PYC-003 Phase 1a data presented at ASN Kidney Week
In its American Society of Nephrology (ASN) Kidney Week presentation on 22 October 2026 (Denver local time, Abstract TH-OR060), Dr. Jagadeesh Kurtkoti of Renal Medicine, South Coast Renal, Southport, Queensland, outlined Part B single ascending dose (SAD) safety and exploratory efficacy data for PYC-003 in patients with autosomal dominant polycystic kidney disease (ADPKD). The study is sponsored by PYC Therapeutics (ASX:PYC), and the company released the presentation on 23 October 2026.
The headline finding was that the presentation reported no treatment-related serious adverse events or discontinuations. It marks human data for a candidate designed to address the underlying cause of polycystic kidney disease.
When big ASX news breaks, our subscribers know first
Why ADPKD is a target for PYC-003
The disease burden
ADPKD is a progressive condition in which cysts steadily enlarge the kidneys and reduce kidney function. According to the presentation, there is no disease-modifying therapy.
Key figures cited in the presentation:
- Affects 1 in 1,000 people, or 12.5 million people worldwide
- Around 80% of cases are driven by PKD1 mutation, which causes PC1 deficiency (PC1 is the protein PYC-003 aims to restore)
- Around 50% of patients progress to end-stage kidney disease by age 60
How PYC-003 works
PYC-003 is a peptide-oligonucleotide conjugate. The presentation states it selectively blocks miR-17 binding to PKD1 mRNA, restoring PC1 expression while preserving miR-17 regulation of other RNAs.
The presentation compared three states:
- Unaffected individual: two functional PKD1 mRNA transcripts and normal PC1 protein levels.
- ADPKD: one functional PKD1 mRNA transcript, reduced PC1 protein levels, and cyst growth with progressive dysfunction.
- PYC-003 treated: the miR-17 binding site on the functional transcript is blocked, PKD1 mRNA is protected, and PC1 protein levels increase.
The approach targets the cause of the disease rather than its symptoms.
Safety and tolerability results
Part B was an open-label SAD study in ADPKD participants across three cohorts: B1 at 0.4 mg/kg, B2 at 1.2 mg/kg and B3 at 2.4 mg/kg, with six participants each. Safety was evaluated through Day 28, with independent safety review before each dose escalation and no protocol-defined stopping rule triggered.
Part A in healthy volunteers (n=32) is complete. Parts C and D (multiple ascending dose, or MAD, plus open-label extension) are ongoing.
| Measure | B1 (0.4 mg/kg, n=6) | B2 (1.2 mg/kg, n=6) | B3 (2.4 mg/kg, n=6) | Part A (HV, n=32) |
|---|---|---|---|---|
| Total TEAEs | **9** | **8** | **12** | **33** |
| Participants with ≥1 related TEAE | **0** | **1** | **1** | **3** |
| Total TESAEs | **1** | **0** | **0** | **0** |
| Treatment-related TESAEs | **0** | **0** | **0** | **0** |
| TEAEs leading to discontinuation | **0** | **0** | **0** | **0** |
TEAE refers to treatment-emergent adverse events, and TESAE to treatment-emergent serious adverse events.
- All adverse events were mild to moderate.
- The single serious adverse event, mild transient amnesia in B1, was considered unrelated to study drug and has resolved.
The presentation stated there were no clinically meaningful treatment-related changes through Day 28 in hepatic markers (ALT, AST), renal and tubular markers (creatinine, urea, KIM-1, NGAL) or electrolytes (potassium, magnesium).
A Day 14 elevation in one B3 participant occurred with flu-like symptoms and was assessed as unrelated to study treatment.
Early efficacy signals in urinary PC1 and kidney volume
Urinary PC1 shows directional increase
The presentation described a directional increase in urinary PC1, an exploratory read-out of renal PC1 expression and a measure of target engagement. The analysis combined the 1.2 mg/kg and 2.4 mg/kg cohorts.
Sample sizes were small. Combined participant numbers were 8 at Day -1 and fell to 3 at Day 42.
The 1.2 mg/kg data come from samples collected after a first dose in the MAD study, and earlier SAD cohorts’ samples were excluded because they were collected under different conditions.
Total kidney volume (TKV) trend
Total kidney volume (TKV) is a measure of kidney size and a marker of disease progression. The presentation compared B1 (n=6) and pooled B2/B3 (n=12 at Month 3; n=11 at Month 4) against an illustrative natural progression of untreated PKD (n=925, mean +6.2% annually).
This is a comparison across studies, not a controlled one. The presentation’s conclusion on early TKV was:
Presentation conclusion
“Early TKV change after a single dose is directionally encouraging but requires confirmation in repeat-dose cohorts.”
What’s next for PYC-003
The presentation set out four conclusions for Part B single-dose PYC-003:
- Safe and well tolerated, with no treatment-related serious adverse events or discontinuations.
- No safety biomarker signal, with no evidence of renal or liver injury.
- Target engagement, shown by a directional increase of exosomal PC1, an exploratory read-out of renal PC1 expression.
- Early TKV that is directionally encouraging, but requires confirmation in repeat-dose cohorts.
Repeat-dose MAD and open-label extension studies are ongoing at 1.2 and 2.4 mg/kg every six weeks (Q6W), with data expected in 2027. For investors, the single-dose safety data provide a clean tolerability record ahead of those repeat-dose readouts, though the efficacy signals remain exploratory.
The study is registered under clinical trial identifier NCT06714006, with 10 sites in Australia, 1 in New Zealand and 1 in Hong Kong.
Don’t Miss the Next Healthcare Breakthrough
Get FREE breaking ASX healthcare news delivered to your inbox within minutes of release, complete with in-depth analysis and the full announcement. Join 20,000+ investors already ahead of the market. Click the “Free Alerts” button at Big News Blast to receive alerts the moment market-moving news breaks.