DiscGenics Announces that the International Journal of Spine Surgery has Published Results of an FDA-Approved Study of Allogeneic Disc Progenitor Cell Therapy for the Treatment of Adults with Lumbar Disc Degeneration

SALT LAKE CITY, July 9, 2024 /PRNewswire/ — DiscGenics, Inc.a privately held, late-stage clinical-stage biopharmaceutical company developing allogeneic, cell-based and regenerative therapies for musculoskeletal degeneration, today announced the publication in the International Journal of Spine Surgery of results from a first-in-human, combined Phase I/Phase II clinical study of allogeneic disc progenitor cell therapy (IDCT or rebonuputemcel) for painful lumbar degenerative disc disease (DDD).

The study met primary efficacy and safety endpoints, showing that a single intradiscal injection of high-dose IDCT (9,000,000 cells/mL) safely increases disc volume and produces statistically significant and clinically meaningful improvements in back pain, disability, and quality of life up to 2 years post-injection in patients with lumbar disc degeneration.

“The results of this study demonstrate the potential of IDCT to safely and effectively reduce pain associated with disc degeneration while producing a regenerative effect within the degenerating disc. MRI image analysis of the disc volume indicated the potential to halt and possibly reverse disease progression,” said Matthew F. Gornet, MD, senior author, board-certified spine surgeon at St. Louis Orthopaedic Center and principal participant in the IDCT study. “I have been a spine surgeon for over 30 years and have participated in over 35 FDA clinical trials, and the patient outcomes in this study are very promising.”

In the FDA-approved, prospective, randomized, double-blind, vehicle- and placebo-controlled, multicenter study, 60 patients with symptomatic single-level lumbar DDD were randomized to receive single intradiscal injections of low-dose cells (n = 20), high-dose cells (n = 20), vehicle alone (n = 10), or placebo (n = 10). The primary endpoint was a mean visual analog scale (VAS) pain improvement of >30% at 52 weeks. Disability and quality of life were assessed via the Oswestry Disability Index (ODI) and EQ-5D, respectively. Disc volume was assessed radiologically. Adverse events (AEs), whether treatment-related or not, were reported. Patients were assessed at baseline and at 4, 12, 26, 52, 78, and 104 weeks after treatment.

At week 52, the main study period, the high-dose group had a mean decrease from baseline in VAS percentage (−62.8%, P = 0.0005), reaching the endpoint of >30% improvement in back pain; the mean change was also significantly greater than the minimal clinically important difference (MID) of a 20-point decrease (−42.8, P = 0.001). This clinical improvement was maintained at week 104. In addition, the high-dose group had clinically meaningful and statistically significant improvements in ODI and EQ-5D at 12 weeks. Clinical improvement was maintained at 26 weeks, 52 weeks, 78 weeks, and 104 weeks after a single intradiscal injection. Only the high-dose group had a significant change in disc volume, with mean increases of 249.0 mm3 (P = 0.028) at 52 weeks and 402.1 mm3 (P = 0.028) at 104 weeks. Overall, a minority of patients (18.3%) reported serious adverse events, with the highest percentage reported in the placebo group. During the trial, 6.7% of patients experienced serious adverse events, all occurring in the vehicle group (n = 1) or placebo group (n = 3), none of which were treatment-related.

“Since DiscGenics’ inception, we have seen consistent evidence of the safety and regenerative potential of the unique IDCT disc cell population to treat disc degeneration,” said Kevin T. Foley, M.D., DiscGenics’ chief medical officer and president of the Semmes-Murphey Neurologic & Spine Institute. “Our early basic science studies, our demonstrated ability to safely use human cells in 14 separate preclinical animal studies conducted in the United States and Japan, and recently, data from our first-in-human safety studies and patient-reported outcomes published in this IJSS manuscript, all support the idea that this cell has the potential to safely regenerate the intervertebral disc from the inside out.”

Chronic low back pain is a chronic, progressive condition in which the intervertebral disc deteriorates, causing pain and disability. It accounts for nearly 40% of chronic low back pain cases in the United States, a serious condition that affects 12% to 30% of American adults at any given time and is estimated to cost the U.S. health care system more than $100 billion annually, placing a significant burden on the economy and patients.

“The significant and durable results from this study demonstrate the incredible potential of IDCT to change the paradigm of care for patients with DDD, a condition with limited treatment options,” said Flagg Flanagan, CEO and Chairman of the Board of DiscGenics. “We are excited about the momentum provided by the publication of these study results as we plan to initiate the Phase III clinical study of this novel therapy in the United States shortly.”

About IDCT

IDCT (Injectable Disc Cell Therapy, or rebonuputemcel) is a single-injection, stand-alone biologic treatment designed to halt the progression of symptomatic lumbar disc degeneration and regenerate the disc from the inside out. The active ingredient (drug substance) in IDCT is a population of live, engineered progenitor cells derived from donated adult human intervertebral disc tissue. These cells are enriched and expanded into discogenic cells through a multi-step manufacturing process in a highly controlled environment under current good manufacturing practice (cGMP) that results in significant proliferation and phenotypic changes of the cells. At the end of the manufacturing process, the discogenic cells undergo extensive testing prior to use, including assessments of identity, purity, potency, and safety. The discogenic cells are then mixed with a viscous solution of sodium hyaluronate and excipients to generate IDCT, the final drug product. IDCT is cryopreserved and stored as individual “ready-to-use” doses for administration by percutaneous injection into the intervertebral disc in an outpatient setting. IDCT has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration.

Disclaimer: IDCT is an investigational product under development by DiscGenics and has not been approved by the FDA or any other regulatory agency for human use.

About DiscGenics

DiscGenics is a privately held, late-stage clinical-stage biopharmaceutical company developing allogeneic cell-based regenerative therapies for musculoskeletal degeneration. Its lead product candidate, IDCT (injectable disc cell therapy, or rebonuputemcel), is a single-injection, stand-alone biologic treatment designed to halt the 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 unique therapies and maintain control over compliance, cost and production timelines, DiscGenics has built and validated a scalable allogeneic cell manufacturing process and cGMP facility in-house at its headquarters in Salt Lake City, Utah. For more information, visit discgenics.com.

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