ProKidney (PROK) Guggenheim’s Inaugural Healthcare Innovation Conference summary

Company overview and lead asset

• Focuses on autologous cell therapy, rilparencel, for advanced chronic kidney disease in type 2 diabetes patients.

• Rilparencel involves kidney biopsy, cell processing, and reinjection into the kidneys.

• Company has over 200 employees and operates from North Carolina and Boston.

• Currently in late-stage clinical development, pre-commercial phase.

Clinical development and regulatory strategy

• Originated from IP at Boston Children's Hospital, shifted focus to kidney cortical cells.

• Went public in 2022 via de-SPAC; new CEO appointed last year.

• Paused phase III and manufacturing in 2023 for facility remediation; achieved GMP compliance by mid-2023.

• Amended phase III protocol to target patients with GFR 20–35, addressing highest unmet need.

• Consolidated phase III efforts to focus on PROACT 1 in the US and pursued accelerated approval discussions with FDA.

Regulatory feedback and approval pathway

• FDA agreed a single, well-conducted randomized trial could suffice for full approval.

• Accelerated approval pathway available under RMAT; FDA open to using slope eGFR as a surrogate endpoint.

• Company to submit a proposal for conditional approval using slope eGFR in 2025.