NEW YORK–(BUSINESS WIRE)–Rumi Therapeutics, a drug-development company within Rumi Scientific Holdings, today announced an option to license agreement with Enthorin Therapeutics to advance RTX-7340 (ENT-7340), an investigational phosphodiesterase 2 (PDE2) inhibitor, for the treatment of Fragile X syndrome (FXS). The agreement brings an IND-ready therapeutic candidate into Rumi Therapeutics’ pipeline and represents a significant milestone in Rumi Scientific Holdings’ expansion from discovery-stage research toward clinical development.

Rumi Scientific Holdings brings together Rumi Therapeutics and Rumi Scientific, whose proprietary human organoid platform supports disease modeling and therapeutic discovery. The RTX-7340 program combines an extensive preclinical development package in Fragile X models, including human organoid studies and evaluation of translational neurophysiological endpoints. Rumi Scientific will explore the relevance of PDE2 inhibition in additional neurodevelopmental disorders.

“This agreement marks an important step in building Rumi Therapeutics into a clinical-stage company. With RTX-7340, we are combining an IND-ready asset with insights generated through Rumi Scientific’s human organoid platform and a development strategy centered on translation to patients. Fragile X is our initial focus, with opportunities to investigate the relevance of this biology across other neurodevelopmental disorders.”

Ignacio Muñoz-Sanjuán, PhD
CEO, Rumi Therapeutics

“We are pleased to partner with Rumi Therapeutics to advance RTX-7340 toward clinical evaluation in Fragile X syndrome. Rumi Scientific’s human organoid capabilities complement the preclinical development work supporting the candidate and create an opportunity to deepen our understanding of its therapeutic potential. Together, we aim to translate that scientific foundation into a focused clinical development program.”

J. Guy Breitenbucher, Ph.D.
Managing Partner, Co-Founder, Enthorin Therapeutics

Connecting human disease biology to therapeutic development

PDE2A activity is increased in Fragile X animal models, and its pharmacological inhibition can improve dendritic spine maturation, synaptic plasticity, social and cognitive endpoints in Fragile-X models. RTX-7340 studies in Fmr1-knockout mice have demonstrated correction of disease-associated EEG abnormalities following PDE2 inhibition, providing a basis for developing a translational biomarker strategy. Rumi Therapeutics intends to use these findings to inform the selection of neurophysiological measures for clinical evaluation, connecting drug exposure and target occupancy with measurable effects on brain activity and, ultimately, determine whether those effects are associated with benefits for individuals living with Fragile X syndrome.

RTX-7340 is investigational, and its human safety and efficacy in Fragile X syndrome have not been established

“A central challenge in Fragile X drug development is connecting a treatment’s effects on disease biology with outcomes that matter to patients. Combining human organoid research with neurophysiological measures creates an opportunity to build that connection more deliberately. A PDE2 program developed around this framework could help us evaluate both whether the drug affects relevant brain function and whether those effects translate into clinical benefit.”

Craig Ericksson, Director, Fragile X Syndrome Research and Treatment Center
Cincinnati Children’s Hospital

About Fragile X Syndrome

Fragile X syndrome is a genetic neurodevelopmental disorder caused by changes in the FMR1 gene that reduce or eliminate production of functional FMRP, a protein important for brain development. It is a leading inherited cause of intellectual disability and can affect learning, language, attention, behavior, and social functioning.

About Rumi Scientific Holdings

Rumi Scientific Holdings includes Rumi Therapeutics and Rumi Scientific. Rumi Therapeutics develops therapies for neurological and neurodevelopmental disorders, while Rumi Scientific uses proprietary human organoid technology to study disease mechanisms and support therapeutic discovery.

About Enthorin Therapeutics

Enthorin Therapeutics is a clinical stage pharmaceutical company founded with the goal of developing innovative therapeutics that target brain circuit dysfunction to improve quality of life for patients, families, and communities. Learn more at www.enthorin.com.

About Liberi Group

Rumi Scientific was supported in its engagement with Enthorin Therapeutics by Liberi Group, a global life sciences business development consultancy serving as Rumi Scientific’s BD advisor. Liberi Group is a global business development and strategic consultancy focused on the life sciences and biotech industries. For more information, please visit www.liberigroup.com.