Akebia Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to Akibia Therapeutics Innovate Topline Data Readout Call and its first quarter financial results and business highlights conference call. As a reminder, this call is being recorded. I would now like to introduce your host for today's conference, Kristen Shepherd.

Thank you, and good morning. My name is Kristen Shepherd, Senior Vice President of Investor Relations at Akibia. Thank you so much for joining us to discuss Akibia's top-line data from Innovate, our global phase three program of our product candidate Vatadustat for the treatment of anemia due to chronic kidney disease in adult dialysis-dependent patients. On today's call, we will also discuss Akibia's first quarter 2020 financial results and other recent business highlights. We issued two press releases this morning, one containing Innovate's top-line data Followed by one containing our financial results and other recent business highlights. Both of these exciting press releases are available on our investor relations website along with the slides for today's call. For your convenience, an audio replay of today's call with the slides will also be available on our website shortly after we conclude today's webcast. Joining our call today are John Butler, President and Chief Executive Officer Dr. Steven Burke, Chief Medical Officer, and Jason Amelo, Chief Financial Officer. Before we begin, I'd like to remind everyone that this conference call includes four looking statements. Each four 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 risk factors and management's discussion and analysis sections of our most recent quarterly and annual reports filed with the SEC and in the cautionary note on forward-looking statements in the slides. The forward-looking statements on this call speak only as of the original date of this call, and we do not undertake any obligation to update or revise any of these statements. With that, I'd like to turn the call over to our CEO, John Butler. John?

Thank you, Kristen, and welcome to everyone. We are thrilled to be able to announce positive top-line data from Innovate, the first of our two global Phase III programs studying Vatadustat, our investigational oral hypoxia-inducible factor pro-hydroxylase inhibitor, or HIF-PHI, to treat the anemia of chronic kidney disease. It is extremely rewarding. to see this program yield clear, consistent, and compelling positive results. This is a very important day for Akibia, but it was not accomplished by the Akibia team alone. I want to sincerely thank our investigators and their staff for participating. But most importantly, I want to thank the nearly 4,000 patients who participated in this program. Dialysis patients are among the most at risk and hardest hit by COVID-19. During these uncertain times, we're reminded of both the critical nature of our work and the significant need to advance care for these patients. I'd also like to thank our collaboration partner, Otsuka, our partner in dialysis before pharma, and our shareholders for supporting Akibia and our efforts thus far. The Akibia team did an extraordinary job closing the studies, collaborating and analyzing the despite the logistical challenges of operating under the present COVID environment, bringing Innovate to a timely and successful conclusion. I'm extremely proud of all of our employees. We believe our collective efforts bring us one step closer to fulfilling our purpose to better the lives of each person impacted by kidney disease. Again, my sincere thanks to everyone. We wouldn't be here today without all of you. In terms of an agenda for today's call, we're going to start with looking at how COVID-19 is impacting our business and how we are working to mitigate potential impacts. We issued our initial response to COVID on March 15th, and today I'll provide some updates. The rest of our discussion will be focused on the positive top-line data that we announced this morning and the exciting pathway that we've developed for Veta-Dustat. We have a lot of exciting data to cover, and so in the interest of time, Jason will not review the Q1 results, but is available for any questions you might have. As always, following our discussions, we'll open the call for Q&A. In this challenging environment, our purpose to better the lives of each person impacted by kidney disease is more relevant and important than ever. Our innovative therapies are critical to dialysis and non-dialysis CKD patients, who are among the most at risk. Continuing to provide and support our critical therapies is a priority, as is protecting the health and safety of our patients, customers, and employees. To that end, we're doing all we can to limit the spread of the virus, as well as support kidney disease patients and our community. Our team has been incredibly productive under our work from home policy. While ensuring business continuity, Our team has continued to support local and national response efforts to the COVID-19 pandemic by donating supplies and meals to frontline healthcare workers in Boston, volunteering to provide medical care at clinics treating COVID patients, and donating to the American Kidney Fund Coronavirus Emergency Fund to support the needs of kidney patients. In terms of our financial and operational performance, to date we have not experienced any significant adverse impact from COVID-19 and our fundamentals have remained strong. As of the end of Q1, our cash runway extends well into 2021. With that said, these are unprecedented times. And due to COVID-19, we have no clear visibility on what to expect for the upcoming weeks and months. We are closely monitoring demand for our marketed therapy. And we're also mindful of the potential macro risks from the impact on the healthcare system and the potential impact on payer mix. Given this uncertainty, we continue to take precautionary measures and actively monitor this evolving situation. Our focus on our clinical programs and ongoing studies is as important as ever. Our PROTECT studies for Vatadustat have advanced significantly, and we continue to expect top-line data mid-year as planned. Also, our Forward 2 trial for Vatadustat is fully enrolled, and we expect top-line