As there is currently no FDA-approved treatment for Alzheimer’s agitation, any progress here could be lucrative. Axsome Therapeutics Inc. (NASDAQ: AXSM) announced on Monday a big step forward in its midstage Alzheimer’s study and was rewarded with a rally in its shares.
According to the update, Axsome’s AXS-05 met the primary endpoint in the ADVANCE-1 Phase 2/3 trial, rapidly, substantially and significantly improving agitation in patients with Alzheimer’s disease as compared to placebo.
AXS-05 met the primary endpoint by demonstrating a statistically significant mean reduction in the Cohen Mansfield Agitation Inventory (CMAI) total score compared to placebo at Week 5. A statistically significantly greater proportion of patients achieved a clinical response on the CMAI, defined as a 30% or greater improvement from baseline.
Some quick background: Alzheimer’s disease is the most common form of dementia and is characterized by cognitive decline, as well as behavioral and psychological symptoms including agitation. Agitation is observed in up to 70% of patients with Alzheimer’s disease and is associated with accelerated cognitive decline, earlier nursing home placement and increased mortality risk.
Note that AXS-05 has been granted FDA Fast Track designation for the treatment of Alzheimer’s disease agitation.
Jeffrey Cummings, M.D., Sc.D., director emeritus of the Cleveland Clinic Lou Ruvo Center for Brain Health, and Chambers Professor of Brain Science at the University of Nevada Las Vegas, commented:
The clinically significant reductions in agitation were accompanied by a favorable safety and tolerability profile. Agitation occurs in the majority of patients with Alzheimer’s disease, is very distressing to patients and their families, and is associated with greater risk of institutionalization and accelerated progression to severe dementia and death. Given the lack of approved treatments for Alzheimer’s disease agitation, and the safety concerns and modest or uncertain efficacy of currently used off-label treatments, the AXS-05 study results represent a meaningful step forward toward urgently needed treatment for this serious complication of Alzheimer’s disease.
Axsome Therapeutics stock traded up about 22% Monday to $93.50, in a 52-week range of $13.64 to $109.94. The consensus price target is $137.75.