Rare Disease Report

BET Inhibitor Being Developed to Treat Fabry Disease

MAY 30, 2017
Mathew Shanley
There is a new drug being developed to treat Fabry disease.

Apabetalone (RVX-208) is a first-in-class, small molecule selective BET (bromodomain and extra-terminal) inhibitor that may help Fabry disease patients.
Earlier today, Resverlogix announced that Health Canada, Therapeutic Products Directorate has granted approval to proceed with a clinical trial to test apabetalone in Fabry disease patients.
Fabry disease is a rare, X-linked lysosomal storage disease that is the result of a mutation in the GLA gene, causing a deficiency in in the alpha galactosidase-A enzyme responsible for breaking down a fatty substance called globotriaosylceramide (Gb3). In patients, this has led to abnormal deposits of Gb3 in blood vessel walls and tissues throughout the body that are capable of damaging major organs while shortening lifespans.
This study is an open-label, exploratory clinical study to evaluate the safety of patients and effect on key biomarkers of apabetalone in subjects with Fabry disease for up to 16 weeks.
The study’s primary objective is to evaluate the safety and tolerability of apabetalone in patients with Fabry disease, while secondary objectives include evaluating the effect of apabetalone in subjects with Fabry disease as decided by change in key biomarkers including alkaline phosphatase (ALP), high-sensitivity C-reactive protein (hs-CRP), and other recognized markers for chronic kidney disease.
"We are extremely pleased to receive approval for this clinical trial in Canada. Canada possesses some of the world's preeminent experts in the orphan Fabry disease field and a well-established patient registry, “stated Donald McCaffrey, Resverlogix President and CEO.
The study population will consist of two cohorts: Cohort 1, which includes patients with Fabry disease receiving enzyme replacement therapy (ERT), and Cohort 2, which includes patients with Fabry disease not receiving ERT.
“Apabetalone is known to regulate key biomarkers associated with this rare disease and may potentially offer an alternative for patients with this rare disorder,” said McCaffrey.

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