How Is PYC Therapeutics Advancing Four RNA Drug Candidates with $121M Cash?

Progress Across Four Genetic Disease Programs

PYC Therapeutics, a clinical-stage biotechnology company specialising in RNA therapeutics, has delivered a robust update for the fourth quarter of 2025. The company is advancing a pipeline of four investigational drug candidates aimed at treating severe genetic diseases with no current therapies. These include Polycystic Kidney Disease (PKD), Autosomal Dominant Optic Atrophy (ADOA), Retinitis Pigmentosa type 11 (RP11), and Phelan-McDermid Syndrome (PMS).

During Q4, PYC completed a critical safety and tolerability review of its PKD drug candidate in healthy volunteers, enabling progression to patient dosing in the combined Phase 1a/1b study. This milestone is a key step toward a registrational Phase 2/3 trial, which could potentially bring a first-in-class treatment to over 10 million people worldwide suffering from PKD.

Advancing Clinical Proof of Concept

In the ADOA program, PYC initiated a global repeat dose Phase 1/2 study designed to establish clinical proof of concept for its lead candidate. ADOA is a progressive blinding eye disease affecting approximately 1 in 35,000 people, and this trial represents a significant step toward validating the therapeutic potential of PYC’s RNA approach.

Meanwhile, the RP11 program, targeting a rare childhood blinding disorder affecting 1 in 100,000 people, showcased encouraging longer-term data. Patients treated with PYC’s drug candidate demonstrated sustained vision improvement up to 18 months post-treatment, reinforcing the promise of this therapy. The company plans to engage with the US Food and Drug Administration (FDA) early in 2026 to align on a registrational trial design, aiming to support a New Drug Application.

Preclinical Advances and Future Trials

For Phelan-McDermid Syndrome, a severe neurodevelopmental disorder, PYC presented compelling preclinical data including successful studies in non-human primates. These findings underpin the company’s plan to submit an Investigational New Drug (IND) application to the FDA in the first half of 2027, paving the way for first-in-human trials.

Financial Position and Outlook

Financially, PYC Therapeutics remains well-capitalised with $121 million in cash at the end of December 2025, bolstered by an expected $20 million R&D rebate in the first quarter of 2026. This strong cash runway supports ongoing clinical studies, regulatory submissions, and discovery programs. Operating cash outflows for the quarter were $13.76 million, reflecting continued investment in research and development.

The company’s strategic focus on RNA therapeutics for monogenic diseases leverages the modality’s precision to increase gene expression without overexpression risks, a factor that could accelerate clinical success. PYC’s upcoming milestones, including trial readouts and regulatory interactions, will be closely watched by investors and industry observers alike.