Unravel Biosciences Announces Submission of RVL-001 Clinical Study Applications for Rett Syndrome and Pitt Hopkins Syndrome to Colombian Health Regulatory Authority

Submissions follow Ethics Committee Approvals for Both Studies Received Earlier This Month and Have Been Accepted for INVIMA Priority Review Under Fast Track Program for Rare Disease

BOSTON, April 29, 2025 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company established to advance drugs for complex diseases, today announced the submission of clinical study applications for Unravel's RVL-001 proof of concept clinical trials for Rett syndrome ("RTT") and Pitt Hopkins syndrome ("PTHS") in Colombia. Both applications have been submitted under a pilot program by the Colombian Health Regulatory Agency ("INVIMA"), allowing for priority review and fast track approval for orphan and high unmet need diseases.

Unravel anticipates study initiation in the early summer for both programs at the Universidad de Antioquia's Center for Technological Development ("PECET"), a clinical study center of excellence and designated INVIMA clinical trial site in Medellin, Colombia. Both RVL-001 proof of concept studies are designed as placebo-controlled, "n-of-1" trials with a target enrollment of 15 patients with RTT and 5 patients with PTHS.

"Today's announcement represents an important milestone towards Unravel's evolution into a clinical-stage company," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "We look forward to INVIMA's expedited review of our study applications and are prepared to start patient dosing soon after we receive INVIMA approval."

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. Unravel's proprietary BioNAV™ drug discovery platform identified RVL-001 as a potentially promising therapeutic drug for Rett Syndrome and 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.

"The Rett and Pitt Hopkins Syndrome communities, including families, caregivers and treating physicians, have been eagerly awaiting the opportunity to participate in meaningful clinical research to find potentially beneficial therapeutics for their loved ones." said Dr. Carolina Lesmes, Pediatric Neurologist and Principal Investigator of the RVL-001 studies. "We are ready to initiate these two important studies to assess the safety and potential efficacy of RVL-001."

About Unravel Biosciences

Unravel Biosciences is a therapeutics company founded on the principle that biological networks inform clinical outcomes. By using primary patient data with AI systems biology computation, rapid in vivo screening, and clinical validation of discovered targets, Unravel enables drug development with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ platform combining target and drug prediction with patient stratification by efficacy and toxicity response to find treatments for complex diseases. Unravel's platform has led to two clinical trials starting in 2025. Unravel's platform developed RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027 and RVL068, molecules targeting two novel mechanisms 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

SOURCE Unravel Biosciences, Inc.