Akebia Reports Third Quarter 2020 Financial Results

11/5/20

Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose of bettering the lives of people impacted by kidney disease, today reported financial results for the third quarter ended September 30, 2020 and provided business updates, including confirming completion of a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) for vadadustat. Vadadustat is Akebia's investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) in development for the treatment of anemia due to chronic kidney disease (CKD) in adult patients on dialysis and not on dialysis.

"We recently completed our pre-NDA meeting with the FDA. This was an important milestone for our vadadustat development program, and we remain on track to submit an NDA to the FDA as early as possible next year. A key component of this NDA is the positive data from our global Phase 3 INNO2VATE program for the treatment of anemia due to CKD in adult patients on dialysis, which we shared most recently at ASN Kidney Week. These data were clear and consistent, and showed that vadadustat achieved both the primary and key secondary efficacy endpoints, as well as the primary and key secondary safety endpoints of the program for patients on dialysis. Based on our pre-NDA meeting, we remain confident that these results support the potential approval of vadadustat for the treatment of anemia due to CKD in adult patients on dialysis," said John P. Butler, President and Chief Executive Officer of Akebia Therapeutics. "Subject to regulatory review and approval, we believe vadadustat has the potential to be a new oral standard of care to help address the unmet needs of adult patients on dialysis, including both incident and prevalent dialysis patients. We believe this could translate into a potential $2 billion market opportunity in the U.S., alone. Together with our collaborator, Otsuka, we look forward to bringing this innovative therapy to patients on dialysis globally, if approved."

Butler continued, "The pre-NDA meeting also allowed us the opportunity to clarify key questions regarding data from PRO2TECT, our global Phase 3 program for the treatment of anemia due to CKD in adult patients not on dialysis, and we look forward to working with the FDA in their review of these data. While the PRO2TECT data showed that vadadustat achieved both the primary and key secondary efficacy endpoints, it did not meet the program's primary safety endpoint for patients not on dialysis, and we remain appropriately cautious in our outlook for potential approval of vadadustat in patients not on dialysis. Importantly, we believe the PRO2TECT data will not adversely impact the potential approvability of vadadustat for the treatment of anemia due to CKD in adult patients on dialysis."

Akebia plans to submit a New Drug Application (NDA) to the FDA for vadadustat as early as possible in 2021 for two indications: (1) the treatment of anemia due to CKD in adult patients on dialysis, and (2) the treatment of anemia due to CKD in adult patients not on dialysis. In addition, Akebia and its collaborator, Otsuka Pharmaceutical Co. Ltd., are working in close collaboration to prepare a Marketing Authorization Application (MAA) for submission to the European Medicines Agency (EMA) next year.

Recent Business Highlights

  • In October, the Company presented data from its global Phase 3 program at American Society of Nephrology Kidney Week 2020 Reimagined (ASN Kidney Week). Akebia's global Phase 3 program consists of two programs that evaluated the efficacy and safety of vadadustat versus darbepoetin alfa for the treatment of anemia due to CKD in adult patients on dialysis (INNO2VATE) and not on dialysis (PRO2TECT).
    • Highlights of INNO2VATE ASN Kidney Week Presentation: As previously reported in May 2020, vadadustat achieved the primary and key secondary efficacy endpoints and the primary safety endpoint of the INNO2VATE program, defined as non-inferiority of vadadustat versus darbepoetin alfa in time to first occurrence of a major adverse cardiovascular event (MACE), which is the composite of all-cause mortality, non-fatal myocardial infarction (MI), or non-fatal stroke, across both INNO2VATE studies. Newly presented INNO2VATE data showed that vadadustat also achieved non-inferiority to darbepoetin alfa on key secondary safety endpoints including expanded MACE, cardiovascular MACE, cardiovascular mortality, and all-cause mortality.
    • Highlights of PRO2TECT ASN Kidney Week Presentation: As previously reported in September 2020, vadadustat achieved the primary and key secondary efficacy endpoints of the PRO2TECT program, but did not meet the primary safety endpoint. Newly presented pre-specified regional analyses of the PRO2TECT program showed vadadustat demonstrated no clinically meaningful increase in cardiovascular risk compared to darbepoetin alfa in analyses of MACE, expanded MACE and all-cause mortality in U.S. patients treated to a target hemoglobin (Hb) range of 10 to 11 g/dL, consistent with U.S. treatment guidelines.
  • In October, Akebia and Otsuka launched Balancing Anemia Due to CKD, a campaign and website designed to increase awareness and education of anemia due to CKD among healthcare providers with the goal of improving the management of this disease for patients.
  • In August, the Company announced the launch of vadadustat in Japan by Mitsubishi Tanabe Pharma Corporation (MTPC), Akebia's partner in Japan, as a treatment for anemia due to CKD in both adult patients on dialysis and not on dialysis under the trade name VAFSEO™.
  • In July, the Company announced an investigator-sponsored research study by The University of Texas Health Science Center at Houston (UTHealth) in Houston, Texas, evaluating the use of vadadustat as a potential therapy to prevent and lessen the severity of acute respiratory distress syndrome (ARDS), a complication of COVID-19. The study is currently underway and actively enrolling patients.

