Full Press Release Details
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 PHASE 3 PROGRAM
COMPLETED 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 PHASE 3 PROGRAM COMPLETED 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.
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 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 Maintained EPO within physiologic range ON DIALYSIS Minimized hemoglobin excursions 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 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 Maintained EPO within physiologic range ON DIALYSIS Minimized hemoglobin excursions 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: 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: 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 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 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: