About the PAH-001 Study

The PAH-001 clinical research study is for men and women over the age of 18 diagnosed with severe PAH (classified as World Health Organization [WHO] Function Class III).

This study will evaluate the optimal dose, safety, and potential therapeutic benefits of an experimental medication, ABI-009, to treat PAH. Everyone in the study will receive the same experimental medication, but the dose may be different. Patients in this study will also continue to receive their specific standard PAH therapies as recommended by their doctor while on the experimental medication.

A total of approximately 20 patients will participate in approximately 6 study centers in the United States.

About ABI-009, the experimental therapeutic in PAH-001

ABI-009 is a mTOR inhibitor. Published data have suggested the importance of the mTOR biological pathway in the development of PAH. Published preclinical studies have shown that an mTOR inhibitor can reverse or control the disease, including the scarring in the small arteries that are indicated in PAH, but this has not been proven in patients with PAH.

For more information

For more information regarding the study please visit the ClinicalTrials.gov website by following the link below:


The study, entitled “A phase 1 clinical trial of ABI-009, an mTOR inhibitor, for patients with severe pulmonary arterial hypertension (PAH)” is listed on the Pulmonary Hypertension Association (PHA) website:


If you are interested in participating in the study the contact details of your nearest study center can be found by searching the study locations.