Target enrollment of 15 Rett patients and 5 Pitt Hopkins patients, with study completion anticipated in the first quarter of 2027.
BOSTON--(BUSINESS WIRE)--#AIxBio--Unravel Biosciences, Inc., ("Unravel"), a clinical stage therapeutics company established to advance drugs for complex diseases through its Predictable Medicine™ platform, today announced the first study participants have been dosed in Unravel’s RVL-001 clinical trials for Rett syndrome (“RTT”) and Pitt Hopkins syndrome (“PTHS”).
RVL-001, a proprietary formulation of vorinostat, targets a novel therapeutic mechanism which will be clinically evaluated for the first time in these studies. In addition to Unravel’s RVL-001 program for RTT and PTHS, the company has also initiated development work on RVL-002, a first-in-class novel molecule for Rett syndrome. Unravel's proprietary BioNAV™ drug discovery engine established a dynamic transcriptome network profile, called “Living Molecular Twins,” to identify RVL-001 as a potentially promising therapeutic drug for Rett Syndrome and PTHS.
The RVL-001 trials are being conducted at the Universidad de Antioquia’s Group for Clinical Trials of PECET (“GIC-PECET”), a clinical study center of excellence and designated INVIMA clinical trial site in Medellin, Colombia. Both RVL-001 proof of concept studies are designed as placebo-controlled, “n-of-1” trials with a target enrollment of 15 patients with RTT and 5 patients with PTHS. More information can be found at https://clinicaltrials.gov/study/NCT07150013 and https://clinicaltrials.gov/study/NCT07150026.
"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."
RTT and PTHS are both rare neurogenetic disorders starting in early childhood that lead to debilitating cognitive, motor and autonomic disability. Despite one approved treatment for RTT, there remains a significant unmet need for novel treatments having meaningful efficacy and acceptable safety and tolerability. There are no known treatments for PTHS.
"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.
About Unravel Biosciences
Unravel Biosciences is a clinical-stage therapeutics company, integrating AI systems biology computation with rapid clinical validation of discovered targets. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, patient stratification, and clinical validation to find treatments for complex diseases using a systematic, data-driven approach called Predictable Medicine™. Unravel's platform identified RVL002, a first-in-class new small molecule targeting mitochondrial metabolism having multiple clinical applications including neurodegenerative and metabolic disorders, and RVL069, a molecule targeting a novel mechanism to treat demyelination. The rareSHIFT™ program provides platform and proprietary datamine access to partners to accelerate and clinically derisk therapeutics. www.unravel.bio and www.rareshift.org
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