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Shares of drugmaker that targets gene mutations soar after positive muscular dystrophy study

  • Sarepta Therapeutics reports positive results from a clinical trial of an experimental medicine for Duchenne muscular dystrophy.
  • DMD is a rare, genetic disease that causes muscle wasting and can be fatal before patients turn 30.
  • The medicine targets a genetic mutation affecting about 8 percent of patients with DMD.

Shares of Sarepta Therapeutics soared 12 percent in early trading Wednesday after the biopharmaceutical company reported positive results from a clinical trial of an experimental medicine for Duchenne muscular dystrophy.

The drug, golodirsen, would be Sarepta's second to treat the rare, genetic disease, which causes muscle wasting and can be fatal before patients turn 30. Sarepta focuses on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases.

The new study, conducted in Europe, involved 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53. Exons are part of the DNA code. The treatment targets a genetic mutation affecting about 8 percent of patients with DMD.

Sarepta's first drug for DMD, Exondys 51 — approved on a conditional basis by the FDA last year pending more testing to confirm results — treats a mutation affecting about 13 percent. Exondys 51 costs about $300,000 per year.

"Our goal is to treat 100 percent" of DMD suffers, Sarepta CEO Doug Ingram told CNBC's "Squawk Box." "The data that we have this morning shows we're on the right path."

The results, announced before Wall Street's open bell, showed that golodirsen increased production of the protein dystrophin to 1.02 percent of normal levels from about 0.095 percent without the drug. Analysts said those results were higher than expected, but scientists wonder whether that's enough to increase muscle strength and have a clinical benefit.

According to Sarepta, the underlying cause of DMD is a mutation in the gene for dystrophin, which is an essential protein involved in muscle fiber function. DMD occurs in one in every 3,500 to 5,000 males worldwide. Symptoms usually start in early childhood, usually between 3- and 5-years old. It primarily affects boys. But in rare cases can affect girls.

"Sarepta is a small company. We have already invested $1 billion fighting Duchenne muscular dystrophy. And we're not done yet," said Ingram, who was appointed as CEO in July. Ahead of Wednesday, Sarepta had a stock market value of $2.6 billion.

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