Akebia and Otsuka Pharmaceutical Announce Collaboration to Develop and Commercialize Vadadustat in the U.S.

  • Funds Vadadustat Global Phase 3 PRO2TECT and INNO2VATE Studies 
  • Committed Capital and Milestone Payments Potentially Exceed $1 Billion 
  • Akebia and Otsuka Share Revenue and Commercialization Costs Equally 
  • Akebia to Host Conference Call at 8:30 a.m. Eastern Time Today 
CAMBRIDGE,  MA  &  TOKYO,  JAPAN  --  December  20,  2016    Akebia  Therapeutics, Inc. (NASDAQ: AKBA) and  Otsuka Pharmaceutical Co., Ltd. today announced they have entered into a collaboration and license agreement in the U.S. for vadadustat, an oral hypoxia-inducible factor (HIF) stabilizer currently in development for the treatment of anemia associated with chronic kidney disease (CKD). Anemia related to CKD affects an estimated 1.8 million patients in the U.S and arises from the kidney’s failure to produce adequate amounts of erythropoietin, a key hormone stimulating  the  production  of  red  blood  cells.1   Left  untreated,  anemia  significantly  accelerates patients' overall deterioration of health with increased morbidity and mortality.2 The collaboration provides capital for the global development program for vadadustat, and commercial resources for a U.S. launch of vadadustat upon approval by the Food and Drug Administration. Under the terms of the agreement, Akebia will receive $265 million in committed funds plus development and commercial milestones, representing a total transaction value that potentially  could  exceed  $1  billion.  The  companies  intend  to  contribute  equally  to commercialization efforts and share equally all costs and revenue in the U.S., where sales of erythropoiesis stimulating agents (ESAs), the current standard of care, are estimated to be $3.5 billion.3   Akebia  will  continue  to  lead  the  ongoing  global  Phase  3  development  program  for vadadustat. “Vadadustat has the potential to significantly change the current standard of care for patients with anemia associated with CKD and addresses a high unmet need for those suffering with this disease,” said Mr. Tatsuo Higuchi, President and Representative Director of Otsuka.   “With Akebia’s renal expertise, this collaboration will enable Otsuka to expand our cardio-renal portfolio while demonstrating our commitment to delivering new treatment options to patients worldwide.” Under the terms of the agreement, Otsuka will pay $265 million or more in committed capital. This includes a payment of $125 million upon signing and a payment of approximately $35 million in the first quarter of 2017. The agreement also provides for Otsuka to pay $105 million or more of the costs of  the  global  development  program  for  vadadustat.  Additionally,  Otsuka  will  pay  potential development and commercial milestones up to $765 million. “This collaboration achieves our goal of funding our global PRO2TECT and INNO2VATE Phase 3 studies for vadadustat while retaining significant long-term value for Akebia,” stated John P. Butler, President and Chief Executive Officer of Akebia. Mr. Butler added, “Our alliance with Otsuka, one of the world’s innovative pharmaceutical leaders, also allows us to prepare an optimal launch of vadadustat, as we will equally share commercial responsibility. Otsuka brings a well-established commercial presence and infrastructure in the U.S., and we share a strong commitment to improve patient lives by delivering important new therapeutic options. This deal also underscores the confidence that we and others have placed in the underlying value of vadadustat and in our ability to bring innovative therapies to patients.” In addition to the collaboration with Otsuka, Akebia has established a collaboration with Mitsubishi Tanabe Pharma Corporation for the development and commercialization of vadadustat in Japan, Taiwan,  South  Korea,  Indonesia,  India  and  other  countries  in  Asia. For other  geographies, including the European Union, Akebia continues advancing discussions with multiple potential partners. Conference Call and Webcast Akebia management will host a conference call to review the details of the transaction beginning at 8:30 a.m. Eastern Time today, Tuesday, December 20, 2016. A live audio webcast of the presentation will   be   available   on   the company's website at http://ir.akebia.com/events.cfm. An   archived presentation will be available for 90 days. To access the conference call, follow these instructions: Dial: (877) 458-0977 (U.S.); (484) 653-6724 (international) Conference ID: 39235865 About Vadadustat Vadadustat is an oral hypoxia-inducible factor (HIF) stabilizer currently in development for the treatment of anemia related to chronic kidney disease. Vadadustat exploits the same mechanism of action used by the body to adapt naturally to lower oxygen availability associated with a moderate increase in altitude. At higher altitudes, the body responds to lower oxygen availability with increased production of HIF, which coordinates the interdependent processes of iron mobilization and erythropoietin production to increase red blood cell production and, ultimately, improve oxygen delivery.  