PYC Therapeutics (PYC) investor relations material

Disease rationale

• ADPKD affects 12.5 million people worldwide; ~50% progress to end-stage kidney disease by age 60.

• PKD1 mutation-driven PC1 deficiency is the main cause in ~80% of cases; cyst growth enlarges kidneys and reduces function.

Mechanism and study design

• PYC-003 blocks miR-17 binding to functional PKD1 mRNA, aiming to restore PC1 while leaving other miR-17 target mRNAs unaffected.

• Global Phase 1a/1b includes placebo-controlled SAD in 32 healthy volunteers and open-label ADPKD SAD cohorts at 0.4, 1.2 and 2.4 mg/kg, with n=6 per cohort.

• Primary endpoint is safety and tolerability; exploratory assessments include plasma and urinary PK, uPC1, TKV and eGFR.

• Open-label MAD and extension studies are ongoing; repeat-dose cohorts include 1.2 and 2.4 mg/kg Q6W, with data expected in 2027.

Single-dose safety

• No treatment-related serious AEs or discontinuations; all AEs were mild to moderate, and no protocol-defined stopping rule was triggered.

• Participants with ≥1 related TEAE: B1 0, B2 1 and B3 1; no treatment-related TESAEs across the three ADPKD cohorts.

• No clinically meaningful treatment-related hepatic, renal or electrolyte marker changes through Day 28; a Day 14 elevation in one B3 participant with flu-like symptoms was assessed as unrelated.