Enthorin Therapeutics has signed an option-to-license agreement with Rumi Therapeutics to advance ENT-7340 for Fragile X syndrome (FXS), as well as other intellectual and developmental disabilities.
Under the deal, Rumi Therapeutics will develop the therapy, renaming it RTX-7340.
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Enthorin Therapeutics will receive an upfront sum, an option exercise fee, and milestone payments.
The company is also eligible for further payments linked to development, regulatory, and commercial targets, alongside royalties on future net product sales.
It will also contribute scientific and technical expertise to assist during the initial stages of the work.
ENT-7340 is an investigational new drug (IND) enabled small molecule that selectively inhibits phosphodiesterase 2A (PDE2A).
It controls cyclic adenosine monophosphate (cAMP) and cyclic guanosine monophosphate signalling pathways (cGMP), which influence neuronal function and synaptic plasticity.
FXS is an inherited neurodevelopmental disorder stemming from Fragile X messenger ribonucleoprotein 1 (FMR1) gene alterations that reduce or prevent the generation of functional FMRP, a protein essential for brain development.
The condition causes varying degrees of cognitive, behavioural, linguistic, and sensory difficulties in both males and females.
Enthorin Therapeutics co-founder and chief scientific officer Marco Peters said: “ENT-7340 represents years of discovery and development work aimed at translating the biology of PDE2 inhibition into effective therapeutics for the treatment of neurodevelopmental disorders such as Fragile X.
“Rumi brings distinctive capabilities for evaluating therapeutics in human cellular and organoid models, providing an opportunity to further validate the biology of RTX-7340 in human systems and explore its mechanism of action in FXS and additional indications.
“We are excited about this agreement and look forward to supporting Rumi during the initial development phase as they advance the programme toward clinical development.”
Rumi Therapeutics, an operating company under parent firm Rumi Holdings alongside Rumi Scientific, plans to advance the therapy into clinical testing by drawing on human organoid disease models.
Enthorin Therapeutics developed the programme with research support from the FRAXA Research Foundation.
