Syndax Pharmaceuticals, Inc.

Good day, everyone, and welcome to the CINDEX fourth quarter and full year 2020 earnings conference call. Today's call is being recorded. At this time, I would like to turn the call over to Ms. Megan Myers of Argo Partners.

Please begin. Welcome, and thank you to those of you joining us on the line and the webcast this afternoon for a review of CINDEX's fourth quarter 2020 financial and operating results. I'm Megan Myers with Argo Partners. And with me this afternoon to discuss the results and provide an update on the company's progress are Dr. Briggs Morickson, Chief Executive Officer, Daphne Kouridis, Chief Financial Officer. Also joining us on the call today for the question and answer session is Michael Metzger, President and COO, Dr. Michael Myers, Chief Medical Officer, and Dr. Peter Ordentlich, Chief Scientific Officer. This call is being accompanied by a slide deck that has been posted to the company's website, so I would ask you to please turn to our forward-looking statements on slide two. Before we begin, I would like to remind you that any statements made during this call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these statements, as a result of various important factors, including those discussed in the risk factor section on the company's most recent quarterly report on Form 10-Q, as well as other reports filed with the SEC. Any forward-looking statements represent our views as of today, March 8th, 2021 only. A replay of this call will be available on the company's website, www.syndax.com, following this call. With that, I'm pleased to turn the call over to Dr. Briggs Morrison, Chief Executive Officer of Syndex.

