CONNECTA Therapeutics to Advance CTH120 into Phase II Pediatric Development for Fragile X Syndrome with EIC Accelerator Support

• CTH120 is a first-in-class neuroplasticity modulator with strong Phase I safety and tolerability data
• Awarded a €2.5M European Innovation Council (EIC) Accelerator grant to prepare for Phase II development in pediatric Fragile X Syndrome (FXS) patients
• CONNECTA leads innovation in neurodevelopmental therapies as a unique European biotech advancing a clinical-stage FXS program

BARCELONA, Spain, Sept. 9, 2025 /PRNewswire/ -- CONNECTA Therapeutics, a clinical-stage biotech company pioneering first-in-class neuroplasticity modulators to address unmet medical needs in central nervous system (CNS) disorders, today announced the launch of studies for the pediatric development program of CTH120, an investigational FXS treatment, following positive results from the Phase I trial.

FXS is a rare, untreatable neurodevelopmental disorder affecting approximately 1 in 7,000 males and 1 in 11,000 females, with a global population of around 1.83 million that is growing as diagnosis improves. Symptoms start in early childhood and include lifelong cognitive delays and behavioral 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 treatment innovation essential.

Supported by a €2.5 million grant from the EIC Accelerator as part of the FRAXCURE project (Grant agreement No. 101217931), CONNECTA is one of just 71 companies selected from over 1,200 applicants in the October 2024 call, highlighting its innovative potential. The grant will enable CONNECTA to complete critical regulatory and preclinical activities, including safety studies, development of an age-appropriate oral formulation, and submission of its Pediatric Investigation Plan to support regulatory approval for a future Phase II trial of CTH120 in pediatric FXS patients. CONNECTA is also planning a Phase IIb trial in adults to further expand clinical evidence supporting CTH120.

CONNECTA's lead program, CTH120, is a first-in-class, disease-modifying therapy for FXS. This novel 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 that have shown limited efficacy and clinically limiting adverse events.

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

CTH120 has strong preclinical and positive Phase I data (NCT06480968) demonstrating excellent safety, tolerability, and pharmacokinetics in healthy adult volunteers. Built on CONNECTA's proprietary neuroplasticity modulation platform, CTH120 is uniquely positioned to address unmet needs in FXS. It serves as a proof of concept for the expansion of this disease-modifying approach into other central nervous system disorders, including Rett syndrome, Down's syndrome, DiGeorge syndrome, autism spectrum disorder (ASD), attention deficit hyperactivity disorder (ADHD), and neuropathic pain.

About CONNECTA Therapeutics

CONNECTA Therapeutics is a clinical-stage biotech company leveraging its small-molecule neuroplasticity modulation platform to address unmet medical needs in Central Nervous System (CNS) disorders with a focus on pediatric neurodevelopmental conditions. Neuroplasticity is the ability of the nervous system to adapt its activity in response to intrinsic or extrinsic stimuli by reorganizing its structure, functions, and connections, and is impaired in the vast majority of these disorders.

The Company's lead program, CTH120, is a first-in-class disease-modifying modulator for Fragile X Syndrome (FXS), a rare and currently untreatable neurodevelopmental disorder that strongly impairs cognition and behavior. CTH120 is a small novel molecule that targets the tropomyosin receptor kinase B (TrkB), a key mediator in brain development and function. Unlike existing symptomatic treatments, CTH120 aims to restore the underlying neuronal architecture. Backed by strong preclinical and Phase I safety data (NCT06480968) and boosted by the FRAXCURE project (co-funded by the European Union), CONNECTA is advancing CTH120 towards Phase II trials in adult and pediatric FXS populations.

CONNECTA's scalable neuroplasticity modulation platform has already yielded additional promising compounds that have shown potential in treating multiple CNS disorders. Preclinical models have demonstrated efficacy for FXS and Rett Syndrome, with the potential to address other high-value indications such as Down's syndrome, DiGeorge syndrome, autism spectrum disorder (ASD), attention deficit hyperactivity disorder (ADHD), and neuropathic pain.

About FRAXCURE Project

FRAgile X syndrome Clinical trial: Unravelling the science behind this Rare disease in Europe | FRAXCURE | Project | Fact Sheet | HORIZON | CORDIS | European Commission. 

Funded by the European Union. Views and opinions expressed are, however, those of the author(s) only and do not necessarily reflect those of the European Union or the European Innovation Council and SMEs Executive Agency (EISMEA). Neither the European Union nor the granting authority can be held responsible for them.

SOURCE CONNECTA Therapeutics