Plans for initial trial of oral RVL001 in Rett syndrome patients advancing

Unravel Biosciences reports 'positive' FDA feedback for study to launch this year

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by Patricia Inácio, PhD |

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The U.S. Food and Drug Administration (FDA) has given “positive” feedback to Unravel Biosciences‘ regarding a proof-of-concept study for RVL001, its investigational therapy for Rett syndrome, the company stated.

Following a pre-investigational new drug (pre-IND) meeting, Unravel received a written response from the FDA providing guidance for the filing of a future IND application that will request clearance to start a trial in Rett patients later this year.

Patient enrollment will be conducted in partnership with TMA Precision Health, a rare disease health platform.

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“We are pleased to have received positive feedback from FDA on our proposed RVL001 clinical study in Rett Syndrome,” Richard Novak, PhD, co-founder and CEO of Unravel, said in a press release. “The successful completion of this key development milestone provides a great validation of Unravel’s ability to rapidly move promising therapies into the clinic, much faster than most conventional approaches to drug development.”

A pre-IND meeting is held to help in planning drug development, with discussions covering regulatory requirements for an IND application. Early interactions with the FDA can help to prevent potential clinical hold issues, according to the agency.

RVL001 is a small molecule oral therapy, Unravel reports. The compound or compounds in the experimental treatment and its target have not been disclosed.

Unravel is working to develop personalized therapies using RNA network analysis.

Scientists at Unravel first analyze patient’s transcriptomics — a global view of how genes are “turned on or off” — to identify specific gene activity signatures. Then, using a proprietary gene interaction network platform called BioNAV and a database of more than 40,000 compounds, they identify potential treatments for a condition.

“RNA tells us exactly what’s going on in the disease in that patient at that time. But rather than look at any one gene, our proprietary model of human health allows us to uncover network-level effects,” the company stated on a webpage.

Promising candidates are screened using Unravel’s frog disease models to confirm that they interact with their intended target, and safety and other checks are conducted on that target, it added.