Unravel Biosciences and Pitt Hopkins Research Foundation Announce Collaboration to Conduct Clinical Study of RVL-001 in Pitt Hopkins Patients

2024-06-11

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Pitt Hopkins Research Foundation to fund proof of concept clinical trial in Colombia to assess the efficacy of vorinostat (RVL-001) in patients with Pitt Hopkins Syndrome (PTHS) BOSTON, June 11, 2024 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company established to advance drugs for complex diseases, and the Pitt Hopkins Research Foundation (the "Foundation") today announced a collaboration to conduct a proof of concept clinical trial of vorinostat (RVL-001) in patients with Pitt Hopkins Syndrome (PTHS). Under the terms of the collaboration, the Foundation will fund the clinical trial conducted by Unravel at its study site in Colombia and Unravel will retain rights for further development and commercialization. Unravel's proprietary BioNAV™ drug discovery platform identified RVL-001 as a potentially promising therapeutic drug for PTHS. Building on a previously announced initiative by Unravel to initiate a proof-of-concept clinical trial of RVL-001 in Colombia for Rett Syndrome, the PTHS study will leverage the clinical study infrastructure in Colombia set up by Unravel for rapid evaluation of BioNAV™ identified drugs in patients. "I am pleased to announce this important collaboration with the Pitt Hopkins Research Foundation," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "A key part of our mission is to rapidly advance promising drugs into clinical testing in multiple orphan disorders, and our partnership with the Foundation will allow us to evaluate the benefit of RVL-001 in this high unmet need patient group." PTHS is a rare genetic disorder of males and females affecting the TCF4 gene in chromosome 18. PTHS is considered an Autism Spectrum Disorder and is characterized by developmental delay, severe cognitive impairment, recurrent seizures/epilepsy, breathing disorders, gastrointestinal issues, lack of speech, and distinctive facial features. There are no known treatments for PTHS. While the overall prevalence of PTHS is not fully known, estimates currently range from 1 in 34,000 to 1 in 300,000 individuals. "In the short time we've been working with Unravel, they've proven that they work with the urgency necessary to develop treatments for our patient population," said Audrey Davidow Lapidus, President of the Pitt Hopkins Research Foundation. "Unravel has put together this trial in record time and we are so grateful- and hopeful.". 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 About Pitt Hopkins Research Foundation The mission of the Pitt Hopkins Research Foundation (PHRF) is to support research dedicated to finding a treatment, and an eventual cure for Pitt Hopkins Syndrome and other similar disorders. The PHRF is also dedicated to supporting the Pitt Hopkins community with resource recommendations, parental support and the latest medical information. For more information, go to

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