CELL AND GENE THERAPY
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.
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.
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.
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.
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.
BriaCell Therapeutics Corp. | December 01, 2022
IPA an advanced biotherapeutic research and technology company, announced that BioStrand BV an AI in silico discovery subsidiary of IPA, has entered into a research collaboration and license agreement with BriaCell Therapeutics Corp. a clinical-stage biotechnology company specializing in targeted immunotherapies for cancer.
The collaboration will leverage BioStrand’s LENSai™ software, built upon IPA’s proprietary HYFT™ Universal Fingerprint™ technologies, and will focus on in silico antibody discovery to generate relevant clinical molecules for potential development. Under the terms of the Agreement, BioStrand and BriaCell will collaborate on the design, discovery, and development of anti-cancer antibodies. Upon successful antibody discovery, BioStrand will receive an upfront payment of US$500,000, and will be eligible to receive future success-based development milestones, including those for the submission of Investigational New Drugs clinical milestone payments, and commercial royalties on net sales of products. Further terms are not disclosed.
“We are very excited to begin this program with BriaCell and apply our technology to the development of biologics that may have a huge clinical impact. Shortening timelines, while also integrating as much information as possible upstream to improve the efficiency of the process, is extremely important in the development of highly targeted therapies. It is a step towards realizing our long-term vision of significantly advancing precision medicine.”
Dr. Ingrid Brands, General Manager and co-founder of BioStrand
“We believe that BioStrand’s revolutionary AI-powered technology, combined with its cutting-edge protein engineering platform, will allow us to design and discover potent anti-cancer therapeutics,” stated Miguel A. Lopez-Lago, PhD, Chief Scientific Officer of BriaCell. “This approach would complement BriaCell’s current immunotherapy pipeline of innovative anti-cancer therapeutics.”
About BriaCell Therapeutics Corp.
BriaCell is an immuno-oncology-focused biotechnology company developing targeted and effective approaches for the management of cancer.
ImmunoPrecise Antibodies Ltd.
ImmunoPrecise Antibodies Ltd. has several subsidiaries in North America and Europe including entities such as Talem Therapeutics LLC, Biostrand BV, ImmunoPrecise Antibodies Ltd. and ImmunoPrecise Antibodies B.V. The IPA Family is a biotherapeutic research and technology group that leverages systems biology, multi-omics modelling and complex artificial intelligence systems to support its proprietary technologies in bioplatform-based antibody discovery. Services include highly specialized, full-continuum therapeutic biologics discovery, development, and out-licensing to support its business partners in their quest to discover and develop novel biologics against the most challenging targets.