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Sarepta (SRPT) DMD Study Shows Proof of Concept for SRP-5051

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Sarepta Therapeutics, Inc. (SRPT - Free Report) announced data from the ongoing multiple ascending dose phase II study — MOMENTUM — evaluating its next-generation candidate, SRP-5051, for the treatment of Duchenne muscular dystrophy (DMD) by skipping exon 51. Data from the study showed that the candidate achieved proof-of-concept and supported continued dose escalation. This is the first clinical data from the company’s next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) platform. Data suggests that this platform will likely lead to improved efficacy and lower dosing compared to its PMO platform. The candidate was well tolerated in the study patient population.

The company’s PMO platform has been used to develop its two marketed drugs — Exondys 51 and Vyondys 53 — and lead pipeline candidate, casimersen, which is currently under review with the FDA. A decision is expected by Feb 25, 2021.

The company reported data from the group that received a monthly-dose of 20 mg/kg of SRP-5051 in the study. Data showed that the patients achieved higher tissue exposure, exon-skipping and dystrophin production at 12-weeks from baseline. Sarepta stated that while comparing MOMENTUM study data with data from previously completed PROMOVI study on Exondys 51, SRP-5051 showed improved efficacy with more convenient or lower dosing. SRP-5051 achieved 1.6-fold increase in exon skipping and a five-fold increase in the percentage of normal dystrophin compared to Exondys 51 at 24 weeks. Moreover, the PROMOVI study evaluated a weekly 30mg/kg of Exondys 51, significantly higher compared to SRP-5051 dose.

Sarepta observed that exon-skipping and dystrophin increases over time based on its experience with PMO-based drugs, suggesting further increase in efficacy for SRP-5051 with the passage of time. Based on these facts along with pre-clinical data, the company intends to continue to escalate doses of SRP-5051 and advance its PPMO exon-skipping pipeline for DMD that includes five other potential therapies.

So far this year, the company’s shares have increased 17% compared with the industry‘s rise of 5.7%.

Sarepta’s Exondys has seen steady growth in sales since its launch, reflecting potential in the DMD segment. SRP-5051, which is also exon-51 skipping candidate, seems to have encouraging potential following its successful launch.

Apart from its exon-skipping candidates, the company is developing several gene therapies targeting muscular dystrophies including DMD and central nervous system disorders. Its lead gene therapy candidate, SRP-9001, an AAV-mediated micro-dystrophin gene therapy candidate, is currently being evaluated in a phase I/II study for treating DMD. The promising candidate also led Roche (RHHBY - Free Report) to sign a collaboration deal with Sarepta related to commercialization of SRP-9001 in ex-U.S. markets.

However, we note that there are there are several other companies developing gene therapies for treating DMD including Solid Biosciences (SLDB - Free Report) , Audentes Therapeutics, and Pfizer (PFE - Free Report) . Successful development of gene therapies for DMD will increase competition in the field. Gene therapies of Solid Biosciences and Pfizer seemed to trail Sarepta’s gene therapy for DMD, based on early-stage study data.

Zacks Rank

Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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