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.
The U.S. FDA’s approval of yet another indication for Dupixent (dupilumab), partnered between Regeneron Pharmaceuticals Inc. and Sanofi SA, could mean another $6.4 billion-plus in sales by the end of the decade. Regulators cleared the drug as an add-on maintenance treatment for chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype who are prone to flare-ups. Dupixent, the first-ever biologic for COPD, entered the market in March 2017.
The FDA’s Oncologic Drugs Advisory Committee (ODAC) met for what chairperson Christopher Lieu called, at the end, “an incredibly long day” to decide whether approval of immune checkpoint inhibitors should be restricted in accordance with expression levels of PD-L1.
With two drugs cleared by the U.S. FDA for Niemann-Pick disease type C (NPC) in less than seven days, Wall Street was pondering the differences between the compounds, given what’s known so far about each. Most recently, the FDA approved Intrabio Inc.’s Aqneursa (levacetylleucine) on its PDUFA date for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing at least 15 kg, making Aqneursa the only approved stand-alone therapy indicated for NPC. On Sept. 20, Zevra Therapeutics Inc. won FDA clearance for Miplyffa (arimoclomol) as the first treatment for NPC.
The U.S. FDA’s Oncologic Drugs Advisory Committee (ODAC) on Sept. 26 will take up a controversy that’s hardly new: whether approval of immune checkpoint inhibitor drugs should be restricted in accordance with PD-L1 expression.
