CELL AND GENE THERAPY

Biogen and Ginkgo Bioworks Collaborate and License Agreement to Develop Novel Gene Therapy Manufacturing Platform

Biogen, Ginkgo Bioworks | May 22, 2021

Biogen and Ginkgo Bioworks announced a partnership in gene therapy today. The companies aim to redefine the industry standard for manufacturing recombinant adeno-associated virus (AAV)-based vectors by working together.

Recombinant AAV-based vectors are extensively used in the development of novel gene therapies with the ability to cure neurological and neuromuscular diseases, as well as other conditions in a variety of therapeutic areas. Manufacturing is now time-consuming and expensive, making it difficult to develop therapies for diseases with high dosage requirements and large patient populations.

Ginkgo intends to address these issues by using its mammalian cell programming platform to increase the performance of AAV-producing plasmid vectors and cell lines, potentially accelerating Biogen's development of novel gene therapies.

“We believe that Ginkgo's unique combination of cell programming expertise, proprietary tools, and biological system awareness makes them a perfect collaborative partner to explore a broad range of design ideas to optimize constructs,” said Alphonse Galdes, Ph.D., Biogen's Head of Pharmaceutical Operations and Technology.“They share our goal of ensuring that approved therapies are not delayed due to manufacturing constraints and are available to patients all over the world.”

“We are delighted to partner with Biogen as they seek to develop treatments that can potentially delay, stop, or cure neurological and neurodegenerative diseases, as well as to improve the industry standard for AAV manufacturing,” said Jason Kelly, CEO of Ginkgo Bioworks. “Synthetic biology is utilizing the power of living cells to develop the next generation of therapeutics, including CAR-T, CRISPR, and gene therapies, which we hope would have a significant effect on the lives of many people.”

Biogen will have access to Ginkgo's proprietary cell programming platform and capabilities under the terms of the agreement. Ginkgo will use its bioengineering facilities and resources to improve Biogen's gene therapy manufacturing processes' AAV output titers. Ginkgo will receive a $5 million initial payment and will be eligible for milestone payments of up to $115 million if the partnership programs achieve certain research, development, and commercial milestones.

About Biogen
Our mission at Biogen is clear: we are neuroscience pioneers. Biogen discovers, develops, and delivers innovative therapies for people suffering from severe neurological and neurodegenerative diseases, as well as related therapeutic adjacencies, around the world. Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel prize winners Walter Gilbert and Phillip Sharp as one of the world's first major biotechnology firms. Biogen currently has the leading portfolio of medicines for the treatment of multiple sclerosis, is the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics, and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimer's disease, and dementia, neuromuscular disorders, movement disorders, ophthalmology, and neuropsychiatry.

About Ginkgo Bioworks
Ginkgo is building a platform for program cells in the same way as we program machines. The platform is enabling the growth of biotechnology across a wide range of markets, from food and agriculture to industrial chemicals and pharmaceuticals. Ginkgo is also closely involved in a variety of COVID-19 response efforts, such as community testing, epidemiological tracing, vaccine development, and therapeutics discovery.

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Watch the story of our pancreatic cancer therapy unfold in this beautifully told documentary. The video introduces us to this hard-to-treat disease, explains the current options available to pancreatic cancer patients, and then it brings to life our treatment for locally advanced pancreatic cancer, all while presenting both an emotional and powerful story.


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MEDICAL

Ambagon Therapeutics Launches With $85 Million Series A to Advance Pioneering Molecular Glue Platform and Progress Pipeline

