Unravel Biosciences and Pitt Hopkins Research Foundation to Study RVL-001 in Pitt Hopkins Patients

Unravel Biosciences, Inc. and the Pitt Hopkins Research Foundation have announced a collaborative effort to conduct a clinical trial assessing the efficacy of vorinostat (RVL-001) in individuals with Pitt Hopkins Syndrome (PTHS). The trial, funded by the Foundation and conducted by Unravel in Colombia, aims to explore the potential benefits of RVL-001 for this rare genetic disorder.

Unravel Biosciences utilizes its proprietary BioNAV™ drug discovery platform, which identified RVL-001 as a promising candidate for treating PTHS. The company, known for its rapid prototyping of therapeutics, integrates AI-driven systems biology with in vivo screening and clinical validation to expedite the development of treatments for complex diseases. This collaboration builds on Unravel's prior initiative to test RVL-001 for Rett Syndrome in Colombia, leveraging the established clinical infrastructure for efficient evaluation.

Dr. Richard Novak, Co-Founder and CEO of Unravel, expressed enthusiasm about the partnership, highlighting the company's commitment to advancing drugs for multiple orphan disorders swiftly. He emphasized the importance of assessing RVL-001 in this patient group, which has significant unmet medical needs.

Pitt Hopkins Syndrome is a rare genetic condition affecting the TCF4 gene on chromosome 18. It manifests as an Autism Spectrum Disorder with symptoms including developmental delay, severe cognitive impairment, seizures, breathing issues, gastrointestinal problems, lack of speech, and distinctive facial features. Current estimates suggest its prevalence ranges from 1 in 34,000 to 1 in 300,000 individuals, but there are no known treatments.

Audrey Davidow Lapidus, President of the Pitt Hopkins Research Foundation, praised Unravel's dedication and efficiency in developing treatments for PTHS. She expressed gratitude and hope about the rapid progress in setting up the trial.

Unravel Biosciences stands out for its integration of AI and rapid screening in drug discovery. The BioNAV™ platform combines target and drug discovery, preclinical screening, and patient stratification to identify treatments for complex diseases. This approach has led to the initiation of four clinical trials in 2024. Among its developments are RVL002, targeting mitochondrial metabolism, and RVL027, focusing on a novel mechanism to treat dystonias. The rareSHIFT™ program further enhances Unravel's capabilities, offering platform access to foundation and biotech partners to accelerate the development of therapeutics.

The Pitt Hopkins Research Foundation is dedicated to advancing research for finding treatments and cures for PTHS and similar disorders. The Foundation also provides resources, parental support, and up-to-date medical information to the Pitt Hopkins community.

This collaboration marks a significant step towards addressing the unmet needs of individuals with Pitt Hopkins Syndrome, leveraging advanced AI-driven drug discovery and swift clinical testing to bring potential treatments closer to reality.