Our lead clinical programmes target the Kv3 family of potassium channels to treat Fragile X syndrome and rare epilepsy syndromes associated with defects in ion channels (channelopathies) or alterations in brain circuits (reduced cortical inhibition).
Increasing the probability of clinical success
To increase the probability of clinical success across our programmes we target rare genetic disorders, where heterogeneity in the patient population is reduced and where there is a strong understanding of the pathophysiology linked to specific ion channels. In addition, we seek out and utilise translational biomarkers that can be used to confirm target engagement, track treatment response and enable patient stratification.
Clinical and preclinical programmes
While our lead clinical programmes are focused on two different rare genetic CNS disorders, we have ongoing preclinical studies engaged in identifying and validating backup Kv3 modulator compounds for a broader range of CNS disorder, including schizophrenia and hearing loss.
- Target / Compound
- Lead Optimization
- Phase I
- Phase II
Fragile X Syndrome
Orphan Epilepsy Syndromes
Back up modulators
linked ion channel
Alzheimer’s Disease, Frontotemporal Dementia and Hyperexcitability Disorders
Lysosomal Channel 1
Alzheimer’s Disease, Parkinson’s Disease and Lysosomal Storage Disorders
Lysosomal channel 2
Parkinson’s Disease and Lysosomal Storage Disorders
Vascular Dementia, Alzheimer’s Disease and Stroke
Early-stage development programmes
At Autifony we are harnessing our ion channel drug discovery platform to build a robust early-stage portfolio of programmes with exclusive chemical equity against a range of novel lysosomal, vascular and neuronal ion channel targets.
These programmes have the potential to deliver novel treatments for neurodegenerative disorders as well as a broader range of CNS disorders, including epilepsy and neuropathic pain.
We work with pharma partners and academic collaborators to complement our internal capabilities to catalyse the progression of our small molecules towards the clinic.