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PTC Therapeutics Drug Doesn't Meet Goal in Duchenne Trial

PTC Therapeutics Inc.’s drug for the rare disorder Duchenne muscular dystrophy failed to help boys with the muscle-wasting disease walk better in a final-stage clinical trial, the company said.

In a trial of 228 patients, PTC measured how far the boys could walk in six minutes. Those on the drug went 15 meters further than those given a placebo, a difference that is not considered statistically significant, according to a statement on Thursday.

Patients with moderate disease were most likely to benefit, going 47 meters farther during the walk test, a significant difference. None of the boys in this subgroup lost their ability to walk during the trial, compared with four getting a placebo, according to the statement. PTC also pooled together data from the phase 3 study and a previous study, and in the meta-analysis, the difference in the six-minute walk test was a significant benefit of 22 meters, PTC said in an e-mail. Shares rose 4 percent to $33 in extended trading in New York.

Christopher Marai, an analyst at Oppenheimer & Co., said the meta-analysis will probably be enough to convince regulators to approve the drug.

“We believe these results will be enough to keep Translarna on the market in the EU and garner FDA approval in the US,” Marai wrote in a note to clients, referring to the U.S. Food and Drug Administration.

‘Totality’ of Data

The company also said the findings may be enough to persuade regulators to back the drug.

“We’re excited because the totality of the clinical data do confirm the benefit we previously saw in the Phase 2 study and, in our perspective, supports the benefit of Translarna,” PTC Chief Financial Officer...


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