US-based therapeutics company Unravel Biosciences has partnered with the Pitt Hopkins Research Foundation to carry out a proof-of-concept trial of vorinostat (RVL-001), a potential treatment for Pitt Hopkins Syndrome (PTHS).

The drug was identified using Unravel's BioNAV drug discovery platform as a potentially promising therapeutic for PTHS.

The Pitt Hopkins Research Foundation will provide funding for the trial, which Unravel will carry out in Colombia.

Unravel Biosciences holds the right to further develop and commercialise vironistat.

The partnership aims to use the clinical study infrastructure established by Unravel in Colombia, which was initially set up for a Rett Syndrome trial, to expedite the evaluation process for RVL-001 in PTHS patients.

Unravel Biosciences co-founder and CEO Richard Novak said: “I am pleased to announce this important collaboration with the Pitt Hopkins Research Foundation.

“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 ailment that is caused by mutations in the TCF4 gene on chromosome 18.

The condition is categorised as an autism spectrum disorder and is characterised by delays in development, cognitive impairments, seizures, breathing and gastrointestinal issues, absence of speech, and unique facial features.

There are currently no known treatments specifically for PTHS, whose prevalence ranges from one in 34,000 to one in 300,000 individuals.

Pitt Hopkins Research Foundation president Audrey Davidow Lapidus said: “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.

“Unravel has put together this trial in record time and we are so grateful ‒ and hopeful.”