2020 ANNUAL REPORT Transformative Realizing the Promise of Precision Medicines to Help Patients with Cancer Lead Better, Longer Lives
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2020 ANNUAL REPORT Transformative Realizing the promise of precision medicines to help patients with cancer lead better, longer lives SIGNIFICANT MARKET POTENTIAL TO TREAT LEVERAGING NEW OPPORTUNITY DEVASTATING DISEASE ADVANCES Novel menin inhibitor, Breakthrough Therapy Next-generation FTI KO-539, with potential Designation from FDA directed at new biology to treat 35% or more for tipifarnib in HNSCC and larger oncology of AML indications MULTIPLE EXPANSION ESTABLISHED STRONG CAPITAL OPPORTUNITIES LEADERSHIP TEAM POSITION Preparing to move into Proven oncology drug $633.3 million in cash, larger populations, development and cash equivalents and combinations and commercialization investments provide earlier lines of therapy expertise runway into 2024 To Our Shareholders This past year has been a truly transformative one for Kura Oncology. Despite the unique challenges we faced in 2020, we came together during the COVID-19 pandemic to adapt, evolve and grow, while remaining focused on our mission to realize the promise of precision medicines to help patients with cancer lead better, longer lives. Here are several highlights: Great Companies Are Built by Great People Despite the challenges of the past year, our organization has nearly doubled in size as we have added key functions. Our leadership team, hiring managers and human resource professionals have done a terrific job recruiting, onboarding, and integrating our newest colleagues virtually as we enlisted them in our mission to improve the lives “We focused our of patients. business around two major pillars, I am particularly pleased that, in August 2020, we recruited Stephen our emerging menin Dale, M.D., a globally recognized leader in drug development as our inhibitor, KO-539, Chief Medical Officer. Stephen joined us from Kyowa Kirin, where he and our late-stage served as SVP and Global Head of Medical Science. Previously, he farnesyl transferase was Global Clinical Vice President and Clinical Head of Oncology at inhibitor, tipifarnib. The results have been AstraZeneca, where among his many accomplishments, he oversaw the truly remarkable, and development of Tagrisso® (osimertinib) for metastatic EGFR-T790M we have positioned mutation-positive non-small cell lung cancer. our programs for continued Optimizing Our Business to Maximize Value for Shareholders advancement in the year ahead.” In early 2020, our leadership conducted an analysis with the goal of creating the greatest value for patients and shareholders, while Troy E. Wilson, Ph.D., J.D. balancing investment, risk and execution. As a result, we focused President & Chief our business and our efforts around two major pillars – our menin Executive Officer inhibitor, KO-539, in AML and our late-stage farnesyl transferase inhibitor, tipifarnib, in HNSCC. The results have been truly remarkable, and we have positioned our programs for continued advancement in the year ahead. KURA ONCOLOGY 2020 ANNUAL REPORT 1 KO-539 Demonstrates Highly Encouraging Preliminary Clinical Data in AML In December 2020, we presented the first clinical data from KOMET-001, our ongoing Phase 1/2 clinical trial of our menin inhibitor, KO-539, at the American Society of Hematology Annual Meeting. These data were highlighted by activity in an all-comer population of patients with relapsed or refractory acute myeloid leukemia (AML), and they support a potentially best-in-class profile both as a monotherapy and in combination. Now we look forward to obtaining a larger clinical dataset as we move into genetically enriched Phase 1b expansion cohorts, including NPM1 mutant AML and KMT2A rearranged relapsed/refractory AML patients. “The preliminary Breakthrough Therapy Designation for Tipifarnib in HRAS Mutant clinical data for Head and Neck Cancer KO-539 suggest it has the potential to be Earlier this year, our farnesyl transferase inhibitor, tipifarnib, received effective for multiple Breakthrough Therapy Designation from the FDA for the treatment genetically defined of patients with recurrent or metastatic HRAS mutant head and neck subgroups of acute squamous cell carcinoma (HNSCC). We appreciate the agency’s myeloid leukemia, affirmation of the potential for tipifarnib to treat this devastating where prognosis disease, and we look forward to working closely with them to bring this remains poor.” therapy to patients as soon as possible. Eunice Wang, M.D. Roswell Park Publication of Data from Our RUN-HN Study in Journal of Comprehensive Cancer Clinical Oncology Center We recently announced the publication of results from our Phase 2 RUN-HN trial of tipifarnib showing an objective response rate (ORR) of 55% with a median progression-free survival (PFS) of 5.6 months and median overall survival (OS) of 15.4 months in recurrent/ metastatic HRAS mutant HNSCC. The results formed the basis of tipifarnib’s Breakthrough Therapy Designation and support the AIM- HN registration-directed in patients with recurrent or metastatic HRAS mutant HNSCC, which is currently recruiting at more than 100 sites around the globe. 