CONNECTA Therapeutics Advances First-in-Class Neuroplasticity Modulator CTH120 to Phase II for Pediatric Fragile X Syndrome
核心洞察
CONNECTA Therapeutics (搜索) received a €2.5 million European Innovation Council (搜索) Accelerator grant to advance CTH120, a first-in-class neuroplasticity modulator, into Phase II pediatric development for Fragile X Syndrome (搜索).
CTH120 targets the tropomyosin receptor kinase B (搜索) (TrkB (搜索)) pathway and demonstrated excellent safety, tolerability, and pharmacokinetics in Phase I trials with healthy adult volunteers.
Fragile X Syndrome (搜索) affects approximately 1 in 7,000 males and 1 in 11,000 females globally, with no approved disease-modifying therapies currently available for this devastating neurodevelopmental disorder.
CONNECTA Therapeutics (搜索) announced the launch of studies for the pediatric development program of CTH120, an investigational treatment for Fragile X Syndrome (搜索) (FXS), following positive results from the Phase I trial. The Barcelona-based clinical-stage biotech company secured a €2.5 million grant from the European Innovation Council (搜索) (EIC) Accelerator to prepare for Phase II development in pediatric FXS patients.
Addressing Critical Unmet Medical Need
FXS represents a significant unmet medical need, affecting approximately 1 in 7,000 males and 1 in 11,000 females, with a global population of around 1.83 million that continues to grow as diagnosis improves. The rare neurodevelopmental disorder manifests in early childhood with lifelong cognitive delays and behavioral challenges including hyperactivity, anxiety, social withdrawal, aggression, and self-injury, severely impacting quality of life.
The disease burden extends beyond patients to families, with only 10% of adult patients living independently and nearly 80% of affected families facing financial hardship. Despite this substantial impact, no disease-modifying therapies currently exist for FXS, making early intervention and treatment innovation essential.
Novel Mechanism of Action
CTH120 represents a first-in-class, disease-modifying therapy that targets the tropomyosin receptor kinase B (搜索) (TrkB (搜索)), a key mediator involved in neuroplasticity. This approach focuses on 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," said Jordi Fàbrega, Co-Founder & CEO of CONNECTA Therapeutics (搜索). "CTH120 has the potential to redefine FXS care through a disease-modifying novel mechanism of action that represents a significant technological advantage."
Strong Clinical Foundation
CTH120 has demonstrated strong preclinical and positive Phase I data (NCT06480968) showing excellent safety, tolerability, and pharmacokinetics in healthy adult volunteers. The Phase I trial results provided the foundation for advancing the compound into pediatric development, representing a critical milestone for families affected by FXS.
Competitive Grant Award
CONNECTA was selected as one of just 71 companies from over 1,200 applicants in the October 2024 EIC Accelerator call, highlighting the innovative potential of its approach. The €2.5 million grant, awarded as part of the FRAXCURE project (Grant agreement No. 101217931), will enable the company to complete critical regulatory and preclinical activities.
These activities include conducting safety studies, developing an age-appropriate oral formulation, and submitting a Pediatric Investigation Plan to support regulatory approval for the 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.
Platform Technology with Broader Applications
Built on CONNECTA's proprietary neuroplasticity modulation platform, CTH120 serves as a proof of concept for expanding this disease-modifying approach into other central nervous system disorders. The company's scalable 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 (搜索), with potential applications extending to other high-value indications including Down's syndrome (搜索), DiGeorge syndrome (搜索), autism spectrum disorder (搜索) (ASD), attention deficit hyperactivity disorder (搜索) (ADHD), and neuropathic pain (搜索).
The neuroplasticity modulation approach addresses a fundamental mechanism impaired in the vast majority of neurodevelopmental disorders, positioning CONNECTA's platform technology for potential broad therapeutic applications across CNS conditions with significant unmet medical needs.