ProKidney Announces Strategic Updates to its Phase 3 Program to Accelerate Rilparencel’s Registrational Path to Potential Approval in the U.S.

• Refined the focus of the Phase 3 program to accelerate path to potential U.S. approval and commercial launch in the Company’s highest priority market
• Discontinued the ex-U.S.-based PROACT 2 trial and focused Company resources on PROACT 1 to expedite enrollment and accelerate estimated topline data readout to Q3 2027

WINSTON-SALEM, N.C., Sept. 03, 2024 (GLOBE NEWSWIRE) -- ProKidney Corp. (Nasdaq: PROK) (“ProKidney” or the “Company”), a late clinical-stage biotech company focused on the development of a first-in-class cell therapy candidate for chronic kidney disease (CKD), today announced strategic updates to its Phase 3 program for rilparencel, an investigational treatment to potentially preserve kidney function in patients with type 2 diabetes and advanced CKD.

ProKidney recently completed a comprehensive internal and external review, including engaging with ex-FDA officials and seasoned regulatory experts, to determine the optimal path to bring rilparencel to patients in the U.S. with type 2 diabetes and advanced CKD – a market where there is high unmet clinical and economic need. An important conclusion of this review is that under the provisions of the Regenerative Medicine Advanced Therapy (RMAT) designation, the Company believes rilparencel is eligible for initial FDA approval under an expedited approval pathway based upon successful completion of the ongoing Phase 3 REGEN-006 (PROACT 1) trial. ProKidney believes that the Phase 3 REGEN-016 (PROACT 2) trial is not required for initial U.S. registration. Thus, the Company will discontinue PROACT 2, which was focused on enrollment outside the U.S. With the discontinuation of PROACT 2, ProKidney now expects current cash to support operating plans into Q1 2027. The Company estimates the revised Phase 3 program will deliver topline results by Q3 2027 and reduce expenses by approximately $150 to $175 million.

“We decided to prioritize PROACT 1 to accelerate potential U.S. registration and commercial launch. We are confident that this strategic shift in our Phase 3 program is the most expeditious and resource efficient approach to bring rilparencel to market in the U.S., our highest priority market,” said Bruce Culleton, M.D., Chief Executive Officer. “The recent positive REGEN-007 interim data update in June further supports the urgency to bring our innovation to patients with advanced CKD. We look forward to continuing our engagement with the FDA, under the RMAT designation, to bring rilparencel to market.”

Today’s update follows a transformational period for ProKidney over the past 10 months. In November 2023, Bruce Culleton, M.D., a nephrologist and seasoned leader of kidney care organizations, was appointed Chief Executive Officer. Under Dr. Culleton’s leadership, ProKidney has made significant progress, including the implementation of improved quality management systems to ensure compliance with global standards for commercial manufacturing and the Phase 3 program, the refinement and restart of the Phase 3 program with a renewed focus on patients with advanced CKD in the U.S., and the appointment of several key executive leaders across clinical operations, manufacturing, human resources, and business operations. The Company also released final data from the Phase 2 RMCL-002 trial and interim data from the Phase 2 REGEN-007 trial. Data from these trials suggest that rilparencel’s greatest potential therapeutic impact is in advanced CKD patients at high risk of kidney failure. This patient population aligns with feedback from payors and providers who have emphasized the need for treatment options in this population.

Rilparencel was granted RMAT designation by the FDA in October 2021. RMAT designation can be granted to regenerative medicine therapies (including cell therapies) that are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and have preliminary clinical evidence that indicate the drug candidate has the potential to address unmet medical needs for such disease or condition. This designation is intended to facilitate an accelerated development and review process similar to the breakthrough therapy designation. With an RMAT designation for rilparencel, ProKidney will continue to work with the FDA to receive guidance on its registrational program, including guidance on clinical trial design, manufacturing, and long-term patient follow-up, as appropriate.

About the Phase 3 REGEN-006 (PROACT 1) Clinical TrialREGEN-006 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 1H 2024 to focus on a subset of patients with stage 4 CKD (eGFR 20-30ml min/1.73m2) and late stage 3b CKD (eGFR 30-35ml min/1.73m2) with accompanying albuminuria (urine albumin-to-creatinine ratio, or UACR less than 5,000 mg/g for patients with eGFR 20-30ml min/1.73m2 and 300-5,000 mg/g for patients with eGFR 30-35ml 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. Subjects in the treatment group are to receive the first rilparencel injection within 18 weeks of kidney biopsy. After three months it is intended that a second rilparencel injection be given into the contralateral kidney. Subjects in the control group, who previously underwent the sham biopsy procedure, are to receive two sham injections at similar time points as the treatment group. The primary objective is to assess the efficacy of up to two rilparencel injections using a minimally invasive percutaneous approach. The primary composite endpoint is the time from first injection to the earliest of: at least 40% reduction in eGFR; eGFR