UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(D)
OF THE SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of earliest event reported):
(Exact name of registrant as specified in its charter)
| (State or other jurisdiction of incorporation) |
(Commission File Number) |
(IRS Employer Identification No.) |
| |
||
| (Address of principal executive offices) | (Zip Code) |
Registrant’s telephone number, including area code:
N/A
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
| Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class |
Trading symbol(s) |
Name of each exchange on which registered | ||
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 7.01 | Regulation FD Disclosure. |
John P. Butler, President and Chief Executive Officer of Akebia Therapeutics, Inc. (the “Company”), plans to virtually present the information in the J.P. Morgan Healthcare Conference Presentation attached hereto as Exhibit 99.1 (the “Presentation”) at the 39th Annual J.P. Morgan Healthcare Conference on January 13, 2021 at 2:50 p.m., Eastern Time. Spokespersons of the Company also plan to present the information in the Presentation at various meetings beginning on January 12, 2021, including investor and analyst meetings.
The information in this Item 7.01 of this Current Report on Form 8-K and Exhibit 99.1 hereto shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities under that Section. The information contained in this Item 7.01 and Exhibit 99.1 hereto shall not be incorporated by reference into any filing with the U.S. Securities and Exchange Commission (the “SEC”) made by the Company under the Securities Act of 1933, as amended, or the Exchange Act, whether made before or after the date hereof, regardless of any general incorporation language in such filing.
By providing the information in Item 7.01 of this Current Report on Form 8-K, including Exhibit 99.1 hereto, the Company is not making an admission as to the materiality of any information herein. The information contained in this Current Report on Form 8-K is intended to be considered in the context of more complete information included in the Company’s filings with the SEC and other public announcements that the Company has made and may make from time to time by press release or otherwise. The Company undertakes no duty or obligation to update or revise the information contained in this Current Report on Form 8-K, although it may do so from time to time as its management believes is appropriate. Any such updating may be made through the filing of other reports or documents with the SEC, through press releases or through other public disclosures.
| Item 9.01 | Financial Statements and Exhibits. |
(d) Exhibits.
| Exhibit |
Exhibit Description | |
| 99.1 | J.P. Morgan Healthcare Conference Presentation | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| AKEBIA THERAPEUTICS, INC. | ||||||
| Date: January 12, 2021 | By: | /s/ John P. Butler | ||||
| Name: | John P. Butler | |||||
| Title: | President and Chief Executive Officer | |||||
Exhibit 99.1 1Exhibit 99.1 1
Statements in this presentation regarding Akebia’s strategy, plans, prospects, populations; the timing of or likelihood of regulatory filings and approvals, including labeling or expectations, beliefs, intentions and goals are forward-looking statements within the meaning of other restrictions, such as the anticipated timing of filing a NDA to the FDA and MAA to the EMA the U.S. Private Securities Litigation Reform Act of 1995, as amended, including but not limited for vadadustat, the potential approval of vadadustat and our outlook related thereto, and to submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and potential indications for vadadustat; the potential direct or indirect impact of the coronavirus 2 the timing thereof; Akebia’s cash runway funding Akebia’s current operating plan beyond the (SARS-CoV-2) pandemic on Akebia’s business, operations, and the markets and communities in expected U.S. launch of vadadustat, assuming regulatory approval; establishing vadadustat as a which Akebia and its partners, collaborators, vendors and customers operate; manufacturing and new oral standard of care for treatment of adult patients with anemia due to chronic kidney quality risks; risks associated with management and key personnel; the actual funding required disease (CKD) on dialysis; establishing vadadustat as a potential oral alternative to injectable to continue to commercialize our commercial product, to develop and erythropoiesis-stimulating agents; the U.S. market opportunity for