One of the milestones flagged for this quarter is the completion of the full pre-clinical data pack for PYC-003 in Autosomal Dominant Polycystic Kidney Disease (ADPKD) and regulatory submission to progress to first-in-human trials.
ADPKD is the most common lethal single-gene disorder in the world, affecting 1 in every 1,000 people worldwide. It is estimated to have an addressable market size of US$10bn, yet still qualifies for FDA and EMA orphan drug status and the substantial benefits that come with that.
PYC intends to submit an application for PYC-003 to commence in clinical trials to both the TGA and FDA. It is anticipated that the trials will commence in Australia early in 2025.
With plans to apply for FDA accelerated approval (that is, early approval for marketing based on Phase 1/2 data with confirmatory Phase 3 data to follow post-approval), PYC hopes to submit an NDA to the FDA (which, as an orphan drug, would be eligible for 6 month priority review) in 2027.
This week, Sangamo Therapeutics announced that it has aligned with the FDA on an accelerated approval pathway for its gene therapy for Fabry disease. Fabry disease is a multisystemic lysosomal disorder which, in the kidneys, leads to proteinuria and chronic kidney failure. Sangamo presented the FDA with promising data from an ongoing Phase I/II study, which includes preliminary evidence of improved kidney function. Sangamo has already finished enrolment in the trial, which has dosed 33 patients.
Based on Sangamo’s Phase 1/2 data, the FDA has agreed that eGFR slope at 1 year can serve as an intermediate clinical endpoint to support a potential Accelerated Approval and that eGFR slope at 2 years may be assessed to verify clinical benefit.
According to Sangamo, the accelerated approval pathway could speed up its gene therapy’s time-to-market by three years.
PYC is proposing to use Total Kidney Volume (TKV) imaging as a surrogate endpoint (changes in TKV are correlated to changes in eGFR) in its Phase 1/2 trial with measurement of eGFR to be used to verify clinical benefit.
The FDA has already listed TKV as a surrogate endpoint which could be appropriate for use as a primary efficacy clinical trial endpoint for marketing approval, even though it has not yet been used to support an approved NDA or BLA.
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