Repair Biotechnologies, Inc. (https://www.repairbiotechnologies.com/), a biotechnology company developing first-in-class therapies capable of rapidly reducing atherosclerotic plaque, is pleased to announce that it has received positive feedback from a Pre-IND meeting with the U.S. Food and Drug Administration (FDA), an important step forward towards opening an IND to conduct a Phase 1b study of its REP-0003 mRNA therapy in patients with homozygous familial hypercholesterolemia (HoFH), a rare condition of accelerated atherosclerosis.
“We appreciated the FDA reviewers’ alignment with our proposed clinical development program, and their positive feedback on our intended clinical trial design, IND-enabling studies, and drug manufacturing processes,” said Reason, Repair Biotechnologies’ CEO and Co-Founder. “HoFH remains a condition of high unmet need, reduced quality of life, and early mortality from plaque rupture leading to heart attack and stroke. We hope to be able to make a real difference for this patient population.”
The Pre-IND meeting focused on the establishment of appropriate clinical endpoints to demonstrate improved function in this patient population following REP-0003 treatment to reduce atherosclerotic plaque volume, such as assessment of exercise capacity. Familial hypercholesterolemia patients typically exhibit a reduced exercise capacity in addition to the other consequences of accelerated atherosclerosis.
In preclinical studies, REP-0003 has produced significant, reliable regression of atherosclerotic plaque and improved exercise capacity in LDLR-knockout mice. As is the case in human patients, this mouse model for HoFH exhibits a loss-of-function mutation in the low-density lipoprotein receptor (LDLR) gene and consequent impairment of liver metabolism. REP-0003 was developed using Repair Biotechnologies’ proprietary Cholesterol Degrading Platform (CDP), and provides a way to safely break down and eliminate the undruggable target of toxic excess intracellular cholesterol in tissues, with potential application to many conditions of aging and obesity.