vadadustat to treat patients commercialize vadadustat, and to operate Akebia; market acceptance and coverage and on dialysis and opportunity for growth of such market; vadadustat supporting and enabling reimbursement of our commercial product and vadadustat, if approved; the risks associated with growth in the U.S. home dialysis market; the potential for vadadustat upon approval and potential generic entrants for our commercial product and vadadustat, if approved; early commercial launch; the potential for rapid adoption of vadadustat in U.S. dialysis patients; termination of any of Akebia’s collaborations; Akebia’s and its collaborators’ ability to satisfy sharing vadadustat clinical data, including in peer reviewed journals, as well as the timing their obligations under Akebia’s collaboration agreements; the competitive landscape for our thereof; submitting the E.U. Marketing Authorization Application (MAA) to the European commercial product and vadadustat; the scope, timing, and outcome of any legal, regulatory and Medicines Agency (EMA) and the timing thereof; the potential launch and commercialization administrative proceedings; changes in the economic and financial conditions of the businesses of vadadustat if approved by regulatory authorities; the potential for Akebia to receive of Akebia and its collaborations partners and vendors; expected reliance on third parties, regulatory and commercial milestone payments upon approval of vadadustat by regulatory including with respect to the development, manufacturing, supply and commercialization of our ® authorities; the growth opportunities for Auryxia (ferric citrate) in 2021 and Akebia’s ability to product and product candidates; and Akebia’s intellectual property position, including its ability execute thereon; the potential for vadadustat to be used as a therapy to prevent and lessen the to obtain, maintain and enforce patent and other intellectual property protection for our severity of ARDS and in other indications; the expansion of Akebia’s pipeline and portfolio of commercial product, vadadustat and any other product candidates. Other risks and uncertainties novel therapeutics, including by leveraging new partnership relationships; and market include those identified under the heading “Risk Factors” in Akebia’s Quarterly Report on Form opportunity, clinical opportunity, commercial potential, prevalence, and the growth in, and 10-Q for the quarter ended September 30, 2020 and other filings that Akebia may make with the potential demand for, vadadustat. The terms “believe,” “expect,” “goal,” “look forward,” U.S. Securities and Exchange Commission in the future. These forward-looking statements “opportunity,” “planned,” “potential,” “will”, derivatives of these words, and similar references (except as otherwise noted) speak only as of the date of this presentation, and except as are intended to identify forward-looking statements, although not all forward- required by law, Akebia does not undertake, and specifically disclaims, any obligation to update looking statements contain these identifying words. Each forward-looking statement is subject any forward-looking statements contained in this presentation. to risks and uncertainties that could cause actual results to differ materially from Vadadustat is an investigational drug and has not yet been approved by the U.S. Food and Drug those expressed or implied in such statement, including, among others, the potential therapeutic Administration or any regulatory authority with the exception of Japan's Ministry of Health, benefits, safety profile, and effectiveness of our product candidates, including vadadustat; the Labour and Welfare. potential indications, demand and market potential and acceptance of our product and product candidates, including our estimates regarding the potential market opportunity for our product, vadadustat or any other product candidates and the size of eligible patient 2Statements in this presentation regarding Akebia’s strategy, plans, prospects, populations; the timing of or likelihood of regulatory filings and approvals, including labeling or expectations, beliefs, intentions and goals are forward-looking statements within the meaning of other restrictions, such as the anticipated timing of filing a NDA to the FDA and MAA to the EMA the U.S. Private Securities Litigation Reform Act of 1995, as amended, including but not limited for vadadustat, the potential approval of vadadustat and our outlook related thereto, and to submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and