Third Quarter Financial Results

  • Revenues: Total revenue was $60.0 million for the third quarter of 2020 compared to $92.0 million for the third quarter of 2019. The decline versus the prior year period was driven by lower collaboration revenue consistent with the Company completing the INNO2VATE and PRO2TECT studies.
    • Collaboration revenue was $25.6 million for the third quarter of 2020 compared to $62.0 million in the third quarter of 2019, and included $0.4 million in royalty revenue related to the commercial sale of vadadustat (VAFSEO™) in Japan from MTPC.
    • Net product revenue for Auryxia® (ferric citrate) was $34.4 million for the third quarter of 2020 compared with $30.0 million in the third quarter of 2019, an increase of 14.6 percent.
  • COGS: Cost of goods sold was $30.3 million for the third quarter of 2020 compared to $38.3 million for the third quarter of 2019 and includes the impact of $9.9 million in non-cash inventory write-downs largely related to a previously disclosed manufacturing quality issue related to Auryxia.
  • R&D Expenses: Research and development expenses were $46.9 million for the third quarter of 2020 compared to $74.5 million for the third quarter of 2019. The decline versus the prior year period was primarily driven by a decrease in costs consistent with the Company completing the INNO2VATE and PRO2TECT studies.
  • SG&A Expenses: Selling, general and administrative expenses were $40.2 million for the third quarter of 2020 compared to $34.2 million for the third quarter of 2019. The increase was primarily a result of higher professional service fees related to marketing and pre-commercialization activities for vadadustat and legal fees.
  • Net Loss: Net loss was $60.0 million for the third quarter of 2020 compared to $54.6 million for the third quarter of 2019. The increase in net loss compared to the prior year period was due primarily to lower total revenue and more specifically, lower collaboration revenue, partially offset by lower operating expenses.
  • Cash Position: Cash, cash equivalents and available-for-sale securities as of September 30, 2020 were $269.3 million. The Company expects its cash resources to fund its current operating plan beyond the expected U.S. launch of vadadustat, assuming regulatory approval.

About Akebia Therapeutics

Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. The Company was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at www.akebia.com, which does not form a part of this release.

About Vadadustat
Vadadustat is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor designed to mimic the physiologic effect of altitude on oxygen availability. At higher altitudes, the body responds to lower oxygen availability with stabilization of hypoxia-inducible factor, which can lead to increased red blood cell production and improved oxygen delivery to tissues. Vadadustat recently completed its global Phase 3 development program for the treatment of anemia due to CKD. Vadadustat is not approved by the U.S. Food and Drug Administration (FDA) or any regulatory authority with the exception of Japan's Ministry of Health, Labour and Welfare (MHLW). In Japan, vadadustat is approved as a treatment for anemia due to CKD in both dialysis-dependent and non-dialysis dependent adult patients.