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Doubts raised over 'read-through' Duchenne drug mechanism

Nature Biotechnology volume 31, pages 771–772 (2013)Cite this article

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Now under regulatory review in Europe for Duchenne muscular dystrophy (DMD), ataluren has had a checkered history. In 2008, PTC secured an upfront payment of $100 million from Genzyme (now part of Paris-based Sanofi) in return for rights to the product outside the US and Canada. But the deal was terminated following lackluster data from a phase 2b trial in DMD. Subsequently, a phase 3 trial in cystic fibrosis also failed to reach statistical significance. Because the drug showed signs of efficacy in each indication, however, PTC pressed ahead. A phase 3 trial in DMD is now underway, and a second phase 3 trial in cystic fibrosis will commence shortly. The recent PLOS Biology study, led by Stuart McElroy, of the University of Dundee, in Dundee, Scotland, arose from the establishment of a drug screening unit. Ataluren was one of several molecules his team used to validate the reporter assays it set up. “We certainly never wanted to get caught in the trap where we optimized a compound on an off-target activity,” McElroy says. “That's one of the challenges of all drug discovery, if you optimize your drug on one activity.”

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    Cormac Sheridan

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Sheridan, C. Doubts raised over 'read-through' Duchenne drug mechanism. Nat Biotechnol 31, 771–772 (2013). https://doi.org/10.1038/nbt0913-771

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