AZD1613 / AstraZeneca - LARVOL DELTA

PIONEER-PKD: Safety, Tolerability and Pharmacokinetics of AZD1613 in Adults With Autosomal Dominant Polycystic Kidney Disease (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: AstraZeneca | Trial completion date: Jan 2027 ➔ May 2027 | Trial primary completion date: Jan 2027 ➔ May 2027

Trial completion date • Trial initiation date • Trial primary completion date • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease

AZD1613, human-specific monoclonal antibody against PAPP-A demonstrates favourable safety and tolerability profile in first-in-human Ph1 study (ERA 2026) - "Method In an inducible, kidney-specific Pkd1 knockout mouse model of ADPKD, we compared weekly subcutaneous dosing of a PAPPA inhibitor for 10 weeks with isotype control; tolvaptan (vasopressin V2receptor antagonism) served as positive control. AZD1613, a monoclonal antibody targeting PAPP ‑ A, showed good tolerability and dose ‑ dependent suppression of PAPP ‑ A activity in first ‑ in ‑ human testing. These results support continued development; the ongoing MAD study will refine safety, PK/PD, and dose selection for future trials."

Clinical • First-in-human • P1 data • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • IGF1 • PKD1 • PRKD1