DiscGenics’ Discogenic Progenitor Cell Therapy Rebonuputemcel Cleared for Phase 3 Program in Lumbar Degenerative Disc Disease
The clearance is based on the agency’s acceptance of clinical trial protocols and a chemistry, manufacturing, and controls clinical development plan for the program.
DiscGenics has received clearance from the FDA to go forward with its phase 3 clinical program for its allogeneic discogenic progenitor cell therapy rebonuputemcel, which is also known as injectable disc cell therapy (IDCT), for the treatment of painful lumbar degenerative disc disease (DDD).1
The clearance is based on the agency’s acceptance of clinical trial protocols and a chemistry, manufacturing, and controls clinical development plan for the program. The phase 3 program will consist of a pivotal study (DGX-A02 or PIVOT) and a confirmatory study (DGX-A03 or CONFIRM), 2 identical randomized, double-blinded, sham-controlled, multicenter clinical trials that will be carried out alongside one another. The 2 trials, which will recruit patients with single-level symptomatic lumbar intervertebral disc degeneration, will last 2 years and will include a primary end point measured at 1-year posttreatment and an additional year of follow-up after that.
“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 revolutionary regenerative and minimally invasive treatment for patients suffering from painful degenerative disc disease,” Nagy Mekhail, MD, PhD, the director of evidence-based pain medicine research and education in the Department of Pain Management at the Cleveland Clinic, said in a statement.1 “We look forward to participating in the continued clinical evaluation of this long waited for, promising therapy.”
DiscGenics anticipates that the first patient in PIVOT will be enrolled within the fourth quarter of this year. Pending positive results, the company intends to use data from the phase 3 program to support a biologics license application for IDCT. IDCT has previously received regenerative medicine advanced therapy (RMAT) designation and fast track designations from the FDA.
IDCT has previously been evaluated in a phase 1/2 clinical trial (NCT03347708), results from which were reported in the International Journal of Spine Surgery.2 According to DiscGenics’ summary of the findings, the study’s primary end point for back pain reduction was met in the high-dose (9,000,000 cells/mL) cohort with a mean visual analog scale (VAS) pain decrease from baseline of −62.8% (P = 0.0005) at 52 weeks posttreatment. Notably, the outcome constituted a significantly greater change than the minimal clinically important difference, which is set at a 20-point decrease (−42.8, P = 0.001), and the clinical improvement was sustained at 104 weeks posttreatment. In terms of safety, severe adverse events (AEs) occurred in 18.3% of patients, but the highest percentage of these were in patients who were treated with the placebo. Furthermore, of the 6.7% of patients who experienced serious AEs, all were in those treated with the vehicle alone or placebo and none of the serious AEs were deemed related to treatment.
“The results from this study demonstrate IDCT’s potential to safely and effectively reduce pain associated with DDD while also producing a regenerative effect within the degenerating disc,” lead study author Matthew F. Gornet, MD, a board certified spine surgeon at The Orthopedic Center of St. Louis and a top enroller in the IDCT study, said in a July 9, 2024 statement.2 “MRI image analysis of disc volume indicated the potential to halt and possibly reverse the progression of the disease. I have been a practicing spine surgeon for more than 30 years and been involved in over 35 FDA clinical trials, and the patient outcomes from this study are very promising.”
