ProKidney (PROK) UBS Virtual Organ Restoration and Cell Therapy Day summary

Company overview and technology

• Publicly traded biotech with ~200 employees and facilities in North Carolina and Boston, listed on Nasdaq since mid-2022.

• Lead asset, rilparencel (formerly REACT), is an autologous cell therapy derived from a patient’s own kidney cells.

• Manufacturing involves biopsy, cell selection, expansion, cryopreservation, and reinjection into the kidney cortex.

• Product evolution from fresh to cryopreserved formulation, now used in late-stage clinical trials.

Preclinical and clinical evidence

• Early preclinical work identified specific renal cell layers linked to improved kidney function and reduced inflammation.

• Phase II RMCL-002 trial showed stabilization of kidney function after rilparencel injection in patients with type 2 diabetes and advanced CKD.

• Strong efficacy signal in high-risk patients (GFR <30, UACR >300), informing Phase III inclusion criteria.

• Safety profile showed no serious adverse events related to the product, with biopsy/injection risks comparable to literature.

Clinical development and trial design

• Phase II REGEN-007 trial tested cryopreserved product, with both kidneys injected three months apart; interim data showed eGFR stabilization.

• Synthetic control group analysis indicated slower kidney function decline in treated patients versus matched external controls.

• Phase III PROACT 1 trial focuses on type 2 diabetes patients with GFR 20–35, using a sham-controlled, blinded design and composite time-to-event endpoints (death, dialysis, transplant, or >40% eGFR decline).

• Protocol amendments narrowed GFR criteria and simplified execution based on Phase II learnings.