Part B of MOMENTUM study of SRP-5051 in patients with DMD

MOMENTUM study of SRP-5051

Sarepta Therapeutics to initiate Part B of MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) amenable to Exon 51 skipping following positive interactions with FDA

Original press release CAMBRIDGE, Mass., Sept. 27, 2021 (GLOBE NEWSWIRE)

  • Company Anticipates Part B of MOMENTUM to Serve as Pivotal Study for SRP-5051 and to Seek Accelerated Approval if Successful
  • Ambulatory and Non-Ambulatory Patients Between the Ages of 7 to 21 Will Be Eligible to Enroll in Part B of MOMENTUM

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced that following positive interactions with the U.S. Food and Drug Administration (FDA), the Company plans to initiate Part B of the MOMENTUM study (Study 5051-201), in the fourth quarter. MOMENTUM is a global trial investigating the use of SRP-5051, the Company’s next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), to treat patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

The study will enroll 20-40 patients between ages 7 to 21 amenable to exon 51 skipping who are naïve to SRP-5051. Additionally, those previously dosed in Study 5051-201, Part A or Study 5051-102 who meet the entrance criteria will be eligible to participate. Both ambulatory and non-ambulatory patients are eligible for participation. The Company will submit the protocol in the next week.


Doug Ingram, Sarepta’s president and chief executive officer – “If proven safe and as efficacious as our initial data suggest it may be, SRP-5051 could be the first in a host of transformative therapies from our next-generation PPMO platform to treat and improve the lives of children living with Duchenne muscular dystrophy. In addition to the fantastic work of our team, and the dedication of Duchenne families and the program’s investigators, I would like to thank FDA’s Division of Neurology I for their diligence and thoughtful advice and input, without which we would not be able to commence Part B of the MOMEMTUM study ahead of schedule.”



About MOMENTUM (Study SRP-5051-201)

MOMENTUM is a multi-arm, ascending dose study of SRP-5051, infused monthly and will assess dystrophin protein level in skeletal muscle tissue following SRP-5051 treatment. The study will enroll both ambulant and non-ambulant patients between the ages of 7 to 21 at sites in the U.S., Canada, Australia, and the European Union. The study will also assess safety and tolerability.

In May of this year, the Company announced results from Part A of the MOMENTUM study showing that after 12 weeks, 30 mg/kg of SRP-5051 dosed monthly resulted in 18 times the exon skipping and eight times the dystrophin production as eteplirsen, dosed weekly for 24 weeks.

Reversible hypomagnesemia was identified in patients taking SRP-5051. The protocol for Part B of MOMENTUM will include magnesium supplementation and monitoring of magnesium levels.


About SRP-5051

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 excluding this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for the production of an internally truncated dystrophin protein. PPMO is Sarepta’s next-generation chemistry platform designed around a proprietary cell-penetrating peptide conjugated to the PMO backbone, intending to increase tissue penetration, increasing exon skipping and significantly increasing dystrophin production. Around 13% of DMD 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.

About Duchenne Muscular Dystrophy (DMD)

DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 – 5,000 male births worldwide. DMD is a devastating and incurable muscle-wasting disease associated with specific gene errors that code for dystrophin. This protein plays a critical structural role in muscle fibre function. Progressive muscle weakness in the lower limbs spreads to the arms, neck, and other body areas. The condition is universally fatal, and death usually occurs before 30 due to respiratory or cardiac failure.

About EXONDYS 51

EXONDYS 51 (eteplirsen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in “skipping” 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 the production of an internally truncated dystrophin protein.

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

This indication is approved under accelerated approval based on increased dystrophin production in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval may be contingent upon verification of a clinical benefit in confirmatory trials.

EXONDYS 51 has met the full statutory standards for safety and effectiveness and, as such, is not considered investigational or experimental.




Source: Sarepta Therapeutics, Inc.

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