The U.S. FDA has approved its second treatment for an ultra-rare disease in the past three days by greenlighting Veopoz (pozelimab-bbfg) from Regeneron Pharmaceuticals Inc. Approval of the priority BLA for Veopoz, a fully human monoclonal antibody to treat Chaple disease, was announced two days ahead of its Aug. 20 PDUFA date. It is the only FDA-approved therapy for the indication.
Six years of collaboration is ending in a buyout, as Decibel Therapeutics Inc. agreed to be acquired by Regeneron Pharmaceuticals Inc. in a deal valued at up to $213 million, including $109 million in equity, with additional payments via contingent value rights linked to clinical and regulatory milestones for DB-OTO, the lead gene therapy program targeting hearing loss.
Regeneron Pharmaceuticals Inc. has disclosed an antibody-drug conjugate consisting of exatecan covalently linked to the antibody trastuzumab targeting HER2 through a linker reported to be useful for the treatment of cancer.
Strong and complete phase III results for Eli Lilly and Co.’s donanemab for treating early Alzheimer's disease (AD) will no doubt inspire more comparisons with recently approved Leqembi (lecanemab). The newly released data for donanemab show it significantly slowed cognitive and functional decline for those with amyloid-positive early symptomatic AD, which lowered the disease-progression risk.
The roadmap and conservative substitution methods Amgen Inc. laid out to “enable” its genus claims for antibodies that inhibit PCSK9 to lower LDL cholesterol are “little more than two research assignments,” the U.S. Supreme Court said in a unanimous opinion handed down May 18 in Amgen Inc. v. Sanofi SA that gave the win to Sanofi. The roadmap “merely describes step-by-step Amgen’s own trial-and-error method for finding functional antibodies — calling on scientists to create a wide range of candidate antibodies and then screen each to see which happen to bind to PCSK9 in the right place and block it from binding to LDL receptors,” the court said in the decision written by Justice Neil Gorsuch.
Dupixent (dupilumab) continues to expand its scope, this time potentially leading it and its developers into a new, multibillion dollar blockbuster market. Dupixent hit the primary and all key secondary endpoints in a phase III study of treating chronic obstructive pulmonary disease (COPD).
As broader markets struggled throughout 2022, the biopharma industry’s largest and most lucrative companies ended the year in a strong position, according to BioWorld’s Biopharmaceutical Index (BBI), which showed the 22 component stocks climbed by 17.3% throughout the year. Neither the Nasdaq Biotechnology Index (NBI) nor the Dow Jones Industrial Average (DJIA) demonstrated such gains. They were down by 10.9% and 8.8%, respectively. All three indices, however, are following the same path in 2023, shooting up throughout the month of January and back down in recent weeks.
Using a near-atomic resolution cryo-electron microscope and imaging techniques that prevent loss of information, scientists at the La Jolla Institute for Immunology (LJI) and Regeneron Pharmaceuticals Inc. have obtained the complete 3D structure of the glycoprotein of the Ebola virus and that of the drug that neutralizes it, Inmazeb, the first FDA-approved treatment for this deadly virus. “The challenge was embracing the inherent asymmetry, the heterogeneity that is really there in biology, understanding it and collecting enough data to get all the images without needing to force any symmetry averaging,” senior author Erica Ollmann Saphire told BioWorld.
The interleukin-2 receptor subunit γ (IL-2RG), also known as γc cytokine receptor or CD132, family of cytokines includes interleukin-2 (IL-2), IL-4, IL-7, IL-9, IL-15 and IL-21. These γc cytokines exert broad pleiotropic effects on the innate and adaptive immune system, and they all share the IL-2RG chain as part of its signaling receptor complex. Researchers from Regeneron Pharmaceuticals Inc. aimed to assess whether targeting γc cytokines may serve as a strategy for the prevention and treatment of T-cell-mediated disease.
Where European regulatory decisions were concerned, there was good news and bad news for pharma today as Sanofi SA and Regeneron Pharmaceuticals Inc. got the go-ahead for expanded approval of Dupixent (dupilumab) in pediatric atopic dermatitis patients whilst Ipsen SA’s ultra-rare bone disease drug palovarotene was left off the shelf as efficacy data failed to impress.
