- 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, UT, USA I July 16, 2024 I 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.
SOURCE: DiscGenics
Post Views: 5,787
- 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, UT, USA I July 16, 2024 I 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.
SOURCE: DiscGenics
Post Views: 5,787
