CONNECTA Therapeutics, CRG, Hospital del Mar Research Institute and I3PT receive €2.7 million from the Spanish Ministry of Science and Innovation to begin Phase IIa trials for CTH120 in Fragile X syndrome
The Phase IIa study will assess the efficacy of CTH120 as an innovative therapy in adults with FXS, the most common form of inherited intellectual disability without a specific treatment.
María Martínez, Jordi Fàbrega, Mara Dierssen, Ana Aldea, Rafael de la Torre and Ana Roche
representing CONNECTA Therapeutics, CRG, Hospital del Mar Research Institute and I3PT
Barcelona, February 12 2024
The public-private consortium made up of biotechnology company CONNECTA Therapeutics, the Centre for Genomic Regulation (CRG), the Hospital del Mar Research Institute (IMIM) and the Institute for Research and Innovation Parc Taulí (I3PT) has been awarded €2.7 million in funding from the Spanish Ministry of Science, Innovation and Universities and the European Union through the NextGeneration EU programme to start Phase IIa clinical trials for CTH120 in the treatment of Fragile X syndrome (FXS).
The funding from the competitive call “Public-private partnership projects” complements the private investment from CONNECTA Therapeutics’ investors, which include Prous Institute for Biomedical Research, Inveready and CDTI Innvierte, and the funds obtained through Retos Colaboración, CDTI NEOTEC and ENISA public research programmes.
CTH120 is an innovative drug designed using artificial intelligence that modulates neuroplasticity. At present, CTH120 is completing Phase I clinical trials at the Hospital del Mar Research Institute in Barcelona, led by Dr Rafael de la Torre and Dr Ana Aldea as principal investigators.
Final results of the Phase I clinical trial are expected in the first half of this year with Phase IIa estimated to start in the first half of 2025. In this new phase, the I3PT, with Dr Ana Roche as the principal investigator, is joining the CRG and Hospital del Mar Research Institute as a strategic partner. The project will also identify specific biomarkers to help assess response to the treatment, led by Dr Mara Dierssen at the CRG.
“We are very happy to make this announcement just days before the Rare Disease Day, as finding therapies for these diseases represents a huge public health challenge. This funding from the Spanish Ministry of Science, Innovation and Universities will allow us to advance clinical development of a specific treatment for FXS, working with world-renowned neuroscience centres and researchers in the Barcelona area,” notes Jordi Fàbrega, co-founder and CEO of CONNECTA Therapeutics.
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 and autism. Its prevalence is estimated to be fewer than 3 in 10,000 inhabitants and it is diagnosed in the first months of life or early childhood using DNA blood tests. There is no specific treatment for the cause of the condition and only symptomatic interventions are available. FXS is currently the most common type of inherited intellectual disability and the second most common genetic one, after Down syndrome.
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 with patients’ intellectual disability and symptoms. CONNECTA Therapeutics’ CTH120, designed using artificial intelligence, has proven in preclinical studies carried out in the group of Dr Mara Dierssen at the Center for Genomic Regulation of Barcelona to be a good modulator of neuroplasticity and also to improve cognitive abilities and reverse behavioural manifestations, such as issues with social interactions. CTH120 has orphan drug designation from the European Medicines Agency.
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, a first-in-class molecule designed using artificial intelligence that modulates neuroplasticity and is being studied as a treatment for Fragile X syndrome and other CNS diseases. Founded in 2019 as a spin-off of Prous Institute for Biomedical Research, CONNECTA Therapeutics is based at the Barcelona Science Park (PCB).
More information: www.connectatherapeutics.com
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 economic prosperity. 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 Barcelona Institute 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 computer sciences, 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 the Government of Catalonia and is accredited by the Carlos III Health Institute. With over 75 years of history, it is the oldest active public research centre.
More information: www.imim.es
The Institute for Research and Innovation Parc Taulí (I3PT) fosters research, innovation and teaching in health to drive knowledge transfer and generate value to bring about social and economic growth. The Institute is renowned in the R&D ecosystem for its history of promoting and implementing its own innovation model, combining research results with the clinical needs of Parc Taulí. This has allowed them to develop custom solutions and implement them in the healthcare system and on the market, through partnerships with key stakeholders in the ecosystem. I3PT is a CERCA institute accredited by the Carlos III Health Institute.
More information: https://www.tauli.cat/en/institut/
Phone +34 667 76 15 24