The Phase IIa study will evaluate the efficacy of CTH120 as an innovative therapy in adult patients with FXS, the most common form of inherited intellectual disability without a specific treatment
The public-private consortium formed by the biotechnology company CONNECTA Therapeutics, the Genomic Regulation Center (CRG), the Hospital del Mar Research Institute and the Parc Taulí Research and Innovation Institute (I3PT) has been awarded 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 start Phase IIa clinical trials for CTH120 in the treatment of Fragile X Syndrome (FXS).
The funding from the competitive call "Projects of public-private collaboration" complements the private investment from CONNECTA Therapeutics investors, which include Prous Institute for Biomedical Research, Inveready and CDTI Innvierte, and the funds obtained through the public funding programs 'Retos Colaboración', CDTI NEOTEC and ENISA.
CTH120 is an innovative therapy designed using artificial intelligence techniqueswhich acts as a modulator of neuroplasticity. Phase I clinical trials for CTH120 are currently being completed at the Hospital del Mar Research Institute in Barcelona, led by Dr. Rafael de la Torre and Dra. Ana Aldea as principal investigators.
Final results of the Phase I clinical trial are expected in the first semester of this year and be able start Phase IIa in the first semester of 2025. In this new phase, the I3PT, with Dra. Ana Roche as principal researcher, joins as strategic partner together with the CRG and the Hospital del Mar Research Institute. During the project, specific biomarkers will also be identified to facilitate the assessment of the response to treatment, an activity led by Dra. Mara Dierssen at CRG.
"We are happy to announce this news a few days before the celebration of the World Day of Rare Diseases, as finding therapies for these diseases represents a huge public health challenge. This funding from the Spanish Ministry of Science, Innovation and Universities allows us progress in the clinical development of a specific treatment for FXS with world-renowned centers and researchers in the field of neuroscience in the Barcelona area" comments Jordi Fàbrega, CEO and co-founder of CONNECTA Therapeutics.
Fragile X syndrome
Fragile X syndrome (FXS) is an inherited genetic disorder tied to the X chromosome that causes intellectual disability and mild to severe emotional and social issues, such as hyperactivity, anxiety, aggressive behaviors or autism. Its prevalence is estimated at approximately 3 patients per 10.000 inhabitants and is diagnosed in the first months of life or early childhood through DNA blood tests. There is no specific treatment for the cause of the pathology, only treatments that help alleviate the symptoms. Currently, the SXF is the first cause of hereditary intellectual disability and the second most prevalent genetic cause after Down syndrome.
The drug CTH120
Fragile X syndrome is caused by a mutation that leads to the lack of the FMRP protein, with an important role in neuroplasticity and the maturation of synaptic connections between neurons, facts that are associated to intellectual disability and the symptoms of patients . The CTH120 drug from CONNECTA Therapeutics, designed using artificial intelligence techniques, has shown in preclinical studies developed in the group of Dra. Mara Dierssen from the Center for Genomic Regulation in Barcelona to be a good modulator of neuroplasticity, while improving cognitive ability and restoring behavioral manifestations such as social interaction issues. CTH120 has the orphan drug designation from the European Medicines Agency.
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