricitinib is approved for RA patients at a 2-mg daily dose; an EUA would potentially authorize a 4-mg dose for COVID-19.

Lilly will review the ACTT-2 data with NIAID and assess any impact on COV-BARRIER, the Phase 3 randomized, double-blind, placebo-controlled study it initiated in June to evaluate the efficacy and safety of baricitinib versus background therapy in hospitalized adults with COVID-19 in the U.S., Europe, Asia and Latin America.

"As a company, we've moved quickly to develop and evaluate medicines for patients for the prevention and treatment of COVID-19," said Daniel Skovronsky, M.D., Ph.D., Lilly senior vice president and chief scientific officer. "These data allow us to better understand baricitinib's role in potentially improving outcomes for hospitalized COVID-19 patients, and we look forward to continuing this research alongside our other initiatives to combat COVID-19."

Baricitinib, a JAK1/JAK2 inhibitor licensed to Lilly from Incyte and marketed as OLUMIANT, is approved in more than 70 countries as a treatment for adults with moderately to severely active RA. Studying baricitinib in controlled trials is important in order to better characterize its potential benefits and understand the safety of its use as a COVID-19 treatment. The U.S. prescribing information for the approved use of baricitinib for RA includes boxed warnings regarding the use of baricitinib, including warnings about risk for developing blood clots and serious infections.

Lilly is also currently supporting ongoing multisite and single-site investigator-initiated trials in Europe and North America for hospitalized patients with COVID-19 infections.

About Lilly's COVID-19 Efforts

Lilly is bringing the full force of its scientific and medical expertise to attack the coronavirus pandemic around the world. Existing Lilly medicines are now being studied to understand their potential in treating complications of COVID-19, and the company is collaborating with two partner companies to discover novel antibody treatments for COVID-19. Lilly intends to test both single antibody therapy as well as combinations of antibodies (sometimes known as antibody cocktails) as potential therapeutics for COVID-19. Click here for media resources related to Lilly's COVID-19 efforts.

Indication and Usage for OLUMIANT (baricitinib) tablets (in the United States) for RA patients

OLUMIANT (baricitinib) 2-mg is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more tumor necrosis factor (TNF) antagonist therapies. Limitation of Use: Not recommended for use in combination with other JAK inhibitors, biologic disease-modifying antirheumatic drugs (DMARDs), or with potent immunosuppressants such as azathioprine and cyclosporine.

IMPORTANT SAFETY INFORMATION FOR OLUMIANT (baricitinib) TABLETS

WARNING: SERIOUS INFECTIONS, MALIGNANCY, AND THROMBOSIS

SERIOUS INFECTIONS: Patients treated with Olumiant are at risk for developing serious infections that may lead to hospitalization or death. Most patients who developed these infections were taking concomitant immunosuppressants such as methotrexate or corticosteroids. If a serious infection develops, interrupt Olumiant until the infection is controlled. Reported infections include:

Carefully consider the risks and benefits of Olumiant prior to initiating therapy in patients with chronic or recurrent infection.

Closely monitor patients for the development of signs and symptoms of infection during and after treatment with Olumiant including the possible development of TB in patients who tested negative for latent TB infection prior to initiating therapy.

MALIGNANCIES: Lymphoma and other malignancies have been observed in patients treated with Olumiant.

THROMBOSIS: Thrombosis, including deep venous thrombosis (DVT) and pulmonary embolism (PE), has been observed at an increased incidence in patients treated with Olumiant compared to placebo. In addition, there were cases of arterial thrombosis. Many of these adverse events were serious and some resulted in death. Patients with symptoms of thrombosis should be promptly evaluated.

WARNINGS AND PRECAUTIONS

SERIOUS INFECTIONS: The most common serious infections reported with Olumiant included pneumonia, herpes zoster and urinary tract infection. Among opportunistic infections, tuberculosis, multidermatomal herpes zoster, esophageal candidiasis, pneumocystosis, acute histoplasmosis, cryptococcosis, cytomegalovirus and BK virus were reported with Olumiant. Some patients have presented with disseminated rather than local disease and were often taking concomitant immunosuppressants such as methotrexate or corticosteroids. Avoid Olumiant in patients with an active, serious infection, including localized infections. Consider the risks and benefits of treatment prior to initiating Olumiant in patients:

Closely monitor patients for infections during and after Olumiant treatment. Interrupt Olumiant if a patient develops a serious infection, an opportunistic infection, or sepsis. Do not resume Olumiant until the infection is controlled.

Tuberculosis Before initiating Olumiant evaluate and test patients for latent or active infection and treat patients with latent TB with standard antimycobacterial therapy. Olumiant should not be given to patients with active TB. Consider anti-TB therapy prior to initiating Olumiant in patients with a history of latent or active TB in whom an adequate course of treatment cannot be confirmed, and for patients with a negative test for latent TB but who have risk factors for TB infection. Monitor patients for TB during Olumiant treatment.

Viral Reactivation Viral reactivation, including cases of herpes virus reactivation (e.g., herpes zoster), were reported in clinical studies with Olumiant. If a patient develops herpes zoster, interrupt Olumiant treatment until the episode resolves.

The impact of Olumiant on chronic viral hepatitis reactivation is unknown. Screen for viral hepatitis in accordance with clinical guidelines before initiating Olumiant.

MALIGNANCY AND LYMPHOPROLIFERATIVE DISORDERS: Malignancies were observed in Olumiant clinical studies. Consider the risks and benefits of Olumiant prior to initiating therapy in patients with a known malignancy other than a successfully treated non-melanoma skin cancer (NMSC) or when considering continuing Olumiant in patients who develop a malignancy. NMSCs were reported in patients treated with Olumiant. Periodic skin examination is recommended for patients who are at increased risk for skin cancer.

THROMBOSIS: Thrombosis, including DVT and PE, has been observed at an increased incidence in Olumiant-treated patients compared to placebo. In addition, arterial thrombosis events in the extremities have been reported in clinical studies with Olumiant. Many of these adverse events were serious and some resulted in death. There was no clear relationship between platelet count elevations and thrombotic events. Use Olumiant with caution in patients who may be at increased risk of thrombosis. If clinical features of DVT/PE or arterial thrombosis occur, evaluate patients promptly and treat appropriately.

GASTROINTESTINAL PERFORATIONS: Gastrointestinal perforations have been reported in Olumiant clinical studies, although the role of JAK inhibition in these events is not known. Use Olumiant with caution in patients who may be at increased risk for gastrointestinal perforation (e.g., patients with a history of diverticulitis). Promptly evaluate patients who present with new onset abdominal symptoms for early identification of gastrointestinal perforation.

LABORATORY ABNORMALITIES:

Neutropenia Olumiant treatment was associated with an increased incidence of neutropenia (absolute neutrophil count [ANC] <1000 cells/mm3) compared to placebo. Avoid initiation or interrupt Olumiant treatment in patients with an ANC <1000 cells/mm3. Evaluate at baseline and thereafter according to routine patient management.

Lymphopenia Absolute lymphocyte count (ALC) <500 cells/mm3 were reported in Olumiant clinical trials. Lymphocyte counts less than the lower limit of normal were associated with infection in patients treated with Olumiant, but not placebo. Avoid initiation or interrupt Olumiant treatment in patients with an ALC <500 cells/mm3. Evaluate at baseline and thereafter according to routine patient management.

Anemia Decreases in hemoglobin levels to <8 g/dL were reported in Olumiant clinical trials. Avoid initiation or interrupt Olumiant treatment in patients with hemoglobin <8 g/dL. Evaluate at baseline and thereafter according to routine patient management.

Liver Enzyme Elevations Olumiant treatment was associated with increased incidence of liver enzyme elevation compared to placebo. Increases of ALT 5x upper limit of normal (ULN) and increases of AST 10x ULN were observed in patients in Olumiant clinical trials.

Evaluate at baseline and thereafter according to routine patient management. Promptly investigate the cause of liver enzyme elevation to identify potential cases of drug-induced liver injury. If increases in ALT or AST are observed and drug-induced liver injury is suspected, interrupt Olumiant until this diagnosis is excluded.

