NEW YORK—Axsome Therapeutics, Inc., a biopharmaceutical company developing novel therapies for the treatment of pain and other central nervous system (CNS) disorders, has enrolled the first patient in the CREATE-1 (CRPS Treatment Evaluation 1) study—a Phase 3 trial evaluating the efficacy and safety of AXS-02 (disodium zoledronate) for the treatment of pain associated with complex regional pain syndrome. AXS-02 is a potent osteoclast inhibitor being developed as an oral, targeted, non-opioid, potentially first-in-class therapeutic for chronic pain.
“CRPS patients live with a level of pain that is unimaginable for most of us. As there are no approved treatments for this serious disease, it represents a high unmet medical need,” said Leonardo Kapural, M.D., Ph.D., professor of anesthesiology at Wake Forest University, and clinical director of the Chronic Pain Center at Wake Forest University Health Sciences Center. “This is an important clinical trial as it may increase the treatment options for those living with CRPS.”
“The Reflex Sympathetic Dystrophy Syndrome Association (RSDSA) supports research to develop better treatments and a cure for this devastating condition,” said Jim Broatch, executive vice president and director of the RSDSA. “Clinical trials such as the CREATE-1 study is an example of the type of research that could yield new options to improve the lives of individuals with CRPS.”
“We are pleased to enroll the first patient in the CREATE-1 trial,” said Randall Kaye, M.D., chief medical officer of Axsome Therapeutics. “This multi-national study will further our understanding of the potential role of AXS-02 in the treatment of pain associated with CRPS. The launch of this Phase 3 trial comes on the heels of our recent FDA Fast Track designation for AXS-02 in CRPS.”
In March of this year, the United States Food and Drug Administration (FDA) granted Fast Track designation for AXS-02 for the treatment of pain associated with CRPS. This designation provides greater access to and more frequent communication with the FDA throughout the entire drug development and review process, with the goal of possibly expediting approval. Fast Track designation also gives Axsome the opportunity to potentially submit sections of the AXS-02 new drug application (NDA) for CRPS on a rolling basis, and allows AXS-02 to be considered for priority review at the time of submission. AXS-02 has also been granted Orphan Drug Designation by the FDA, and Orphan Medicinal Product Designation by the European Medicine Agency (EMA) for the treatment of CRPS.
“As an organization, we aim to research and bring to market innovative therapies for sufferers of chronic pain and CNS diseases,” said Herriot Tabuteau, M.D., CEO of Axsome Therapeutics. “We are committed to working to find solutions for the CRPS patient community.”
The Phase 3 CREATE-1 multi-national, multi-center, randomized, double-blind, placebo-controlled trial is designed to evaluate the efficacy and safety of AXS-02 in the treatment of pain associated with CRPS. The study is expected to enroll 190 patients at sites in the United States, Canada, Europe, and Australia. Eligible patients will be randomized in a 1:1 ratio to be treated with AXS-02 or placebo. The primary efficacy measure is the change in patient reported pain intensity, measured using the 0-10 Numerical Rating Scale (NRS). Secondary outcome measures include assessments of the change in the Brief Pain Inventory (BPI) Pain Score, Patients’ and Clinicians’ Global Impression of Change (PGI-C and CGI-C, respectively), quality of life measures, and bone turnover markers.
AXS-02 (disodium zoledronate) is a potent osteoclast inhibitor being developed as an oral, targeted, non-opioid, potentially first-in-class therapeutic for chronic pain, including pain associated with CRPS. AXS-02 has a high affinity for bone mineral, and reduces osteoclast activity by inhibiting the farnesyl pyrophosphate synthase (FPPS) enzyme.
AXS-02 is an investigational medication not approved by the FDA. The safety and efficacy of AXS-02 have not yet been established.
CRPS, previously known as Reflex Sympathetic Dystrophy (RSD), is a debilitating condition characterized by severe and continuous pain in a limb, accompanied by autonomic, sensory, motor and trophic changes. For many patients the pain and associated loss of function result in significant and sometimes permanent disability. There is currently no drug approved for the treatment of CRPS in the United States or the European Union.
Axsome Therapeutics, Inc. is a biopharmaceutical company developing novel therapies for the management of pain and other CNS disorders. By focusing on this therapeutic area, Axsome is addressing growing markets where current treatment options are limited or inadequate. Axsome has a portfolio of clinical development stage as well as research stage products. Axsome Therapeutics aims to become a fully integrated biopharmaceutical company that develops and commercializes differentiated products, which increase the armamentarium of caregivers and improve the lives of patients. While the focus of the company is on developing its internally derived product candidates, Axsome also evaluates potential in-licensing opportunities to supplement its current portfolio.