ProKidney Updates Phase 3 Program to Accelerate Rilparencel's U.S. Approval

ProKidney Corp., a biotech company at an advanced clinical stage, recently unveiled strategic changes to its Phase 3 program aimed at accelerating the potential approval and commercial launch of its cell therapy candidate, rilparencel, for chronic kidney disease (CKD) in the U.S. market. The company decided to concentrate its resources on the PROACT 1 trial (REGEN-006) and discontinue the PROACT 2 trial (REGEN-016), which was being conducted outside the U.S. This move is expected to expedite the development process and reduce expenses significantly.

The decision came after a thorough internal and external review, involving consultations with former FDA officials and regulatory experts. The review concluded that rilparencel might qualify for an expedited approval pathway under the Regenerative Medicine Advanced Therapy (RMAT) designation. This pathway could potentially lead to initial FDA approval based on the successful completion of the ongoing Phase 3 REGEN-006 (PROACT 1) trial. The company believes that the PROACT 2 trial is not essential for the initial U.S. registration of rilparencel.

By focusing on PROACT 1, ProKidney aims to accelerate the U.S. registration and commercial launch of rilparencel, which targets patients with type 2 diabetes and advanced CKD—a group with a significant unmet clinical need. The revised Phase 3 program is projected to deliver topline results by the third quarter of 2027, and the company expects its current cash reserves to support operations until the first quarter of 2027. This shift is anticipated to reduce expenses by approximately $150 to $175 million.

Bruce Culleton, M.D., CEO of ProKidney, emphasized the importance of this strategic realignment, noting that it is the most efficient way to bring rilparencel to the U.S. market. The recent positive interim data from the REGEN-007 trial further underscores the urgency of making this treatment accessible to patients with advanced CKD. ProKidney plans to continue collaborating with the FDA under the RMAT designation to facilitate the approval and market introduction of rilparencel.

The past ten months have been transformative for ProKidney, marked by significant developments under the leadership of Dr. Culleton, who took over as CEO in November 2023. The company has made considerable progress, including enhancing its quality management systems to meet global standards for commercial manufacturing and refining its Phase 3 program to focus on U.S. patients with advanced CKD. ProKidney also welcomed several key executive leaders across various departments, further strengthening its organizational capabilities.

Rilparencel, ProKidney's lead product candidate, is an innovative autologous cellular therapy aimed at preserving kidney function in diabetic patients at high risk of kidney failure. The drug received RMAT designation from the FDA in October 2021, a status that facilitates an accelerated review process for regenerative medicine therapies showing potential to address unmet medical needs in serious or life-threatening conditions.

The ongoing Phase 3 REGEN-006 (PROACT 1) trial is a randomized, blinded, sham-controlled study assessing the safety and efficacy of rilparencel in patients with type 2 diabetes and advanced CKD. The trial includes a specific focus on patients with stage 4 CKD and late-stage 3b CKD, with a planned enrollment of approximately 685 subjects. The primary objective is to evaluate the efficacy of up to two rilparencel injections using a minimally invasive percutaneous approach. The study aims to measure the time to significant kidney function decline or other critical renal and cardiovascular events.

ProKidney, established in 2015, has been a pioneer in developing cellular therapies for CKD. With over a decade of research behind its innovations, the company continues to drive forward in its mission to offer new treatment options for patients facing this debilitating condition.