Biotech company CONNECTA Therapeutics, member of Catalonia.health, has announced the start of a Phase IIa clinical trial with its most advanced program, CTH120, in adult males with fragile X syndrome (FSX). The milestone comes after the positive safety results obtained in Phase I and represents a key step in the company's evolution.
CTH120 is a first-in-class novel small molecule that acts on the tropomyosin kinase B (TrkB) receptor, a key regulator of neuroplasticity, the brain's ability to form and adapt neuronal connections. SXF is the most common inherited cause of intellectual disability, with an estimated prevalence of approximately 3 in 10,000 individuals, and currently has no specific approved treatment.
Essay design and objectives
The Phase IIa clinical trial is a multicenter, randomized, double-blind, parallel-group study designed to evaluate twice-daily CTH120 versus placebo. It will include 30 male adults between the ages of 18 and 45 in a 1:1 ratio. The primary objective is to evaluate the safety and tolerability of the compound, while the secondary objectives include the characterization of pharmacokinetics and the evaluation of clinical efficacy.
The study will be carried out at the Hospital del Mar Research Institute (HMRIB) in Barcelona and at the Fundació Parc Taulí Research and Innovation Institute (I3PT) in Sabadell, two centers with extensive experience in SXF and neurodevelopmental disorders and also members of Catalonia.health. The trial is led by Dr. Rafael de la Torre Fornell (HMRIB), together with Dra. Ana Aldea Perona (HMRIB) and Dr. Ana Roche Martínez (I3PT). Under the leadership of Dr. Mara Dierssen, at the Center for Genomic Regulation (CRG), the study will also contribute to the identification of specific biomarkers linked to the neurobiology of SXF.
The study has been authorized by the Spanish Agency for Medicines and Health Products (AEMPS) and is funded by the Spanish Ministry of Science, Innovation and Universities and the NextGenerationEU program of the European Union (CPP2022-009659).
Statements of those responsible
Jordi Fàbrega, co-founder and CEO of CONNECTA Therapeutics, emphasized that the start of Phase IIa represents a very significant milestone for the company. Fàbrega noted that the trial was designed to evaluate the safety and tolerability of CTH120 and its potential therapeutic benefit, and expressed the team's confidence that the study will contribute to the development of a new SXF-modifying treatment and serve as a proof of concept for the company's neuroplasticity modulation platform.
Dr. Rafael de la Torre Fornell, principal researcher and coordinator of the study at the HMRIB, has shown that SXF has a profound impact on cognitive and behavioral functions and severely affects the quality of life of the people who live with it and that of their families. De la Torre emphasized that this Phase IIa trial is an important opportunity to evaluate a new and very promising mechanism of action and advance clinical knowledge of this condition.
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