Degenerative Disc Disease
Degenerative disc disease is a chronic and progressive condition that is characterized by inflammation and breakdown of tissue within the intervertebral disc.
Located between the vertebral bones in the spine, the intervertebral disc acts like a pillow to cushion and evenly distribute pressure across the vertebrae. Each disc is comprised of an outer fibrous ring, the annulus fibrosus, which surrounds an inner gel-like center, the nucleus pulposus.
When the intervertebral disc becomes inflamed and loses its structure, the disc tissue becomes rigid and weakened, and begins to collapse. This can result in pressure being applied to the surrounding nerves in the spinal column, causing pain and loss of function.
Disc degeneration in the lower back can cause acute or chronic pain at the site of degeneration and may also be felt radiating into the hips, buttocks, thighs or legs. Pain is most commonly felt or made worse by movements such as sitting, bending, lifting or twisting.
Current treatment options include pain management (including opioid use), physical therapy, steroid injections, ablation, chiropractic care and other alternative remedies in the earlier stages, and surgical intervention in the later stages.
Clinical Trials
DiscGenics is preparing to initiate two identical Phase III clinical trials running in parallel: the pivotal trial (DGX-A02 or PIVOT) and the confirmatory trial (DGX-A03 or CONFIRM). Both are randomized, double-blinded, Sham-controlled, multicenter studies in adult subjects with single-level symptomatic lumbar intervertebral disc degeneration.
Through these studies, 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.
This accelerated clinical pathway is enabled by IDCT’s regenerative medicine advanced therapy (RMAT) and Fast Track designations by the U.S. Food and Drug Administration.
Subject recruitment is expected to begin in Q1 2025.
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Past Clinical Trials
DiscGenics has completed two first-in-human, prospective, randomized, double-blinded, controlled, multicenter clinical studies of IDCT (rebonuputemcel) in the U.S. and Japan. Both studies met their primary endpoints.
Results from the U.S. combined Phase I/Phase II study have been published in the peer-reviewed International Journal of Spine Surgery. To summarize, a single intradiscal injection of high-dose IDCT (9,000,000 cells/mL) safely increased disc volume and produced statistically significant, clinically meaningful improvements in back pain, disability, and quality of life out to 2 years post-injection in patients with lumbar disc degeneration.
Expanded Access Policy
DiscGenics is committed to developing cell therapies for patients with disc degeneration. Our goal is to bring these treatments to patients as safely and quickly as possible. We are currently conducting clinical trials to assess the safety and efficacy of our investigational product. DiscGenics believes that participation in our ongoing clinical trial is the best way for patients to access the product. Data generated from these studies is provided to regulatory authorities as part of the product approval process.
We recognize that there are patients with disc degeneration who do not qualify for our trial and these patients may be interested in accessing the investigational cell therapy prior to regulatory approval through expanded access (i.e., compassionate use). Expanded access refers to the use of an investigational therapy (one that has not been approved by the U.S. Food & Drug Administration (FDA) or other applicable regulatory authority) outside of a clinical trial where more comprehensive safety and efficacy data are collected.
According to the US FDA and other regulatory agencies, expanded access may be appropriate if all of the following apply:
The patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition;
There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
Patient enrollment in a clinical trial is not possible;
Potential patient benefit based on available safety and efficacy information justifies the potential risks of treatment;
Providing the investigational medical product will not interfere with, or delay, investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
In the interest of ensuring material availability for clinical enrollment programs DiscGenics does not offer an expanded access program at this time. We believe participating in our trial is the most appropriate way to access our investigational product.
Consistent with the 21st Century Cures Act, DiscGenics may revise this policy at any time.
Understanding Cell Therapy Regulations
While DiscGenics is advancing its DDD treatment through a stringent regulatory process, not all cell therapies are required to adhere to the same level of regulatory rigor.
Some cell-based therapies, like IDCT, undergo significant transformation during the manufacturing process and are subject to clearance by one or more regulatory bodies (e.g. FDA, EMA, PMDA, etc.). On the other hand, some cell therapies may be only minimally manipulated prior to application and thereby are not required to abide by regulations that ensure the safety and efficacy of a treatment.
Before beginning treatment of any kind, we encourage patients and their families to understand the rigorous levels of testing for safety and efficacy a product has undergone before considering it as a treatment for themselves or loved ones.
Disclaimer: IDCT (rebonuputemcel) 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.