Continuous exposure to prostacyclin is well-established as the gold-standard treatment for late-stage Pulmonary Arterial Hypertension (PAH) as “rescue therapy” for deteriorating patients. However, despite current treatment guidelines recommending prostacyclin analog therapy as mono or combination therapy in patients with PAH, clinicians frequently delay the initiation of prostacyclin therapy due to inconvenient administration and side effects.
Current administration of prostacyclin therapy is often through a subcutaneous infusion which causes infusion site pain and injection site reactions in a majority of patients. These symptoms can be severe and lead to the discontinuation of the treatment. Intravenous infusion of prostacyclin analogs requires the use of a surgically implanted central venous catheter and, if strict aseptic preparation procedures are not followed, patients receiving intravenously infused prostacyclin may face sepsis, a life threatening bloodstream infection. Prostacyclin therapy can also be administered through inhaled or oral routes, but these therapies provide a lower cumulative dose of prostacyclin than the dose received with continuous infusion therapy.
TransCon Treprostinil is designed as a once-daily self-administered subcutaneous injection of treprostinil, the active agent in Remodulin®, and is intended to offer the same efficacy as continuously-infused prostacyclin analogs, but with a safer and more convenient route of administration and without the infusion site reaction and bloodstream infection risks associated with the currently approved routes of treprostinil administration. Proof of principle for the TransCon Treprostinil product profile was obtained in a Phase 1 study in healthy volunteers with a first generation formulation of TransCon Treprostinil. New product formulations with improved characteristics are being investigated for future development; in addition, this product candidate is currently a partnering opportunity.
PAH is a life-threatening disease characterized by elevated blood pressure in the pulmonary arteries. As the disease worsens, the right side of the heart works harder to pump blood to the lungs, which may eventually lead to right heart failure and, potentially, death.