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 | Read time : 04:00 min

Emergent BioSolutions
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.

Spotlight

Cell culture is practiced extensively throughout the world today. The techniques required to allow cells to grow and be maintained outside the body have been developed throughout the 20th century. In the 50 years since the publication of the first human cancer cell line, HeLa (1), thousands of cell lines representing most of the spectrum of human cancer have been derived.

Spotlight

Cell culture is practiced extensively throughout the world today. The techniques required to allow cells to grow and be maintained outside the body have been developed throughout the 20th century. In the 50 years since the publication of the first human cancer cell line, HeLa (1), thousands of cell lines representing most of the spectrum of human cancer have been derived.

Related News

CELL AND GENE THERAPY

Biocytogen Enters into Antibody Agreement with ADC Therapeutics

Biocytogen | November 28, 2022

Biocytogen Pharmaceuticals Co., Ltd. announced that it has entered into an evaluation and option agreement with ADC Therapeutics SA. Biocytogen will grant ADC Therapeutics a license to evaluate Biocytogen’s proprietary antibodies against three tumor targets, with an option to license selected antibodies at a later date for global ADC development and commercialization. Biocytogen reserves all global rights for these antibodies beyond ADC development. Biocytogen will receive an upfront payment. For each option exercised, Biocytogen will be entitled to an option-exercise fee, and development and commercial milestone payments, which potentially total tens of millions of US dollars, as well as single-digit royalties on net sales. Under the agreement, Biocytogen will provide ADC Therapeutics with monoclonal and/or biparatopic antibodies directed against three tumor targets, generated through Biocytogen’s Project Integrum. ADC Therapeutics will carry out feasibility studies and be responsible for further development and commercialization of the ADC products if the company exercises an option. “ADC Therapeutics is a leader in the development and commercialization of ADCs. We believe this collaboration with ADC Therapeutics will underscore the value of Biocytogen’s proprietary antibody discovery and screening platforms. We’ve been dedicated to expanding the possibilities of antibody-based therapeutics in disease treatment, through the exploration of new types of targets and modalities through Project Integrum. The combination of Biocytogen’s strength in antibody development with ADC Therapeutics’ expertise and track record in payload and linker technology may lead to the generation of novel ADC medicines for unmet medical needs in the future.” Dr. Yuelei Shen, President and CEO of Biocytogen “We are pleased to enter this collaboration with Biocytogen as we continue expanding our toolbox to realize the full potential of our ADC platform,” said Patrick van Berkel, PhD, Chief Scientific Officer of ADC Therapeutics. “We look forward to evaluating the fully human antibodies from Biocytogen’s proprietary discovery platform, which have the potential to accelerate the development of our novel ADCs.” About Biocytogen Biocytogen is a global biotechnology company that drives the research and development of novel antibody-based drugs with innovative technologies. Using its proprietary RenMabTM /RenLite® mice platforms for fully human monoclonal and bispecific antibody development, Biocytogen has integrated its in vivo drug efficacy screening platforms and strong clinical development expertise to streamline the entire drug development process. Biocytogen is undertaking a large-scale project to develop first-in-class and/or best-in-class antibody drugs for more than 1000 targets, known as Project Integrum. This project has resulted in 28 drug co-development agreements and 16 RenMiceTM licensing agreements with companies around the world, including several partnerships with multinational pharmaceutical companies (MNCs). The company's pipeline includes 12 core products, among which two products are in phase II multi-regional clinical trials and two products are in phase I. Headquartered in Beijing, Biocytogen has branches in Haimen Jiangsu, Shanghai, Boston, USA and Heidelberg, Germany. About ADC Therapeutics ADC Therapeutics is a commercial-stage biotechnology company improving the lives of those affected by cancer with its next-generation, targeted antibody drug conjugates. The Company is advancing its proprietary PBD-based ADC technology to transform the treatment paradigm for patients with hematologic malignancies and solid tumors. ADC Therapeutics’ CD19-directed ADC ZYNLONTA is approved by the FDA for the treatment of relapsed or refractory diffuse large b-cell lymphoma after two or more lines of systemic therapy. ZYNLONTA is also in development in combination with other agents. In addition to ZYNLONTA, ADC Therapeutics has multiple ADCs in ongoing clinical and preclinical development.

Read More

CELL AND GENE THERAPY

Rocket Pharmaceuticals to Acquire Renovacor, Extending Leadership in AAV-based Cardiac Gene Therapy

