The approval of Ascendis Pharma A/S’ hormone replacement therapy Yorvipath (palopegteriparatide) hypoparathyroidism – the first and only treatment for adults with the rare endocrine disease – did little to sate the market’s appetite for new drugs in the indication, where a number of players are busy in various stages of development.
DBV Technologies SA CEO Daniel Tasse said his firm will meet “very shortly” with the U.S. FDA for talks that will formalize an accelerated approval process for the Viaskin Peanut allergy patch. “Did this take longer than expected? Yes, it did,” Tasse said during a conference call update. “But this was a choice we made, and it was a necessary choice” in order to nail down precise requirements for the product.
Targeting NMDA in mental health has chalked wins but not universally, as shown by Sage Therapeutics Inc.’s failure of the placebo-controlled phase II Lightwave study testing dalzanemdor in Alzheimer's disease, which missed the primary outcome measure, another bit of bad luck from the company that was disclosed Oct. 8.
Wall Street promptly began trying to weigh the compound’s marketplace odds after Merck & Co. Inc. detailed positive data from the phase IIb/III trial known as MK-1654-004 with clesrovimab, an investigational prophylactic monoclonal antibody designed to protect infants from respiratory syncytial virus (RSV) disease during their first RSV season. The results, plus interim findings from the ongoing phase III experiment dubbed MK-1654-007 were offered during IDWeek 2024 in Los Angeles.
This summer’s IPO by Artiva Biotherapeutics Inc. highlighted early stage efforts with natural killer cells in autoimmune disease, where a handful of companies are advancing programs. The firm raised $167 million through an upsized financing, with funds aimed at the development of its lead AlloNK program for systemic lupus erythematosus and other indications in the same category.
Shares of Scholar Rock Holding Corp. (NASDAQ:SRRK) soared $26.86, or 362%, to close Oct. 7 at $34.28, after the Cambridge, Mass.-based firm disclosed positive top-line data from the phase III Sapphire study testing apitegromab in patients with spinal muscular atrophy (SMA). Apitegromab, which Wainwright analyst Andres Maldonado said will “transform SMA” therapy, met the primary endpoint with statistically significant and clinically meaningful improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded.
Judo Bio Inc. emerged from stealth mode and rolled out data showing the value of using megalin receptors for intracellular delivery of ligand-small interfering RNA (siRNA) therapeutics to the kidney as a way of reducing expression of the targeted genes.
Engene Holdings Inc.’s recent disclosure of pivotal phase II data with nonviral gene therapy detalimogene voraplasmid (also known as detalimogene, and previously as EG-70) – along with a planned protocol refinement – added intrigue to the non-muscle invasive bladder cancer space, which continues to percolate.
Shattuck Labs Inc. opted, as one analyst put the matter, to do “the right thing early” by ending the clinical program with phase I-stage SL-172154 and shift resources to SL-325, a death receptor 3 antagonist, initially for inflammatory bowel disease, where TL1A/DR3-blocking antibodies have shown compelling monotherapy efficacy.
Cereno Scientific AB’s positive top-line results from the phase IIa trial of histone deacetylase inhibitor CS1 in pulmonary arterial hypertension provide a “clear path forward” in the debilitating, fatal disease, the company said.
