CymaBay Therapeutics Inc.

Good day, ladies and gentlemen, and welcome to SEMA Bay's third quarter 2020 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 the call for your questions. Please be advised that the call will be recorded at the company's request. It is also being webcast live on the investor section at the SEMA Bay website at www.semabay.com. Now I'd like to turn the call over to Mr. Dan Mendel, Vice President of Finance at FEMA Bay. Mr. Mendel, please proceed.

Thank you, Operator, and good afternoon, everyone. I hope that you've had a chance to review the press release we issued announcing our third quarter 2020 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 and plans on the development program for Stella Delpar before I provide a brief summary of our financials. Following our prepared remarks, we will open up the call 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, and cash runway, 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 can 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 statement 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 statement. 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.

Good afternoon. And thank you for joining us. We will keep today's prepared remarks brief with a focus on two key highlights from the most recent quarter. First, the significant progress made to reinitiate our late stage development program of StellaDelPAR for patients with primary biliary cholangitis or PBC. And second, our continued efforts to effectively manage our cash responsibly with an emphasis on ensuring we are able to deliver on our core operating plan. The third quarter of 2020 got off to a quick start with the mid-July FDA lifting of the clinical holds on the Celadelfar program after the agency's review and acceptance of a thorough safety review by a panel of independent, world-renowned liver experts. Just a week later, we announced positive top line results from our enhanced phase three study of Celadalpar in PBC. Despite having halted enhanced prior to patients completing the full 52 week treatment period, data for 167 patients who completed at least 12 weeks of treatment demonstrated that Celadalpar 10 milligrams provided patients with a statistically significant benefit on the primary and two key secondary endpoints in the trial. The results for the 55 patients on Celadal PAR10 milligrams who were evaluated at week 12 revealed a nearly 80% response on our primary composite endpoint, an accepted regulatory approval endpoint when measured at 52 weeks, versus 12.5% for the 56 patients on placebo. almost 30% response on alkaline phosphatase normalization versus zero on placebo, and a meaningful and significant placebo-controlled effect on reducing puritis, a key clinical symptom of PBC, in patients with moderate to severe puritis at baseline measured by the numerical rating scale, or NRF. In addition, Celadilpar appeared to be safe and well-tolerated in this study. We have been studying Celadelpar in PBC since 2015, having completed four clinical trials with over 300 patients dosed with Celadelpar and a subset of these treated for two years or longer. We believe a profile has emerged from our program in which Celadelpar at 10 milligrams is an optimal dose having anticholestatic, anti-inflammatory, and antipyretic activities and good overall safety to date. This pattern of effect suggests the potential of Celadalpar to provide patients with improvements in biochemical markers of disease severity and progression and to reduce symptom burden. Our ultimate goal is that these benefits translate into improved outcomes and quality of life for many patients with PBC. Although enhanced was halted early, Its results reinforce our near-term priority of restarting celadalpar development in PBC. Its results are invaluable because they allowed us to optimize the design of our next phase three study. They confirmed the endpoint, let us select the optimal dose, and gave us confidence to properly size the study so that it is overpowered on its key endpoints while still enrolling as quickly as possible. The study is named RESPONSE, and it is 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 who have an inadequate response to or intolerance to ursodeoxycholic acid in a two to one randomization to oral once daily Celadelpar 10 milligrams or placebo. The primary outcome measure is the composite responder rate after 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level below 1.67 times the upper limit of normal with at least a 15% decrease from baseline and has normal level of total bilirubin. Additional key outcomes of efficacy will compare the rate of normalization of alkaline phosphatase at 52 weeks and the change in puritis from baseline to six months for patients with a baseline NRS of four or greater or moderate to severe puritis. Puritis will be assessed using the same numerical rating scale and daily electronic diary as we used successfully in enhanced. Importantly, response will evaluate the same patient population, the same 10 milligram optimal dose of cell at LPAR, and the same primary and key secondary endpoint as evaluated in enhanced. The targeted size of the