There are hundreds of people facing unsatisfactory treatments for CNS disorders. Due to the limitation of treatment options, Axsome Therapeutics Inc (NASDAQ:AXSM) has taken on a new journey of developing differentiated, new therapies based on novel mechanisms of action and innovation. The company has several candidates in its CNS portfolio, including AXS-12, AXS-07, AXS-05, and AXS-14, under development for multiple indications.
The biopharma has reported a new status and plans for AXS-05, the confirmation of pivotal development. Following a successful Breakthrough Therapy meeting with the FDA, the novel, oral, investigational NMDA receptor was picked as a potential treatment for Alzheimer’s disease (AD) agitation.
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The Support of the Breakthrough Therapy Designation
AXS-05 received the Breakthrough Therapy designation earlier in the year. The FDA’s approval was based on positive results from a pivotal Phase 2/3 ADVANCE-1 study, which enrolled 366 Alzheimer’s disease patients. The patients treated with AXS-05 had significant and rapid improvement in agitation compared to the placebo. There was also a significant mean reduction from baseline in the Cohen Mansfield Agitation Inventory (CMAI).
More often than not, a Breakthrough Therapy designation, when granted, accelerates the development and evaluation timelines for a promising investigational medicine. At that point, the preliminary clinical evidence should have an indication of extensive improvement on clinical endpoints over available therapies for life-threatening illnesses.
Filing of an NDA (New Drug Application) for Approval of AXS-05
“Axsome is very pleased with the FDA feedback from our recent Breakthrough Therapy meeting, which confirms a streamlined path to NDA submission for AXS-05 in Alzheimer’s disease agitation…’’ the CEO, Herriot Tabuteau announced.
This means the company is ready to file an NDA. The good news is that only one additional Phase 3 efficacy trial will be necessary to support the filing and only a placebo control for an indication. A randomized-withdrawal design will be applied to carry out the Phase 3 efficacy trial.
Meanwhile, the biopharma says it is ready to initiate the fourth quarter of the year’s efficacy trial. This will go hand in hand with the fourth quarter of an open-label safety extension trial of AXS-05.