A 100B Dollars settlement? Or much less? Opioid payouts a mystery until J and J judge rules

Fiercepharma | July 23, 2019

After Johnson and Johnson failed in its latest bid to toss out the opioid suit playing out in an Oklahoma courtroom, the possibility of a settlement continues to grow. For analysts watching the case as a bellwether for other opioid suits, the question is how big the payout could get. Short answer: Who knows? Monday, a judge threw out J&J’s final attempt to dismiss the illegal marketing lawsuit it faces in Oklahoma over sales of its opioid products in the state. J&J, which prosecutors described as the “kingpin” of the state’s opioid epidemic in their closing arguments last week, argued its portrayal by the state as a public nuisance was invalid and violated its First Amendment rights. The bench judge and state attorney Brad Beckworth weren’t having it. "No man or woman has ever laid down their life for this country so that a pharmaceutical company could come out and lie or hook kids on opioids,” Beckworth said.

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Manufacturing drugs for paediatric applications comes with a unique set of challenges. Notably, the need to counteract the bitterness of active pharmaceutical ingredients, and enhance solubility to reduce the risk of delayed onset of action and local tissue injury.


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Cellusion and Celregen a member of Fosun Pharma, Enter into Exclusive License Agreement of CLS001 for a Corneal Endothelial Cell Regenerative

Cellusion and Celregen | September 12, 2022

Cellusion Inc. a Japanese regenerative medicine startup aiming to solve the global corneal transplant waiting list problem with a unique differentiation induction method from iPS cells to Corneal Endothelial Cell Substitute from iPS Cells for bullous keratopathy novel treatment and Hangzhou Celregen Therapeutics Ltd. a member of Shanghai Fosun Pharmaceutical Co., Ltd. and focus on regenerative medicine and cell therapy and incubated by New Drug Fund of Fosun Health Capital announced an exclusive license agreement in the Greater China region for Celregen to develop, manufacture and commercialize CLS001. Under the Agreement, Celregen will have the exclusive right to develop, manufacture and commercialize CLS001 for bullous keratopathy in the Greater China region, including Mainland China, Hong Kong, Macau and Taiwan. On the other hand, Cellusion retains the rights of development, manufacturing and commercialization of CLS001 in other regions including Japan, the USA, and EU. Under the terms of the agreement, Cellusion will potentially receive over 100 million US dollars including upfront payment, development milestones and sales milestones as well as tiered royalties. “We are very enthusiastic about combining our knowledge of the underlying iPS cellular biology with Celregen and Fosun Pharma in the development and commercialization of innovative medicine in China. Together, we are committed to make best efforts on launching CLS001 for the patient suffering from bullous keratopathy due to the cornea donor shortage in the greater China region.” Shin Hatou, M.D. Ph.D., CEO of Cellusion “Today’s announcement with a global industry leader in iPS cells derived corneal endothelial regenerative therapy such as Cellusion, is an important and strategic step to build a long-term foundation for cell therapy in China,” said Cui Zhiping, the Chairman of Celregen, Fosun Global Partner, Chief Consultant of VC investment of Fosun Pharma and CEO of Fosun New Drug Fund. “This partnership, which leverages each company’s respective strengths, will help us bring novel regenerative medicine products to patients in greater China.” According to reports, there are about millions of people in China who are blind due to corneal diseases, and this number is increasing at a rate of 100,000 per year. Most of the patients with corneal blindness need corneal transplantation to restore their sight, but only less than 20,000 corneal transplants are conducted due to severe donor cornea shortage issues. Cellusion has already announced that the patient recruitment began for the First-in-Human Investigator-Initiated Study of CLS001 to initiate in 2022 at the Keio University Hospital and has been preparing to start Cellusion initiated clinical trial in Japan within a few years followed by global studies in the US and EU region. Cellusion is developing CLS001, CECSi cells for Corneal Endothelial regeneration to cure Bullous Keratopathy which is applied to more than half of all cases of corneal transplantation. CLS001 is expected to replace the current supply limitations by combining “CECSi Cells made from iPS cells with excellent proliferative properties” and “a simple injection cell delivery procedure without needs of human expertise.” About Celregen Celregen, incubated by New Drug Fund of Fosun Health Capital, focuses on stem cell and transformed cellular regenerative medicine. This platform adopts a variety of modes of cooperation with leading biotech and scientists. It is expected to be a head biotech in regenerative medicine in China. About Fosun Pharma Founded in 1994, Fosun Pharma is a global innovation-driven pharmaceutical and healthcare industry group deep-rooted in China. Fosun Pharma directly operates businesses including pharmaceutical manufacturing, medical devices, medical diagnosis, and healthcare services. As a shareholder of Sinopharm Co., Ltd., Fosun Pharma expands its areas in the pharmaceutical distribution and retail business. Fosun Pharma is patient-centered and clinical needs-oriented. The company enriches its innovative product pipeline through diversified and multi-level cooperation models such as independent research and development, cooperative development, license-in, and in-depth incubation. Fosun Pharma has formed technological platforms for innovative small molecule drugs, antibody drugs, and cell therapy with a focus on key disease areas including oncology and immunomodulation, metabolism and digestive system, and central nervous system. About Fosun New Drug Fund New Drug Fund, under the management of Fosun Health Capital, is the first VC established by Fosun Pharma. The Fund focuses on innovative biopharmaceutical-based drug fields at the start-up and expansion stages, including gene therapy, cell therapy, bio-conjugate drugs, and medical aesthetics, etc. The Fund has committed to incubate cutting-edging science and revolutionary technologies from world top universities, research institutes and biotech, through which path we can establish and adopt a variety of models of cooperation.

