FDA Allows Q-Cells(R) for First-In-Human Transverse Myelitis Trial

SALT LAKE CITY, UT--(Marketwired - Jul 25, 2017) - Q Therapeutics, Inc., developer of clinical-stage cell therapies for central nervous system (CNS) disease and injury, announced U.S. Food and Drug Administration (FDA) allowance of its Investigational New Drug (IND) application to proceed with a Phase 1/2a clinical trial of Q-Cells® in patients with Transverse Myelitis™. TM is a crippling inflammation of the spinal cord that affects approximately 30,000 people in the United States, with more than 1,700 new cases diagnosed each year. Similar to Multiple Sclerosis (MS), TM destroys the myelin sheath surrounding nerve fibers in the spinal cord, compromising muscle control and affecting sensation.

"This is another milestone in our quest to bring effective treatments for devastating CNS diseases and injuries to the clinic," said Steven Borst, the company's CEO and chairman. Clinical trials in the nine-person, dose-escalation TM safety study will pave the way to developing Q-Cells for treatment of several debilitating disorders caused by a lack of healthy glial cells. "Our approach uses the glial cell's natural ability to repair and support nerve cells in the CNS. Q-Cells hold great promise not only for those people with rare diseases such as TM and ALS, but for the many people worldwide who live with MS, spinal cord injury and stroke." The company also has FDA allowance to initiate a Phase 1/2a clinical trial in Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease).

Q-Cells are human glial-restricted progenitor cells (GRPs) and the Company's first patented cellular therapeutic candidate. As reported in the May 2017 issue of Experimental Neurology, Piotr Walczak, MD, PhD, and his team at The Johns Hopkins University showed that Q-Cells exert a robust therapeutic effect when transplanted into the CNS of animals born unable to produce healthy myelin. The Q-Cells performed multiple repair and support functions resulting in myelination and restoration of animals to normal function with extended life span. These data, along with other published pre-clinical data from animal models of CNS disease and injury, show that delivering healthy glial cells into the brain and spinal cord can alter the course of currently incurable CNS conditions. 

"The FDA's clearance of this IND is yet another validation of our collaborative and purposeful approach to move Q-Cells into the clinic," said James Campanelli, PhD, Vice President of Research and Development at Q Therapeutics. "We developed this IND application leveraging our pre-clinical and therapeutic manufacturing experience, and achieved regulatory allowance in under a month. This approach will serve us well as we explore other disease indications for Q-Cells. We are grateful to all of our team members, academic collaborators and corporate partners without whom this step forward would not be possible."

"We have long believed that Q-Cells' unique ability to repair and support CNS nerve cells is fundamental to treating many CNS disorders. The ability of these cells to replicate once injected, migrate, differentiate into mature glial cells and repair myelin, as demonstrated by Dr. Walczak's lab, further highlights the power of this therapeutic approach," said Mahendra Rao, MD, PhD, Q Therapeutics scientific co-founder and chief strategy officer. "We are eager to move forward with this trial and optimistic that Q-Cells will prove effective in treating human CNS injury and disease."

Collaborative development of the Q-Cells therapeutic approach with Dr. Walczak and Nicholas Maragakis, MD of The Johns Hopkins University has received substantial support from the National Institute of Neurological Disorders and Stroke Translational Research Program (U01-NS06713) and the Maryland Stem Cell Research Fund. Q Therapeutics worked with MPI Research, Inc., to carry out preclinical safety studies, Goodwin Biotechnology, Inc., to manufacture the antibody used in cell purification, and Biologics Consulting Group for regulatory guidance. The company also partnered with the Cell Therapy and Regenerative Medicine Facility (CTRM) at the University of Utah in developing Good Manufacturing Processes to create the clinical-grade product.

About Q Therapeutics, Inc. -- Headquartered in Salt Lake City, Q Therapeutics is a clinical-stage company developing adult stem cell therapies to treat debilitating central nervous system (CNS) disease and injury. 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 ™, cerebral palsy and stroke, as well as other neurodegenerative diseases and injuries such as Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease), Huntington's disease, spinal cord injury, traumatic brain injury, and Alzheimer's disease. Q Therapeutics' initial clinical targets are TM and ALS, with INDs in both indications now accepted to proceed by the FDA. The Company's proprietary product pipeline also includes neural cell products derived from induced pluripotent stem cells (iPSC). 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.