CymaBay Therapeutics Inc.

Good day, ladies and gentlemen, and welcome to SEMA Bay first quarter 2021 financial results and business update conference call. At this time, all participants are in a listen-only mode. Following the formal remarks, we will open up the call for questions. Please be advised that this call will be recorded at the company's request. It is also being webcast live on the investor section of SEMA Bay's website, at www.semabay.com. Now I would like to turn the call over to Mr. Dan Menel, Vice President of Financial at SEMA Bay. Mr. Menel, you may begin.

Thank you, Operator, and good afternoon, everyone. I hope that you've had a chance to review the press release we issued announcing our first quarter 2021 financial results and business updates. You can access that release on our website under the Investors tab. Joining me on the call today are Sujal Shah, Chief Executive Officer, Dr. Chuck McWhirter, Chief Scientific Officer, and Clara Dickinson, Chief Regulatory Officer. Sujal will provide an update on recent progress in the build-out of our leadership team to deliver on our value creation strategy. Chuck will discuss an update on our pipeline diversification, and I will provide a brief summary of our financials. Following our prepared remarks, we'll be available for Q&A. Before we begin, I'd like to remind everyone the statements made during this conference call, including the Q&A session relating to CIMA Bay's expected future performance, business prospects, events or plans, including clinical plans, regulatory approvals, and anticipated timelines and data release dates, cash runway, and planning for commercialization of any future products are forward-looking statements as defined under the Private Securities Litigation Reform Act of 1995. Although the company believes that the expectations reflected in such forward-looking statements are based upon reasonable assumptions, actual outcomes and results are subject to risks and uncertainties and could differ materially from those forecast due to the impact of many factors. The company assumes no obligation to update or supplement any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law. Participants are directed to the cautionary statements set forth in today's press release, as well as the risk factors set forth in SEMA Bay's quarterly and annual reports filed with the SEC for factors that could cause actual results to differ materially from those anticipated in the forward-looking statements. This conference call is the property of SEMA Bay, and any recording or rebroadcast is expressly prohibited without the written consent of SEMA Bay. At this time, I'd like to turn the call over to Sujal.

