Phase 3 heiGHt trial met primary objective in children with pediatric growth hormone deficiency
Ascendis Pharma (ASND) announced positive top-line results from the phase 3 heiGHt Trial, a randomized, open-label, active-controlled trial that compared once-weekly TransCon Growth Hormone to a daily growth hormone in children with pediatric growth hormone deficiency.
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 at 52 weeks.
In the primary analysis of the intent-to-treat population using ANCOVA, TransCon hGH demonstrated an AHV of 11.2 cm/year compared to 10.3 cm/year for the daily hGH.
The treatment difference was 0.86 cm/year with a 95% confidence interval of 0.22 to 1.50 cm/year.
The AHV for TransCon hGH was significantly greater than the daily hGH.
The AHV was greater for TransCon hGH than for the daily hGH at each visit, with the treatment difference reaching statistical significance from and including week 26 onward.
The incidence of poor responders was 4% and 11% in the TransCon hGH and daily hGH arms, respectively. All sensitivity analyses completed from the trial support the primary outcome, indicating the robustness of these results.
Results from the trial indicate that TransCon hGH was generally 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.
ASND is up $44.64 to $113.95.
Cantor Fitzgerald analyst Alethia Young raised her price target for Ascendis Pharma to $185 from $100 as she was “positively surprised” that the TransCon hGH study for growth hormone achieved a superior result on annualized growth velocity of .86cm.
The analyst thinks that the expectation had been non-inferiority with a slightly favorable numerical trend. Young also thinks safety was clean as well, which has been a challenge with other long acting growth hormones.
She would expect shares to trade up 50%-100% based on her model, and sees continued upside from shares as additional programs, such as TransCon PTH, achieve proof of concept and more candidates from the proprietary TransCon platform enter the clinic. The analyst reiterates an Overweight rating on the shares.
Growth hormone (GH) has been available for management of the short stature associated with growth hormone deficiency (GHD) for more than 60 years; recombinant DNA-derived human growth hormone (rhGH) has been available since 1985. While availability is no longer a problem, there still remains a number of difficulties with the diagnosis of GHD, resulting mainly from the lack of appropriate tools to make (or exclude) the diagnosis reliably.
The incidence of short stature associated with GHD has been estimated to be about 1:4000 to 1:10000 . It is the primary indication for GH treatment in childhood, which presently requires daily subcutaneous injections for the patient and substantial cost for the healthcare system. Based on these premises, it is clear that an accurate diagnosis is essential
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