Lipid Elevations Treatment with Olumiant was associated with increases in lipid parameters, including total cholesterol, low-density lipoprotein cholesterol and high-density lipoprotein cholesterol. Assess lipid parameters approximately 12 weeks following Olumiant initiation. Manage patients according to clinical guidelines for the management of hyperlipidemia.

VACCINATIONS: Avoid use of live vaccines with Olumiant. Update immunizations in agreement with current immunization guidelines prior to initiating Olumiant therapy.

HYPERSENSITIVITY: Reactions such as angioedema, urticaria, and rash that may reflect drug sensitivity have been observed in patients receiving Olumiant, including serious reactions. If a serious hypersensitivity reaction occurs, promptly discontinue Olumiant while evaluating the potential causes of the reaction.

ADVERSE REACTIONSMost common adverse reactions include: upper respiratory tract infections (16.3%, 11.7%), nausea (2.7%, 1.6%), herpes simplex (0.8%, 0.7%) and herpes zoster (1.0%, 0.4%) for Olumiant 2 mg and placebo, respectively.

USE IN SPECIFIC POPULATIONS

PREGNANCY AND LACTATION: No information is available to support the use of Olumiant in pregnancy or lactation. Advise women not to breastfeed during treatment with Olumiant.

HEPATIC AND RENAL IMPAIRMENT: Olumiant is not recommended in patients with severe hepatic impairment or in patients with severe renal impairment.

Please click to access full Prescribing Information, including Boxed Warning about Serious Infections, Malignancies, and Thrombosis, and Medication Guide.

BA HCP ISI 09JUL2020

About OLUMIANTOLUMIANT is a once-daily, oral JAK inhibitor approved in the U.S. for the treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more TNF inhibitor therapies, and approved outside of the U.S. for patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more DMARDs.i There are four known JAK enzymes: JAK1, JAK2, JAK3 and TYK2. JAK-dependent cytokines have been implicated in the pathogenesis of a number of inflammatory and autoimmune diseases.ii OLUMIANT has greater inhibitory potency at JAK1, JAK2 and TYK2 relative to JAK3; however, the relevance of inhibition of specific JAK enzymes to therapeutic effectiveness is not currently known.i

In December 2009, Lilly and Incyte announced an exclusive worldwide license and collaboration agreement for the development and commercialization of baricitinib and certain follow-on compounds for patients with inflammatory and autoimmune diseases.

About Eli Lilly and Company Lilly is a global health care leader that unites caring with discovery to create medicines that make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us at lilly.com and lilly.com/newsroom. P-LLY

About Incyte Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about OLUMIANT (baricitinib) as a potential treatment for patients with COVID-19 and as a treatment for patients with rheumatoid arthritis, and about the supply of OLUMIANT, and reflects Lilly's and Incyte's current beliefs. This press release also contains a forward-looking statement about Lilly's potential antibody treatments for COVID-19. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of development and commercialization. Among other things, there can be no guarantee that OLUMIANT will receive additional regulatory approvals or continue to be commercially successful, that we can provide an adequate supply of OLUMIANT in all circumstances, or that potential antibody treatments will be safe and effective. For further discussion of these and other risks and uncertainties, see Lilly's and Incyte's most recent respective Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission. Except as required by law, Lilly and Incyte undertake no duty to update forward-looking statements to reflect events after the date of this release.

i Olumiant Prescribing Information, 2020.ii Walker JG and Smith MD. J Rheumatol. 2005;32;1650-1653.

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SOURCE Eli Lilly and Company

Company Codes: NASDAQ-NMS:INCY, NYSE:LLY

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Baricitinib in Combination with Remdesivir Reduces Time to Recovery in Hospitalized Patients with COVID-19 in NIAID-Sponsored ACTT-2 Trial - BioSpace

Neurocrine Biosciences and Voyager Therapeutics Present New Long-Term Three-Year Data Demonstrating that One-Time Treatment with an Investigational…

Posted: September 12, 2020 at 8:53 am


-- Data for Investigational Gene Therapy Treatment NBIb-1817 (VY-AADC) Presented at the MDS Virtual Congress 2020 --

SAN DIEGO and CAMBRIDGE, Mass., Sept. 11, 2020 (GLOBE NEWSWIRE) -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) and Voyager Therapeutics, Inc. (Nasdaq: VYGR) today announced data from PD-1101, a Phase Ib open-label, three-year efficacy and safety study, demonstrating that a one-time treatment with investigational gene therapy, NBIb-1817 (VY-AADC), showed sustained improvement in motor function including greater On time without troublesome dyskinesia, reduction in Unified Parkinsons Disease Rating Scale (UPDRS) Part III scores, and reduction in the amount of medications in patients with Parkinsons disease. In the PD-1101 study, NBIb-1817 reduced average Off time by up to -1.91 hours and improved average On time without troublesome dyskinesia by up to +2.23 hours in patients with advanced Parkinsons disease after three years across three cohorts. In addition, 14 out of 15 patients treated with NBIb-1817 continued to show an improvement in disease staging after three years, as assessed by the modified Hoehn & Yahr scale. These new data, along with two-year data from another open-label Phase Ib trial, PD-1102, were presented today at the MDS Virtual Congress 2020, September 1216, 2020 (www.mdscongress.org/Congress/Registration.htm).

In data from the three-year PD-1101 trial, the one-time treatment with NBIb-1817 showed sustained reduction in diary Off time by an average of -0.15 to -1.91 hours (baseline 4.28 to 4.93 hours) and improved diary On time without troublesome dyskinesia by an average of +0.26 to +2.23 hours (baseline 10.32 to 10.46 hours) across the cohorts as reported by 15 patients with advanced Parkinsons disease. NBIb-1817 also showed sustained improvement in motor function after three years, as measured by UPDRS Part III off medication scores, by -10.2 to -19.0 points (baseline 35.8 to 38.2 points) across the cohorts, per clinician assessment. Requirements for Parkinsons disease medications were also reduced in cohorts 2 and 3 (daily levodopa-equivalent dose reductions, average of -322.0 and -441.2 mg/day, respectively; baseline 1507.0 and 1477.0 mg/day, respectively). Two-year data from the PD-1102 trial for 7 patients showed that NBIb-1817 reduced diary Off time by an average of -3.2 hours and increased diary good On time by +2.1 hours (baselines 9.3 hours and 6.6 hours, respectively). In this study, NBIb-1817 showed sustained improvement in motor function after two years, with improved UPDRS Part III off medication scores of -12.0 points (baseline 34.4). Requirements for Parkinsons disease medications were also reduced (daily levodopa-equivalent dose reduction, average pf -439.5 mg/day; baseline 1500.9 mg/day). Preliminary safety data from both studies suggest that NBIb-1817 was well-tolerated, with no study drug-related serious adverse events (SAEs) reported. The most common adverse events reported were headache, hypoesthesia, and musculoskeletal pain (PD-1101), and upper respiratory tract infection, headache, nausea, and depression (PD-1102).

It is promising to see that after three years, a single administration of one-time investigational gene therapy treatment NBIb-1817 showed sustained reduction in Off time, as well as improvement in On time without troublesome dyskinesia and other measures of motor function in patients with Parkinsons disease, said Chad Christine, M.D., primary author, a lead investigator of the study and Professor of Neurology at the University of California, San Francisco (UCSF) Weill Institute for Neurosciences. Parkinsons disease patients motor function would be expected to worsen over three years, making these results very encouraging. The standard of care for advanced Parkinsons disease has not significantly changed in decades and it is our hope that NBIb-1817 has the potential to become the first gene therapy for Parkinsons disease.

Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the U.S. and six million people worldwide. It is characterized by a loss of dopamine from neuronal degeneration, with a concomitant loss of the aromatic L-amino acid decarboxylase (AADC) enzyme required to synthesize dopamine in the brain, leading to associated impairment in motor, neuropsychiatric, and autonomic functions.

We are pleased that the results from these studies show that one-time treatment with investigational NBIb-1817 may help restore the brains ability to convert levodopa into dopamine, said Eiry Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. Our hope is that NBIb-1817 will help patients experience less Off time and more On time and improve motor symptom control. We plan to re-initiate enrollment in our registrational RESTORE-1 clinical trial with NBIb-1817 this year and look forward to further evaluating NBIb-1817 in patients with Parkinsons disease.

