NEW YORK - Axsome Therapeutics, Inc. (NASDAQ: NASDAQ:AXSM), a biopharmaceutical company, announced today positive results from its Phase 3 SYMPHONY trial, indicating that its investigational drug AXS-12 (reboxetine) significantly reduced narcolepsy symptoms, including cataplexy attacks and excessive daytime sleepiness (EDS), compared to a placebo.
The SYMPHONY trial, which spanned multiple centers and was double-blind and placebo-controlled, enrolled 90 patients diagnosed with narcolepsy. Over five weeks, participants received either AXS-12 or a placebo. The primary endpoint was the change in frequency of weekly cataplexy attacks, which are sudden reductions or losses of muscle tone triggered by strong emotions, commonly associated with narcolepsy.
AXS-12 met the primary endpoint, showing a statistically significant reduction from baseline in weekly cataplexy attacks at Week 5, with an 83% reduction for those treated with AXS-12 compared to a 66% reduction for the placebo group. Additionally, 33% of patients treated with AXS-12 achieved remission of cataplexy by Week 5, in contrast to 9.5% of those on placebo.
Improvements were also noted in the severity of EDS, with AXS-12 patients experiencing a mean reduction of 1.8 points on the Clinician Global Impression of Severity scale for EDS, versus a 0.9 point reduction for placebo patients. Cognitive function, as measured by the Cognitive Function Items of the Functional Outcomes of Sleep Questionnaire, also improved significantly with AXS-12 treatment.
The drug demonstrated a favorable safety profile, with the most common adverse events being dry mouth, nausea, and constipation, which were generally mild to moderate. The trial reported low rates of discontinuation due to adverse events.
Dr. Herriot Tabuteau, CEO of Axsome Therapeutics, highlighted the rapid and substantial reduction of cataplexy events as a promising development for narcolepsy patients, who often report persistent symptoms despite existing treatments. The company plans to present detailed results of the SYMPHONY trial at upcoming scientific meetings and is looking forward to completing the ongoing open label safety extension trial of AXS-12.
AXS-12 has been granted Orphan Drug Designation by the FDA for the treatment of narcolepsy and is protected by issued patents until at least 2039. This designation may provide seven years of marketing exclusivity in the U.S. upon potential FDA approval.
The information is based on a press release statement from Axsome Therapeutics, Inc.
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