ProKidney's Rilparencel Receives Positive FDA Feedback on Phase 3 Trial Design and Accelerated Approval Pathway
Key Insights
The FDA has confirmed that ProKidney's PROACT 1 Phase 3 study could be sufficient for full U.S. regulatory approval of rilparencel, a cellular therapy for chronic kidney disease (search) (CKD).
ProKidney may pursue accelerated approval for rilparencel, with the FDA indicating that eGFR (search) slope could serve as an acceptable surrogate endpoint.
ProKidney's cash reserves of $406.8 million are expected to support operations into 2027, facilitating the continued development of rilparencel.
ProKidney Corp. (Nasdaq: PROK) announced positive feedback from the FDA regarding its lead product candidate, rilparencel, for the treatment of chronic kidney disease (search) (CKD). Following a Type B meeting with the FDA, the agency confirmed that the ongoing Phase 3 PROACT 1 study could be sufficient to support a Biologics License Application (BLA) submission and potential full regulatory approval in the U.S. The FDA also indicated that the accelerated approval pathway is available for rilparencel, with the potential use of estimated glomerular filtration rate (eGFR (search)) slope as a surrogate endpoint. This regulatory update coincides with ProKidney's report of $406.8 million in cash reserves, expected to fund operations into 2027.
Regulatory and Clinical Development Updates
The FDA's feedback on the PROACT 1 trial design and the potential for accelerated approval represents a significant milestone for ProKidney. The PROACT 1 trial, also known as REGEN-006, is a Phase 3, randomized, blinded, sham-controlled study evaluating the safety and efficacy of rilparencel in patients with type 2 diabetes (search) and advanced CKD. The trial focuses on patients with stage 4 CKD (eGFR (search) 20-30ml/min/1.73m²) and late-stage 3b CKD (eGFR 30-35ml/min/1.73m²) with albuminuria. The primary composite endpoint is the time from first injection to the earliest of a 40% reduction in eGFR, eGFR <15 mL/min/1.73m², chronic dialysis, renal transplant, or renal/cardiovascular death.
In late October, ProKidney presented five posters at the American Society of Nephrology’s (ASN) Kidney Week. These included a late-breaking clinical trial presentation on the Phase 2 REGEN-007 study and four posters detailing rilparencel’s mechanism of action (MOA) and product characteristics.
Financial Highlights
ProKidney reported having $406.8 million in cash, cash equivalents, and marketable securities as of September 30, 2024, compared to $363.0 million on December 31, 2023. The company anticipates these funds will support operating expenses and capital expenditure requirements into 2027. Research and development expenses for the third quarter of 2024 were $31.3 million, a slight decrease from $32.2 million for the same period in 2023. General and administrative expenses were $17.7 million for the third quarter of 2024, compared to $14.4 million for the same period in 2023, primarily driven by a non-cash impairment charge of $5.3 million related to the Greensboro facility and increases in cash compensation.
About Rilparencel
Rilparencel, also known as REACT®, is an autologous cellular therapy being investigated in Phase 2 and Phase 3 studies for preserving kidney function in diabetic patients at high risk of kidney failure. It has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.