Shares of Axsome Therapeutics Inc AXSM, the best-performing biotech of the year, were ripping higher Monday following a positive clinical readout.
Axsome Evaluating Candidate In 4 Indications
A Phase 3 study dubbed GEMINI that evaluated Axsome's AXS-05 — an oral investigational NMDA receptor antagonist with multimodal activity — in major depressive disorder met the primary endpoint, the company said Monday.
The study was a double-blind, placebo-controlled, multicenter U.S. trial in which 327 adult patients with confirmed moderate to severe MDD were randomized to receive either AXS-05 or placebo once daily for the first three days, and twice daily thereafter for a total of six weeks.
AXS-05, chemically dextromethorphan and bupropion, showed a highly statistically significant reduction in the Montgomery-Asberg Depression Rating Scale, or MADRS, total score compared to placebo at week six, with mean reductions from the baseline of 16.6 points for the control arm compared to 11.9 points for the placebo, with a pie value of 0.002.
The investigational asset continued to show statistically significant improvement relative to placebo right from the first week, Axsome said.
Additionally, the rates of remission from depression were statistically significant for AXS-05 compared to placebo at week two and at every point thereafter.
AXS-05 was well-tolerated in the trial and was not associated with psychomimetic effects or weight gain, the company said.
Axsome is evaluating AXS-05 for four indications: treatment-resistant depression, MDD, agitation in Alzheimer's disease and smoking cessation.
Stock Rallies By 70%
MDD is a debilitating, chronic, biologically based disorder. In the U.S., about 7.1% adults or about 17 million people experience MDD each year, Axsome said, citing National Institutes of Health estimates.
"Clinical depression is a potentially life-threatening condition. Currently marketed antidepressants fail to provide adequate treatment response in almost two thirds of treated patients, and may take up to six to eight weeks to provide clinically meaningful response," Professor Maurizio Fava, a psychiatrist, said in a statement.
"These data suggest that AXS-05, as an oral NMDA receptor antagonist with multimodal activity, may represent a novel treatment for major depressive disorder."
Following positive Phase 2 and Phase 3 trials, Axsome said it expects to file an NDA for AXS-05 in MDD in the coming year.
"With its modulation of glutamate neurotransmission, if approved, AXS-05 would represent the first mechanistically novel oral pharmacotherapy for depression in over 30 years," said Cedric O'Gorman, the senior vice president of clinical development and medical affairs at Axsome.
Axsome shares, which have soared about 1,560% year-to-date, were rallying by 70.09% to $79.58 at the time of publication.
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