FDA Grants Orphan Drug Status to RESTEM's ULSC Program for Polymyositis and Dermatomyositis Treatment

CORONA, Calif., Dec. 03, 2024 – RESTEM, a biotechnology firm at the clinical trial stage, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its umbilical cord outer lining stem cells (ULSCs) program. This program aims to treat Polymyositis (PM) and Dermatomyositis (DM), both debilitating autoimmune disorders.

Polymyositis is characterized by the inflammation and weakening of skeletal muscles, which are critical for movement. Dermatomyositis, a variant of Polymyositis, not only involves muscle inflammation but also presents with a skin rash. Both conditions severely affect the quality of life and present significant treatment challenges. The prevalence of PM/DM is estimated to be between 5 and 22 cases per 100,000 individuals. Currently, the primary treatments for these conditions involve prolonged use of corticosteroids and immunosuppressive therapies, which often come with substantial side effects and toxicity.

Keith March, MD, PhD, Chief Medical Officer of RESTEM, remarked that receiving ODD represents a crucial step forward for the company’s goal to offer safer and more effective treatment solutions for PM/DM patients. He expressed optimism that RESTEM's innovative ULSC therapy could provide significant benefits by modulating the immune system, potentially decreasing dependency on steroids. He also indicated the company's commitment to furthering the ULSC platform and collaborating closely with regulators to bring this therapy to those in need.

Previous announcements have highlighted RESTEM’s ULSC therapy demonstrating promising outcomes in early clinical trials. The therapy was well-tolerated, showed safety, and indicated initial clinically significant improvements. Moreover, it suggested a potential reduction in the need for steroid use among patients. RESTEM is gearing up for Phase 2/3 trials, expected to commence in the first quarter of 2025.

The FDA’s Office of Orphan Products Development awards ODD to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 individuals in the United States. This designation offers several benefits to drug developers, including support in the drug development process, tax credits for qualified clinical costs, waivers from specific FDA fees, and seven years of marketing exclusivity.

RESTEM is a pioneering biotechnology company dedicated to creating next-generation, off-the-shelf cell therapies aimed at modulating the immune system. The company leverages proprietary products, substantial clinical development proficiency, and advanced manufacturing capabilities. RESTEM is advancing two key programs: ULSCs for autoimmune diseases and natural killer cell (NK) therapies for senescence and age-related disorders. These therapies are designed to treat various disabling diseases and enhance patient outcomes and overall health. RESTEM is headquartered in Miami, Florida.