December 18, 2024

 

Dear U.S. Limb-girdle community,

As 2024 comes to a close, we want to take an opportunity to share some brief updates related to Sarepta’s clinical programs that are focused on LGMD 2E/R4, LGMD 2D/R3, and LGMD 2C/R5, or what are collectively called sarcoglycanopathies.  

Importantly, 2024 has marked important progress in our ability to leverage available tools provided by US regulators to seek the fastest and most responsible pathway to make our LGMD therapies available in the US. This includes the use of innovative trial designs that are informed by natural history and the use of surrogate endpoints such as expression of the missing protein. We are maximizing the synergies across this platform from both an R&D and manufacturing perspective. We know this tangible progress is not due to science alone. Your milestone engagement efforts, like the FDA Patient Listening session, the Externally-led Patient-Focused Drug Development meeting, and, most recently, this year’s scientific workshop have helped inform FDA and our path. Together, we will continue to advance therapies at the frontier of LGMD, in 2E, and across the sarcoglycanopathies and additional subtypes. We plan to maximize use of the accelerated approval pathway for sarcoglycanopathies to help address the unmet need as quickly as possible. We will work with our collaborators outside the US, seeking the community’s input and generating data that may meet the varying requirements of global regulators.

  • LGMD 2E/R4: Earlier today, Sarepta announced that enrollment and dosing is complete in EMERGENE (Study SRP-9003-301), a Phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec). SRP-9003 is an investigational gene therapy for the treatment of limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E/R4), or beta-sarcoglycanopathy. Data from EMERGENE are expected in the first half of 2025. Assuming a positive pre-Biologics License Application (BLA) meeting and supportive data from EMERGENE, Sarepta anticipates submitting a BLA in 2025 to the U.S. Food and Drug Administration seeking accelerated approval for SRP-9003.
  • LGMD 2D/R3: Study SRP-9004-102 (DISCOVERY) for LGMD 2D/R3, or alpha-sarcoglycanopathy, is initiating in December of 2024. DISCOVERY is a small US study in which we will evaluate an investigational gene therapy delivered through an intravenous line. It is a phase 1, proof-of-concept study evaluating safety and expression of the alpha-sarcoglycan protein after treatment. The study’s status on clinicaltrials.gov will be listed as “enrolling by invitation”, which means that clinical trial sites will enroll the study from their own clinic populations.  
  • LGMD 2C/R5: Study SRP-9005-101, Sarepta’s first human study of an investigational gene therapy designed for people living with LGMD2C/R5, is expected to initiate in early 2025. Sarepta looks forward to sharing further updates about the program when they become available.
  • JOURNEY: The JOURNEY natural history study continues to collect incredibly important data that will help us understand different forms of LGMD, especially as new therapies and studies are developed. We would like to thank those who continue to participate in the JOURNEY natural history study for their incredible contribution to science and to the growing body of information about LGMD.  The JOURNEY Natural History is currently enrolling a limited number of patients for the sarcoglycanopathy cohorts (LGMD 2C, 2D, and 2E). More information about the JOURNEY study may be found on clinicaltrials.gov (NCT04475926).
    *[Though this community letter is specifically about the sarcoglycanopathy clinical programs, we would like to clarify that the JOURNEY study continues to enroll people living with LGMD2A/R1, or calpainopathy, at multiple sites globally. More information may be found on clinicaltrials.gov (NCT04475926).

Our programs are on track, and this would not be possible without the engagement, energy and interest of the LGMD community members who are eager to enter a natural history study and/or an investigational gene therapy study.  We recognize our studies unfortunately are not able to reach every individual or family that wishes to participate. But in reaching these important milestones, we are encouraged and hopeful that continued rapid progress of the studies underway will lead to faster and broader access to potentially approved therapies in the future.  

We look forward to sharing more information, broader program updates, and details about studies in our LGMD pipeline in 2025, including at the International LGMD conference in Orlando, FL, in July. Meanwhile, if you have questions about Sarepta’s programs you may reach out to [email protected].

 

Wishing you warm and happy holidays,

 

The Patient Affairs Team