PYC Therapeutics (ASX:PYC) has named its fourth clinical drug candidate, PYC-002, targeting Phelan-McDermid syndrome (PMS), a rare neurodevelopmental disorder for which there are currently no treatment options.

The Perth-based biotechnology company is progressing PYC-002 through the investigational new drug (IND) pathway, with human trials expected to begin in the first half of 2026. This decision follows the successful completion of preclinical studies that demonstrated the drug’s potential. Restores normal gene expression associated with PMS.

PMS affects approximately 1 in 10,000 people and is caused by underexpression of the SHANK3 gene in brain neurons. This defect disrupts communication between neurons, causing serious developmental problems. PYC-002 addresses this underlying cause and has been shown in animal models to increase SHANK3 gene expression in key brain regions. In neurons from PMS patients, the drug was able to restore SHANK3 expression to levels seen in unaffected individuals.

PYC Chief Executive Dr Rohan Hockings said this was an important milestone for the PMS community. “We are very pleased that the PMS community is advancing this drug candidate into human clinical trials. The data supporting this milestone show great potential for the first RNA therapy in this indication. It shows that there is.

He also said this advance builds on insights from other RNA therapies in similar diseases, which could accelerate the impact on patients.

PYC Therapeutics is a clinical-stage biotechnology company focused on RNA therapeutics for genetic diseases. The company’s development pipeline includes treatments for retinitis pigmentosa, autosomal dominant optic atrophy, and autosomal dominant polycystic kidney disease.

Shares closed 4.33% lower at $1.44 on Monday. However, it is up 44.95% since the beginning of the year.