PYC Therapeutics announced the results of a study conducted in human 3-dimensional models derived from patients with end-stage renal failure due to Autosomal Dominant Polycystic Kidney Disease (PKD). The results demonstrate that an investigational drug candidate designed by PYC (known as PYC-003) to address this disease at its root cause is effective. These 3D patient-derived cyst models represent the 'gold-standard' pre-clinical assay for evaluating drug candidates in this indication.

PYC-003 is the fourth program in the Company's development pipeline ? joining three other first-in-class RNA drug candidates with disease-modifying potential currently progressing into or through human trials. PYC plans to accelerate PYC-003 into human trials following these results.

An Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) to enable the commencement of human trials for this drug candidate is planned for H2 2024. PYC-003 is expected to have an accelerated pathway through human trials due to the extent of the unmet patient need in PKD. A New Drug Application in support of this candidate could be submitted following two clinical trials rather than the conventional three.

PKD is a monogenic disease (meaning that it is caused by a mutation in a single gene). Drugs targeting monogenic diseases have the highest likelihood of demonstrating efficacy in clinical trials[12] and lower probability of off-target safety issues. PKD affects 1 in every 1,000 people across the globe.

There are currently no drugs available that address the underlying cause of the disease and approximately 50% of PKD patients will progress to end-stage renal failure by the age of 60. PKD is characterised by the formation of multiple fluid filled cysts throughout the kidney and, to a lesser extent, other organs. Progression of the cyst frequency and volume over time ultimately leads to destruction of the internal architecture and function of the kidney.

The data demonstrating that PYC-003 is effective in 3D patient-derived cyst models complements existing data from animal models highlighting the ability of this drug candidate to reach the cells affected in PKD[14]. PYC's drug delivery platform has already demonstrated the ability to deliver an RNA therapy to the target cells affected by the disease in vivo in high concentration.