PYC Therapeutics (ASX:PYC) has held a pre-IND meeting with the US FDA and announced it has agreed on a proposed pathway through clinical trials for its second investigational candidate.
The candidate is being investigated for the treatment of a blinding eye disease of childhood called Autosomal Dominant Optic Atrophy (ADOA).
ADOA affects one in every 35,000 people, and no treatment options are currently available. The company says it expects to progress the candidate to human trials in the first half of 2024
ADOA is caused by a mutation in one copy of the OPA1 gene. In around 85 per cent of patients, the mutation leads to insufficient levels of OPA1 gene expression to support normal cellular function in the retinal ganglion cells of the eye. Loss of retinal ganglion cells due to cell death interrupts the normal processing of the visual signal from the retina to the brain leading to the loss of vision in ADOA patients.
PYC says its investigational drug candidate for ADOA - known as PYC-001 - is a precision therapy that aims to restore the expression of the OPA1 gene back to levels required for the normal function of the retina.