AXS-12 Meets Primary Goal in Phase 3 Narcolepsy Study

Axsome Therapeutics, a biopharmaceutical firm, has reported positive results from its Phase 3 SYMPHONY trial for the drug AXS-12, which is being studied for the treatment of narcolepsy. Narcolepsy is a chronic sleep disorder affecting the sleep-wake cycle, characterized by excessive daytime sleepiness and sudden loss of muscle tone, known as cataplexy. The study involved 90 patients who received either AXS-12 or a placebo for five weeks.

The trial showed that AXS-12 significantly reduced the frequency of cataplexy attacks by 83% compared to a 66% reduction in the placebo group at week five. The drug also demonstrated a rapid onset of action, with a 56% reduction in weekly cataplexy attacks by week one. Furthermore, AXS-12 induced remission of cataplexy in 33% of the patients by week five, compared to only 9.5% in the placebo group. The drug also increased the percentage of cataplexy-free days to 84.5% by week five, compared to 22.6% in the placebo group.

In addition to its effects on cataplexy, AXS-12 also significantly reduced the severity of excessive daytime sleepiness (EDS), as assessed by the Clinician Global Impression of Severity (CGI-S) scale. The drug showed improvements in cognitive function, including concentration and memory, as measured by the Functional Outcomes of Sleep Questionnaire (FOSQ-10). AXS-12 also led to a significant reduction in the overall severity of narcolepsy and improved the patients' overall function and quality of life.

The drug was well-tolerated, with the most common side effects being dry mouth, nausea, and constipation, which were generally mild to moderate. The discontinuation rate due to adverse events was low.

AXS-12 has been granted Orphan Drug Designation by the FDA, indicating its potential for treating a rare disease. The designation could provide Axsome with seven years of marketing exclusivity in the U.S. upon FDA approval.

Axsome plans to present detailed results of the SYMPHONY trial at upcoming scientific meetings and is looking forward to completing an ongoing open-label safety extension trial for AXS-12. The company is committed to bringing this new treatment option to individuals living with narcolepsy, addressing the significant unmet needs of patients suffering from the condition.