Sarepta Therapeutics Completes Enrollment in EMERGENE, a Phase 3 Clinical Study of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4

Rhea-AI Summary

Sarepta Therapeutics (NASDAQ:SRPT) has completed enrollment and dosing in EMERGENE (Study SRP-9003-301), a Phase 3 clinical trial of SRP-9003 for treating limb-girdle muscular dystrophy Type 2E/R4. The global study's primary endpoint focuses on beta-sarcoglycan protein expression, with data expected in the first half of 2025.

The company plans to file for accelerated approval of SRP-9003 in 2025, following a positive pre-Biologics License Application meeting. This development serves as a precedent for Sarepta's other LGMD pipeline programs, including LGMD2D and LGMD2C programs, scheduled to initiate in Q1 2025.

Positive

• Completed enrollment in Phase 3 EMERGENE trial ahead of anticipated 2025 approval filing
• Study design serves as template for company's broader LGMD pipeline programs
• Potential first-mover advantage in untreated LGMD2E/R4 market

Negative

• No treatment efficacy data available yet from Phase 3 trial
• Approval timeline extends to 2025
• Success depends on positive pre-BLA meeting and supportive trial data

Insights

Medical Research Analyst

The completion of enrollment in EMERGENE represents a significant milestone for Sarepta's gene therapy program targeting LGMD2E/R4. The study's design focusing on beta-sarcoglycan protein expression as the primary endpoint is strategically sound, as this biomarker directly addresses the underlying cause of the disease. The anticipated BLA submission in 2025 positions SRP-9003 as a potential first-in-class treatment for this ultra-rare condition.

The trial's design sets a precedent for Sarepta's broader LGMD pipeline, particularly for their LGMD2D and LGMD2C programs. The use of the AAVrh74 vector with MHCK7 promoter demonstrates sophisticated engineering, targeting not just skeletal muscles but also critical cardiac tissue - a important feature for addressing the cardiopulmonary complications common in LGMD2E/R4 patients.

Market Research Analyst

This development strengthens Sarepta's position in the rare disease space, particularly in muscular dystrophies. With a market cap of $12 billion, successful commercialization of SRP-9003 could open up a new revenue stream in the ultra-rare disease market. The company's expanding LGMD pipeline, including LGMD2D and LGMD2C programs, suggests a strategic focus on building a comprehensive rare disease portfolio. The study's completion also validates Sarepta's gene therapy platform, potentially increasing investor confidence in their broader development pipeline of over 40 programs.

– Sarepta anticipates filing for accelerated approval of SRP-9003 in 2025

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that enrollment and dosing is complete in EMERGENE (Study SRP-9003-301), a Phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec). SRP-9003 is an investigational gene therapy for the treatment of limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E/R4), or beta-sarcoglycanopathy. EMERGENE is a global study, and the primary endpoint is the biomarker expression of beta-sarcoglycan protein, the absence of which is the sole cause of LGMD2E/R4.

“The completion of enrollment in the EMERGENE study marks a significant milestone to bring a potentially disease-modifying treatment to individuals living with LGMD2E, an ultra-rare form of LGMD with no treatments beyond symptom management. We are committed to securing approval of SRP-9003 as quickly as possible and are now closer to reaching that goal for patients and their families,” said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics. “The design of EMERGENE is an important precedent that informs development plans for Sarepta’s other LGMD pipeline programs, including our LGMD2D program which we just initiated and our LGMD2C program which we expect to initiate in the first quarter of 2025, and serves as a pathfinder for heterogenous neuromuscular gene therapies more broadly.”

Data from EMERGENE are expected in the first half of 2025. Assuming a positive pre-Biologics License Application (BLA) meeting and supportive data from EMERGENE, Sarepta anticipates submitting a BLA to the U.S. Food and Drug Administration seeking accelerated approval for SRP-9003 in 2025.

About Study SRP-9003-301 (EMERGENE)

EMERGENE, Study 9003-301 is a Phase 3, multinational, open-label study of SRP-9003 for the treatment of LGMD2E/R4 in ambulatory and non-ambulatory participants, ages 4 and older. EMERGENE’s primary endpoint is expression of beta-sarcoglycan 60 days after dosing. Secondary outcomes and endpoints include functional measures through month 60 and safety.

About SRP-9003 (bidridistrogene xeboparvovec)

SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy that uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases. SRP-9003 is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart^1,2,3 which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

About Limb-girdle Muscular Dystrophy

Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with LGMD Type 2E/R4 (beta-sarcoglycanopathy) typically begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, frequently progresses to loss of ambulation in the teen years and often leads to early mortality.

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on LinkedIn, X (formerly Twitter), Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, technologies and scientific approaches, business plans, priorities, research and development programs, the potential benefits of SRP-9003, including SRP-9003 serving as a pathfinder for certain gene therapy more broadly, and expected plans and milestones, including data from EMERGENE in the first half of 2025 and potentially submitting a BLA in 2025, and initiating our LGMD2C program in the first quarter of 2025.

Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: our data may not be sufficient for obtaining regulatory approval; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons, including, among others, denial by the regulatory agencies of permission to proceed with our clinical trials, or placement of a clinical trial on hold, challenges in identifying, recruiting, enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials; different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, many of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2023, and Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.

Source: Sarepta Therapeutics, Inc.

References:

1. Pozsgai ER, et al. Systemic AAV-Mediated b-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice. Mol. Ther. 2017 Apr 5;25(4):855-869.
2. Mendell JR, et al. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;77(9):1-10.
3. Salva MZ, et al. Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol Ther. 2007;15(2):320-329.

 

View source version on businesswire.com: https://www.businesswire.com/news/home/20241218625378/en/

Investor Contact:

Ian Estepan, 617-274-4052

iestepan@sarepta.com

Media Contact:

Tracy Sorrentino, 617-301-8566

tsorrentino@sarepta.com

Source: Sarepta Therapeutics, Inc.

FAQ

When will Sarepta (SRPT) release Phase 3 EMERGENE trial data for SRP-9003?

Sarepta expects to release data from the Phase 3 EMERGENE trial in the first half of 2025.

What is the primary endpoint of Sarepta's EMERGENE Phase 3 trial for SRPT?

The primary endpoint is the biomarker expression of beta-sarcoglycan protein, measured 60 days after dosing.

When does Sarepta (SRPT) plan to file for SRP-9003 accelerated approval?

Sarepta plans to submit a BLA seeking accelerated approval for SRP-9003 in 2025, pending positive pre-BLA meeting and supportive trial data.

What age group is eligible for Sarepta's EMERGENE trial of SRP-9003?

The EMERGENE trial includes both ambulatory and non-ambulatory participants ages 4 and older.

How long will Sarepta's EMERGENE trial track secondary outcomes for SRPT?

The trial will track functional measures and safety as secondary outcomes through month 60 (5 years).