Small interfering RNA (siRNA) gained another vote of confidence, albeit back end-loaded, from big pharma as privately held Adarx Pharmaceuticals Inc. signed a deal with Abbvie Inc. related to disease areas that include neuroscience, immunology, and oncology.
Xenon Pharmaceuticals Inc. is delaying the phase III data readout from the X-tole2 trial with azetukalner in focal onset seizures (FOS) from the second half of 2025 to early 2026 but remains busy with NDA plans.
Eli Lilly and Co. gained more oomph in its weight-loss battle with Novo Nordisk A/S, as the firm highlighted another batch of results from Surmount-5, a phase IIIb open-label trial evaluating the safety and efficacy of Zepbound (tirzepatide), a glucose-dependent insulinotropic polypeptide receptor and glucagon-like peptide-1 (GLP-1) receptor agonist compared to the Novo prospect Wegovy (semaglutide), a mono GLP-1 receptor agonist, in adults with obesity, or overweight with at least one weight-related medical problem and without diabetes.
Verastem Oncology Inc. CEO Dan Paterson said he is “not expecting a huge bolus [of ovarian cancer patients] at the beginning” of Avmapki/Fakzynja’s launch, but momentum will build over time. “Based on our market research, this [drug] is the most likely thing for them to go on next,” he added, noting that patients tend to be on the drug for an average of 18 months.
Revolution Medicines Inc. followed the results offered last month with zoldonrasib (RMC-9805), a RAS G12D (ON) inhibitor, with plenty more about pipeline progress – “an embarrassment of riches,” CEO Mark Goldsmith said – during the firm’s first-quarter update. Shares of the firm (NASDAQ:RVMD) closed May 8 at $41.91, up $4.25, or 11%.
The appointment May 6 of Vinay Prasad as the head of the U.S. FDA’s Center for Biologics Evaluation and Research (CBER) “bodes poorly” for Sarepta Therapeutics Inc.’s development-stage pipeline, said Wainwright analyst Mitchell Kapoor – and Wall Street reflected as much, as the stock (NASDAQ:SRPT) ended that day down 26.6% vs. an XBI drop of 6.6% – this ahead of the after-hours earnings disclosure that pushed the Cambridge, Mass.-based firm down even farther by more than another 20%, with the XBI unchanged.
Aldeyra Therapeutics Inc. is making another run at U.S. FDA clearance for reproxalap in dry eye disease (DED) after nailing the primary endpoint in a phase III randomized, double-masked, vehicle-controlled dry eye chamber trial of 0.25% ophthalmic solution.
PTC Therapeutics Inc. CEO Matthew Klein said the firm “achieved all we set out to [achieve] in phase II” with PTC-518 in Huntington’s disease (HD), but it wasn’t enough to excite Wall Street, as some questioned whether the level of associations between trial findings and efficacy would appease the U.S. FDA. Shares (NASDAQ:PTCT) ended May 5 at $40.65, down $9.30, or 18.6%. Warren, N.J.-based PTC rolled out the most recent data from the Pivot-HD effort in stage 2 and stage 3 HD with PTC-518, an oral, centrally as well as peripherally distributed huntingtin (HTT) pre-mRNA splicing modifier.
Wall Street immediately set to weighing the long-term import of Cytokinetics Inc.’s regulatory delay with aficamten, but the short-term impact was evident, as shares (NASDAQ:CYTK) dipped to close May 2 at $37.35, a loss of $5.57 or 13%.
Clinical results offered at the recent meeting of the American Urological Association in Las Vegas signal that better treatments may lie ahead for non-muscle invasive bladder cancer.
