Biopharma Spotlight: Pfizer’s Hemophilia B Gene Therapy, Bernie Sanders’ Novo Nordisk Investigation, Regeneron and Mammoth’s Partnership
A look at three of today’s top biopharma stories
Spotlight
The U.S. FDA approved Pfizer’s Beqvez (fidanacogene elaparvovec-dzkt) for adults with moderate to severe hemophilia B who use factor IX (FIX) prophylaxis therapy, or who have current or a history of life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and who do not have neutralizing antibodies to AAV serotype Rh74var capsid. Whew!
Beqvez is believed to be a one-time treatment, an AAV-based gene therapy that introduces a functional copy of the FIX gene encoding a high-activity FIX variant.
The first gene therapy approved for hemophilia B was CSL’s Hemgenix, approved in 2022. Hemgenix costs $3.5 million. Although Pfizer didn’t announce a price, it has indicated it is offering a warranty program based on length of patient response.
Bernie Sanders Launches Investigation into Price of Ozempic and Wegovy in the US
Sen. Bernie Sanders (I-Vt), who is also Chairman of the Senate Health, Education, Labor, and Pensions (HELP) Committee, is launching an investigation into what he calls “outrageously high prices” of Novo Nordisk’s type 2 diabetes and obesity drugs, Ozempic and Wegovy, in the U.S. Currently, according to Sanders office, the company charges patients in the U.S. with type 2 diabetes $969 a month for Ozempic, which is only $155 in Canada and $59 in Germany (hello, socialized medicine!). The company also charges obesity patients in the U.S. $1,349 a month for Wegovy while only charging $140 in Germany and $92 in the UK.
In a statement, Sanders said, “In my view, the American people should not have to pay up to $1,349 a month for prescription drugs that cost less than $5 to manufacture and can be purchased at a fraction of this price in other developed countries.”
Regeneron and Mammoth Partner on Next-Gen CRISPR-Based Gene Editing
Regeneron Pharmaceuticals (Tarrytown, NY) announced it inked a partnership with Mammoth Biosciences. Regeneron is working on AAV vectors using antibody-based targeting to improve gene therapy delivery. Mammoth is developing novel ultracompact nucleases and related gene editing systems that are generally smaller than other CRISPR-based systems. Under the deal, Regeneron is paying Mammoth $100 million inclusive of $95 million in equity investment at signing, and an upfront payment. Mammoth is eligible for $370 million per target in various milestones as well as royalties ranging from single digits to mid-teens. Mammoth also has the right to opt-in to co-funding and profit sharing on a majority of collaboration programs instead of the milestones and royalties. Regeneron picks up 5-1/2 years of access to Mammoth’s editing tech.
“We believe in the incredible power of gene editing, which we are utilizing in our diverse preclinical and clinical genetic medicines pipeline,” said Christos Kyratsous, PhD, SVP and co-Head of Regeneron Genetic Medicines. “After years spent developing our next-generation delivery approaches, we are eager to combine them with Mammoth’s gene editing systems to better match payload, delivery system and disease type.”