Allogene, Notch Partner to Develop iPSC-Derived Blood Cancer Therapies

GEN | November 05, 2019

Allogene Therapeutics will partner with Notch Therapeutics to research and develop induced pluripotent stem cell (iPSC) AlloCAR™ therapies for initial blood cancer indications that include non-Hodgkin lymphoma, leukemia and multiple myeloma, the companies said today, through an exclusive worldwide collaboration and license agreement that could generate more than $300 million for Notch. Under their partnership, Allogene and Notch plan to create allogeneic cell therapy candidates from T cells or natural killer (NK) cells using Notch’s Engineered Thymic Niche (ETN) platform, created to offer potential flexibility and scalability for the production of stem cell-derived immune cell therapies. ETN is designed to enable the expansion of induced pluripotent stem cells (iPSCs) and differentiation of iPSCs from mature immune cells in scalable, fully defined, feeder-free and serum-free cultures into T cells that can be genetically tailored for any T cell-based immunotherapeutic application. The platform uses genetically tailored stem cells as a renewable source for creating allogeneic T cell therapies that are designed, according to Notch, to deliver safer, consistently manufactured and more cost-effective cell immunotherapies to patients.

Spotlight

A convergence of biological data and computing power is yielding amazing technological progress in clinical research and development. Vast amounts of data, often from disparate sources, are enabling the transformation of treatment paradigms. In many therapeutic areas, with oncology chief among them, traditional broad-spectrum therapies that produce variable or unsatisfactory outcomes in large populations are being supplanted by precision medicines that produce improved patient outcomes in targeted populations.

Spotlight

A convergence of biological data and computing power is yielding amazing technological progress in clinical research and development. Vast amounts of data, often from disparate sources, are enabling the transformation of treatment paradigms. In many therapeutic areas, with oncology chief among them, traditional broad-spectrum therapies that produce variable or unsatisfactory outcomes in large populations are being supplanted by precision medicines that produce improved patient outcomes in targeted populations.

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INDUSTRIAL IMPACT

Rocket Pharmaceuticals Completes Acquisition of Renovacor

Rocket Pharmaceuticals, Inc. | December 02, 2022

Rocket Pharmaceuticals, Inc. a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that it has completed the previously announced acquisition of Renovacor, Inc. a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. Under the terms of the merger agreement entered into on September 19, 2022, Renovacor shareholders received, for each share of Renovacor common stock, 0.1763 shares of Rocket common stock. The acquisition provides Rocket with Renovacor’s most advanced program, REN-001, an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. BAG3-DCM represents a significant unmet medical need in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist. Additionally, Rocket gains access to world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities altogether further extend Rocket’s leadership position in AAV-based cardiac gene therapy and help advance the Company’s goal of pursuing gene therapy cures for patients living with rare and devastating diseases. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has a preclinical AAV-based gene therapy program in BAG3-associated dilated cardiomyopathy.

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CELL AND GENE THERAPY

Emergent BioSolutions Completes Acquisition of Exclusive Worldwide Rights to TEMBEXA® First FDA-Approved Smallpox Oral Antiviral for All Ages

TEMBEXA and Smallpox | September 27, 2022

Emergent BioSolutions Inc. announced that it has completed its acquisition of exclusive worldwide rights to TEMBEXA® the first oral antiviral approved by the U.S. Food and Drug Administration for all age groups for the treatment of smallpox, from Chimerix. TEMBEXA was approved in June 2021 and is indicated for the treatment of human smallpox disease in adult and pediatric patients, including neonates. The completion of the acquisition follows the satisfaction or waiver by the parties, as applicable, of all closing conditions, including expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act), as amended, and receipt of consent from the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services, for a sub-contract agreement between Chimerix and Emergent. “The addition of TEMBEXA to our smallpox medical countermeasure franchise, which consists of our smallpox vaccine and therapeutic for smallpox vaccine complications, creates a more comprehensive offering to combat this deadly public health threat. We look forward to supporting the U.S. government’s smallpox preparedness strategy on a broader scale by executing on this BARDA contract.” Paul Williams, SVP government/MCM business at Emergent The 10-year contract valued at up to $680 million, to supply up to 1.7 million treatment courses of tablet and suspension formulations of TEMBEXA® to the U.S. government, was awarded to Chimerix on August 29, 2022. The contract includes an initial product procurement valued at approximately $115 million, with optional future procurement, valued at up to approximately $551 million, exercisable at the sole discretion of BARDA. In addition to product procurement, the contract includes reimbursed post marketing activities of approximately $12 million. Financial Terms Based on the terms of the final BARDA agreement, Emergent is expected to pay Chimerix An upfront payment of $238 million; Potential milestone payments of up to $124 million contingent on the potential exercise by the U.S. government of procurement options following the base period; 15% royalty on gross profit from sales of TEMBEXA outside the U.S.; 20% royalty on gross profit from sales of TEMBEXA in the U.S. that are in excess of the 1.7 million treatment courses as contemplated in the existing BARDA contract; and Up to an additional $12.5 million upon achievement of certain development-based milestones. ABOUT TEMBEXA TEMBEXA is an oral antiviral approved by the FDA in June 2021 for the treatment of human smallpox disease caused by variola virus in adult and pediatric patients, including neonates. TEMBEXA is formulated as 100 mg tablets and 10 mg/mL oral suspension dosed once weekly for two weeks. The oral suspension formulation is particularly important for patients who have difficulty swallowing due to age or medical status. TEMBEXA is not indicated for the treatment of diseases other than human smallpox disease. The effectiveness of TEMBEXA for the treatment of smallpox disease has not been determined in humans because adequate and well-controlled field trials have not been feasible and inducing smallpox disease in humans to study the drug’s efficacy is not ethical. TEMBEXA efficacy may be reduced in immunocompromised patients based on studies in immune deficient animals. TEMBEXA has a BOXED WARNING for increased risk for mortality when used for longer duration. About Smallpox Smallpox is a highly contagious disease caused by the variola virus. Historically, smallpox was one of the deadliest diseases in history with a case fatality rate of approximately 30%. Despite successful eradication of smallpox in the 1970s, there is considerable concern that variola virus could reappear, either through accidental release or as a weapon of bioterrorism. According to the U.S. Centers for Disease Control and Prevention variola virus is ranked in the highest risk category for bioterrorism agents due to its ease of transmission, high mortality rate, and potential to cause public panic and social disruption. Based on a recent report – The Department of Health and Human Services Fiscal Year 2023 Public Health and Social Services Emergency Fund Justification of Estimates for Appropriations Committee – smallpox remains a threat of high concern to both the domestic and international community. About Emergent BioSolutions At Emergent, our mission is to protect and enhance life. For over 20 years, we’ve been at work defending people from things we hope will never happen—so we are prepared just in case they ever do. We provide solutions for complex and urgent public health threats through a portfolio of vaccines and therapeutics that we develop and manufacture for governments and consumers. We also offer a range of integrated contract development and manufacturing services for pharmaceutical and biotechnology customers.

