Allogeneic discogenic cell therapy was well tolerated and produced clinically meaningful, statistically significant improvements in low back pain, function, and quality of life by 12 weeks following intradiscal injection; improvements were sustained at the one-year interim readout with additional data to follow at 18 and 24 months.
Salt Lake City, UT – February 25, 2022 – DiscGenics, Inc., a clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine, today announced the presentation of positive interim data from its ongoing Phase 1/2 clinical trial of IDCT (rebonuputemcel), an allogeneic injectable discogenic cell therapy for degenerative disc disease (DDD), at Spine Summit 2022.
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 >30% as measured on a 100mm Visual Analog Scale (VAS) at Weeks 12 (‑54.53% [‑69.46, ‑39.60], p=0.0056), 26 (‑50.94% [‑66.10, ‑35.78], p=0.0140), and 52 (‑62.79% [‑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.
Importantly, 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.
“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, Chief Executive Officer and Chairman of the Board 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 in the process of analyzing data from patient follow-up visits at weeks 78 and 104. The Company intends to submit the full dataset to the U.S. Food & Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) for consideration in an End of Phase 2 meeting targeted for the second half of 2022.
In the meantime, 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.
About IDCT
IDCT (rebonuputemcel) is a homologous, allogeneic, injectable cell therapy that utilizes proprietary Discogenic Cells, which are biomedically engineered progenitor cells that have been derived from human intervertebral disc tissue. Discogenic Cells are reproducibly manufactured in a highly-controlled environment under current good manufacturing practices (cGMP). They are subjected to extensive testing throughout production and prior to use, including identity, purity, potency, and safety analyses. The final product is cryopreserved and maintained as individual “off-the-shelf” doses for administration via percutaneous injection in an outpatient setting. IDCT has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA).
About the IDCT Trial
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 U.S. 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.
Through this study, IDCT is being evaluated under an investigational new drug (IND) allowance by the U.S. Food and Drug Administration (FDA) and will be regulated as a drug-biologic through a therapeutics biologics license application (BLA). Importantly, DiscGenics announced in August 2019 that the FDA granted Fast Track designation for IDCT as a potential treatment option for chronic low back pain. For more information on the U.S. study, please visit: https://clinicaltrials.gov/ct2/show/NCT03347708.
About Chronic Low Back Pain and Degenerative Disc Disease
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 12-30% 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% of patients, low back pain is caused by DDD, a chronic and progressive condition where the intervertebral disc breaks down and causes pain.
About DiscGenics
DiscGenics is a privately held, clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. As the only company in the world to develop an allogeneic cell therapy derived from intervertebral disc cells to treat diseases of the disc, DiscGenics believes it has a unique opportunity to harness the restorative potential of the human body to heal millions of patients suffering from the debilitating effects of back pain. DiscGenics’ first product candidate, IDCT (rebonuptemcel), is a homologous, allogeneic, injectable cell therapy that utilizes biomedically engineered progenitor cells derived from intervertebral disc tissue, known as Discogenic Cells, to offer a non-surgical, potentially regenerative solution for the treatment of patients with mild to moderate degenerative disc disease. For more information, visit discgenics.com.