AMO Pharma Limited, a US-based privately held clinical-stage specialty biopharmaceutical company involved in rare genetic disorders with limited or no treatment options, on Monday announced a licence agreement with Population Health Research Institute (PHRI), a joint global health research institute of McMaster University and Hamilton Health Sciences, and Venca Research Inc.

The agreement is intended to support the development and potential manufacturing, and commercialisation of AMO-02 in arrhythmogenic right ventricular cardiomyopathy (ARVC). Recruitment is now underway with the first patient randomised in the PHRI TaRGET Phase 2 proof-of-concept clinical trial evaluating AMO-02 for the treatment of ARVC. AMO Pharma says that the study will be the largest clinical trial of a therapeutic ever conducted in this rare, heritable cardiomyopathy.

The TaRGET study is a randomised, double-blind, placebo-controlled trial evaluating AMO-02 in patients with genotype-positive ARVC. The primary endpoint is the change from baseline in mean premature ventricular contractions per 24 hours, measured by seven-day Holter monitoring compared to placebo at six months of treatment. Secondary endpoints include right ventricular strain on echocardiography, frequency of implantable cardioverter-defibrillator therapies, and episodes of sustained ventricular tachycardia.

Under the terms of the agreement, AMO Pharma will work closely with PHRI on regulatory interactions and study oversight.

AMO Pharma anticipates first data from the trial in the second quarter of 2027.