Axsome Therapeutics initiates Phase III COAST-1 trial of AXS 02 for treatment of pain associated with knee osteoarthritis-

Axsome Therapeutics, Inc. enrolled the first patient in the COAST-1 (Clinical Knee Osteoarthritis Symptom Treatment 1) study, a Phase III trial evaluating the efficacy and safety of AXS-02 for the treatment of the pain of knee osteoarthritis (OA) associated with bone marrow lesions (BMLs).

AXS-02 is a potent osteoclast inhibitor being developed as an oral, targeted, non-opioid, potentially first-in-class therapeutic for chronic pain. The COAST-1 study is being conducted pursuant to a FDA Special Protocol Assessment (SPA). An SPA documents the FDA's agreement that the design and planned analysis of a clinical trial adequately address scientific and regulatory objectives that, if met, would support a regulatory submission for approval of a drug.

COAST-1 (Clinical Knee Osteoarthritis Symptom Treatment 1) is a randomized, double-blind, placebo-controlled trial to assess the efficacy and safety of AXS-02 administered orally to patients with knee osteoarthritis (OA) associated with bone marrow lesions (BMLs). This trial is anticipated to enroll approximately 346 patients with clinically diagnosed knee OA and at least one confirmed BML in the affected knee on MRI. Eligible patients must be at least 50 years of age, either male or postmenopausal female, and have at least moderate pain intensity. After a baseline period, patients meeting the entry criteria will be randomized in a 1:1 ratio to receive either (1) AXS-02 tablets once per week or (2) matching placebo tablets once per week, under fasting conditions for 6 weeks. Randomized patients will remain blinded for an additional 18 weeks, totaling 24 weeks for the double-blind phase. The primary endpoint is the change in pain intensity from baseline to week 24, measured using a 0-10 numerical rating scale (NRS). Axsome has reached agreement with the FDA regarding a Special Protocol Assessment (SPA) for the COAST-1 study. The SPA provides agreement that the design and planned analysis of the COAST-1 study adequately address objectives that, if met, would support a regulatory submission for approval of AXS-02 for the treatment of the pain of knee OA associated with BMLs.