If everything continues as before, by mid-year, Connecta Therapeutics will be able to ensure that its Fragile X Syndrome (FXS) Drug Is Safe in Healthy People. The signs are so positive that this Catalan company has already started working on the testing funding necessary to demonstrate that this medication is safe, also in patients suffering from this genetic disorder. And, for now, it has already tied up 2.7 million euros from the Ministry of Science, Innovation and Universities, which they hope will be just the beginning.
As detailed in a statement by the company, fragile X syndrome is due to a mutation that causes a lack of a protein (FMRP), which is key in synaptic connections between neurons. This translates into people with intellectual disability and/or emotional and social problems such as hyperactivity, anxiety, aggressive behaviors or autism. Currently, the SXF is the leading cause of hereditary intellectual disability and the second most prevalent genetic cause after Down syndrome.
The proposal from Connecta Therapeutics (a company that was born as a result of research by the Center for Genomic Regulation of Barcelona) is a medicine that modulates neuroplasticity, improves cognitive ability and restores behaviors such as social interaction problems. This drug has been designed with the help of tools of artificial intelligence that allow us to simulate scenarios, conditions or situations and not have to recreate them in the laboratory, which speeds up research and makes the product tested on animals much safer.
Thus, the company is about to pass Phase I of its clinical trial, carried out at the Hospital del Mar Research Institute in Barcelona with “positive preliminary results“. It will be in the middle of this year when it is completely confirmed that the treatment does not generate significant side effects on healthy people. And the next step, the one that these 2.7 million euros begin to finance, will be to test the same but with patients adults. This Phase II will begin during the first half of 2025.
In any case, this company does not rule out having to turn to private capital to obtain much more money. Connecta Therapeutics already has Prous Institute for Biomedical Research, Inveready and the CDTI among its shareholders.