Connecta Therapeutics drug for fragile X syndrome receives orphan designation from European Medicines Agency
The Connecta Therapeutics CTH120 molecule has received approval as orphan drug from the European Medicines Agency (EMA) to treat fragile X syndrome (FXS), the leading cause of hereditary intellectual disability. The company -founded in 2019 in the Barcelona Science Park by Prous Institute for Biomedical Research, Josep Prous, David Prous and Jordi Fàbrega- is accelerating the development of the molecule in order to begin clinical trials in 2022.
Currently, fragile X syndrome has no cure. CTH120 doesn’t just palliate the symptoms but addresses the origin of the pathology through an innovative therapeutic strategy based on modulating neuroplasticity.
The co-founder and CEO of Connecta, Jordi Fàbrega, explains that “promoting treatments of rare diseases is a public health priority as there are almost 7,000 rare diseases that affect 7% of the world population”. To Connecta Therapeutics, this recognition “will help to accelerate the company’s scientific and business development and to facilitate the start of new avenues of research in central nervous system diseases”, says Fàbrega.
The EMA grants this designation to support the development of drugs for diseases that affect fewer than five patients out of every 10,000 inhabitants in the European Union. Companies that develop these drugs enjoy additional benefits such as market exclusivity for 10 years.
In order to bring CTH120 to clinical trials, Connecta Therapeutics closed an investment round of €1.7 million in 2020, led by Inveready and the CDTI Innvierte program. It also received a grant of almost €2 million from “Retos Colaboración” of the Spanish Ministry of Science and Innovation through the public-private consortium made up of the company, the Centre for Genomic Regulation (CRG) and the Hospital del Mar Medical Research Institute (IMIM).
A first-in-class drug to treat fragile X síndrome based on an innovative strategy
Fragile X syndrome (FXS) is a hereditary genetic disorder linked to the X chromosome that causes intellectual disability and emotional and social problems ranging from mild to severe issues, such as hyperactivity, anxiety, aggressive behaviours, and autism. Its prevalence is estimated at less than 3 patients per 10,000 inhabitants. The syndrome is diagnosed in newborns or in childhood through DNA blood tests, but there is no specific treatment directed at the cause of the disease, only treatments to help relieve its symptoms.
FXS is due to a mutation that leads to a FMRP protein deficiency, which plays an important role in the plasticity and maturation of synaptic connections between neurons, facts that seem to be related to the intellectual disability and symptoms of those affected. Preclinical studies have demonstrated Connecta Therapeutics’ CTH120, designed using artificial intelligence techniques, is a good neuroplasticity modulator. The drug has also been shown to enhance cognitive capacity and improve autistic signs and symptoms such as social interaction issues.