Axsome’s Alzheimer’s Drug Promises Blockbuster Potential Despite Mixed Trial Results

Axsome Therapeutics (NASDAQ: AXSM) is pressing ahead with plans to seek U.S. FDA approval for AXS-05 (Auvelity) in the treatment of Alzheimer’s disease agitation, despite mixed results in late-stage trials announced in December. Of the two recent Phase III studies, one met its primary endpoint, while the other failed to outperform a placebo. However, with two earlier trials having met their primary endpoints, the biotech firm believes it has sufficient data to support an FDA filing in the second half of 2025.

Already approved for major depressive disorder (MDD), AXS-05 is also in Phase II trials for smoking cessation. The drug has received the FDA’s Breakthrough Therapy designation, which could accelerate the approval timeline, potentially allowing for an earlier market launch.

Market expectations remain high. Visible Alpha consensus estimates show a 76.25% probability of success (POS) for AXS-05 in Alzheimer ’s-related agitation in the U.S. If approved, risk-adjusted sales are projected to rise from $94 million in 2026 to blockbuster status at $1 billion by 2031, with peak global sales reaching $1.9 billion by 2036. By then, AXS-05—across its approved and potential indications—is expected to account for 77% of Axsome’s total revenue.

Separately, Axsome reached a settlement agreement this week with Teva Pharmaceuticals (TLV: TEVA) over a patent dispute related to Auvelity. The agreement protects Axsome’s MDD treatment from generic competition in the U.S. market. Auvelity is expected to generate $477 million in sales in fiscal 2024, with peak global revenue projected to hit $1.7 billion by 2034.