Connecta Therapeutics Raises Funds to Advance Potential Fragile X Therapy

Connecta Therapeutics raised €1.7 million (about $2 million) in investments that will enable it to bring a potential fragile X syndrome (FXS) therapy to Phase 1 clinical trials in late 2021, and to Phase 2a stage by 2023.

Fragile X is caused by mutations in the FMR1 gene, leading to low-to-no levels of the fragile X mental retardation protein (FMRP). Largely active in the brain, FMRP regulates the production of other proteins, including several at synapses — the sites where nerve cells communicate typically through structures such as dendritic spines.

Connecta’s potential therapy, CTH120, seeks to improve neuroplasticity, which is the brain’s ability to adapt to change by modifying its connections. Abnormal neuroplasticity is thought to underlie the symptoms of fragile X, including cognitive and behavioral problems. CTH120 is intended to target proteins implicated in nerve cell communication and address the current lack of treatments specific to fragile X.

“What sets this drug apart is that it modulates various proteins involved in the anomalies in the dendritic spines of cortical neurons in FXS,” Mara Dierssen, PhD, a research advisor to Connecta, said in a press release.

Fragile X is the most common genetic cause of autism. According to Connecta, preclinical studies have suggested CTH120 to improve cognitive abilities and to reverse autism traits, such as social interaction difficulties.

Atypical neuroplasticity plays a role in other neurological disorders, such as Rett syndrome and Down syndrome, which share some cognitive and behavioral characteristics with fragile X. As such, CTH120 also could be effective in treating people with these disorders, Connecta stated.

Inveready led the current funding round. Other investors included CDTI, through its Innvierte program for Spanish technology companies; Connecta’s founders, Josep and David Prous, who also founded the Prous Institute for Biomedical Research, of which Connecta is a spin-off; and Jordi Fàbrega, Connecta’s CEO.

“We’re very happy that Inveready and CDTI have joined the company, not only as strategic partners and investors, but also bringing their experience in advancing projects with a huge impact on R&D [research and development]. We would like to start clinical trials on this first-in-class drug as soon as possible to prove the efficacy of this therapeutic strategy” for fragile X, Fàbrega said.