NBIb-1817 is an investigational recombinant adeno-associated viral serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine.

We are encouraged by the congruence of long-term data, including clinician- and patient-reported clinical outcomes in our clinical studies, said Omar Khwaja, M.D., Ph.D., Chief Medical Officer and Head of Research and Development at Voyager Therapeutics. These results are promising and show that the approach has the potential to transform the treatment of Parkinsons disease, and help improve the lives of patients and their families.

Additional information about PD-1101 and PD-1102 will be available on demand for registered participants through October 1, 2020 on the MDS meeting website (www.mdscongress.org/Congress/Registration.htm).

About Parkinsons Disease and NBIb-1817 (VY-AADC) Parkinsons disease is a chronic, progressive and debilitating neurodegenerative disease that affects approximately one million people in the U.S. and six million people worldwide. It is characterized by a loss of dopamine and neuronal degeneration with a concomitant loss of the aromatic L-amino acid decarboxylase (AADC) enzyme required to synthesize dopamine in the brain, leading to associated impairment in motor, neuropsychiatric, and autonomic functions. Dopamine is a chemical messenger that is produced in the brain and is involved in the control of movement. It is made when AADC converts the chemical levodopa to dopamine. As Parkinsons disease progresses, there is less AADC enzyme in parts of the brain where levodopa is converted to dopamine.

NBIb-1817 is an investigational recombinant adeno-associated viral (AAV) serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine. NBIb-1817 is administered into the brain using intraoperative monitoring with magnetic resonance imaging (MRI)-facilitated targeted delivery.

About the RESTORE-1 Clinical TrialPaused temporarily in April 2020 due to the COVID-19 pandemic, Neurocrine Biosciences and Voyager Therapeutics plan to re-initiate RESTORE-1, a Phase 2, randomized, placebo-surgery controlled, double-blinded, multi-center clinical trial, to evaluate the safety and efficacy of NBIb-1817 in patients who have been diagnosed with Parkinson's disease for at least four years and have at least three hours of Off time during the day as measured by a validated self-reported patient diary.

For more information about the RESTORE-1 clinical trial, including eligibility criteria, please visit clinicaltrials.govand restore1study.com.

About the RESTORE-2 Clinical TrialPreparations are ongoing for the RESTORE-2 global registrational trial that will include clinical sites within and outside the U.S.

About Neurocrine Biosciences and Voyager Therapeutics Strategic CollaborationIn 2019, Neurocrine Biosciences and Voyager Therapeutics entered into a strategic collaboration focused on the development and commercialization of gene therapy programs, VY-AADC for Parkinsons disease and VY-FXN01 for Friedreichs ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine Biosciences expertise in neuroscience, drug development and commercialization with Voyagers innovative gene therapy programs targeting severe neurological diseases.

About Neurocrine BiosciencesNeurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinsons disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

About Voyager TherapeuticsVoyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information on Voyager, please visit the companys website atwww.voyagertherapeutics.comor follow@VoyagerTxon Twitter andLinkedIn.

Voyager Therapeuticsis a registered trademark of Voyager Therapeutics.

Neurocrine Biosciences Forward-Looking StatementsIn addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are risks that the product candidates licensed from Voyager may not obtain regulatory approval from the FDA or other regulatory agencies, or such approval may be delayed or conditioned; risks that development activities related to the product candidates licensed from Voyager may not be completed on time or at all; risks associated with the Company's dependence on Voyager for research, development and manufacturing activities; risks that ongoing or future clinical trials may not be successful or replicate previous clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for product candidates licensed from Voyager; risks that the product candidates licensed from Voyager may be precluded from commercialization by the proprietary rights of third parties; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our business; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; and other risks that are described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

Voyager Therapeutics Forward-Looking StatementsThis 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 may, might, will, would, should, expect, plan, anticipate, believe, estimate, undoubtedly, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager Therapeutics makes regarding the potential impact or significance of the long-term medical data for patients treated in the PD-1101 and PD-1102 clinical trials; the re-initiation of RESTORE-1 Phase 2 clinical trial prior to year-end, the initiation of the RESTORE-2 Phase 3 clinical trial during the first half of 2021; the initiation, timing, progress, activities, goals and reporting of results of other activities associated with the PD program, and the potential benefits, timing and future operation of the collaboration with Neurocrine Biosciences are forward looking. All forward-looking statements are based on estimates and assumptions by Voyagers management that, although Voyager believes such forward-looking statements 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 Voyager expected. Such risks and uncertainties include, among others, risks that ongoing or future clinical trials may not be successful or replicate previous clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for product candidates now being evaluated in clinical trials; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our clinical trials and our business generally; risks related to the initiation and conduct of preclinical studies and clinical trials; the sufficiency of preclinical and clinical data to support applications for additional studies and marketing approval of our PD drug development candidates; changes in expectations from the FDA and other regulatory authorities as to the requirements for obtaining product approvals; the decisions of the FDA and other regulatory authorities in response to applications we file in connection with our product candidates under our PD program and otherwise in our conduct of PD drug development activities; the priorities, capabilities, diligence and efforts of Neurocrine Biosciences, our collaboration partner for the PD program, and other collaborators and vendors supporting our PD program; and the commercial potential of PD product candidates that may be developed as part of our PD program. These statements are also subject to a number of material risks and uncertainties that are described in Voyager Therapeutics Annual Report on Form 10K, Voyager Therapeutics Quarterly Reports on Form 10-Q and other reports filed by Voyager Therapeutics with theSecurities and Exchange Commission, as may be updated by its subsequent filings with theSecurities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager Therapeutics undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contact: Neurocrine BiosciencesNavjot Rai (Media)858-617-7623media@neurocrine.com

Todd Tushla (Investors)858-617-7143ir@neurocrine.com

Contact: Voyager TherapeuticsPaul Cox (Investors)857-201-3463pcox@vygr.com

Sheryl SeapyW2Opure949-903-4750sseapy@purecommunications.com

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Neurocrine Biosciences and Voyager Therapeutics Present New Long-Term Three-Year Data Demonstrating that One-Time Treatment with an Investigational...

Electronic Clinical Outcome Assessment (eCOA) Solutions Market – Growing adoption of cloud based platforms across and the emergence of cloud…

Posted: September 11, 2020 at 5:53 am


Electronic clinical outcome assessment (eCOA) is an umbrella term encompassing patient-reported outcomes (PROs), observer-reported outcomes (ObsROs), clinician-reported outcomes (ClinROs), and performance-related outcomes. The eCOA solutions can be used to measure the efficacy of a health intervention on patients populations. eCOAs take into account persons mental condition, symptoms of a disease, and the effect of a disease condition on the patients function. eCOA solutions facilitate the clinical diagnosis of diseases with complex etiology and help in designing effective therapies; for instance ClinROs and PROs are commonly used in the assessment of schizophrenia. The solutions make the extensive use of an array of electronic devices and digital technologies such as smartphones, tablets, and computers to help clinicians, patients, and their caregivers make the desired reporting. These solutions are instrumental in improving the accuracy of results in clinical trials by making reporting intelligent, reducing the margin of error, and giving the benefit of an extended sample. In addition, the ability offered by eCOA solutions to gather clinical data in real time and the benefit offered to users in making quick changes are compelling concerns bolstering the popularity among various end users.

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The need for improving patient safety and safety in clinical trials has gained more significance than ever. eCOA with several of its advantages underpinned by digital healthcare system are increasingly being adopted by hospitals, healthcare centers, and contact research organizations (CROs). Constant advancements in eCOA modalities and applications bode well for the market. Furthermore, the rising adoption of cloud solutions is remarkable expanding the horizon of the electronic clinical outcome assessment market. The advent of innovative data visualization and reporting tools is a noteworthy factor unlocking promising prospects in the market.

Global Electronic Clinical Outcome Assessment (eCOA) Solutions Market: Overview

Electronic clinical outcome assessment (eCOA) solutions quantify a patients overall state of mind, symptoms in a patient, the impact of a particular disease, and how the patient functions in this condition. Electronic clinical outcome assessment can be employed for determining if a drug has been established to deliver treatment benefits. Electronic clinical outcome assessment solutions include patient-reported outcome (PRO), clinician-reported outcome (ClinRO), and observer-reported outcome (ObsRO). There are several end users of electronic clinical outcome assessment such as in hospitals, healthcare centers, and Contact Research Organizations (CROs) among others. The end user segment that held the leading share in 2016 and is expected to exhibit exponential growth over the coming years. This growth can be attributed to the rising pressure on pharmaceutical companies in order to save the total clinical development studies costs.

