Connecta Therapeutics starts clinical trials for its Fragile X syndrome drug

Connecta Therapeutics, a clinical stage biotechnology company focusing on the development of new treatments for central nervous system (CNS) disorders without therapeutic options, has been granted authorisation from the Spanish Agency of Medicines andMedical Devices (AEMPS) to start Phase I clinical trials on its drug CTH120 for the treatment of Fragile X syndrome (FXS).

The study will take place at Hospital del Mar Research Institute in Barcelona, with Dr Rafael de laTorre, 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 andhead 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, afirst-in-class molecule designed using artificial intelligence that modulates neuroplasticity and has proven in preclinical studies to improve cognitive capacity and reverse behavioural manifestations ofneurodevelopmental 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 advisoryboard. 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, whichhas 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.

Fragile X syndrome

Fragile X syndrome (FXS) is an inherited genetic disorder tied to the X chromosome that causes mild to severe intellectual disabilities and emotional and social issues, such as hyperactivity, anxiety, aggressive behaviour andautism. Its prevalence is estimated to be fewer than 3 in 10,000 inhabitants. It is currently diagnosed at birth or in childhood using DNA blood tests, but there is no specific treatment for the cause of the condition, only ones thathelp 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

Fragile X syndrome is caused by a mutation that leads to a lack of FMRP protein, which plays an important role in neuroplasticity and maturation of synaptic connections between neurons, facts that are associated withpatients’ intellectual disability and symptoms. Connecta Therapeutics’ CTH120, designed using artificial intelligence, has proven in preclinical studies to be a good modulator of neuroplasticity and also to improve cognitive abilities and reverse behavioural manifestations, such as issues with social interactions.

About Connecta Therapeutics

Connecta Therapeutics is a clinical stage biotechnology company focusing on the development of new treatments for central nervous system (CNS) disorders without therapeutic options. Its main drug is CTH120, afirst-in-class molecule designed using artificial intelligence that modulates neuroplasticity and is being studied as a treatment for Fragile X syndrome (Phase I) and other CNS diseases (preclinical). Founded in 2020 as a spin- off of Prous Institute for Biomedical Research, Connecta Therapeutics is based at the Barcelona Science Park (PCB) and has already raised €5 million in funding to develop its programmes.

More information: www.connectatherapeutics.com

About Centre for Genomic Regulation

The Centre for Genomic Regulation (CRG) is a renowned international biomedical research centre located in Barcelona. Its mission is to discover and promote knowledge to benefit society, public health, and economicprosperity. The centre 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 BarcelonaInstitute of Science and Technology (BIST) and is a CERCA research centre of the Government of Catalonia.

More information: www.crg.eu

About Hospital del Mar Research Institute

The Hospital del Mar Research Institute of Barcelona is a research centre in biomedicine and health sciences, organised across five large programmes: cancer, epidemiology and public health, biomedical computersciences, neurosciences, and translational clinical research. Featuring around 700 professionals, it is among the top ten Spanish institutions in terms of scientific impact on healthcare. It is a CERCA research centre of theGovernment of Catalonia and is accredited by the Carlos III Health Institute.

More information: www.imim.es