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Profile: Sarepta Therapeutics Inc (SRPT.OQ)

SRPT.OQ on NASDAQ Stock Exchange Global Select Market

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Sarepta Therapeutics, Inc., incorporated on June 5, 2013, is a biopharmaceutical company. The Company focuses on the discovery and development of ribose nucleic acid (RNA)-targeted therapeutics for the treatment of rare neuromuscular diseases. The Company operates through discovering, developing, manufacturing and delivering therapies to patients with Duchenne muscular dystrophy (DMD). The Company is primarily focused on the development of its disease-modifying DMD drug candidates.

The Company has received accelerated approval for its product, EXONDYS 51, indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. EXONDYS 51 is studied in clinical trials under the name of eteplirsen. The Company is in the process of planning several studies for eteplirsen and product candidates targeting skipping of exon 53 and/or exon 45. It is also conducting an open label extension of the Phase IIb study for which patients can transition to commercial drug after certain criteria are met (Study 4658-us-202), an open label study on ambulatory patients with a concurrent untreated control arm (Study 4658-301/PROMOVI), a study evaluating the safety and tolerability of eteplirsen in participants with advanced stage DMD (Study 4658-204) and a study evaluating the safety and tolerability of eteplirsen in participants with early stage DMD (Study 4658-203), each of which will allow for patients to transition to commercial drug after meeting certain criteria. The Company is also planning two additional Phase I studies.

The Company is enrolling and dosing patients in ESSENCE (Study 4045-301), its Phase III placebo controlled confirmatory trial in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 or 53 skipping using SRP-4045 and SRP-4053, respectively. SRP-4053, an exon 53-skipping product candidate that it has selected for development through the SKIP-NMD consortium, is in the clinic in European Union (EU) as part of a Phase I/II study. Part I has been completed and Part II, an open label portion of this study, is ongoing (Study 4053-101). It is also enrolling and dosing patients amenable to exon 45 skipping in ESSENCE, its placebo controlled study for eteplirsen. SRP-4045, an exon 45-skipping product candidate that it selected for development in collaboration with Children's National Medical Center (CNMC) in Washington, D.C. and the Carolinas Medical Center (CMC) in Charlotte, N.C. In addition to EXONDYS 51, the Company's DMD pipeline includes other product candidates, which are at various stages of development.

The Company's next generation phosphorodiamidate morpholino oligomer (PMO)-based compounds are synthetic compounds that bind to complementary sequences of RNA by standard Watson-Crick nucleobase pairing. When targeted to messenger RNA (mRNA), PMO-based compounds down-regulate protein translation by steric blockade. Its PMO-based chemistries include peptide conjugated PMO (PPMO), PMO-X and PMOplus. PPMO technology features covalent attachment of a cell-penetrating peptide to a PMO with the goal of cellular delivery into the cytosol and the nucleus. PMOplus features the selective introduction of positive charges to the PMO backbone and it has potentially therapeutic applications, especially for anti-viral therapeutics. PMO-X incorporates chemical modifications to the PMO backbone linkages.

Company Address

Sarepta Therapeutics Inc

215 1st St Ste 415
CAMBRIDGE   MA   02142-1213
P: +1617.2744000
F: +1302.6365454

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