United Therapeutics Corporation, a biotechnology company focused on the development and commercialization of unique products, has signed an exclusive agreement with Ascendis Pharma A/S, an emerging specialty pharmaceutical company, 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).

Remodulin is a prostacyclin vasodilator indicated in the United States for the treatment of pulmonary arterial hypertension (PAH) (WHO Group 1) to diminish symptoms associated with exercise.  Studies establishing effectiveness included patients with NYHA Functional Class II-IV symptoms and etiologies of idiopathic or heritable PAH (58%), PAH associated with congenital systemic-to-pulmonary shunts (23%), or PAH associated with connective tissue diseases (19%).

"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.