The Centers for Medicare & Medicaid Services (CMS) disclosed 10 drugs covered under part D of Medicare that, for the first time, the agency can negotiate under the Inflation Reduction Act. Bargaining with drug companies will take place this year and next, CMS said, and the arrived-at prices will become effective starting in 2026. People on Medicare who have used the 10 drugs paid a total of $3.4 billion out-of-pocket for them in 2022.

With a new approval, BMS’s Reblozyl expands anemia treatment 

The U.S. FDA has awarded Bristol Myers Squibb Co. (BMS) with its third approval for treating anemia with Reblozyl (luspatercept-aamt). Specifically, the approval is for treating anemia without previous erythropoiesis stimulating agent use in adults with very low- to intermediate-risk myelodysplastic syndromes who may require regular red blood cell (RBC) transfusions. The FDA accepted the sBLA in May, which is about the same time the EMA validated the drug’s type II variation application, and then set an Aug. 28 PDUFA date. BMS announced the approval late in the evening of the PDUFA date. Reblozyl was first approved in November 2019, partnered with Celgene Corp. for anemia in adults with beta-thalassemia who require regular RBC transfusions. Added to the label in April 2020 was anemia in patients failing an erythropoiesis stimulating agent and requiring two or more RBC units over eight weeks in very low-to- intermediate-risk myelodysplastic syndromes with ring sideroblasts as well as those with myelodysplastic-myeloproliferative neoplasm with ring sideroblasts and thrombocytosis.

Lib’s lerodalcibep hits co-primary endpoints in phase III HeFH trial

Heterozygous familial hypercholesterolemia (HeFH) patients treated with lerodalcibep achieved a 58.6% reduction in LDL-cholesterol at week 24 and a 65% reduction at the mean of weeks 22 and 24 in the phase III trial Liberate-HeFH. The developer, Lib Therapeutics Inc., was founded in 2015 when it licensed the technology from Bristol Myers Squibb Co. but is just now emerging from stealth. In the past eight years, it has advanced its only product through preclinical and early clinical studies to phase III trials, with regulatory filings in both the U.S. and Europe targeted for early 2024. “Our drug is not a monoclonal antibody,” said Evan Stein, CEO and chief scientific officer of Lib. “It’s a unique small binding protein that blocks PCSK9 in the bloodstream just like a monoclonal antibody (MAb), but it’s a small injection.” In the trial, lerodalcibep was dosed at 300 mg monthly in a 1.2-mL subcutaneous injection. “It’s also stable at room temperature. MAbs have to be refrigerated.”

Verastem, Genfleet to develop compounds targeting RAS pathways in deal worth up to $625M

Verastem Oncology Inc. and Genfleet Therapeutics Ltd. have inked a discovery and development deal to advance three oncology discovery programs targeting RAS pathway-driven cancers. The collaboration brings together Verastem’s clinical and regulatory expertise and Genfleet’s discovery capabilities, while also leveraging Verastem’s experience in RAS biology and RAS pathway-dependent cancers and Genfleet’s accomplishments with its KRAS G12C inhibitor program. The agreement provides for up-front, research support and option payments to Genfleet, with potential total deal size across all three programs up to $625.5 million.

Methodological, target breadth begets optimism for movement disorder progress

At the 2023 International Congress of Parkinson’s Disease and Movement Disorders, which is being held in Copenhagen this week, Lorraine Kalia, a scientist at Toronto Western Hospital’s Krembil Brain Institute and at the University of Toronto’s Tanz Centre for Research in Neurodegenerative Diseases, gave an overview of “Emerging targets in the clinic” in a plenary session on “Therapeutic strategies for the future.”

Also in the news

Abbvie, Amgen, Anixa, Apellis, Aprinoia, Arbor, Atsena, Biostax, Bluerock, Bridgebio, Celularity, Citryll, Confo, Cybin, Daiichi Sankyo, Deargen, Elite, Ena Respiratory, Evaxion, Hanchorbio, Harbour, Henlius, I2o, Immgenuity, Inhibrx, Lytix, Mallinckrodt, Medipacific, Mithra, Neurocrine, Northstar, Novartis, Nrx, Omega, Onl, Pardes, Pharma Two B, Recce, Regeneron, Serengen, Sinovac, Small, Tourmaline, Ubiquigent, Valneva, Xylocor