Akebia Therapeutics (NASDAQ:AKBA) executives outlined the company’s commercial priorities and pipeline plans at the Guggenheim Emerging Outlook Biotech Summit, highlighting ongoing efforts to expand adoption of its anemia drug VAFSEO in U.S. dialysis centers while advancing a new rare kidney disease pipeline.
Company focus: kidney disease, two commercial products
CEO John Butler described Akebia as a “purpose-driven” company focused on improving the lives of people with kidney disease. He noted the company has two commercial products: Auryxia, a phosphate binder that has been contributing revenue “as it nears the end of its life,” and VAFSEO, a hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) approved to treat anemia in chronic kidney disease patients on dialysis.
VAFSEO launch: access expanded, discontinuation challenges and a TIW shift
Butler said Akebia launched VAFSEO in January and saw “very, very strong initial uptake,” but emphasized that the dialysis market is highly protocol-driven. Unlike retail prescribing models, dialysis providers control which drugs are available in clinics, and physicians can use only what is on the provider’s formulary.
He contrasted early traction at US Renal—where he cited clinical advocacy and comfort with reimbursement dynamics, including transitional drug add-on payment adjustment (TDAPA)—with slower uptake at other providers, including DaVita.
A key early issue, Butler said, was higher-than-expected discontinuation. VAFSEO is a once-daily oral drug, while dialysis centers have managed anemia for decades with in-chair injections. He said many patients experience an initial dip in hemoglobin after starting VAFSEO at the standard starting dose before titration brings hemoglobin into range. Akebia observed that nurses and “anemia managers” would often stop therapy when the initial dip appeared, contributing to what he described as more patients “coming out the bottom of the funnel” despite new starts.
To address this, Akebia supported a shift toward three-times-weekly (TIW) dosing in some centers so the drug can be administered as observed dosing during dialysis sessions. Although Butler said TIW dosing is not currently in the label, he described early operational benefits: in centers adopting TIW, the share of patients not receiving a second prescription fell from “upwards of 30%” to “less than 10%” in the first month.
Butler also said Akebia ended 2025 with access to approximately 275,000 patients and framed the near-term focus as helping providers refine implementation now that access is broader.
Clinical readouts: VOCAL, VOICE and label discussions
Akebia executives said they expect upcoming clinical data to support broader adoption and potentially future labeling discussions around TIW dosing.
- VOCAL: Chief Medical Officer Steve Burke said the VOCAL study is sponsored by Akebia and run with DaVita, with data expected by the end of the year. The study compares VAFSEO to Mircera, a long-acting erythropoiesis-stimulating agent (ESA), and Burke said he expects “excellent hemoglobin control,” stability, and reduced need for rescue therapies with less hemoglobin variability.
- VOICE: Management described VOICE as a randomized study at US Renal Care enrolling more than 2,100 patients, comparing VAFSEO to Epogen (a short-acting ESA). Butler said enrollment completed last June, the company expects database lock by the end of this year, and results are planned for “early in 2027.” Burke said the primary endpoint is a composite of death and hospitalization using a win ratio approach, with a non-inferiority goal that can “flip” to superiority.
Burke also highlighted a VOCAL sub-study examining red blood cell phenotypes, including proteomics, metabolomics, lipidomics, membrane fluidity, and resistance to oxidative stress. He said Akebia has observed that red blood cells produced under VAFSEO tend to be larger and carry more hemoglobin with a narrower distribution of widths, which he characterized as favorable.
Separately, Butler referenced an analysis presented at ASN using a “win odds” technique from a Phase III dataset, which he said showed a statistically significantly lower risk of death or hospitalization with VAFSEO versus darbepoetin, a long-acting ESA. He said the company intends to publish and further communicate the findings.
On labeling, Butler said Akebia plans to engage with FDA regarding TIW dosing and cited existing studies (MODIFY and FOCUS) that used TIW dosing, with VOCAL and VOICE also using TIW. He suggested FDA may want VOCAL results first, with VOICE results also potentially relevant to the discussion.
Non-dialysis CKD: FDA discussions and limited path forward
Butler said Akebia continues to engage FDA on the non-dialysis chronic kidney disease population, which he described as a large group where a once-daily oral anemia therapy “makes perfect sense.” However, he said that at an October Type C meeting, FDA’s position was that Akebia would need to redo the PROTECT trial, which he described as a 3,000+ patient study taking five years, a path the company said did not make sense economically or from a timing perspective.
Butler said Akebia discussed potential subpopulations with high unmet need where benefits could outweigh perceived risk and that the company continues to explore those possibilities, while taking a conservative view. He also said the company expects some “non-promoted use” in non-dialysis settings, though it would require physicians to pursue medical exceptions and reimbursement.
Pipeline: praliciguat in FSGS and AKB-097 basket study
Butler said Akebia’s rare kidney disease pipeline includes praliciguat—licensed from Cyclerion in 2021—and AKB-097, in-licensed from Q32 Bio in December.
Burke said praliciguat is a small molecule that stimulates soluble guanylate cyclase to increase cyclic GMP, supporting nitric oxide signaling. He described the drug as beneficial to podocytes and having anti-inflammatory and anti-fibrotic effects. Akebia initiated a Phase II study in focal segmental glomerulosclerosis (FSGS) in December and has dosed the first patients.
For the Phase II FSGS trial, Burke described a randomized, double-blind, placebo-controlled design in roughly 60 patients, with six months of treatment and a crossover option for placebo patients. The primary endpoint is reduction in urine protein-to-creatinine ratio (UPCR). Management said it has not guided to timing for data readout due to enrollment considerations in a competitive and heterogeneous disease area; the trial will focus on primary FSGS and genetically attributable FSGS while excluding secondary forms.
On AKB-097, Burke described the molecule as a fusion protein that targets C3d at sites of complement activation and includes Factor H components intended to inhibit the alternative pathway locally. He said the approach may avoid systemic complement inhibition associated with marketed agents that carry boxed warnings for serious infections, and he suggested dosing could be weekly or every two weeks with an auto-injector over time. Butler said Akebia plans to start a basket study in the second half of the year in IgA nephropathy (IgAN), C3 glomerulopathy (C3G), and lupus nephritis, following a protocol Q32 had already aligned with FDA. The company expects to begin sharing data in 2027.
Butler also noted an internal research HIF compound, AKB-9090, with a Phase I in healthy volunteers expected to start and be completed by the end of the year, targeting acute kidney injury associated with cardiac surgery as an initial indication.
On financial resources, CFO Erik Ostrowski said Akebia had $166 million in cash as of the end of the third quarter and has guided to at least two years of cash runway. Management pointed to VAFSEO launch progress, VOCAL and VOICE readouts, and advancement of praliciguat and AKB-097 as key events investors are likely to watch over the coming year.
About Akebia Therapeutics (NASDAQ:AKBA)
Akebia Therapeutics, Inc, a clinical-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, is focused on the development and commercialization of therapies for patients with kidney disease. The company’s lead product candidate, vadadustat, is an investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor designed to treat anemia associated with chronic kidney disease in both dialysis-dependent and non-dialysis patients. Akebia’s research and development efforts also extend to preclinical programs targeting nephrology and related metabolic disorders.
Since its founding in 2007, Akebia has pursued strategic collaborations to advance its clinical pipeline and expand its market reach.
