Safety Committee Greenlights Higher Dose in Untreated Blinding Eye Disease Trial

Clinical Milestone in ADOA Treatment Development

PYC Therapeutics (ASX:PYC), a clinical-stage biotech focused on precision medicines for genetic diseases, has announced a significant advancement in its clinical trial for Autosomal Dominant Optic Atrophy (ADOA). The Safety Review Committee (SRC) overseeing the Single Ascending Dose (SAD) study has approved escalating the dose of PYC-001 from 3 micrograms per eye to 10 micrograms per eye. This approval follows a thorough evaluation of safety and tolerability data from the first patient cohort after four weeks of follow-up.

This dose escalation represents a critical step in the clinical development of PYC-001, a first-in-class investigational RNA therapy designed to restore the expression of the OPA1 gene, which is deficient in ADOA patients. The disease, which leads to progressive vision loss and eventual legal blindness, currently has no approved treatments, underscoring the unmet medical need PYC aims to address.

PYC-001’s Precision Approach and Market Potential

PYC-001 leverages the company’s proprietary drug delivery platform to ensure effective intracellular delivery of RNA therapeutics, overcoming a major hurdle in this drug class. By targeting the root genetic cause of ADOA, PYC-001 holds promise as a disease-modifying therapy rather than merely symptomatic relief.

With an estimated patient population of around 7,500 in the Western world and orphan drug pricing benchmarks, the addressable market for ADOA treatments is projected to exceed AUD 2 billion annually. This market potential adds commercial significance to the clinical progress reported.

Broader Pipeline and Strategic Outlook

Beyond ADOA, PYC Therapeutics is advancing multiple RNA-based programs targeting other monogenic diseases, including Retinitis Pigmentosa type 11, Autosomal Dominant Polycystic Kidney Disease, and Phelan McDermid Syndrome. Each program is at various stages of clinical or preclinical development, reflecting PYC’s strategic focus on high-impact genetic disorders with limited or no treatment options.

The company anticipates continued generation of human safety and efficacy data for PYC-001 throughout 2025, with potential further dose escalations and regulatory discussions to optimize the clinical pathway. This measured approach aims to balance patient safety with the urgency of addressing a debilitating condition.

Implications for Investors and the Sector

PYC’s dose escalation approval is a positive signal for investors, indicating that early safety data supports advancing the trial. However, the ultimate clinical and commercial success will depend on forthcoming efficacy results and regulatory milestones. The broader RNA therapeutics sector continues to attract attention for its transformative potential, and PYC’s progress in ADOA adds a noteworthy chapter to this evolving story.