Sarepta Therapeutics Announces Topline Results from EMBARK, a Global Pivotal Study of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy

– Results support submission of an efficacy supplement to the BLA; US FDA has indicated openness to reviewing the data for label expansion based on the totality of evidence from EMBARK

– In EMBARK, participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met

– Robust, statistically significant results on all key pre-specified secondary endpoints, including time to rise (p=0.0025), and 10-meter walk test (p=0.0048), demonstrated evidence of a clinically meaningful treatment benefit that was similar in magnitude and statistical significance across all age groups

– No new safety signals were observed

– Sarepta to host investor call on October 30 at 4:30 p.m. Eastern time

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced topline results from EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl) in patients with Duchenne muscular dystrophy between the ages of 4 through 7 years.

“The results of EMBARK, our double-blind, placebo-controlled trial, support the conclusion that ELEVIDYS modifies the trajectory of Duchenne and benefits patients across age groups living with this ferociously degenerative disease. The results favored ELEVIDYS across all endpoints in the study, including achieving statistical significance on all pre-specified key secondary endpoints and in each age subgroup of the key secondary endpoints. Indeed, passing 5 seconds on time to rise is the strongest predictor of early loss of ambulation and in EMBARK, ELEVIDYS reduced those odds over 52 weeks by greater than 90 percent,” said Doug Ingram, president and chief executive officer, Sarepta. “Based on the EMBARK results, we intend to move swiftly to request an update to expand the labeled indication to treat all patients. Importantly, we have shared the EMBARK topline results with FDA leadership and they have confirmed that, based on the totality of the evidence, they are open to such label expansion if supported by review of the data, and that they intend to proceed rapidly with consideration of the submission.”

In the study, ELEVIDYS-treated patients improved 2.6 points on their North Star Ambulatory Assessment (NSAA) total score 52 weeks after treatment compared to 1.9 points in placebo-treated patients. The difference of 0.65-points between treated and placebo groups did not reach statistical significance (n=125; p=0.24).

All key pre-specified functional secondary endpoints demonstrated robust evidence for a clinically meaningful treatment benefit that was consistent across age groups in ELEVIDYS-treated patients compared to placebo at 52 weeks. These include:

*least squared means

All other timed functional endpoints – including stride velocity 95th centile (SV95C) and time to ascend 4 steps – demonstrated consistent treatment benefit in favor of ELEVIDYS. Full results from EMBARK will be shared at future medical meetings and publication will be pursued in a medical journal.

“The strong prognostic power of time to rise, and the particular importance of the 5 second milestone in predicting functional decline and future loss of ambulation, is clearly demonstrated in natural history.1 In EMBARK, the reduction in patients progressing past this milestone when treated with ELEVIDYS is highly clinically relevant,” said Craig McDonald, M.D., professor and chair of the UC Davis Health Department of Physical Medicine and Rehabilitation, and an investigator in the EMBARK study. “The consistency of the positive effect across all timed function tests and age groups provides evidence of a meaningful treatment effect. In addition, it is important to note that this is the first clinical trial in the history of DMD trials to show a statistically significant and meaningful improvement on the novel measure of 95th centile stride velocity derived from an objective community wearable activity monitor.”

There were no new safety signals in the EMBARK study, reinforcing the favorable and manageable safety profile observed with ELEVIDYS to date. The most common treatment-related adverse events were gastrointestinal events (vomiting, nausea, and decreased appetite) and pyrexia. Seven participants (11.1%) experienced a treatment-related serious adverse event (SAE) and there were no clinically meaningful changes observed in SAEs associated with known risks of ELEVIDYS.

Conference call details

On October 30, 2023, at 4:30 p.m. Eastern time, Sarepta will host a conference call and webcast to discuss these 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.

As part of a collaboration agreement signed in 2019, Roche is working with Sarepta Therapeutics to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function, keeping them stronger for longer. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Together, the companies are implementing a comprehensive joint clinical development plan to maximize the chances of broad approval and access so that ELEVIDYS can reach as many individuals with Duchenne as rapidly as possible.

About EMBARK, Study 9001-301

Study SRP-9001-301, also known as EMBARK, is a multinational, phase 3, randomized, two-part crossover, placebo-controlled study of ELEVIDYS in individuals with Duchenne muscular dystrophy between the ages of 4 to 7 years. The primary endpoint is change from baseline in NSAA Total Score at Week 52 following treatment. Eligible participants received a single dose of ELEVIDYS during either Part 1 or Part 2 of the study.

In Part 1, participants (n=125) were randomized according to age (≥4 to 16 to