PYC Therapeutics (ASX:PYC) has announced significant progress in its efforts to develop RNA-based precision medicines for genetic diseases. The company is in the midst of clinical trials for its leading drug candidates targeting rare eye conditions, including Retinitis Pigmentosa type 11 (RP11) and Autosomal Dominant Optic Atrophy (ADOA). In a recent update, PYC reported positive outcomes from early-stage studies, with multiple patients showing improvements in vision after receiving a single dose of the investigational drug for RP11. This drug, VP-001, has received Orphan Drug Designation from the US Food and Drug Administration, providing benefits such as tax credits and potential market exclusivity.
RP11 and ADOA are genetic eye disorders that lead to progressive vision loss. RP11, caused by mutations in the PRPF31 gene, results in the gradual degeneration of the retina, leading to night blindness and a narrowing field of vision. In contrast, ADOA, linked to OPA1 gene mutations, causes the optic nerves to deteriorate, affecting central vision and colour perception.
CEO Glenn Noronha highlighted the importance of these milestones, stating, “We are excited by the progress of VP-001, especially as it remains the first drug candidate targeting RP11 to advance into human trials. The FDA’s recognition of its potential is a testament to our commitment to bringing precision RNA therapies to patients who have no other options”.
The company's pipeline extends beyond eye diseases, with upcoming trials planned for Autosomal Dominant Polycystic Kidney Disease (ADPKD) and Phelan-McDermid Syndrome, a severe neurodevelopmental disorder. Both programs are expected to advance in early 2025.
Financially, PYC remains stable, with $45.5 million in cash reserves as of 30 September 2024. The company also anticipates receiving a $17 million R&D tax rebate in the December quarter.