The global market for electronic clinical outcome assessment solutions is expected to witness significant growth during the forecast period owing to the growing adoption of cloud based platforms across and the emergence of cloud healthcare solutions.

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Global Electronic Clinical Outcome Assessment (eCOA) Solutions Market: Drivers and Restraints

The global market for electronic clinical outcome assessment solutions is expected to rise at an exponential rate in the upcoming years owing to factors such as the growing adoption of electronic clinical outcome assessment by medical research experts. For instance, in 2015, the Journal of Clinical Studies projected that recently 7% of all the trials are integrating electronic clinical outcome assessment solutions and is expected to rise by almost 20% by the end of 2018. With the healthcare sector witnessing substantial growth, the need for research studies for developing novel treatments and drugs is also rising. The growing number of research studies has further intensified the demand for centralized data capture equipment and technologies. Thus, the above factors are expected to propel the demand for electronic clinical outcome assessment solutions in the near future.

Furthermore, rising weight on pharmaceutical manufacturers in order to expurgate overall prices for new drug development processes has caused in their rising predisposition from paper-based processes towards electronic data capturing. Data capturing through electronic clinical outcome assessment solution platforms enhances the quality of information which is received, complements the data collection processes, and provides noteworthy value to its subscribers, such as data analysis. Electronic based services for capturing data and assessing it to address the challenges of paper based records is rising with increasing patient compliance. They are also known for eliminating the challenges related to data variance and minimizing the cost of site monitoring.

Global Electronic Clinical Outcome Assessment (eCOA) Solutions Market: Market Potential

Companies operating in the global electronic clinical outcome assessment solutions market are implementing strategies such as geographic enhancement, launch of new product, enhancement of distribution channel, partnership agreements, and acquisitions. For instance, in February 2017, CRF Health proclaimed a partnership with Vodafone for enhancing the service offered to clinical trial service providers and their patients. This particular agreement allows CRF to employ the Machine-to-Machine platform of Vodafone.

Global Electronic Clinical Outcome Assessment (eCOA) Solutions Market: Regional Outlook

Region-wise, North America is expected to lead the market in terms of consumption in the coming years owing to the prevalence of manufacturers of medical device, advanced technology research centers, and hospitals and universities along with enhanced healthcare establishments.

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Global Electronic Clinical Outcome Assessment (eCOA) Solutions Market: Vendor Landscape

Some of the top drawer companies present in the market are OmniComm Systems, Inc., ERT Clinical, CRF Health, eClinical Solutions, BioClinica, Merge Healthcare Incorporated, Medidata Solution, Inc., Paraxel International Corporation, and Oracle Corporatio.

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Electronic Clinical Outcome Assessment (eCOA) Solutions Market - Growing adoption of cloud based platforms across and the emergence of cloud...

Orasis Raises $30 Million to Advance the Clinical Development of Novel Treatment for Presbyopia – GlobeNewswire

Posted: at 5:53 am


HERZLIYA, Israel, Sept. 10, 2020 (GLOBE NEWSWIRE) -- Orasis Pharmaceuticals, an emerging ophthalmic pharmaceutical company focused on developing an innovative pharmaceutical solution for the treatment of presbyopia symptoms, today announced the closing of a $30 million Series C financing. The financing was co-led by new investor Bluestem Capital and returning investor Visionary Ventures, with participation from other returning investors Sequoia Capital, SBI (Japan) Innovation Ventures, Maverick Ventures Israel, LifeSci Venture Partners and additional investors. Tyler J. Stowater, partner and vice president of Bluestem Capital, will join theOrasisBoard of Directors in conjunction with the financing.

Proceeds from the financing will be used to advance Orasis lead eye drop candidate for the treatment of presbyopia symptoms through completion of its Phase 3 clinical trials. The funds will also be used for pre-commercialization activities ahead of potential product launch.

This successful funding round completes the capitalization for the Phase 3 clinical trials and fuels the initial growth strategy for commercialization, said Elad Kedar, chief executive officer of Orasis. We aspire to make near vision clear again for people with presbyopia by empowering them with an unparalleled solution, an eye drop that will provide them with comfort and control of their near vision. We are very grateful for and highly encouraged by the validation from a diverse range of investors, including the co-lead investors Bluestem Capital and Visionary Ventures, and our returning investors. Our product candidate has demonstrated excellent efficacy, safety and comfort profiles in previous clinical studies and we look forward to initiating our Phase 3 clinical trials to further evaluate the effectiveness of the product in the near future.

Tyler J. Stowater, partner and vice president of Bluestem, added, With almost two billion people in the world living with presbyopia, the market potential for a novel, non-invasive option is highly anticipated by eyecare providers and patients. We have high confidence in the Orasis team, to successfully complete its clinical program, and apply their informed commercial approach, which will make Orasis a leader in the presbyopia space.

About Presbyopia

Presbyopiais the loss of ability to focus on near objects as a result of the natural aging process. It occurs mostly after the age of 40 when the crystalline lens of the eye gradually stiffens and loses flexibility. There are almost two billion people globally and more than 120 million people in the U.S. living with presbyopia. People with presbyopia experience blurred vision when performing daily tasks that require near visual acuity, such as reading a book, a restaurant menu or messages on a smartphone. Presbyopia cannot be prevented or reversed, and it continues to progress gradually. All existing treatment options are either cumbersome or invasive, presenting a significant unmet need for quality of life improvement for people with presbyopia.

About Orasis Pharmaceuticals

Orasis is an emerging ophthalmic pharmaceutical company committed to making near vision clear again for people with presbyopia. Orasis novel proprietary formulation, designed to achieve an optimal balance between efficacy, safety and comfort,has the potential to position the company as an emerging leader in the presbyopia space. Orasis is based in Herzliya, Israel. For more information, visithttps://www.orasis-pharma.com and connect with us on LinkedIn: https://www.linkedin.com/company/orasis-pharmaceuticals/.

About Bluestem Capital

With more than 95 years of collective experience, Bluestem Capital is a private equity and venture capital firm with 25 portfolio companies. Healthcare is a leading investment area for the firm, with continued emphasis on eyecare innovation. For more information, visit https://www.bluestemcapital.com.

About Visionary Ventures

Visionary Ventures is a leading venture capital fund investing in pharmaceuticals and devices for the optometric vertical. The firm has over 75 key opinion leaders in ophthalmology and optometry who invest, advise and provide unique insights into the best opportunities for eyecare. By building a team of specialists, professionals and industry leaders at the forefront of innovation, Visionary is able to leverage a unique, competitive advantage to our venture investments. For more information, visit https://visionaryvc.com.

Media ContactAlison ChenLifeSci Communications +1 646-876-4932achen@lifescicomms.com

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Orasis Raises $30 Million to Advance the Clinical Development of Novel Treatment for Presbyopia - GlobeNewswire

Operation Warp Speed turns to DoD to host phase 3 clinical trial of AstraZeneca’s COVID-19 vaccine – Homeland Preparedness News

Posted: at 5:53 am


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The U.S. Department of Defense (DoD) will host a phase three trial of the vaccine candidate AZD1222, developed by AstraZeneca, at five locations selected by Operation Warp Speed.

For the trial, the selected sites include Naval Medical Center San Diego, Joint Base San Antonio Brooke Army Medical Center, Wilford Hall Ambulatory Surgical Center in San Antonio, Walter Reed National Military Medical Center in Maryland, and Fort Belvoir Community Hospital in Virginia. There, researchers seek volunteers at higher risk of SARS-CoV-2 infection, be they essential workers, from dense urban areas or communities hardest hit by the pandemic, or residents and workers of crowded facilities.

The Department of Defense continues to play a key role in the development of a potential COVID-19 vaccine, Tom McCaffery, Assistant Secretary of Defense for Health Affairs, said. Now that vaccines have passed the first phases of testing for safety, dosing, and response, we are ready to move into the next phase where volunteers are needed to join large clinical studies.

