Can PYC’s RNA Therapies Overcome Clinical and Regulatory Hurdles in 2025?

Clinical Progress Across Four Genetic Disease Programs

PYC Therapeutics, an ASX-listed biotechnology company focused on RNA therapeutics, has delivered a robust update for the second quarter of 2025. The company is advancing four drug candidates targeting severe genetic diseases with no current treatment options. These include Polycystic Kidney Disease (PKD), Retinitis Pigmentosa type 11 (RP11), Autosomal Dominant Optic Atrophy (ADOA), and Phelan-McDermid Syndrome (PMS).

In PKD, a condition affecting over 10 million people worldwide, PYC has initiated dosing in the combined Phase 1a/1b clinical trial, completing dosing in the first two cohorts of the single ascending dose study. This early-stage trial is a precursor to a registrational Phase 2 trial, which the FDA has indicated could support drug approval based on a 12-month study.

Encouraging Data and Regulatory Alignment in Eye Disease Programs

The company’s lead asset, targeting RP11, a rare blinding eye disease of childhood, has shown clinical proof of concept with improved vision in treated patients. PYC presented these promising results at major international conferences and has aligned with the FDA on the framework for registrational trials to support a New Drug Application. Meanwhile, the ADOA program has progressed through dosing of the third patient cohort in its Phase 1 single ascending dose study, with plans to initiate a combined Phase 1/2 multiple ascending dose study later this year.

Advancing Preclinical Programs Toward Human Trials

On the neurodevelopmental front, PYC’s PMS program has presented compelling preclinical data demonstrating restoration of missing gene expression in brain cells. This supports the planned transition into human clinical trials in 2026. The company is also progressing through necessary toxicology studies to enable this transition.

Financial Position and Outlook

Following a successful $146 million capital raise earlier this year, PYC reported $153 million in cash at the end of June 2025, with an additional $20 million expected from R&D rebates in the second half of the year. This strong cash position extends the company’s runway well into 2027, providing ample resources to fund ongoing clinical development and regulatory activities.

Looking ahead, PYC anticipates multiple key milestones in the second half of 2025, including completion of dose escalation studies, initiation of patient dosing in later trial phases, updated clinical data releases, and further regulatory engagements with the FDA. These developments will be critical in advancing PYC’s transition from clinical-stage to commercial-stage biotechnology company.