Pharmaceutical Technology Editors

FDA announced the approval of CSL’s Hemgenix (etranacogene dezaparvovec), the first one-time gene therapy for adults with hemophilia B, on Nov. 22, 2022. The treatment is approved for use in adults with hemophilia B who currently use factor IX prophylaxis therapy, or those who have had life-threatening hemorrhage or repeated spontaneous bleeding episodes. The therapy will cost approximately $3.5 million, making it the most expensive single-use therapy in the United States.

According to the agency press release, Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding. In an ongoing clinical trial, etranacogene dezaparvovec reduced the rate of annual bleeds by 54%. Additionally, 94% percent of patients were able to discontinue factor IX prophylaxis and remained prophylaxis-free.

“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,” said Peter Marks, director, FDA Center for Biologics Evaluation and Research, in the release. “Today’s approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia.”

“As part of our promise to patients, CSL is committed to delivering innovative and groundbreaking solutions to address unmet medical needs, and we are proud to introduce the next wave of breakthrough medicines for people living with hemophilia B,” said Paul Perreault, CEO and managing director, CSL, in a company press release. “We recognize and thank all trial participants, scientists and investigators—without whom this important achievement would not have been possible—and look forward to seeing the positive impact of HEMGENIX on the hemophilia B community.”

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CSL’s Hemgenix (etranacogene dezaparvovec) will cost approximately $3.5 million, making it the most expensive single-use medicine in the United States.

FDA Approves $3.5 Million Hemophilia B Gene Therapy

Merck, known as MSD outside of the United States and Canada, announced its intention to acquire Imago BioSciences, a clinical-stage biopharmaceutical company focused on bone marrow disorders, on Nov. 21, 2022. Per the terms of the definitive merger agreement, Merck will acquire Imago for approximately $1.35 billion in total equity. The transaction is expected to close in the first quarter of 2023.

According to a company press release, Imago specializes in developing new medicines for the treatment of myeloproliferative neoplasms and other bone marrow diseases. Imago’s lead candidate is bomedemstat, an investigational, orally available lysine-specific demethylase 1 (LSD1) inhibitor. Due to the role that LSD1 plays in the function of malignant blood cells, targeting it is intended to provide a new mechanism of treatment for various blood cancers. It is currently being evaluated in multiple Phase II clinical trials for the treatment of essential thrombocythemia, myelofibrosis, and polycythemia vera, among other indications.

“We continue to invest in our pipeline with a focus on applying our unique capabilities to unlock the value of breakthrough science for the patients we serve,” said Robert M. Davis, president and CEO, Merck, in the release. “This acquisition of Imago augments our pipeline and strengthens our presence in the growing field of hematology.”

“This milestone is a testament to more than a decade of pioneering research by Imago scientists and the entire Imago team’s unwavering dedication to improving the lives of patients,” said Hugh Y. Rienhoff Jr., founder and CEO, Imago BioSciences, in the release. “This agreement leverages Merck’s industry-leading clinical development expertise to maximize the therapeutic potential of bomedemstat while providing important value for shareholders.”

Merck will purchase Imago BioSciences, a biotechnology company focused on bone marrow disorders, for approximately $1.35 billion. 

Merck Announces $1.35 Billion Acquisition of Imago BioSciences

Sartorius BIA Separations (BIA), a bio-chromatography company, and Exopharm, a company focused on medicines using exosomes, or extracellular vesicles, announced on Nov. 22, 2022 that they have entered into a formal collaboration. The companies have signed a Material Transfer Agreement that aims to use Exopharm’s patented LEAP technology alongside BIA’s Convective Interaction Media chromatography for improved large-scale therapeutic exosome production and commercialization.

According to a company press release, exosomes have advantages over alternative nanoparticle delivery technologies, as they are non-toxic, efficient at delivering RNA cargoes into cells, and able to do so without eliciting an immune response. Exosomes can also be engineered to provide specific delivery as well as repeated dosing.

“BIA sees the potential in exosomes for new and innovative therapeutic applications and understands that purification of exosomes has held back their adoption as a non-viral drug-delivery technology,” said

Aleš Strancar, co-founder and managing director of BIA, in the release. “We believe that BIA’s CIM monolith technology and Exopharm’s exosome-specific LEAP ligands can work synergistically together to enhance specificity, efficiency and capacity of industrial exosome purification processes. This work with Exopharm has the potential to be a game-changer in the emerging exosome field.”

The agreement aims to develop integrated technology for large-scale exosome production. 

Sartorius BIA Separations and Exopharm Sign Joint Research Agreement

CSL Vifor and Fresenius Kabi announced on Nov. 28. 2022 that China’s National Medical Products Administration (NMPA) has approved Ferinject (ferric carboxymaltose), a preparation for intravenous iron therapy for the treatment of iron deficiency in adult patients for whom oral iron preparations are ineffective or cannot be used. Ferinject has now received marketing authorization in 85 countries worldwide.

