RESTEM Doses First Patient in Muscular Dystrophy Trial

Biotechnology company RESTEM announced the start of a Phase 1/2a clinical trial for Restem-L, a cell therapy targeting facioscapulohumeral muscular dystrophy (FSHD). The first patient has now been dosed in the study, which is designed to assess the safety and preliminary efficacy of the UMPCs in FSHD patients.

The clinical study is funded by SOLVE FSHD, an organization focused on accelerating treatments for the rare neuromuscular disease. FSHD is characterized by progressive muscle weakness, primarily affecting the face, shoulder blades, and upper arms. There are currently no approved therapies for the condition.

RESTEM's Restem-L therapy utilizes umbilical lining modified progenitor cells (UMPCs). The Phase 1/2a trial is a randomized, placebo-controlled study that will enroll 16 patients. The primary endpoint is safety, with secondary outcomes including immune and inflammatory markers, functional outcomes reported by patients and clinicians, and MRI and muscle biopsy biomarkers.

"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," said Andres Isaias, CEO of RESTEM. The company is developing off-the-shelf cell therapies designed to modulate the immune system.

FSHD affects an estimated 1 in 8000 people worldwide. The current focus for managing the condition is on symptom management, slowing disease progression, and improving quality of life.