ProKidney (PROK) Jefferies London Healthcare Conference 2024 summary

Company and therapy overview

• Focuses on phase 3 cell therapy rilparencel for advanced diabetic chronic kidney disease, addressing high unmet need.

• Therapy process involves renal cortex biopsy, cell expansion, and reinjection as a personalized treatment.

• Mechanism of action shows reduced inflammatory and fibrotic markers in final product; further updates expected in the second half of next year.

Clinical evidence and trial results

• Phase 2 trials (RMCL-002, REGEN-007) showed stabilization of kidney function, especially in stage 4 patients with elevated UACR.

• Bilateral kidney injections in REGEN-007 led to eGFR decline rates similar to healthy adults over 18 months.

• Safety profile aligns with standard kidney biopsy; patients and physicians report comfort with the procedure.

• Enrollment for phase 3 PROACT 1 is active, with sites being recruited.

Regulatory and trial design updates

• FDA confirmed a single phase 3 trial (PROACT 1) could support full U.S. approval, replacing the previous two-trial plan.

• PROACT 1 is a 685-patient, randomized, sham-controlled study with at least 80% power, targeting 120 events and a hazard ratio of 0.6.

• eGFR slope suggested as a surrogate endpoint for accelerated approval; details under internal discussion.

• No interim analyses planned due to proximity of final data readout.