CONNECTA Therapeutics and Centre for Genomic Regulation to Present First-in-Class Program CTH120 for Fragile X Syndrome at FENS Forum 2026
Preclinical data highlight restoration of cognitive, behavioral and synaptic deficits in Fragile X syndrome models
Barcelona, Spain – 2 July 2026 – CONNECTA Therapeutics, a clinical-stage biotech company pioneering first-in-class neuroplasticity modulators to address unmet medical needs in central nervous system (CNS) disorders, today announced that it will present a poster, together with the Centre for Genomic Regulation (CRG), on CONNECTA’s lead, first-in-class program, CTH120, at the FENS Forum 2026, taking place 6-10 July 2026 in Barcelona, Spain.
The presentation entitled “CTH120, a novel treatment for Fragile X syndrome and other neurodevelopmental disorders” will provide details of the preclinical data evaluating the therapeutic potential of CTH120 in Fragile X syndrome (FXS). CTH120 is a first-in-class small molecule targeting TrkB (NTRK2) receptor, a key mediator of neuroplasticity. By modulating neuronal connectivity, it is designed to restore disrupted brain architecture in neurodevelopmental disorders such as FXS. CTH120 is currently recruiting adult males with FXS in the Phase IIa clinical trial (NCT07654114), following the Phase I study demonstrating favorable safety and tolerability.
Development highlights include:
• Restoration of cognitive and behavioral deficits in the Fmr1-KO model, demonstrating improvement in key functional impairments associated with FXS
• Normalization of dendritic spine density and morphology in treated FXS mice models, supporting the TrkB receptor modulation mechanism of action and its relevance to neuronal connectivity and function
• Current status of clinical development of CTH120 to progress as a potential therapy in FXS and other neurodevelopmental disorders
These studies were designed to assess whether modulation of neuroplasticity via TrkB (NTRK2) receptor can improve core disease features, including cognitive, behavioral and synaptic deficits. Using in vivo and ex vivo approaches in the Fmr1-KO mouse model of FXS, the effects of both acute and chronic oral administration of CTH120 were evaluated across a range of behavioral, cognitive and neurobiological endpoints, including dendritic spine structure and morphology.
“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,” said Dr. Mara Dierssen, group leader of CRG.
Dr. Josep Prous, Jr., Co-Founder and CSO of 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.”
Dr. Marta Pascual-Gilabert, Director of Research & Development at CONNECTA Therapeutics, commented, “Fragile X syndrome remains a lifelong condition with significant unmet need, particularly given the lack of approved disease-modifying therapies. Targeting neuroplasticity and the underlying biology of FXS represents a promising approach beyond symptom management, and the continued advancement of CTH120 is an important step in exploring this potential.”
Details of the poster presentation are as follows:
Abstract title: “CTH120, a novel treatment for Fragile X syndrome and other neurodevelopmental disorders”
Abstract number: 4498
Date: 9 July 2026
Session number: 6
Session time: 14:00–17:30 CEST
Poster authors in attendance: 14:00–15:30 CEST
Poster number: PS06-09PM-102
Topic: G. Disorders of the Nervous System; G.15 Neurodevelopmental disorders
Main topic: G.15.b Intellectual disability
Secondary topic: G.19 Rare diseases
Authors: Marta Pascual-Gilabert1,†, Álvaro Fernández-Blanco2,†, Irene Domingo-Pascual1, María Martínez de Lagrán2, Jordi Fàbrega1, Josep Prous1,* and Mara Dierssen2,*
1CONNECTA Therapeutics, S.L., Barcelona, Spain 2 Centre for Genomic Regulation, The Barcelona Institute for Science and Technology, Barcelona, Spain
† Co-authors * Corresponding authors
Abstracts are available via the FENS Forum 2026 website here.
Dr. Marta Pascual-Gilabert, Director of Research & Development at CONNECTA Therapeutics, Dr. Álvaro Fernández-Blanco, Researcher at CRG and Ms. Irene Domingo Pascual, Senior R&D Project Manager at CONNECTA Therapeutics, will be in attendance and are available for meetings.
For more information, please contact:
Optimum Strategic Communications
Zoe Bolt, Katie Flint, Nellie Stephens
Tel: +44 (0) 20 3882 9621
Email: CONNECTA@optimumcomms.com
About CONNECTA Therapeutics
CONNECTA Therapeutics is a clinical-stage biotech company leveraging its small-molecule neuroplasticity modulation platform to address unmet medical needs in central nervous system (CNS) disorders with a focus on pediatric neurodevelopmental conditions. Neuroplasticity is the ability of the nervous system to adapt its activity in response to intrinsic or extrinsic stimuli by reorganizing its structure, functions, and connections, and is impaired in the vast majority of these disorders.
The Company’s lead program, CTH120, is a potential first-in-class disease-modifying treatment for Fragile X syndrome (FXS), a rare and currently untreatable neurodevelopmental disorder that strongly impairs cognition and behavior. CTH120 is a small novel molecule that targets the tropomyosin kinase B (TrkB) receptor, a key mediator in brain development and function. Unlike existing symptomatic treatments, CTH120 aims to restore the underlying neuronal architecture. Backed by strong preclinical research, the Phase I safety data (NCT06480968) and the assessment of neural biomarkers in FXS (NCT06957054), CONNECTA has initiated a Phase IIa trial of CTH120 currently recruiting adult FXS males, and is preparing to advance the program into FXS pediatric population, supported by the FRAXCURE project (co-funded by the European Union, GA101217931).
CONNECTA’s scalable neuroplasticity modulation platform has already yielded additional promising compounds that have shown potential in treating multiple CNS disorders. Preclinical models have demonstrated efficacy for FXS and Rett syndrome, with the potential to address other high-value indications such as Down’s syndrome, DiGeorge syndrome, autism spectrum disorder (ASD), attention deficit hyperactivity disorder (ADHD), and neuropathic pain. For more information, please visit
www.connectatherapeutics.com
About Centre for Genomic Regulation (CRG)
The Centre for Genomic Regulation (CRG) is a renowned international biomedical research center located in Barcelona. Its mission is to discover and promote knowledge to benefit society, public health, and economic prosperity. The center employs more than 400 interdisciplinary scientists focused on understanding the complexity of life, from the genome to the cell and the entire organism. The CRG forms part of the Barcelona Institute of Science and Technology (BIST) and is a CERCA research center of the Government of Catalonia. More information: www.crg.eu
About the FRAXCURE project
FXS is a neurodevelopmental rare disease without a cure affecting around 1.8 M patients worldwide. FXS is responsible for 30% of intellectual disability and cognitive deterioration in both males and females, starting at paediatric age and chronically persisting for life, impacting in learning abilities, attention problems, hyperactivity, autism and social impairments. Early intervention in children is crucial to provide the greatest chance of developing a full range of neurodevelopmental skills, and more opportunities for their social inclusion.
The FRAXCURE project embraces all the activities to confirm CTH120 as a breakthrough disease modifying treatment for the FXS paediatric population. For further information visit the CORDIS website.
Co-funded by the European Union. Views and opinions expressed are, however, those of the author(s) only and do not necessarily reflect those of the European Union or the European Innovation Council and SMEs Executive Agency (EISMEA). Neither the European Union nor the granting authority can be held responsible for them.