Thanks very much, Megan, and thank you to everyone for joining us on today's call and webcast. Slide three provides a high-level summary of our current corporate priorities as we strive to realize a future in which people with cancer live longer and better than ever before. 2020 was a transformational year for Syndex. Over the course of the year, we've made great progress in our two clinical stage programs, both of which are on track to be in registrational programs this year. Thanks to the support of our many committed investors, we ended the year well financed to vigorously pursue both of our existing programs and to aggressively look for additional opportunities to bring targeted oncology drugs into our pipeline. Let's now turn to slide four in S&DX 5613. are genetically targeted agent for the treatment of leukemia. As we've noted previously, there's extensive validation of both MLLR and NPM1 mutations as molecular targets in leukemia. And well-established diagnostic tests routinely identify patients with these genetic mutations. Premier publications provide the scientific rationale and preclinical validation of our ongoing clinical trial. and historic precedents support a rapid regulatory path for such targeted agents in acute leukemias. Slide 5 briefly summarizes the ongoing AUGMNT 101 trial, the first in human phase 1-2 trial in the accelerated understanding of MENIN or AUGMNT program. Consistent with what we communicated on our last call, we are on track to present the completed phase 1 portion of the trial in the near future. and to start the Phase II expansion cohorts soon thereafter. We have received several questions about the upcoming data disclosure, and so I just want to provide some guidance. Our intent is to present the completed Phase I trial. The primary objectives of Phase I are to determine the safety, tolerability, the maximum tolerated dose, and the recommended Phase II dose, of S and DX5613 in patients with relapsed or refractory acute leukemia. We anticipate addressing these primary objectives in our data presentation. We will also have characterized the PK parameters of 5613. We anticipate having data for at least 30 patients with relapsed refractory leukemia. There will be patients whose leukemia has an MLR rearrangement. There will be patients whose leukemia has an NPM1C mutation. and there will be patients whose leukemia has neither of these genetic lesions. Although clinical efficacy is not an objective of the Phase I trial, we anticipate summarizing the efficacy that has been observed. We anticipate that the data will be presented by the principal investigator of the Augment 101 trial, Dr. Eitan Stein from the Memorial Sloan Kettering Cancer Institute, an extremely well-respected international expert in the field of adult leukemias, who has extensive experience developing novel targeted agents. Dr. Stein's presentation will be similar in content to a typical presentation at a scientific congress, such as ASH, and the opportunities for Q&A. We are working with Dr. Stein to find a suitable date, but anticipate securing a date prior to the end of April. We have, of course, been busy preparing for the phase two portion of the trial, which will enroll three distinct expansion cohorts, each of which consists of a specific genetically defined relapsed or refractory acute leukemia, and which is on track to initiate over the next couple of months. The three cohorts will include patients with MLLR acute lymphoid leukemia, ALL, patients with MLLR acute myeloid leukemia, AML, in patients with NPM1 mutant AML. The phase two portion will further characterize the safety of 5613 and will provide a robust estimate of the complete response rate as the primary measure of therapeutic benefit. The phase two portion will enroll both pediatric and adult patients, thereby providing us a potential path to regulatory approval with a broad label including both adults and pediatrics. I should note that if the results are positive, this phase two portion of Augment 101 could potentially support a regulatory filing, giving existing regulatory precedents. Over the last period, we've also been conducting extensive research into the broad landscape of clinical opportunities for 5613 beyond the initial approval and relapse refractory acute leukemias, as illustrated on slide six. While we are not in a position today to lay out the specific next steps in building out the 5613 franchise, I can say that we are using this ongoing analysis to inform specific combination regimens that we anticipate starting to study later this year. Several investigators and leading companies have already reached out to us with novel, exciting ideas for future development of S&DX 5613, and we are evaluating those opportunities. Let me now turn to slide seven, and axotilamab, formerly known as SNDX6352, are potentially best-in-class monoclonal antibody therapy targeting the CSF1 receptor. As you know, we recently presented our phase one chronic graft-versus-host disease data at ASH and have initiated our pivotal trial for axotilamab in chronic graft-versus-host disease. This trial is the axotilamab for graft versus host disease trial called Agave 201, and it's outlined on this slide. The trial will enroll patients with chronic graft versus host disease whose disease has progressed after two prior therapies. Patients must be at least six years of age and have met overall entry criteria. This is a pivotal dose-ranging trial in which patients will be randomized to one of three treatment groups, each investigating a distinct dose of axotilamab given either every two weeks or every four weeks. The primary endpoint is overall response rate using the 2014 NIH consensus criteria for chronic graft-versus-host disease. Secondary endpoints will include duration of response and validated quality of life assessments using the Lee symptom scale. Enrollment to the study is underway. and we are on track to deliver top-line data in 2023. We believe that chronic graft-versus-host disease represents a high unmet medical need and an important commercial opportunity, with approximately 14,000 patients suffering from a chronic graft-versus-host disease in the U.S. today. With the recent positive pivotal results from both Insight's JAKA-Fi and Cadman's KD025, we may seem soon see regulatory approvals and commercial launches that will begin to delineate the commercial opportunity to chronic graft-versus-host disease. Despite recent advances in this area, to our knowledge, axotilumab is the only agent in clinical development that specifically targets the monocyte macrophage lineage. We believe the data generated state with axotilumab suggests it has the potential to play an important role in the treatment of chronic graft-versus-host disease, both as monotherapy and given its safety profile, potentially in combination with complementary medicines. We've also been working extensively with experts in the field of fibrotic diseases, and have found a strong consensus that the scientific rationale for the efficacy of axotilamab in chronic graft-versus-host disease firmly supports its potential in a wide variety of fibrotic diseases, such as idiopathic pulmonary fibrosis and scleroderma. We are actively evaluating options by which to build the Axotelamab franchise beyond chronic graft-versus-host disease and take advantage of what we believe are a significant set of opportunities that could material enhance shareholder value. Finally, slide eight summarizes the transactions that led to the acquisition of the Mennon MLR and Axotelamab programs. We believe that we will be able to continue to expand our pipeline through the acquisition or in-licensing of quality, differentiated assets. We believe that we have the necessary skills to evaluate and identify high-quality assets and the clinical development experience to bring these campaigns through valuable inflection points. We expect to remain among preferred partners for such transactions. I'll now turn the call over to Daphne to review our financial results. Daphne.