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10/06/2026 - Press release

CONNECTA Therapeutics Initiates Phase IIa Clinical Trial of CTH120, a First-in-Class Neuroplasticity Modulator, in Fragile X Syndrome

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 the initiation of its Phase IIa clinical trial evaluating its lead first-in-class program, CTH120, in adult males with fragile X syndrome (FXS).

CTH120 is a novel small molecule targeting the tropomyosin receptor kinase B (TrkB), a key regulator of neuroplasticity, the brain's ability to form and adapt neural connections. Impaired neuroplasticity is a core pathophysiological feature in neurodevelopmental disorders and contributes to the clinical manifestations observed in conditions such as FXS. FXS is the most common inherited cause of intellectual disability, with an estimated prevalence of approximately 3 per 10,000 individuals.

"Initiating this Phase IIa study represents a significant milestone for CONNECTA," said Jordi Fàbrega, Co?Founder and CEO of CONNECTA Therapeutics. "Building on encouraging Phase I safety data, this trial is designed to further evaluate the safety and tolerability of CTH120 and to generate critical insights into its potential therapeutic benefit by modulating pathways implicated in disrupted neuronal function in FXS. We believe this study could support the development of a novel, disease?modifying approach for FXS and serve as an important proof point for our broader neuroplasticity modulation platform."

The Phase IIa randomized, double?blind, placebo?controlled, parallel?group, multicenter study (EU CT number: 2025?522972?97?00) is designed to evaluate CTH120 administered twice daily versus placebo. The first clinical trial will be conducted in individuals most severely affected by FXS, who are typically adult males due to the X-linked nature of the condition. The primary endpoint is the assessment of safety and tolerability, with secondary endpoints including characterization of pharmacokinetics and evaluation of clinical efficacy. The trial will randomize 30 adult males aged 18-45, in 1:1 ratio to receive CTH120 or placebo. Successful completion of the study will represent an important step toward later?stage clinical development of CTH120.

"FXS has a profound impact on cognitive and behavioral function, severely affecting the quality of life of individuals living with the condition and their families. It remains a condition with significant unmet medical need, with no approved disease?modifying treatments," said Dr. Rafael de la Torre Fornell, PharmD, PhD, Principal Investigator and Study Coordinator at Hospital del Mar Research Institute (HMRIB). "This Phase IIa trial provides an important opportunity to evaluate a promising new mechanism of action and advance clinical understanding of this lifelong condition."

The Phase IIa study will be conducted at Hospital del Mar Research Institute (HMRIB) in Barcelona and Parc Taulí Research and Innovation Institute Foundation (I3PT) in Sabadell, two leading centers with broad expertise in FXS and neurodevelopmental disorders. The study is led by Dr. Rafael de la Torre Fornell, PharmD, PhD, as well as Ana Aldea Perona, MD, PhD (HMRIB) and Ana Roche Martínez, MD, PhD (I3PT). Under the leadership of Mara Dierssen, PhD, at the Centre for Genomic Regulation (CRG), the clinical trial will also contribute to advancing the identification of disease-specific biomarkers underlying the neurobiology of FXS and to assessing treatment response. 

The study has been authorized by the Spanish Agency of Medicines and Medical Devices (AEMPS), with approvals from the relevant ethics committees, and is funded by the Spanish Ministry of Science, Innovation and Universities and the European Union's NextGeneration EU program (Project reference: CPP2022?009659).

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