ProKidney cancels one Phase 3 study, seeks 'expedited approval' with another

ProKidney has recently decided to streamline its efforts to obtain FDA approval for its kidney disease cell therapy, rilparencel, by focusing solely on a single Phase 3 trial. The biotechnology company announced on Tuesday that it will discontinue its PROACT 2 study, which was primarily targeting patients outside the United States. Instead, the company will direct its resources towards a parallel Phase 3 trial in the U.S. aimed at treating chronic kidney disease.

This strategic decision was made following an extensive internal and external review. The review concluded that only one Phase 3 study would be necessary to qualify for an expedited approval pathway with the FDA. ProKidney highlighted that the Regenerative Medicine Advanced Therapy (RMAT) designation for rilparencel includes provisions that support the conclusion that a second registrational trial is not required for initial approval.

A spokesperson for ProKidney mentioned that the expedited pathway could potentially include accelerated approval, contingent on the results from the Phase 3 study. The company's focus on a single trial is also a decision influenced by financial constraints. By discontinuing the second Phase 3 study, ProKidney expects to save between $150 million and $175 million. This adjustment in their plans is projected to extend their cash runway into the first quarter of 2027, although the Phase 3 results are not anticipated until the third quarter of that year.

The spokesperson further indicated that ProKidney would require a modest amount of additional capital to fund the topline results of the trial. This funding could be sourced through business development activities or public offerings.

CEO Bruce Culleton expressed confidence in this strategic shift, asserting that it represents the most efficient and resourceful approach to bring rilparencel to the U.S. market, which remains their highest priority. This confidence is grounded in recent positive developments for the company. Less than three months ago, ProKidney raised $140 million through a public offering, driven by promising Phase 2 data. The open-label Phase 2 results revealed that rilparencel injections in each kidney, administered three months apart, stabilized kidney function in 13 diabetic patients with chronic kidney disease over an 18-month period. The data were consistent in 10 out of the 13 patients who met the updated inclusion criteria for the Phase 3 program.

Earlier this year, ProKidney revised the trial protocol to focus on recruiting patients at a higher risk of kidney failure. The aim was to align the trial population more closely with prior Phase 2 data and to address the concerns of payers and healthcare providers. This adjustment underscores the company's commitment to ensuring that rilparencel can be effectively and efficiently brought to market, providing a new treatment option for those suffering from chronic kidney disease.

Overall, ProKidney's decision to concentrate on a single Phase 3 trial is a strategic move intended to accelerate the approval process while managing financial resources prudently. This approach is expected to streamline their efforts and provide a clearer path to bringing their innovative therapy to patients in need.

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