PR99503

SALT LAKE CITY, Jan. 25, 2023 /PRNewswire=KYODO JBN/ --

-- 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.

DiscGenics, Inc. (

https://c212.net/c/link/?t=0&l=en&o=3764995-1&h=3788254259&u=https%3A%2F%2Fwww.discgenics.com%2F&a=DiscGenics%2C+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).

Photo - https://mma.prnewswire.com/media/1989076/Discgenics_IDCT_injection.jpg

Logo - https://mma.prnewswire.com/media/561675/DiscGenics_Logo.jpg

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

       (

https://c212.net/c/link/?t=0&l=en&o=3764995-1&h=3750864484&u=https%3A%2F%2Fwww.discgenics.com%2Fnews-posts%2F2022%2F2%2F25%2Fdiscgenics-announces-presentation-of-positive-interim-clinical-data-from-phase-12-study-of-cell-therapy-for-degenerative-disc-disease&a=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 (

https://c212.net/c/link/?t=0&l=en&o=3764995-1&h=3326979980&u=https%3A%2F%2Fwww.americanacademyns.org%2FDefault.aspx&a=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

       (

https://c212.net/c/link/?t=0&l=en&o=3764995-1&h=2508384701&u=https%3A%2F%2Fwww.spine.org%2Fam&a=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

       (

https://c212.net/c/link/?t=0&l=en&o=3764995-1&h=3174166431&u=https%3A%2F%2Fwww.jpmorgan.com%2Fsolutions%2Fcib%2Finsights%2Fhealth-care-conference&a=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, 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

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 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 (

https://c212.net/c/link/?t=0&l=en&o=3764995-1&h=1468027561&u=https%3A%2F%2Fwww.discgenics.com%2Fnews-posts%2Fdiscgenics-receives-fda-allowance-of-ind&a=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:

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

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 discgenics.com (

https://c212.net/c/link/?t=0&l=en&o=3764995-1&h=1883600422&u=https%3A%2F%2Fwww.discgenics.com%2F&a=discgenics.com

).

SOURCE DiscGenics, Inc.

CONTACT: Lindsey Saxon, lindsey@discgenics.com