Genzyme, PTC: Mid-stage trial for DBMD treatment yields mixed results

SOUTH PLAINFIELD, N.J. A mid-stage clinical trial of a drug for muscular dystrophy got mixed results, developers Genzyme Corp. and PTC Therapeutics announced.

The two companies recently announced results of a 174-patient phase 2b trial of ataluren, for treating nonsense mutation Duchenne/Becker muscular dystrophy, also known as DBMD, which can cause loss of walking ability as early as age 10 and life-threatening lung and heart complications by the late teens and early 20s. The disease affects around 1,900 patients in the United States. A “nonsense mutation” is a mutation of the DNA that causes incomplete manufacture of proteins.

The companies said the main goal of the study –– change the distance patients could walk within six minutes –– did not reach statistical significance, but that the drug was well-tolerated and no patients discontinued treatment due to adverse side effects. The drug also is undergoing studies as a treatment for nonsense mutation cystic fibrosis and hemophilia.

“The quality of the data from this well-conducted study and additional analyses will help to inform the clinical development of ataluren in other indications,” Genzyme SVP genetic diseases Geoffrey McDonough said. “We remain committed to the development of ataluren and will continue to collaborate with PTC to advance its development for the treatment of genetic disorders.”