PTC Therapeutics, Inc. (PTCT) has announced encouraging results from two long-term extension studies involving vatiquinone for treating Friedreich ataxia (FA). The studies successfully met their primary endpoints, leading to a pre-market share increase of over 5 percent for the company.
Friedreich ataxia is a rare inherited disorder that progressively impairs the nervous system. Over a 144-week period, the MOVE-FA long-term extension study demonstrated that vatiquinone treatment yielded a 3.7-point improvement on the modified Friedreich Ataxia Rating Scale (mFARS), which evaluates disease progression through a series of physical examinations. According to PTC, the observed difference in the primary endpoint signifies a 50 percent reduction in disease progression over a span of three years.
Furthermore, PTC analyzed long-term, open-label data from an earlier study of vatiquinone in adults with FA. After 24 months of treatment, participants experienced a 4.8-point benefit on the mFARS.
The company intends to submit a New Drug Application (NDA) for vatiquinone in December of this year.
On Monday, PTC shares closed at $34.99, reflecting a slight decrease of 0.31 percent. Over the past year, the stock has ranged between $17.53 and $40.69.
