Apabetalone is well tolerated and lowers pulmonary vascular resistance in patients with pulmonary arterial hypertension (PAH), according to the results of a pilot study called APPRoAcH-p.

These findings set the stage for a larger multicenter phase 2 trial to further assess the efficacy of apabetalone in patients with PAH.

PAH is a rare and progressive disease characterized by the narrowing and thickening of the pulmonary arteries, causing increased resistance to blood flow. Research has shown that bromodomain-containing protein 4 (BRD4) plays a key role in PAH pathology and progression. Moreover, inhibiting BRD4 can reverse PAH in animal models of the disease.

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Apabetalone is an oral available BRD4 inhibitor created by Resverlogix Corp. designed to affect disease-causing biological processes.

Read more about PAH therapies

The main objective of the pilot study was to extend the preclinical findings on the roles of epigenetics and DNA damage and on BRD4 inhibition as a potential therapy for PAH. Seven patients with PAH were administered 100 mg of apabetalone twice a day for 16 weeks alongside their existing standard of care treatments. 

The primary endpoint was any change in pulmonary vascular resistance. Secondary endpoints included changes in other metrics of cardiac function, quality-of-life measures, and plasma biomarkers.

The results showed that apabetalone was well tolerated and all patients completed the trial. Moreover, the trial met its primary endpoint, with apabetalone lowering pulmonary vascular resistance.

“The study successfully achieved its primary objective of confirming the feasibility of the clinical study design and demonstrated encouraging results of apabetalone treatment in patients with PAH,” Steeve Provencher, MD, MSc, FRCPC, the director of pulmonary research at the Institute of Cardiology and Respirology of Quebec at Laval University and the lead investigator and sponsor of the trial, said in a press release. “This strongly sets the ground for the planned larger double-blind study.”


Resverlogix announces apabetalone meets primary endpoint in a pulmonary arterial hypertension pilot study. News release. Resverlogix Corp.; September 8, 2021.

Apabetalone for pulmonary arterial hypertension: a pilot study (APPRoAcH-p). ClinicalTrials.gov. August 31, 2018. Updated August 30, 2021. Accessed September 22, 2021.