ProKidney Corp. (Nasdaq: PROK) announced its financial results and business highlights for the year ended December 31, 2024, reporting significant progress in its clinical development program for rilparencel, a cellular therapy for chronic kidney disease (CKD).
The company made substantial advancements across multiple fronts in 2024, including refining its Phase 3 program to accelerate rilparencel's path to market, releasing positive Phase 2 data, restarting manufacturing operations, and securing $140 million in equity funding to extend its cash runway into mid-2027.
FDA Confirms Accelerated Approval Pathway
In a significant regulatory development, the U.S. Food and Drug Administration (FDA) confirmed during a Q4 2024 Type B meeting that an accelerated approval pathway is available for rilparencel if an acceptable surrogate endpoint is used. The FDA indicated that estimated glomerular filtration rate (eGFR) slope could serve as this surrogate endpoint.
"We refined our Phase 3 program to focus on advanced CKD patients with the highest unmet need, engaged in discussions with the FDA to accelerate rilparencel's path to market, improved and restarted our manufacturing process, and secured $140 million in equity funding," said Bruce Culleton, M.D., CEO of ProKidney.
The FDA also confirmed that REGEN-006 (PROACT 1), a single, large, multi-center, well-controlled Phase 3 study, could be sufficient to support a potential Biologics License Application (BLA) submission. Based on this feedback, ProKidney discontinued its Phase 3 REGEN-016 (PROACT 2) study, which had focused on enrollment outside the U.S.
Additional details on the potential accelerated approval pathway are expected in mid-2025 after a planned Type B meeting with the FDA.
Promising Phase 2 Data
ProKidney expects to release full data from Group 1 of the Phase 2 REGEN-007 study in Q2 2025. This data will include approximately 20 patients who have received two rilparencel injections, with an average follow-up of approximately 18 months.
Interim results from REGEN-007 announced in June 2024 showed stabilization of kidney function for 18 months in 13 patients with advanced CKD and diabetes. These early results suggest rilparencel's potential to preserve kidney function in patients at high risk of kidney failure.
Phase 3 PROACT 1 Trial Design
The REGEN-006 (PROACT 1) trial is an ongoing Phase 3, randomized, blinded, sham-controlled safety and efficacy study of rilparencel in subjects with type 2 diabetes and advanced CKD. The study protocol was amended in the first half of 2024 to focus on patients with stage 4 CKD (eGFR 20-30 mL/min/1.73m²) and late stage 3b CKD (eGFR 30-35 mL/min/1.73m²) with accompanying albuminuria.
The trial has a planned enrollment of approximately 685 subjects, randomized 1:1 to receive either rilparencel or sham treatment. Patients in the treatment group receive two rilparencel injections, one in each kidney, three months apart.
The primary composite endpoint is the time from first injection to the earliest of: at least 40% reduction in eGFR; eGFR <15 mL/min/1.73m², and/or chronic dialysis, and/or renal transplant; or renal or cardiovascular death.
Financial Position
ProKidney reported a solid financial position with $358.3 million in cash, cash equivalents, and marketable securities as of December 31, 2024, compared to $363.0 million on December 31, 2023. The company expects these funds to support its operating expenses and capital expenditure requirements into mid-2027.
Research and development expenses increased to $127.7 million for 2024, up from $106.7 million in 2023. This $21.0 million increase was primarily driven by higher cash-based compensation costs as the company expanded its clinical development, quality, manufacturing, and biostatistics teams to support ongoing clinical trials.
General and administrative expenses rose to $56.1 million in 2024 from $44.8 million in 2023, an increase of $11.3 million. This was primarily due to increased cash-based compensation, an impairment charge of $5.3 million related to the company's Greensboro, North Carolina facility, and higher equity-based compensation.
The net loss before noncontrolling interest was $163.3 million for 2024, compared to $135.4 million for 2023.
About Rilparencel
Rilparencel (also known as REACT®) is ProKidney's lead product candidate, a first-in-class, proprietary autologous cellular therapy being evaluated in Phase 2 and Phase 3 studies. It is designed to preserve kidney function in diabetic patients at high risk of kidney failure.
The therapy has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, which can help expedite development and review of regenerative medicine therapies intended to treat serious conditions.
Looking Ahead
"Looking ahead, 2025 will be a critical inflection point, with the expected release of full Group 1 data from the Phase 2 REGEN-007 study and an update on the accelerated approval pathway for the Phase 3 PROACT 1 study after our planned Type B meeting with the FDA," said Dr. Culleton. "Our objective is clear: expedite rilparencel's path to market in the U.S. to bring a new therapeutic option to patients with advanced CKD and diabetes at high risk of requiring dialysis."
As ProKidney advances its clinical program, the company is positioned to potentially address a significant unmet need in the treatment of chronic kidney disease, a condition affecting millions of patients worldwide.
