Rilparencel

The trials enrol patients with type 2 diabetes and chronic kidney disease (CKD). CKD can be a complication of type 2 diabetes. Credit: NTshutterth via Shutterstock. ProKidney is dropping an ex-US Phase III study for its chronic kidney disease (CKD) cell therapy rilparencel, following discussions with regulatory experts and ex-US Food and Drug Administration (FDA) officials. The US-based biotech’s Phase III program consisted of two studies; the REGEN-006 (PROACT 1) (NCT05099770) study in the US and the REGEN-016 (PROACT 2) (NCT05286853) study in ex-US countries. Both trials are designed to investigate the agent, which is also known as REACT, in type 2 diabetes and CKD. Following discussions to finalise rilparencel’s regulatory plans in the US, North Carolina-based ProKidney will drop the REGEN-016 trial, a move which the company says will save between $150m and $175m. The company closed a $140m public offering of class A ordinary shares in June 2024, which would extend its cash runway to mid-2026. Rilparencel, which was granted regenerative medicine advanced therapy (RMAT) designation by the FDA in October 2021, is a regenerative therapy. Administered through intra-renal injection, it utilises autologous renal cells embedded in a gelatin-based hydrogel. These cells are designed to stimulate the regeneration of functional kidney tissue, aiming to restore kidney function based on organ regeneration technology. Enrolment into the REGEN-006 study was put on hold last year after ProKidney announced that it was updating the protocol. The study, which previously enrolled patients with mild-moderate to severe CKD, was updated to focus on patients with the most severe CKD. The two trials were expected to enrol up to 600 patients each. Subjects were randomised 1:1 into either a rilparencel treatment arm and undergo a kidney biopsy, or a sham control arm. See Also: BR-6002 by Boryung Pharmaceutical for Duodenal Ulcer: Likelihood of Approval BR-6002 by Boryung Pharmaceutical for Gastric Ulcers: Likelihood of Approval In the announcement accompanying the update, ProKidney’s CEO Bruce Culleton said: “We are confident that this strategic shift in our Phase III programme is the most expeditious and resource efficient approach to bring rilparencel to market in the US, our highest priority market.” Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Rilparencel is an autologous cell therapy creating by identifying progenitor cells in a patient’s biopsy. ProKidney has stopped one of a pair of phase 3 trials for its cell therapy for kidney disease after deciding it wasn’t essential for securing FDA approval.The product, called rilparencel or REACT, is an autologous cell therapy creating by identifying progenitor cells in a patient’s biopsy. A team formulates the progenitor cells for injection into the kidney, where the hope is that they integrate into the damaged tissue and restore the function of the organ.The North Carolina-based biotech has been running two phase 3 trials of rilparencel in Type 2 diabetes and chronic kidney disease: the REGEN-006 (PROACT 1) study within the U.S. and the REGEN-016 (PROACT 2) study in other countries.The company has recently “completed a comprehensive internal and external review, including engaging with ex-FDA officials and seasoned regulatory experts, to decide the optimal path to bring rilparencel to patients in the U.S.”Rilparencel received the FDA’s regenerative medicine advanced therapy (RMAT) designation back in 2021, which is designed to speed up the development and review process for regenerative medicines. ProKidney’s review concluded that the RMAT tag means rilparencel is eligible for FDA approval under an expedited pathway based on a successful readout of its U.S.-focused phase 3 trial REGEN-006. As a result, the company will discontinue the REGEN-016 study, freeing up around $150 million to $175 million in cash that will help the biotech fund its plans into the early months of 2027. ProKidney may still need a top-up at some point, however, as on current estimates the remaining phase 3 trial may not read out top-line results until the third quarter of that year.ProKidney, which was founded by Royalty Pharma CEO Pablo Legorreta, closed a $140 million underwritten public offering and concurrent registered direct offering in June, which had already extending the biotech’s cash runway into mid-2026.“We decided to prioritize PROACT 1 to accelerate potential U.S. registration and commercial launch,” CEO Bruce Culleton, M.D., explained in this morning’s release.“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.”The phase 3 trials were on pause during the early part of this year while ProKidney amended the PROACT 1 protocol as well as its manufacturing capabilities to meet international standards. Manufacturing of rilparencel and the trials themselves resumed in the second quarter.