UTHR, Ascendis collaborate for self-injectable PAH medication

The partnership will focus on developing self-injectable treprostinil molecule, the active ingredient in Remodulin (treprostinil) injection, for pulmonary arterial hypertension (PAH).

The TransCon technology platform is expected enhance the delivery profile of treprostinil by providing a controlled, long-acting release of carrier-linked product.

United Therapeutics chairman and chief executive officer Martine Rothblatt said, "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."

As a part of the agreement, United Therapeutics gains exclusive rights to develop prostacyclin, prostacyclin analogue and prostacyclin-related products as PAH therapies leveraging the TransCon technology, and retains worldwide commercial rights to products resulting from the collaboration.

United Therapeutics president and chief operating officer Roger Jeffs said pre-clinical studies in multiple animal models with a lead compound of TransCon-linked treprostinil have 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," Jeffs added.