ProKidney Corporation【PROK】株式ニュース

From July 15th
ProKidney Announces Alignment with the FDA on the Accelerated Approval Pathway for Rilparencel
• FDA confirmed that eGFR slope is an acceptable surrogate endpoint for accelerated approval of rilparencel in patients with CKD and
type 2 diabetes
• FDA also confirmed that the ongoing Phase 3 PROACT 1 study can be used for both accelerated approval and for full approval
• Nearly half of the patients required for the accelerated approval analysis using eGFR slope have been enrolled
• Topline data to support an application for accelerated approval is anticipated in Q2 2027
WINSTON-SALEM, N.C., July 15, 2025 – ProKidney Corp. (Nasdaq: PROK) (“ProKidney” or the “Company”), a leading late clinical-stage cellular
therapeutics company focused on chronic kidney disease (CKD), today announced confirmation of alignment with the U.S. Food and Drug
Administration (FDA) on the accelerated approval pathway for rilparencel. Rilparencel is an autologous cellular therapy that received Regenerative
Medicine Advanced Therapy (RMAT) designation from the FDA and currently is being evaluated in the ongoing Phase 3 REGEN-006 (PROACT 1)
trial to demonstrate the therapy’s potential to preserve kidney function in patients with advanced CKD and type 2 diabetes.
In a recent Type B meeting, the FDA confirmed that the slope of estimated glomerular filtration rate (eGFR) in patients from the ongoing Phase 3
PROACT 1 study can serve as the surrogate endpoint and primary basis for a Biologics License Application (BLA) submission of rilparencel under the
accelerated approval pathway. The FDA agreed that a rilparencel effect size (versus sham controls) of at least 1.5 mL/min/1.73m2/year improvement
would be an acceptable demonstration of efficacy in the setting of patients receiving appropriate standard of care therapies. ProKidney anticipates
topline data readout of eGFR slope as the surrogate endpoint to support an application for accelerated approval in Q2 2027. To date, ProKidney has
enrolled nearly half of the patients required for the accelerated approval analysis. The FDA also confirmed that the ongoing Phase 3 PROACT 1 study
may serve as the confirmatory study to support full approval of rilparencel based on the primary time-to-event composite endpoint specified in the
protocol. Updated guidance on the expected timing of the confirmatory readout will be provided in 1H 2026.
“We are very encouraged by the FDA’s support of the accelerated approval pathway for rilparencel using eGFR slope, marking a critical regulatory
milestone for ProKidney,” said Bruce Culleton, M.D., CEO of ProKidney. “This alignment allows us to move forward with clarity and conviction as we
advance enrollment in our pivotal Phase 3 PROACT 1 study on the heels of the positive Phase 2 REGEN-007 topline results announced last week. We
look forward to our ongoing dialogue with the FDA, under RMAT designation, to bring rilparencel to patients with advanced CKD and diabetes, a
patient population with high unmet clinical and societal needs.”
About Chronic Kidney Disease
CKD is a progressive condition characterized by the gradual decline of kidney function, which can ultimately lead to end-stage kidney disease (ESKD)
requiring dialysis or transplantation. An estimated 37 million adults in the U.S. have CKD, though many remain undiagnosed in the early stages.
Diabetes is the leading cause of CKD, and individuals with both conditions face significantly elevated risks of cardiovascular events, hospitalization,
and mortality. ProKidney is developing rilparencel for patients with Stage 3b/4 CKD and diabetes, a population that includes 1 to 2 million people in the
U.S. While current treatment options aim to slow disease progression, there remains a substantial unmet need for therapies that can stabilize kidney
function and delay or prevent the need for dialysis in patients with advanced CKD.
About the Phase 3 REGEN-006 (PROACT 1) Clinical Trial
REGEN-006 is an ongoing Phase 3, randomized, blinded, sham controlled safety and efficacy study of rilparencel in subjects with advanced CKD and
type 2 diabetes. The study protocol was amended in 1H 2024 to focus on a subset of 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 (UACR less than 5,000 mg/g for patients with eGFR 20-30
mL/min/1.73m2 and 300-5,000 mg/g for patients with eGFR 30-35 mL/min/1.73m2). The total planned enrollment is approximately 685 subjects.
Subjects are randomized (1:1) to the treatment group and the sham control group prior to kidney biopsy or a sham biopsy procedure, respectively. The
primary objective is to assess the efficacy of up to two rilparencel injections (one in each kidney) using a minimally invasive percutaneous approach.
The surrogate endpoint for accelerated approval is eGFR slope, and the primary composite endpoint is the time from first injection to the earliest of: at
least 40% reduction in eGFR; eGFR