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BUSINESS INSIGHTS

Saudi Ministry of Investment and Insilico Medicine sign agreement to expand region’s biotechnology capabilities

Insilico Medicine | September 15, 2022

Saudi Arabia’s Ministry of Investment and clinical stage artificial intelligence (AI)-driven drug discovery company Insilico Medicine have signed a memorandum of understanding (MoU) to support the development of a robust AI-driven biotech industry in the Kingdom. The agreement was signed during the Global AI Summit, held from the 13th through the 15th of September in Riyadh, which featured over 200 speakers representing 90 countries discussing the future of AI, including Insilico Medicine founder and CEO Alex Zhavoronkov, PhD. Sara Althari, PhD, senior researcher and MISA advisor, spearheaded the agreement. MISA and Insilico Medicine will work on shared initiatives that build on the capabilities of Insilico’s end-to-end AI-powered drug discovery platform, PharmaAI. Drawing on trillions of data points, the platform has three distinct but interconnected elements – novel AI target discovery novel AI drug design and AI clinical trial prediction. Under the terms of the MoU, Insilico will share its expertise and software platform with local biotech companies to support and accelerate their research and drug development efforts. Through the partnership, the MoU also seeks to spur the development of new biotech companies. Insilico will also work with Saudi companies on robotics-aided drug design and clinical trial design and best practices. The Company is launching a fully autonomous AI-run robotics lab that will perform experiments, capture data, and further validate its AI-discovered and designed drugs later this fall. The Company’s clinical trial prediction tool, InClinico, is designed to help researchers and companies plan trials with greater statistical likelihood of Phase 2 success. There will be additional partnership opportunities in other areas where Insilico’s AI platform has shown promise, including the support and initiation of projects related to healthy longevity and support for green chemistry initiatives, including the development of sustainable chemicals and crop protection solutions. “As someone who decided to dedicate his life to extending healthy productive longevity for everyone on the planet, I applaud Saudi Arabia’s new initiatives in this most impactful area of research. As a company, we are encouraged by Saudi Arabia’s vision to increase its digital economy and play a meaningful role in the future of drug discovery using AI and robotics. We look forward to working together to expand the region’s biotech sector and partnering with established and emerging companies as well as academic and research institutions in order to explore the full potential of our end-to-end AI platform.” Alex Zhavoronkov, PhD, founder and CEO of Insilico Medicine Over the past year, MISA has signed MoUs with a number of pharmaceutical and technology companies to grow its biotech sector, including Novartis, Janssen, AstraZeneca, GSK and Roche, as well as Amazon Saudi Arabia. Insilico Medicine, a company which bridges pharma and tech, is the first AI-driven drug discovery company to sign such an agreement in the region. About Insilico Medicine Insilico Medicine, a clinical stage end-to-end artificial intelligence (AI)-driven drug discovery company, is connecting biology, chemistry, and clinical trials analysis using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques to discover novel targets and to design novel molecular structures with desired properties. Insilico Medicine is delivering breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, immunity, central nervous system diseases and aging-related diseases.

