Country for PR: United States
Contributor: PR Newswire New York
Wednesday, January 25 2023 - 13:09
DiscGenics Announces Positive Two-Year Clinical Data from U.S. Study of Discogenic Progenitor Cell Therapy for Degenerative Disc Disease
SALT LAKE CITY, Jan. 25, 2023 /PRNewswire-AsiaNet/ --

-- Discogenic progenitor cell therapy safely increased disc volume and provided 
rapid, durable improvements in low back pain, function, quality of life, and 
pain medication usage out to two years post-injection in patients with lumbar 

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 positive 
two-year clinical data from its first-in-human clinical study of IDCT 
(rebonuputemcel), an allogeneic discogenic progenitor cell therapy for lumbar 
degenerative disc disease (DDD).

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In the FDA-allowed prospective, randomized, double-blind, vehicle- and 
placebo-controlled, multicenter clinical study, high dose IDCT (9,000,000 
cells/mL; n=20) met the primary safety and efficacy endpoints and demonstrated 
statistically significant improvements in low back pain, function, quality of 
life, and disc volume, suggesting a regenerative effect following a single 
injection into the intervertebral disc.

Key findings include:

    -- The primary safety endpoint of the study was achieved with no 
       subjects in the IDCT treatment groups experiencing treatment
       -emergent serious adverse events (TESAEs).

    -- As previously reported 
       the primary efficacy endpoint of the study was achieved, with 
       statistically significant improvement in back pain scores by >30% as 
       measured on a 100mm Visual Analog Scale (VAS) observed in the high 
       dose IDCT group at 52 weeks (–62.79%, p=0.0005). A smaller, 
       significant decrease in VAS was also observed in the vehicle group.

    -- Clinically meaningful, statistically significant improvements in low 
       back pain (VAS), function (ODI), and quality of life (EQ-5D) were 
       observed by 12 weeks following intradiscal injection with high dose 
       IDCT in subjects with symptomatic lumbar disc degeneration.

    -- These clinical improvements were sustained at six months, one year, 
       1.5 years, and two years post-injection and statistically exceeded the 
       Minimal Clinically Important Difference (MCID) in each respective 
       outcome measure, which reflect changes following a clinical 
       intervention that are meaningful for the patient.

    -- In the low dose IDCT group (3,000,000 cell/mL; n=20), there was a 
       trend in improvement of clinical outcomes, though inconsistent. While 
       the vehicle control group (n=10) resulted in some pain relief, it was 
       not associated with clinically meaningful improvements in function or 
       quality of life. No consistent or durable statistically significant or 
       clinically meaningful outcomes were observed in the saline placebo 
       control group (n=10).

    -- Statistically significant improvements in disc volume were also 
       observed in the high dose IDCT group, where MRI imaging-derived mean 
       change in disc volume increased steadily from baseline and reached 
       statistical significance at Week 52 (249.01 mm(3), p=0.0284) and Week 
       104 (402.1 mm(3), p=0.028).

    -- In contrast, changes in disc volume for the control groups decreased, 
       although not at a statistically significant level.

    -- Importantly, the high dose IDCT treatment group was the only group in 
       this study to show a decrease in both opioid and nonsteroidal anti-
       inflammatory drug (NSAID, e.g. aspirin, ibuprofen, etc.) use.

    -- At 2 years, overall patient follow-up was 85.0%.

"These clinical results demonstrate the incredible potential of DiscGenics's 
IDCT to safely treat not only the pain and disability associated with DDD with 
a single injection, but also to address the underlying cause of the disease—the 
degenerating disc. This is unlike any treatment I have seen in 30 years of 
practice and unlike any treatment currently available on the market," said 
Matthew F. Gornet, M.D., Board Certified Spine Surgeon at The Orthopedic Center 
of St. Louis and top enroller in the IDCT study. "The improvements we observed 
in disc volume through MRI image analysis suggest DiscGenics's IDCT produces a 
regenerative effect within the degenerating disc which indicates the ability to 
halt and possibly reverse the progression of DDD."

