DiscGenics Announces Positive Two-Year Clinical Data from U.S. Study of Discogenic Progenitor Cell Therapy for Degenerative Disc Disease

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 DDD.

SALT LAKE CITY,Jan. 25,2023 -- 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).

During treatment,a single dose of IDCT is injected into the painful disc percutaneously.

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,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 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 mm3,p=0.0284) and Week 104 (402.1 mm3,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,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,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 11,2023.

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,double-blinded,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 usage.

Upon enrollment,eligible subjects were randomized to one of four treatment cohorts: low dose IDCT (3,000 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) allowanceby 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: 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 economyand 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,visitdiscgenics.com.

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