Kidney Week

Abstract: TH-PO1177

Rilparencel Autologous Cell Therapy in Patients with Diabetes and Advanced CKD: Phase 2 Interim Results

Session Information

• Late-Breaking Science Posters
  October 24, 2024 | Location: Exhibit Hall, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: CKD (Non-Dialysis)

• 2302 CKD (Non-Dialysis): Clinical, Outcomes, and Trials

Authors

• Stavas, Joseph, ProKidney, Raleigh, North Carolina, United States

Joseph Stavas, MD, MPH

• Silva, Arnold L., Boise Kidney & Hypertension Institute, Boise, Idaho, United States

Arnold L. Silva, MD, PhD

• Saad, Theodore F., Nephrology Associates, Newark, Delaware, United States

Theodore F. Saad, MD, FASN

• Butler, Emily Lynn, ProKidney, Raleigh, North Carolina, United States

Emily Lynn Butler, PhD

• Tangri, Navdeep, University of Manitoba, Winnipeg, Manitoba, Canada

Navdeep Tangri, MD, PhD

• Culleton, Bruce F., ProKidney, Raleigh, North Carolina, United States

Bruce F. Culleton, MD

Background

Cell-based therapies in patients with chronic kidney disease (CKD) may repair diseased nephrons and stabilize kidney function. We describe an interim analysis of an open label phase 2 trial (NCT05018416) with rilparencel, an autologous cell product made from kidney biopsy tissue.

Methods

The study objectives are to evaluate efficacy and safety measured by change in eGFR slope from baseline to after last injection and procedure- and product-related serious adverse events. Patients with type 1 or 2 diabetes, eGFR 20-50 ml/min/1.73/m² and UACR 30-5000 mg/g are randomized into one of two cohorts. Cohort 1 subjects receive two percutaneous rilparencel injections: one into the cortex of each kidney 3 to 5 months apart; Cohort 2 subjects receive at least one injection, and a 2^nd injection only after a pre-defined biochemical trigger. eGFR slope is presented for only Cohort 1 given its alignment with an ongoing phase 3 trial. Safety data is aggregated across both cohorts. A post-hoc analysis compares eGFR slope in Cohort 1 with a 10:1 matched synthetic control group. The control group was identified from a subject-level clinical trial database and matched with Cohort 1 based on risk of kidney failure (Klinrisk model).

Results

49 subjects received the 1^st injection (n=24 Cohort 1; n=25 Cohort 2) and 34 subjects received a 2^nd injection (n=22 Cohort 1; n=12 Cohort 2); 28 subjects have not completed the study and remain in follow up (n=11 Cohort 1; n=17 Cohort 2). 13 Cohort 1 subjects have greater than 12 months follow up post 2^nd injection. Mean age is 63 yrs; 54% are female. At baseline, mean (SD) eGFR is 27.9 (9.5) ml/min/1.73/m²; median [IQR] UACR is 239 [4, 2392] mg/g. Annualized eGFR slope in Cohort 1 is -1.1 ml/min/1.73/m²/year (95% CI -3.5, 1.3). Average change in eGFR in the matched control group is -4.0 ml/min/1.73/m² over 12-months. Two hematomas and 2 episodes of acute kidney injury occurred after kidney biopsy; 1 hematoma occurred after 83 injections. There were no product-related SAEs.

Conclusion

Interim findings suggest rilparencel can stabilize kidney function in patients with diabetes and advanced CKD. Procedure-related AEs are infrequent and typical given the interventions. A global phase 3 study is ongoing to determine efficacy and safety of rilparencel.

Funding

• Commercial Support – ProKidney