Akebia Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by. And welcome to Akebia's Therapeutics Fourth Quarter of 2020 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you'll need to press star 1 on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today, Kristen Shepard, Senior Vice President of Investor Relations. Thank you. Please go ahead.

Thank you, and welcome to Akebia's fourth quarter and fiscal year 2020 Financial Results and Business Update conference call. Please note that the press release detailing our results for the fourth quarter and fiscal year 2020 was issued earlier this morning and is available on our Investor Relations website. For your convenience, a replay of today's call will also be available on our website shortly after we conclude today's event. Joining me for today's event is John Butler, our Chief Executive Officer, and David Spellman, our Chief Financial Officer. Before we begin, I'd like to remind everyone that this call includes forward-looking statements. Each forward-looking statement contained in this call is subject to risks and uncertainties that could cause actual results to differ materially from those described in these statements. Additional information regarding these factors is described in the forward-looking statements section of the press release we issued earlier today, as well as in the risk factors in management's discussion and analysis sections of our most recent quarterly and annual reports filed with the FCC. The four looking statements on this call speak only of the original date of this call, and except as required by law, we do not undertake any obligation to update or revise any of these statements. With that, I'd now like to introduce our CEO, John Butler. John?

Thank you, Kristen, and thank you all for joining us today. 2020 was a year of focused execution for Akibia. We set out to accomplish a lot in 2020, and we did what we said we would do, advancing Vatadustat, our lead product candidate, and laying the groundwork for a number of catalysts in 2021. More specifically, we achieved key clinical and regulatory milestones, including completing our global Phase III clinical development program for Vatadustat, and completing our pre-NDA meeting with the FDA. We also executed well against our commercial priorities. After securing the first regulatory approval of Vatadustat with our partner, MTPC, earlier in the year, we began supporting the commercialization of Vatadustat in Japan under the trade name Vafseo in Q3. As a company whose purpose is to better the lives of people impacted by kidney disease, I've been saddened to watch the disproportionate impact COVID-19 has had on patients on dialysis. Our team did a tremendous job of helping ensure that these patients who continue to be at the highest risk have access to our therapies despite the pandemic. As a result of these and other efforts, we grew Eurexia revenue over 2019. I'm incredibly proud of our entire team for all their work across 2020. and their continued commitment to both our purpose and our patients. Importantly, we achieved all this and more while keeping true to our goal of maintaining a strong balance sheet. With forward momentum from 2020, we have line of sight to significant milestones this year that we believe will further position Akibia to deliver on our strategy and build long-term value for our shareholders. Our highest priority is submitting our NDA for Vatadustat for the treatment of anemia due to CKD with the FDA. Since completing our pre-NDA meeting in late October, our team has been hard at work putting together a very detailed and comprehensive data package in support of Vatadustat's NDA submission. We remain firmly on track to achieve this milestone by the middle of the second quarter of this year. We also expect to follow this with Vatadustat's MAA submission to the EMA in collaboration with Otsuka this year. As we advance Vatadustat towards commercialization subject to approval, we continue to execute on key commercial, development, and financial priorities across our business. As dialysis is a primary focus, I'll start there. Data from Innovate, our global phase three program that evaluated the efficacy and safety of Vatadustat in patients on dialysis were clear, consistent, and compelling. The data showed that Vatadustat achieved non-inferiority to darbopoietin alpha on both the primary and key secondary hematologic efficacy endpoints, as well as the primary and key secondary safety endpoints that included MACE, expanded MACE, cardiovascular MACE, cardiovascular mortality, and all-cause mortality. Going a little deeper, Our clinical data also demonstrated that Vatadustat maintained EPO within a physiologic range, increased hemoglobin in a predictable and controlled manner, and minimized hemoglobin excursions above that target range. We believe dialysis represents a potential $2 billion market opportunity in the U.S. alone. We're confident that upon approval, we have the potential to address the unmet needs of the over 500,000 adult patients on dialysis in the U.S. and rapidly establish Vatadustat as the new oral standard of care for the treatment of anemia due to CKD. We anticipate that the strength of our data, which is clear and consistent across both prevalent and incident dialysis patients, will play a meaningful role in helping develop treatment protocols within dialysis providers. which are critical to driving adoption in the dialysis market, subject, of course, to regulatory approval. To further strengthen Vatadustat's potential profile for in-center dialysis patients, we're working to demonstrate that Vatadustat can also be dosed three times a week and plan to have that data available at launch, which will allow us to submit an SNDA for this regimen post-approval. Although the significant majority of dialysis patients are cared for in-center, recently several factors, including the COVID-19 pandemic, changing patient preferences, government initiatives, and reimbursement changes are supporting a shift towards home dialysis. We believe that as a convenient, once-daily oral therapeutic, Vatadustat has the potential to offer an important value proposition both to the growing number of home dialysis patients and to dialysis providers looking to better support these patients and grow their home programs. We believe we are well positioned to support a strong Vatadustat launch in dialysis in the U.S. upon approval. Our go-to-market strategy includes leveraging our existing nephrology-focused commercial organization with our partner Otsuka sharing in the launch costs and responsibilities. As well, we have an exclusive distribution relationship with V4 Pharma that positions Vatadustat for potential rapid adoption in up to 60% of the U.S. dialysis market. Adding to this is a unique reimbursement model in the U.S. dialysis market with TDAPA, an add-on payment to the bundle that's intended to encourage adoption of innovative therapies by clinicians and dialysis providers. To maximize our market potential and ensure commercial readiness upon U.S. approval, we're executing across many different work streams within medical affairs, scientific communications, disease state education, patient services, and manufacturing. We also recently added Leanne Zumwalt to our Board of Directors. Many of you may know Leanne as she spent over 