Operation Warp Speed hopes that efforts like this will yield safe and effective vaccines and therapeutics by January 2021. Supporting this particular trial are the DoDs Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense, along with the Office of the Assistant Secretary of Defense for Health Affairs and the Defense Health Agency.

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Operation Warp Speed turns to DoD to host phase 3 clinical trial of AstraZeneca's COVID-19 vaccine - Homeland Preparedness News

Avera to start clinical studies on experimental antibody therapy – KELOLAND.com

Posted: September 3, 2020 at 8:58 am


SIOUX FALLS, S.D. (KELO) Avera is getting a new opportunity in the fight against COVID-19. The health care system has been chosen as a clinical site for studies on experimental antibody therapy to prevent and treat the virus.

Finding a way to treat and prevent COVID-19 could be beneficial in the fight against the virus.

Now Avera will be participating in three Phase 2 and 3 clinical studies evaluating whether a combination of two lab-made antibodies can do just that.

The clinical studies have already been approved to move forward following positive Phase 1 safety results.

Two of the studies, both the inpatient and the outpatient are already treating people that are already COVID positive, and so in those cases, we are trying to decrease the severity of their symptoms, so if they are in the hospital, their length of stay, chief clinical research officer, Avera Research, Amy Elliott said.

The other study involves people who were exposed to someone with COVID-19.

For those patients, then it is a prevention study, to try to keep the virus from entering their cells in the first place, and if they would get it, to keep the severity from getting too bad, Elliot said.

The clinical studies are sponsored by Regeneron Pharmaceuticals.

If it is successful, weve already had experience with it so as it moves into approved therapy, which is where the company would like it to go, then we are comfortable with how to give it, so it gives us kind of a prelook to make sure its effective, chief medical officer of research, Avera, John Lee said.

To bring these trials to Avera and to this region I think helps continue to put South Dakota on the map for high quality research, Elliot said.

Elliott says they are hoping to recruit about 45 to 50 people in all three of the studies. Eligible participants must be 18 years or older and live in the Sioux Falls area. You can call 605-504-3154 if you think you or a family member may meet the criteria.

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Avera to start clinical studies on experimental antibody therapy - KELOLAND.com

North America Healthcare CRO Services Industry 2020-2027 – Increased Need for Therapeutics/Vaccines to Treat COVID-19 – GlobeNewswire

Posted: at 8:58 am


Dublin, Sept. 03, 2020 (GLOBE NEWSWIRE) -- The "North America Healthcare Contract Research Organizations Services Market to 2027 - Service Type; Therapeutic Indication; End User, and Country" report has been added to ResearchAndMarkets.com's offering.

The North America healthcare contract research organizations (CRO) services market is expected to reach US$ 40,863.26 million by 2027 from US$ 19,498.79 million in 2019. The market is estimated to grow at a CAGR of 9.9% during 2020-2027.

The healthcare contract research organizations (CRO) services market is growing primarily due to the growing number of clinical trials and increasing expenditure in R&D and outsourcing activities in the North America region. Factors such as stringent regulations for approval of drugs damage the growth of the market. Additionally, rising biosimilars and biologics market and globalization of clinical studies are likely to fuel the growth of the healthcare contract research organizations (CRO) services market during the forecast period.

Exponentially developing healthcare infrastructure and increasing adoption of advanced technologies by several countries have led to the globalization of clinical studies during the past decade. Additionally, economic globalization in developed countries play a key role in globalization. For instance, according to a study published in the International Journal of Clinical Trials in 2016, the number of countries, which serve as clinical study locations outside the US, has doubled in the last 10 years.

Furthermore, according to a data published by clinicaltrails.gov, as of June 2020, an estimated 61% of the total studies were recruited at out of the US locations. Factors such as cost optimization, infrastructure availability, patient recruitment rate, government regulations, and market potential offer a major contribution for the transition from US to other countries. Globalization of these clinical studies has opened new avenues and market potential for CROs.

North America is witnessing the growing number of COVID-19 cases. Due to the outbreak of COVID-19 pandemic there is an increased need for therapeutic or vaccine for this highly contagious viral disease. Hence, more COVID-19 clinical trials are being planned and initiated, whereas, the large number of clinical trials in US for non-COVID-19 indications are being delayed. Therefore, it is likely to affect the healthcare contract research organizations (CRO) services market owing to the above mention points.

Based on service type, the healthcare contract research organizations (CRO) services market is further segmented into early phase development, laboratory services, consulting services, and clinical research services. Early phase development market is further categorized into discovery studies, chemistry, marketing & manufacturing, and preclinical market. The laboratory services segment is further sub segmented as bio-analytical testing and analytical testing. The clinical research services segment is further bifurcated into phase I, phase II, phase III, and phase IV. In 2019, the clinical research segment accounted for the largest share of the market. Growth of this segment is attributed to the increasing clinical trials and growing R&D investment.

Key Topics Covered

1. Introduction1.1 Scope of the Study1.2 Research Report Guidance1.3 Market Segmentation

2. North America Healthcare Contract Research Organizations (CRO) Services Market - Key Takeaways

3. Research Methodology3.1 Coverage3.2 Secondary Research3.3 Primary Research

4. North America Healthcare Contract Research Organizations (CRO) Services Market - Market Landscape4.1 Overview4.2 PEST Analysis4.2.1 Healthcare contract research organizations (CRO) services Market - North America PEST Analysis4.3 Expert Opinion

5. North America Healthcare Contract Research Organizations (CRO) services Market - Key Market Dynamics5.1 Key Market Drivers5.1.1 Increasing Expenditure in R&D and Outsourcing Activities5.1.1 Rising Number of Clinical Trials5.2 Key Market Restraints5.2.1 Stringent Regulations For Approval Of Drugs5.3 Key Market Opportunities5.3.1 Rising Biosimilars and Biologics Market5.4 Future Trends5.4.1 Globalization of Clinical Studies5.5 Impact Analysis

6. Healthcare Contract Research Organizations (CRO) Services Market - North America Analysis6.1 North America Healthcare Contract Research Organizations (CRO) Services Market Revenue Forecasts and Analysis

7. North America Healthcare Contract Research Organizations (CRO) Services Market Analysis and Forecasts To 2027 - By Service Type7.1 Overview7.2 North America Healthcare Contract Research Organizations (CRO) Services Market, by Service Type 2019 & 2027 (%)7.3 Early Phase Development7.4 Laboratory Services7.5 Consulting Services7.6 Clinical Research Services

8. North America Healthcare Contract Research Organizations (CRO) Services Market Analysis - by Therapeutic Application8.1 Overview8.2 North America Healthcare Contract Research Organizations (CRO) Services Market, by Therapeutic Application 2019-2027 (%)8.3 Oncology8.4 Infectious Diseases8.5 Cardiovascular Disease8.6 Respiratory Diseases8.7 Neurological Disorders8.8 Others

9. North America Healthcare Contract Research Organizations (CRO) Services Market Analysis - by End-user9.1 Overview9.2 North America Healthcare Contract Research Organizations (CRO) Services Market, by End-user 2019-2027 (%)9.3 Pharmaceutical and Biopharmaceutical Companies9.4 Medical Device Companies9.5 Others (Academic Institutes)

10. Healthcare Contract Research Organizations (CRO) Services Market Revenue and Forecasts to 2027 - Geographical Analysis

11. Impact of COVID-19 Pandemic on North America Healthcare Contract Research Organizations (CRO) Services Market

12. Company Profiles12.1 Charles River Laboratories, Inc.12.2 IQVIA Inc.12.3 Laboratory Corporation of America Holdings (Covance)12.4 Parexel International Corporation12.5 PRA Health Sciences12.6 Syneos Health12.7 Medpace12.8 PPD Inc.

For more information about this report visit https://www.researchandmarkets.com/r/6skjge

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North America Healthcare CRO Services Industry 2020-2027 - Increased Need for Therapeutics/Vaccines to Treat COVID-19 - GlobeNewswire

Clinerion, Maxer Consulting partner to boost identification and recruitment of patients into clinical trials in Italy, promoting safer, more effective…

Posted: at 8:58 am


BASEL, Switzerland (PRWEB) September 03, 2020

Clinerion and Maxer will cooperate to engage clinical trial sponsors to fully exploit Patient Network Explorers potential and provide added-value services arising from Maxers experience in clinical data management, regulatory affairs, medical affairs, clinical trial disclosure and data transparency, and medical writing services.

