The U.S. Food and Drug Administration (FDA) has sanctioned a Phase III clinical trial program for DiscGenics Inc.’s allogeneic, injectable disc progenitor cell therapy (IDCT or rebonuputemcel) for painful lumbar degenerative disc disease (DDD).

Chronic low back pain is a serious medical condition that represents a leading cause of disability worldwide and is the most common non-cancer reason for opioid prescription in the United States. It affects 12% to 30% of U.S. adults at any given time and is estimated to cost the U.S. healthcare system more than $100 billion each year, according to industry statistics. In nearly 40% of patients, low back pain is caused by DDD, a chronic and progressive condition where the intervertebral disc breaks down and causes pain.
 
“There are very few credible treatments on the market for mechanical back pain, and we believe early clinical evidence suggests DiscGenics’ cell therapy has the potential to shift the paradigm of care from ablative/destructive procedures or fusion surgery to a regenerative and minimally invasive treatment for patients suffering from painful degenerative disc disease,” said Nagy Mekhail, M.D., Ph.D., director of evidence-based pain medicine research and education, Department of Pain Management, at the Cleveland Clinic. “We look forward to participating in the continued clinical evaluation of this long waited for, promising therapy.”
 
The Phase III clinical program agreed upon with the FDA will consist of two identical studies running in parallel: the pivotal study (DGX-A02 or PIVOT) and the confirmatory study (DGX-A03 or CONFIRM). Both studies are randomized, double-blinded, sham-controlled, multicenter trials involving patients suffering from single-level symptomatic lumbar intervertebral disc degeneration.
 
“The Phase III clinical program builds on a strong foundation of clinical data from the combined Phase I/Phase II study of IDCT showing promising safety and efficacy results, recently published in the International Journal of Spine Surgery and is informed by several discussions with the FDA to ensure continued safe evaluation of IDCT in this patient population where the unmet clinical need is high,” stated DiscGenics Chief Medical Officer Kevin T. Foley, M.D., chairman of Semmes-Murphey Neurologic & Spine Institute.
 
Each study will have a one-year primary endpoint with another one-year follow-up period (two-year total duration). Patient enrollment in the pivotal study is expected to begin in the fourth quarter this year.
 
“To get to this point has been a long, but very exciting journey. From the early founding of this cell technology by the late Dr. Valery Kukekov to the successful first-in-human clinical evaluation of IDCT, we have become more confident not only around the safety of the cell, but also its regenerative potential,” DiscGenics CEO/Board Chairman Flagg Flanagan said. “Our team has been persistent in achieving this regulatory milestone, which allows us to continue fulfilling our responsibility as stewards of this technology.”
 
Through this study, IDCT is being evaluated under an investigational new drug (IND) allowance by the FDA. If successful, this Phase III clinical program will support a biologics license application.
 
IDCT is a standalone, single-injection biologic treatment designed to stop the progression of symptomatic lumbar disc degeneration and regenerate the disc from the inside out. The active ingredient in IDCT is a live, manufactured progenitor cell population derived from donated adult human intervertebral disc tissue. These cells are enriched and expanded into discogenic cells through a multistep manufacturing process in a highly controlled environment under current good manufacturing practices (cGMP) that results in significant proliferation and phenotypic changes to the cells. After they are manufactured, the discogenic cells are subjected to extensive testing prior to use, including identity, purity, potency, and safety evaluations. The discogenic cells are then mixed with a viscous sodium hyaluronate solution and excipients to generate IDCT, which is cryopreserved and maintained as individual “off-the-shelf” doses for administration through percutaneous injection into the intervertebral disc in an outpatient setting. IDCT has been granted regenerative medicine advanced therapy (RMAT) and Fast Track designations by the FDA.
 
DiscGenics is a privately held, late-stage clinical biopharmaceutical company developing allogeneic, cell-based regenerative therapies for musculoskeletal degeneration. It’s lead product candidate, IDCT,  is a standalone, single-injection biologic treatment designed to halt progression of lumbar disc degeneration and regenerate the disc from the inside out. DiscGenics is also developing a follow-on allogeneic cell platform to enable new musculoskeletal indications. To further develop these therapies and to maintain control over compliance, cost, and production timelines, DiscGenics has built and validated an in-house scalable, allogeneic cell manufacturing process and cGMP facility at its headquarters in Salt Lake City, Utah.