Cognition Therapeutics Inc. evolved from the work of a neuroscientist and a chemist working in the San Francisco Bay area, seeking out targets to block the effects of Alzheimer’s disease. Since the company’s 2007 inception, it has received close to $200 million in U.S. NIH grant funding. Investors often tell CEO Lisa Ricciardi, who joined the company in 2020: “’That’s because you have a relationship with the FDA.’ Well, no. It’s because it’s competitive” and the company’s research has met the muster. “You have to apply two or three times. … It’s with rigor that these results are generated and that we’re able to get more funding.”
A mysterious turn in the phase IIb story with idiopathic pulmonary fibrosis (IPF) candidate bexotegrast sent shares of Pliant Therapeutics Inc. (NASDAQ:PLRX) down 60.6%, or $4.72, to close Feb. 10 at $3.07.
Eli Lilly and Co.’s end last year to its PI3Kα inhibitor program didn’t mean the pharma player was giving up on the target – far from it, as signaled by the potential $2.5 billion deal signed recently to take over Scorpion Therapeutics Inc. and gain rights to phase I/II-stage STX-478 for breast cancer and other solid tumors. The list of developers at work in the space includes Roche AG plus smaller entities such as Celcuity Inc., Menarini Group, Onkure Therapeutics Inc., Relay Therapeutics Inc. and Totus Medicines Inc.
Touting its novel approach for treating glaucoma, Qlaris Bio Inc. reported promising top-line data from two phase II studies testing QLS-111 in patients with primary open-angle glaucoma and ocular hypertension, showing the drug met all primary and secondary endpoints, reducing intraocular pressure with a clean safety profile that could encourage patients to remain on treatment.
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
George Medicines, a new spinout from Australia’s George Institute for Global Health, could offer patients better control of their blood pressure as well as fewer side effects thanks to an ultra-low-dose triple combination.
The first successful phase II trial in Duchenne muscular dystrophy (DMD) heart disease rolled out from Cumberland Pharmaceuticals Inc., which said top-line findings from the experiment called Fight DMD showed promising results in the indication that represents the main cause of death for such patients.
New and positive data for Maia Biotechnology Inc.’s lead candidate for a particularly deadly and advanced lung cancer propelled the stock modestly higher on Feb. 4. The pivotal phase II THIO-101 study of Thio, a telomere-targeting agent sequenced with Regeneron Inc.’s immune checkpoint inhibitor, Libtayo (cemiplimab), as a third-line treatment for advanced non-small-cell lung cancer (NSCLC) showed median overall survival of 16.9 months for the 22 NSCLC patients who received at least one dose of Thio in parts A and B.
A week after the first IPO of the year was priced, obesity treatment developer Metsera Inc. and renal specialist Maze Therapeutics Inc. have begun trading on Nasdaq. Metsera (NASDAQ:MTSR) surged 47% on Jan. 31 to close at $26.50 per share while Maze (NASDAQ:MAZE) barely budged, closing three-tenths of a percentage point lower on the day.
An effort two decades in the making, Vertex Pharmaceuticals Inc.’s suzetrigine gained U.S. FDA approval as the first drug targeting the NaV1.8 pain signal. Branded Journavx, the oral small molecule is cleared for use as a non-opioid option for treating moderate to severe acute pain.
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