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CELL AND GENE THERAPY

Lunaphore and Nucleai announce a partnership to provide AIpowered spatial biology analysis to accelerate drug development

Lunaphore and Nucleai | September 09, 2022

Lunaphore, a Swiss life sciences company developing technology to enable spatial biology in every laboratory, and Nucleai, a leader in AI-powered spatial biology transforming precision medicine by unlocking the power of pathology data announced a collaboration to accelerate the discovery of novel biomarkers and drug targets using the latest spatial imaging and machine learning technologies. “We are thrilled to announce the partnership with Lunaphore and combine Lunaphore’s best-in-class flagship COMET™, a hyperplex staining and imaging platform, with Nucleai’s ATOM platform that uniquely supports multiplex, IHC, and H&E data. This strategic partnership will allow us to utilize multiplex technology and provide a complete, actionable, and scalable solution to improve drug target discovery and development of our pharma and biotech partners.” Avi Veidman, Chief Executive Officer of Nucleai Mapping biological microenvironments with spatial mapping technology is an exciting area of discovery. Lunaphore’s novel COMET™ technology unlocks the power of immunofluorescence spatial biology with a robust and user-friendly system, permitting the use of any non-conjugated antibodies and enabling the wide adoption of spatial biology in laboratories. Nucleai has built a platform that makes spatial analysis scalable and operational, enabling the next generation of actionable insights from massive pathology data sets that have not been analyzed to their fullest potential and could provide significant value to pharmaceutical companies and diagnostic labs. The partnership will utilize Lunaphore’s innovative COMET™ platform for hyperplex staining and imaging with Nucleai’s cutting-edge AI spatial models to derive new insights from tissue biopsies, including novel drug targets, mechanisms of action, and biomarkers to advance the field of precision medicine. The combined solutions will provide laboratories with an integrated end-to-end spatial biology workflow from automated, hyperplex sequential immunofluorescence staining and imaging to AI-enabled, state-of-the-art image processing, and data analytics. As part of the partnership, the companies also plan to develop predictive and prognostic spatial biomarker assays. “Our partnership with Nucleai is based on our shared vision to advance next-generation spatial multiplex immunofluorescence imaging to accelerate drug and biomarker discovery and development,” said Déborah Heintze, Chief Marketing Officer of Lunaphore. “Connecting Nucleai’s solution with COMET™, we have the potential to more precisely characterize the immune system and disease microenvironment to provide deeper biological insights to drug developers.” “Nucleai brings innovative spatial biology and machine learning platform (ATOM) to empower researchers with novel insights into drug discovery,” said Mridula Iyer, Ph.D., Vice President of Strategic Partnerships at Nucleai. “The technology is designed to unlock and analyze valuable data from pathology slides previously inaccessible, leading to the development of new precise targeted therapy that is important for patient outcomes. This collaboration is another example of how both Lunaphore and Nucleai are accelerating efforts to partner with pharmaceutical and biopharmaceutical companies, as well as medical research institutions and other biomedical organizations.” About COMET™ COMET™ is a fully automated sequential immunofluorescence instrument, able to perform hyperplex staining and imaging, producing high-quality data in a robust and reproducible manner. With superior tissue profiling capabilities, the system allows multiplex analysis of up to 40 different spatial markers per tissue slide without human intervention. COMET™ has a wide range of research applications, allowing for a dramatic improvement in the understanding of disease pathology in areas such as immuno-oncology, neuroscience, and infectious diseases. The technology has the ability to revolutionize clinical applications such as drug discovery and biomarker development. About Lunaphore Lunaphore Technologies S.A. is a Swiss company born in 2014 with the vision of enabling spatial biology in every laboratory. Lunaphore has developed a game-changing chip technology that can extract spatial proteomic and genomic data from tumors and transform any simple assay into multiplex spatial biology without complexity. Lunaphore empowers researchers to push the boundaries of research to ultimately develop the next generation personalized therapies. About Nucleai Nucleai is an AI-powered spatial biology company with a mission to transform drug development and clinical treatment decisions by unlocking the power of pathology data. Nucleai provides pharmaceutical companies, Contract Research Organizations, and diagnostics laboratories with a state-of-the-art AI platform to improve clinical trials and clinical decision-making.

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