Akebia Therapeutics has begun its Phase 2 study of praliciguat, an oral soluble guanylate cyclase stimulator, by dosing the first patient with biopsy‑confirmed focal segmental glomerulosclerosis (FSGS). The trial, which started on January 6 2026, enrolls approximately 60 adults who are already receiving maximally tolerated ACE inhibitor or ARB therapy and randomizes them 1:1 to praliciguat or placebo for 24 weeks, followed by an open‑label extension.
The study’s primary endpoint is the change from baseline in urine protein‑to‑creatinine ratio (UPCR) at week 24, with a key secondary endpoint of partial remission defined as a ≥40 % reduction in UPCR and an absolute UPCR of less than 1.5 g/g. These metrics are standard for evaluating disease‑modifying activity in FSGS and align with regulatory expectations.
Praliciguat has a well‑documented safety profile, having completed Phase 1 safety studies in healthy volunteers and Phase 2 studies in heart failure and diabetic kidney disease, where it was tolerated without significant safety signals. The drug’s mechanism—stimulating soluble guanylate cyclase to increase cyclic GMP—offers potential anti‑fibrotic and vasodilatory effects that may address the underlying pathophysiology of FSGS.
FSGS affects roughly 40,000 U.S. patients and currently has no approved targeted therapies. The disease is characterized by scarring of the glomeruli, leading to proteinuria and progressive kidney failure. A first‑in‑class treatment would fill a substantial unmet medical need and could capture a sizable share of the 40‑k patient market.
Akebia’s rare‑kidney‑disease pipeline extends beyond praliciguat. In late 2025 the company acquired global rights to AKB‑097, a tissue‑targeted complement inhibitor for C3‑driven disorders, and continues to develop a vasodilator for FSGS. These assets position Akebia to address multiple rare kidney indications and to diversify its therapeutic portfolio.
Commercially, Akebia’s current products include Vafseo, a vadadustat anemia therapy launched in early 2025, and Auryxia, a ferric citrate phosphate binder whose revenue has been declining due to patent expiration. The introduction of a potential FSGS therapy could broaden the company’s revenue base beyond its dialysis‑focused products and reduce reliance on the aging Auryxia market.
The initiation of the Phase 2 trial is a strategic milestone that signals progress toward a first‑in‑class therapy for an orphan disease. Successful outcomes could accelerate regulatory review, open new revenue streams, and enhance Akebia’s long‑term growth prospects by shifting the company’s focus from primarily dialysis products to a broader rare‑kidney‑disease portfolio.
While no immediate market reaction data are available, the trial start is likely to be viewed positively by investors as a tangible step toward diversifying Akebia’s revenue and addressing an unmet medical need.
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