PYC Therapeutics (ASX:PYC) has nominated a fourth clinical drug candidate, PYC-002, targeting Phelan-McDermid Syndrome (PMS), a rare neurodevelopmental disorder with no current treatment options.
The Perth-based biotechnology company is progressing PYC-002 into an Investigational New Drug (IND) pathway, with human trials expected to begin in the first half of 2026. The decision follows the successful completion of pre-clinical studies that demonstrated the drug’s potential to restore normal gene expression linked to PMS.
PMS, which affects approximately 1 in 10,000 people, is caused by insufficient expression of the SHANK3 gene in brain neurons. This deficiency disrupts communication between neurons, leading to severe developmental issues. PYC-002 addresses this underlying cause, with animal models showing it increases SHANK3 gene expression in critical brain regions. In neurons derived from PMS patients, the drug was able to restore SHANK3 expression to levels seen in unaffected individuals.
PYC's Chief Executive Officer, Dr Rohan Hockings, called the milestone significant for the PMS community. “We are very pleased for the PMS community to be progressing this drug candidate into human trials. The data supporting this milestone show great potential for the first RNA therapy in this indication.”
He also noted that the progress draws on insights from other RNA therapies in similar diseases, which could expedite the path to patient impact.
PYC Therapeutics is a clinical-stage biotechnology firm focused on RNA therapies for genetic diseases. The company's development pipeline includes treatments for conditions such as retinitis pigmentosa, autosomal dominant optic atrophy, and autosomal dominant polycystic kidney disease.
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