20 years serving in key leadership roles with DaVita, most recently as Group Vice President of Government Affairs before her retirement earlier this year. Leanne's perspective and extensive operating experience with one of the largest dialysis providers in the U.S., will help ensure that our market and commercial strategies are well aligned with the needs of dialysis providers and their patients. We, along with the Executive Steering Committee, who has overseen our Phase III development program, believe it is critical to publish our data. And collectively, we continue to execute on our robust publication plan. To date, this has included the presentation of our Phase III data at ASN, and publication of both our Innovate and Protect Methods papers in peer-reviewed journals during the fourth quarter. Looking ahead, we plan to present additional data at the upcoming NKF spring clinical meetings in April, and we have line of sight to publication of our Innovate program in a prestigious peer-reviewed publication expected in the near term. Protect has been submitted earlier this year for publication as well. Again, there is a significant unmet need among patients with anemia due to CKD, and we see a promising opportunity in Vatadustat to advance the care of patients on dialysis. Turning to non-dialysis, as we discussed last quarter, we plan to submit data from PROTECT together with additional analyses and pursue a broad label for adult patients not on dialysis as part of our Vatadustat NDA submission. However, as PROTECT missed the primary MACE endpoint, we are remaining cautious on potential approval in non-dialysis. Now, in compiling the NDA, we've been able to take a deeper dive into the data to better understand PROTECT's MACE result. The cardiovascular outcomes reported in PROTECT contrast with those reported in the Innovate program, as well as with the safety data from other clinical and preclinical studies of Atadustat in both dialysis and non-dialysis adult patients. Additional in-depth analyses conducted by our team continue to demonstrate that the greater number of MACE events observed among Vatadustat patients not on dialysis, as compared with those on Darbopo and Alpha in the PROTECT program, was primarily related to an excess of non-cardiovascular deaths and deaths of unknown cause in regions outside of the United States where significant differences in treatment patterns for patients not on dialysis were observed. As we've previously discussed, we believe these and other analyses inform the cardiovascular safety profile of Vatadustat in U.S. patients not on dialysis. We continue to believe that pursuing approval in non-dialysis is the right thing to do for patients, and we look forward to working with the FDA in their review. In addition, I want to share that as part of our NDA work, we undertook a review of hepatic safety across the Vatadustat clinical programs. which includes data from over 8,000 patients across 36 completed Phase I, II, and III clinical studies conducted by Akibia and our partner, MTPC. The review of hepatic safety included a blinded assessment of all hepatic events in the studies by a panel of hepatic experts and analysis by an independent hepatic expert and our team. I'm very pleased to report that, based on this review, we concluded that the data across the clinical program showed that the hepatic safety profiles of Vatadustat and Darbopoietin-alpha in injectable ESA and the current standard of care appear to be similar, and that there were no HISE law events identified across the entire Vatadustat clinical development program. More specifically, we concluded that a 2014 case previously reported by us as meeting the biochemical criteria for Heise law, while complicated, was in fact not a case of Heise law. Our conclusion on this case will be reflected in our NDA for Vatadustat. We wanted to share this report finding as we believe it reinforces what we expect will be a high-quality NDA submission for Vatadustat. As well, this finding reinforces our confidence around the long-term strategic vision for Vatadustat. An important aspect of this strategy is focused on innovation. So let me shift a little bit and talk about our future development plans. Innovation is a core capability at Akebia that we have built over the past decade with robust research and clinical development resources. This engine has yielded significant scientific advancements, including Vatadustat, which is based on Nobel Prize winning science. While the near-term and largest commercial opportunity for Vatadustat is within anemia due to CKD, we believe there is the potential for additional opportunities for Vatadustat. We're encouraged that the current study evaluating the use of Vatadustat as a potential therapy to prevent and lessen the severity of acute respiratory distress syndrome, or ARDS, in adult patients who've been hospitalized due to COVID-19 is progressing well. As a reminder, This is a clinical investigator-sponsored study led by UT Health that we provided supportive funds for last year. UT Health has shared that over 120 patients are enrolled and recently announced that they have been awarded $5.1 million in funding from the US Department of Defense to expand this clinical trial at their facilities. If the results of this study are positive, we believe this COVID-related clinical experience could offer possibilities not only to move forward and help COVID patients, but also to explore Vatadustat for a large and enduring opportunity as a treatment for ARDS beyond COVID. After being relatively quiet in 2020, we look to share more information on potential development opportunities for Vatadustat later this year and where possible, updates from UT Health. To sum up, as we transition to 2021, we plan to continue executing to prepare for the expected commercialization of Vatadustat while investing in potential opportunities to help position Akibia for the long term. We're pleased to be doing all this from a place of financial strength, with a cash runway that extends beyond the expected U.S. launch of Vatadustat, assuming timely regulatory approval and the receipt of associated regulatory milestones. And as Dave will discuss, we remain committed to both maintaining a strong balance sheet and improving our cost structure moving forward. In furtherance of these objectives, we recently announced a $60 million transaction with healthcare royalty partners to monetize our Vafseo royalties and sales milestones under our collaboration agreement with MTPC. While Dave will provide more details shortly, we believe this transaction is strong validation of the value that Vafseo can bring to patients in Japan. As well, we believe it underscores our commitment to preserving both our strategic and financial flexibility while investing for the successful launch of Adadustat upon approval. As we head into the next chapter of Akebia, there's a lot of important work ahead of us in 2021. We remain confident that our hard work in 2020 has laid the foundation for the year ahead and the journey that we're on for the longer term. We're excited about the future, and together with our collaborator, Otsuka, we look forward to bringing Vatadustat for the treatment of anemia due to CKD to patients globally upon approval. I'll now turn the call over to Dave, who will review our financial results. Dave?