ProKidney (PROK) Study Update summary

Clinical study background and rationale

• Rilparencel (REACT) is an autologous cell therapy aimed at preserving kidney function in advanced CKD patients, particularly those with diabetes, who have limited options beyond dialysis or transplantation.

• The therapy involves harvesting, expanding, and reinjecting a patient's own kidney cells, with pivotal phase III and multiple phase II trials underway.

• The clinical program targets stage 3b/4 CKD caused by type 2 diabetes, with potential for label expansion to long-term dialysis prevention.

• No current therapies provide long-term kidney function preservation for high-risk CKD patients, who often progress to dialysis.

• The program is supported by a leadership team with over 150 years of combined experience in biotechnology and clinical development.

Study design and patient population

• REGEN-007 phase II trial enrolled 53 subjects, with 27 randomized to Group 1; 24 received at least one injection, and 13 had 12 months follow-up after two injections.

• Group 1 dosing mirrors phase III protocol, with key entry criteria including CKD with diabetes, eGFR 20–50, and HbA1c <10%.

• A subgroup of 10 subjects met phase III inclusion criteria, with similar demographics and baseline kidney function.

• Baseline eGFR averaged ~30 mL/min; less than one-third used SGLT2 inhibitors at baseline.

• Group 2 included 25 patients, with re-dosing triggered by sustained decline in eGFR or increase in UACR.

Efficacy and safety outcomes

• In Group 1, average eGFR change at 18 months post-1st injection was -1.3 ml/min/1.73m²; the phase III eligible subgroup (n=10) showed -0.6 ml/min/1.73m².

• Matched external controls experienced a mean eGFR decline of -6.2 ml/min/1.73m² over 18 months.

• No rilparencel-related serious adverse events were observed; safety profile was consistent with prior studies and comparable to kidney biopsy.

• Only one early termination (cardiovascular death, unrelated to therapy) occurred in Group 1.

• Group 2 patients showed variable responses, with some experiencing significant declines after re-dosing triggers.