Ascendis Pharma A/S Announces Initiation of a Phase 1 Single Ascending Dose Study of TransCon Treprostinil

Copenhagen, Denmark, January 14, 2015 – Ascendis Pharma A/S, a clinical stage biotechnology company that applies its innovative TransCon technology to address significant unmet medical needs, today announced that it has initiated a Phase 1 single ascending dose study of TransCon Treprostinil in healthy volunteers. TransCon Treprostinil is being developed to treat pulmonary arterial hypertension, a life-threatening orphan disease characterized by elevated blood pressure in the pulmonary arteries.

TransCon Treprostinil has been designed as an inert prodrug that enables a sustained release of unmodified treprostinil in circulation following a once-daily self-administered subcutaneous injection. TransCon Treprostinil is intended to produce a pharmacokinetic profile comparable to continuously infused prostacyclins, with the benefit of a more convenient route of administration, while avoiding the infusion site pain and risk of bloodstream infections associated with currently marketed infused prostacyclin therapies.
“The initiation of this Phase 1 study for our second wholly-owned clinical program is an important milestone for Ascendis," stated Jan Mikkelsen, President and Chief Executive Officer of Ascendis Pharma. "Our clinical stage pipeline is now comprised of long-acting TransCon prodrugs that incorporate both small molecules and proteins, demonstrating the broader potential of the TransCon technology platform.” The Phase 1 study is a single ascending dose study that will evaluate pharmacokinetic, injection tolerability, and safety parameters in a healthy volunteer population. Ascendis expects to report results from this study in mid-2015.

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology, which combines the benefits of prodrug and sustained release technologies, to develop a pipeline of best-­‐in-­‐class therapeutics that address significant unmet medical needs. The TransCon technology can be applied to existing drug therapies, including proteins, peptides and small molecules, to create prodrugs that provide for the predictable and sustained release of an unmodified parent drug.

The Ascendis Pharma pipeline includes TransCon Growth Hormone, a proprietary program that has completed a Phase 2 study in adults with growth hormone deficiency, or GHD. Ascendis Pharma is currently conducting a Phase 2 study of TransCon Growth Hormone in children with GHD. Ascendis Pharma is also developing its wholly-owned TransCon Treprostinil for the treatment of pulmonary arterial hypertension, or PAH. TransCon Treprostinil is currently in a Phase 1 single ascending dose study in healthy volunteers. In addition to its proprietary programs, Ascendis Pharma has formed collaborations focused on leading products in large markets that are of strategic importance to its collaboration partners. These collaborations are with Sanofi in diabetes and Genentech in the field of ophthalmology.

Contact:
Investor Relations:
Martin Auster, M.D.
Chief Business Officer
(650) 617-3403
MA@AscendisPharma.com
Media Relations:
Marion Janic
Rooney & Associates
212.223.4017
mjanic@rooneyco.com