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

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 in 15 ambulatory and non-ambulatory participants, ages 4 and older. The EMERGENE design incorporates a six-month natural history lead-in. The primary endpoint is expression of beta-sarcoglycan 60 days after dosing. Other endpoints include functional measures through month 60 and safety.

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. Sarepta’s six LGMD gene therapy programs in development include LGMD2E/R4, LGMD2D, LGMD2C, LGMD2B, LGMD2L and LGMD2A, which together represent more than 70 percent of known LGMD cases.

Patients with LGMD2E (beta-sarcoglycanopathy) 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, progresses to loss of ambulation in the teen years and often leads to early mortality. There are currently no disease modifying treatments for LGMD2E.

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 Twitter, LinkedIn, Instagram and Facebook.

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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; and the potential benefits of SRP-9003.