In a verbal sparring over who can deliver the lowest drug prices in the U.S., several Senate Democrats are urging President Donald Trump to immediately release the list of second-round Medicare-negotiated drug prices, instead of doing what they characterize as “ambiguous” and “opaque” pricing deals with individual biopharma companies.
“Our goal is that by the end of this call, you’ll share our conviction in the totality of the data.” So said Agios Pharmaceuticals Inc. CEO Brian Goff as the company reported top-line data from its 52-week Rise Up trial testing mitapivat in sickle cell disease showing the oral pyruvate kinase activator hit statistical significance on one of two primary endpoints and two of three key secondary endpoints.
Arrowhead Pharmaceuticals Inc. won U.S. FDA approval of plozasiran in familial chylomicronemia syndrome (FCS), the second drug to gain clearance for use in the rare genetic disease following the late 2024 nod for Ionis Pharmaceuticals Inc.’s Tryngolza (olezarsen), setting up a battle in the marketplace even as the firms wage a patent dispute regarding the two RNA-based therapies.
Shanghai Henlius Biotech Inc. and Organon & Co. announced Nov. 17 that the U.S. FDA cleared Poherdy (pertuzumab-dpzb) as the first and only interchangeable biosimilar to Perjeta (pertuzumab, Genentech Inc./Roche Holding AG).
Top-line results from a midstage study of Vanda Pharmaceuticals Inc.’s tradipitant hit its primary endpoint by preventing the nausea and vomiting that can be caused by the GLP-1 receptor agonist Wegovy (tirzepatide, Eli Lilly and Co.) in overweight and obese adults. The positive phase II study data prompted Vanda to look at a phase III study of the oral neurokinin-1 receptor antagonist next year.
Roche AG’s Genentech unit landed another clinical victory with giredestrant that provided a major stock boost for competitor Olema Pharmaceuticals Inc. Made public were positive phase III data from the Lidera study testing selective estrogen receptor degrader and complete estrogen receptor antagonist (CERAN) as an adjuvant endocrine treatment for people with ER-positive, HER2-negative, early stage breast cancer (BC).
Over a span of five-and-a-half months this year, 3.5% of the more than 11,000 clinical trials funded by the U.S. NIH had their grants terminated, according to an article published in the Nov. 17 JAMA Internal Medicine. That’s 383 trials that lost funding.
The U.S. FDA gave the thumbs up to Kura Oncology Inc./Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor, ziftomenib, to treat relapsed, refractory (r/r) nucleophosmin1 (NMP1)-mutant acute myeloid leukemia (AML). The approval of the drug, branded Komzifti, came more than two weeks ahead of the Nov. 30 PDUFA date.
In an article that reads like informal guidance, U.S. FDA Commissioner Marty Makary and CBER Director Vinay Prasad discussed the criteria for using the agency’s novel plausible mechanism pathway for personalized treatments when a randomized trial isn’t feasible, as well as future uses of the approval path that could expand beyond gene and cell therapies to other biologics and even small molecules.
Korro Bio Inc.’s latest update on RNA editing prospect KRRO-110 may mean one less competitor in alpha-1 antitrypsin deficiency (AATD), and shares of the firm (NASDAQ:KRRO) closed Nov. 13 at $6.50, down $24.92, or 79%. As part of the third-quarter earnings report, Korro said KRRO-110 produced functional protein in AATD patients but fell short of projected levels of functional protein after a single administration.
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