potential indications for vadadustat; the potential direct or indirect impact of the coronavirus 2 the timing thereof; Akebia’s cash runway funding Akebia’s current operating plan beyond the (SARS-CoV-2) pandemic on Akebia’s business, operations, and the markets and communities in expected U.S. launch of vadadustat, assuming regulatory approval; establishing vadadustat as a which Akebia and its partners, collaborators, vendors and customers operate; manufacturing and new oral standard of care for treatment of adult patients with anemia due to chronic kidney quality risks; risks associated with management and key personnel; the actual funding required disease (CKD) on dialysis; establishing vadadustat as a potential oral alternative to injectable to continue to commercialize our commercial product, to develop and erythropoiesis-stimulating agents; the U.S. market opportunity for vadadustat to treat patients commercialize vadadustat, and to operate Akebia; market acceptance and coverage and on dialysis and opportunity for growth of such market; vadadustat supporting and enabling reimbursement of our commercial product and vadadustat, if approved; the risks associated with growth in the U.S. home dialysis market; the potential for vadadustat upon approval and potential generic entrants for our commercial product and vadadustat, if approved; early commercial launch; the potential for rapid adoption of vadadustat in U.S. dialysis patients; termination of any of Akebia’s collaborations; Akebia’s and its collaborators’ ability to satisfy sharing vadadustat clinical data, including in peer reviewed journals, as well as the timing their obligations under Akebia’s collaboration agreements; the competitive landscape for our thereof; submitting the E.U. Marketing Authorization Application (MAA) to the European commercial product and vadadustat; the scope, timing, and outcome of any legal, regulatory and Medicines Agency (EMA) and the timing thereof; the potential launch and commercialization administrative proceedings; changes in the economic and financial conditions of the businesses of vadadustat if approved by regulatory authorities; the potential for Akebia to receive of Akebia and its collaborations partners and vendors; expected reliance on third parties, regulatory and commercial milestone payments upon approval of vadadustat by regulatory including with respect to the development, manufacturing, supply and commercialization of our ® authorities; the growth opportunities for Auryxia (ferric citrate) in 2021 and Akebia’s ability to product and product candidates; and Akebia’s intellectual property position, including its ability execute thereon; the potential for vadadustat to be used as a therapy to prevent and lessen the to obtain, maintain and enforce patent and other intellectual property protection for our severity of ARDS and in other indications; the expansion of Akebia’s pipeline and portfolio of commercial product, vadadustat and any other product candidates. Other risks and uncertainties novel therapeutics, including by leveraging new partnership relationships; and market include those identified under the heading “Risk Factors” in Akebia’s Quarterly Report on Form opportunity, clinical opportunity, commercial potential, prevalence, and the growth in, and 10-Q for the quarter ended September 30, 2020 and other filings that Akebia may make with the potential demand for, vadadustat. The terms “believe,” “expect,” “goal,” “look forward,” U.S. Securities and Exchange Commission in the future. These forward-looking statements “opportunity,” “planned,” “potential,” “will”, derivatives of these words, and similar references (except as otherwise noted) speak only as of the date of this presentation, and except as are intended to identify forward-looking statements, although not all forward- required by law, Akebia does not undertake, and specifically disclaims, any obligation to update looking statements contain these identifying words. Each forward-looking statement is subject any forward-looking statements contained in this presentation. to risks and uncertainties that could cause actual results to differ materially from Vadadustat is an investigational drug and has not yet been approved by the U.S. Food and Drug those expressed or implied in such statement, including, among others, the potential therapeutic Administration or any regulatory authority with the exception of Japan's Ministry of Health, benefits, safety profile, and effectiveness of our product candidates, including vadadustat; the Labour and Welfare. potential indications, demand and market potential and acceptance of our product and product candidates, including our estimates regarding the potential market opportunity for our product, vadadustat or any other product candidates and the size of eligible patient 2
OUR HISTORY Innovation and Potential ® Auryxia AKEBIA TODAY 2021 F O RW ARD MOME N T U M Auryxia In the U.S., vadadustat is an investigational HIF PH inhibitor that is not approved by the FDA. 3 3OUR HISTORY Innovation and Potential ® Auryxia AKEBIA TODAY 2021 F O RW ARD MOME N T U M Auryxia In the U.S., vadadustat is an investigational HIF PH inhibitor that is not approved by the FDA. 3 3
AKEBIA TODAY 2021 F OR WA R D M OM E N TUM V AD AD U S T A T ADOPTION OF VADADUSTAT IN JAPAN FOR BOTH DIALYSIS AND NON-DIALYSIS AN ORAL HIF-PH INHIBITOR FOR THE ADULT PATIENTS TREATMENT OF ANEMIA DUE TO CHRONIC KIDNEY DISEASE (CKD) NEPHROLOGY FOCUSED COMMERCIAL TEAM IN THE U.S. WITH AURYXIA® ü PHASE 3 PROGRAM COMPLETED ü U.S. NDA SUBMISSION ON TRACK; FUNDING BEYOND THE EXPECTED EXPECTED AS EARLY AS 1 U.S. LAUNCH OF VADADUSTAT POSSIBLE IN 2021 HIF-PH or HIF-PHI is hypoxia-inducible factor prolyl hydroxylase inhibitor. NDA is New Drug Application. 1. 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. 4AKEBIA TODAY 2021 F OR WA R D M OM E N TUM V AD AD U S T A T ADOPTION OF VADADUSTAT IN JAPAN FOR BOTH DIALYSIS AND NON-DIALYSIS AN ORAL HIF-PH INHIBITOR FOR THE ADULT PATIENTS TREATMENT OF ANEMIA DUE TO CHRONIC KIDNEY DISEASE (CKD) NEPHROLOGY FOCUSED COMMERCIAL TEAM IN THE U.S. WITH AURYXIA® ü PHASE 3 PROGRAM COMPLETED ü U.S. NDA SUBMISSION ON TRACK; FUNDING BEYOND THE EXPECTED EXPECTED AS EARLY AS 1 U.S. LAUNCH OF VADADUSTAT POSSIBLE IN 2021 HIF-PH or HIF-PHI is hypoxia-inducible factor prolyl hydroxylase inhibitor. NDA is New Drug Application. 1. 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. 4
BURDEN OF DISEASE UNMET NEEDS CLINICAL IMPACT >500K Anemia due to CKD can contribute to PEOPLE IN THE U.S. risk of ESKD, cardiovascular (CV) ON DIALYSIS WITH disease, stroke, cognitive impairment, 2 ANEMIA DUE TO CKD CV-related complications and death ESKD is End Stage Kidney Disease. ESA is erythropoiesis-stimulating agent. Sources: 1 . Stauffer et al, PLOSONE, 2014. 2 2020 USRDS Annual Data Report: https://adr.usrds.org/2020/reference-tables. 5BURDEN OF DISEASE UNMET NEEDS CLINICAL IMPACT >500K Anemia due to CKD can contribute to PEOPLE IN THE U.S. risk of ESKD, cardiovascular (CV) ON DIALYSIS WITH disease, stroke, cognitive impairment, 2 ANEMIA DUE TO CKD CV-related complications and death ESKD is End Stage Kidney Disease. ESA is erythropoiesis-stimulating agent. Sources: 1 . Stauffer et al, PLOSONE, 2014. 2 2020 USRDS Annual Data Report: https://adr.usrds.org/2020/reference-tables. 5
L E AD I N VE S T I G AT I O N AL V AD ADU S TAT P R O D U C T CA N D ID AT E INNOVATIVE CONVENIENT ORAL ROBUST CLINICAL COMMERCIAL MoA DOSING RESULTS BREADTH An oral hypoxia-inducible Potential oral alternative to Positive Phase 3 clinical Strong commercial factor prolyl hydroxylase injectable ESAs results in adult patients with partnerships with industry inhibitor (HIF-PHI) designed anemia due to CKD on leaders to stimulate endogenous dialysis EPO production. Based on Nobel Prize winning science MoA is mechanism of action. EPO is erythropoietin. In the U.S., vadadustat is an investigational HIF PH inhibitor that is not approved by the FDA. 6L E AD I N VE S T I G AT I O N AL V AD ADU S TAT P R O D U C T CA N D ID AT E INNOVATIVE CONVENIENT ORAL ROBUST CLINICAL COMMERCIAL MoA DOSING RESULTS BREADTH An oral hypoxia-inducible Potential oral alternative to Positive Phase 3 clinical Strong commercial factor prolyl hydroxylase injectable ESAs results in adult patients with partnerships with industry inhibitor (HIF-PHI) designed anemia due to CKD on leaders to stimulate endogenous dialysis EPO production. Based on Nobel Prize winning science MoA is mechanism of action. EPO is erythropoietin. In the U.S., vadadustat is an investigational HIF PH inhibitor that is not approved by the FDA. 6
