Kura Oncology, Inc.

Ladies and gentlemen, thank you for standing by and welcome to the Cura Oncology fourth quarter and full year 2020 earnings conference call. At this time, all participant lines are in a listen-only mode. After the speaker presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. Please be advised that today's conference is being recorded and if you require any further assistance, please press star 0. I would like to hand the conference to your speaker today, Pete DeSpain. Please go ahead, sir.

Great. Thank you, Victor. Good morning, and welcome to CURL Oncology's fourth quarter and full year 2020 conference call. Joining me on the call are Dr. Troy Wilson, our President and Chief Executive Officer, and Dr. Mark Grasso, our Chief Financial Officer and Chief Business Officer. Jim Bosta, our Chief Legal Officer, Dr. Steven Dale, our Chief Medical Officer, Kirsten Flowers, our Chief Commercial Officer, and Kathy Ford, our Chief Operating Officer, are also with us and available to answer questions. Before I turn the call over to Dr. Wilson, I would like to remind you that today's call will include forward-looking statements based on current expectations. Such statements represent management's judgment as of today and may involve risks and uncertainties that could cause actual results to differ materially from expected results. Please refer to Cura's filings with the SEC, which are available from the SEC or on the Cura Oncology website for information concerning risk factors that could affect the company. I'd also like to point your attention to our newly updated corporate presentation, which can be found in the Investors section of our website. With that, I'll now turn the call over to Dr. Troy Wilson, President and CEO of Cura Oncology. Troy?