Ambagon Therapeutics | January 07, 2022

Ambagon Therapeutics, a biotechnology company unlocking intrinsically disordered proteins and other difficult-to-target protein classes, announced an $85 million Series A to augment its drug discovery platform and to advance its pipeline of molecular glues. The financing was led by Nextech Invest. Ambagon was previously seeded by RA Capital Management, Droia Ventures, Inkef Capital, AbbVie Ventures, MRL Ventures Fund, and Mission BioCapital. Investors from the seed round joined the A round, along with new investor Surveyor Capital. Many disease-relevant proteins have regions of intrinsic disorder that cannot be targeted by conventional small molecule drugs. Several thousand proteins with such intrinsically disordered regions have been identified that interact with the hub protein 14-3-3. In binding to a disordered protein region, 14-3-3 induces order, conferring druggability. By stabilizing naturally occurring 14-3-3:client interactions, Ambagon aims to rapidly develop novel therapeutic candidates for difficult-to-drug proteins. The pipeline currently focuses on oncology, where many opportunities exist to engage currently-undruggable targets. Ambagon was founded by world leaders in 14-3-3 biology and drugging protein-protein interactions: Michelle Arkin, Professor and Chair of Pharmaceutical Chemistry and co-Director of the Small Molecule Discovery Center at UCSF, Luc Brunsveld, Professor of Chemical Biology at the Eindhoven University of Technology (TU/e), and Ambagon Chief Technology Officer Christian Ottmann, Associate Professor of Molecular Cell and Structural Biology, TU/e. Ambagon’s seasoned leadership team includes Chief Executive Officer Scott Clarke, previously CEO of Tizona Therapeutics and Trishula Therapeutics, and Chief Scientific Officer Nancy Pryer, previously CSO at Day One Biopharmaceuticals and Chief Development Officer at Nurix Therapeutics. “Our deep understanding of 14-3-3 biology has broad applications for drug discovery as it opens up disordered protein regions as therapeutic targets. Combined with our proprietary structural insights, curated chemical library, and bespoke drug discovery tools, our experienced drug development team is well placed to bring forward new medicines addressing previously undruggable targets.” Scott Clarke “Ambagon’s modular approach for leveraging 14-3-3 biology to enable drug discovery is not just unique, but uniquely well-conceived,” said Melissa McCracken, Partner, Nextech Invest. “We are excited to see such an exciting platform translate into a very rich pipeline.” “We are proud to continue to support Ambagon as it works to create first-in-class and best-in-class drugs through targeted stabilization of protein complexes,” said Adam Rosenberg, Ambagon’s Chair and RA Capital Venture Partner. “Since our seed investment, the team has developed an impressive proprietary dataset and systemic understanding of 14-3-3 interactions. Ambagon truly has the potential to change the narrative for disordered targets.” About Ambagon Therapeutics Ambagon Therapeutics is a biotechnology company pioneering methods to unlock intrinsically disordered protein targets using small molecules. Ambagon applies deep knowledge of 14-3-3 proteins and a proprietary suite of drug discovery tools to create molecular glues that stabilize 14-3-3:target complexes. These molecular glue stabilizers amplify native biology to restore or inhibit target function, potentiate target activity, or promote or block target degradation. Ambagon’s initial focus is on oncology, with five programs in discovery. It has locations in San Carlos, California, and Eindhoven, the Netherlands. About 14-3-3 Biology Ambagon’s platform harnesses the biology of the regulatory hub protein 14-3-3, which reads serine/threonine phosphorylation. With more than 3000 client proteins reported, 14-3-3 has a vast interactome, enabling the manipulation of a broad range of biology across indications. In binding its clients, 14-3-3 imposes order on disordered client protein sequences, conferring druggability on otherwise undruggable proteins and protein regions. About the Modular Ambagon Drug Discovery Platform By selectively stabilizing the interaction between 14-3-3 and a 14-3-3 client protein, Ambagon can create first-in-class drugs addressing high value targets inaccessible by other means, particularly those with a high degree of disorder, including transcription factors, adaptor/scaffolding proteins, and RNA binding proteins.

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OPKO Health Acquires ModeX Therapeutics, Gains Proprietary Immunotherapy Technology with a Focus on Oncology and Infectious Diseases

