DiscGenics Gets FDA Nod for Phase III Trials of Disc Cell Therapy for Lumbar Disc Disease

DiscGenics, Inc., a biopharmaceutical company based in Salt Lake City, has announced that the U.S. Food and Drug Administration (FDA) has accepted its Phase III clinical protocols and Chemistry, Manufacturing, and Controls (CMC) clinical development plan. This acceptance allows the company to proceed with the clinical program for its innovative allogeneic, injectable disc progenitor cell therapy known as IDCT or rebonuputemcel. This therapy targets painful lumbar degenerative disc disease (DDD).

The treatment involves a single dose of IDCT being injected directly into the affected disc using a minimally invasive procedure. Dr. Nagy Mekhail, the Director of Evidence-Based Pain Medicine Research and Education at the Cleveland Clinic, expressed optimism about the therapy's potential to revolutionize the treatment of mechanical back pain. He noted that current treatments often involve more invasive surgical procedures, and DiscGenics' cell therapy could offer a less destructive and regenerative alternative for patients with painful degenerative disc disease.

The Phase III clinical program, which has been designed in consultation with the FDA, includes two parallel studies: the pivotal DGX-A02 (PIVOT) and the confirmatory DGX-A03 (CONFIRM). Both studies are randomized, double-blinded, sham-controlled, and multicenter trials focusing on patients with single-level symptomatic lumbar intervertebral disc degeneration. The studies will each have a primary endpoint at one year, followed by an additional one-year observation period, making the total study duration two years. Enrollment for the pivotal study is anticipated to begin in the fourth quarter of 2024.

Dr. Kevin T. Foley, Chief Medical Officer of DiscGenics and Chairman of Semmes-Murphey Neurologic & Spine Institute, emphasized that the Phase III program leverages robust data from previous Phase I/Phase II trials. These earlier studies demonstrated promising safety and efficacy results, and the new program aims to continue this rigorous evaluation to address the significant unmet clinical need in this patient population.

Flagg Flanagan, Chief Executive Officer and Chairman of the Board for DiscGenics, reflected on the journey that led to this regulatory milestone. From the initial development of the cell technology by the late Dr. Valery Kukekov to the successful first-in-human clinical trials, the team at DiscGenics has grown increasingly confident about the safety and regenerative potential of IDCT. The company is committed to advancing this technology responsibly and diligently.

IDCT, or injectable disc cell therapy (rebonuputemcel), is a biologic treatment intended to halt the progression of symptomatic lumbar disc degeneration and regenerate the disc from within. The active ingredient consists of progenitor cells derived from donated adult human intervertebral disc tissue. These cells undergo a multistep manufacturing process under current good manufacturing practices (cGMP), resulting in significant cell proliferation and phenotypic changes. The final product, IDCT, combines these cells with a Sodium Hyaluronate Solution and other excipients to form a cryopreserved, "off-the-shelf" treatment administered via percutaneous injection. IDCT has been granted regenerative medicine advanced therapy (RMAT) and Fast Track designations by the FDA.

DiscGenics is dedicated to developing cell-based regenerative therapies for musculoskeletal conditions. The company's lead product, IDCT, aims to provide a single-injection solution for lumbar disc degeneration. Additionally, DiscGenics is working on an allogeneic cell platform to address other musculoskeletal issues. To support these developments, the company has established an in-house scalable manufacturing process and cGMP facility at its headquarters in Salt Lake City, Utah.

DiscGenics' advancements in the field of regenerative medicine and cell therapy represent a significant potential shift in the treatment of chronic low back pain and degenerative disc disease, conditions that affect millions and place a substantial economic burden on the healthcare system.