Phase III fliGHt trial shows TransCon Growth Hormone safe in pediatric growth hormone deficiency

Ascendis Pharma announced preliminary results from the phase III fliGHt Trial of TransCon Growth Hormone (hGH). The results indicated treatment with TransCon hGH was safe and well-tolerated in subjects with pediatric growth hormone deficiency (GHD) who were previously treated with commercially-available daily growth hormone therapies. In the trial, the adverse event profile of TransCon hGH was similar to the published safety profile of daily growth hormone therapies with no drug-related discontinuations or drug-related serious adverse events, no neutralizing antibodies, and a low level of low-titer non-neutralizing antibodies. These fliGHt Trial data include new information demonstrating safety and tolerability in treatment-naïve subjects under three years of age.

Along with data from the pivotal heiGHt Trial and the enliGHten (long-term extension) Trial, these results form a safety database of approximately 300 subjects treated with TransCon hGH (approximately 300 for six months, 120 for 12 months and 45 for 24 months), consistent with input from regulatory authorities. The phase III, open label, 26-week single arm fliGHt Trial enrolled 143 subjects with pediatric GHD who were previously treated with commercially-available daily growth hormone therapy (average dose was 0.29 mg/kg/week reflecting typical treatment patterns in the U.S.), and three treatment-naïve subjects under three years of age. The company plans to present detailed results from the fliGHt Trial at its upcoming R&D Update on Wednesday, June 26 in New York City, and at future medical conferences.

Comment: Ascendis plans a clinical database lock for the TransCon hGH phase III program in the third quarter of 2019. Subsequently, the company intends to submit a Biologics License Application (BLA) with the U.S. Food and Drug Administration for TransCon hGH to treat pediatric GHD in the first half of 2020.