Therapeutic PYC (ASX:PYC) has named a 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 advancing PYC-002 toward an investigational new drug (IND) pathway, with human trials expected to begin in the first half of 2026. The decision follows successful preclinical studies who demonstrated the potential of the drug. to restore normal expression of genes linked to premenstrual syndrome.
Premenstrual syndrome, which affects approximately 1 in 10,000 people, is caused by insufficient expression of the SHANK3 gene in neurons in the brain. This deficit disrupts communication between neurons, leading to serious developmental problems. PYC-002 addresses this underlying cause, with animal models showing that it increases SHANK3 gene expression in critical regions of the brain. In neurons derived from PMS patients, the drug was able to restore SHANK3 expression to levels seen in unaffected individuals.
PYC CEO Dr. Rohan Hockings called the milestone an important one for the PMS community. “We are very pleased that the PMS community is advancing this drug candidate toward human trials. The data supporting this milestone show great potential for the first RNA therapy in this indication.
He also noted that the advances build on knowledge of other RNA therapies for 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 conditions such as retinitis pigmentosa, autosomal dominant optic atrophy and autosomal dominant polycystic kidney disease.
Shares closed Monday down 4.33% at $1.44. However, they are up 44.95% year to date.
