On 31 August, the company Axsome Therapeutics, which develops therapies for the management of central nervous system disorders, confirmed the pivotal development status for its drug candidate AXS-05 for the treatment of Alzheimer’s disease (AD) agitation.
AXS-05 received the Breakthrough Therapy designation from the US Food and Drug Administration (FDA) earlier in the year. A Breakthrough therapy designation is a process to expedite the development and review of drugs for serious or life-threatening conditions. The Breakthrough therapy designation for AXS-05 in AD agitation was supported by positive results from the pivotal ADVANCE-1 Phase II/III study, which enrolled 366 US participants with agitation associated with AD. This trial met its primary endpoint by demonstrating a statistically significant improvement in AD agitation.
Following a recent successful Breakthrough Therapy meeting with the FDA, Axsome Therapeutics has announced confirmation of the pivotal development status for AXS-05. This means the company is ready to file a New Drug Application (NDA), which is a comprehensive document that must be submitted to the FDA in order to request regulatory approval for new drugs. Only one Phase III clinical trial will be needed to support the filling of this NDA. This Phase III placebo-controlled and randomised-withdrawal efficacy trial is on track for Q4 2020. In addition, the company plans to initiate a long-term safety trial of AXS-05 in AD agitation to supplement the existing AXS-05 database. This study is expected to start by the end of the year.