DMD Research Veers Toward Gene Therapy: 3 Stocks In Focus

Duchenne muscular dystrophy (“DMD”) is a rare genetic disease and a severe form of muscular dystrophy, primarily affecting males. It progressively weakens and degenerates skeletal and heart muscles.

The symptoms of muscle weakness generally start to appear as early as four years of age in boys and progresses quickly.

Duchenne muscular dystrophy is one of the nine types of muscular dystrophy caused by the absence of a particular gene, dystrophin, which the body uses to keep the muscle cells intact. The disease starts to affect the muscles in the hips, pelvic area, thighs and shoulders in early stages and leads to degeneration of voluntary muscles in the arms, legs and trunk gradually. As the disease progresses, it affects the heart and respiratory muscles in early teen patients.

Life expectancy of DMD patients is as low as early adulthood. However, the scenario is changing with advances in cardiac and respiratory care with patients living till their 50s.

Due to the severity and rarity of DMD, the FDA is also incentivizing the companies developing treatments to treat this rare muscular degenerative disease by granting orphan drug designation to several candidates. This designation gives seven years of marketing exclusivity upon approval, research funding eligibility, tax credits for certain research costs, and a waiver of the FDA application user fee.

However, there are very few companies developing DMD treatments due to lower number of patients. Sarepta Therapeutics, Inc.’s (NASDAQ:SRPT) Exondys 51 is the first approved drug in the United States, which has been available since late 2016. The drug is a small molecule, which targets mutation of the DMD gene susceptible to exon 51 skipping. Exondys 51 and PTC Therapeutics, Inc.’s (NASDAQ:PTCT) Emflaza are among the approved treatments of DMD in the United States.

The focus is currently shifting to development of gene therapies for the treatment of the disease. Gene therapies deliver a functional copy of the dystrophin to muscle cells to restore its production.

3 Pharma/Biotech Companies Developing Gene Therapy to Treat DMD

Here we discuss three companies, which are researching and developing advance treatments for DMD.

Sarepta has about eight exon-skipping candidates in its pipeline. Its lead candidate, golodirsen, is an exon-53 skipping candidate, which has demonstrated better efficacy than Exondys 51 with 100% response rate in a mid-stage study. A new drug application (“NDA”) is expected to be filed by the end of this year.

Apart from exon-skipping candidates, Sarepta is focusing on gene therapies for developing treatments for DMD. The company also initiated two phase I/II studies in the fourth quarter of 2017, which will evaluate gene therapies for DMD. Earlier this week, it announced promising preliminary results from an early-stage study evaluating the gene therapy, AAVrh74.MHCK7.micro-dystrophin. (Read more: Sarepta's Stock Soars on Encouraging Gene Therapy Results )

Sarepta currently carries a Zacks Rank #4 (Sell).

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Written By: Zacks Investment Research