Enthorin Licenses Fragile X Drug Candidate ENT-7340 to Rumi

Enthorin Therapeutics has signed a licensing agreement with Rumi Therapeutics to advance ENT-7340, an experimental drug candidate being developed for Fragile X syndrome and other neurological disorders.

Under the agreement, Rumi Therapeutics will take responsibility for the further development of ENT-7340, which will now be known as RTX-7340. The companies aim to advance the drug towards clinical development and explore its potential as a treatment for Fragile X syndrome.

As part of the agreement, Enthorin will receive an upfront payment, an option exercise fee and milestone payments. The company will also be eligible for additional payments linked to development, regulatory and commercial progress, along with royalties on future net sales.

Enthorin will continue to support the programme by providing scientific and technical expertise during the early stages of development.

ENT-7340 is a small-molecule drug designed to block an enzyme called phosphodiesterase 2A (PDE2A). This enzyme plays a role in regulating two important signalling pathways in the brain, known as cAMP and cGMP. These pathways help control communication between brain cells and support processes such as learning, memory and brain function.

Researchers believe that changes in these signalling pathways may contribute to Fragile X syndrome. By targeting PDE2A, RTX-7340 is designed to help restore normal cellular signalling and improve brain function.

In preclinical studies involving mouse models of Fragile X syndrome, the drug candidate showed activity across several behavioural and brain-function measures. The studies reported improvements in cognitive and autism-related behavioural abnormalities.

The compound has also shown target engagement through PET imaging, providing researchers with additional evidence that the drug reaches and interacts with its intended target in the brain.

Rumi Therapeutics plans to build on these findings using its human cellular and organoid research platforms. These technologies allow researchers to study disease processes in laboratory-grown human tissue models and investigate how potential treatments work.

Fragile X syndrome is a genetic condition caused by changes in the FMR1 gene, which can reduce or prevent the production of a protein called FMRP. This protein is important for normal brain development and function.

The condition is one of the leading inherited causes of intellectual disability. It can affect learning, language, attention, behaviour and social development. Symptoms and their severity can vary between individuals.

The agreement marks another step in Enthorin’s efforts to develop treatments for neurological and neurodevelopmental conditions. The company has also been working on other drug programmes targeting brain signalling pathways, including treatments for Parkinson’s disease and Dup15q syndrome.

With the transfer of ENT-7340 to Rumi Therapeutics, the companies aim to build on the existing research and move RTX-7340 closer to clinical evaluation. However, the drug remains an investigational candidate, and further studies will be needed to establish its safety and effectiveness in people with Fragile X syndrome.