The 60-subject study was designed to evaluate the safety and preliminary 
efficacy of IDCT for the treatment of symptomatic lumbar degenerative disc 
disease versus vehicle and saline controls. Subjects were enrolled at 13 
centers across 12 states.

In this study, low back pain was measured on a 100-mm Visual Analog Scale 
(VAS), function was measured via the Oswestry Disability Index Questionnaire 
(ODI), and quality of life was measured using the EQ-5D Index Score.

"We are very encouraged by the final two-year results of this study," said 
Flagg Flanagan, Chief Executive Officer and Chairman of the Board for 
DiscGenics. "The significant and durable improvements we saw in pain, function, 
quality of life, disc volume, and concomitant pain medication usage are 
critical indicators of the potential for IDCT to change the paradigm of care 
for patients with DDD."

DiscGenics has submitted a full clinical study report to the U.S. Food & Drug 
Administration's (FDA) Office of Tissues and Advanced Therapies (OTAT).

Simultaneously, DiscGenics is scaling up its in-house manufacturing 
capabilities so it will have cells ready for future application, pending the 
FDA's review of the data.

A summary of this data has been presented at:

    -- The American Academy of Neurological Surgery (AAcNS) 84th Annual 
       Meeting ( 
       by Kevin T. Foley, MD, Professor of Neurosurgery at the University of 
       Tennessee Health Science Center and Chairman of Semmes-Murphey Clinic 
       on September 29, 2022.

    -- The North American Spine Society (NASS) 37th Annual Meeting 
       by Matthew F. Gornet, MD, Board Certified Spine Surgeon at The 
       Orthopedic Center of St. Louis on October 12, 2022.

    -- The 41st Annual J.P. Morgan Healthcare Conference 
       by Flagg Flanagan, CEO and Chairman of DiscGenics on January 11, 

About IDCT
IDCT (rebonuputemcel) is an allogeneic injectable discogenic progenitor cell 
therapy intended for patients with symptomatic early to moderate degenerative 
disc disease. The active ingredient (Drug Substance) of IDCT is a live 
discogenic progenitor cell population derived from the intervertebral disc 
tissue of adult organ donors. These cells are enriched and expanded into 
Discogenic Cells through a multistep manufacturing process in a highly 
controlled environment under current good manufacturing practices (cGMP) that 
results in significant proliferation and phenotypic changes to the cells. At 
the completion of the manufacturing process, the Discogenic Cells are subjected 
to extensive testing prior to use, including identity, purity, potency, and 
safety evaluations. The Discogenic Cells are then mixed with a viscous Sodium 
Hyaluronate Solution and excipients to generate IDCT, the Final Drug Product. 
IDCT is cryopreserved and maintained as individual "off-the-shelf" doses for 
administration via percutaneous injection into the intervertebral disc in an 
outpatient setting. IDCT has been granted Fast Track designation by the U.S. 
Food and Drug Administration (FDA). 

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

About the IDCT Clinical Study (DGX-A01)
DGX-A01 was 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. Sixty subjects were enrolled in 13 centers 
across 12 U.S. states.

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 

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 were 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).

For more information on the U.S. study, please visit:

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 
developing regenerative cell-based therapies that alleviate pain and restore 
function in patients with degenerative diseases of the spine. DiscGenics's 
first product candidate, IDCT (rebonuputemcel), is an allogeneic, injectable 
discogenic progenitor cell therapy for symptomatic, mild to moderate lumbar 
disc degeneration. IDCT is a mixture of live Discogenic Cells, which are a 
manufactured progenitor cell population derived from donated adult human 
intervertebral disc tissue, and a viscous carrier. 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 has a unique opportunity to 
offer a non-surgical, potentially regenerative solution for the treatment of 
patients suffering from the debilitating effects of back pain. For more 
information, visit ( 

SOURCE DiscGenics, Inc.

CONTACT: Lindsey Saxon,