Ascendis Pharma A/S (NASDAQ: ASND) was another biotech firm that saw its shares rise sharply on Monday (after a buyout announcement). In this case, Ascendis shares were up as the result of positive top-line results from its late-stage growth hormone study in children.
Specifically, results came from the Ascendis phase 3 heiGHt Trial that compared once-weekly TransCon Growth Hormone (hGH) to a daily growth hormone (Genotropin) in children with pediatric growth hormone deficiency.
Ultimately, the trial met its primary objective demonstrating that TransCon hGH was observed to be non-inferior and, additionally, superior to the daily hGH on the primary endpoint of annualized height velocity (AHV) at 52 weeks.
In the primary analysis of the intent-to-treat population, TransCon hGH demonstrated an AHV of 11.2 cm per year compared to 10.3 cm per year for the daily hGH. The treatment difference was 0.86 cm per year with a 95% confidence interval of 0.22 to 1.50 cm per year. The AHV for TransCon hGH was significantly greater than the daily hGH.
Results from the trial indicate that TransCon hGH generally was safe and well-tolerated, with adverse events consistent with the type and frequency observed with daily hGH therapy and comparable between arms of the trial.
Jan Mikkelsen, Ascendis Pharma’s president and CEO, commented:
The heiGHt Trial results announced today represent a potential breakthrough for patients and future treatment options for growth hormone deficiency. The heiGHt Trial demonstrated that TransCon hGH had superior efficacy, as well as comparable safety and tolerability to daily growth hormone. We believe these results provide a validation of our TransCon technology platform, which forms the basis of our endocrinology pipeline and has potential application in other therapeutic areas.
Shares of Ascendis were last seen up about 67% at $115.73, in a new 52-week range of $53.21 to $116.00. The consensus price target is $85.78.