Syndax Pharmaceuticals, Inc.

Good day, everyone, and welcome to the Syndax fourth quarter 2022 conference call. Today's call is being recorded. At this time, I would like to turn the call over to Sharon Clary, head of investment relations at Syndax Pharmaceuticals.

Thank you, Operator. Welcome, and thank you all for joining us today for a review of Syndax's fourth quarter and full year 2022 financial and operating results. I'm Sharon Clary, and with me this afternoon to provide an update on the company's progress and to discuss financial results are Michael Metzger, Chief Executive Officer, Dr. Briggs-Morrison, President and Head of R&D, and Keith Goldan, Chief Financial Officer. Also joining us on the call today for question and answer session are Dr. Vitora Dentler, Chief Scientific Officer, and Dr. Anjali Kangula, Chief Business Officer. This call is accompanied by a slide deck that has been posted on the investor page of the company's website. You can now turn to our forward-looking statement on slide two. Before we begin, I'd 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 factors section in the company's most recent annual report on Form 1010. as well as other reports filed with the SEC. Any forward-looking statements may represent our views as of today, February 28, 2023 only. A replay of this call will be available on the company's website, www.cindex.com, following its completion. With that, I'm pleased to turn the call over to Michael Metzger, Chief Executive Officer, CINDEX.

Thank you, Sharon, and thank you to all of you joining on the webcast today. Now, turning to slide three, 2022 was a year of growth and strong execution for Syndex. We made great progress working towards our long-term goals, all of which put us firmly on the path to becoming a fully integrated commercial-stage biopharmaceutical company, delivering two best-in-class products to patients in areas of high unmet medical need. On Revumentive, we made significant progress in enrolling patients in the Augment 101 pivotal trials, announced breakthrough therapy designation for patients with relapsed refractory KMT2A rearranged acute leukemia, and presented robust updated phase one data that further supported Revimenid's best-in-class clinical profile in two oral sessions at the 2022 American Society of Hematology Annual Meeting. Further, we initiated additional phase one trials designed to test Revimenid in a variety of settings, including combinations with standard of care regimen. For axotilumab, we completed enrollment in the Pivotal Agave 201 trial, published the initial Phase 1-2 chronic graft-versus-host disease dataset in the Journal of Clinical Oncology, and made progress expanding the axotilumab development program in collaboration with our partner, Insight. We are fortunate to have Insight as a strong collaborator to help us successfully advance axotilumab development. Additionally, in December, we bolstered our balance sheet with a 172 $5 million follow-on offering. Our cash runway is now expected to extend into the second half of 2025, allowing us to appropriately invest to maximize the value of our pipeline, prepare for two potential U.S. commercial launches in 2024, and pursue potential business development opportunities. Our focus, determination, and unending commitment to patients has propelled us forward to this critical year. A year that we expect will be marked by pivotal readouts and registration filings for our two lead drug candidates, both of which are first and potentially best-in-class treatments. 2023 is an incredibly significant year for Syntax, and I'm confident that we have the expertise and resources to execute on our goals, along with the determination to realize a future in which people with cancer live longer and better than ever before. Turning to slide four, we provide a high-level summary of our current corporate priorities. Starting with Revimentum, our highly selected menin inhibitor. Our pivotal Phase II Augment 101 trial evaluating Revimentum in patients with relapsed refractory NPM1 mutant or KMT2A rearranged acute leukemia is progressing well. We are happy to report that we have completed enrollment of a sufficient number of patients with a KMT2A rearrangement to support a registration filing, and we are on track to report top-line data from the TRIAL IN THE THIRD QUARTER OF 2023. THIS DATA WOULD PROVIDE THE BASIS FOR AN NDA FILING BY YEAR END 2023. I WILL PROVIDE MORE DETAILS ON AUGMENT 101 LATER IN THE CALL. IN ADDITION TO THE AUGMENT 101 TRIAL, WE HAVE ONGOING TRIALS TESTING REVUMENTIV IN EARLIER LINES OF THERAPY AND IN COMBINATION WITH STANDARD OF CARE MEDICINES TO TREAT THESE FORMS OF ACUTE LEUKEMIA. WE ARE COMMITTED TO UNLOCKING THE FULL POTENTIAL OF THIS IMPORTANT THERAPY. With that goal in mind, we expect to have several additional data readouts in 2023 for Revumented. These include initial top-line data from Augment 102 chemocombination trial in relapsed refractory acute leukemia patients, safety data from the dose escalation phase of the BEAT-AML cooperative trial of Revumented in combination with venetoclax and astatidine in newly diagnosed AML patients, and initial top-line data from the first solid tumor proof-of-concept trial in metastatic colorectal cancer. Moving to axotilumab, our antibody against CSF1R. We remain on track to report top-line data in mid-2023 from the pivotal Phase II Agave201 trial, evaluating axotilumab in patients with chronic graft-versus-host disease, or CGBHD, and plan to submit a BLA filing by the end of 2023. To maximize the value of the axotilumab program, we anticipate initiating additional trials in 2023, including a combination of axotilumab and ruxolitinib in frontline CGBHD that we expect insight to be in later this year, as well as a phase two trial of axotilumab in idiopathic pulmonary fibrosis, or IPF, that we anticipate kicking off in the first half of 2023. We continue to evaluate potential opportunities to in-license earlier-stage targeted oncology compounds that we believe could become high-value differentiated assets. We believe that we have sufficient capital to bring in new early-stage assets, but our bar for doing so is quite high, as any compound would need to complement our current pipeline and fit in with our long-term corporate strategy. Let's now turn to slide five, and I'll provide further details on the recommended program. As you recall, the Phase II portion of Augment 101 is designed as three single-arm pivotal trials with distinct patient populations that enroll independently. These patient populations are KMT2A rearranged ALL, KMT2A rearranged AML, and MPM1 mutant AML. The primary endpoint of each pivotal trial is the percentage of patients achieving CR, CRH, with secondary endpoints including durability of CR-CRH response, transfusion independence, overall survival, and safety. In December, we received breakthrough therapy designation, or BTD, for remunerated for the treatment of all adult and pediatric patients with relapse or refractory KMT2A rearranged acute leukemia, whether presenting as acute myeloid or acute lymphoid phenotype. The receipt of BTD underscores the potential, if approved, for Revimenib to be the first drug to address the breadth of unmet medical needs in KMT2AR leukemia, accounting for up to 10% of all acute leukemias. Based on the broad breakthrough therapy designation and our ongoing conversations with regulators, we will pool the data generated from both the AML and ALL KMT2AR cohorts into a single NDA filing aimed at an indication to treat all relapsed refractory acute leukemia patients with a KMT2A rearrangement. Thanks to the enthusiastic support of our investigators, we have already enrolled a sufficient number of patients with KMT2A rearrangements in the phase two portion of the Augment 101 trial to enable this filing strategy. We expect to share top line data from this population in the third quarter and file an NDA for the treatment of relapsed refractory, KMT2AR, acute leukemia in adult and pediatric patients by the end of 2023. Separately, we continue to enroll relapsed refractory NPM1 mutant AML patients and expect to report completion of enrollment for these patients in the second half of 2023. These trials are enrolling well, and there was significant excitement for review by investigators at the ASH annual meeting in December, where we presented updated phase one data that Briggs will take you through on slide six. Briggs?