Sarepta Therapeutics, Inc. to Initiate Part B of Momentum Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Following Positive Interactions with FDA
September 27, 2021 at 08:35 am EDT
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Sarepta Therapeutics, Inc. 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 between 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. 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 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.
Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company. The Company is focused on helping patients through the discovery and development of ribonucleic acid (RNA)-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. It has developed multiple approved products for the treatment of Duchenne muscular dystrophy (Duchenne) and is developing potential therapeutic candidates for a range of diseases and disorders, including Duchenne, Limb-girdle muscular dystrophies (LGMDs) and other neuromuscular and central nervous system (CNS) related disorders. It has developed and commercialized four approved products for the treatment of Duchenne: EXONDYS 51 (eteplirsen), Injection (EXONDYS 51), VYONDYS 53 (golodirsen) Injection (VYONDYS 53), AMONDYS 45 (casimersen) Injection (AMONDYS 45), and ELEVIDYS. Its pipeline includes approximately 40 programs at various stages of discovery, pre-clinical and clinical development.
Sarepta Therapeutics, Inc. to Initiate Part B of Momentum Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Following Positive Interactions with FDA