About Anemia Associated with CKD Approximately 30 million people in the U.S. have chronic kidney disease (CKD), with an estimated 1.8 million of these patients suffering from anemia. Anemia results from the body's inability to coordinate red blood cell production in response to lower oxygen levels due to the progressive loss of kidney function with inadequate erythropoietin production. Left untreated, anemia significantly accelerates patients' overall deterioration of health with increased morbidity and mortality. Anemia is  currently  treated  with  injectable  recombinant erythropoiesis  stimulating  agents,  which  are associated with inconsistent hemoglobin responses and well-documented safety risks.4 About Akebia Therapeutics Akebia Therapeutics, Inc. is a biopharmaceutical company headquartered in Cambridge, Massachusetts, focused on delivering innovative therapies to patients with kidney disease through hypoxia-inducible factor biology. Akebia’s lead product candidate, vadadustat, is an oral therapy in development for the treatment of anemia related to chronic kidney disease in both non-dialysis and dialysis patients. Akebia’s global Phase 3 program for vadadustat, which includes the PRO2TECT studies for non-dialysis patients with anemia secondary to chronic kidney disease and the INNO2VATE studies for dialysis-dependent patients, is currently ongoing. For more information, please visit our website at www.akebia.com. About Otsuka Otsuka Pharmaceutical is a global healthcare company with the corporate philosophy: “Otsuka – people   creating   new  products   for better   health   worldwide.”   Otsuka   researches,   develops, manufactures and markets innovative and original products, with a focus on pharmaceutical products for the treatment of diseases and nutraceutical products for the maintenance of everyday health. Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC), based in Princeton, N.J., discovers and develops new compounds that address unanswered medical needs and advance human health, with a focus on neuroscience, oncology, and cardio-renal treatments. For more information about Otsuka in the U.S., visit www.otsuka-us.com and on Twitter at @OtsukaUS. Otsuka Pharmaceutical is a subsidiary of Otsuka Holdings Co., Ltd., headquartered in Tokyo, Japan, with 2015 consolidated sales of $11.9 billion. Otsuka welcomes you to visit its global website at https://www.otsuka.co.jp/en. Forward-Looking Statements This press release includes forward-looking statements. Such forward-looking statements include those  about  Akebia's  strategy,  future plans  and  prospects,  including  statements  regarding  the potential indications and benefits of vadadustat, the satisfaction of Akebia’s funding needs for the PRO2TECT and INNO2VATE Phase 3 programs, the potential commercialization of vadadustat if approved by the FDA, anticipated contributions from Otsuka pursuant to the Collaboration and License  Agreement,  and  the  progress  toward  securing  a  European  collaboration.  The  words “anticipate,” “appear,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including the risk that existing preclinical and clinical data may not be predictive of the results of ongoing  or  later  clinical  trials;  the  ability  of  Akebia  to  successfully  complete  the  clinical development of vadadustat; the funding required to develop Akebia's product candidates and operate the company, and the actual expenses associated therewith; the actual costs incurred in the Phase 3 studies of vadadustat and the availability of financing to cover such costs; the timing and content of decisions made by the FDA and other regulatory authorities; the rate of enrollment in clinical studies of vadadustat; the actual time it takes to initiate and complete clinical studies; Akebia’s ability to satisfy its obligations under the Collaboration and License Agreement; early termination of the Collaboration  and  License  Agreement  by  Akebia  or  Otsuka, Akebia's  ability  to  negotiate commercially reasonable terms with a European collaboration partner; the success of competitors in developing product candidates for diseases for which Akebia is currently developing its product candidates; and Akebia's ability to obtain, maintain and enforce patent and other intellectual property protection for vadadustat. Other risks and uncertainties include those identified under the heading “Risk Factors” in Akebia's Annual Report on Form 10-Q for the quarter ended September 30, 2016, and other filings that Akebia may make with the Securities and Exchange Commission in the future. Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release. Contacts: Akebia
AJ Gosselin
Manager, Corporate Communications
617-844-6130
agosselin@akebia.com Otsuka Pharmaceutical Development & Commercialization, Inc. (in U.S.)
Kimberly Whitefield
Corporate Communications  
609-535-9259 
kimberly.whitefield@otsuka-us.com Otsuka Pharmaceutical Co., Ltd. (in Japan)
Jeffrey Gilbert 
Leader, Pharmaceutical Public Relations 
81-3-6361-7379 
Gilbert.jeffrey@otsuka.jp December 2016     01US16EUC0228

References

1Stages 1-4: JAMA 2007 Coresh et al. (Prevalence of CKD in the US). NHANES 1988-94 and 1999-2004. Stage 5: USRDS 2013 report (ESRD). Iseki K and Kohagura. Anemia as a risk factor for chronic kidney disease K. Kidney Int Suppl. 2007; 107:S4-9. 2Culleton B, Manns B, Zhang J, Tonelli M, Klarenbach S, et al. Impact of anemia on hospitalization and mortality in older adults. Blood 2006; 107(10):3841-3846. Portolés J, Gorriz J, Rubio E, de Alvaro F, García F, et al. The development of anemia is associated to poor prognosis in NKF/KDOQI stage 3 chronic kidney disease. BMC nephrology 2013; 14(1):2. 3Global sales of injectable erythropoiesis-stimulating agents as reported in public filings. 4Singh AK. What is causing the mortality in treating the anemia of chronic kidney disease: erythropoietin dose or hemoglobin level? Curr Opin Nephrol Hypertens 2