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PHARMA TECH

Bluestar Genomics Receives CAP Accreditation for Its Clinical Laboratory

Bluestar Genomics | October 11, 2022

Bluestar Genomics, Inc., an early cancer detection company leading the development and commercialization of next-generation liquid biopsy tests focused on non-invasive detection of high-mortality cancers in high-risk patient populations, today announced it received accreditation for its clinical laboratory from the College of American Pathologists (CAP). The certification enables the company to serve physicians and patients globally as it works towards broad commercialization of its first test for the detection of pancreatic cancer. CAP Accreditation is an internationally recognized program that awards laboratories that meet stringent requirements of quality, accuracy, and consistency. This milestone further solidifies Bluestar Genomics’ commitment to providing the highest standards of testing results. The company’s proprietary approach to detecting cancer early is anchored in epigenomic process of tracking dynamic changes in cells in the body by measuring levels of the biomarker 5-hydroxymethylcytosine (5hmC) in a person’s blood. “This accreditation—on the heels of our CLIA certification and combined with our growing body of scientific evidence supporting the utility of our 5hmC-based epigenomic testing—positions us to become the first cancer detection company to offer non-invasive epigenomic blood-based testing for early pancreatic cancer detection. These milestones are a testament to our focus on preparing our company to serve customers around the world, helping millions of patients avoid late-stage, untreatable cancer diagnosis.” David Mullarkey, chief executive officer of Bluestar Genomics Bluestar Genomics’ pancreatic cancer early detection test has been analytically validated and previously received FDA Breakthrough Device Designation. It is designed to analyze a person’s changing biology and uses novel 5hmC-based epigenomic analysis to detect when cells become cancerous. The company has a prospective, large clinical validation study underway to further study its test. About Bluestar Genomics Bluestar Genomics is an early cancer detection company focused on the development and commercialization of non-invasive epigenomic tests to detect cancer through a standard blood draw, earlier than existing methods and when the disease is still treatable. The company uses its one-of-a-kind epigenomic platform that combines best-in-class bioinformatics and genomic technologies to analyze individuals’ changing biology that is yet to produce symptoms. Leveraging its novel liquid biopsy technology, Bluestar Genomics is initially focused on high-mortality cancers with the greatest need for early detection, starting with pancreatic and women’s cancers. With locations in San Diego and the San Francisco Bay Area, Bluestar Genomics collaborates with top research institutions and is supported by multiple global healthcare and technology investors and pharmaceutical collaborations.

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BUSINESS INSIGHTS

BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A

BioMarin Pharmaceutical Inc. | November 24, 2022

BioMarin Pharmaceutical Inc. announced advancements in the U.S. Food and Drug Administration review of the Biologics License Application of ROCTAVIAN™ for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date. The Company also remains on track to host the scheduled FDA Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility located in Novato, CA. "The review of a BLA is a dynamic process, and we appreciate FDA's ongoing engagement as we work toward delivering a potentially transformative treatment choice to those patients with severe hemophilia A. We look forward to further dialogue with the Agency as it reviews our application." Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin About valoctocogene roxaparvovec The FDA granted Regenerative Medicine Advanced Therapy designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017. In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN™ on August 24, 2022. Robust Clinical Program BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A. Also ongoing is a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors. About Hemophilia A People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate or mild disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life. Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases.

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Spotlight

Manufacturing drugs for paediatric applications comes with a unique set of challenges. Notably, the need to counteract the bitterness of active pharmaceutical ingredients, and enhance solubility to reduce the risk of delayed onset of action and local tissue injury.

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