It was a battle of the companies with drugs for transthyretin-mediated amyloidosis on the first day of the J.P. Morgan 2026 Healthcare Conference with Alnylam Inc., Bridgebio Pharma Inc. and Pfizer Inc. all presenting at the annual kickoff conference. One company disclosed 2025 sales as well as 2026 revenue guidance, the second only looked back, while the third was too big to do either for a specific drug.
On the last day of the year, shares of Axsome Therapeutics Inc. rose 22% on news that the U.S. FDA accepted and granted priority review of the company’s supplemental NDA for AXS-05 to treat Alzheimer’s disease agitation, a $1 billion-plus opportunity, scheduling the PDUFA date for April 30.
Despite missing the primary endpoint in a phase III study of solriamfetol in major depressive disorder, Axsome Therapeutics Inc. still sees a path to another phase III study. It’s a path some analysts and investors are concerned about, though its precision-medicine approach got plenty of nods of approval.
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.
An asset that some investors may have overlooked came through for Axsome Therapeutics Inc., which rolled out phase III data from the trial called Symphony with AXS-12 (reboxetine), which hit the primary endpoint by significantly reducing cataplexy attacks in narcolepsy patients vs. placebo.
Axsome Therapeutics Inc.’s AXS-05 (dextromethorphan + bupropion) has notched another success by hitting its phase III primary endpoint in treating a notoriously difficult Alzheimer’s disease (AD) indication when compared to placebo. Newly released data from the Accord study showed AXS-05, an oral, NMDA receptor antagonist with multimodal activity, statistically significantly delayed time to relapse of AD agitation vs. placebo (p=0.014), which was the primary endpoint.
Gate Neurosciences Inc. was first founded in 2019, but officially launched last week with two clinical-stage assets and a bold goal: to develop better drugs for CNS disorders and identify better-suited patients for those drugs. The company’s first molecular target is the NMDA receptor. Gate has acquired the rights to two NMDA receptor modulators, zelquistinel and apimostinel.
A year from the U.S. FDA’s August 2021 citation of deficiencies in the labeling for Axsome Therapeutics Inc.’s major depressive disorder therapy, Auvelity (dextromethorphan + bupropion), the agency has approved the drug for use by adults. The New York-based company’s stock (NASDAQ:AXSM) took flight on the news, with shares rising 40% to close at $59.55 Aug. 19, surpassing their previous 52-week high of $48.82 and starkly contrasting with a same-period low of $19.38.
Axsome Therapeutics Inc. and the U.S. FDA appear to be working out their differences. The company said it received the proposed labeling from the agency for AXS-05 (dextromethorphan + bupropion), an oral NMDA receptor antagonist with multimodal activity, for treating major depressive disorder.
A quartet of companies on May 2 announced three complete response letters (CRLs) that left them scrambling to get back on the path to approval. Hutchmed Ltd.'s surufatinib met with word from the U.S. FDA that two positive phase III studies in China and a bridging study in the U.S. would not support approval of the drug for pancreatic and extra-pancreatic neuroendocrine tumors. A multi-regional clinical trial in the U.S. is needed, the regulator said, brushing aside China-only studies when seeking a U.S. approval.