Rocket Pharmaceuticals, Inc. | September 21, 2022

Rocket Pharmaceuticals, Inc. a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, and 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, today announced a definitive agreement under which Rocket will acquire Renovacor in an all-stock transaction for an implied value of approximately $2.60 per share, based on the volume weighted average trading price of Rocket shares of $15.51 for the 30 trading days through and including Monday, September 19, 2022. The boards of directors of both companies have unanimously approved the transaction, which is currently expected to close by the first quarter of 2023. “The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for cardiac disease and gives us a perfect opportunity to continue on our mission to transform the lives of heart failure patients through the power of gene therapy. Building on our success in Danon Disease to date, I am particularly excited to expand our cardiology focus and capabilities and address a clear unmet medical need in BAG3-associated dilated cardiomyopathy. By combining Renovacor’s compelling preclinical work with our joint clinical, regulatory and CMC expertise, we believe we will be well-positioned to bring the highest impact gene therapy with the best chance for success to these patients in the most productive and efficient manner possible.” Gaurav Shah, M.D., Chief Executive Officer of Rocket Dr. Shah continued, “Given the positive pediatric safety data previously announced from our Phase 1 RP-A501 Danon Disease program, and the upcoming pediatric efficacy data and longer-term adult cohort data we anticipate presenting at the Heart Failure Society of America (HFSA) Scientific Meeting at the end of this month, this strategic acquisition gives us what we believe is the broadest platform in the field to address these devastating rare cardiac diseases. Furthermore, the acquisition will bring to Rocket key personnel, namely a team of leading cardiology drug development experts, critical capabilities, and valuable IP to support continued development of the BAG3 as well as other potential cardiac programs, including a gene therapy research collaboration for arrhythmogenic cardiomyopathy.” Renovacor’s most advanced program, REN-001, is 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. Renovacor has deep technical expertise in the development of precision therapies that address genetically driven cardiac diseases. Further, Renovacor is supported by world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities, all together, represent tremendous value and will enhance Rocket’s leading position in cardiac AAV-based gene therapy. “Renovacor has made tremendous progress in advancing targeted gene therapies to address the high unmet medical needs of patients living with genetically driven forms of heart disease,” said Magdalene Cook, MD, Chief Executive Officer of Renovacor. “Our experienced team is excited to join Rocket in a shared vision of broadening patient access to precision medicines for cardiovascular disease and addressing common barriers jointly. We look forward to combining the considerable resources and expertise of Renovacor and Rocket in creating a category leader in the precision cardiology field. As a result of this combination, we will be suspending current guidance regarding preclinical and clinical timelines for our programs as we evaluate these items with the Rocket team.” It is currently anticipated that the transaction will close by the first quarter of 2023, subject to approval by Renovacor and Rocket shareholders, receipt of any required customary regulatory approvals and the satisfaction of other customary closing conditions. RTW Investments, LP, a significant shareholder of both Rocket and Renovacor, has entered into a voting agreement with Renovacor, pursuant to which they have agreed, among other things, and subject to the terms and conditions of the agreement, to vote in favor of the Renovacor acquisition as a Rocket stockholder. SVB Securities is serving as exclusive financial advisor and Goodwin Procter LLP is serving as legal counsel to Rocket. Wells Fargo Securities is serving as exclusive financial advisor and Troutman Pepper Hamilton Sanders LLP is serving as legal counsel to Renovacor. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of genetic therapies that 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. About Renovacor Renovacor is 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. The company’s lead program in BAG3-associated dilated cardiomyopathy (DCM) uses gene transfer technology to address the monogenic cause of this severe form of heart failure. Renovacor’s vision is to bring life-changing therapies to patients living with serious genetic cardiovascular and related diseases, by developing medicines that target the underlying cause of disease and provide a transformative benefit and significant improvement to quality of life.

Read More

INDUSTRIAL IMPACT

Alloy Therapeutics Raises $42 Million Series D Financing to Accelerate Discovery Technology Development Across Biologic Modalities