OPKO Health | May 10, 2022

OPKO Health, Inc. (NASDAQ: OPK), a multinational biopharmaceutical and diagnostics company, today announced the acquisition of ModeX Therapeutics, Inc. a privately held biotechnology company focused on developing innovative multi-specific immune therapies for cancer and infectious diseases. OPKO acquired ModeX for $300 million in OPKO common stock. Founded in October 2020 with headquarters in Natick, Mass., ModeX Therapeutics has developed highly flexible multi-specific antibody technology platforms with broad targeting and functional capabilities, simpler manufacturing and potentially better specificity and safety, providing significant differentiation from competing platforms. The design of these multi-specifics is amenable to gene-based delivery by mRNA or DNA vectors. The ModeX product portfolio includes cancer immunotherapies that combine four specificities into one protein to improve targeting and immune killing, as well as masking or “stealth” technology to improve tumor-specific killing and reduce side effects. For viral diseases, the lead targets of a broad and potent multi-specific antibody portfolio include HIV and SARS-CoV-2. A vaccine for Epstein-Barr virus is also in development. The acquisition of ModeX Therapeutics significantly broadens our technology foundation and expands our product pipeline to include multi-specific multi-functional antibodies focused on a range of cancers and infectious diseases, with applicability to other therapeutic areas. We believe the promise of better outcomes for patients treated with these multi-specific antibodies represents a next generation of large molecule therapeutics and the next chapter of OPKO, We welcome ModeX’s co-founders Dr. Zerhouni and Dr. Nabel, as well as Alexis Borisy to our Board of Directors, and Dr. Zerhouni and Dr. Nabel to OPKO’s executive management team. The ModeX executive team brings to OPKO a wealth of experience, knowledge and industry contacts, which we expect will have a tremendous long-term positive impact on OPKO as we advance their technology and product pipeline and leverage potential synergies with our current portfolio in diagnostics and therapeutics.” Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO. Elias Zerhouni, M.D., Co-Founder and Chairman of the Board of ModeX, has been appointed President and Vice Chairman of the Board of Directors of OPKO. Dr. Zerhouni brings extensive experience in academia, government and industry as a leading authority on emerging trends and issues in medical care and biomedical research and development. A physician scientist with an academic background in imaging and biomedical engineering, Dr. Zerhouni most recently served as President of Global Research & Development and Executive Vice President of Sanofi. Dr. Zerhouni also served as Director of the National Institutes of Health (NIH), Senior Fellow for Global Health Research at the Bill and Melinda Gates Foundation, Presidential U.S. Envoy for Science and Technology, and Professor and Chair of the Russell H. Morgan Department of Radiology and Biomedical Engineering, Executive Vice Dean and Dean for Research at the Johns Hopkins School of Medicine. Dr. Zerhouni was elected to the National Academy of Medicine and to the National Academy of Engineering. He serves on the Board of the Lasker Foundation, the Foundation for NIH, the Davos Alzheimer’s Collaborative and Research!America. He received the 2017 Scripps Executive of the Year Award for the pharmaceutical industry and the French Legion of Honor in 2008. He has been a director of Danaher Corporation since 2009. Being part of OPKO represents a transformative opportunity for both companies. We anticipate it will accelerate ModeX’s product pipeline focused on unmet needs in oncology and infectious diseases and the development of our innovative technologies, and also will take advantage of synergies with OPKO’s programs, ModeX has operated quietly since we were founded 18 months ago on the basis of over 10 years of prior foundational work. We have assembled a world-class executive team with outstanding public and private sector leadership experience to advance our programs. Our lead drug candidate is already in the clinic while several others in late preclinical stages are expected to enter clinical development in 2023. We were enticed to join OPKO by its leadership’s vision and enthusiasm for strengthening our mutual potential for breakthrough innovation.” Dr. Zerhouni. Gary Nabel, M.D., Ph.D., Co-Founder, President and Chief Executive Officer of ModeX, has been appointed Chief Innovation Officer of OPKO and joins OPKO’s Board of Directors. Dr. Nabel, a renowned virologist and immunologist, served as Chief Scientific Officer and Senior Vice President of Sanofi where he directed the breakthrough laboratory that developed tri-specific products now in early clinical development. He is the founding director of the NIH’s Vaccine Research Center, working on vaccines and broadly neutralizing antibodies against HIV, influenza, SARS, Ebola, Chikungunya and Epstein-Barr virus. He was previously an investigator at the Howard Hughes Medical Institute at the University of Michigan. In recognition of his expertise at the forefront of virology, immunology, gene therapy and molecular biology, Dr. Nabel was elected to the National Academy of Medicine, is a fellow of the American Association of Physicians and the American Academy of Arts Sciences, and was awarded the Geoffrey Beene Builders of Science Award from Research!America. Alexis Borisy, the Lead Independent Director of ModeX, also joins OPKO’s Board of Directors. Mr. Borisy is a leading biotechnology entrepreneur and investor with more than 25 years of experience, including founding, serving as Chief Executive Officer and/or Chairman of nine NASDAQ-listed companies. He co-founded and served as either the Chief Executive Officer or Chairman of Blueprint Medicines, Foundation Medicine, Relay Therapeutics, Tango Therapeutics, Celsius Therapeutics and CombinatoRx. With the additions of Dr. Zerhouni, Dr. Nabel and Mr. Borisy to OPKO’s Board, the number of Directors expands to 13. About OPKO Health, Inc. OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies.