R O B U S T CLIN ICA L D ATA V AD ADU S TAT An oral HIF-PH inhibitor designed to stimulate endogenous EPO production : Phase 3 Program ü Demonstrated efficacy and cardiovascular safety of vadadustat for the treatment of anemia due to CKD in POTENTIAL NEW ORAL adult patients on dialysis STANDARD OF CARE FOR ANEMIA DUE TO Clinical data also demonstrated that vadadustat: CKD IN ADULT PATIENTS ü Maintained EPO within physiologic range ON DIALYSIS ü Minimized hemoglobin excursions ü Increased hemoglobin in predictable and controlled Vadadustat is not approved by the FDA. manner Sources: Data from: Akebia's global INNO VATE program which included two separate Phase 3 studies (Correction/Conversion and Conversion), and collectively enrolled 3,923 adult patients on dialysis with anemia 2 due to CKD. Both INNO VATE studies were global, multicenter, open label (sponsor blind), active-controlled (darbepoetin alfa - an injectable erythropoiesis stimulating agent (ESA)), non-inferiority studies; Akebia’s 2 Phase 1 Study in Normal Healthy Volunteers (CI-0002); and, Akebia’s Phase 2b Study in Dialysis-Dependent CKD Patients (CI-0011). 7R O B U S T CLIN ICA L D ATA V AD ADU S TAT An oral HIF-PH inhibitor designed to stimulate endogenous EPO production : Phase 3 Program ü Demonstrated efficacy and cardiovascular safety of vadadustat for the treatment of anemia due to CKD in POTENTIAL NEW ORAL adult patients on dialysis STANDARD OF CARE FOR ANEMIA DUE TO Clinical data also demonstrated that vadadustat: CKD IN ADULT PATIENTS ü Maintained EPO within physiologic range ON DIALYSIS ü Minimized hemoglobin excursions ü Increased hemoglobin in predictable and controlled Vadadustat is not approved by the FDA. manner Sources: Data from: Akebia's global INNO VATE program which included two separate Phase 3 studies (Correction/Conversion and Conversion), and collectively enrolled 3,923 adult patients on dialysis with anemia 2 due to CKD. Both INNO VATE studies were global, multicenter, open label (sponsor blind), active-controlled (darbepoetin alfa - an injectable erythropoiesis stimulating agent (ESA)), non-inferiority studies; Akebia’s 2 Phase 1 Study in Normal Healthy Volunteers (CI-0002); and, Akebia’s Phase 2b Study in Dialysis-Dependent CKD Patients (CI-0011). 7
Mean Change from Baseline in HGB Levels: Dialysis Dependent CKD Patients V AD AD U S TAT 1 INNO VATE Phase 3 Conversion (3,554 Prevalent DD-CKD Patients) 2 G L O B A L PHASE 3 Vadadustat efficacy demonstrated in two P R OG R A M Phase 3 studies: ü Achieved primary and key secondary efficacy endpoints (mean change in hemoglobin Mean Change from Baseline in HGB Levels: (HGB) levels) Dialysis Dependent CKD Patients • Weeks 24 to 36 1 INNO VATE Phase 3 Correction/Conversion (369 Incident DD-CKD Patients) 2 (primary) • Weeks 40 to 52 Two Phase 3 Studies (secondary) 3,923 Adult Patients Source: 1. K.-U. Eckardt, et al. Global Phase 3 Clinical Trials of Vadadustat on Dialysis for Treatment of Anemia in Patients With Dialysis-Dependent Chronic Kidney Disease (DD-CKD). Presented at: American Society of Nephrology Kidney Week; October 22, 2020. (Akebia’s Phase 3 randomized, open-label, active-controlled non-inferiority study assessed the efficacy and safety of (Prev(a Plr e e n vta a le nd I nt a nc nd I ide nc nt i P de on pul t ations) vadadustat compared to darbepoetin alfa in 3,923 dialysis-dependent subjects with anemia due to CKD, with a treatment duration of 52 weeks.) 8 Populations)Mean Change from Baseline in HGB Levels: Dialysis Dependent CKD Patients V AD AD U S TAT 1 INNO VATE Phase 3 Conversion (3,554 Prevalent DD-CKD Patients) 2 G L O B A L PHASE 3 Vadadustat efficacy demonstrated in two P R OG R A M Phase 3 studies: ü Achieved primary and key secondary efficacy endpoints (mean change in hemoglobin Mean Change from Baseline in HGB Levels: (HGB) levels) Dialysis Dependent CKD Patients • Weeks 24 to 36 1 INNO VATE Phase 3 Correction/Conversion (369 Incident DD-CKD Patients) 2 (primary) • Weeks 40 to 52 Two Phase 3 Studies (secondary) 3,923 Adult Patients Source: 1. K.-U. Eckardt, et al. Global Phase 3 Clinical Trials of Vadadustat on Dialysis for Treatment of Anemia in Patients With Dialysis-Dependent Chronic Kidney Disease (DD-CKD). Presented at: American Society of Nephrology Kidney Week; October 22, 2020. (Akebia’s Phase 3 randomized, open-label, active-controlled non-inferiority study assessed the efficacy and safety of (Prev(a Plr e e n vta a le nd I nt a nc nd I ide nc nt i P de on pul t ations) vadadustat compared to darbepoetin alfa in 3,923 dialysis-dependent subjects with anemia due to CKD, with a treatment duration of 52 weeks.) 8 Populations)
