Study site selection and finalization of clinical trial agreement final step toward initiation of RVL-001 proof-of-concept clinical studies early next year

BOSTON, Oct. 24, 2024 /PRNewswire/ — Unravel Biosciences, Inc., (“Unravel”), an AI-enabled therapeutics company established to advance drugs for complex diseases, and the clinical research unit of the PECET of the University of Antioquia, certified in Good Clinical Practices by the Colombian Health Regulatory Authority (“INVIMA”) and located in in Medellín, Colombia, today announced the finalization of a clinical trial agreement enabling PECET to be the designated study site for Unravel’s RVL-001 proof of concept clinical trials for Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS).

Unravel’s proprietary BioNAV drug discovery platform identified RVL-001 as a potentially promising therapeutic drug for Rett Syndrome and PTHS. Building on previously announced initiatives by Unravel to initiate a proof-of-concept clinical trials of RVL-001 in Colombia for RTT as well as a collaboration by the Pitt Hopkins Research Foundation (PTHF) to conduct an extension  study of RVL-001 in PTHS, today’s agreement with PECET in Colombia formally establishes the clinical infrastructure for Unravel to rapidly evaluate BioNAV identified drugs in patients.

“We are excited to collaborate with the highly skilled PECET team to accelerate therapeutics development for rare disease patients,” said Richard Novak, Ph.D., Unravel Co-Founder and CEO. “By conducting multiple clinical trials in parallel at a single clinical trial site enables faster and less costly drug development to support more patients with unmet needs.”

RTT and PTHS are both rare neurogenetic disorders starting in early childhood that lead to debilitating cognitive, motor and autonomic disability.  Despite one approved treatment for RTT, there remains a significant unmet need for novel treatments having meaningful efficacy and acceptable safety and tolerability.  There are no known treatments for PTHS.  In addition to Unravel’s RVL-001 program for RTT and PTHS, the company has also initiated development work on RVL-002, a first-in-class novel molecule for Rett syndrome.

Initiation of patient dosing for the RVL-001 trial in RTT and extension trial in PTHS are both anticipated in early 2025, with study startup activities including clinical trial material manufacturing and patient recruitment initiated earlier this year as previously announced. 

“We are pleased to be the designated clinical trial center for Unravel, it is a great challenge that we will take on with great enthusiasm to contribute to the search for solutions for this type of disease,” said Iván Darío Vélez, Professor Emeritus of the University of Antioquia and founder of PECET. “PECET has extensive expertise conducting clinical studies with international  biopharma sponsors including US to support worldwide registration and approval and we look forward to supporting Unravel in their development efforts”.

About Unravel Biosciences

Unravel Biosciences revolutionizes drug development, uncovering disease-modifying targets from patient data powered by AI systems biology computation, rapid in vivo screening, and clinical validation of discovered targets with unprecedented efficiency. Unravel leverages its proprietary BioNAV foundational model of human health combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases. Unravel’s platform has led to four clinical programs. Unravel’s platform developed RVL001, a novel formulation to treat neurodevelopmental disorders, 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 using existing drugs as a key stepping stone to validating novel targets and mechanisms in real patient populations. www.unravel.bio and www.rareshift.org

SOURCE Unravel Biosciences, Inc.

WANT YOUR COMPANY’S NEWS FEATURED ON PRNEWSWIRE.COM?

440k+
Newsrooms &
Influencers

9k+
Digital Media
Outlets

270k+
Journalists
Opted In