Celltrion Inc. scored a hat-trick of deals to license new antibody candidates, including a $744 million deal with Kaigene Inc. Nov. 3, and a near $500 million deal with Mustbio Co. Ltd. Oct. 31.
Centessa Pharmaceuticals plc’s unremarkable third-quarter 2025 earnings report was greatly overshadowed by an early readout from its orexin receptor 2 (OX2R) agonist program in narcolepsy, particularly promising phase IIa data demonstrating ORX-750’s efficacy across a group of sleep disorders, though investors seek further data to differentiate Centessa’s program from potential competitors such as Takeda Pharmaceutical Co. Ltd.’s oveporexton and Alkermes plc’s alixorexton.
Tulyp Medical Inc. has emerged from stealth mode with a pressure-driven perfusion system designed to improve vascular procedures. The company, born out of Sofinnova Partners' medical device accelerator, has submitted the technology to the U.S. FDA for 510(k) clearance following initial clinical results that demonstrated safety and functionality.
Lumosa Therapeutics Co. Ltd.’s intravenous odatroltide (LT-3001) met the primary endpoints in a phase IIb trial in China in patients with acute ischemic stroke, paving the way for a pivotal phase III study.
Just as investors were looking ahead to news by year-end on Prelude Therapeutics Inc.’s SMARCA2-targeted degraders, the firm said work in the space will be halted, with efforts shifting toward the mutant selective JAK2V617F JH2 inhibitor program by way of a new deal with Incyte Corp.
Five-year follow-up data for Santhera Pharmaceuticals AG’s Duchenne muscular dystrophy (DMD) drug, Agamree (vamorolone), confirm that its efficacy in preserving muscle function is comparable to standard-of-care corticosteroids, but that the overall side-effect profile is more benign. There was less positive DMD news from Sarepta Therapeutics Inc.
Results of the Shortcut study, presented last week at the Transcatheter Cardiovascular Therapeutics meeting in San Francisco, indicated that cutting balloon angioplasty matched intravascular lithotripsy in coronary artery preparation of calcification for stent placement.
The U.K. Medicines and Healthcare products Agency (MHRA) has committed to major reforms of how it regulates drugs for rare diseases, making it easier to run clinical trials and get approvals. The new rules will be published in full early in 2026 and come into effect later in the year, but following consultation with industry, academics and patients’ groups, the agency has released a position paper setting out its plans.
Early stage data from Terns Pharmaceuticals Inc.’s lead candidate showed a large reduction in the number of leukemia cells in those with previously treated chronic myeloid leukemia. Results from the ongoing phase I Cardinal study of TERN-701, an oral, allosteric BCR-ABL tyrosine kinase inhibitor, were “unprecedented,” according to Terns.
Uniqure NV is regrouping after a surprise switcheroo by the havoc-beset U.S. FDA regarding phase I/II studies with AMT-130 vs. external control in Huntington’s disease (HD) – news of which pushed down the Lexington, Mass.-based firm’s shares (NASDAQ:QURE) Nov. 3 by $33.40, a loss of 49%, at the closing price of $34.29.
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