Akebia Therapeutics Advances Praliciguat Development With Phase 2 Enrollment For FSGS Treatment

Akebia Therapeutics, Inc. (AKBA) has initiated patient enrollment in a Phase 2 clinical trial examining Praliciguat as a potential therapy for Focal Segmental Glomerulosclerosis (FSGS), marking a significant step forward in addressing a critical unmet medical need. The first patient was dosed this week, signaling the beginning of this pivotal study phase.

The Clinical Challenge: FSGS and the Treatment Gap

Focal Segmental Glomerulosclerosis represents a severe kidney disorder characterized by progressive scarring of the glomeruli—the kidney’s primary filtering structures. Approximately 40,000 patients in the United States live with this condition, yet currently, there are no FDA-approved treatments specifically designed to address this disease. This therapeutic void underscores the urgent need for innovative drug candidates like Praliciguat to fill the gap in clinical management options.

Understanding Praliciguat’s Mechanism

Akebia’s lead candidate operates as a soluble guanylate cyclase (sGC) stimulator, a mechanism of action that has demonstrated therapeutic promise in related renal conditions. The company licensed Praliciguat from Cyclerion Therapeutics, and preliminary research has been encouraging. Earlier Phase 1 studies in healthy volunteers and Phase 2 evaluations in heart failure and diabetic kidney disease patients revealed no significant safety concerns, establishing a favorable baseline safety profile for further investigation.

Trial Design and Patient Population

The ongoing Phase 2 investigation is structured as a randomized, double-blind, placebo-controlled multicenter study focused on adults with biopsy-confirmed FSGS. Approximately 60 patients already receiving maximum tolerated doses of angiotensin-converting enzyme inhibitors or angiotensin receptor blockers will be randomized at a 1:1 ratio to receive either Praliciguat or placebo over a 24-week treatment window.

The study’s primary outcome measure centers on the change from baseline in the urine protein-to-creatinine ratio (UPCR) at Week 24. Secondary endpoints include the percentage of patients achieving partial remission, defined as a 40% reduction in UPCR with values below 1.5 gram per gram. Following the 24-week blinded phase, all participants will enter an open-label extension where they will receive Praliciguat for an additional 24 weeks, allowing extended efficacy and safety observation.

Broader Pipeline and Future Prospects

Beyond Praliciguat, Akebia’s development portfolio reflects a multi-pronged approach to kidney and related disorders. Vadadustat (Vafseo) is currently in Phase III development for treating anemia associated with chronic kidney disease in both dialysis-dependent and non-dialysis dependent populations. Additionally, Akebia is developing AKB-9090 to address cardiac surgery-related acute kidney injury and acute respiratory distress syndrome, as well as AKB-10108 for retinopathy of prematurity in newborns.

Market Perspective

On Tuesday’s trading session, AKBA closed at $1.47, reflecting a 2.65% decline. The market’s response will likely depend on interim efficacy and safety data from this Phase 2 trial and the company’s ability to demonstrate meaningful clinical benefit in the FSGS patient population.