ProKidney (NasdaqCM:PROK) FY Conference Transcript

Summary of ProKidney Conference Call Company Overview - Company: ProKidney - Headquarters: Winston-Salem and Boston - Employee Count: Approximately 250 - Public Offering: Went public in 2022 - Product: Autologous cell therapy named Reparencel aimed at keeping patients with advanced chronic kidney disease (CKD) off dialysis - Current Study: In a phase 3 study called ProAct One [2][3] Phase 3 Study Details - Study Design: Randomized, sham-controlled, multicenter study primarily in the U.S., with some sites in Mexico and Taiwan - Sample Size: Targeting over 600 patients to achieve 122 events, which is significant for a cell therapy [3][4] - Regulatory Progress: Achieved RMAT designation in 2021, allowing for regulatory flexibility; discussions with the FDA have led to alignment on an accelerated approval process based on eGFR slope [4][5] - Expected Readout: Top-line results anticipated by Q2 2027 [5][11] Efficacy and Effect Size - Effect Size: FDA agreed on a meaningful effect size of at least 1.5 mL/min/year difference between treatment and control groups [7][8] - Context: This target is 50% greater than the differences observed in SGLT2 trials, which typically show less than 1 mL/min/year [8][9] - Phase 2 Results: In the phase 2 study, a decline of -5.8 mL/min/year was observed pre-treatment, reduced to -1.3 mL/min/year post-treatment, indicating a substantial difference [9][10] Market Opportunity - Target Population: Focus on high-risk CKD patients, particularly those with eGFR around 30 or less and high albuminuria [15][16] - Market Size: Estimated over 1 million patients in stage 4 CKD and 3B with high UACR, with around 500,000 being diabetics [29] Financials and Cash Runway - Cash Position: As of June, ProKidney had $295 million, expected to last until mid-2027, covering the phase 3 data and accelerated approval [33] - Cost Management: Strategic changes, including halting another phase 3 study (ProAct Two), saved approximately $150 million [36] Competitive Landscape - Therapeutic Positioning: ProKidney's therapy is expected to complement standard care, targeting patients who do not respond adequately to existing treatments like ACE inhibitors, ARBs, SGLT2s, and GLP-1 agonists [28][29] - Manufacturing Capabilities: Own manufacturing facility in North Carolina, capable of supporting both phase 3 and commercial launch [29] Regulatory and Strategic Considerations - FDA Relationship: Strong relationship with FDA, with RMAT designation providing a favorable regulatory environment [47] - Tariffs Impact: Minimal impact from tariffs due to primarily U.S.-sourced operations [48] Future Directions - Potential for Earlier CKD Populations: While there is interest in targeting earlier stages of CKD, the current focus remains on the high-risk population due to sufficient demand [31] - AI Utilization: ProKidney is cautiously exploring AI for administrative and cybersecurity tasks, with careful consideration in regulatory contexts [44][46] Conclusion ProKidney is positioned to address a significant unmet need in the chronic kidney disease market with its innovative cell therapy, Reparencel. The company is making substantial progress in its phase 3 study and has a solid financial foundation to support its upcoming milestones.