The phase IIb study results of Supernus Pharmaceuticals Inc.’s SPN-820 in treatment-resistant depression had pulled the company’s stock (NASDAQ:SUPN) down 15.6% on Feb. 19. The randomized, double-blind, placebo-controlled study of adults failed to show a statistically significant improvement on the primary endpoint of change from baseline on the Montgomery-Åsberg Depression rating scale. Shares closed at $33.52 apiece on Feb. 19.
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.”
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.
Major strides for the company on two fronts – psychedelic therapies and treatment-resistant depression (TRD) – came in the form of Dublin-based GH Research plc’s phase IIb data with GH-001, an inhalable mebufotenin (5-MeO-DMT) activator of serotonin 5-HT2A and 5-HT1A receptors that met not only its primary endpoint but all secondary goals in the TRD experiment. Shares of GH (NASDAQ:GHRS) closed Feb. 3 at $17.99, up $7.39, or 69.7%, on word that the fast-acting, short-duration compound led to a significant reduction from baseline of -15.2 points in Montgomery-Åsberg Depression Rating Scale total score on the eighth day of treatment compared with +0.3 points in the placebo group (a difference of -15.5 points, p<0.0001).
Complete response letter hurdles overcome and U.S. FDA clearance in hand, Axsome Therapeutics Inc. will disclose pricing in about four months, closer to the launch of Symbravo to treat adult migraine with or without aura.
EMA approval of the Alzheimer’s disease therapy Leqembi (lecanemab) has stalled once again, after the European Commission did not as usual nod through the agency’s recommendation, but told it to examine safety data that have recently become available.
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.
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.
Harness Therapeutics Ltd. has raised fresh financing to further develop its technology for upregulating the translation of mRNA into proteins, and in particular to take on a previously undruggable target in Huntington’s disease.
Lantheus Holdings Inc. is making waves in M&A with its second big deal of the month – and year – the acquisition of Evergreen Theragnostics Inc. for $250 million in cash up front and up to an additional $752.5 million in contingent payments. The transaction follows announcement of a definitive agreement to acquire Life Molecular Imaging Ltd., a subsidiary of Life Healthcare Group Holdings Ltd., on Jan. 13 for $350 million in cash at closing and a further $400 million in milestone payments.