News | May 28, 2024

Unravel Biosciences Receives FDA Orphan Drug Designation For Vorinostat (RVL-001) As A Treatment For Rett Syndrome

Initiation of global clinical development program for RVL-001 in Rett syndrome underway, with recently announced Rett studies in the US led by Vanderbilt and in Colombia led by Unravel.

Boston, MA /PRNewswire/ - Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company established to advance drugs for complex diseases, today announced that it has received Orphan Drug Designation from the United States Food and Drug Administration (FDA) for vorinostat (RVL-001) as a treatment for Rett Syndrome.

Unravel's proprietary BioNAV™ drug discovery platform identified RVL-001 as a promising therapeutic for Rett Syndrome. Over the past 18 months, Unravel has moved efficiently and rapidly towards clinical testing of RVL-001 in Rett patients. Last year, Unravel announced that it had received positive pre-IND meeting feedback from the FDA for its proposed proof of concept Rett study. In March of this year, Unravel announced the initiation of RVL-001 clinical trial material manufacturing for use in upcoming Rett syndrome clinical trials in the US and Colombia. And last month, Unravel announced its collaboration with Vanderbilt University on a landmark multicenter US clinical study that will evaluate RVL-001 in Rett syndrome patients.

"Receiving an Orphan Drug Designation from the FDA is a validation of our team's commitment to developing new treatment options that can benefit the lives of patients with rare diseases," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "Today's milestone, along with our recently announced Rett clinical trial collaboration with Vanderbilt and our upcoming POC Rett study in Colombia, illustrates the paradigm-shifting speed by which Unravel can rapidly prototype drugs and evaluate their efficacy in patients."

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, with a continued unmet need for novel treatments having meaningful efficacy and acceptable safety and tolerability. In addition to Unravel's RVL-001 program, the company has also initiated development work on RVL-002, a first-in-class novel molecule for Rett syndrome.

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. and

Source: Unravel Biosciences, Inc.

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