DiscGenics Touts Positive Clinical Data for DDD Cell Therapy

A treatment for degenerative disc disease is showing promise in clinical trials.

DiscGenics Inc.’s injectable cell therapy reduced back pain and improved patients’ quality of life, according to interim data. The company’s IDCT (rebonuputemcel) is a homologous, allogeneic, injectable cell therapy that uses proprietary discogenic cells—biomedically engineered progenitor cells that have been derived from human intervertebral disc tissue. IDCT has been granted Fast Track designation in August 2019 by the U.S. Food and Drug Administration (FDA).

The primary efficacy endpoint of the study (n=60) was achieved, with statistically significant improvement in back pain observed in the high dose IDCT group. For these patients, low back pain scores improved more than 30 percent as measured on a 100mm Visual Analog Scale (VAS) at Weeks 12 (–54.53 percent [–69.46, –39.60], p=0.0056), 26 (–50.94 percent [–66.10, –35.78], p=0.0140), and 52 (–62.79 percent [–77.13, –48.46], p=0.0005). At these same timepoints, high dose IDCT also produced clinically meaningful, statistically significant improvements in function as measured by the Oswestry Disability Index (ODI) and quality of life as measured by the EQ-5D Index Score. No subjects in the IDCT treatment groups have experienced serious treatment-emergent adverse events (TEAEs).
 
“We are very encouraged by this interim clinical data. Not only are we seeing a strong safety profile and dramatic pain reduction with a dose response that occurs early and continues at the one-year time point in patients who received the high dose IDCT, we’re also seeing rapid, significant, and durable improvements in function and quality of life,” said Kevin T. Foley, M.D., chief medical officer for DiscGenics and chairman of Semmes-Murphy Neurologic & Spine Institute. “As we await the 78-week and 104-week final data readouts, we hope to see sustained and meaningful improvement in each of these life-changing measures.”
 
The improvements in pain, function, and quality of life at one-year post-injection exceeded what are known as minimum clinically important differences (MCIDs), which reflect changes in a clinical intervention that are meaningful for the patient. DiscGenics is in the process of analyzing data from patient follow-up visits at weeks 78 and 104. The company plans to submit data to the FDA’s Office of Tissues and Advanced Therapies (OTAT) in the second half of this year.
 
“The significant and durable improvements we’re seeing in pain, function, and quality of life are critical indicators of the potential IDCT has to change the paradigm of care for patients with DDD,” said Flagg Flanagan, CEO and board chairman for DiscGenics. “As stewards of this technology, the dedicated and talented team here at DiscGenics is working tirelessly to usher IDCT through the clinical and regulatory process to commercialization, with the ultimate goal of improving the lives of millions of people suffering from the debilitating effects of chronic low back pain.”
 
DiscGenics is continuing to scale up its in-house manufacturing capabilities so it will have cells ready for future clinical and/or commercial application, depending on the outcome of the FDA’s review of the data.
 
The IDCT trial is a prospective, randomized, double-blinded, vehicle- and placebo-controlled, multicenter clinical study to evaluate the safety and efficacy of IDCT in subjects with single-level, symptomatic lumbar intervertebral disc degeneration. The trial is underway in 14 centers across 12 states in the United States and has completed enrollment of all 60 subjects with no safety issues reported to date.
 
Prior to enrollment, each participating subject was screened and verified to have met all eligibility criteria, including: having early to moderate symptomatic, single-level DDD from L3-S1, no previous lumbar spine surgery, no radiculopathy (pinched nerve) or leg pain, and no comorbidities, such as tumors, fibromyalgia, systemic disease, osteoarthritis or chronic opioid usage. Upon enrollment, eligible subjects were randomized to one of four treatment cohorts: low dose IDCT (3,000,000 cells/mL; n=20), high dose IDCT (9,000,000 cells/mL; n=20), vehicle alone (n=10) or saline placebo (n=10). Each subject received a single intradiscal injection of his or her assigned treatment into the target symptomatic lumbar intervertebral disc. In accordance with the trial design, subjects in all cohorts will be observed and evaluated for two years. Primary outcome measures include safety and reduction in pain. Secondary outcome measures include reduction in disability and radiographic improvement. 

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 U.S. It affects 12 percent to 30 percent of U.S. adults at a given time and is estimated to cost the U.S. healthcare system over $100 billion each year, creating a significant burden on the economy and individual patients dealing with the condition. In nearly 40 percent of patients, low back pain is caused by DDD, a chronic and progressive condition where the intervertebral disc breaks down and causes pain.
 
DiscGenics develops regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. It claims to be the only company in the world to develop an allogeneic cell therapy derived from intervertebral disc cells to treat diseases of the disc.