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(Photo: Connecta)

Connecta Therapeutics starts clinical trials for its Fragile X syndrome drug

Connecta Therapeutics, a biotechnology company based in the Science Park in the clinical phase that develops new treatments for unmet medical needs of the central nervous system (CNS), has received authorization from the Spanish Agency for Medicines and Health Products start of the Phase I clinical trial of the drug CTH120 for the treatment of fragile X syndrome (XFS).

The study will take place at Hospital del Mar Research Institute in Barcelona, with Dr Rafael de la Torre, director of the Neuroscience Research Programme at Hospital del Mar Research Institute, and Dr Ana Aldea, coordinator of the Clinical Research Unit at Hospital del Mar Research Institute and head of the Clinical Pharmacology Section at Hospital del Mar, as principal investigators.

It is a double-blind, placebo-controlled trial that aims to assess the safety and tolerability of CTH120, a first-in-class molecule designed using artificial intelligence that modulates neuroplasticity and has proven in preclinical studies to improve cognitive capacity and reverse behavioural manifestations of
neurodevelopmental disorders. The study will recruit 70 healthy adults, both men and women.

CTH120 has been developed with contributions from the team led by Dr Mara Dierssen, head of the Cellular and Systems Neurobiology group in the Synthetic and Systems Biology Programme of the Centre for Genomic Regulation (CRG) and member of the Connecta Therapeutics scientific advisory
board. The researchers Juan Luis Musoles and María Martínez de Lagrán have participated in these studies.

“We are very proud that Hospital del Mar Research Institute will be carrying out the clinical trial for an FXS treatment, which is a hereditary genetic neurodevelopmental disorder related to the X chromosome that causes mild to severe intellectual disability,” said Dr Rafael de la Torre. FXS, which has no specific treatment, is estimated to affect fewer than 3 in 10,000 inhabitants.

“It is very important to find a therapeutic solution for FXS, currently considered the most common cause of inherited intellectual disability. CTH120 brings new hope for patients with this minority disease all over the world,” highlighted Dr Ana Aldea.

“The polypharmacological profile of CTH120 is a novel approach that acts simultaneously on cognitive and sociability aspects characteristic of neurodevelopmental diseases,” noted Dr Mara Dierssen.

“In preclinical studies, we’ve seen that CTH120 has great potential as an effective, safe therapeutic option for FXS patients, not only to alleviate their symptoms but also to tackle the origin of the condition,” explained Dr Josep Prous, co-founder and Chief Scientific Officer of Connecta Therapeutics.

“The final results of the Phase I trial are expected in the second quarter of 2024. If they’re positive and back the safety of our drug, we’ll continue with Phase II clinical trials to study the efficacy of the drug in patients as soon as possible,” said Jordi Fàbrega, co-founder and CEO of Connecta Therapeutics.

CTH120 has been granted orphan drug designation from the European Medicines Agency.

The company was recently granted a participatory loan for €600,000 from the Spanish National Innovation Company (Enisa). This funding reinforces Connecta Therapeutics’ CNS research activities. Since it was founded in 2019, Connecta Therapeutics has raised over €5 million, including funds from the founders, venture capitalists (Inveready and CDTI through the Innvierte programme) and various competitive public programmes, including Retos Colaboración 2019 and NEOTEC.