According to a company press release, China has the world’s largest iron deficiency anemia population, with an estimated prevalence of 15%. The treatment’s approval is intended to benefit the Chinese healthcare system, among others, through implementation of a more effective patient blood management. The launch of Ferinject is expected in the first half of 2023.

“We are thrilled about the marketing authorization of Ferinject, which is a milestone for Chinese patients living with iron deficiency or iron deficiency anemia,” said Hervé Gisserot, general manager of CSL Vifor, in the release. “There is a high unmet need in China, which has the world’s largest iron deficiency population, and we are confident that Ferinject can make a meaningful difference in the treatment of these patients. At the same time, this is another important step in our goal to globalizing and maximizing the performance of our iron franchise as we look forward to bring this treatment to market as soon as possible.”

NMPA has approved Ferinject in China for the treatment of iron deficiency or iron deficiency anemia. 

Ferinject Approved in China for the Treatment of Iron Deficiency in Adult Patients

MillporeSigma announced on Nov. 16, 2022 that its life science business sector is investing more than $286 million in its biosafety testing capacity at the company’s site in Rockville, Maryland. A new, state-of-the-art, 250,000 ft2 facility at the site will house biosafety testing, analytical development, viral clearance suites, and cell bank manufacturing services.

According to a company press release, the goal of this investment is to significantly increase the company’s ability to conduct biosafety testing and analytical development services. Over the next four years, the company expects the expansion to create more than 500 new jobs.

“MilliporeSigma is harnessing its broad portfolio of modern technologies and enabling services to help ensure the safety of the world’s medicines. These significant investments in Rockville and other sites across our global network will help us meet growing demand for the robust testing studies that biologics manufacturers depend upon to comply with stringent regulatory guidelines,” said Belén Garijo, chair of the executive board and CEO, Merck KGaA, Darmstadt, Germany, in the release. “This is a prime example of our focus on sustainable growth in areas with strong underlying upwards dynamics.”

“This is the largest investment in contract testing in Merck KGaA, Darmstadt, Germany’s history,” said Matthias Heinzel, member of the executive board, Merck KGaA, Darmstadt, Germany, and CEO, Life Science. “We thank the State of Maryland and Montgomery County, Maryland, for their collaboration in making this new campus a reality for our employees and clients. Together, we are impacting life and health with science.”

The $286 million investment ability is designed to elevate MillioporeSigma’s ability to conduct biosafety testing and analytical development services.

MilliporeSigma Invests $286 Million in US Drug Safety Testing Capacity

Regeneron and CytomX Therapeutics, a clinical-stage biopharmaceutical company focused on oncology, announced a collaboration and licensing agreement on Nov. 17, 2022. The collaboration will see the companies partner to develop investigational next-generation bispecific immunotherapies for cancer using CtyomX’s Probody therapeutic platform and Regeneron’s Veloci-Bi bispecific antibody development platform.

According to a company press release, the agreement will see Regeneron and CytomX collaborate on the discovery activities to identify and validate conditionally active bispecific antibodies. Regeneron will be responsible for funding preclinical and clinical development and commercialization activities. CytomX will receive a $30 million upfront payment with the potential for up to $2 billion in research, development, regulatory, and sales-based milestones, as well as tiered global net sales royalties.

The partnership intends to use CytomX’s biologic masking strategies to develop investigational Regeneron bispecifics that remain inactive until activated by proteases in the tumor microenvironment. According to the release, this technology would have the potential to widen the therapeutic window and help minimize off-target effects for next-generation T-cell engaging therapies, allowing them to potentially address tumor types typically unresponsive to immunotherapy.

"At Regeneron, we're focused on developing a paradigm-changing portfolio of oncology medicines for patients, by combining a deep understanding of cancer biology with cutting-edge technologies," said John Lin, senior vice-president of Immuno-oncology, head of Bispecifics, Regeneron, in the release. "This collaboration will enable Regeneron and CytomX to combine our collective oncology expertise with two premier platforms – Probody and Veloci-Bi – to develop novel immunotherapies and research their potential to transform patient lives."

"CytomX has pioneered the field of conditionally-activated therapeutics through high quality and differentiated science, leading to broad experience in biologic masking strategies and a deep understanding of the protease tumor microenvironment," said Sean McCarthy, CEO and chairman, CytomX, in the release. "We are thrilled that our scientific expertise has attracted Regeneron as our newest collaborator, and we look forward to working closely together to further optimize T-cell engager strategies and push the boundaries of cancer immunotherapy to new levels."

The collaboration will see the companies develop investigational next-generation bispecific immunotherapies for cancer.