Sarepta May Seek Partnership Advice for Experimental Drug

Sarepta Therapeutics Inc., a developer of an experimental drug for Duchenne muscular dystrophy, may seek advisers to find an overseas partner for the therapy, Chief Executive Officer Christopher Garabedian said.

The biotechnology company is talking with more than a dozen drugmakers about a partnership for international sales of eteplirsen, Sarepta’s medicine for the rare muscle-wasting disease, Garabedian said in an interview at the JPMorgan Chase & Co. health-care conference in San Francisco. Cambridge, Massachusetts-based Sarepta is considering hiring an advisory firm, such as Centerview Partners LLC or JSP Partners, to help with the talks. The company isn’t for sale, he said.

While successful testing of eteplirsen may make Sarepta, a 32-year-old company with no marketed products, an acquisition target, Garabedian said he wants it to become a rival to large biotechnology companies such as Gilead Sciences Inc., the world’s biggest maker of AIDS medicines and Celgene Corp., the maker of the $3 billion cancer drug, Revlimid.

“I’m building a team that knows how to create a successful global biopharmaceutical company,” Garabedian said. “I’m hiring people from Genzyme, Gilead, Celgene, Shire, and Vertex. Those people like myself want to work on the next break-out biotech.”

Duchenne muscular dystrophy is a form of the disease that affects boys and worsens quickly. It is caused by a defective gene for a protein in the muscles and occurs in about one of every 3,600 male infants, according to the National Institutes of Health.

Sarepta declined less than 1 percent to $26.18 at the close of New York trading.

Gene Repair

Eteplirsen repairs a gene mutation in about 13 percent of patients, helping them produce the missing protein and gain strength, the company has said. The drug is made by manipulating RNA, or ribonucleic acid, which controls protein synthesis.

Sarepta’s shares have increased more than fourfold in the 12 months through yesterday, including tripling in a single day last October when the company announced positive results for the drug in a test of eight patients.

Investors have driven up the share price with an acquisition in mind though no deal will happen until a clear regulatory path is established for eteplirsen in the first quarter of the year, said Kimberly Lee, an analyst at Janney Montgomery Scott LLC. GlaxoSmithKline Plc, Pfizer Inc. and Sanofi would be likely bidders as they are looking to acquire drugs to expand their products for rare diseases, Lee said.

Sarepta’s next key marker is its meeting to discuss mid-stage testing with the U.S. Food and Drug Administration, which will allow the company to decide if it can seek accelerated approval, Garabedian said.

Further Testing

Lee said Sarepta probably won’t receive accelerated approval based on current results in so few patients and likely will be required to run a larger, pivotal study. Prosensa, a Dutch biotechnology company, and Glaxo are conducting a Phase 3 study of a treatment called drisapersen for Duchenne muscular dystrophy patients with data expected by the fourth quarter. Drisapersen was once known as PRO-051.

“Investors are focused on whether the regulatory pathway is going to be accelerated approval versus standard approval,” Lee said.

Sarepta also must smooth out eteplirsen’s intellectual property in Europe, where the company has a patent dispute with Prosensa that may affect partnership talks, Garabedian said.

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