Sarepta Therapeutics Inc’s market leading gene therapy for Duchenne muscular dystrophy moves ahead as Pfizer’s results are not compelling and include safety concerns.

Bloomberg reports that RBC analyst Brian Abrahams advised clients to buy Sarepta shares as the negative Pfizer data help the former position against the latter. Baird’s Brian Skorney noted “Pfizer fell way short on both safety and efficacy” and emphasized the positive for Sarepta’s safety.

Current Financial Situation

Sarepta is presently priced by the market at $129.76 per share. Its 52 week high low is $165.87 and $95.21. With revenues of just around $323.44 million they have a staggering market capitalization of $11.27 billion and a 5 year expected PEG ratio of 143.20. The company will lose about $403 million but maintains just about $1.4 billion cash in the bank so they have some cushion.


Founded in 1980 and based in Cambridge, MA they focus on the discovery and development of RNA-based therapeutics, gene therapy and other genetic medicine approaches for the treatment of rare diseases. The company offers EXONDYS 51, a disease-modifying therapy for Duchenne muscular dystrophy (DMD). Its products pipeline includes Golodirsen, a product candidate that binds to exon 53 of dystrophin pre-mRNA, which results in exclusion or skipping of exon during mRNA processing in patients with genetic mutations, and Casimersen, a product candidate that uses phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the DMD gene.

Additionally, the company’s pipeline comprises SRP-5051, a peptide conjugated PMO that binds to exon 51 of dystrophin pre-mRNA. It has collaboration agreements with Nationwide Children’s Hospital (TrialSite News has written that Nationwide is a leader in gene therapy) to advance a micro-dystrophin gene therapy program under the research and license option agreement; Galgt2, a gene therapy program for the treatment of DMD; and Neutrophin 3, a gene therapy program to treat Charcot-Marie-Tooth neuropathies.

The company also has a license agreement with Lysogene to develop LYS-SAF302, a gene therapy program to treat Charcot-Marie-Tooth neuropathies. They are also leveraging LYS-SAF302 for mucopolysaccharidosis IIIA; a license option and agreement with Lacerta to development treatments for CNS-targeted and lysosomal storage diseases; and research collaboration and option agreement with Genethon to develop micro-dystrophin gene therapy products.  The company inked a R&D agreement with Duke University to advance gene editing CRISPR/Cas9 technology for restoring dystrophin expression, a collaboration agreement with Summit (Oxford) Ltd. to commercialize products in Summit’s utrophin modulator pipeline, a strategic collaboration with Paragon Bioservices, and a strategic collaboration with CENTOGENE for the identification of patients with DMD in the Middle East and North Africa region. Sarepta Therapeutics, Inc. was founded in 1980 and is headquartered in Cambridge, Massachusetts.

Source: Bloomberg

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