CONNECTA highlights Fragile X data as Phase 2a trial advances
CONNECTA Therapeutics will present preclinical data supporting its Phase 2a Fragile X syndrome therapy CTH120 at the FENS Forum 2026, highlighting improvements in cognition, behaviour and neuronal connectivity.
CONNECTA Therapeutics and the Centre for Genomic Regulation (CRG) will present new preclinical findings on CTH120, the company’s lead neuroplasticity modulator, at the FENS Forum 2026 in Barcelona next week.
The poster will showcase data supporting the therapeutic potential of CTH120 in Fragile X syndrome (FXS), a rare inherited neurodevelopmental disorder with no approved disease-modifying treatments. The programme is already recruiting adult males with FXS into a Phase 2a clinical trial following positive Phase 1 safety and tolerability results.
According to the company, preclinical studies demonstrated restoration of cognitive and behavioural deficits in Fmr1 knockout mouse models of Fragile X syndrome. Researchers also reported normalisation of dendritic spine density and morphology, findings that support the proposed mechanism of action targeting the TrkB (NTRK2) receptor, which plays an important role in neuroplasticity and neuronal connectivity.
The studies evaluated both acute and chronic oral administration of CTH120 using in vivo and ex vivo models to assess cognitive, behavioural and synaptic outcomes.
Mara Dierssen, group leader at the Centre for Genomic Regulation, said: “Presenting CTH120 at FENS Forum 2026 provides an important opportunity to highlight the scientific rationale behind our neuroplasticity-focused approach in Fragile X syndrome and other neurodevelopmental disorders.”
The company believes modulation of the TrkB receptor may help restore disrupted neuronal architecture associated with Fragile X syndrome and potentially other neurodevelopmental disorders.
Josep Prous, co-founder and chief scientific officer at CONNECTA Therapeutics, added: “CTH120 targets TrkB (NTRK2) receptor, a key mediator of neuroplasticity, with the goal of restoring disrupted neuronal architecture underlying the cognitive and behavioral features of FXS. We look forward to engaging with the neuroscience community as we continue to advance this program.”
Fragile X syndrome is the most common inherited cause of intellectual disability and is associated with a range of cognitive, behavioural and developmental challenges. Current treatment options focus largely on symptom management, leaving a significant unmet need for therapies that address the underlying biology of the condition.
CONNECTA said CTH120 is designed to restore neuronal connectivity by modulating neuroplasticity, representing a novel therapeutic approach that differs from existing symptomatic treatments.
The preclinical findings will be presented during a poster session at the FENS Forum 2026 on 9 July in Barcelona as the company continues to progress the programme through Phase 2a clinical development.




