Axsome Therapeutics Initiates Phase 3 Study of AXS-02 for Knee Osteoarthritis Associated with Bone Marrow Lesions
First Patient Enrolled in the COAST-1 Study
Non-Opioid, Oral, Targeted, Potentially First-in-Class Mechanism for Pain
Novel Biomarker-Based Approach for the Treatment of Knee Osteoarthritis Pain
COAST-1 Study Being Conducted under FDA Special Protocol Assessment
“BMLs, which are visible on MRI, are linked to knee pain and cartilage damage, and are present in a significant percentage of patients with knee OA,” said
The COAST-1 study is being conducted pursuant to a
“Knee OA is a significant source of chronic disability for millions of patients. A hallmark of the condition is joint pain, often associated with bone changes, including osteophytes and BMLs. These processes can lead to loss of normal joint function and may progress to eventual joint failure resulting from progressive cartilage loss,” said
“We believe that AXS-02 holds promise in the treatment of knee OA due to its unique, non-opioid mechanisms of action for pain,” said
“We are pleased to initiate a Phase 3 trial in this important indication which touches the lives of so many,” said
About the COAST-1 Study
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
About AXS-02
AXS-02 (disodium zoledronate tetrahydrate) is a potent osteoclast inhibitor being developed as an oral, targeted, non-opioid, potentially first-in-class therapeutic for chronic pain. AXS-02 has a high affinity for bone mineral, and reduces osteoclast activity by inhibiting the farnesyl pyrophosphate synthase (FPPS) enzyme. AXS-02 is being developed for the treatment of complex regional pain syndrome (CRPS), the pain of knee osteoarthritis (OA) associated with bone marrow lesions (BMLs), and chronic low back pain (CLBP). Phase 3 trials are underway with AXS-02 in CRPS and knee OA associated with BMLs, and are planned in CLBP. AXS-02 is an investigational medication not approved by the
About Knee Osteoarthritis (OA) associated with Bone Marrow Lesions (BMLs)
Knee OA is a disorder characterized by periarticular bone changes, progressive loss of articular cartilage, joint space narrowing, and eventual total joint failure. It is clinically manifested by knee pain, significant physical disability, and reduced quality of life. BMLs are regions of increased signal intensity on magnetic resonance imaging (MRI) of the knee in patients with knee OA. BMLs are strongly associated with the presence and severity of knee pain, and predict disease severity and structural progression in patients with knee OA, based on published studies. Results of epidemiological studies suggest that there are approximately 7 million symptomatic patients in
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Certain matters discussed in this press release are “forward-looking statements”. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the success, timing and cost of our ongoing clinical trials and anticipated clinical trials for our current product candidates, including statements regarding the timing of initiation and completion of the trials; the timing of and our ability to obtain and maintain
Axsome Contact:Mark Jacobson Vice President, OperationsAxsome Therapeutics, Inc. 25 Broadway , 9th FloorNew York, NY 10004 Tel: 212-332-3243 Email: mjacobson@axsome.com www.axsome.com Trout Group Contact: Marcy Beth Nanus Senior Vice PresidentThe Trout Group LLC Tel: 646-378-2927 Email: mnanus@troutgroup.com