Rocket Pharmaceuticals Inc. CEO Gaurav Shah said his firm is investigating how its gene therapy for Danon disease may have created an “unexpected and paradoxical” effect that led to problems for a phase II patient who ultimately died.
News from Tectonic Therapeutic Inc. in January took away some of Wall Street’s jitters about the relaxin pathway brought about by Eli Lilly and Co.’s recent moves, but another big pharma player – Astrazeneca plc – is still providing suspense in pulmonary hypertension in heart failure with preserved ejection fraction.
Investor hopes rose sharply for Merus NV’s phase III trials – data should roll out next year – with bispecific antibody petosemtamab after mid-stage results impressed Wall Street in head and neck squamous cell carcinoma. Shares of Utrecht, the Netherlands-based Merus (NASDAQ:MRUS) jumped, too, closing May 23 at $55.14, up $13.54, or 33%, on interim data as of the Feb. 27 cutoff date.
Sanofi SA made good on its plan to bear down on M&A by agreeing to buy Vigil Neuroscience Inc. for $8 per share (NASDAQ:VIGL). Included in the transaction is a non-tradeable contingent value right entitling the holder to potentially collect $2 per share more in cash, payable following the first commercial sale of the phase II-ready VG-3927, a small-molecule triggering receptor expressed on myeloid cells 2 (TREM2) antagonist for Alzheimer’s disease, if achieved within a specific period. Watertown, Mass.-based Vigil’s stock closed May 22 at $7.88, up $5.57, or 241%.
The morning’s negative stock reaction to Tourmaline Bio Inc.’s phase II news with heart drug pacibekitug had some onlookers scratching their heads, even as analysts congratulated the company on results from the ongoing study – a good-news dataset to which the market gradually caught on as the day progressed.
Frets about how the new federal administration might affect prospects for vaccines were quelled at least somewhat by the U.S. FDA green light for Novavax Inc.’s COVID-19 vaccine Nuvaxovid, indicated for adults 65 and over and people 12-64 years old with at least one underlying condition that puts them at risk of severe outcomes from infection by the virus.
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.
