FDA DESIGNATION WOULD PROVIDE 7 YEARS OF MARKET EXCLUSIVITY UPON MARKET APPROVAL OF Q-CELLS®
SALT LAKE CITY, Sept. 26, 2018 (GLOBE NEWSWIRE) -- Q Therapeutics, Inc., a developer of clinical-stage cell therapies for central nervous system (CNS) diseases announced that its Q-Cells® product (human glial restricted progenitor cells and their progeny) has received the FDA’s orphan drug designation for the treatment of transverse myelitis.
“Orphan drug designation of Q-Cells for transverse myelitis is a great step toward our goal of advancing glial cell therapy for demyelinating and neurodegenerative diseases,” said Steven Borst, President and CEO of Q Therapeutics, Inc. “People who are hit hardest by transverse myelitis face a lifetime of paralysis and wheelchair confinement. Building upon the remarkable, disease-modifying results that have been achieved in pre-clinical studies, we aim to someday reverse that paralysis, not only in these patients, but all who have disabilities that result from damaged or destroyed myelin.”
Transverse myelitis (TM) is a severely debilitating autoimmune disease that destroys key components of the spinal cord and leads to total paralysis in many of the people it afflicts. It is similar to multiple sclerosis, but TM paralyzes healthy people over a very short period of time – sometimes just 24 to 36 hours – when the immune system attacks the myelin sheaths surrounding nerve fibers. No effective treatments yet exist to restore myelin once it has been destroyed.
Last year, collaborators at The Johns Hopkins University showed that Q-Cells replaced missing myelin in mice, restoring the animals to normal function and life expectancy. These robust data supported swift FDA clearance of Q Therapeutics’ proposed TM clinical trial. Similar experiments in animal models of spinal cord injury also have demonstrated the restorative capabilities of Q-Cells.
Orphan Drug Designation is granted to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 patients in the United States, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases and conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare disease and to further advance scientific development of such promising medical products. The office also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry and rare disease patient groups.
About Q Therapeutics, Inc.
Q Therapeutics is a clinical-stage company developing adult stem cell therapies to treat debilitating CNS disease and injury. The Company has patented processes to derive and manufacture glial-restricted progenitor (GRP) cells from any tissue source, including iPSC. The Company’s first therapeutic product candidate, Q-Cells®, is intended to restore or preserve normal CNS activity by supplying essential nerve cell functions. Q-Cells may be suitable to treat a range of CNS disorders, including demyelinating conditions such as multiple sclerosis (MS), transverse myelitis (TM), cerebral palsy and stroke, as well as neurodegenerative diseases and injuries such as amyotrophic lateral sclerosis (ALS), spinal cord injury, traumatic brain injury, and Alzheimer’s disease. Q Therapeutics has obtained Investigational New Drug (IND) clearance from the FDA for Phase 1/2a trials in both ALS and TM for its adult cell product, Q-CELLS. In addition, the FDA has granted orphan drug status and fast track designation to Q-CELLS for ALS. For more information, see www.qthera.com.
Cautionary Statement Regarding Forward Looking Information
This news release may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Q Therapeutics’ technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of its intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect results and other risks and uncertainties are detailed from time to time in Q Therapeutics’ periodic reports.
Name: Steven Borst
Telephone: 801-582-5400, ext. 104