data by year-end as planned. COVID precautions are, however, causing a delay in enrolling new clinical trials. For ongoing trials, we're using remote monitoring and performing remote patient visits where possible. Our commercial and customer-facing teams are leveraging tools and technology to interact virtually with healthcare providers, including dialysis centers, and responding to their needs. We believe Akebia can play an important role in supporting our customers and our patients during this crisis, and we're here to help enable continuity of care with important programs like Akibia Cares. We're fortunate that even before the COVID pandemic, we had already begun implementing new business continuity plans across our supply chain with the goal of safeguarding our ability to provide our therapies to patients who rely on them and driving operating efficiencies across our business. These measures look to ensure that critical materials are not sourced from any single supplier for either Orixia or Vatadustat. At this time, our supply chain is functioning, our manufacturers are operating, and while we don't currently anticipate a supply disruption, we believe that we have inventory to help mitigate the impact should one occur. In sum, this uncertain COVID environment has presented new risks to our business. While we're working hard to mitigate any potential impact, were mindful that many of these risks and the impact to the larger healthcare market are outside our control. The bright spot is that our team is more committed than ever to deliver on our purpose, and we believe we have tremendous value-enhancing opportunities ahead. And that's a great segue to our Innovate data. Again, we are very excited with the clear, consistent, and compelling efficacy and safety, particularly MACE data, that we announced today. Vatadustat succeeded in meeting the primary and key secondary efficacy endpoint in each of the two Innovate Phase III studies, confirming that once daily, oral therapy with Vatadustat can increase and maintain hemoglobin in the target range similar to the current standard of care, in this case, ARIN-S for darbacoitin alpha. Vatadustat also achieved the primary safety endpoint of the Innovate program. defined as non-inferiority in time to first occurrence of major adverse cardiovascular events, or MACE, which is a composite of all-cause mortality, non-fatal myocardial infarction, or non-fatal stroke, confirming Vatadustat safety and demonstrating no increased cardiovascular risk of Vatadustat compared with the standard of care in adult dialysis patients with anemia due to CKD. These results were clear and remarkably consistent across all patient populations in dialysis with anemia due to CKD, whether the patient was new to dialysis, an incident patient, or had been on for many years, a prevalent patient. Importantly, all of these analyses were based on a statistical analysis plan and non-inferiority margins agreed to with regulators. Today, there are approximately 500,000 dialysis patients in the U.S., In general, this is a population with significant health issues. Approximately 90% of dialysis patients are being treated with erythropoiesis stimulating agents, or ESAs, to manage their anemia. The impact of this disease on patients with CKD is profound. In addition to the well-known symptoms of fatigue, dizziness, and shortness of breath, anemia has been associated with more severe adverse outcomes, such as cardiovascular complications, including left ventricular hypertrophy and congestive heart failure. Multiple large peer reviewed studies have demonstrated the increased cardiovascular risk associated with ESA use in both dialysis and non-dialysis patients. Physicians associate that risk with the EPO levels achieved by the doses of ESA, the speed of hemoglobin increase, and excursions of hemoglobin above 12 grams per deciliter. InnovateData further support what our Vatadustat development program has consistently demonstrated and what research suggests nephrologists want in a new treatment option for their patients. Vatadustat treatment was designed with the goal of maintaining physiologic EPO levels, increasing hemoglobin in a predictable manner, minimizing hemoglobin excursions, and providing a convenient oral dose. We believe our data uniquely positions Vatadustat as a potential new oral standard of care for treating all populations of dialysis patients, including both incident and prevalent dialysis patients with anemia due to CKD subject to approval. The headline for our Global Phase III Innovate program continues to be that we believe it is designed for clinical, regulatory, and commercial success, and the results continue to support that belief. Innovate consists of two non-inferiority studies designed to evaluate the efficacy and cardiovascular safety during long-term treatment of anemia without a DUSAT using an active control, darbatholatin, and injectable ESA, which is the current standard of care. Simply put, we believe that in order to change the standard of care, you need to compare to the standard of care. We thoughtfully constructed this program and our trial design after extensive dialogue with the FDA and European regulators. We have a straightforward statistical analysis plan, prospectively defined and agreed to non-inferiority margins with the FDA and EMA, and we also agreed with the FDA on the key components of our statistical analysis plan. Our innovative trial design has enabled a straightforward collection and analysis of MACE across our program and yielded a clear and compelling data readout on both efficacy and safety. We can't wait to share these data with regulators and ultimately with physicians, dialysis providers, and payers. We are more confident than ever that the clinical success we've demonstrated with Innovate supports Vatavustat's potential for success on the regulatory front. We are also confident that the Innovate data will be highly informative for physicians, patients, dialysis providers, and payers as they make important decisions about patient care once Vatavustat is approved. I know we're all excited to get into the specifics, so let me hand the call over to our Chief Medical Officer, Dr. Steven Burke. Steve?