Sarepta Looks To Focus On Exondys 51 & DMD Pipeline In 2017

We issued an updated research report on Cambridge, MA-based Sarepta Therapeutics, Inc. (NASDAQ:SRPT) on Jun 28.

Sarepta is a commercial-stage biopharmaceutical company that focuses on the discovery and development of RNA-based therapeutics targeting rare and infectious diseases, especially Duchenne muscular dystrophy (DMD).

So far this year, Sarepta’s share price has outperformed the Zacks categorized Medical-Biomed/Genetics industry. The stock has surged 21.6% compared with an increase of 10.0% for the broader industry.

Sarepta received a boost with the FDA’s accelerated approval for Exondys 51 (eteplirsen) in the U.S. in Sep 2016. Exondys 51 is the first approved disease-modifying therapy for DMD in the U.S. as well as the company’s first product to receive marketing approval. The drug is under review in the EU.

The company is conducting a confirmatory phase III study - PROMOVI - evaluating the clinical benefits of the drug as part of the accelerated approval process. The study evaluates whether Exondys 51 improves motor function in DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.

In the first quarter, Sarepta recorded revenues of $16.3 million, up 201% sequentially, primarily due to sales of Exondys 51. Also, the company recognized a narrower per share loss of 70 cents from the year-ago loss of $1.31. However, selling, general & administrative (SG&A) expenses have increased due to higher legal fees and commercial initiatives, compensation and other personnel expenses related to commercialization of Exondys 51.

Moreover, based on sales trends witnessed in the first quarter, the company expects Exondys 51 to generate $95 million in revenues in 2017, exceeding its prior guidance of $80 million.

Meanwhile, Sarepta is focused on building its DMD pipeline beyond Exondys 51. The company has eight exon-skipping candidates in its pipeline that is estimated to treat 75%–80% of the DMD population. The company is also evaluating candidates targeting exon 53 (Study 4053-101—6 to15 years, ambulatory; data expected this year) and exon 45/53 (Study 4045-301—7 to16 years, ambulatory). In addition, Sarepta is conducting Study 4045-101 in non-ambulatory boys aged 7 to 21 years amenable to skipping-exon-45.

Meanwhile, after the approval of its DMD drug, Sarepta collaborated with Catabasis Pharmaceuticals in 2016 to conduct a joint research to explore a combination-drug treatment approach for DMD.

Apart from DMD, the company develops treatments for infectious diseases including Marburg virus, Ebola virus and influenza, which is primarily funded and supported by the National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID). Along with it, Sarepta has an exclusive license and collaboration agreement with Summit Therapeutics plc (NASDAQ:SMMT) for the rights to the latter’s utrophin modulator pipeline.

However, Pfizer Inc. (NYSE:PFE) , BioMarin, PTC Therapeutics, Inc. (NASDAQ:PTCT) and many more are developing RNA-based treatments for DMD. With small and major companies developing their own pipeline, the DMD market is set to become competitive in the future.

Zacks Rank

Sarepta currently holds Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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