ProKidney (PROK) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic vision and clinical focus

• Aims to transform treatment for advanced chronic kidney disease (CKD) with rilparencel, an autologous cell therapy targeting high-risk patients facing kidney failure.

• FDA alignment on accelerated approval pathway using eGFR slope as a surrogate endpoint; pivotal Phase III topline results expected in Q2 2027.

• Over 150 patients treated in phase II trials, showing kidney function stabilization and favorable safety profile; phase III pivotal readout expected in Q2 2027.

• Focused on a single product, with robust clinical data, experienced leadership, and a cash runway extending past mid-2027.

• Manufacturing expansion underway in North Carolina, with two new buildings totaling 180,000 sq ft to support commercial launch and BLA submission.

Clinical development and study design

• Phase III PROACT 1 study targets advanced CKD and type 2 diabetes patients, using a randomized, sham-controlled, blinded design.

• Enrollment for accelerated approval efficacy analysis expected to complete by mid-2026, with top-line readout in Q2 2027.

• Power calculations for phase III are conservative, assuming a 1.5 ml/min difference in eGFR slope, despite phase II showing >4 ml/min difference.

• Subgroup analyses and safety are key components of the upcoming top-line readout.

• Target population for phase III narrowed to eGFR 20–35 to focus on highest-risk patients.

Rilparencel manufacturing and mechanism of action

• Manufactured from patient’s own kidney cells, with no gene editing, preconditioning, or immunosuppression required.

• Process involves kidney biopsy, cell expansion to over a billion cells, and bilateral kidney injections three months apart.

• Final product primarily contains tubular and epithelial cells with reduced inflammatory and fibrotic markers.

• Working hypothesis: injected cells exert anti-inflammatory effects and leverage innate kidney repair mechanisms, but do not form new nephrons.

• Ongoing R&D and mechanism of action studies, with data expected throughout 2026 and presentations at major conferences.