Ascendis Pharma A/S (ASND) Monday said the pivotal ApproaCH study of TransCon CNP, in children with achondroplasia met its primary goal. The company's shares were more than 17 percent up in pre-market.
Achondroplasia is a genetic disorder that causes short-limbed dwarfism.
The study evaluated 84 children aged 2-11 years with achondroplasia. They were randomized to receive either TransCon CNP or placebo. Children treated with TransCon CNP demonstrated an LS mean annualized growth velocity (AGV) of 5.89 cm/year compared to 4.41 cm/year in the placebo arm, at week 52, achieving the primary endpoint. Further, TransCon CNP was generally well-tolerated.
Ascendis stock had closed at $119.22, up 4.75 percent on Friday. It has traded in the range of $85.29 - $161.00 in the last 1 year.
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