R&D

R&D IS AT THE HEART OF CONNECTA

We are an innovation-oriented, R&D-intensive organization, committed to the development of innovative treatments for patients.

R&D APPROACH

CONNECTA follows a network pharmacology-based approach by evaluating all the targets involved in CNS properties and processes, and modulating different pathways that lead to specific disorders.

The traditional one drug–one target–one disease approach in CNS drug discovery has become increasingly inefficient. CONNECTA’s integrative in silico approach is based on identifying multiple actionable targets and designing drug candidates with disease-modifying potential.

THERAPEUTIC FOCUS

CONNECTA is currently focused on the clinical development of new treatments for patients suffering from neurodevelopmental disorders with an affection of protein networks involved in synaptic and structural plasticity (neuroplasticity imbalance).

PIPELINE

CTH120 – NEUROPLASTICITY MODULATOR

CONNECTA’s leading program, CTH120, is a first-in-class small molecule, NCE, for neuroplasticity-mediated disorders with social behaviour and pro-cognitive properties.

CTH120 was designed after the study of more than 30 molecular and cellular targets for neuroplasticity. The molecule has been proven, in in vitro tests and in vivo in animal models, to be an excellent neuroplasticity modulator with a great extension profile for different neurodevelopmental disorders (fragile X syndrome, Rett syndrome, Down syndrome and autism spectrum disorders, among others).

Initially focused on the treatment of fragile X syndrome (FXS), the most common inherited cause of intellectual disability and considered a major unmet need. FXS is caused by a mutation in the X chromosome on the Fragile X Messenger Ribonucleoprotein 1 (FMR1) gene. The full gene mutation can cause significant intellectual disability, greatly reduce functional abilities, educational achievement and social-emotional skills.

    • Excellent preclinical efficacy results: CTH120 rescues the dendritic pathology of FXS, clearly improving cognitive ability and completely restoring the social abnormalities linked to the disorder.
    • Different approach from other FXS candidates: CTH120 is a multi-target drug with pleiotropic effects (network pharmacology), including catecholamine transport inhibition and neurotrophic factors modulation.
    • International patent family.
    • Orphan drug designation: The EMA has granted orphan drug status for CTH120 in FXS, as orphan medicinal product nr EU/3/21/2432 (May 2021).
    • Entering FIH studies: CTH120 has finalized the regulatory toxicology studies and is entering Phase I clinical trials in 2022.

 

ABOUT FRAGILE X SYNDROME

Fragile X syndrome (FXS) is a genetic condition that causes a range of developmental problems including learning disabilities and cognitive impairment. FXS’s computed prevalence for male and female patients is approximately of 3/10.000.

Affected individuals usually exhibit delayed development of speech and language by age 2. Most males with FXS have mild to moderate intellectual disability, while about one-third of affected females are intellectually disabled. Children with FXS may also have anxiety and hyperactive behavior such as fidgeting or impulsive actions. They may have attention deficit disorder (ADD), which includes an impaired ability to maintain attention and difficulty focusing on specific tasks. About one-third of individuals with FXS have features of autism spectrum disorder that affect communication and social interaction. Seizures occur in about 15% of males and about 5% of females with FXS.

Source: https://ghr.nlm.nih.gov/condition/fragile-x-syndrome