ProKidney (PROK) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic Reset and Operational Progress

• Underwent a major reset in 2024, pausing and restarting manufacturing and phase 3 clinical programs, repositioning the product, and improving stakeholder communications.

• Remediation efforts led to resumption of patient treatment and significant progress in clinical studies.

• Enhanced focus on diversity in patient recruitment for ongoing trials.

• Cash runway now extends into early 2027, supported by a $140M stock offering in June 2024 and $407M in cash as of September 30, 2024.

• Headquarters in Boston, MA, and Winston-Salem, NC.

Clinical Development and Trial Updates

• Phase 2 study 002 showed stabilization of kidney function for up to 30 months in some groups, especially those with high risk (GFR <30 and high proteinuria).

• Interim analysis from REGEN-007 Phase 2 trial showed kidney function preserved for 18 months post-second injection, with no rilparencel-related serious adverse events.

• Externally matched controls experienced continuous decline, while rilparencel-treated subjects showed stabilization.

• Phase 3 PROACT 1 is a pivotal, sham-controlled study in over 600 patients with diabetes and CKD (eGFR 20–35), with topline results expected in Q3 2027.

• FDA validated the focus on a single phase 3 study and opened the possibility for accelerated approval using eGFR slope as a surrogate endpoint.

Product and Scientific Insights

• Therapy involves harvesting kidney tissue, expanding kidney cells, and re-injecting them into the patient’s kidneys, with dosing based on kidney size.

• Product is primarily composed of tubular and epithelial cells, which become more de-differentiated during manufacturing.

• Ongoing research focuses on understanding the mechanism of action and ensuring potency assurance for regulatory submissions.

• Safety profile is consistent with or better than historical kidney biopsy data, aided by controlled procedures and specialized training.

• No rilparencel-related serious adverse events observed in recent trials.