Unravel Announces Collaboration with Vanderbilt University Medical Center to Support Upcoming Multicenter U.S. Clinical Study in Rett Syndrome that Includes RVL-001

News provided by

May 14, 2024, 08:45 ET

Multisite trial run by Vanderbilt will evaluate repurposed FDA-approved drugs as treatment options for Rett syndrome patients, including vorinostat (RVL-001) predicted by Unravel's BioNAV platform.

BOSTON, May 14, 2024 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company established to advance drugs for complex diseases, today announced a collaboration with Vanderbilt University Medical Center (VUMC) to provide support for VUMC's upcoming multisite clinical trial to evaluate three repurposed drugs, including an oral liquid formulation of vorinostat (Unravel's RVL-001), for the treatment of patients with Rett Syndrome.

Vanderbilt's $13 million Department of Defense grant funded study was previously announced by VUMC in January of this year (link below). As per VUMC's announcement, the study is an umbrella trial that will compare three FDA-approved drugs, ketamine, vorinostat and donepezil, to a common placebo. Participants will be randomized equally to one of the four treatment arms for a 16-week treatment to assess safety, tolerability, efficacy and biomarker response.

As part of the collaboration announced today, Unravel will provide analytical and formulation support for the oral liquid vorinostat (RVL-001) clinical test material to be used in the study as well as support to the VUMC clinical and regulatory teams. In exchange for the company's support of the trial, Unravel will have rights to the clinical data generated for vorinostat for its own future development and commercialization purposes.

"We are pleased to announce our collaboration today with VUMC and Dr. Jeff Neul on this landmark study in Rett Syndrome," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "Having RVL-001, a drug prioritized by our BioNAV platform in Rett syndrome, selected by the VUMC team for inclusion in this major study is further validation of the promise and potential of our core platform technology."

Rett syndrome is a rare neurogenetic disorder starting in early childhood and leading to debilitating cognitive, motor and autonomic disability. With a global incidence of one in 10,000 female births, Rett Syndrome represents an orphan disease with widespread impact and only one FDA-approved treatment. Unravel's proprietary drug discovery platform identified RVL-001 as a promising therapeutic for Rett Syndrome, as well as RVL-002, a novel small molecule therapeutic currently being developed.

"Having industry support to help with further development and commercialization of therapies that have therapeutic value in Rett Syndrome identified by this study will be a benefit to patients and the clinical community," said Jeffrey Neul, M.D., Ph.D., Annette Schaffer Eskind Professor, Director of the Vanderbilt University Kennedy Center and the Principal Investigator of the study.

About Unravel Biosciences

Unravel Biosciences is the first rapid prototyping therapeutics company, integrating AI systems biology computation with rapid in vivo screening and clinical validation of discovered targets with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases. Unravel's platform has led to four clinical trials starting in 2024. Unravel's platform developed RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027, a molecule targeting a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform access to foundation and biotech partners to accelerate and clinically derisk therapeutics. www.unravel.bio and www.rareshift.org

For further information, see January 9, 2024 Press Release: "VUMC receives $13 million grant to coordinate multisite clinical trial for Rett syndrome treatment," VUMC News, https://news.vumc.org/2024/01/09/vumc-receives-13-million-grant-to-coordinate-multisite-clinical-trial-for-rett-syndrome-treatment/

Media Contact: [email protected].

SOURCE Unravel Biosciences, Inc.