SRC Approves Dose Escalation in ADOA Clinical Trial

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Stock PYC Therapeutics Ltd (PYC.ASX)
Release Time 11 Jun 2025, 9:17 a.m.
Price Sensitive Yes
 SRC Approves Dose Escalation in ADOA Clinical Trial
Key Points
  • PYC is developing a precision therapy (PYC-001) for the blinding eye disease Autosomal Dominant Optic Atrophy (ADOA)
  • The Safety Review Committee has approved escalation of the PYC-001 dose from 10 to 30 micrograms per eye
  • PYC will continue to generate human safety and efficacy data for this first-in-class drug candidate through 2025
Full Summary

PYC Therapeutics, a clinical-stage biotechnology company, announced that the Safety Review Committee (SRC) monitoring the Single Ascending Dose (SAD) study for its investigational drug candidate PYC-001 has approved the escalation of the PYC-001 treatment dose from 10 micrograms per eye (cohort 2) to 30 micrograms per eye (cohort 3). This decision was made following the evaluation of the safety and tolerability data for patients in cohort 2 through 4-weeks of follow-up. PYC-001 is being developed as a precision therapy to address the underlying cause of Autosomal Dominant Optic Atrophy (ADOA), a blinding eye disease that begins in childhood and leads to legal blindness in middle age in most patients. There are currently no treatment options available for ADOA, which represents an estimated >$2 billion per annum addressable market. PYC-001 utilizes the company's proprietary drug delivery technology to overcome the major challenge for RNA drugs by ensuring sufficient drug reaches its target inside the cells affected by ADOA. The ongoing SAD study is followed by a Multiple Ascending Dose (MAD) study and an Open-Label Extension (OLE) study, which will facilitate repeat dosing and evaluation of the optimal dosing regimen of PYC-001, as well as an assessment of its safety/tolerability and efficacy profile. Successful completion of the SAD and MAD studies will lead to the initiation of a registrational Phase 2/3 trial aimed at supporting a New Drug Application for PYC-001. PYC will continue to update shareholders on the progress of this high-velocity clinical development program.

Outlook

PYC will continue to generate human safety and efficacy data for this first-in-class drug candidate PYC-001 through the course of 2025.