Vadadustat cardiovascular safety demonstrated across two V AD AD U S TAT Phase 3 studies: G L O B A L ü Achieved primary and key secondary safety endpoints PHASE 3 Primary and Key Secondary Safety Endpoints: Dialysis Dependent CKD Patients P R OG R A M 1 INNO VATE Phase 3 Studies (Prevalent and Incident DD-CKD Patients) 2 Two Phase 3 Studies 3,923 Adult Patients on Dialysis All MACE safety endpoints were adjudicated by a committee blinded to treatment assignment. (Prevalent and Incident Source: 1. K.-U. Eckardt, et al. Global Phase 3 Clinical Trials of Vadadustat for Treatment of Anemia in Patients With Dialysis-Dependent Chronic Kidney Disease. Presented at: American Society of Nephrology Kidney Week; October 22, 2020. (Akebia’s Phase 3 randomized, open-label, active-controlled, non-inferiority study assessed the efficacy and safety of vadadustat compared to darbepoetin alfa in 3,923 dialysis- dependent subjects with anemia due to CKD, with a treatment duration of 52 weeks.) MACE is the composite of all-cause mortality, non-fatal myocardial infarction, or non-fatal stroke. 9 Populations)Vadadustat cardiovascular safety demonstrated across two V AD AD U S TAT Phase 3 studies: G L O B A L ü Achieved primary and key secondary safety endpoints PHASE 3 Primary and Key Secondary Safety Endpoints: Dialysis Dependent CKD Patients P R OG R A M 1 INNO VATE Phase 3 Studies (Prevalent and Incident DD-CKD Patients) 2 Two Phase 3 Studies 3,923 Adult Patients on Dialysis All MACE safety endpoints were adjudicated by a committee blinded to treatment assignment. (Prevalent and Incident Source: 1. K.-U. Eckardt, et al. Global Phase 3 Clinical Trials of Vadadustat for Treatment of Anemia in Patients With Dialysis-Dependent Chronic Kidney Disease. Presented at: American Society of Nephrology Kidney Week; October 22, 2020. (Akebia’s Phase 3 randomized, open-label, active-controlled, non-inferiority study assessed the efficacy and safety of vadadustat compared to darbepoetin alfa in 3,923 dialysis- dependent subjects with anemia due to CKD, with a treatment duration of 52 weeks.) MACE is the composite of all-cause mortality, non-fatal myocardial infarction, or non-fatal stroke. 9 Populations)
Summary of Treatment Emergent Adverse Events (TEAEs) V AD AD U S TAT and TEAEs Occurring in >10% of Patients in Either Treatment Group G L O B A L 1 INNO VATE Phase 3 Studies (Prevalent and Incident DD-CKD Patients) 2 PHASE 3 P R OG R A M Two Phase 3 Studies 3,923 Adult Patients on Dialysis Source: 1. K.-U. Eckardt, et al. Global Phase 3 Clinical Trials of Vadadustat for Treatment of Anemia in Patients With Dialysis-Dependent Chronic Kidney Disease. Presented at: American Society of Nephrology (Prevalent and Incident Kidney Week; October 22, 2020. (Akebia’s Phase 3 randomized, open-label, active-controlled non-inferiority study assessed the efficacy and safety of vadadustat compared to darbepoetin alfa in 3,923 dialysis-dependent subjects with anemia due to CKD, with a treatment duration of 52 weeks.) 10 Populations)Summary of Treatment Emergent Adverse Events (TEAEs) V AD AD U S TAT and TEAEs Occurring in >10% of Patients in Either Treatment Group G L O B A L 1 INNO VATE Phase 3 Studies (Prevalent and Incident DD-CKD Patients) 2 PHASE 3 P R OG R A M Two Phase 3 Studies 3,923 Adult Patients on Dialysis Source: 1. K.-U. Eckardt, et al. Global Phase 3 Clinical Trials of Vadadustat for Treatment of Anemia in Patients With Dialysis-Dependent Chronic Kidney Disease. Presented at: American Society of Nephrology (Prevalent and Incident Kidney Week; October 22, 2020. (Akebia’s Phase 3 randomized, open-label, active-controlled non-inferiority study assessed the efficacy and safety of vadadustat compared to darbepoetin alfa in 3,923 dialysis-dependent subjects with anemia due to CKD, with a treatment duration of 52 weeks.) 10 Populations)
ADVANCING VADADUSTAT FOR PATIENTS ON DIALYSIS 2021 INNO VATE Data 2 Positive Top-line Presented at ASN Completed Pre-NDA MI LE S T O N E S INNO VATE Data 2 Meeting with FDA Kidney Week Reported • Publication of Inno2vate Data in Peer Reviewed Journal; Expected in 2021 • U.S. NDA Submission to FDA Expected as Early as Possible in TM Launch of Disease INNO VAT Methods 2021 (VAFSEO ) 2 Awareness Resources Manuscript Published in launched in Japan “Balancing Anemia Nephrology Dialysis • Working with Otsuka to Prepare Due to CKD” with. Transplantation EU MAA Submission to EMA; ……. Expected in 2021 FDA is U.S. Food and Drug Administration. NDA is New Drug Application. MAA is Marketing Authorization Application. EMA is European Medicines Agency. Otsuka Pharmaceutical Co. Ltd. (“Otsuka”) is Akebia’s collaborator. In the U.S., vadadustat is an investigational HIF PH inhibitor that is not approved by the FDA. 11ADVANCING VADADUSTAT FOR PATIENTS ON DIALYSIS 2021 INNO VATE Data 2 Positive Top-line Presented at ASN Completed Pre-NDA MI LE S T O N E S INNO VATE Data 2 Meeting with FDA Kidney Week Reported • Publication of Inno2vate Data in Peer Reviewed Journal; Expected in 2021 • U.S. NDA Submission to FDA Expected as Early as Possible in TM Launch of Disease INNO VAT Methods 2021 (VAFSEO ) 2 Awareness Resources Manuscript Published in launched in Japan “Balancing Anemia Nephrology Dialysis • Working with Otsuka to Prepare Due to CKD” with. Transplantation EU MAA Submission to EMA; ……. Expected in 2021 FDA is U.S. Food and Drug Administration. NDA is New Drug Application. MAA is Marketing Authorization Application. EMA is European Medicines Agency. Otsuka Pharmaceutical Co. Ltd. (“Otsuka”) is Akebia’s collaborator. In the U.S., vadadustat is an investigational HIF PH inhibitor that is not approved by the FDA. 11