2 KURA ONCOLOGY 2020 ANNUAL REPORT New Approaches to Address Larger Patient Populations and Pursue Earlier Lines of Therapy As we continue to advance tipifarnib as a monotherapy in HNSCC, we are leveraging new advances and insights to expand its use in combination with other oncology therapeutics. Specifically, we have prioritized the combination of tipifarnib and an inhibition of the PI3Kα proto-oncogene in patients with HNSCC. We believe this combination has the potential to treat between 20-50% of HNSCC patients, a devastating disease for which there are no FDA approved small molecule targeted therapies, and we look forward to initiating this trial later this year. Strengthened Balance Sheet Along with executing against our ambitious R&D goals, we took the “We are encouraged opportunity last year to strengthen Kura’s balance sheet. I am pleased by the compelling to say that, with more than $600 million in cash, we have runway into at efficacy and safety least 2024 and the resources we need to reach critical value-inflection profile of tipifarnib in points for our programs. patients with recurrent or metastatic HRAS Well Positioned to Advance Our Drug Candidates in the Year Ahead mutant head and neck squamous cell On behalf of Kura’s leadership and board of directors, I would like carcinoma, a disease of to thank the patients in our clinical studies, our employees for their high unmet need.” willingness to adapt in unusual circumstances with courage and positivity, and our shareholders for your continued encouragement and Alan Ho, M.D., Ph.D. support as we navigated the storm of 2020. We remain committed to Memorial Sloan Kettering Cancer Center realizing the promise of precision medicines for the treatment of cancer, and I look forward to updating you on our progress in the year ahead. Sincerely, Troy E. Wilson, Ph.D., J.D. President & Chief Executive Officer KURA ONCOLOGY 2020 ANNUAL REPORT 3 Drug Candidate Pipeline Registration Program Preclinical Phase 1 Phase 2 Directed Acute Myeloid Leukemia (AML) KO-539 Menin Inhibitor • Enrollment in Phase 1 expansion cohorts expected to begin in mid-2021 HRASm Head and Neck Squamous Cell Carcinoma (HNSCC) Tipifarnib • Enrollment in AIM-HN registration-directed trial ongoing Farnesyl Transferase Inhibitor PI3Kɲ / HRAS dependent HNSCC • Initiation of PI3Kɲ inhibitor combination study expected in 2H 2021 Next-Generation Solid tumors Farnesyl Transferase Inhibitor • Nomination of development candidate expected in mid-2021 Relapsed/Refractory AML is a Challenging Disease “We are in a stronger Associated with Poor Outcomes financial position than ever before, with more NPM1-Mutant AML KMT2A(MLL)-Rearranged AML than $600 million in cash at the end of Estimated 6,000 new cases Estimated 1,000-2,000 new 2020, which we believe in the U.S. per year1 cases in the U.S. per year1 provides us with (~30% of AML) ( 5-10% of AML) sufficient resources to Known co-mutations confer NCCN guidelines denote advance our programs worse prognosis2 and represent that MLL-r confers 3 through multiple value- rational combination approaches poor prognosis inflection points.” Marc Grasso, M.D. Tipifarnib Has the Potential to be the First Small Chief Financial Officer Molecule Targeted Therapy for HNSCC Patients and Chief Business Officer Globally, ~885,000 people Outcomes with currently develop head and neck cancer available therapies (including annually and ~450,000 die of I-O therapy) are poor6 4 HNSCC each year OS First line: 10-15 mo; Second line: 5-8 mo 60,000+ cases of HNSCC per PFS year in the U.S.5 First line: 3-5 mo; Second line: 2-3 mo ORR First line: 20-36%; Second line: 13-16% 1 SEER statistics for AML in the US, accessed April 2020 2 Döhner et al. Blood. 2017 Jan 26;129(4):424-447 3 NCCN. AML Guidelines (version 3.2020). Accessed May 2020 4 Bray et al. CA Cancer J Clin. 2018;68(6):394-424 5 Cramer et al. Nat Rev Clin Oncol. 2019 Nov;16(11):669-683 | ACS Cancer Facts and Figures 2020 6 N Engl J Med. 2008 Sep 11;359(11):1116-27 | Keytruda & Opdivo package inserts | J Clin Oncol. 2007 Jun 1;25(16):2171-7 | J Clin Oncol. 2012 30:15_suppl, 5574-5574 4 KURA ONCOLOGY 2020 ANNUAL REPORT UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ց ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 OR տ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD