Urgent pleas from parents greet biotech's new drug

There's a hard, urgent logic to Jenn Mcnary's plea: Her 13-year-old son, Austin, is wasting away from an incurable genetic disease, while her 10-year-old with the same disorder has been helped by testing a cutting-edge drug that's not yet approved for sale.

"I would love to get him on this medication to give him as good a chance as Max has gotten," the Vermont mom says. "At this stage anything that can stop the progression is a lifesaver."

There's an equally compelling logic to the strategy of Sarepta Therapeutics CEO Chris Garabedian, who recently reported clinical-trial results that suggest the drug may be the first effective treatment for slowing Duchenne muscular dystrophy.

"We could spend all our time answering phone calls from parents who would like our drug, and putting in the paperwork to the FDA" for individual "compassionate use" cases in which Sarepta can't charge money, he says. "That is the surest path to insolvency."

Sarepta -- until recently called AVI BioPharma and based in Bothell but now headquartered outside Boston -- may be one of those biotech fairy tales that sustains belief in the entire industry: A little company that brings a novel lifesaving drug to market.

But its steps toward that goal can't come fast enough for parents whose sons are losing the ability to move their own feet. It makes for a complicated relationship.

The degenerative Duchenne disorder, which affects an estimated 13,000 U.S. boys with a faulty X chromosome, usually leads to loss of mobility in the teens, then difficulty in breathing and eventually heart failure, usually by age 30.

The current version of Sarepta's treatment targets a subset of about 15 percent of that population, making it an "ultra-orphan" drug.

Sarepta in late July unveiled the results from a 36-week Phase 2 clinical trial of the drug, Eteplirsen, in 12 boys aged 7 to 13. The principal investigator on the trial, at Nationwide Children's Hospital in Columbus, Ohio, called the drug's effect "unprecedented."

That set off a surge in shares of Sarepta, which shot from $3.46 to a high above $15 over six weeks.

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Urgent pleas from parents greet biotech's new drug