The chief executive officer, Edward Kaye, of Cambridge, Mass.-based Sarepta Therapeutics (SRPT) recently indicated the company is looking to acquire a drug to treat Duchenne muscular dystrophy (DMD).


On June 29, 2015, Sarepta announced it had completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for eteplirsen for DMD. Eteplirsen utilizes the company’s proprietary exon-skipping technology to “skip” exon 51 of the dystrophin gene, which allows the cell’s repair mechanism to fix the specific genetic mutation that affects about 13 percent of those with the disease.

Sarepta is in a head-to-head race with competitor BioMarin Pharmaceutical Inc. (BMRN), which is headquartered in San Rafael, Calif. BioMarin beat Sarepta to the FDA with its NDA in April for drisapersen, also an exon-skipping drug candidate for DMD.

“We believe drisapersen may offer a meaningful benefit to boys living with DMD whose mutations are amenable to exon 51 skipping,” said Camilla Simpson, BioMarin’s global head of regulatory affairs, pharmacovigilance in an April 27 statement. “The totality of data on drisapersen contains three randomized, placebo-controlled, efficacy trials and two long term extension studies, which include some boys treated for approximately 3.4 years.”

If Sarepta does acquire another drug for DMD, the strategy would be to potentially mix the new drug with eteplirsen for a cocktail of treatments. In a recent interview with Bloomberg Business, Kay said, “We are going to own DMD,” adding that the company hoped to offer “a menu of options” to patients.

It’s unlikely that BioMarin would take the challenge lying down. BioMarin has $1.01 billion in cash, whereas Sarepta has about $166 million. Sarepta may have an advantage in terms of debt, only $6.4 million compared to BioMarin’s $656 million. If there’s any hint of a collaboration between the two companies, nobody’s talking about it. At a San Francisco investor meeting in January, the two companies’ chief executives sniped at each other over data claims, which suggests any deal would be unlikely.

Other companies working in the DMD space include Pfizer Inc. (PFE), Nicox SA, PTC Therapeutics Inc. (PTCT)), Santhera Pharmaceutical Holding AG, Catabasis Pharmaceuticals, Inc., Summit Therapeutics plc, and Capricor Therapeutics, Inc..

BioMarin most recently announced positive results from a Phase II clinical study for BMN-111 (vasoritide) for the treatment of achondroplasia, the most common form of human dwarfism. Raluca Pancratov of SunTrust Robinson Humphrey wrote in a research note that if that drug were approved, it could bring in about $700 million worldwide.

BioMarin Pharmaceutical stock has been on a steady rise for the last year. On July 17, 2014, shares traded for $55.78 and rose to $67.27 on Oct. 14, 2014. On Mar. 30, 2015, shares traded for $129.14 and are trading today at $144.50.

Sarepta Therapeutics stock has been much more volatile. Shares traded for $23.56 on Oct. 24, 2014, dropped to $11.61 on Jan. 13, 2015 and rose slightly to $12.20 on April 30, 2015. On May 20, 2015 shares took a big jump, rising to $26.24, then moving to $30.43 on June 30, 2015. Shares are trading currently at $30.90.

Duchenne muscular dystrophy is a genetic disorder that results in progressive muscle degeneration and weakness. It is caused by an absence of a protein called dystrophin, which helps to keep muscle cells intact. The disease primarily affects boys and onset is typically between the ages of 3 and 5. Although survival rates have improved greatly recently, the most common age of mortality is in the early 30s, although there are some cases of men surviving into their 40s and 50s.

Sarepta’s drug, eteplirsen, may be approved by early 2016. BioMarin’s drug, drisapersen, might be approved by December 2015.


July 14, 2015

By Mark Terry, Breaking News Staff

Source: BioSpace Featured News