AKBA

• Pipeline and R&D Progress

  • Initiated a Phase II basket trial for abribifus (EBRI), a next-generation complement inhibitor, in three rare complement-mediated kidney diseases: IgA nephropathy, lupus nephritis, and C3 glomerulopathy. The trial will enroll up to 30 patients receiving once-weekly subcutaneous dosing for 26 weeks, with initial data expected in 2027. Abribifus is designed to target kidney glomeruli directly without systemic blood complement inhibition, which the company believes will give it a better long-term safety profile than existing therapies.
  • Enrollment is ongoing for the Phase II trial of perlisiguat (PRALI), a small molecule soluble guanylate cyclase stimulator, in focal segmental glomerulosclerosis (FSGS). The randomized, double-blind, placebo-controlled trial will enroll up to 60 patients, with primary endpoint measured at 24 weeks. A Phase II study of perlisiguat in diabetic kidney disease conducted by partner Cyclarion has already demonstrated rapid, sustained proteinuria reduction.
  • AKB 9090 is progressing through its Phase I SAD/MAD study in healthy volunteers, with data expected to be reported early 2027. After this readout, 2027 R&D resources will be prioritized to abribifus and perlisiguat, the company's highest near-term value opportunities. A Phase II study of abribifus in ANCA-associated vasculitis is planned to start in 2027.

• Vafsio Commercial and Clinical Progress

  • The independent VOICE trial of Vafsio for anemia due to CKD in dialysis patients was stopped early after an interim analysis found it met pre-specified stopping criteria, demonstrating statistically significant and clinically meaningful reduction in the composite endpoint of all-cause mortality and hospitalization, driven by a 10% reduction in hospitalization. This is the first head-to-head ESA study ever to show a significant hospitalization benefit, consistent with prior post-hoc Phase III data.
  • Vafsio hit a milestone of over 10,500 active patients in Q2 2026, a 41% sequential increase from Q1. Quarterly new patient starts were the highest since launch, showing strong adoption momentum.
  • The prescriber base is diversified, with approximately one-third of prescribers from non-USRC dialysis organizations. Mid-sized dialysis organizations USRC, IRC, and DCI drove most patient growth in the quarter, and all three still have significant room for further expansion. DaVita, the single largest growth opportunity, saw increased new patient and new prescriber growth in Q2, with heightened senior clinical engagement on operational implementation of Vafsio following the VOICE trial results.
  • A streamlined, more targeted commercial strategy was implemented in Q2 to improve field team efficiency, with increased focus on large group practices and strategic partners.

• After Vafsio's period of regulatory exclusivity expires on December 31, 2026, the company plans to reduce Vafsio's price to fall within the range of existing ESAs, which is significantly lower than its current price. As a result, although Vafsio unit sales are expected to increase in 2027 compared to 2026, total 2027 Vafsio revenue is expected to decrease year-over-year.
• Arixia revenues are expected to continue to decrease in 2026 due to ongoing generic competition and pricing pressure.
• The company expects its existing $155.5 million in cash and cash equivalents (as of June 30, 2026), plus expected future revenue from products, royalties, supply and licensing agreements, along with planned refinancing of its senior secured term loan, will be sufficient to fund its current operating plan for at least the next two years.

Total revenues for Q2 2026 were $49.1 million, down from $62.5 million in Q2 2025. The decrease was driven by lower Arixia revenues, partially offset by growth in Vafsio revenue.

• Vafsio: Net product revenue was $21.3 million in Q2 2026, representing a 60% year-over-year increase (from $13.3 million in Q2 2025) and a 34% sequential increase from Q1 2026. Vafsio accounted for ~43.4% of total Q2 2026 revenue.
• Arixia: Net product revenue was $25.5 million in Q2 2026, down from $47.2 million in Q2 2025, due to generic competition. Arixia accounted for ~51.9% of total Q2 2026 revenue.
• License, Collaboration, and Other: Revenues increased to $2.4 million in Q2 2026, up from $2 million in Q2 2025. This segment accounted for ~4.9% of total Q2 2026 revenue.

Additional cost and profit metrics:

• Cost of goods sold: $10.4 million in Q2 2026 vs $9.9 million in Q2 2025
• R&D expenses: $14.1 million in Q2 2026 vs $11 million in Q2 2025
• SG&A expenses: $28.2 million in Q2 2026 vs $26.6 million in Q2 2025
• Net loss: $8.9 million in Q2 2026 vs a net income of $0.2 million in Q2 2025

• Forward-looking statements about pipeline progress, Vafsio adoption, and future financial performance are inherently subject to risks and uncertainties that could cause actual results to differ materially from expectations, with additional details available in the company's SEC filings.
• The rare kidney disease pipeline space is competitive, which could slow enrollment for clinical trials and impact future commercial positioning if approved.
• Adoption of Vafsio by large dialysis organizations such as DaVita and Fresenius takes significant time and organizational alignment, and there is uncertainty around the exact timing and magnitude of future adoption growth, even with positive clinical data.

Q: Near-term Vafsio growth: will it come more from new patients or from expanding in-center protocols for existing patients? / A: Most growth is driven by new patients from expanding numbers of active clinics and prescribers, which grew 17% quarter-over-quarter. About 25% of patients who discontinued therapy in 2025 have restarted after switching to observed in-center dosing protocols, which also contributes to growth. The VOICE trial results are expected to further accelerate all sources of growth.

Q: Can you share 80-90 day Vafsio persistence rates and discontinuation reasons, and what is the timeline for rolling out VOICE data to medical organizations? / A: First refill adherence is 89%, which is strong. Discontinuation after that aligns with normal churn in the dialysis population, with the most common reasons being hospitalization, patient intolerance (rare GI issues), and lack of efficacy in some patients. While the data has not been formally presented or published yet, principal investigator Jeff Block and the company's medical team are already sharing results with provider leadership across major dialysis organizations, with formal presentation and peer-reviewed publication targeted as quickly as possible. Large provider groups including DaVita have already begun operational planning for expanded adoption.

Q: How is perlisiguat Phase II enrollment progressing, and what is the opportunity for combination therapy with newly approved products for FSGS and complement-mediated kidney diseases? / A: Enrollment is progressing well, but the company will not commit to a specific data readout timeline until all 60 patients are fully enrolled. There is room for multiple therapies in the heterogeneous FSGS market; perlisiguat works via a unique soluble guanylate cyclase pathway that is distinct from newly approved sparsentin, making combination therapy a promising potential opportunity. For abribifus in complement-mediated diseases, the tissue-targeted safety profile makes it well-suited for combination with B-cell directed therapies, as it avoids the systemic infection risk of existing systemic complement inhibitors.

Q: How much Vafsio growth comes from non-USRC uptake, have protocols changed since VOICE, and what is the timeline for DaVita adoption? / A: Roughly one-third of Vafsio prescribers are now non-USRC; USRC, IRC, and DCI combined have ~66,000 total dialysis patients, with only ~10,500 total Vafsio patients across all providers, leaving significant room for growth. DaVita rolled out a system-wide observed dosing protocol for Vafsio in early June 2026, which was the first major protocol change after VOICE. DaVita physician awareness, likelihood to recommend, and preference for Vafsio are at all-time highs, but large organizational change takes time. The company expects meaningful adoption growth to occur by the end of 2026, setting up strong growth for 2027.