DiscGenics Announces Positive Two-Year Clinical Data from U.S. Study of Discogenic Progenitor Cell Therapy for Degenerative Disc Disease
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. (
), 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 (
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
(
)
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 (
) 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 (
).
SOURCE DiscGenics, Inc.
CONTACT: Lindsey Saxon, lindsey@discgenics.com
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