Kidney Week

Abstract: FR-PO0498

Renal Autologous Cell Therapy for CKD with Diabetes: Pooled Safety and Efficacy Results from a Phase 2 Program in a Subset of Participants with Key Phase 3 Eligibility Characteristics

Session Information

• CKM: Clinical - Trials, Epidemiology, and Biomarkers
  October 23, 2026 | Location: Exhibit Hall A, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Cardiovascular-Kidney-Metabolic Health

• 602 Cardiovascular-Kidney-Metabolic Health: Clinical

Authors

• Ali, Farah N., ProKidney LLC, Winston-Salem, North Carolina, United States

Farah N. Ali, MD, MS

• Butler, Emily Lynn, ProKidney LLC, Winston-Salem, North Carolina, United States

Emily Lynn Butler, PhD

• Holmes, Ashley A., ProKidney LLC, Winston-Salem, North Carolina, United States

Ashley A. Holmes, MS

• Stavas, Joseph, ProKidney LLC, Winston-Salem, North Carolina, United States

Joseph Stavas, MD, MPH

• Cizman, Borut, ProKidney LLC, Winston-Salem, North Carolina, United States

Borut Cizman, MD

• Culleton, Bruce F., ProKidney LLC, Winston-Salem, North Carolina, United States

Bruce F. Culleton, MD

Background

Despite new treatments to delay disease progression in pts with diabetes and CKD, progression to ESKD remains a concern. Rilparencel is under investigation as a potential autologous cell therapy to preserve kidney function. We sought to understand long term safety and efficacy in a subset of pts from a pooled Ph2 program in which baseline characteristics align with key eligibility criteria of the ongoing Ph3 registrational trial, REGEN-006.

Methods

We analyzed pooled efficacy and safety data from Ph2 studies in pts with T2DM and CKD that met key eligibility criteria of REGEN-006. Study RMCL-002 and study REGEN-003 administered 2 injections over 6 months. Study REGEN-007 had two cohorts: Cohort 1 received 2 injections over 3 months; Cohort 2 received 1 injection and a 2^nd injection conditionally for decline in eGFR and/or increase in UACR. Study REGEN-008 is a non-interventional long term follow up study that enrolled previously treated pts.

Results

61 pts met key eligibility criteria of REGEN-006. Baseline mean age was 65 yrs (SD 9) and mean eGFR was 24 ml/min/1.73m²(SD 5). Of the 61 pts, 51 pts had an eGFR measurement 12 months after 1^st injection; the average change from baseline (SD) in eGFR was -0.29 (5.8). Of those, 42 pts had an eGFR measurement 18 months after 1^st injection and 25 pts had an eGFR 24 months after 1^st injection; the average change from baseline (SD) was -1.76 (5.6) and -2.43 (6.5), respectively. The pooled annualized eGFR slope after first injection was -1.78 ml/min/1.73m²/year.

Of 61 biopsies (n=61), related and possibly related SAEs (as determined by the PI) occurred in 3 pts; of 111 injections, procedure related and possibly related SAEs occurred in 4 pts; possibly related rilparencel SAEs occurred in 1 pt (cardiac arrest, acute respiratory failure, ventricular tachycardia, septic shock). 48% of pts experienced a related or possibly related TEAE; most common were injection site pain, subcapsular renal haematoma, renal haematoma, fatigue, procedural nausea and procedural pain, each reported in <10% of pts. No procedure- or product-related deaths were reported.

Conclusion

Cortical rilparencel injections may preserve kidney function with an acceptable safety profile. A multi-center, blinded, Ph3 randomized controlled trial is ongoing.

Funding

• Commercial Support – ProKidney