Slide 1: Rilparencel Renal Autologous Cell Therapy for Patients with Stage 3-4 CKD and Type 2 Diabetes
Rilparencel Renal Autologous Cell Therapy for Patients with Stage 3-4 CKD and Type 2 Diabetes: Results from a Phase 2 Clinical Trial David Gerber1, George Bakris2, Arnold Silva3, Steven Coca4, Anna Burgner5, Emily Butler6, Bruce Culleton6, and Joseph Stavas6 1University of Cincinnati College of Medi
Slide 2: About ProKidney
About ProKidney Cell-based biotechnology company located in the United States 18+ years of proprietary discovery in kidney disease cell therapy 11+ years Phase 1-3 clinical trials in subjects with moderate to severe CKD Company owned manufacturing and operations Investigational product, rilparencel
Slide 3: Trial Objectives and Endpoints
Trial Objectives and Endpoints • To assess the safety and efficacy of rilparencel in subjects with Trial Objective type 2 diabetes (T2DM) and moderate-to-severe chronic kidney disease (CKD) • Change in kidney function, determined by serial measurements Primary Efficacy Endpoint of eGFR • Procedural-
Slide 4: Study Design
Study Design EOS* 1st 2nd Month 24 follow- rilparencel rilparencel up after last Active Cohort Injection Injection injection n=41 EOS* Screening R 1st 2nd Month 12 follow- Visit and rilparencel rilparencel Biopsy 1:1 Injection Injection up after last injection Day –60 to Day 0 *EOS = end-of-st udy D
Slide 5: Methods
Methods Rilparencel Statistical Trial Timeline Procedures Analysis • First Subject, First Visit • Ex vivo cultured expansion of • Analyses were performed on • February 2017 cells from percutaneous kidney subjects who were randomized cortex biopsy and biopsied • Last Subject, Last Visit • Selected Re
Slide 6: Subject Flow
Subject Flow 1st ACTIVE COHORT Biopsy rilparencel Active Cohort Injection EOS 41 subjects were randomized to the Active n=41 41 Cohort and biopsied 39 Before 1st Injection: 2 subjects withdrew 41 39 31 R Before End of Study: 3 subjects withdrew 1:1 1st 31 consent, 3 subjects died, 2 were lost to fol
Slide 7: Screening Demographics
Screening Demographics Group 1 Group 2 Characteristics (Active Treatment, n=41) (Deferred Treatment, n=42) Age, years (mean +/- SD) 66.0 +/- 10.0 64.5 +/- 8.9 Male (n) 29 27 Race (n) White 39 31 African American, Black, Asian, Other 2 11 Body Mass Index, kg/m2 33.4 +/- 6.1 33.7 +/- 5.5 Systolic bloo
Slide 8: Procedure and Product Safety Results
Procedure and Product Safety Results 3 SAEs Related 10 SAEs Related 0 SAEs Related to Biopsy to Injection to Product (n=83) (n=132) (n=132) 1 hematoma • 2 hematoma • • none • 2 pain (back & • 1 Page Kidney abdominal) • 1 AKI • 1 AKI • 1 anaemia • 1 pyrexia • 1 pneumonia • 1 blood creatinine increase
Slide 9: Non-Procedure Related SAEs
Non-Procedure Related SAEs Area of Interest n (%) Cardiac Death 2 (2.7) Serious cardiac disorders 14 (19.2) Acute myocardial infarction 7 (9.6) Cardiac arrest 5 (6.8) Coronary artery disease 4 (5.5) Serious Renal and Urinary Disorders 19 (26.0) Acute kidney injury 11 (15.1) End stage renal disease 5
Slide 10: Primary Efficacy Result - Change in eGFR
Primary Efficacy Result - Change in eGFR 0.1 -0.1 -1.1 -3.7 Average baseline eGFR (SEM) -3.7* Group ml/min/1.73m2 -4.3* 1 32.8 (1.4) 2 31.7 (1.2) 3 28.8 (1.4) Group 1 Group 2 Group 3 (Active Group) (Deferred Treatment: (Deferred Treatment: SoC only) rilparencel injection) *T-Test of Change from Base
Slide 11: Long Term eGFR Change from Baseline
Long Term eGFR Change from Baseline Average Change from Baseline in eGFR ml/min/1.73m2 18 Months 24 Months 30 Months Group 1 (Active -3.1 (n=26) -4.5* (n=29) -7.0* (n=24) Treatment) Group 3 (Deferred -1.8* (n=24) -0.1 (n=15) -2.8 (n=12) Treatment: rilparencel injection) *T-Test of Change from Baseli
Slide 12: eGFR Slope Change
eGFR Slope Change Annualized eGFR Slope* Annualized eGFR Slope* (95% CI) (95% CI) Over 12 months Through End of Study ml/min/1.73m2/year ml/min/1.73m2/year Group 1 (Active Treatment) -3.0 (-5.7, -0.2) -3.0 (-4.3, -1.8) Group 2 (Deferred Treatment: -2.3 (-4.5, -0.1) -2.8 (-4.5, -1.1) SoC Only) Group
Slide 13: Post-hoc Subgroup Analysis
Post-hoc Subgroup Analysis 2.1 1.6 0.8 0.5 Average baseline eGFR (SEM) Group ml/min/1.73m2 -3.0* -5.5* 1 25.4 (1.4) 2 26.1 (0.8) 3 20.9 (0.9) Group 1 Group 2 Group 3 (Active Group) (Deferred Treatment: (Deferred Treat ment: SoC only) rilparencel inject ion) *T-Test of Change from Baseline: P-value <
Slide 14: Key Study Limitations
Key Study Limitations • Open label trial design • Randomized control group (SoC comparator) had only 12-month follow-up • No upper threshold on UACR inclusion criteria • Limited subject diversity • COVID pandemic - reduced clinical site visits, lab assessments and limited facility access for procedu
Slide 15: Summary and Conclusion
Summary and Conclusion • Subjects randomized to the deferred treatment arm experienced less decline in kidney function after rilparencel treatment versus standard of care Key Findings • Kidney function stabilized in a subgroup of subjects with Stage 4 CKD and severe UACR • Observed procedure and pro
Slide 16: Thank you to our RMCL-002 Sites and Investigators
Thank you to our RMCL-002 Sites and Investigators • UNC - Randal Detwiler • Lehigh Valley Health Network - Nelson Kopyt • University of Chicago - Benjamin Ko • University of Wisconsin - Ali Gardezi • Vanderbilt - Anna Burgner • University of Michigan - Alexander Yevzlin • Mt. Sinai NYC - Steven Coca