PYC Therapeutics says studies show program effective in key models

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PYC Therapeutics has announced the results of safety and efficacy studies conducted in non-human primates (NHPs) in its second drug development program.

The company said the program aims to be the first treatment available for a progressive and irreversible blinding eye disease of childhood called Autosomal Dominant Optic Atrophy (ADOA).

ADOA affects 1 in every 35,000 people. It is caused by insufficient levels of a specific protein called OPA1 in the retina of patients with the condition.

This NHP study aimed to show that PYC’s drug candidate (PYC001) could increase the amount of OPA1 protein in the retina of NHPs at a safe and well-tolerated dose. The company said the results demonstrated that PYC-001 can successfully achieve this objective.

“These results are very promising for patients with ADOA,” said PYC’s CEO Dr Rohan Hockings. “Drugs targeting monogenic diseases already have the highest likelihood of success in human trials. Now we can link this data from NHPs with the results that we have generated in the ‘retina in a dish’ models from patients with ADOA to demonstrate a fully integrated data pack suggesting that we can stop ADOA in its tracks.”