Shares of Ascendis Pharma A/S jumped more than 70% upon the release of clinical trial results that showed the company's once-weekly TransCon Growth Hormone therapy encouraged more growth in pediatric patients with growth hormone deficiency than Pfizer Inc.'s daily Genotropin medicine.
Daily injections of Genotropin, or height growth hormones, are the only available treatment option in the U.S. and Europe for children with growth hormone deficiency, or GHD. Pediatric GHD is a rare condition that occurs when the pituitary gland does not produce enough growth hormones, causing children with the disease to have shorter statures.
The Ascendis treatment uses the same growth hormone in Genotropin, but the TransCon technology attaches a carrier that protects the drug from being eliminated by the body.
Ascendis compared a weekly-dose of TransCon height growth hormone with the once-daily standard treatment in a 52-week phase 3 trial called heiGHt. The study enrolled 161 children who had not previously been treated for GHD. Average height velocities for the weekly and daily injections were 11.2 centimeters and 10.3 centimeters per year, respectively.
The treatment is also being evaluated in two other phase 3 trials; results from fliGHt are expected in the second quarter of 2019, while enliGHten is a long-term extension of the other two trials.
In a presentation at the J.P. Morgan Healthcare Conference in January, Ascendis President and CEO Jan Møller Mikkelsen said 100% of the patients enrolled in the heiGHt trial — those taking the weekly therapy and daily Genotropin — moved over to the once-weekly therapy and enrolled in the follow up enliGHten trial. The company will introduce an auto-injector application during the enliGHten trial.
"Our goal is to alleviate the burden of daily injections so every child has a better opportunity to achieve normal adult height and overall endocrine health — and to look forward to a healthy future," said Ascendis Chief Medical Officer Jonathan Leff in a statement.
The Danish company plans to apply for a biologics license with the U.S. Food and Drug Administration for TransCon height growth hormone to treat pediatric GHD in the first half of 2020.
Shares of Ascendis climbed 70.44% to $118.13 as of 1:33 p.m. ET. on March 4.