Roche Holding Ltd. (ADR) (OTCMKTS:RHHBY) has suspended dosing in Phase II trial of PTC Therapeutics, Inc. (NASDAQ:PTCT) . The trial was for the development of spinal muscular atrophy (SMA) therapy. The suspension is due to safety problems stemming from the preclinical study.
The drug, named RG7800 is currently in a 64 patient Phase II trial that is designed to determine the efficacy and safety of the drug in the treatment of SMA. The drug showed positive results in Phase I study. PTC gave dosage dependent on SMN2 gene that affects the disease.
In long term study on animals, the researchers ran into “eye finding” and therefore as a precautionary measure both the companies decided to suspend the dosing of more patients to research the drug. The step has led to a suspension of Phase II trial of the drug.
The exact nature of the finding was not disclosed however it was stated that the dosage given to animals was more than any patient in the study and that the issue had not come up in human trials. Further details were not given by PTC.
Roche bought the exclusive license to the program in 2011 for $30 million an up to $460 million in future to partner with PTC and its partners at the SMA Foundation. The Swiss pharmaceutical company has agreed to pay up to $545 million for Trophos, a French biotech company working on Phase II SMA Treatment on SMA.
PTC is persisting with its controversial Duchenne muscular dystrophy (DMD), though rumours persist that PTC is looking for potential buyers. The biotech company won early European approval for the DMD despite earlier failure. PTC is currently working on Phase III and plans to post data by the end of the year. The success in Phase III will determine whether a taluren will stay in the European markets or not.
