United Therapeutics Announces Collaboration with Ascendis Pharma to Develop Self-Injectable Treprost

SILVER SPRING, Md. - United Therapeutics Corporation (NASDAQ: UTHR) announced today that it has signed an exclusive agreement with Ascendis Pharma A/S to apply Ascendis Pharma's proprietary TransCon technology platform to United Therapeutics' treprostinil molecule, the active ingredient in Remodulin® (treprostinil) injection.  United Therapeutics believes that the TransCon technology platform may enable a controlled, long-acting release of a novel, carrier-linked product, significantly enhancing the delivery profile of treprostinil by establishing a self-injectable alternative for patients who currently use the drug via a continuous infusion pump for the treatment of pulmonary arterial hypertension (PAH).

"We are thrilled to enter into this license agreement with Ascendis Pharma,"

said Martine Rothblatt, Ph.D., United Therapeutics' Chairman and Chief Executive Officer.  

"The potential to bring another novel therapeutic option to PAH patients represents an exciting new opportunity for Remodulin delivery as we constantly re-charge our mission to better the lives of patients suffering from PAH."

"Pre-clinical studies in multiple animal models with a lead compound of TransCon-linked treprostinil have already produced encouraging results, demonstrating a sustained release profile supportive of once-daily injection.  The application of TransCon technology to treprostinil may also minimize injection site pain and reactions that frequently occur with continuously infused subcutaneous Remodulin,"

commented Roger Jeffs, Ph.D., United Therapeutics' President and Chief Operating Officer.  

"We are very excited to be working with Ascendis Pharma to further develop and advance this promising product into the clinic."

Under the terms of the agreement, United Therapeutics will also have exclusive rights to develop prostacyclin, prostacyclin analog and prostacyclin-related products for treatment of PAH using the TransCon technology, and will hold worldwide commercial rights to products resulting from the collaboration.  

About United Therapeutics
United Therapeutics Corporation is a biotechnology company focused on the development and commercialization of unique products to address the unmet medical needs of patients with chronic and life-threatening conditions. 

About Ascendis Pharma and TransCon
Ascendis Pharma A/S is an emerging specialty pharmaceutical company which creates improved, patentable versions of marketed drugs.  The company is headquartered in Palo Alto, California and Copenhagen, Denmark, with research in Heidelberg, Germany.

Ascendis Pharma's prodrug technology platform, TransCon, is a proprietary technology enabling transient conjugation of peptides, proteins and small molecules to various carriers.  The technology utilizes Ascendis Pharma's proprietary TransCon Linker families, which are a diverse group of structures with inherent self-cleaving properties, making drug release independent of enzyme activity and tissue conditions. Ascendis Pharma's transient conjugation is designed to release drug in the body in a precise, time-controlled fashion, thus creating a long-acting effect for the original unmodified drug.