In Race to Treat Muscle-Wasting Disease, Sarepta Seeks Purchases

Sarepta Therapeutics Inc. is looking to buy another drug in an escalating battle with bigger rival BioMarin Pharmaceutical Inc. to treat Duchenne muscular dystrophy, the rare and deadly muscle-wasting disease.

The two companies are in a race to bring the products to market, and buying a drug could let Sarepta eventually combine therapies into a more effective cocktail. BioMarin submitted its drug to the U.S. Food and Drug Administration in April, beating Cambridge, Massachusetts-based Sarepta by a matter of weeks.

With the two drugmakers racing neck-and-neck at the FDA, Sarepta’s interim Chief Executive Officer Edward Kaye said the company’s next move will be to buy another therapy, potentially to use in a cocktail of treatments for the muscle-wasting disease, which occurs in about one of 3,500 boys born worldwide, according to the U.S. National Institutes of Health.

“We are going to own DMD,” Kaye said in an interview. The company, he said, wants “a menu of options” to offer patients, and is looking for an acquisition within a year.

When Kaye goes shopping, he may run into BioMarin, which is willing to do deals of its own. BioMarin Chief Medical Officer Henry Fuchs says that his company, not Sarepta, will lead treatment of the disease.

“With the Duchenne muscular dystrophy space you betcha we’re going to be creating and owning franchise spaces,” Fuchs said in a phone interview. BioMarin, he said, has the resources “that are necessary to actually deliver important medical advances to patients -- as opposed to just talking about that.”

BioMarin has a bigger war chest to make acquisitions, with $1.01 billion in cash, compared with Sarepta’s $166 million. Sarepta has a small amount of debt -- $6.4 million -- while BioMarin owes $656 million.

Sarepta’s shares have gained 41 percent in the last year, while BioMarin’s have more than doubled.

Bad Blood

Adding to the rivalry is a history of bad blood between the companies that bubbled over at a San Francisco investor meeting in January, when BioMarin CEO Jean-Jacques Bienaime said its DMD drug’s data was superior to Sarepta’s. Sarepta’s then-CEO Chris Garabedian shot back at the time that the comments were “inappropriate” and criticized Bienaime for blending data from two different studies.

Behind the war of words is a deadly and tragic disease. A genetic disorder, Duchenne causes muscle degeneration starting at an early age, deforming bones and causing the heart to swell. Few patients live past their 30s.

The two companies are among many pursuing treatments for DMD, for which there are currently no approved drugs to halt or reverse the muscle damage. Among the firms are PTC Therapeutics Inc., Pfizer Inc., Summit Therapeutics Plc, Santhera Pharmaceutical Holding AG, Catabasis Pharmaceuticals Inc., Capricor Therapeutics Inc. and Nicox SA.

Drug Cocktail

“The goal here is to address the entire DMD population,” Sarepta Chief Financial Officer Sandy Mahatme said by phone. “There is some thought that a cocktail of therapies would help stabilize the disease, or perhaps even go along the way to curing the disease.” Beyond acquisitions Sarepta is looking for partnerships and areas to invest in early research, Mahatme said.

Sarepta’s path to submit its first drug was a difficult one. FDA regulators delayed the company’s application in October, asking for more data, and Garabedian resigned in March.

The drug, eteplirsen, received priority review from the FDA and could be approved by the beginning of 2016, according to the company. BioMarin’s drug drisapersen could be approved by December.

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