The partners' joint mission is to liaise with study sites to increase patient participation in trials to its full potential by offering tools to optimize protocol design, supporting the efficient start-up of local trials, and planning and consistently hitting realistic patient recruitment targets to enable the achievement of total timeline targets.

Clinerion makes real-world patient data accessible for analysis so that study protocols can be optimized with real patients in mind. It also maintains a cloud platform that brings together participants from across the clinical study ecosystem, comprising sponsors, hospitals and clinicians, and treating physicians and patients.

Maxer will support commercial and non-commercial sponsors in registering their clinical trials, uploading the results of the clinical trials on the public databases (EU and US), anonymizing the clinical data, and editing relevant scientific papers.

The geographic scope of the partnership is primarily focused on Italy, but will extend to other countries as opportunities arise.

"There are objective complementarities between Clinerion's and Maxer's business models," says Massimo Zaninelli, CEO Of Maxer. "This condition lays the foundations for cooperation that will contribute to carrying out clinical studies on targeted patient populations, reducing the times and costs of implementation, and enabling innovative approaches to personalized medicine.

We are eager to bring the full support of our services to the patients and physicians in Italy, says Ian Rentsch, CEO of Clinerion. Clinerions Patient Network Explorer matches patients to clinical research, creating greater access to international trials for Italian trial sites and offering physicians more treatment options.

About Clinerion

Clinerion accelerates clinical research and medical access to treatments for patients. We generate real-world data from our global network of partner hospitals for Real World Evidence analyses. Clinerion's Patient Network Explorer radically improves the efficiency and effectiveness of clinical trial recruitment by offering data-driven protocol optimization, site feasibility evaluation and real-time patient search and identification to match patients to treatments.

Clinerion facilitates the participation of partner hospitals in leading-edge, industry-sponsored trials and time savings in patient recruitment. Researchers gain access to real-time, longitudinal patient data from electronic health records for analysis. We enable pharmaceutical companies, CROs and SMOs to shorten patient recruitment and save costs by streamlining operations and leveraging strategic intelligence. Clinerions Patient Network Explorer also provides a platform for integration of diverse patient data sources into real-world data ecosystems. Clinerions proprietary technologies comply with international patient privacy and data security regulations. Clinerion is a global data technology service company headquartered in Switzerland.

Clinerion website: http://www.clinerion.com Clinerions Patient Network Explorer: http://www.clinerion.com/index/PatientNetworkExplorerSolutions.html

For more information, please contact:Le Vin ChinDirector, Head of Marketing & CommunicationsClinerion LtdElisabethenanlage 11, 4051 Basel, SwitzerlandTel.: +41 61 865 60 54media@clinerion.com

About Maxer Consulting

Maxer applies its pharmaceutical expertise, medical communication techniques, change management method, and training programmes to help its customers meet the continually increasing expectations for new therapeutic solutions.

Maxer business model is based on interacting with its customers in an open, collaborative, and proactive way with high standards of quality. Putting people at the centre does apply to both patients and the principle of responsibility that is part of any entrepreneurial initiative.

The creation of economic and social value is Maxer's purpose and ensures the independence that allows operating successfully.

The constant quest for innovation guarantees that Maxer provides its clients with the most advanced solutions.

Maxer Consulting website: http://www.maxerconsulting.it/en

For more information, please contact:Erika OrnagoPharma DirectorMaxer Consulting srlVia Leon Battista Alberti, 520149 Milan ItalyTel.: +39 83421186info@maxerconsulting.com

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Innovent Announces First Subject Dosed in the Phase 1 Clinical Trial of Anti-IL-23 Monoclonal Antibody – PRNewswire

Posted: September 1, 2020 at 5:53 pm


SAN FRANCISCO,and SUZHOU, China, Aug. 31, 2020 /PRNewswire/ -- Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, announced that the Phase I clinical study (CIBI112A101) of recombinant anti-interleukin 23p19 subunit antibody injection (IBI112) has completed the first healthy subject dosing in China.

The study CIBI112A101 is a randomized, double-blind, placebo-controlled, dose-escalation Phase I clinical trial to evaluate the safety and tolerability (primary objective) in addition with PK/PD of IBI112 when administered as a single subcutaneous or intravenous dose in healthy Chinese subjects.

IBI112 is an interleukin-23 (IL-23) antibody, a cytokine with pro-inflammatory effect secreted by activated macrophages and dendritic cells. IL-23 promotes Th17 cell expansion, and the inflammatory response caused by various cytokines and chemokines such as IL-17 and IL-22 secreted by Th17 cells is associated with a variety of autoimmune diseases. IL-23 is composed of two subunits, p19 and p40, of which the p40 subunit is common to IL-23 and IL-12. IBI112 is a monoclonal antibody that selectively targets the p19 subunit of IL-23 but not the p40 subunit, so it can treat autoimmune diseases such as psoriasis by inhibiting the IL-23/Th17 pathway without affecting IL-12-mediated pathogenic defense and tumor immune surveillance.

Dr. Lei Qian, Senior Medical Director of Medical Science and Strategic Special Diseases Department of Innovent, stated: "Currently, there are 2-3% of the world's population suffering from psoriasis, and about 6 million psoriasis patients in China. Regarding inflammatory bowel disease (IBD), the incidence trend is also rising significantly in the last few years. However, neither psoriasis nor IBD therapies have met the huge medical needs. Recently, IL-23 and other biological drugshave been attracting more and more attention due to the superior efficacy and safety profiles, but there are still no drugs targeting IL-23p19 independently developed in China yet. IBI112 is an innovative monoclonal antibody, independently developed by Innovent, that can specifically bind to IL-23p19 subunit and block IL-23-mediated signaling pathway to achieve anti-inflammatory effect. It has potential to treat psoriasis, IBD and other autoimmune diseases. We saw good safety and efficacy data in the preclinical studies and believe this clinical progress of IBI112 is of great importance. We look forward to the results of subsequent clinical studies of IBI112 and hope to provide new treatment options for patients in need."

About IBI112

IBI112 is a monoclonal antibody independently developed by Innovent, with independent intellectual property rights. This product specifically binds to IL-23p19 subunit thereby preventing IL-23 from binding to cell surface receptors, blocking the IL-23 receptor-mediated signaling pathway. Preclinical data demonstrated a clear target, clear mechanism of action, and significant anti-inflammatory effect of IBI112, which indicate that IBI112 may provide a more effective treatment option for patients with autoimmune diseases.

About the CIBI112A101 study

The study CIBI112A101 is a randomized, double-blind, placebo-controlled, dose-escalation Phase I clinical study to evaluate the safety and tolerability (primary objective) in addition with PK/PD of IBI112 when administered as a single subcutaneous or intravenous dose in healthy Chinese subjects.

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 oncology, 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 oncology, metabolic, autoimmune diseases and other major therapeutic areas, with 2 products, TYVYT (sintilimab injection) and BYVASDA (bevacizumab injection), on market, 2 assets under NDA review with priority review status, 4 assets in Phase III or pivotal clinical trials, and additional 15 molecules in or close to clinical trials. TYVYT (sintilimab injection) has been the only PD-1 inhibitor included in the NRDL since 2019.

Innovent has built an international team with broad expertise in high-end biological drug development and commercialization. The company has also entered into strategic collaborations with Eli Lilly and Company, Adimab, Incyte, Alector, Hanmi and other international pharmaceutical companies. For more information, please visit: http://www.innoventbio.com.

