Green light for Ascendis’ Phase III in Growth Hormone Deficiency

It seems like the €48M Series D financing and the public introduction on the NASDAQ have paid off. Danish Ascendis Pharma announced positive top-line results from a Phase II study that evaluated the once-weekly TransCon Growth Hormone in children with growth hormone deficiency.

After Zosano, Ascendis was the second European company that went public at the NASDAQ this year. The raised $111.5M (€102M) mainly paid the development of the company’s in-house growth hormone treatment. Today, this project is one step closer to the market.

Great Phase II results get the all-clear for the next level. The six month Phase 2 study was conducted in 53 treatment-naïve, pre-pubertal children with growth hormone deficiency. Three doses of Ascendis’ TransCon Growth Hormone were compared to Pfizer’s approved treatment Genotropin. The latter is one out of two approved long-acting growth hormone products on the market. Both use unmodified growth hormone as the active agent.

Ascendis uses its TransCon technology to create enhanced prodrug versions of already existing drugs. That means, the company’s drug is likewise an unmodified growth hormone combined with its unique platform. The big advantage of Ascendis’ potential treatment would be the once-weekly injections, in contrast to Pfizer’s required daily injections.

The goal of therapy was to restore safe and effective levels of growth hormone throughout the body so that the child has the potential to normalize metabolism and body composition and achieve a normal adult height. In terms of efficacy, safety and tolerability, once-weekly TransCon Growth Hormone appeared comparable to the gold-standard daily growth hormone therapy.

Ascendis’ pipeline.

Besides its Growth Hormone treatments, the company’s technology contributes to partnerships with Genentech and Sanofi – the latter dumbed another TransCon insulin project in April. Taken together with the same treatment in adults, which completed Phase II last year, the company may soon be facing a $3Bn market.

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