DiscGenics Announces Completion of Enrollment in U.S. Phase 1/2 Clinical Trial of Discogenic Cell Therapy for Degenerative Disc Disease

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Mar 04, 2020, 08:00 ET

SALT LAKE CITY, March 4, 2020 /PRNewswire/ -- 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 that it has completed enrollment in its Phase 1/2 first-in-human U.S. clinical study of IDCT, an allogeneic, injectable Discogenic Cell therapy for degenerative disc disease (DDD).

IDCT is reproducibly manufactured in a cGMP environment and is subjected to extensive testing, including identity, purity, potency and safety evaluations prior to use. In preparation for an eventual commercial program for IDCT, DiscGenics is in the process of scaling up its cGMP manufacturing process. Learn more on this guided tour of our research laboratories in Salt Lake City, Utah with DiscGenics Chairman and CEO, Flagg Flanagan and Sr. Director of R&D, Dr. Lara Silverman.

This prospective, randomized, double-blinded, vehicle- and placebo-controlled, multicenter clinical study is designed to evaluate the safety and efficacy of IDCT in patients with symptomatic, single-level, mild to moderate lumbar DDD. All 60 subjects have been treated and no safety issues have been reported. Subjects will be followed for a period of two years.

"DDD is a complex medical condition characterized by inflammation and breakdown of intervertebral disc tissue, making it a highly complicated environment to heal," said Domagoj Coric, M.D. of Carolina Neurosurgery and Spine Associates and Spine Division Chief of Atrium's Musculoskeletal Institute as well as the study's principal investigator. "We are thrilled to be participating in the clinical evaluation of IDCT, as we believe it has the potential to reduce inflammation and restore disc height, as demonstrated in preclinical studies. If these findings can be replicated in humans, it could result in reduced pain and disability, making IDCT an extremely attractive therapeutic candidate for the unmet medical need of one of the most common causes of chronic low back pain."

Chronic low back pain is a serious medical condition that represents a leading cause of disability worldwide (1) and is the most common non-cancer reason for opioid prescription in the U.S. (2). It affects 12-30% of U.S. adults at a given time (3) and is estimated to cost the U.S. healthcare system over $100 billion each year (1), 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 (4-6).

"The completion of patient enrollment in our first-in-human U.S. study of IDCT for DDD represents a significant milestone and an important step in the development of this potentially revolutionary treatment for a truly significant unmet medical need," said Flagg Flanagan, Chief Executive Officer and Chairman of the Board of Directors for DiscGenics. "We are looking forward to successful completion of the study. In the meantime, we are focused on the scale up and scale out of our commercial cGMP manufacturing facility in Salt Lake City to meet all regulator requirements and anticipated commercial demand for IDCT."

IDCT is a homologous, allogeneic, injectable cell therapy that utilizes proprietary Discogenic Cells, which are biomedically engineered progenitor cells that have been derived from intervertebral disc tissue. Discogenic Cells are reproducibly manufactured in a highly controlled environment under current good manufacturing practices (cGMP) and subjected to extensive testing throughout production and prior to use, including identity, purity, potency and safety. The final product is cryopreserved and maintained as individual "off-the-shelf" doses for administration in an out-patient setting.

About the IDCT Trial
The IDCT trial is a prospective, randomized, double-blinded, vehicle- and placebo-controlled, multicenter clinical study to evaluate the safety and preliminary efficacy of IDCT in subjects with single-level, symptomatic lumbar intervertebral disc degeneration. The trial is underway in 14 centers across 12 states in the U.S. and has completed enrollment of all 60 subjects with no safety issues reported to-date.

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 (n=20), high dose IDCT (n=20), vehicle (n=10) and 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 will be 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). Importantly, DiscGenics announced in August 2019 that the FDA granted Fast Track designation for IDCT as a potential treatment option for chronic low back pain. For more information on the U.S. study, please visit: https://clinicaltrials.gov/ct2/show/NCT03347708.

IDCT is also being evaluated in a multicenter safety study in Japan, which is supported by a Clinical Trial Notification (CTN) approved by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). For more information on the Japanese study, please visit: https://clinicaltrials.gov/ct2/show/NCT03955315.

About DiscGenics
DiscGenics is a privately held, 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. 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 believes it has a unique opportunity to harness the restorative potential of the human body to heal millions of patients suffering from the debilitating effects of back pain. DiscGenics' first product candidate, IDCT, is a homologous, allogeneic, injectable cell therapy that utilizes biomedically engineered progenitor cells derived from intervertebral disc tissue, known as Discogenic Cells, to offer a non-surgical, potentially regenerative solution for the treatment of patients with mild to moderate degenerative disc disease. For more information, visit discgenics.com.

References

Hoy, D., March, L., Brooks, P., Blyth, F., Woolf, A., Bain, C., Williams, G., Smith, E., Vos, T., Barendregt, J., Murray, C., Burstein, R., and Buchbinder, R. The global burden of low back pain: estimates from the Global Burden of Disease 2010 study. Ann Rheum Dis 73, 968, 2014.
Ringwalt, C., Gugelmann, H., Garrettson, M., Dasgupta, N., Chung, A.E., Proescholdbell, S.K., and Skinner, A.C. Differential prescribing of opioid analgesics according to physician specialty for Medicaid patients with chronic noncancer pain diagnoses. Pain Res Manag 19, 179, 2014.
Davis, M.A., Onega, T., Weeks, W.B., and Lurie, J.D. Where the United States spends its spine dollars: expenditures on different ambulatory services for the management of back and neck conditions. Spine (Phila Pa 1976) 37, 1693, 2012.
Freemont, A.J. The cellular pathobiology of the degenerate intervertebral disc and discogenic back pain. Rheumatology (Oxford) 48, 5, 2009.
Anderson, D.G., and Tannoury, C. Molecular pathogenic factors in symptomatic disc degeneration. Spine J 5, 260S, 2005.
Zhang, Y.G., Guo, T.M., Guo, X., and Wu, S.X. Clinical diagnosis for discogenic low back pain. Int J Biol Sci 5, 647, 2009.