Thank you, Pete, and thank you, everyone, for joining us this morning. We're a precision medicine company with two clinical stage oncology drug candidates for which we own global commercial rights. Our menin inhibitor, KO539, and our farnesyl transferase inhibitor, Tipifarnib. Each of our drug candidates targets oncogenic driver mutations in indications of high unmet need, and we're pursuing an accelerated development and fast-to-market strategy. Our pipeline also includes an emerging next-generation farnesyl transferase inhibitor program that we believe will target innovative biology to address indications of high unmet need through rational combinations. We're also in a stronger financial position than ever before with more than $600 million in cash, which we believe provides us with sufficient resources to advance our programs through multiple value inflection points. And we have a talented and proven team with the oncology drug development and commercialization expertise required to be successful. Now, let me take you through each of our programs, beginning with our menin inhibitor, KL539. In December, we reported preliminary clinical data from our Phase I-II Comet 001 clinical trial of KL539 at the American Society of Hematology annual meeting. These data were highlighted by single-agent activity in an all-comer population of patients with relapsed or refractory acute myeloid leukemia, including patients with NPM1 mutations, and KMT2A rearrangements. KO539 also demonstrated a favorable safety and tolerability profile with no drug discontinuations due to treatment-related adverse events and no evidence of QTC prolongation. Since the ASH presentation, we've continued to enroll patients in the phase one dose escalation portion of the trial as KO539 continues to demonstrate compelling clinical activity and a wide therapeutic window. we've completed the 600 milligram dose cohort and are currently evaluating an 800 milligram dose cohort. Meanwhile, we recently sought FDA feedback regarding the design of the registration-directed portion of our trial. In the context of those discussions, FDA guided that we should consider determining a minimum safe and biologically effective dose in our ongoing phase one trial. FDA also agreed we should modify our primary endpoint from CR-CRI to CR-CRH, and incorporate transfusion independence as a secondary endpoint in order to align the endpoints with those that would be meaningful for patients and acceptable for registrational intent. Based on this feedback, we plan to amend our COMET-001 trial protocol to include two Phase I expansion cohorts while we continue to evaluate KO539 in dose escalation. We expect these Phase I expansion cohorts to include a minimum of 12 patients each at doses that have already met the safety threshold to help us to determine a minimum safe and biologically effective dose. Furthermore, we plan to enrich each of these Phase I expansion cohorts with both NPM1 mutant and KMT2 rearranged relapsed or refractory AML patients. This should help us to further characterize the efficacy of KL539 in these target populations and better inform a recommended phase two dose. Determination of an optimal recommended phase two dose is among the most critical decisions for an early stage clinical program. With these changes to the trial protocol, we have the opportunity to move into genetically defined expansion cohorts prior to reaching a maximum tolerated dose to obtain a larger data set in an enriched population and to more confidently determine a recommended Phase II dose, thereby increasing the likelihood of success for the program. We believe that the clean safety and tolerability profile, the encouraging signs of clinical activity, and the wide therapeutic window of KL539 support a potential best-in-class profile, both as a monotherapy and in combination. We continue to actively engage with our key opinion leaders and global steering committees to further define a comprehensive clinical development plan for KL-539, including a potential third expansion cohort, combination studies in the front line, and a pediatric development strategy. We intend to move these efforts forward aggressively, pending determination of a recommended phase two dose, and we look forward to providing updates to you along the way. Now let's turn our attention to our farnesyl transferase inhibitor, tififarnic. Earlier this morning, we were very pleased to announce that tibifarnib has been granted breakthrough therapy designation from FDA for the treatment of patients with recurrent or metastatic atrius mutant head and neck squamous cell carcinoma, or HNSCC, with a variant allele frequency greater than or equal to 20% after disease progression on platinum-based chemotherapy. The breakthrough therapy designation is based upon data from our Phase II RUN-HN trial which was recently accepted for publication in an upcoming issue of the Journal of Clinical Oncology. As a reminder, the RUN-HN trial showed an objective response rate of approximately 50%, a progression-free survival of six months, and a median overall survival of 15 months. As a point of reference, the objective response rate for the three FDA-approved therapies for the treatment of HNSCC in the second line ranged from 13% to 16%, with a median progression-free survival of two to three months and a median overall survival of five to eight months. This breakthrough therapy designation acknowledges both the dire unmet need for patients with recurrent or metastatic HRAS mutant HNSCC and the promise of tipifarnib to provide clinical benefits to these patients. Benefits of breakthrough therapy designation include more frequent meetings and communications with FDA, intensive guidance on an efficient drug development program, eligibility for rolling review of an NDA submission, and an organizational commitment involving senior managers from FDA. We anticipate the breakthrough therapy designation will help to facilitate the development and ultimate approval of tibifarnib for the treatment of HNSCC patients. To that end, we remain focused on conducting our AIM-HN registration-directed trial and bringing tipifarnib to the market as quickly and as efficiently as possible. In addition, we're also leveraging new advances to expand the use of tipifarnib in combination with other oncology therapeutics to address larger patient populations and pursue earlier lines of therapy. Among these potential combinations, we've prioritized the combination of tipifarnib and an inhibitor of the enzyme PI3 kinase alpha in patients with HNSCC. Our preclinical data suggests that HRAS and PI3 kinase alpha are codependent oncogenes in HNSCC and that combining tipifarnib with a PI3 kinase alpha inhibitor has the potential to provide meaningfully better antitumor activity than inhibiting either target alone. We believe the total addressable population for tipifarnib may be as high as 50% of HNSCC. We continue to prepare for a Phase I-II proof-of-concept study of tipifarnib in combination with a PI3 kinase alpha inhibitor in patients who have HRAS overexpressing, PIK3CA mutated, and or PIK3CA amplified HNSCC, and we expect to initiate this study in the second half of 2021. Breakthrough therapy designation from FDA is the latest milestone in our effort to pioneer the use of farnesyl transferase inhibitors to treat patients with cancer. We view farnesyl transferase inhibition in oncology as a potentially valuable therapeutic and commercial franchise, one that has the potential to deliver multiple opportunities for additional indications. Over the past several years through our internal efforts and a network of academic collaborations, we've uncovered some compelling opportunities for farnesyl transferase inhibitors in combination with other targeted therapies. These efforts have both revealed some exciting new areas of biology and underscored to us the opportunity for a greater investment in this therapeutic class. Last year, we initiated a discovery stage program to develop a next-generation farnesyl transferase inhibitor. Our goal is to deliver a drug candidate that has comparable potency and selectivity and improved pharmacokinetic and physical chemical properties relative to tipifarnib. I'm pleased to report we've already identified multiple advanced lead compounds and expect to nominate a development candidate for IMD-enabling studies in mid-2021. We intend to direct this next-generation FTI at new biology and larger oncology indications, and we look forward to sharing our progress and our plans with you later this year. With that, I'll now turn the call over to Mark Grasso for a discussion of our financial results for the fourth quarter and full year 2020.