Alloy Therapeutics, Inc. | October 04, 2022

Alloy Therapeutics, a biotechnology ecosystem company, has closed $42 million in Series D financing led by its existing investors 8VC and Mubadala Capital and joined by return investors Thiel Capital, Presight Capital, Founders Fund, and other unnamed family offices and sovereign wealth funds. Alloy will use proceeds from the Series D financing to further support the drug discovery community with new pre-competitive drug discovery technologies in new biologic modalities and new partnership models designed to fuel innovation. Since Alloy’s Series C fundraise in March 2021, Alloy has expanded from its foundational antibody discovery technologies and discovery services into T-cell receptors with the launch of its Keyway TCR Discovery division, and genetic medicines through its collaboration with Dr. Sudhir Agrawal, the inventor of gapmer technology. Alloy has further expanded with three new research sites, in Basel, CH; Athens, GA; and San Francisco, CA. “After three years of hard work and planning, we were proud to formally launch Keyway TCR Discovery in January 2022. We envision a future in 20 years where engineered TCRs and TCR mimics are as successful a modality as monoclonal antibodies are today. Keyway believes this is possible by providing unrestricted access to enabling technologies and discovery services without pharma having to spend billions of dollars to acquire companies for proprietary technologies and people.” Dongxing Zha, CEO of Keyway and Alloy’s CTO for TCR Modalities In response to growing engagement from partners, Alloy will further leverage its Series D funds to continue to invest in expanded offerings across all six of its modalities. These will support the drug discovery community in the pursuit of multi-modal therapeutics while reducing or eliminating stacked downstream payments. Through its affiliated venture studio, 82VS, Alloy supports scientist-entrepreneurs with seamless access to its cutting-edge technologies and discovery services. 82VS companies are often the first customers of Alloy’s innovations, having full access to the Alloy suite of technologies and discovery services to advance the best drug candidates and invent new applications that can benefit the broader Alloy ecosystem and, ultimately, patients and their caregivers. To best serve the vast discovery needs of pharmaceutical companies, Alloy is pioneering an Innovation Subscription offering that gives its collaborators access to all current and future Alloy technology platforms for one flat, annual fee with no milestones or royalties. Partners benefit from Alloy as an ever-growing source of pre-competitive, foundational discovery technology. Alloy also supports complementary CROs, CDMOs and other discovery and development service providers as a critical part of the collaborative ecosystem to empower pharma in its mission to cure disease. In this subscription model, Alloy provides a new home for promising pre-competitive technologies and academic breakthroughs that have traditionally been underfunded or unavailable to all. “Our Innovation Subscription model is available now as an exciting next step in furthering our collaborative mission and unlocking the potential for sharing foundational, pre-competitive drug discovery technologies with the global drug discovery community,” said Alloy Therapeutics Chief Executive Officer and Founder Errik Anderson. “Including this financing, Alloy will have invested hundreds of millions of dollars in innovation that the largest pharma companies can access through a flat, annual subscription with no additional milestones or royalties. Emerging biotech and academics can access our tech through collaboration and more traditional and creative structures. Combined with the reinvestment of 100% of our revenue, including our subscription revenue, back into innovation and access to innovation, Alloy is a new for-profit business model that plays for the collective long game in a non-zero-sum way. The value of a subscription goes up every year, while the fees stay flat or even will decrease with scale. We price this so a single avoided royalty will pay for the entire platform access over a 20-plus-year period. We are grateful for our longstanding investors for supporting this vision of arming the drug discovery industry with the tools they need to discover and develop the best medicines for patients more quickly.” Alloy launched in 2017 to democratize foundational, pre-competitive technologies and capabilities for the discovery of therapeutic biologics and has since grown with over 130 partners across academia, biotech, and large biopharma. Its first platform was the ATX-Gx™, a suite of transgenic mouse strains for human antibody discovery that was originally in-licensed from a major pharma company and made broadly available to the drug discovery ecosystem by Alloy, with open access to newly introduced strains. Through its DeepImmune™ Antibody Discovery services, Alloy helps partners find the best therapeutic antibody candidates against targets of interest, by deploying a bespoke blend of its proprietary in vivo, in vitro, and in silico technologies and workflows. In 2022, Alloy launched Keyway™ TCR Discovery as its second fully integrated technology and services offering for the discovery of therapeutic engineered TCRs and TCR mimics. “Alloy has successfully expanded its flagship discovery offering in antibodies to multiple promising modalities to better serve the global drug discovery community,” said Alaa Halawa, Head of Mubadala Capital’s US Ventures business, which first backed Alloy as an investor in its Series C financing. “We are proud to partner with the company as it executes on its promise of enabling scientists through the development of transformative drug discovery technologies. We look forward to seeing how the partnership with large pharma partners and other venture firms will leverage Alloy’s platform to enable their therapeutic discovery teams and emerging startups to advance the best medicines.” About Alloy Therapeutics aAlloy Therapeutics is a biotechnology ecosystem company empowering the global scientific community to make better medicines together. Through a community of partners across academia, biotech, and the largest biopharma, Alloy democratizes access to pre-competitive tools, technologies, services, and company creation capabilities that are foundational for discovering and developing therapeutic biologics across six modalities, including antibodies, TCRs, genetic medicines, peptides, cell therapies, and drug delivery. Partners may access all current and future technologies for a flat annual fee through Alloy’s Innovation Subscriptions offering. As a reflection of Alloy’s relentless commitment to the scientific community, Alloy reinvests 100% of its revenue in innovation and access to innovation. About Alloy’s Antibody Discovery Offering Alloy’s first foundational platform, the ATX-Gx™ mice, is a suite of proprietary transgenic mice strains for human therapeutic antibody discovery, which can be accessed in your lab or through DeepImmune™ Antibody Discovery. This service integrates Alloy’s full complement of proprietary in vivo, in vitro, and in silico discovery and optimization technologies into one comprehensive service offering for fully human monoclonal and bispecific antibody discovery. The technologies and protocols can be replicated in a partner’s organization through a platform transfer and as part of an Alloy Innovation Subscription. Alloy is a leader in bispecific antibody discovery and engineering services, utilizing its proprietary ATX-CLC common light chain platform integrating novel transgenic mice and phage display. About Keyway™ TCR Discovery In 2022, Alloy launched Keyway™ TCR Discovery, Alloy's second fully integrated platform and service offering. Led by a team of TCR therapeutic pioneers and experts and leveraging the full resources of the Alloy ecosystem, Keyway provides comprehensive proprietary technology and capabilities for end-to-end discovery of TCR mimics and engineered TCRs, which includes production and quality control of MHC/peptide complexes, biophysical characterization, specificity testing, reformatting, and engineering, affinity maturation, and in vivo and in vitro preclinical testing. Partners may access Keyway's technology and capabilities through a standard or bespoke one-stop-shop discovery campaign, platform transfer, or Innovation Subscription.

Read More