CTH120 Medication Granted Orphan Designation in EU

Yedida Y Bogachkov PhD avatar

by Yedida Y Bogachkov PhD |

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CTH120 granted orphan designation in EU/Fragile X News Today/scientist using microscope in lab illustration

Connecta Therapeutics‘ CTH120 molecule has been granted orphan designation by the European Medicines Agency (EMA) for treating fragile X syndrome, the leading cause of hereditary intellectual disability and autism spectrum disorders.

The company said it intends to begin human clinical trials as early as next year to test CTH120, which is designed to improve cognitive ability and ease the symptoms of autism.

Orphan designation given by the EMA is used for medications that are intended to treat diseases that affect fewer than 5 of every 10,000 citizens in the European Union. This designation allows Connecta to benefit from incentives, such as protection from competition for 10 years, if its investigational medicine is approved and hits the market.

For Connecta, orphan designation for CTH120 “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,” Jordi Fàbrega, co-founder and CEO of Connecta, said in a press release.

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“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,” Fàbrega said.

Fragile X is caused by mutations in the FMR1 gene that lead to impaired production of a protein called FMRP, commonly found in the brain and essential for normal cognitive development. This protein helps to mature the synaptic connections between neurons (nerve cells) and plays an important role in neuroplasticity. Of note, synapses are the junctions between two nerve cells that allow them to communicate, while synaptic plasticity refers to the ability of synapses to strengthen or weaken over time.

CTH120 works not only to ease the symptoms of fragile X, but to address their cause, using artificial intelligence techniques.

Preclinical studies have indicated that CTH120 can modulate the brain’s inherent neuroplasticity. In animal models of fragile X, CTH120 also has been shown to improve cognitive abilities and to ease symptoms of autism linked to the disorder, such as social interaction issues.

The start of clinical trials is driven, in part, by €1.7 million (almost $2 million) in financing led by Inveready and CDTI Innvierte, the company said. Additional funding of €2 million (about $2.3 million) was granted by “Retos Colaboración,” of the Spanish Ministry of Science and Innovation, to further the development of CTH120, according to Connecta.