H I GH GR O W TH O PPO R TU N I TY V AD ADU S TAT I N U . S. DI ALY SI S MARK E T Unmet clinical needs of CKD patients on dialysis $2 Billion Approx. 556K U.S. patients on dialysis; 90% Estimated U.S. 1 treated for anemia due to CKD Dialysis Market 2 Opportunity Unique market dynamics with dialysis center clinical protocols Emphasis on growing home dialysis market 12 Sources: 1. 2020 USRDS Annual Data Report: https://adr.usrds.org/2020/reference-tables. 2 Based on internal estimates and industry reports estimating ESA pricing. 12H I GH GR O W TH O PPO R TU N I TY V AD ADU S TAT I N U . S. DI ALY SI S MARK E T Unmet clinical needs of CKD patients on dialysis $2 Billion Approx. 556K U.S. patients on dialysis; 90% Estimated U.S. 1 treated for anemia due to CKD Dialysis Market 2 Opportunity Unique market dynamics with dialysis center clinical protocols Emphasis on growing home dialysis market 12 Sources: 1. 2020 USRDS Annual Data Report: https://adr.usrds.org/2020/reference-tables. 2 Based on internal estimates and industry reports estimating ESA pricing. 12
WEL L P OS I T I ON ED U P ON V AD ADU S TAT A P P R O VA L A N D LA UN C H Go-to-market ü Convenient oral dosing for all dialysis patients with unique opportunity to support and grow U.S. home dialysis strategy positions market vadadustat for 1 ü Favorable reimbursement model with TDAPA potential rapid adoption in up to ü Experienced nephrology focused salesforce in the U.S. with 60% of U.S. * dialysis patients ü Exclusive U.S. distribution channel into Fresenius Medical * Care with Vifor Pharma *Pursuant to the Vifor Amended Agreement, Akebia granted Vifor (International) Ltd. (“Vifor Pharma”) an exclusive license to sell vadadustat to Fresenius Kidney Care and to certain other third party dialysis organizations in the U.S., upon approval of vadadustat by the FDA, the earlier of reimbursement under TDAPA (defined below) or inclusion in the ESRD bundle and a milestone payment from Vifor. Source: 1 TDAPA: Transitional drug add-on payment adjustment, CMS Ruling CMS-1691-F. Medicare Program; End-Stage Renal Disease Prospective Payment System, Payment for Renal Dialysis Services Furnished to Individuals with Acute Kidney Injury, End-Stage Renal Disease Quality Incentive Program, Durable Medical Equipment, Prosthetics, Orthotics and Supplies (DMEPOS) Competitive Bidding Program (CBP) and Fee Schedule Amounts, and Technical Amendments to Correct Existing Regulations Related to the CBP for 13 Certain DMEPOS. WEL L P OS I T I ON ED U P ON V AD ADU S TAT A P P R O VA L A N D LA UN C H Go-to-market ü Convenient oral dosing for all dialysis patients with unique opportunity to support and grow U.S. home dialysis strategy positions market vadadustat for 1 ü Favorable reimbursement model with TDAPA potential rapid adoption in up to ü Experienced nephrology focused salesforce in the U.S. with 60% of U.S. * dialysis patients ü Exclusive U.S. distribution channel into Fresenius Medical * Care with Vifor Pharma *Pursuant to the Vifor Amended Agreement, Akebia granted Vifor (International) Ltd. (“Vifor Pharma”) an exclusive license to sell vadadustat to Fresenius Kidney Care and to certain other third party dialysis organizations in the U.S., upon approval of vadadustat by the FDA, the earlier of reimbursement under TDAPA (defined below) or inclusion in the ESRD bundle and a milestone payment from Vifor. Source: 1 TDAPA: Transitional drug add-on payment adjustment, CMS Ruling CMS-1691-F. Medicare Program; End-Stage Renal Disease Prospective Payment System, Payment for Renal Dialysis Services Furnished to Individuals with Acute Kidney Injury, End-Stage Renal Disease Quality Incentive Program, Durable Medical Equipment, Prosthetics, Orthotics and Supplies (DMEPOS) Competitive Bidding Program (CBP) and Fee Schedule Amounts, and Technical Amendments to Correct Existing Regulations Related to the CBP for 13 Certain DMEPOS.