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MEDICAL

Kintor Pharma Provides Update on One of its Three Multi-Regional Phase 3 Trials of Proxalutamide for COVID-19

Kintor Pharma | December 29, 2021

Kintor Pharmaceutical Limited, a clinical-stage biotechnology company developing innovative small molecule and biological therapeutics, provided an update on its multi-regional study of proxalutamide for the treatment of COVID-19 infection. Statistical criteria were not met at an interim analysis of the phase III outpatient study, designed for testing efficacy and safety of proxalutamide for treating COVID-19 in non-hospitalized COVID-19 patients. At the interim analysis, 348 patients with mild-to-moderate COVID-19 symptoms were enrolled. Kintor Pharma will seek for Health Authorities including the U.S. Food & Drug Administration's consent to amend the protocol and continue to enroll higher risk COVID-19 patients only with multiple comorbidities and/or patients with no COVID-19 vaccination history. Based on the interim analysis, there were no safety concerns and no drug-related serious adverse events reported during the study. As of December 23, 2021, Kintor Pharma had completed the enrollment of this pivotal study according to the original protocol. More than 95 percent of the enrolled patients were from the U.S. For the interim analysis, all patients were from the U.S., where the hospitalization rate is very low. "The COVID-19 pandemic continues to evolve with the Omicron variant highlighting the need for therapeutics. It is important to investigate new modalities to treat those infected with the virus. We believe proxalutamide could become an important tool in the fight against COVID-19 infection and will continue to investigate its use. We will provide updates on the final data analysis from this outpatient clinical trial in due course." Dr. Youzhi Tong, founder, Chairman, and Chief Executive Officer of Kintor Pharma The outpatient study is a randomized, double-blind, placebo-controlled Phase III study designed to evaluate the efficacy and safety of proxalutamide in outpatients with mild to moderate COVID-19 illness. For the interim analysis, the trial enrolled 348 male and female patients with one or more mild COVID-19-related symptoms within five days of symptoms onset. Participants were randomized to receive proxalutamide or placebo orally twice daily for 14 days. The enrolment covers the whole population including patients with a vaccination history, as well as without any risk factors. Both the treatment arm and placebo arm were given standard of care. The primary endpoint was the percentage of all-cause death and hospitalization for a period exceeding 24 hours by day 28. About Kintor's Multi-Regional Clinical Trials for Proxalutamide Kintor Pharma is conducting two registered Phase III multi-regional clinical trials of proxalutamide for the treatment of COVID-19 outpatients, and one Phase III MRCT for COVID-19 inpatients in countries and regions that include the United States, South America, Asia and the European Union. The second outpatient study plans to enroll 724 patients, with the primary endpoint of percentage of subjects requiring oxygen by day 28. The inpatient study is a randomized, double-blind, placebo-controlled Phase III study. Male and non-pregnant female hospitalized adults are included. The primary endpoint is time to sustained recovery, and the key secondary endpoint is percentage of all-cause mortality. Kintor Pharma was granted an emergency use authorization for proxalutamide for the treatment of COVID-19 infection in hospitalized patients in Paraguay. About Proxalutamide Proxalutamide is a nonsteroidal antiandrogen — specifically, a selective high-affinity silent antagonist of the androgen receptor — that is under development for the potential treatment of COVID-19, prostate cancer, and breast cancer. Targeting AR-ACE2/TMPRSS2 signal axis by proxalutamide could significantly inhibit the entry of the virus into host cells by transcriptionally down-regulating the expression of TMRPSS2 and ACE2. Proxalutamide promotes the clearance of pathogens and decreases inflammation by activating the Nrf2 pathway, which inhibits the overproduction of IL-6, proinflammatory cytokines, and chemokines. This mechanism of action reduces the intensity of the cytokine response, which may be of clinical benefit to patients with COVID-19 infection. About Kintor Pharmaceutical Limited Kintor Pharmaceutical Limited is developing and commercializing a robust pipeline of innovative small molecule and biological therapeutics for androgen-receptor-related disease areas with unmet medical needs, including COVID-19, prostate, breast and liver cancer, alopecia and acne.

