Proof-of-concept trial for Rett treatment expected this summer
Pending regulatory OK, Colombia study of RVL001 to enroll 15 patients
Unravel Biosciences is asking authorities in Colombia to allow the launch — which could be as early as this summer, if cleared — of proof-of-concept clinical trials in the country to test RVL001, its treatment candidate for Rett syndrome.
The company has submitted an application seeking regulatory clearance for a trial of RVL001, involving Rett patients, at the Universidad de Antioquia’s Center for Technological Development in Medellin.
The trial is expected to enroll 15 people with Rett syndrome and will test the experimental therapy against a placebo, according to a company press release. No further details have been provided by Unravel.
Richard Novak, PhD, the company’s cofounder and CEO, said the trial clearance request “represents an important milestone [toward] Unravel’s evolution into a clinical-stage company.”
Developer using AI to ID novel Rett treatment targets
Unravel said it plans to use artificial intelligence (AI) techniques for trial analyses of genetic activity. These analyses will be paired with validation in preclinical models to identify novel treatment targets, per the company, which previously announced a partnership to supply RVL-001 for clinical testing.
RVL-001 is a formulation of vorinostat, a drug approved in the U.S. under the name Zolinza to treat skin problems due to cancer — specifically ones caused by a cancer type known as cutaneous T-cell lymphoma. Vorinostat belongs to a class of therapies called histone deacetylase inhibitors.
Histones are proteins that help to organize a cell’s DNA; strands of DNA wrap around histones sort of like thread wrapped around a spool. DNA can be wrapped tightly or more loosely around histones, and this alters the activity of genes that are contained within the DNA.
Per the company, histone deacetylases are a type of enzyme that helps to control how tightly DNA is wrapped around histones. By blocking these enzymes, histone deacetylase inhibitors can act to modulate the activity of genes within cells.
We look forward to [an] expedited review of our study applications and are prepared to start patient dosing soon after we receive [regulatory] approval.
The company is also developing another therapy, RVL002, for Rett syndrome. That treatment candidate targets the metabolism of mitochondria, which are the cellular structures responsible for producing energy.
Unravel also submitted an application asking for permission to start clinical testing of RVL001 in Pitt Hopkins syndrome, which, like Rett syndrome, is a rare genetic disorder marked by issues with development.
Both applications were submitted under a pilot program from the National Food and Drug Surveillance Institute (INVIMA) that aims to accelerate development of treatments designed to fill unmet needs in rare diseases. This means that Unravel’s applications will get priority review, however no time frame was specified by the company.
“We look forward to INVIMA’s expedited review of our study applications and are prepared to start patient dosing soon after we receive [regulatory] approval,” Novak said.
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