ProKidney believes it will only need one Phase 3 trial to win an FDA approval for its kidney disease cell therapy, and will discontinue another to save cash.
On Tuesday, the biotech said it will wind down its PROACT 2 study, which was primarily focused on patients outside the US, and focus resources on a parallel US trial of the therapy, rilparencel, as a treatment for chronic kidney disease.
The decision was based on “a comprehensive internal and external review” that concluded, in part, that the company would only need one Phase 3 study to qualify for “an expedited approval pathway” at the FDA. ProKidney cited “provisions” in rilparencel’s Regenerative Medicine Advanced Therapy (RMAT) designation as evidence that a second registrational trial wouldn’t be needed for an initial approval.
A spokesperson for ProKidney said the expedited pathway could include accelerated approval, depending on the Phase 3 data.
The company will be operating with a narrow fiscal window as it works to advance its lead asset. It expects the revised plans to extend its cash runway into the first quarter of 2027, but the Phase 3 results aren’t expected until the third quarter of that year. Cutting the second Phase 3 study is expected to save the company between $150 million to $175 million.
The spokesperson said it will need “a modest amount of incremental capital” to fund the topline trial results, which could come from either business development or public offerings.
Bruce Culleton“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 CEO Bruce Culleton in a statement.
ProKidney is less than three months removed from a $140 million public offering on the back of open-label Phase 2 data. Those results showed that after receiving a shot of rilparencel in each kidney, three months apart, 13 patients with diabetes and chronic kidney disease showed “stabilized kidney function” for 18 months. The company said the data were similar in 10 out of 13 patients that met updated inclusion criteria for the Phase 3 program.
ProKidney amended the trial protocol earlier this year to focus on recruiting patients at a higher risk of kidney failure. The rationale then was that the new population would “better align” with prior Phase 2 data and appease payers and providers.