Axsome Therapeutics Narcolepsy Drug AXS-12 Achieves Phase III Success, Paving Way for US FDA Filing

Phase III Success:
Axsome Therapeutics' narcolepsy drug AXS-12 has successfully met its primary endpoint in the Phase III ENCORE trial, demonstrating a statistically significant reduction in cataplexy attacks compared to placebo[1][2].

Efficacy and Safety:
The trial showed that AXS-12 reduced cataplexy attacks by 72% at one month and 82% at six months, with improvements also seen in excessive daytime sleepiness and cognition. The drug was well tolerated with a favorable long-term safety and tolerability profile[1][2].

NDA Filing:
Following the positive results, Axsome Therapeutics plans to submit a new drug application (NDA) to the US Food and Drug Administration (FDA) and intends to request a pre-NDA meeting[1][2].

Orphan Drug Designation:
AXS-12 has been granted Orphan Drug Designation for the treatment of narcolepsy, which may provide seven years of marketing exclusivity in the US upon FDA approval[2].

Patent Protection:
AXS-12 is covered by issued patents providing protection until at least 2039[2].