RESTEM Announces First-Patient-Dosed in the Phase 1/2a Study of Restem-L in Facioscapulohumeral Muscular Dystrophy

− First patient with facioscapulohumeral muscular dystrophy (FSHD) dosed with Restem-L umbilical lining modified progenitor cells (UMPCs)

− The Phase 1/2a study will evaluate the safety and preliminary efficacy of Restem-L in patients with FSHD

− Clinical study is funded by SOLVE FSHD

MIAMI and VANCOUVER, British Columbia, Dec. 08, 2025 (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 the initiation of a Phase 1/2a study of Restem-L, the Company’s umbilical lining modified progenitor cells (UMPCs) off-the-shelf platform, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The study is funded by SOLVE FSHD, a venture philanthropic organization dedicated to accelerating treatments for FSHD.

“We are excited to partner with SOLVE FSHD and initiate this Phase 1/2a clinical trial of Restem-L in patients with FSHD. This clinical trial allows us to explore Restem-L’s potential beyond autoimmune diseases, and expand our reach to other inflammatory-driven serious conditions with high unmet medical needs such as FSHD - a genetic neuromuscular disease with no approved therapies,” said Andres Isaias, Chief Executive Officer of RESTEM. “Our collaboration with SOLVE FSHD highlights the growing interest in Restem-L, and further underscores the increasing recognition of these cells as a potentially potent, efficacious and safe next generation, immune-modulatory cell therapy. We are grateful for this collaboration and look forward to further building on the already strong safety profile and compelling results demonstrated with Restem-L in different indications.”

“We are delighted to partner with RESTEM and have the first patient with FSHD dosed with Restem-L in the Phase 1/2a trial, marking an important milestone in our efforts to advance potential therapies for this debilitating condition, where there are currently no effective available treatments,” commented Eva Chin, Ph.D., Executive Director of SOLVE FSHD. “FSHD is a rare neuromuscular disease characterized by immune cell infiltration and inflammation in skeletal muscles, ultimately leading to their degeneration, progressive weakness and loss of facial and upper body function and eventually impaired mobility. We were very impressed with the ability of RESTEM’s UMPCs to modulate key inflammatory factors in related muscle inflammatory indications, and chose to fund this study after reviewing numerous other technologies and platforms. The initiation of this trial inches us closer to bringing hope to the up to 1 million patients estimated to suffer from FSHD worldwide.”

The Phase 1/2a study (NCT07086521) is a double-blind, randomized, dose-repeating, placebo-controlled, cross over study to assess the safety and preliminary efficacy of allogeneic UMPCs on disease severity in patients with FSHD. The trial, led by John Day, M.D., Ph.D. in Stanford University, aims to enroll 16 patients, randomized 1:1 to receive either Restem-L or placebo for 6 months, and then crossed-over to receive either placebo or Restem-L, respectively for additional 6 months, with a treatment duration of 12 months and study follow up at 15 and 21 months. The primary endpoint of the study is safety with secondary and exploratory outcomes including serum markers of immune/inflammatory response, patient and clinician reported functional outcomes, MRI and muscle biopsy biomarkers.

About Facioscapulohumeral Muscular Dystrophy (FSHD)

FSHD is a genetic muscle disorder characterized by progressive muscle weakness, primarily affecting the face, shoulder blades, and upper arms. It is one of the most common forms of muscular dystrophy and typically begins in adolescence or early adulthood, though one can vary. It affects approximately 1 in 8000 people worldwide, and while there is currently no cure, available treatments focus on managing symptoms, slowing disease progression, and enhancing quality of life.

About RESTEM

RESTEM is a leading clinical-stage biotechnology company focused on developing off-the-shelf, next-generation cell therapies for autoimmune, inflammatory, and age-related diseases. Leveraging proprietary products, deep clinical expertise, and advanced manufacturing capabilities, RESTEM is advancing two potentially transformative programs, Restem-L, our umbilical cord lining progenitor cells (UMPCs) therapy for autoimmune diseases, and activated natural killer cell (aNK) therapeutics targeting senescence and age-associated disorders.  Our therapies are designed to reprogram the immune system rather than focusing solely on symptom management, offering patients with limited options the potential to address underlying disease mechanisms. RESTEM is headquartered in Miami, Florida. For more information, please visit www.restem.com and follow us on X and LinkedIn.

About SOLVE FSHD
SOLVE FSHD is a venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD. Established by renowned Canadian entrepreneur and philanthropist Chip Wilson, widely known as the founder of lululemon and part owner of Amer Sports which holds renowned brands such as Arc’teryx, Salomon and Wilson Sports. Chip has committed $100 million to kick-start funding into projects that support the organization’s mission to find a cure for FSHD by 2027. The goal of SOLVE FSHD is to find a solution that can stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD. For more information, visit  https://solvefshd.com/

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