Ascendis banks $60M to get its orphan drug into Phase III

Danish biotech Ascendis Pharma has put together a $60 million D round, cash that'll help get its once-a-week treatment for growth hormone deficiency (GHD) into late-stage testing.

Ascendis' bedrock is a technology called TransCon, a hydrogel made of prodrug linkers that seek out targeted molecules in the body and then release their therapeutic payload. The biotech's top prospect is TransCon growth hormone, a weekly GHD therapy designed to spare patients from the daily injections that make up the standard of care.

In a Phase II study in adults with GHD, the weekly treatment met all of its primary and secondary endpoints, demonstrating itself to be as effective as daily growth hormone shots. And, in interim results from a Phase II pediatric study revealed in September, TransCon growth hormone is performing comparably to Pfizer's ($PFE) daily Genotropin. Ascendis expects to release top-line results from that study in mid-2015, using the results to inform a late-stage program and its latest fundraise to pay for it.

Sofinnova Ventures, OrbiMed and Vivo Capital co-led the new round, joined by a syndicate of investors including Janus Capital Management, Venrock, RA Capital Management and the Paris-headquartered Sofinnova Partners, which is Ascendis' largest shareholder.

For Sofinnova Ventures, which closed a $500 million new fund over the summer, pitching in on Ascendis' latest round affirms its confidence not only in the biotech's lead program but also the promise of TransCon as a whole, General Partner Dr. James Healy said. Beyond GHD, Ascendis has plans to get a TransCon-powered pulmonary arterial hypertension treatment into the clinic next year, and the technology has further applications in ophthalmology and diabetes, according to the company.

"One of the important things for patients with pulmonary arterial hypertension or growth hormone deficiency is being able to take effective therapies and be able to comply with them, so if you can increase compliance by decreasing frequency of injections and thereby have better outcomes, it's a win for everyone," Healy said. And the biotech's core mission--"taking existing drugs and longer acting prodrugs that are able to create best-in-class therapeutics"--has wide applications in other diseases, he said.

Healy, who is taking a seat on Ascendis' board, isn't disclosing whether the biotech has its eye on an IPO or is feeling around for a buyout.

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