- Receives FDA acceptance on Phase III clinical protocols for
parallel pivotal and confirmatory trials
- Achieves alignment with FDA on Chemistry, Manufacturing, and
Controls (CMC) aspects of Phase III program
SALT
LAKE CITY, July 16, 2024 /PRNewswire/ -- DiscGenics,
Inc., a privately held, late-stage clinical, biopharmaceutical
company developing allogeneic, cell-based, regenerative therapies
for musculoskeletal degeneration, today announced it has gained
acceptance from the U.S. Food and Drug Administration (FDA) for the
clinical protocols and Chemistry, Manufacturing, and Controls (CMC)
clinical development plan for Phase III clinical program of its
allogeneic, injectable disc progenitor cell therapy (IDCT or
rebonuputemcel) for painful lumbar degenerative disc disease (DDD),
allowing the study to proceed.
"There are very few credible treatments on the market for
mechanical back pain, and we believe early clinical evidence
suggests DiscGenics' cell therapy has the potential to shift the
paradigm of care from ablative / destructive procedures or fusion
surgery to a revolutionary regenerative and minimally invasive
treatment for patients suffering from painful degenerative disc
disease," said Nagy Mekhail, MD,
PhD, the Director of Evidence-Based Pain Medicine Research and
Education, Department of Pain Management at the Cleveland Clinic.
"We look forward to participating in the continued clinical
evaluation of this long waited for, promising therapy."
The Phase III clinical program agreed upon with the FDA will
consist of two identical studies running in parallel: the pivotal
study (DGX-A02 or PIVOT) and the confirmatory study (DGX-A03 or
CONFIRM). Both studies are randomized, double-blinded,
Sham-controlled, multicenter studies in subjects with single-level
symptomatic lumbar intervertebral disc degeneration.
"The Phase III clinical program builds on a strong foundation of
clinical data from the combined Phase I/Phase II study of IDCT
showing promising safety and efficacy results, recently published
in the International Journal of Spine Surgery and is informed
by several discussions with the FDA to ensure continued safe
evaluation of IDCT in this patient population where the unmet
clinical need is high," said Kevin T.
Foley, MD, Chief Medical Officer of DiscGenics and Chairman
of Semmes-Murphey Neurologic & Spine Institute.
Each study will have a one-year primary endpoint with another
one-year follow-up period (two-year total duration). The first
subject in the pivotal study is estimated to be enrolled during Q4
2024.
"To get to this point has been a long, but very exciting
journey. From the early founding of this cell technology by the
late Dr. Valery Kukekov to the successful first-in-human clinical
evaluation of IDCT, we have become more confident not only around
the safety of the cell, but also its regenerative potential," said
Flagg Flanagan, Chief Executive
Officer and Chairman of the Board for DiscGenics. "Our team has
been passionately persistent in achieving this important regulatory
milestone, which allows us to continue fulfilling our
responsibility as stewards of this technology."
Through this study, IDCT is being evaluated under an
investigational new drug (IND) allowance by the U.S. Food and
Drug Administration (FDA). If successful, this Phase III clinical
program will support a biologics license application (BLA) to be
filed with the FDA.
About IDCT
IDCT (injectable disc cell therapy, or
rebonuputemcel) is a standalone, single-injection biologic
treatment designed to halt progression of symptomatic lumbar disc
degeneration and regenerate the disc from the inside out. The
active ingredient (Drug Substance) in IDCT is a live, manufactured
progenitor cell population derived from donated adult human
intervertebral disc tissue. 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 regenerative medicine
advanced therapy (RMAT) and Fast Track designations by
the U.S. Food and Drug Administration.
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 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,
late-stage clinical biopharmaceutical company developing
allogeneic, cell-based regenerative therapies for musculoskeletal
degeneration. It's lead product candidate, IDCT (injectable disc
cell therapy, or rebonuputemcel), is a standalone, single-injection
biologic treatment designed to halt progression of lumbar disc
degeneration and regenerate the disc from the inside out.
DiscGenics is also developing a follow-on allogeneic cell platform
to enable new musculoskeletal indications. To further development
of these unique therapies, and to maintain control over compliance,
cost, and production timelines, DiscGenics has built and validated
an in-house scalable, allogeneic cell manufacturing process and
cGMP facility at its headquarters in Salt
Lake City, Utah. For more information,
visit discgenics.com.
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SOURCE DiscGenics, Inc.