Editor’s Take: The partnership may provide a new option for a disorder that is a leading cause of intellectual disability.
Enthorin Therapeutics announced an option-to-license agreement it entered with Rumi Therapeutics, for a candidate for Fragile X syndrome treatment.
ENT-7340, an Investigational New Drug (IND)-enabled, highly potent, selective small-molecule inhibitor, will be advanced by Rumi as RTX-7340.
In the deal, Enthorin receives an upfront payment—exact terms undisclosed—milestones, and an option exercise fee. Rumi assumes responsibility for RTX-7340’s development. In the initial stages of that development, Enthorin will assist Rumi with scientific and technical expertise.
What is Fragile X?
Fragile X syndrome is a genetic neurodevelopmental disorder caused by changes in the FMR1 gene. The condition reduces or eliminates production of functional FMRP, a protein that has relevance to brain development.
As such, Fragile X is a leading inherited cause of intellectual disability, affecting learning, language, attention, behavior, and social function. Fragile X syndrome affects both males and females, and has substantial variation in its severity.
To treat this, ENT-7340 selectively inhibits PDE2A, an enzyme regulating both cAMP and cGMP (cyclic AMP and GMP, respectively) pathways. These each have a part to play in neuronal function and synaptic plasticity.
The program builds on Enthorin’s previous work implicating disrupted cyclic nucleotide signaling and its role in Fragile X syndrome.
Enthorin Founder Talks Rumi Deal
Enthorin Co-Founder and Chief Scientific Officer Marco Peters, PhD, points to ENT-7340 as representative of years of discovery and development.
“Rumi brings distinctive capabilities for evaluating therapeutics in human cellular and organoid models,” Peters said. “[It’s] an opportunity to further validate the biology of RTX-7340 in human systems and explore its mechanism of action … We are excited about this agreement and look forward to supporting Rumi during the initial development phase.”