Monte Rosa Therapeutics advances MRT-6160, MRT-2359, and MRT-8102 programs.

Monte Rosa Therapeutics is advancing multiple programs toward Phase 2 studies, including MRT-6160 for immune-mediated diseases and MRT-2359 for castration-resistant prostate cancer. MRT-8102, targeting IL-1β and the NLRP3 inflammasome, is on track for IND filing in H1 2025. Cyclin E1 and CDK2-directed MGD programs are advancing toward the clinic, with IND submission anticipated in 2026. The company has a strong cash position expected to fund operations into 2028.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) has announced positive topline results from Part 2 of the EMBARK study, a global, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy (DMD). The study, Study SRP-9001-301, demonstrated sustained benefits and disease stabilization in ambulatory individuals with DMD following treatment with ELEVIDYS.

Key findings from the study include:

- Crossover-treated patients, who received a placebo in Part 1 and crossed over at 52 weeks to receive ELEVIDYS in Part 2, showed statistically significant improvements in the North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10-meter walk/run (10MWR) compared to a pre-specified, well-matched external control (EC) group. The improvements were 2.34 points on the NSAA, -2.70 seconds on the TTR, and -1.07 seconds on the 10MWR, all with P-values 0.0001.
- Patients treated in Part 1 of the EMBARK study showed sustained expression of ELEVIDYS micro-dystrophin at week 64, and functional improvements on the NSAA, TTR, and 10MWR were sustained two years after treatment. The least square means (LSM) differences between the ELEVIDYS-treated patients and the EC group increased from year one to year two for all three functional outcomes, indicating a continued divergence from the natural history of DMD.
- Skeletal muscle MRI conducted on patients treated in Part 1 showed minimal progression in underlying muscle pathology, aligning closely with the observed functional benefits.

Louise Rodino-Klapac, Ph.D., executive vice president, Head of R&D, and Chief Scientific Officer at Sarepta, commented, "We’re very encouraged to see the results from Part 2 of EMBARK as they further elucidate the impact ELEVIDYS has on disease progression in a blinded, controlled study. Skeletal muscle MRI demonstrates the importance of preserving muscle, and the functional outcome results show disease stabilization sustained through two years after treatment."

The company will host an investor call and webcast today at 8:30 a.m. ET to discuss these results in more detail. Detailed results from Part 2 of the EMBARK study will be shared at future medical meetings.

References:
[1] https://www.businesswire.com/news/home/20250126244525/en/Sarepta-Therapeutics-Announces-Results-from-Part-2-of-the-EMBARK-Study-Demonstrating-Sustained-Benefits-and-Disease-Stabilization-in-Ambulatory-Individuals-with-Duchenne-Muscular-Dystrophy-Following-Treatment-with-ELEVIDYS