DiscGenics Announces Publication of FDA-Approved Study on Allogeneic Disc Therapy for Lumbar Degeneration

In a significant development for the treatment of lumbar degenerative disc disease (DDD), DiscGenics, Inc., a privately held, late-stage clinical biopharmaceutical company, announced the publication of results from their first-in-human clinical study in the International Journal of Spine Surgery. The study evaluated an allogeneic disc progenitor cell therapy, known as IDCT or rebonuputemcel, designed to alleviate pain and improve function in patients suffering from this debilitating condition.

The clinical trial was a combined Phase I/Phase II study, conducted to assess the safety and efficacy of IDCT in patients with painful lumbar degenerative disc disease. The therapy involves a single injection of IDCT directly into the affected disc. The study met its primary endpoints, demonstrating that a high-dose injection of IDCT (9,000,000 cells/mL) not only increased disc volume but also led to statistically significant and clinically meaningful improvements in back pain, disability, and quality of life for up to two years post-injection.

Dr. Matthew F. Gornet, the lead author of the study and a Board Certified Spine Surgeon at The Orthopedic Center of St. Louis, noted the promising outcomes. He highlighted that the MRI analysis revealed a potential to halt and even reverse the progression of DDD. Dr. Gornet, with over 30 years of experience and involvement in more than 35 FDA clinical trials, emphasized the encouraging patient outcomes from this study.

The multicenter study was prospective, randomized, double-blinded, and vehicle- and placebo-controlled. It included 60 patients with symptomatic, single-level lumbar DDD who were divided into four groups. These groups received either a low-dose cell injection, a high-dose cell injection, a vehicle alone, or a placebo. The primary endpoint was an improvement in back pain as measured by the visual analog scale (VAS), requiring a greater than 30% improvement at 52 weeks. Secondary assessments included the Oswestry Disability Index (ODI) for disability and the EQ-5D for quality of life, alongside radiological evaluation of disc volume.

At the 52-week mark, the high-dose group showed a mean VAS pain reduction of 62.8% from baseline, surpassing the minimal clinically important difference (MCID) of a 20-point decrease. This pain relief was sustained through the 104-week follow-up. Moreover, the high-dose group exhibited significant improvements in ODI and EQ-5D scores by 12 weeks, which were maintained throughout the study period. Notably, the high-dose group was the only one to show a significant increase in disc volume, with a mean increase of 249.0 mm3 at 52 weeks and 402.1 mm3 at 104 weeks.

Adverse events (AEs) were reported by 18.3% of patients, with the highest percentage occurring in the placebo group. Serious adverse events were observed in 6.7% of patients, but none were related to the IDCT treatment.

Kevin T. Foley, MD, Chief Medical Officer of DiscGenics, highlighted the consistent evidence of safety and regenerative potential seen in both pre-clinical and clinical studies. Foley emphasized the unique capability of IDCT to regenerate intervertebral discs from the inside out.

DDD is a widespread condition, affecting 12-30% of U.S. adults and accounting for nearly 40% of chronic low back pain cases. It poses a substantial economic burden, with an estimated cost of over $100 billion to the U.S. healthcare system annually.

Flagg Flanagan, CEO and Chairman of DiscGenics, expressed enthusiasm about the study's results, indicating that IDCT has the potential to revolutionize treatment for DDD, a condition with limited therapeutic options. He also mentioned plans to initiate Phase III clinical trials in the U.S. soon.

In summary, IDCT represents a promising therapeutic advancement for patients with lumbar degenerative disc disease. The study results underscore its potential to provide significant, long-lasting pain relief and functional improvement, paving the way for new treatment paradigms in musculoskeletal degeneration.