PYC Therapeutics Secures Safety Committee Approval for Increased 2.4 mg/kg Dose in Phase 1b PKD Trial with 24-Month Extension Greenlit

PYC Therapeutics Advances PYC-003 Development in Polycystic Kidney Disease

PYC Therapeutics is advancing PYC-003, designed to target the root cause of polycystic kidney disease, through a structured clinical program. The Safety Review Committee overseeing the ongoing Phase 1b MAD study has approved dose escalation to 2.4 mg/kg, marking a key milestone following thorough safety and tolerability evaluations from earlier trial phases.

This decision was based on detailed safety assessments from Phase 1a Cohort B3, where PKD patients received 2.4 mg/kg and were monitored for four weeks post-dose, alongside safety data from the first three patients dosed at 1.2 mg/kg in Phase 1b Cohort C1 with one week follow-up after the second dose. This cautious dose escalation aligns with established clinical trial safety protocols.

24-Month Open-Label Extension Study Approved for Extended Patient Treatment

Alongside dose escalation, PYC announced approval for a 24-month open-label extension (Part D) of the Phase 1b MAD trial, allowing continuous PYC-003 dosing for participants completing Part C. This extension mirrors the anticipated duration of the planned registrational trial, contingent on regulatory approval.

The extension study facilitates collection of long-term safety, tolerability, and efficacy data under repeat dosing conditions. It also provides ongoing treatment access for trial participants. Human Research Ethics Committee and regulatory approvals have been secured, enabling eligible patients to continue PYC-003 treatment beyond the main Phase 1b period.

Upcoming Data Milestones and Clinical Development Timeline

Safety and efficacy data from the Phase 1a SAD study are expected in the second half of 2026. This study evaluates PYC-003’s safety and preliminary efficacy at escalating single doses in PKD patients, offering initial human data on the drug’s profile.

Data from the ongoing Phase 1b MAD study and the open-label extension are projected for 2027. These results will inform the design and initiation of a combined Phase 2/3 registrational trial aimed at supporting a New Drug Application for PYC-003 in PKD. All timelines and plans remain subject to regulatory alignment and inherent clinical development risks as outlined in prior ASX disclosures.

Precision Medicine Focus on Monogenic Genetic Diseases

PYC Therapeutics is committed to developing precision RNA therapies for genetic diseases with no current treatments. Utilizing a proprietary drug delivery platform, PYC enhances the potency of RNA therapeutics, targeting monogenic diseases caused by single-gene mutations.

Polycystic kidney disease, a monogenic disorder, aligns with PYC’s strategic focus. The company currently advances three clinical-stage programs, with PYC-003 for PKD among the most progressed candidates.

Safety Review Committee’s Role and Dose Escalation Strategy

The Safety Review Committee’s approval to escalate to 2.4 mg/kg in Phase 1b follows standard clinical governance, reviewing safety data from Phase 1a Cohort B3 patients dosed at 2.4 mg/kg and initial Phase 1b patients dosed at 1.2 mg/kg. The committee also approved escalation to 4 mg/kg in the Phase 1a SAD study but will consider a third Phase 1b dose cohort (0.4 mg/kg or 4 mg/kg) only after assessing dose dependency and accumulating further safety data expected in 2027.

Phase 1b Study Design and Objectives

The Phase 1b MAD study evaluates safety, tolerability, and optimal dosing of repeated PYC-003 administration, differing from the single-dose design of Phase 1a SAD. This repeat dosing is critical to establish appropriate regimens for longer-term patient use.

Successful Phase 1b completion and regulatory alignment aim to enable initiation of a combined Phase 2/3 registrational trial to support PYC-003’s New Drug Application for PKD. Progression depends on regulatory approvals and compliance.

Addressing Unmet Needs in Polycystic Kidney Disease

PKD remains an area of significant unmet medical need, with limited therapies targeting its underlying cause. PYC-003 is developed to address PKD’s pathophysiology rather than just symptom management. PYC’s RNA therapeutic platform aims to deliver enhanced potency compared to traditional treatments.

The open-label extension allowing up to 24 months of continuous dosing underscores PYC’s commitment to generating robust long-term safety and efficacy data to support PYC-003’s clinical value.

Clinical Trial Registration and Additional Resources

PYC’s Phase 1 study of PYC-003 in autosomal dominant polycystic kidney disease is registered under clinical trial identifier NCT06714006, providing transparency on protocol, eligibility, and sites for patients, clinicians, and researchers.

Further corporate and investor information is available at pyctx.com, with investor inquiries directed to [email protected]. PYC also maintains updates via LinkedIn on clinical progress and corporate news.

Forward-Looking Statements and Risk Factors

PYC’s update includes forward-looking statements regarding clinical timelines, data outcomes, and regulatory pathways, subject to risks and uncertainties that may cause actual results to differ materially. These include factors beyond PYC’s control inherent in drug development and regulatory review.

Investors should note that PYC has no obligation to update forward-looking statements based on new information or future events. The timing of data releases, regulatory approvals, and commercialization remain uncertain. Additional risk disclosures are detailed in PYC’s previous ASX filings, including the February 2, 2026 release.