Dosing underway in clinical trial of Rett syndrome therapy RVL-001
Participants being recruited at site in Colombia; results expected in early 2027
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Unravel Biosciences announced it has begun dosing in a proof-of-concept clinical trial evaluating its experimental therapy, RVL-001, in people with Rett syndrome.
Richard Novak, PhD, Unravel’s co-founder and CEO, said this marks “an important milestone for our company and for [people with] Rett syndrome” in a company press release.
The Phase 1 study (NCT07150013) aims to enroll 15 girls and young women with Rett syndrome, ages 6 to 21, who have a confirmed MECP2 gene mutation and are on stable treatment. All participants will initially receive a placebo for four weeks, then a low dose of RVL-001 (80 mg/m2/day) for eight weeks, followed by a higher dose (160 mg/m2/day) for another eight weeks.
The study’s main goals are to evaluate the safety and tolerability of RVL-001 in people with Rett syndrome and to assess the therapy’s effects on genetic activity in patients’ cells. The trial is currently enrolling participants at a site in Colombia, and results are expected in early 2027.
In parallel with the Rett trial, Unravel has also launched a similar study to test RVL-001 in five people with Pitt-Hopkins Syndrome, another rare genetic disease marked by developmental abnormalities.
“We are pleased to be able to initiate these important clinical studies for Rett and Pitt Hopkins patients in Colombia,” said Carolina Lesmes, MD, principal investigator. “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.”
Vorinostat blocks activity of key proteins
Rett syndrome is a rare genetic disorder caused mainly by mutations in the MECP2 gene, which normally helps coordinate the activity of other genes in cells. RVL-001 is a formulation of vorinostat, a drug approved in the U.S. under the name Zolinza as a treatment for skin problems associated with a specific type of cancer called cutaneous T-cell lymphoma.
Vorinostat works by blocking the activity of histone deacetylases, a class of proteins that regulate cellular genetic activity by modulating how DNA is physically packaged within the cell. Unravel identified this drug as a potential treatment for Rett and Pitt Hopkins using its BioNAV platform, which the company defines as a network model of human health that “predicts molecules to restore a disease back to health.”
“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,” Novak said.
Unravel is also developing a second experimental treatment for Rett syndrome, among other indications, called RVL-002. According to the company, RVL-002 targets cellular energy-generating structures called mitochondria.