SOURCE Innovent Biologics, Inc.

http://www.innoventbio.com

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Innovent Announces First Subject Dosed in the Phase 1 Clinical Trial of Anti-IL-23 Monoclonal Antibody - PRNewswire

Immunomic Therapeutics Announces Publication of Results from 3 ATTAC Studies of CMV-Specific Dendritic Cell Vaccines for the Treatment of GBM -…

Posted: at 5:53 pm


Sept. 1, 2020 12:05 UTC

~25-35 % survival over 5 years from diagnosis in vaccinated GBM patients vs a historical rate of ~ 5%

ROCKVILLE, Md. & SAN DIEGO--(BUSINESS WIRE)-- Immunomic TherapeuticsInc., (ITI), a privately-held clinical-stage biotechnology company pioneering the study of nucleic acid immunotherapy platforms, announced today that results from multiple ATTAC clinical studies of dendritic cell vaccines have been published online by American Association for Cancer Research (AACR) in an article titled, Once, Twice, Three Times a Finding: Reproducibility of Dendritic Cell Vaccine Trials Targeting Cytomegalovirus in Glioblastoma.

ITI is developing several dendritic cell vaccines for the treatment of cancer, including ITI-1000 for glioblastoma (GBM), with leaders in cancer immunotherapy for brain tumors, John Sampson, M.D., Ph.D. from Duke University and Duane Mitchell, M.D., Ph.D. from the University of Florida. ITIs dendritic cell vaccine is designed to target the pp65 viral antigen of Cytomegalovirus (CMV) that is expressed in GBM, but not in normal brain cells.

In the ATTAC studies, the GBM patients white blood cells are removed, matured into dendritic cells (DCs), and modified to generate a vaccine to the pp65 viral protein when fused to the LAMP1 protein for antigen presentation. This DC vaccine is then returned to the patient. As observed in the ATTAC studies, ITI believes this approach may harness the bodys immune system to recognize, attack and destroy tumor cells that express CMV in GBM and potentially other cancers. The published results from the three original ATTAC clinical studies are summarized below:

Three separate clinical trials conducted by Drs. Mitchell and Sampson utilized Cytomegalovirus specific dendritic cell vaccines in patients with newly diagnosed glioblastoma.

The three small studies (total n = 26; NCT#s 00639639, 2366728) revealed that overall 5-year survival increased from a historical low of 5% to 25%. However, in two of the studies, vaccination site pre-conditioning with either Td or GM-CSF:

This data is preliminary, and additional studies are needed.

These study results not only advance our understanding of a virus role in cancer, but they also signal tremendous hope to patients and their families suffering from this devastating disease, Sampson said. I look forward to continued evaluation of ITIs dendritic cell vaccines, including ITI-1000, in the ongoing, randomized, placebo-controlled ATTAC-II study.

GBM is the most aggressive form of brain cancer, often resulting in a patients death within one to two years from diagnosis. Historically, it is a very difficult disease to treat and current treatment options offer limited benefit to extend survival, added Mitchell. The results demonstrated with CMV-specific dendritic vaccines in the ATTAC studies are very encouraging, particularly in the observation of a significant fraction of long-term survivors and favorable safety profile of the vaccine platform. We remain steadfast in our pursuit to identify effective treatments for patients with GBM and look forward to the continued evaluation of ITIs vaccines in addressing this clear and pressing unmet medical need.

The AACR article can be found here.

About Glioblastoma (GBM)

According to the American Association of Neurological Surgeons, GBM is an aggressive brain cancer that often results in death within 15 months of diagnosis. GBM develops from glial cells (astrocytes and oligodendrocytes), grows rapidly, and commonly spreads into nearby brain tissue. GBM is classified as Grade IV, the highest grade, in the World Health Organization (WHO) brain tumor grading system. The American Brain Tumor Association reports that GBM represents about 15% of all primary brain tumors and approximately 10,000 cases of GBM are diagnosed each year in the U.S.

About ITI-1000 and the Phase 2 (ATTAC-II) Study

ITI-1000 is an investigational dendritic cell vaccine therapy currently in a Phase 2 clinical trial (ATTAC-II) for the treatment of GBM. ITI-1000 was developed using Immunomics proprietary investigational lysosomal targeting technology, UNITE, in the context of cell therapy. In May 2017, Immunomic exclusively licensed a patent portfolio from Annias Immunotherapeutics for use in combination with UNITE and ITI-1000, allowing Immunomic to combine UNITE with a patented and proprietary CMV immunotherapy platform. The ATTAC-II study (NCT02465268) is a Phase II randomized, placebo-controlled clinical trial enrolling patients with newly diagnosed GBM that will explore whether dendritic cell (DC) vaccines, including ITI-1000, targeting the CMV antigen pp65 improves survival. This study is enrolling up to 120 subjects at 3 clinical sites in the United States. For more information on the ATTAC-II study, please visit http://www.clinicaltrials.gov.

About UNITE

ITIs investigational UNITE platform, or UNiversal Intracellular Targeted Expression, works by fusing pathogenic antigens with the Lysosomal Associated Membrane Protein 1, an endogenous protein in humans, for immune processing. In this way, ITIs vaccines (DNA or RNA) have the potential to utilize the bodys natural biochemistry to develop a broad immune response including antibody production, cytokine release and critical immunological memory. This approach puts UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy and infectious diseases. UNITE is currently being employed in a Phase II clinical trial as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP1 nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.

About Immunomic Therapeutics, Inc.

Immunomic Therapeutics, Inc. (ITI) is a privately-held, clinical stage biotechnology company pioneering the development of vaccines through its proprietary technology platform, UNiversal Intracellular Targeted Expression (UNITE), which is designed to utilize the bodys natural biochemistry to develop vaccines that generate broad immune responses. UNITE has a robust history of applications in various therapeutic areas, including infectious diseases, oncology, allergy and autoimmune diseases. ITI is primarily focused on applying the UNITE platform to oncology, where it could potentially have broad applications, including viral antigens, cancer antigens, neoantigens and antigen-derived antibodies as biologics. The Company has built a large pipeline from UNITE with six oncology programs and two allergy programs. ITI has entered into a significant allergy partnership with Astellas Pharma and has formed several academic collaborations with leading Immuno-oncology researchers at Fred Hutchinson Cancer Research Institute, Johns Hopkins University of Medicine, University of Florida, and Duke University. ITI maintains its headquarters in Rockville, Maryland. For more information, please visit http://www.immunomix.com.

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Immunomic Therapeutics Announces Publication of Results from 3 ATTAC Studies of CMV-Specific Dendritic Cell Vaccines for the Treatment of GBM -...

Revive Therapeutics Announces IRB Approval for Phase 3 Clinical Trial Protocol for Bucillamine in COVID-19 – GlobeNewswire

Posted: at 5:53 pm


TORONTO, Aug. 31, 2020 (GLOBE NEWSWIRE) -- Revive Therapeutics Ltd. (Revive or the Company) (CSE: RVV, USA: RVVTF), a specialty life sciences company focused on the research and development of therapeutics for medical needs and rare disorders, is pleased to announce that the Companys Phase 3 clinical trial protocol to evaluate the safety and efficacy of Bucillamine in patients with mild-moderate COVID-19 received approval from the independent Institutional Review Board ("IRB") at Advarra, a premier IRB services company in North America.

With the IRB approval of the Phase 3 study protocol for COVID-19, we can recruit U.S. clinical sites efficiently, allowing us to move forward with providing Bucillamine to patients under our IND that was approved by the FDA last month, said Michael Frank, Revives Chief Executive Officer.

An IRB operates under FDA regulations and is an FDA registered constituted group that has been formally designated to review and monitor biomedical research involving human subjects. In accordance with FDA regulations, an IRB has the authority to approve, require modifications (to secure approval), or disapprove research. The purpose of IRB review is to assure, both in advance and by periodic review, that appropriate steps are taken to protect the rights and welfare of humans participating as subjects in the research. To accomplish this purpose, IRBs use a group process to review research protocols and related materials (e.g., informed consent documents and investigator brochures) to ensure the protection of the rights and welfare of human subjects of research.

