PTC Therapeutics' lead drug ataluren failed a Phase IIb trial, flunking a test of its ability to improve the physical performance of patients with a rare genetic disorder. Genzyme is partnered on the program.
Researchers tested the therapy in patients with nonsense mutation Duchenne/Becker Muscular Dystrophy. The primary endpoint--a change in 6-minute walk distance--did not reach statistical significance within the 48-week duration of the study, according to preliminary results.
PTC has pinned some big hopes on commercializing ataluren. The developer highlighted its clinical trial work on the therapy just two months ago, when it closed on a $50 million financing round. "We believe the commercial launch of ataluren will represent a new paradigm in the treatment of genetic disorders," noted Delphi Ventures' Deepa Pakianathan at the time. And PTC showed no signs of backing away from its commitment at this stage of the game.
"These results further demonstrate the safety profile of ataluren and support continued development," remarkd Langdon Miller, PTC's chief medical officer. "DBMD is a progressive, debilitating, and life-threatening neuromuscular disorder. The variability of symptom onset, disease progression, and 6-minute walk distance creates challenges for clinical development. Importantly, this trial does provide a wealth of valuable data about ataluren and DBMD. Additional analyses will guide the overall clinical and regulatory path forward."
- check out the PTC's release