Hospital del Mar Research Institute Hospital del Mar Research Institute


19/02/2024 - General information

Nearly 3 million euros to initiate Phase IIa of CTH120 drug for Fragile X syndrome

The Ministry of Science, Innovation and Universities and the European Union, through the Next Generation EU program will finance this new phase. It will evaluate the efficacy of CTH120 as an innovative therapy in adult patients with SXF, the most common form of hereditary intellectual disability without specific treatment.

María Martínez, Jordi Fàbrega, Mara Dierssen, Ana Aldea, Rafael de la Torre and Ana Roche representing CONNECTA Therapeutics, CRG, the Hospital del Mar Research Institute and I3PT.

The public-private consortium formed by the biotechnology company CONNECTA Therapeutics, the Hospital del Mar Research Institute, the Center for Genomic Regulation (CRG), and the Parc Taulí Research and Innovation Institute (I3PT) will receive 2,7 million euros in funding from the Ministry of Science, Innovation and Universities and the European Union, through the Next Generation EU program, to initiate Phase IIa clinical trials of the drug CTH120 for the treatment of Fragile X syndrome (Fragile X syndrome).

CTH120 is an innovative therapy designed using artificial intelligence techniques that acts as a modulator of neuroplasticity. Phase I clinical trials for CTH120 are currently being finalized at the Hospital del Mar Research Institute in Barcelona, led by Dr. Rafael de la Torre and Dr. Ana Aldea as principal investigators. The final results of the Phase I clinical trial are expected to be available during the first half of this year and the Phase IIa trial is expected to start in the first half of 2025. During the project, specific biomarkers will also be identified to facilitate the assessment of the response to treatment.

Fragile X syndrome and the drug CTH120

Fragile X syndrome is due to a mutation that involves the lack of the FMRP protein, which plays an important role in neuroplasticity and the maturation of synaptic connections between neurons, facts that are related to intellectual disability and symptomatology of patients. CONNECTA Therapeutics' drug CTH120, designed using artificial intelligence techniques, has been shown in preclinical studies developed in Dr. Mara Dierssen's group at the Center for Genomic Regulation in Barcelona to be a good modulator of neuroplasticity, while improving cognitive capacity and restoring behavioral manifestations such as social interaction problems. CTH120 has orphan drug designation from the European Medicines Agency.

This syndrome is an X-linked inherited genetic disorder that causes intellectual disability and mild to severe emotional and social problems such as hyperactivity, anxiety, aggressive behavior or autism. Its prevalence is estimated at approximately 3 patients per 10,000 population and is diagnosed during the first months of life or early childhood by DNA blood tests. There is no specific treatment aimed at the cause of the pathology, only treatments that help to alleviate the symptoms. Currently, SXF is the leading cause of hereditary intellectual disability and the second most prevalent genetic cause after Down syndrome.

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