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: Contact@Unravel.bio.
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SOURCE Unravel Biosciences, Inc.