RESTEM Receives FDA Orphan Drug Designation for its ULSC Program for the Treatment of Polymyositis and Dermatomyositis

CORONA, Calif., Dec. 03, 2024 (GLOBE NEWSWIRE) — RESTEM – a clinical-stage biotechnology company that develops off-the-shelf, next-generation cell therapies designed to modulate the immune system, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its umbilical cord outer lining stem cells (ULSCs) program for the treatment of Polymyositis (PM) and Dermatomyositis (DM).

Polymyositis is an autoimmune disease that causes inflammation and weakness of the skeletal muscles responsible for movement. Dermatomyositis is a form of polymyositis that is associated with skin rash, in addition to muscle inflammation. Both disorders can significantly compromise quality of life and are very challenging to treat. The estimated incidence of PM/DM is approximately 5 to 22 cases per 100,000 individuals¹. Current treatments include long-term use of corticosteroids and immunosuppressive therapies, which can be associated with significant toxicity and other side effects.

“Receiving the Orphan Drug Designation (ODD) is a significant milestone in our mission to provide effective and safer treatment options for patients suffering from PM/DM,” said Keith March, MD, PhD, Chief Medical Officer of RESTEM. “We believe that our novel ULSC therapy has the potential to offer meaningful benefits for patients by modulating the immune system, which may reduce the reliance on steroids. We look forward to the continued development of the ULSC platform and working closely with regulators to bring its potential to patients in need.”

As previously announced, Restem’s ULSC therapy has shown promising results in early clinical trials, demonstrating safety, tolerability and initial clinically significant improvements, as well as the potential to significantly reduce the need for steroid use in patients. The company is currently preparing for Phase 2/3 trials with anticipated initiation in 1Q 2025.

The FDA’s Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for qualified clinical costs, exemptions from certain FDA fees, and seven years of marketing exclusivity.

1. Myopathies. [Updated 2023 May 25]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK532860/

About RESTEM

RESTEM is a leading clinical-stage biotechnology company focused on developing off-the-shelf, next-generation cell therapies designed to modulate the immune system. Leveraging our proprietary products, robust clinical development expertise, and cutting-edge-manufacturing capabilities, we advance two potentially groundbreaking programs, umbilical cord lining stem cells (ULSCs) for autoimmune diseases and natural killer cell (NK) therapeutics targeting senescence and age-associated disorders. Our therapies are intended to treat a broad range of disabling diseases and are designed to improve patient outcomes, as well as overall health and wellness. RESTEM is headquartered in Miami, Florida. For more information, please visit www.restem.com and follow us on X and LinkedIn.

Investor Contact

Daniel Ferry
LifeSci Advisors
+1.617.430.7576
daniel@lifesciadvisors.com

Media Contact

Nelson Cabautan
Restem Group, Inc.
+1.800.490.0924
ncabatuan@restem.com