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.
Scientists in the U.K. are developing glucose-powered bioelectronics to advance the use and capabilities of implantable medical devices. A research team, led by the University of Bath, received £2.1 million (US$3.3 million) in funding from the government to develop miniature, lightweight and long-lasting glucose fuel cells to help address the invasiveness and limitations of current battery-powered implants.
Vera Therapeutics Inc.’s atacicept gained still more airtime at the Cantor Fitgerald health care conference held Sept. 4, and some on Wall Street believe the appetite for the BAFF and APRIL dual inhibitor could bode well for others in the same mechanistic space.
Neurodegeneration specialist Vesper Bio ApS has announced positive results from the phase Ib/II trial of its oral sortilin inhibitor VES-001 in frontotemporal dementia. The small study involved six participants who had not progressed to symptomatic disease but were carriers of mutations in the GRN gene that codes for progranulin, a growth factor that is essential for neuronal health.
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