2016 has been a busy year for PAH. There was exciting progress and some disappointment as well. Reata Pharmaceuticals launched their phase 3 study of Bardoxylone Methyl in connective tissue disease related PAH (The CATALYST Study) after showing promising results in their phase 2 study. Actelion has started their phase 4 study (TRITON) comparing triple vs double up-front therapy (Opsumit-Macitentan and Adcirca-Tadalafil compared to Opsumit, Adcirca and Uptravi-Selexipag). Bellerophon began enrolling their phase 3 study of inhaled nitric oxide in PAH.
The PHA began their long-awaited pulmonary hypertension registry project. This will hopefully yield important information about PAH patients and best care practices over time. We are expanding on our understanding of differences in gene expression between PAH patients and people without PAH. Hopefully this will result in new treatments in the future.
In the disappointment category, Gilead’s phase 2 study of their ASK-1 inhibitor did not show positive results. The underlying mechanism of action remains very exciting and there were no reported safety concerns. We hold onto a glimmer of hope that the molecule will continue development in a more targeted subset of patients with PAH.
The biggest frustration by far for 2016 has been the changes in Caring Voices Coalition. This has resulted in many patients no longer qualifying for charitable support to afford their medications.
Looking Forward to 2017
We hope that United Therapeutics’ FREEDOM EV study of Orenitram in combination with other oral therapies will be completed and we will have additional data on the value of this therapy in PAH. Perhaps the most exciting near term expectation for 2017 is that the FDA will approve United Therapeutics and Medtronic collaborative effort on the fully implanted Remodulin pump system. Bayer will begin enrolling a study comparing Adempas (Riociguat) to Adcirca (Tadalafil) or Revatio (sildenafil).
Lastly, we remain optimistic that the drug companies that produce the important medications for PAH patients will be able to work with the federal government to find a solution to the cost barriers that are threatening to prevent many patients from being able to afford their medications.