CONNECTA Therapeutics announces positive results from Phase I study of neurodevelopmental disorders drug candidate, CTH120
CONNECTA Therapeutics has announced positive results from its Phase I clinical trial to evaluate the safety, tolerability and pharmacokinetics profile of its drug candidate CTH120 in adult volunteers.
CTH120 is an investigational drug that modulates neuroplasticity, a property of the brain affected in many neurodevelopmental disorders, including attention-deficit/hyperactivity disorder (ADHD), Down syndrome and Fragile X syndrome (FXS). These conditions affect between 5% and 10% of the population and are characterised by delayed or altered acquisition of cognitive and social abilities. Preclinical studies have shown CTH120 to improve cognitive abilities and reverse behavioural manifestations of these disorders.
CONNECTA’s randomised, double-blind, placebo-controlled, Phase I clinical trial recruited 76 healthy adults (men and women aged 18 to 55) at Hospital del Mar Research Institute (HMRI) in Barcelona. CTH120 was studied in single and multiple orally administered doses, determining its pharmacokinetic profile and potential food interactions (ClinicalTrials.gov ID: NCT06480968)1.
“The results confirm that CTH120 has proven to be safe and well tolerated throughout the clinical trial,” explained Dr Rafael de la Torre and Dr Ana Aldea, principal investigators of the study.
Dr Josep Prous, Co-Founder and Chief Scientific Officer of CONNECTA Therapeutics, added that “the results are very solid and encourage us to evaluate the efficacy of CTH120 in adults with FXS in Phase II clinical trials, starting in the second half of 2025.”
“We’re very pleased to move forward the clinical development of CTH120 and excited that our investigational drugs may improve the quality of life for patients with neurodevelopmental disorders and their families, currently a high unmet medical need”, concluded Jordi Fàbrega, Co-Founder and CEO of CONNECTA Therapeutics.
CONNECTA Therapeutics is currently conducting an observational study on the biomarkers that will be used in the Phase IIa clinical trial with Hospital del Mar Research Institute (HMRI), the Parc Taulí Research and Innovation Institute (I3PT) and the Centre for Genomic Regulation (CRG)2.
Fragile X syndrome
Fragile X syndrome (FXS) is an inherited genetic disorder caused by a mutation in the X chromosome that causes mild to severe intellectual disabilities and emotional, behavioural and social manifestations, such as hyperactivity, anxiety, aggressive behaviour and autism. Its prevalence is estimated to be 3 in 10,000 people and it is diagnosed around the first months of life or early childhood using DNA blood tests. There is no specific treatment for the cause of the condition, only ones that help alleviate its symptoms. FXS is currently the most common type of inherited intellectual disability and the second most common genetic one, after Down syndrome.
CTH120
CONNECTA Therapeutics’ CTH120 is a first-in-class molecule designed using artificial intelligence technologies that modulates neuroplasticity, improving cognitive abilities and reversing behavioural manifestations. In FXS animal models, CTH120 reverses pathological characteristics of dendritic spines, which show greater density and less maturity. CTH120 has the orphan drug designation for FXS from the European Medicines Agency.
1 Project funded by the Ministry of Science, Innovation and Universities and the State Research Agency (MCIN/AEI /10.13039/501100011033) under the RETOS-COLABORACIÓN sub-programme, 2019 call. The project is jointly funded by the European Union to help promote technological development, innovation and quality research.
2 Project funded by the Ministry of Science, Innovation and Universities and the State Research Agency (MCIN/AEI /10.13039/501100011033) under the COLABORACIÓN PÚBLICO-PRIVADA sub-programme, 2022 call, and by the European Union through the “NextGenerationEU / Plan for a stronger, more resilient Europe”.
