PYC Therapeutics (ASX:PYC), a clinical-stage precision medicine company, has reported new data that strengthen the case for its lead drug candidate, PYC-001, as a potential disease-modifying treatment for Autosomal Dominant Optic Atrophy or ADOA.
The company announced that a single administration of PYC-001 produced a sustained increase in OPA1 protein expression in the retina of non-human primates four months after dosing, and that patients treated in ongoing Phase 1/2 trials have shown sustained improvements in visual acuity alongside reductions in retinal stress.
The non-clinical results come from animals that received a 15 microgram dose per eye, a regimen that corresponds to the human equivalent of a 30 microgram dose under the study’s volumetric scaling. Immunofluorescence quantification of retinal ganglion cells showed a statistically significant increase in OPA1 signal at Day 113 following a single dose. Representative retinal images demonstrated markedly stronger OPA1 staining in the treated animals compared with controls, providing visual confirmation of the effect measured by the assays.
The company said that patients in the MYRTLE trial who have received repeated doses of PYC-001 have experienced improvements in low contrast visual acuity as well as decreases in mitochondrial stress measured by Flavoprotein Fluorescence. The company highlighted the association between reduced retinal stress and the vision gains, noting that these improvements have occurred with a favourable safety and tolerability profile and no treatment-related serious adverse events reported to date.
Patients are currently dosed every two to three months. The new data indicate the possibility of moving to a schedule of once every greater than four months. PYC says it will review and amend the clinical protocol where appropriate and subject to regulatory alignment and the usual risks and uncertainties.