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MEDICAL

Emmaus Life Sciences Announces Launch of Full-Service Telehealth Solution

Emmaus Life Sciences, Inc. | April 12, 2022

Emmaus Life Sciences, Inc. (OTQXC: EMMA), a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease, today announced the launch of an innovative full-service telehealth solution with its strategic partners, including Asembia LLC, US Bioservices Corporation and UpScriptHealth. The telehealth program capitalizes on the expansion of telemedicine in the U.S. to afford patients and providers on-line access to Endari®, the company's prescription-grade L-glutamine oral powder, for the treatment of sickle cell disease. The launch of our full-service telehealth solution reflects our stated commitment to make Endari accessible to sickle cell patients in need and we are excited to be launching the program, Our solution will allow patients to see a doctor without leaving home, thereby eliminating unnecessary travel time and expense and the risk of infection that can occur with hospital visits. Eligible patients will be able to receive a same-day physician authorization and prescription for Endari and to have the prescription delivered to their homes within just a few days. As the telehealth solution grows, we are optimistic that it will help us to reach new patients and add meaningful revenue for Emmaus." Yutaka Niihara, M.D., M.P.H., Emmaus' Chairman and Chief Executive Officer. According to a December 3, 2021 report from the U.S. Department of Health & Human Services (HHS), available at the HHS website at https://www.hhs.gov, the share of Medicare visits conducted through telehealth in 2020 increased 63-fold, from approximately 840,000 in 2019 to 52.7 million. The report noted that telehealth was particularly helpful in offsetting potential foregone behavioral health care and that states with the highest use of telehealth in 2020 included Massachusetts, Vermont, Rhode Island, New Hampshire, and Connecticut. Other industry sources suggest that the trend toward telehealth accelerated in 2021 and is likely to be sustained. We believe that affording patients and telehealth prescribers access to Endari in a quick and convenient way will improve their experience and potentially increase adherence rates, We look forward to working with our strategic partners on this important project." George Sekulich, Senior Vice President of Global Commercialization of Emmaus. About Emmaus Life Sciences Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease. The company currently markets U.S. Food and Drug Administration approved Endari® (L-glutamine oral powder) indicated to reduce the acute complications of sickle cell disease in adults and children 5 years and older. The company is also engaged in the discovery and development of innovative treatments and therapies for certain rare and orphan diseases as well as those affecting larger populations, such as diverticulosis. About Sickle Cell Disease There are approximately 100,000 people living with sickle cell disease (SCD) in the United States and millions more globally. The sickle gene is found in every ethnic group, not just among those of African descent; and in the United States an estimated 1-in-365 African Americans and 1-in-16,300 Hispanic Americans are born with SCD.1 The genetic mutation responsible for SCD causes an individual's red blood cells to distort into a "C" or a sickle shape, reducing their ability to transport oxygen throughout the body. These sickled red blood cells break down rapidly, become very sticky, and develop a propensity to clump together, which causes them to become stuck and cause damage within blood vessels. The result is reduced blood flow to distal organs, which leads to physical symptoms of incapacitating pain, tissue and organ damage, and early death

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Spotlight

Watch the story of our pancreatic cancer therapy unfold in this beautifully told documentary. The video introduces us to this hard-to-treat disease, explains the current options available to pancreatic cancer patients, and then it brings to life our treatment for locally advanced pancreatic cancer, all while presenting both an emotional and powerful story.

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