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 will be released 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 is possible using eGFR as a surrogate endpoint, pending FDA agreement.

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

Clinical trial design and patient population

• Focus is 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 slowing of decline in this population.

• Standard of care event rate for the phase 3 program is set at 13%.

• Background therapies like SGLT2s and GLP-1s have limited impact in late-stage CKD, with adherence and cost being 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 late-stage, fast-progressing patients, but unclear if earlier-stage patients would benefit more.

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

• Switch from fresh to cryopreserved product validated with no difference in efficacy.