Thank you, Dan. Good afternoon and thank you for joining us today. Less than two months ago on our last call, we highlighted the initiation of response. Our global phase three registration study of Stella del Par for patients with primary biliary cholangitis or PVC. And the expansion of our clinical pipeline with MBX 2982 and CB 0406. This past Monday, And then again earlier today, we announced further significant progress with the strengthening of our executive team with the additions of Louis Stewart as Chief Commercial Officer and Dr. Dennis Kim as Chief Medical Officer. These seasoned biotech executives come with considerable talent and energy to apply their leadership and experience as we move into a period in which we plan to complete development and regulatory submissions for CELADELPAR, begin pre-commercial preparations, and continue efforts to diversify our research and development. I am extremely pleased to have Dennis Kim joining CIMA Bay as our CMO. Dennis is a physician scientist trained in endocrinology and with significant clinical development and executive experience in an emerging biotech environment that he acquired during substantial tenures at Amelin, Orexigen, and Zapgen. Dennis will join the executive team reporting to me and will lead all clinical functions, including development, clinical operations, biometrics, and medical affairs. His track record fits precisely FEMA Bay's need to have him lead a highly motivated and productive clinical function that is integrated with research, regulatory, manufacturing, business development, and commercial efforts. I'm confident that many of you will learn that Dennis also brings an acumen well-suited to articulate the science, medicine, plans, and opportunities of our programs to broad audiences, including medical experts, investigators, patient groups, investors, and analysts. I am equally delighted to welcome Louis Stewart as our Chief Commercial Officer. Just a little over two years before CELADELPAR's potential launch, this timing is ideal as we add a recognized commercial leader to implement a thoughtful and efficient build of both a strategy and an organization. Louis comes with deep experience in commercial leadership and product launches in the emerging biotech setting. We were deeply impressed by his knowledge and experience in a variety of situations, including at CV Therapeutics and most recently at MyoVan, both of which required the self-reliant approach of standing up a product launch, but also post-launch, they ultimately involved integrating marketing and sales efforts into much larger entities. Drawing upon this exposure to a broad set of practices, Louis will lead all aspects of the commercial function, including marketing and sales. He will join the senior team reporting to me and will be intimately involved in strategic planning across many functions. There will be many opportunities to get to know Louis as one of our spokespersons as he moves our commercial plans forward. I expect many will gain an appreciation for the energy and enthusiasm he brings that is grounded in his aptitude for acquiring knowledge and using critical thinking. The announcement of these critical C-level hires serves as a capstone to the continued progress we made as discussed in March with the initiation of two studies in patients with PBC, the Phase III Response Study and the Long-Term Safety Assure Study. Our remarks today will be brief, mainly reiterating our guidance on timing for these studies while providing some additional color on study population. We will also describe progress being made on MBX 2982 and CBO 406, our other pipeline projects, for which we expect to be able to provide further updates in the second half of 2021 as we seek to bring additional novel treatment alternatives to patients. We will conclude with key corporate and financial updates. Completing the development of CeladalPAR for patients with PBC remains our top priority. In the first quarter, we initiated response, a 52-week placebo-controlled randomized global Phase III registration study evaluating the safety and efficacy of CeladalPAR in patients with PBC. Response is intended to enroll 180 patients in a two-to-one randomization to oral once-daily Feladilpar 10 milligrams or placebo as an add-on therapy to patients with an inadequate response or intolerance to first-line ursodeoxycholic acid therapy. The primary outcome measure is the composite biochemical responder rate after 52 weeks for alkaline phosphatase and bilirubin, the same endpoint used to register Ocalibum. the only approved second-line treatment alternative for PBC patients. We expect our strategy for differentiation to potentially be significantly strengthened by two key secondary endpoints, the rate of normalization of alkaline phosphatase at 52 weeks and the change in puritis from baseline to six months in patients with moderate to severe puritis as reflected in a baseline puritis numerical rating scale value of four or greater. As many of you will know, the design for response and confidence in the selection of endpoints is significant given the shared population, endpoints, and the safety and efficacy demonstrated by the Celadal PAR 10 milligram dose in the completed Phase III study, Enhance, which was stopped early. The results for Enhance were presented in a late-breaking presentation at ASLD last November. Celadelpar 10 milligrams achieved a high degree of statistical significance for all three of the same endpoints being used in response after only 12 weeks of treatment. Our approach in the development program for Celadelpar is to collect data necessary to characterize the benefit-risk of Celadelpar across the broadest population of patients with PBC. We have been asked recently about patients with PBC and their underlying stage of disease as it relates to cirrhosis. Broad categories of increasing severity of fibrosis can be described as non-cirrhotic, compensated cirrhotic, and decompensated cirrhotic. Within compensated cirrhotic are sub-stages of patients with and without clinically significant portal hypertension. Clinically significant portal hypertension develops as the fibrotic liver becomes increasingly stiff and is thought to often be a precursor to decompensating liver-related events. Measuring portal hypertension is an invasive burdensome procedure, and so a clinical signature for clinically significant portal hypertension can come from a number of less invasive criteria as recommended in published practice guidelines. Across two significant studies with Celadelpar enrolling more than 360 patients with PBC, there were over 50 patients with compensated cirrhosis, some of which were treated for two years or more. And clinical data from some of these have been highlighted in published abstracts and presentations given at past medical meetings. Due to the identical eligibility criteria and overlap with clinical sites, we expect a majority of patients that enroll in response to also be non-serotic, but would nonetheless expect somewhere around 15 to 20% to be compensated serotics. Patients in this population would eventually be in the target population, and so it is important that they be studied for safety and efficacy. In this quarter, we continue to make headway with our clinical development program for Celadalpar, including activities for other NDA-enabling drug-drug interaction studies and special population studies for patients with renal impairment and PBC patients with hepatic impairment. Results from the hepatic impairment study in particular will provide valuable insights into the exposure and potentially the safety and efficacy of Celadalpar in PBC patients with various degrees of cirrhosis. In the first quarter, we also initiated Assure, an open-label long-term study of CeladalPAR in patients with PBC in order to collect additional safety data to support registration. We expect CeladalPAR to have one of the most robust safety databases in PBC patients ever submitted for an NDA. Our most important focus remains to accelerate site activations, screenings, and randomizations in response over the coming quarters. Our efforts in response with 80 fewer patients are nonetheless on the same scale globally as they were for enhanced. This is being done in an effort to mitigate challenges posed by the pandemic and greater competition for patients. It remains our goal to enroll the study by the end of the year, and we will look to provide additional updates on timelines later this year. Now, let me turn the call over to our Chief Scientific Officer, Dr. Chuck McWherter, to cover updates on our early clinical stage MBX2982 and CB0406 programs. Chuck?