Sarepta Finishes Enrollment in Phase 3 Study of SRP-9003 for LGMD Type 2E/R4

Sarepta Therapeutics, a leader in the field of precision genetic medicine for rare diseases, has announced a significant advancement in their ongoing efforts to develop a gene therapy for limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E/R4), also known as beta-sarcoglycanopathy. The company has completed enrollment and dosing for their global Phase 3 clinical trial, known as EMERGENE (Study SRP-9003-301), investigating the efficacy and safety of SRP-9003 (bidridistrogene xeboparvovec). This gene therapy aims to address the root cause of LGMD2E/R4, which is the absence of beta-sarcoglycan protein.

SRP-9003 utilizes the AAVrh74 vector to robustly deliver a full-length beta-sarcoglycan transgene to skeletal, diaphragm, and cardiac muscles. By employing the MHCK7 promoter, it ensures strong gene expression in the heart, which is vital for patients suffering from LGMD2E/R4 due to the potential for pulmonary or cardiac complications.

The completion of the EMERGENE study’s enrollment marks a crucial step towards providing a potentially transformative treatment for individuals with this ultra-rare disease, which currently lacks treatment options beyond managing symptoms. Dr. Louise Rodino-Klapac, Sarepta’s Executive Vice President and Chief Scientific Officer, emphasized the significance of this milestone. She highlighted that the EMERGENE study's design informs the pathways for other treatments in Sarepta's LGMD pipeline, with plans to initiate trials for LGMD2D and LGMD2C in the near future.

The primary goal of the EMERGENE trial is to assess the expression of beta-sarcoglycan protein 60 days post-treatment. Additionally, secondary objectives will evaluate functional improvements and safety over a period of 60 months. Results from the study are anticipated in the first half of 2025. Should these results be favorable, Sarepta plans to file for accelerated approval of SRP-9003 with the U.S. Food and Drug Administration within the same year.

Limb-girdle muscular dystrophies, including LGMD2E/R4, are genetic disorders characterized by progressive muscle weakness and wasting, initially affecting muscles around the hips and shoulders before advancing to the limbs. Symptoms typically appear before age 10, with patients experiencing difficulties in activities such as running and climbing stairs. This autosomal recessive condition often leads to a loss of mobility during the teenage years and an increased risk of early mortality.

Sarepta Therapeutics is dedicated to advancing precision genetic medicine to tackle rare diseases that severely impact lives. The company holds a leading position in developing treatments for Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs). With over 40 programs in various stages of development, Sarepta is harnessing its Precision Genetic Medicine Engine, focusing on gene therapy, RNA, and gene editing technologies to address unmet medical needs.