Axsome Therapeutics Announces AXS-12 Achieves Primary Endpoint in the SYMPHONY Phase 3 Trial in Narcolepsy - Seite 4

AXS-12 was well tolerated in the trial. The most commonly reported adverse events in the AXS-12 arm were dry mouth (n=6), nausea (n=6), and constipation (n=4), which were overall mild to moderate. The rates of discontinuation due to adverse events was low (n=1 in each of AXS-12 and placebo arms). There were no serious adverse events in the trial.

AXS-12 was granted Orphan Drug Designation for the treatment of narcolepsy in October 2018. Orphan Drug Designation is granted to promising drugs intended for the safe and effective treatment of rare diseases, defined as those affecting fewer than 200,000 people in the U.S. This designation may entitle Axsome to a period of seven years of marketing exclusivity in the U.S. upon FDA approval and a waiver of the Company’s obligation to pay the FDA application user fees for the product as required by the Prescription Drug User Fee Act. AXS-12 is covered by issued patents providing protection to at least 2039.

Axsome plans to present the detailed results of the SYMPHONY trial at upcoming scientific meetings.

Summary of Topline Results of the SYMPHONY Trial

Effect on Cataplexy

• AXS-12 demonstrated a substantial and statistically significant reduction from baseline in weekly cataplexy attacks compared to placebo at Week 5, with reductions of 83% for AXS-12 and 66% for placebo (p=0.018).
• AXS-12 demonstrated a statistically significant reduction from baseline in weekly cataplexy attacks compared to placebo at Week 1, with reductions of 56% for AXS-12 and 31% for placebo (p=0.007).
• AXS-12 achieved remission of cataplexy (100% reduction from baseline) at Week 5 in 33% of patients compared to 9.5% of placebo patients (p=0.008).
• Remission at Week 2 was achieved by 24% of AXS-12 treated patients compared to 4.5% of placebo patients (p=0.008).
• AXS-12 increased the percentage of cataplexy free days (days with zero cataplexy attacks) to 84% at Week 5 compared to 22% for placebo (p=0.014).
  

Effect on Excessive Daytime Sleepiness (EDS)

• AXS-12 significantly reduced EDS severity compared to placebo at Week 5 with mean reductions on the CGI-S for EDS of 1.8 points for AXS-12 compared to 0.9 points for placebo (p=0.027).
  
• Improvement on the CGI-S for EDS was seen as early as Week 1 compared to placebo (p=0.006).
  
• Concurrent EDS and cataplexy response was achieved at Week 5 by 57% of patients treated with AXS-12 compared to 33% of placebo patients (p=0.029).