CONNECTA Therapeutics, a clinical-stage biotech company located at the Barcelona Science Park (PCB), which leads the development of first-in-class neuroplasticity modulators to address unmet medical needs in central nervous system (CNS) disorders, has announced the start of the study for the development of its pediatric program CTH120, an investigational treatment for Fragile X Syndrome (FXS) with positive results from the Phase I trial.

Fragile X Syndrome (FXS) is a rare neurodevelopmental disorder with no approved treatment, affecting approximately 1 in 7,000 males and 1 in 11,000 females, with a global population of 1.83 million people that continues to grow as diagnosis improves. Symptoms begin in early childhood, including lifelong cognitive delays and behavioural challenges such as hyperactivity, anxiety, social withdrawal, aggression, and self-injury, severely impacting quality of life.

Only 10% of adult patients live independently, and nearly 80% of affected families face financial hardship. Despite this burden, no disease-modifying therapies exist, making early intervention and therapeutic innovation essential.

With the support of a €2.5 million grant from the EIC Accelerator under the FRAXCURE project, CONNECTA is one of only 71 companies selected from over 1,200 applications in the October 2024 call, highlighting its innovative potential. The grant will enable the company to complete critical regulatory and preclinical activities, including safety studies, the development of an age-appropriate oral formulation, and submission of the Pediatric Investigation Plan to support regulatory approval for a future Phase II trial in pediatric FXS patients.

Additionally, the biotech company also plans a Phase IIb trial in adults to further expand the clinical evidence supporting CTH120.

“There are no approved treatments for FXS, a devastating disorder that affects children’s cognitive development, behaviour, and quality of life for them and their families, lasting a lifetime. CTH120 has the potential to redefine FXS care through an innovative, disease-modifying mechanism of action that represents a significant technological advantage. The EIC Accelerator grant is a game-changer — accelerating our mission to bring hope and lasting impact to families. We are honored to have been selected for funding in one of the most competitive application rounds since the launch of the EIC Accelerator program under Horizon Europe,” said Jordi Fàbrega, Co-Founder and CEO of CONNECTA Therapeutics.

Its lead program, CTH120, is a first-in-class, disease-modifying therapy for FXS. This innovative small molecule targets the tropomyosin receptor kinase B (TrkB), a key mediator involved in neuroplasticity — the brain’s ability to reorganize, adapt, and form new neural connections. By modulating neuroplasticity, CTH120 aims to restore the disrupted neuronal architecture underlying FXS, offering a transformative approach beyond current symptomatic treatments, which have shown limited efficacy and clinically restrictive adverse effects.

CTH120 has a solid preclinical data package and positive Phase I results demonstrating an excellent safety, tolerability, and pharmacokinetic profile in healthy adult volunteers. Developed from CONNECTA’s proprietary neuroplasticity modulation platform, CTH120 is uniquely positioned to address the unmet needs in FXS.

Furthermore, it serves as a proof of concept for extending this disease-modifying approach to other central nervous system disorders, including Rett syndrome, Down syndrome, DiGeorge syndrome, autism spectrum disorder (ASD), attention deficit hyperactivity disorder (ADHD), and neuropathic pain.

» Link to the news: CONNECTA Therapeutics website [+]