ProKidney (PROK) 7th Annual Evercore ISI HealthCONx Healthcare Conference summary

Regulatory and clinical development updates

• Regulatory update on accelerated approval pathway expected in the first half of 2025, following FDA meetings.

• Full Cohort 1 REGEN-007 data anticipated in the first half of 2025.

• FDA confirmed a single trial (PROACT 1) is sufficient for submission, leading to discontinuation of PROACT 2.

• Accelerated approval pathway possible using eGFR as a surrogate endpoint, pending FDA agreement.

• Phase 3 PROACT 1 readout now expected in Q3 2027, with potential for earlier results depending on follow-up.

Clinical trial design and patient population

• Focus on late-stage, fast-progressing stage 4 CKD patients, with some stage 3B included.

• Stabilization of kidney function is the primary goal, with prior studies showing significant effect in high-risk populations.

• Standard of care event rate for phase 3 set at 13%, based on current data and evolving treatment landscape.

• Background therapies (SGLT2s, GLP-1s, MRAs) have limited impact in late-stage CKD due to adherence and cost challenges.

• Patients in late-stage CKD continue to have high unmet needs despite recent therapeutic advances.

Mechanism of action and dosing strategy

• Early data suggested regenerative effects, but current focus is on stabilization and anti-inflammatory, anti-fibrotic mechanisms.

• Stronger effects observed in fast-progressing, late-stage patients; unclear if earlier-stage patients would benefit more.

• Two-dose regimen (one per kidney) is currently optimal; redosing may be explored in the future.

• Switch from fresh to cryopreserved product validated and accepted by regulators.