BOSTON — Unravel Biosciences said the first patients have been dosed in proof-of-concept clinical studies of RVL-001 for Rett syndrome and Pitt Hopkins syndrome, two rare neurogenetic disorders that begin in early childhood.
The Boston-based clinical-stage therapeutics company said the placebo-controlled studies are targeting enrollment of 15 patients with Rett syndrome and five patients with Pitt Hopkins syndrome. Study completion is expected in the first quarter of 2027.
RVL-001 is a proprietary formulation of vorinostat and is designed to target a novel therapeutic mechanism being clinically evaluated for the first time in the studies. Unravel said its BioNAV drug discovery engine identified RVL-001 as a potentially promising therapeutic for both Rett syndrome and Pitt Hopkins syndrome through a dynamic transcriptome network profile the company calls “Living Molecular Twins.”
“Dosing our first patients in our RVL-001 trials is an important milestone for our company and for Rett syndrome and Pitt Hopkins syndrome patients,” said Richard Novak, Ph.D., Unravel Co-Founder and CEO. “Our ability to rapidly identify a promising therapeutic for these two debilitating diseases and in less than two years progress into clinical trials clearly highlights the remarkable capability of our BioNAV™ platform to hasten drug discovery and evaluation.”
The studies are being conducted at Universidad de Antioquia’s Group for Clinical Trials of PECET, known as GIC-PECET, in Medellin, Colombia. The center is a designated INVIMA clinical trial site.
Unravel said both RVL-001 studies are designed as placebo-controlled “n-of-1” trials. The company has also started development work on RVL-002, a first-in-class novel molecule for Rett syndrome.
Rett syndrome and Pitt Hopkins syndrome can cause serious cognitive, motor and autonomic disabilities. Unravel said there remains a significant unmet need for new Rett syndrome treatments with meaningful efficacy and acceptable safety and tolerability, despite one approved therapy. The company said there are no known treatments for Pitt Hopkins syndrome.
“We are pleased to be able to initiate these important clinical studies for Rett and Pitt Hopkins patients in Colombia, for the families of the patients, this study is a silver lining. Our study site has been actively preparing to work on these studies for the past several months and our clinical team looks forward to supporting the trials’ progress,” said Dr. Carolina Lesmes, Principal Investigator, GIC-PECET.