About the Phase 3 Confirmatory Clinical Study

The Phase 3 confirmatory clinical study titled, A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study of Bucillamine in Patients with Mild-Moderate COVID-19, will enroll up to 1,000 patients that will be randomized 1:1:1 to receive Bucillamine 100 mg three times a day (TID), Bucillamine 200 mg TID or placebo TID for up to 14 days. The primary objective is to compare the frequency of hospitalization or death in patients with mild-moderate COVID-19 receiving Bucillamine therapy with those receiving placebo. The primary endpoint is the proportion of patients meeting a composite endpoint of hospitalization or death from the time of the first dose through Day 28 following randomization. Efficacy will be assessed by comparing clinical outcomes (death or hospitalization), disease severity using the 8-category NIAID COVID ordinal scale, supplemental oxygen use, and progression of COVID19 between patients receiving standard-of-care plus Bucillamine (high dose and/or low dose) and patients receiving standard-of-care plus placebo. Safety will be assessed by reported pre-treatment adverse events and treatment-emergent adverse events (including serious adverse events and adverse events of special interest), laboratory values (hematology and serum chemistry), vital signs (heart rate, respiratory rate, and temperature), and peripheral oxygen saturation.

An interim analysis will be performed by an Independent Data and Safety Monitoring Board (DSMB) after 210 patients have been treated and followed up for 28 days after randomization. The better performing Bucillamine dose at the interim analysis will be selected and patients will then be randomized 2:1 to the selected Bucillamine dose or placebo. Additional interim analyses will be performed after 400, 600, and 800 patients have reached this same post-treatment timepoint. The independent DSMB will actively monitor interim data for the ongoing safety of patients and will recommend continuation, stopping or changes to the conduct of the study based on the interim analysis reports.

The Company is not making any express or implied claims that its product has the ability to eliminate or cure COVID-19 (SARS-2 Coronavirus) at this time.

Scientific Rationale of Bucillamine for COVID-19

Preclinical and clinical studies have demonstrated that reactive oxygen species contribute to the destruction and programmed cell death of pulmonary epithelial cells.1 N-acetyl-cysteine (NAC) has been shown to significantly attenuate clinical symptoms in respiratory viral infections in animals and humans, primarily via donation of thiols to increase antioxidant activity of cellular glutathione2,3,4,5. Bucillamine (N-(mercapto-2-methylpropionyl)-l-cysteine) has a well-known safety profile and is prescribed in the treatment of rheumatoid arthritis in Japan and South Korea for over 30 years. Bucillamine, a cysteine derivative with two thiol groups, has been shown to be 16 times more potent as a thiol donor in vivo than NAC 6. The drug is non-toxic with high cellular permeability. The basis of the clinical study will analyze if Bucillamine has the potential, via increasing glutathione activity and other anti-inflammatory activity, to lessen the destructive consequences of SARS-CoV2 infection in the lungs and attenuate the clinical course of COVID-19.

About Revive Therapeutics Ltd.

Revive is a life sciences company focused on the research and development of therapeutics for infectious diseases and rare disorders, and it is prioritizing drug development efforts to take advantage of several regulatory incentives awarded by the FDA such as Orphan Drug, Fast Track, Breakthrough Therapy and Rare Pediatric Disease designations. Currently, the Company is exploring the use of Bucillamine for the potential treatment of infectious diseases, with an initial focus on severe influenza and COVID-19. With its recent acquisition of Psilocin Pharma Corp., Revive is advancing the development of Psilocybin-based therapeutics in various diseases and disorders. Revives cannabinoid pharmaceutical portfolio focuses on rare inflammatory diseases and the company was granted FDA orphan drug status designation for the use of Cannabidiol (CBD) to treat autoimmune hepatitis (liver disease) and to treat ischemia and reperfusion injury from organ transplantation. For more information, visit http://www.ReviveThera.com.

For more information, please contact:

Michael FrankChief Executive OfficerRevive Therapeutics Ltd.Tel: 1 888 901 0036Email: mfrank@revivethera.comWebsite: http://www.revivethera.com

Neither the Canadian Securities Exchange nor its Regulation Services Provider have reviewed or accept responsibility for the adequacy or accuracy of this release.

Cautionary Statement

This press release contains forward-looking information within the meaning of applicable Canadian securities legislation. These statements relate to future events or future performance. The use of any of the words could, intend, expect, believe, will, projected, estimated and similar expressions and statements relating to matters that are not historical facts are intended to identify forward-looking information and are based on Revives current belief or assumptions as to the outcome and timing of such future events. Forward looking information in this press release includes information with respect to the Offering, including the intended use of proceeds. Forward-looking information is based on reasonable assumptions that have been made by Revive at the date of the information and is subject to known and unknown risks, uncertainties, and other factors that may cause actual results or events to differ materially from those anticipated in the forward-looking information. Given these risks, uncertainties and assumptions, you should not unduly rely on these forward-looking statements. The forward-looking information contained in this press release is made as of the date hereof, and Revive is not obligated to update or revise any forward-looking information, whether as a result of new information, future events or otherwise, except as required by applicable securities laws. The foregoing statements expressly qualify any forward-looking information contained herein. Reference is made to the risk factors disclosed under the heading Risk Factors in the Companys annual MD&A for the fiscal year ended June 30, 2019, which has been filed on SEDAR and is available under the Companys profile at http://www.sedar.com.

References

1. S Ye et al, Inhibition of Reactive Oxygen Species Production Ameliorates Inflammation Induced by Influenza A Viruses via Upregulation of SOCS1 and SOCS3., American Society for Microbiology. 2015 Mar;89(5):2672-2683).

2. L. Carati et al, Attenuation of influenza-like symptomatology and improvement of cell-mediated immunity with long-term N-acetylcysteine treatment., Eur Respir J. 1997 Jul;10(7):1535-41).

3. M Mata et al, N-acetyl-L-cysteine (NAC) inhibit mucin synthesis and pro-inflammatory mediators in alveolar type II epithelial cells infected with influenza virus A and B and with respiratory syncytial virus (RSV)., Biochem Pharmacol. 2011 Sep;82(5):548-55.

4. D Ungheri et al, Protective effect of n-acetylcysteine in a model of influenza infection in mice., Int J Immunopathol Pharmacol. 2000 Sep-Dec;13(3):123-128.

5. RH Zhang et al, N-acetyl-l-cystine (NAC) protects against H9N2 swine influenza virus-induced acute lung injury., Int Immunopharmacol. 2014 Sep;22(1):1-8).

6. LD Horwitz, Bucillamine: a potent thiol donor with multiple clinical applications, Cardiovasc Drug Rev. 2003 Summer;21(2):77-90).

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Revive Therapeutics Announces IRB Approval for Phase 3 Clinical Trial Protocol for Bucillamine in COVID-19 - GlobeNewswire

Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I – BioSpace

Posted: at 5:53 pm


Data on all eight patients demonstrate sustained engraftment and supranormal IDUA enzyme expression

Translation of metabolic correction to clinical outcomes in first two patients continues to support potential of hematopoietic stem cell gene therapy in a second neurometabolic disorder

Data support planned initiation of registrational trial in 2021

BOSTON and LONDON, Sept. 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced additional interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at theSan Raffaele Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The readout from the primary endpoint at one year of follow-up is expected in 2021. Today's results are being shared virtually in an invited oral presentation at the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).

We continue to see encouraging data from the ongoing clinical trial in MPS-I, including promising preliminary clinical effects on motor development, acquisition of cognitive skilIs and growth in the first two patients that were treated now 1.5 and 2 years ago, respectively. Additionally, new preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI, which we look to confirm with longer follow-up, saidMaria Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget. "These data, taken together with those from clinical studies of HSC gene therapy for other metabolic disorders and leukodystrophies, support the potential for this therapeutic approach to correct a wide spectrum of multisystemic manifestations of the disease, bringing clinically meaningful benefits for patients.

Interim Study Results

Eight patients with the severe Hurler subtype of MPS-I had been treated with OTL-203 in the ongoing proof-of-concept study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Treatment with OTL-203 was generally well-tolerated with a safety profile consistent with the selected conditioning regimen. Consistent with previous analyses, treatment across all eight patients continued to demonstrate:

We continue to see positive trends in all biomarker and clinical measures as we follow patients in the OTL-203 proof of concept study for longer periods of time, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With a growing amount of data to support advancing this program, we have recently convened a panel of disease experts to develop a design for a registrational trial that we intend to take to the regulators in advance of initiating the study in 2021 and ultimately progressing towards commercialization.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigationalex vivoautologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard

Orchard Therapeuticsis a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Ourex vivoautologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy inMilan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be successfully developed, approved or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates or that long-term adverse safety findings may be discovered; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedJune 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Read more:
Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - BioSpace

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