CymaBay Therapeutics Inc.

Good day, ladies and gentlemen, and welcome to Simba Bay's second quarter 2021 financial results and business update conference call. At this time, all participants are on 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 Simba Bay website at www.simbabay.com. Now I'd like to turn the call over to Mr. Dan Menold, Vice President of Finance at Simba Bay. 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 second 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 Dr. Dennis Kim, Chief Medical Officer. Following our prepared remarks, we will open up the call for Q&A. Before we begin, I'd like to remind everyone that 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, funding and repayment schedules, anticipated timelines and trial enrollment 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 forecasts 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 risk factors set forth in the company'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. One year ago, we successfully gained regulatory clearance to restart clinical development of Cellidelpar. We did this with a staff of approximately 20 employees in the midst of the global pandemic that continues even to this day. The second quarter saw us achieve significant milestones that have positioned us once again to attain our core objectives of delivering innovative treatments to patients in need while also providing value to our shareholders. We added experienced talent across the functional areas vital to getting CeladalPAR to patients with primary biliary cholangitis or PBC. This includes those executing all the necessary studies for global regulatory submissions including response, our global phase three study, and the many who are preparing us for regulatory filings, manufacturing products, and commercial launch. Paramount to our plans, we have now also secured the funding needed to complete Celladelpar's development through a non-dilutive risk-sharing development funding agreement with Abingworks. In this agreement, we have a partner with a long history of funding innovative and successful life sciences companies and who importantly shares our belief that Celadalpar has the potential to meaningfully improve the treatment of patients with PBC. Underscoring this potential are the results in our latest presentation featured at the International Liver Congress in June in those patients with cirrhosis. a more advanced stage of this rare life-threatening disease. On today's call, I will begin by summarizing key aspects of the development financing agreement with Abingworth and then ask Dennis to discuss the progress in response and our most recent presentations at the International Liver Congress sponsored by the European Association for the Study of Liver Disease. Chuck will provide a brief update on our early stage pipeline programs, followed by Dan's summary of key financial highlights in the second quarter and first six months of the year. We will have Q&A following our presentations. Our announcement of a non-dilutive risk sharing funding agreement with Abingworth highlights our commitment to find innovative means to support our mission of improved treatment alternatives to patients with chronic inflammatory liver diseases like PBC. Proceeds from this transaction are expected to support the completion of our development program for Celadelpar in PBC, including the ongoing Global Phase III Response Study and the long-term Open Label Assure Study up to the time of regulatory filing, and various NDA-enabling studies as well. By securing this capital and strengthening our balance sheet, we are well-positioned to deliver on the promising opportunity ahead of us to deliver value to patients and shareholders. Under the terms of this strategic development funding transaction, we will receive from Abingworth up to $100 million to fund the development of Celadal PAR for PBC, of which $75 million will be received in three installments over approximately the next six months. We have the option to receive an additional $25 million within approximately two months of the completion of enrollment of response. In exchange, we will make fixed payments equal to two times the funded amount spread over a six-year period following the first regulatory approval in either the U.S. or the EU. And in addition, sales milestone payments capped at 1.1, the funded amount, based on U.S. product sales. Specific payment schedules for the regulatory and sales milestones are outlined in the 8K filed with the SEC last week. We believe the terms including the repayment caps and back-end weighted repayment schedule, in this agreement are some of the most favorable found in recent precedent transactions of this kind. We also retain the ability to accelerate payment at a reduced amount upon regulatory approval. FEMA Bay retains upside potential for CELA Del Par in the U.S., along with full worldwide commercial rights. We examined a wide variety of funding vehicles and worked closely with outside financial and legal advisors in a competitive process before making a final selection. Commonly, risk sharing funding agreements in biotech are for programs that are commercial or at least have regulatory approval. We believe the level of interest from multiple parties reflects the significant efficacy and safety data generated to date for Celadelpar in PBC. Finding a financial partner in Abingworth with the background to conduct significant diligence and come to a shared view of the long-term potential for Celadelpar was key to consummating the deal. We are excited to have Abingward's support as they have deep scientific, operating, and financial experience in biotech. Moving on to our key business updates. Let me begin by reviewing response before turning the call over to Dennis to discuss key milestones achieved to date and our focus going forward. Completing the development of Celadal PAR for patients with PBC remains our top priority. As a reminder, response is a 52-week placebo-controlled randomized global phase 3 registration study evaluating the safety and efficacy of Celadal PAR in patients with PBC. The study is intended to enroll 180 patients in a two-to-one randomization to oral once-daily Celadelpar 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 Ocalibus. the only approved second-line treatment alternative for PVC. Two key secondary endpoints include 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 know, the design for response is based off our enhanced phase three study in which after only three months of dosing, Celadelpar 10 milligrams demonstrated meaningful, statistically significant improvements in biochemical markers of disease and reductions in symptom burden in patients with PBC. In addition to enrolling the same patient population, evaluating the same primary and key secondary endpoints, and studying the same optimal 10 milligram dose of Celadilpar as we did in Enhance. Our plan is to leverage our prior experience and success by taking response to over 20 countries and over 100 sites around the world. In 2019, we randomized 265 patients in Enhance, from first patient in to last patient enrolled in approximately 11 months. Screening in response, where we are intending, as mentioned, to enroll 180 patients, was initiated at the end of the first quarter this year, with our first patient randomization occurring in April. We set an aggressive goal for response And although our priority remains to complete enrollment in response as quickly as possible with a continued focus on year end, we were always aware that we would have to work through challenges presented by the global pandemic. As with any global study of this size in a rare disease such as PBC, enrollment begins slowly and accelerates as site activations and screenings begin to accelerate. Study startup activities including IRB and ethics committee approvals and site activations have been affected by the pandemic, even as effective vaccines have recently become available. Lately, the spread and evolving risks related to the Delta variant have added uncertainty and complexity to clinical research. As we assess the impact of these developments, We have to be prepared to keep sites active and enrolling patients into the first half of 2022. We will provide additional guidance around enrollment timelines on a quarterly basis as we progress and as we gain greater clarity into the impacts of the pandemic and the effectiveness of initiatives we are implementing to address these challenges. I'll now turn the call over to Dennis to review our progress to date and our strategies to complete our Phase III program. Dennis?