PTC Therapeutics, Inc. (PTCT) Tuesday announced positive data from two different long-term extension studies of vatiquinone in Friedreich ataxia (FA). The primary endpoints of the studies were met. Following the news, the company's shares were trading more than 5 percent up in pre-market.
Friedreich ataxia (FA) is a rare, genetic disorder that causes progressive damage to the nervous system.
Results from the MOVE-FA long-term extension study showed that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit on the modified Friedreich Ataxia Rating Scale (mFARS). mFARS is a series of physical examination assessments to measure disease progression in patients with FA. PTC said this treatment difference on the primary endpoint represents a 50 percent slowing in disease progression over 3 years.
Additionally, PTC analyzed long-term open-label data from an earlier study of vatiquinone in adults with FA. Following 24-months of treatment with vatiquinone, subjects had a 4.8-point benefit on the mFARS.
The company plans to submit NDA for vatiquinone in December this year.
PTC shares had closed at $34.99, down 0.31 percent on Monday. It has traded in the range of $17.53 - $40.69 in the last 1 year.
For comments and feedback: editorial@rttnews.com