Akebia Therapeutics (NASDAQ:AKBA) executives outlined the company’s near- and mid-term priorities at the Leerink Partners Global Healthcare Conference, emphasizing the commercialization of its anemia drug Vafseo, continued investment in kidney-disease programs, and smaller exploratory efforts outside nephrology.
Three-part capital allocation framework
Chief Executive Officer John Butler said the company manages spending “thoughtfully” as a commercial-stage business with a pipeline, and described three strategic spending buckets:
- Commercial execution and evidence generation for Vafseo, including commercialization efforts and ongoing Phase IV/Phase 3b-type studies intended to support broader adoption.
- Expansion of a kidney-focused pipeline, which Butler characterized as central to Akebia’s identity. He highlighted praliciguat, AKB-097, and AKB-9090 (in acute kidney injury).
- Smaller investments beyond kidney disease, including work involving Aduhelm in ARDS with UT Health to “prove the mechanism,” and AKB-10108 in retinopathy of prematurity, where toxicology work is underway.
Vafseo: standard-of-care ambition supported by outcomes and economic analyses
He also highlighted clinical outcomes analyses presented in recent conferences. At the American Society of Nephrology meeting in November, Akebia presented a post hoc “win odds” analysis from its Phase III INNO2VATE program, which Butler said evaluated prospectively collected endpoints of mortality and hospitalization. He said the analysis showed a statistically significant reduction in the risk of dying or being hospitalized with Vafseo. Butler added that the work has been accepted by a “top-tier nephrology journal,” with publication expected “very shortly,” which he said would allow the company’s medical team to educate physicians on the findings.
At a subsequent annual dialysis conference, Butler said Akebia presented an economic analysis focused on hospitalization. He cited an approximately 8% decrease in hospitalizations with Vafseo, driven by cardiovascular- and infection-related admissions, along with a 16% reduction in length of stay. Using “standard Medicare costs,” he said those factors translated to about a 15% cost reduction—roughly $3,700 per patient per year—and about $0.9 billion in savings if all eligible ESA-treated patients were switched to Vafseo. Butler said the company is working toward publication of that economic work.
Observed TIW dosing, and the VOICE and VOCAL studies
A key commercial and clinical topic was “TIW” (three-times-weekly) observed dosing, which Butler said is not yet in Vafseo’s label and is expected to be discussed with the FDA. He said daily dosing performs well, but sending medication home raised compliance concerns in a setting where patients have historically received in-chair injections. Butler said internal and collaborative work suggests TIW dosing is an option that can help anemia managers and nurses confirm adherence.
He pointed to the FO2CUS study as demonstrating TIW dosing and said two TIW studies—VOICE and VOCAL—are ongoing. Butler said VOICE investigator Geoffrey Block recommended transitioning patients at U.S. Renal to TIW dosing, and Butler expects the transition to be complete by the end of the quarter. He added that physicians have told the company that after an initial titration phase, patients tend to maintain hemoglobin well, contrasting this with ongoing dose adjustments associated with ESAs.
Discussing VOCAL, Butler said the 350-patient study is being run at DaVita centers and is sponsored by Akebia, potentially supporting a regulatory filing for TIW dosing if required by FDA. He said non-inferiority in hemoglobin management is the primary expectation. Butler also emphasized a VOCAL sub-study in roughly 30 patients evaluating red blood cell characteristics, including omics and metabolomics. He said the company aims to provide physicians with a mechanistic “why,” including the hypothesis that Vafseo produces a more physiologic red blood cell—larger and carrying more oxygen—and plans future work on red blood cell lifespan in dialysis patients.
On VOICE, Butler said he expects a similar magnitude of mortality and hospitalization differences as seen in the INNO2VATE win odds analysis. He also discussed ESA “rescue” use as a factor in earlier studies, noting that in FO2CUS, ESA rescue was about half, and said the VOICE design eliminates a dosing-related dip seen previously.
Reimbursement and the post-TDAPA transition
Butler and Chief Financial Officer Erik Ostrowski addressed how the company views reimbursement dynamics as TDAPA winds down. Butler said Akebia always viewed TDAPA as a limited period and expects longer-term pricing to be “in and around the ESAs.” He described ESA pricing as roughly $2,000 to $2,500 per year and said Vafseo can still be a “very pro-profitable product” at that level, particularly if it demonstrates savings to dialysis providers, including those operating under capitated arrangements.
Butler said investors may struggle with the complexity of dialysis reimbursement, noting there are relatively few TDAPA drugs. He framed a key question as whether volume growth can offset the revenue decline after TDAPA, and said Akebia does not expect to fully offset that near-term decline, describing a likely “divot” in revenue even as volume continues to rise.
Ostrowski said that at ESA-level pricing, the post-TDAPA market opportunity is about $1 billion. Butler added that broader availability after TDAPA ends could expand the eligible patient population. He also reiterated the company’s view that achieving standard of care in a $1 billion market could translate to a “$500-plus million product.”
Pipeline updates: praliciguat in FSGS and AKB-097 in complement-mediated diseases
Beyond Vafseo, Butler highlighted two kidney-focused development programs. For praliciguat, he said Akebia began a Phase II study in focal segmental glomerulosclerosis (FSGS) late last year after delays tied to a complete response letter period and drug supply issues with a partner. He described FSGS as high unmet need and said prior diabetic kidney disease data from Cyclerion showed reduced proteinuria, while Akebia’s animal studies supported potential efficacy in FSGS. Butler noted the PARASOL FDA-academic effort helped clarify approvable endpoints. He said the FSGS study has started and enrollment is progressing, but the company has not yet guided timing.
For AKB-097, which Akebia acquired in December from Q32 Bio, Butler said initial skepticism about another complement inhibitor gave way to enthusiasm after discussions with kidney medical experts. He described the asset as tissue-targeted, potentially enabling efficacy consistent with leading complement inhibitors while improving safety and dosing convenience. Butler said the company plans to initiate an open-label basket study in IgA nephropathy (IgAN), lupus nephritis, and C3 glomerulopathy (C3G) in the second half of the year, with data expected to begin emerging in 2027.
Ostrowski added that operational metrics the company is monitoring for Vafseo include expanding both the breadth and depth of prescribing, along with adherence rates, which he said appear to be improving with TIW dosing.
Butler said Akebia plans to host a virtual R&D day on April 2 featuring external key opinion leaders, which he said is intended to provide a deeper look at the pipeline.
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.
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