CONNECTA Therapeutics

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.

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