Ascendis Pharma Reveals Results of Phase 1 Study of Pulmonary Arterial Hypertension Therapy TransCon Treprostinil

Ascendis Pharma A/S, a clinical stage biotechnology company focused on creating drug candidates by improving existing drugs, recently announced results from its Phase 1 single ascending dose trial of TransCon Treprostinil, which produced a dose-dependent increase in plasma treprostinil levels. However, results from the study showed that the tolerability of treprostinil injection-site did not meet the criteria described in the target product profile.

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The mixed results from the trial are a setback for the PH community, given the therapy’s potential benefits to improve upon a currently-used treatment for the disease. TransCon Treprostinil is being developed to treat Pulmonary Arterial Hypertension, a life-threatening disease characterized by elevated blood pressure in the pulmonary arteries. As the condition progresses, the right side of the heart works harder to pump blood to the lungs, which eventually leads to right heart failure and ultimately death. Treatment with prostacyclin is the gold-standard therapy for PAH patients.

Intravenous infusion of prostacyclin analogs requires the use of a surgically implanted central venous catheter, but it can also be administered through inhaled or oral routes. However, these therapies provide a lower cumulative dose of prostacyclin as compared to the dose received with continuous infusion therapy. This results in overall systemic exposure of prostacyclin. To address these issues, TransCon Treprostinil is designed as a once-daily self-administered subcutaneous injection of treprostinil, the active agent in Remodulin®, and offers the same efficacy as continuously-infused prostacyclin analogs, however 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.

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The company is applying its pioneering TransCon technology to the development of Treprostinil, which combines the benefits of prodrug and sustained release technologies that company believes will lead to a pipeline of best-in-class therapeutics that address significant unmet medical needs. Ascendis plans to conduct more research on new product formulations of TransCon Treprostinil and also expects to continue clinical development when improvements in the product address the limitations revealed in the recent clinical trial results.

Jan Mikkelsen, Chief Executive Officer of Ascendis, stated in a recent news release “While we are pleased with the pharmacokinetic results observed in this study, and believe they affirm the potential of our TransCon technology, TransCon Treprostinil did not reproduce the tolerability profile observed in preclinical studies.”  Mr. Mikkelsen continued, “As we look forward to the remainder of 2015, we plan to finalize and report topline data from our Phase 2 pediatric study of TransCon Growth Hormone by mid-year and prepare for the initiation of our Phase 3 pediatric study in mid-2016.  We will also continue to develop our internal pipeline of therapeutics for orphan diseases.”