Sarepta Therapeutics Announces Positive Data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51 - Seite 2

• 2.81% mean dystrophin expression as measured by western blot
• 1.60% mean change from baseline, P= 0.0012
• Mean exon skipping of 2.47%, as measured by ddPCR, and a mean change from baseline in exon skipping of 2.00%
• The changes from baseline represent a 4.3-fold increase in dystrophin expression and a 4.9-fold increase in exon skipping compared to a weekly 30 mg/kg dose of eteplirsen at 24 weeks.

“The dystrophin production delivered by SRP-5051 in the MOMENTUM study is very encouraging. Importantly, with effective supplementation and monitoring, we have not seen additional complications from the hypomagnesemia,” said Eugenio Mercuri, M.D., Ph.D., head of the Neuromuscular Unit, Catholic University, Rome, Italy, and an investigator in the study. “The results with SRP-5051 to date suggest it could play an important role in the treatment of Duchenne patients with a confirmed exon 51-amenable mutation.”

There were seven serious, treatment-emergent adverse events in MOMENTUM Part B, four serious events of hypomagnesemia and three serious cases of hypokalemia. Hypomagnesemia has been seen in earlier clinical studies of SRP-5051 and, throughout MOMENTUM Part B, supplemental magnesium was administered prophylactically. No treatment-related discontinuations occurred in the study.

Conference call details

At 8:30 a.m. Eastern time on Jan. 29, 2024, Sarepta will host a conference call and webcast to discuss the results.

The event will be webcast live under the investor relations section of Sarepta’s website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

About SRP-5051 (vesleteplirsen)

SRP-5051 is an investigational agent using Sarepta’s PPMO chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally shortened dystrophin protein. PPMO is Sarepta’s next-generation chemistry platform designed around a proprietary cell-penetrating peptide conjugated to the PMO backbone, with the goal of increasing tissue penetration, increasing exon skipping, and significantly increasing dystrophin production. Around 13% of Duchenne patients have mutations that make them amenable to skipping exon 51. If successful, the PPMO offers the potential for improved efficacy and less frequent dosing for patients.