FU T U RE RE VE N U E S T RU C T U RE S V AD ADU S TAT In addition to potential regulatory and commercial milestone payments, Akebia will receive the following on net sales of vadadustat, subject to approval: Akebia to share profit equally on all U.S. sales of vadadustat with Otsuka (50/50) • Akebia and Otsuka to share (50/50) a majority of the profit from United States Vifor Pharma’s sales of vadadustat to Fresenius Kidney Care and 1 other third party dialysis organizations 100% of Product Revenue Royalties to Royalties to Akebia tiered Akebia tiered up to up to 30% 20% International 2 3 Europe Japan Latin America 1. Pursuant to the Vifor Amended Agreement, Akebia granted Vifor (International) Ltd. (“Vifor Pharma”) an exclusive license to sell vadadustat to Fresenius Kidney Care and to certain other third party dialysis organizations in the U.S., upon approval of vadadustat by the FDA and other conditions discussed in Akebia’s SEC filings. Akebia will receive a majority of the profit from Vifor Pharma’s sales of vadadustat to FKC and other third party dialysis organizations. Akebia will then share revenue from this profit share with Otsuka pursuant to the Otsuka U.S. Agreement. Akebia currently retains rights to commercialize vadadustat for use in other dialysis organizations in the U.S., which will be done in collaboration with Otsuka following FDA approval. 2. Also includes Russia, China, Canada, Australia and Middle East . 3. As of August 2020, Akebia receives royalties on net sales of VAFSEO (vadadustat) in Japan. 14FU T U RE RE VE N U E S T RU C T U RE S V AD ADU S TAT In addition to potential regulatory and commercial milestone payments, Akebia will receive the following on net sales of vadadustat, subject to approval: Akebia to share profit equally on all U.S. sales of vadadustat with Otsuka (50/50) • Akebia and Otsuka to share (50/50) a majority of the profit from United States Vifor Pharma’s sales of vadadustat to Fresenius Kidney Care and 1 other third party dialysis organizations 100% of Product Revenue Royalties to Royalties to Akebia tiered Akebia tiered up to up to 30% 20% International 2 3 Europe Japan Latin America 1. Pursuant to the Vifor Amended Agreement, Akebia granted Vifor (International) Ltd. (“Vifor Pharma”) an exclusive license to sell vadadustat to Fresenius Kidney Care and to certain other third party dialysis organizations in the U.S., upon approval of vadadustat by the FDA and other conditions discussed in Akebia’s SEC filings. Akebia will receive a majority of the profit from Vifor Pharma’s sales of vadadustat to FKC and other third party dialysis organizations. Akebia will then share revenue from this profit share with Otsuka pursuant to the Otsuka U.S. Agreement. Akebia currently retains rights to commercialize vadadustat for use in other dialysis organizations in the U.S., which will be done in collaboration with Otsuka following FDA approval. 2. Also includes Russia, China, Canada, Australia and Middle East . 3. As of August 2020, Akebia receives royalties on net sales of VAFSEO (vadadustat) in Japan. 14
1 3 • Ongoing investigator-sponsored • U.S. NDA submission to FDA clinical study by UTHealth expected as early as possible in 2021 2021 Creating evaluating vadadustat as • EU MAA submission expected in 2021 potential therapy to prevent and lessen the severity of acute Future Value for respiratory distress syndrome (ARDS) Akebia STRATEGIC OBJECTIVES NOVEL 2 4 • Leverage Auryxia as clinical • Explore partnerships to expand catalyst and commercial our portfolio and leverage our beachhead for vadadustat in CKD expertise in R&D In the U.S., vadadustat is an investigational HIF PH inhibitor that is not approved by the FDA. 151 3 • Ongoing investigator-sponsored • U.S. NDA submission to FDA clinical study by UTHealth expected as early as possible in 2021 2021 Creating evaluating vadadustat as • EU MAA submission expected in 2021 potential therapy to prevent and lessen the severity of acute Future Value for respiratory distress syndrome (ARDS) Akebia STRATEGIC OBJECTIVES NOVEL 2 4 • Leverage Auryxia as clinical • Explore partnerships to expand catalyst and commercial our portfolio and leverage our beachhead for vadadustat in CKD expertise in R&D In the U.S., vadadustat is an investigational HIF PH inhibitor that is not approved by the FDA. 15
