Unravel Biosciences and PECET to Collaborate on Clinical Trials for Rett and Pitt Hopkins Syndromes in Colombia

Study site selection and finalization of the clinical trial agreement mark a significant step forward for the initiation of RVL-001 proof-of-concept clinical studies early next year.

BOSTON, October 24, 2024 – Unravel Biosciences, Inc., an AI-powered therapeutics company dedicated to developing treatments for complex diseases, in partnership with the clinical research unit of PECET at the University of Antioquia, has finalized a clinical trial agreement. This agreement designates PECET, certified in Good Clinical Practices by the Colombian Health Regulatory Authority (INVIMA) and situated in Medellín, Colombia, as the study site for Unravel's RVL-001 clinical trials for Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS).

The BioNAV™ drug discovery platform, developed by Unravel, identified RVL-001 as a promising therapeutic candidate for RTT and PTHS. Building on prior initiatives to begin proof-of-concept clinical trials of RVL-001 in Colombia for RTT and collaborating with the Pitt Hopkins Research Foundation (PTHF) to extend an RVL-001 study in PTHS, this agreement with PECET formally establishes the necessary clinical infrastructure. This infrastructure will enable Unravel to swiftly evaluate drugs identified by BioNAV™ in patient trials.

Richard Novak, Ph.D., Co-Founder and CEO of Unravel, expressed enthusiasm about working with PECET’s team, stating, "Collaborating with PECET's highly skilled team allows us to accelerate the development of therapeutics for patients with rare diseases. Conducting multiple clinical trials simultaneously at a single site helps expedite drug development, making it more cost-effective to support patients with unmet needs."

RTT and PTHS are rare neurogenetic disorders that begin in early childhood, resulting in severe cognitive, motor, and autonomic disabilities. While there is one approved treatment for RTT, there remains a substantial need for new treatments with meaningful efficacy, safety, and tolerability. Currently, no treatments are available for PTHS. Alongside the RVL-001 program for RTT and PTHS, Unravel is developing RVL-002, a novel molecule for treating Rett syndrome.

Patient dosing for the RVL-001 trial in RTT and the extension trial in PTHS is expected to commence in early 2025. Study startup activities, including manufacturing clinical trial materials and patient recruitment, began earlier this year as previously announced.

Iván Darío Vélez, Professor Emeritus at the University of Antioquia and founder of PECET, shared his excitement about the collaboration, stating, "We are honored to be the designated clinical trial center for Unravel. This is a significant challenge that we embrace enthusiastically as we contribute to finding solutions for these diseases. PECET has extensive experience conducting clinical studies with international biopharma sponsors, including those from the US, to support global registration and approval, and we look forward to supporting Unravel’s development efforts."

Unravel Biosciences aims to revolutionize drug development by identifying disease-modifying targets using AI systems biology computation, rapid in vivo screening, and clinical validation of discovered targets. Their BioNAV™ platform integrates target and drug discovery, preclinical screening, and patient stratification to identify treatments for complex diseases. This platform has led to four clinical programs, including RVL001, a novel drug formulation for neurodevelopmental disorders, RVL002, a first-in-class 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.