study balances our two most important objectives, including demonstrating efficacy on biochemical markers of disease and on reducing pruritus, along with supporting overall safety, with the goal of enrolling and completing the study in a timely fashion. As we have done and enhanced, we will also encourage patients to volunteer for a baseline biopsy in response. The key difference in response will be that these patients will also have the option of a 52-week biopsy instead of at three years, as had been planned for enhanced. The 52-week biopsy will provide required additional safety information to support registration. Based on our own dialogue with the FDA, the incorporation of paired liver biopsies in PBC Phase III studies is part of their new posture towards broader safety assessments in PBC and is unrelated to specific sponsor or compound issues. I am pleased by the progress our internal team has made in startup activities for response, and we will continue to offer refined guidance as we make further progress. We believe we will commence response in the first quarter of next year, but want to caution that the impact of the COVID pandemic may introduce unknown risks to study startup and enrollment. We'll continue to provide updates as we progress and develop experience in the study. In addition to response, we have also initiated study startup activities for Assure, an open-label, long-term study of Celadalpar in patients with PBC, intended to collect additional safety data to support registration. Like response, this study is expected to begin enrolling patients in Q1 2021. The study will first enroll patients who have participated in our prior studies of Celadalpar and PBC, including the open-label Phase II study, enhanced, and our prior long-term safety study. As patients complete response, and potentially other future PBC studies would sell at LPAR, they will also have the opportunity to enroll in Assure. For each of these studies, we also plan to incorporate necessary procedures to ensure patient safety in consideration of the current global pandemic involving COVID-19. In addition to providing valuable input into design considerations for response, The results from Enhance have formed the basis for raising awareness and excitement around the reinitiation of the CellDelPAR development program in PBC among patients and physicians. We believe the positive data from Enhance has the potential to drive interest from investigators and their patients in enrolling response. Earlier this week, we announced data from Enhance will be featured in an oral late-breaking presentation delivered by Professor Gideon Hirshfield from the University of Toronto at the liver meeting sponsored by the American Association for the Study of Liver Diseases on November 16th. We are looking forward to having a significant presence once again at this important meeting, which will also include a poster of distinction featuring data from our Phase II study of Celadalpar in patients with non-alcoholic steatohepatitis, or NASH, given by Dr. Stephen Harrison. As we have previously discussed, we believe these data support the potential for Celadalpar to offer meaningful anti-inflammatory and anti-fibrotic effects while improving metabolic aspects of the disease in a combination approach to treating patients with NASH. This poster presentation will offer us a platform to continue exploring opportunities to collaborate or partner with others that may have complementary mechanisms for NASH. While our focus remains on completing development of Celadelpar in PBC, we continue to evaluate Celadelpar and our other early stage clinical assets for other indications and development opportunities. Earlier today, we announced plans to conduct a study to evaluate the potential for MBX2982, our GPR119 agonist, to prevent hypoglycemia in patients with type 1 diabetes. Insulin-induced hypoglycemia in diabetics is a significant limiting factor in achieving the desired glucose control and is the cause of significant morbidities. In recent preclinical studies, GPR119 agonists were shown to enhance glucagon secretion in response to low glucose levels and were able to prevent hypoglycemia in animal models. The Phase IIa proof of pharmacology study will assess whether MBX2982 can enhance glucagon secretion during insulin-induced hypoglycemia in subjects with type 1 diabetes. While SEMA-BAY retains full rights to MBX-2982, the study will be led by the Advent Health Translational Research Institute in Orlando, Florida, and will be fully funded by the Helmsley Charitable Trust. We appreciate the opportunity to contribute to this effort to evaluate MBX-2982 for its potential to treat individuals at risk for insulin-induced hypoglycemia. one of the most challenging and potentially life-threatening complications of insulin therapy and diabetes. In addition to the significant momentum behind our efforts to restart and complete development of CELADELPAR for PBC, a key highlight of the quarter is the successful management of our overall costs for yet another quarter. On that note, I'll ask Dan to provide a brief summary of our key financial highlights from the third quarter. Dan?