Syndax Pharmaceuticals, Inc.

Good day and welcome to the Syndex first quarter 2022 earnings conference call. Today's call is being recorded. At this time, I would like to turn the call over to Megan Myers of Argot Partners. Please begin.

Thank you, Operator. Welcome and thank you to those of you joining us on the line and the webcast this afternoon for a review of Syndex's first quarter 2022 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 Michael Metzger, Chief Executive Officer, Dr. Briggs Morrison, President and Head of R&D, and Alex Milti, Chief Accounting Officer. Also joining us on the call today for the question and answer session is Dr. Peter O'Dutlick, Chief Scientific Officer, and Dr. Anjali Ganguly, Chief Business Officer. This call is being accompanied by a slide deck that has been posted on 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 statement made during this call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation and 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 in 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, May 9th, 2022 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 Michael Metzger, Chief Executive Officer of Syndax.

Thank you, Megan, and thank you to everyone joining us on today's call and webcast. On the heels of a transformational 2021, we're off to a strong start for 2022, and I look forward to sharing with you today our recent progress and several key developments. I'm especially excited to report that Syndax is now on pace to deliver two U.S. applications for potential drug approvals in 2023, a distant and aspirational goal for most biotech companies, and yet a near-term and highly achievable one for Syndax. Despite the challenging market backdrop, we currently find ourselves with a solid balance sheet and the support of a strong pharmaceutical partner to advance the development of one of our lead molecules. We are well positioned to expand both of our lead assets into new indications and to build out a commercial organization to support the potential launch of these first and potentially best-in-class products. We also have the flexibility to selectively pursue business development opportunities to augment our pipeline with additional promising molecules to complement the ones we have. The momentum is building at Syntax, and it is indeed a very exciting time for the company. Now, turning to slide three, We provide a high-level summary of our current corporate priorities as we strive to realize a future in which people with cancers live longer and better than ever before. Starting with Revumenib, our official generic name for SMDX5613, our highly selective menin inhibitor. Enrollment in our three pivotal phase two trials in patients with relapsed refractory MPM1 or MLR acute leukemia which we call Augment 101, 2A, 2B, and 2C, remains on track. We're also pleased to announce that we have enrolled our first patients in the BEAT AML and Augment 102 trials, which represent important expansion opportunities for the franchise in combination with other approved agents and begin to move us into newly diagnosed patients. Beyond acute leukemia, we will be initiating a proof-of-concept trial later this year in colorectal cancer to our knowledge, represents the first assessment of a menin inhibitor in patients with solid tumors. This is an exciting development, and Briggs will discuss this effort further in a few minutes. Moving to axotilumab, or antibody against CSF1R. Enrollment is ongoing in our pivotal Agave 201 trial in patients with chronic graft-versus-host disease, with top-line data expected in 2023. In addition, we are excited to announce today that we recently received FAST-TRACK designation for axotilumab for the treatment of CGBHD. We are currently making progress, working closely with our partner, Insight, to maximize the value of this important program and expect to begin trials to move axotilumab into earlier lines of CGBHD later this year. And beyond graft-versus-host disease, we remain on track to commence a Phase II proof-of-concept trial of FAST-TRACK axotilamab, and idiopathic pulmonary fibrosis, or IPF, in the fourth quarter of this year. Again, I want to emphasize that we now have two registrational programs ongoing for two first-in-class and potentially best-in-class medicines in two areas of unmet medical need. With filings for both programs expected in 2023, we are starting to expand the organization to support the potential commercial launch of both molecules in the U.S., Later this year, we expect to hire the company's first chief commercial officer to lead the additional build-out of our commercial organization. Let's now turn to slide four and provide further details on where we are with REVUMENIT. First, we are conducting three single-arm phase two trials that FDA has agreed may each serve as a pivotal trial. Each of these single-arm phase two trials represent an independent path to a separate indication. The Augment 101-2A trial is enrolling patients with relapsed refractory MLLR-ALL, 2B is enrolling patients with relapsed refractory MLLR-AML, and 2C is enrolling patients with relapsed refractory MPM-1-AML. Each trial is open to patients aged one month or older, and each trial will enroll independent of the other two. We may seek initial regulatory approval for RevuMeta based on the results of any one of these trials should one trial enroll faster than the others, or we may seek initial regulatory approval with any two or three, depending on when they complete enrollment. We are happy to report that additional site initiation and patient accrual across the individual trials is going well, and we are optimistic that we will be able to complete enrollment in at least one of these trials this year. Given our current trajectory and our view of the competitive landscape, we anticipate being the first company to achieve regulatory approval for amended inhibitor. Needless to say, we believe being first to market is important and accretive to the long-term value of Syndax. We have agreement with FDA that for each trial, the primary endpoint will be the percentage of patients achieving CR-CRH, with secondary endpoints including durability of CR-CRH response, transfusion independence, overall survival, and safety. Importantly, the trial design allows patients to be treated with Revumentib after bone marrow transplant a design feature that allows us to start to understand the role of Revumentib in the post-transplant maintenance setting. We also have agreement with FDA on the statistical design of each trial. Each trial will enroll 64 adult patients and up to 10 pediatric patients. Beyond the augment pivotal program in relapsed refractory disease, slide five highlights some of the additional opportunities we are exploring with Revumentib, all of which build on the excellent safety and efficacy profile we have seen thus far with Revumentib. As I mentioned at the start of today's remarks, we are excited to share that enrollment is now underway in both the BEAT AML and Augment 102 trials. The Phase 1 BEAT AML Umbrella Trial is a collaboration with Leukemia and Lymphoma Society. As part of the collaboration, Revumeneb, the first MEN inhibitor to be included in the trial, will be combined with venetoclax and the azacitidine in diagnosed, newly diagnosed AML patients who are unfit for induction chemotherapy. This trial will assess safety as well as initial efficacy with top-line data expected next year. We expect a Phase 2-3 trial, which could serve as the basis for a regulatory filing, to follow soon after completion of the Phase 1 trial. The middle panel of the slide highlights our Augment 102 trial, which is designed to assess Revumenev in combination with standard salvage chemotherapies used for pediatric patients with ALL or AML. I am pleased to report that patients have begun accruing in this trial as well. Both the Augment 102 and Beat AML trials are supported by preclinical data, which demonstrate the potential benefit of a Mennon inhibitor used in combination regimens in these settings, and we are excited to see how these data translate in the clinic. The third panel on the right illustrates the Intercept trial, which is designed to explore the activity of Revimentib in patients with AML who have MRD-positive disease. This trial is being conducted as part of the Intercept Master Clinical Trial being led by the Australian Leukemia and Lymphoma Group, and we expect patients to begin enrolling into that trial later this quarter. The Intercept Trial is focused on investigating novel therapies to target early relapse and clonal evolution as a preemptive therapy in AML. The trial will enroll patients with measurable residual disease progression following initial treatment, a group of patients at very high risk of relapse, that represents an important unmet medical need. A general tenet in cancer treatment is that the earlier you treat the patient's disease, the better patients do and the longer the patients stay on medicine. The Intercept trial is a very creative approach to treating patients early in their disease course. I'll also note that Revumena is the first menin inhibitor to be included in the Intercept AML Master Clinical Trial. We believe the selection of Revumeneb for inclusion in two master clinical trial protocols highlights the enthusiasm that investigators have shown for treating patients with Revumeneb. As we have previously communicated, we are committed to unlocking the full potential of Revumeneb beyond the relapsed refractory acute leukemia setting. Slide six highlights our goals as a company to be the first to market in the relapsed refractory disease setting. Beyond that, we intend to pursue approval in additional value-enhancing indications in patients with MLR and MPM1 mutant acute leukemias, including the newly diagnosed and maintenance settings. As we continue to develop Revumentive for use beyond treatment of relapsed refractory patients and into the first-line treatment setting, we see the opportunity to treat up to 12,500 patients identified each year with these two forms of mutant acute leukemia. NPM1 and MLR represent up to 40% of the overall AML population, which, to our knowledge, will be the largest subpopulation of AML to be addressed by a new targeted therapy. Our goal is to engage patients early in their treatment journey, get them into remission, and maintain them in that state for months, if not years. That emerging potential for Revumentum is why we believe it could be one of the most important new franchises and largest market opportunities in hematology and certainly worthy of significant investment. As I mentioned earlier, we are also preparing to start an initial proof-of-concept trial in solid tumors, yet another exciting new area of exploration for Revumenta. I will now turn the call over to Briggs, who will walk through the biologic rationale, development strategy, and market opportunity for Revumenta in colorectal cancer. Briggs?