Summary of Kura Oncology Conference Call Company Overview - **Company**: Kura Oncology - **Event**: Jefferies Global Healthcare Conference 2025 - **Key Participants**: CEO Troy, Commercial Officer Brian, CMO Molly Industry Focus - **Industry**: Oncology, specifically Acute Myeloid Leukemia (AML) and Menin inhibitors Key Highlights from the Call 1. **Positive Trial Results**: Kura Oncology reported two positive trials indicating that their mechanism works in AML, suggesting that their drugs are likely approvable [4][10] 2. **Clinical Data**: - Phase 2 trial showed a 23% complete response (CR) rate and a median overall survival of 16.4 months for responders, compared to typical survival of 3 months in relapsed refractory leukemia [5] - Zifdomenib demonstrated consistent activity regardless of prior treatments or therapy lines [6] 3. **Safety Profile**: - No myelosuppression differentiation syndrome was reported, and QT prolongation was not clinically meaningful [7][8] - Once-daily oral dosing was highlighted as a convenience factor for patients and physicians [8][15] 4. **Regulatory Milestones**: - Kura has a PDUFA date set for November 30, indicating a priority review status [10][12] - The company is preparing for a significant data presentation at EHA on June 12 [9] 5. **Market Opportunity**: - Approximately 20,000 AML patients are diagnosed annually, with an estimated 10,000 potentially eligible for Menin inhibitors [52] - The potential market size for Kura's product in AML is estimated between $5 billion to $10 billion, with Kura aiming to capture around $3 billion [53] 6. **Combination Therapies**: - Kura is exploring combination therapies, including a study combining Zifdomenib with Imatinib for GIST, which could enhance treatment durability [56][59] - The company is also investigating combinations in breast cancer and other solid tumors [60][61] 7. **Pipeline and Funding**: - Kura has $703 million in cash and is eligible for an additional $375 million in near-term milestones, ensuring funding through the AML program [67] - The company is focused on advancing its pipeline, including FTI programs and diabetes treatments [67] Additional Insights - **Physician Feedback**: Physicians are excited about the introduction of Menin inhibitors, indicating a competitive landscape but also a growing acceptance of new therapies [21][22] - **Regulatory Environment**: Kura does not anticipate changes in FDA perspectives that would affect their accelerated approval pathway [33][36] - **Operational Efficiency**: Kura is combining two phase three trials under a single protocol to streamline operations and enhance patient eligibility [39][40] This summary encapsulates the critical points discussed during the conference call, highlighting Kura Oncology's advancements, market potential, and strategic direction in the oncology space.

Financial Data and Key Metrics Changes - Collaboration revenue from the Kyowa Kirin partnership for Q1 2025 was $14.1 million compared to no revenue in Q1 2024 [20] - Research and development expenses for Q1 2025 were $56 million compared to $36.3 million for Q1 2024 [20] - General and administrative expenses for Q1 2025 were $22.8 million compared to $18.2 million for the same period in 2024 [20] - Net loss for Q1 2025 was $57.4 million compared to a net loss of $49.5 million for Q1 2024 [20] - As of March 31, 2025, the company had cash, cash equivalents, and short-term investments of $658.2 million compared to $727.4 million as of December 31, 2024 [21] Business Line Data and Key Metrics Changes - The Ziftamenib development program achieved its primary CRCRH endpoint in the Phase II trial for relapsed/refractory NPM1 mutant AML, with data to be presented at the 2025 ASCO Annual Meeting [11][12] - The company announced the first patients with advanced gastrointestinal stromal tumors (GIST) were dosed with the combination of Ziftamenib and imatinib [8] Market Data and Key Metrics Changes - The company is focusing on the NPM1 mutant AML market, which represents approximately 30% of new AML cases annually, indicating a significant unmet need [10] - The company is also targeting advanced GIST patients, where current treatment options are limited [15] Company Strategy and Development Direction - The company aims to commercialize Ziftamenib in acute myeloid leukemia (AML) and is preparing for multiple clinical data updates throughout the year [6][7] - The strategic partnership with Kyowa Kirin is expected to provide greater development, commercial, and operational resources, enhancing financial stability [19] - The company plans to initiate the COMMENT-seventeen Phase III trials in the second half of 2025, focusing on both intensive and non-intensive treatment regimens for AML [14][23] Management's Comments on Operating Environment and Future Outlook - Management remains optimistic about the potential for Ziftamenib to impact patients with relapsed/refractory NPM1 mutant AML, citing a collaborative relationship with the FDA [10] - The company is monitoring the macro landscape and believes the impact from tariffs will be negligible [19] Other Important Information - The company has a strong cash position, with sufficient capital to fund the Ziftamenib AML program to commercialization and advance its pipeline [21] - The company is preparing for upcoming milestones, including presentations at major oncology meetings and the initiation of new trials [23] Q&A Session Summary Question: Expectations for combo data coming later this year - Management emphasized safety as the top priority for the combination data and confirmed they are on track to start the study in the second half of 2025 [26][29] Question: Impact of regulatory changes on approval process - Management stated there have been no disruptions from regulatory changes and they expect to receive notification from the FDA regarding the application review in the second quarter [33][36] Question: Market share strategy for NPM1 market - Management believes they are not necessarily second to market and are prepared to compete aggressively for patients [66][70] Question: Patient segments responsive to GIST combination - The combination approach is expected to be mutationally agnostic, potentially benefiting both imatinib-refractory and naive patients [74][75] Question: Enrollment timeline for pivotal trials - Management indicated that there should not be a drag on enrollment for frontline trials, as no menin inhibitor is currently approved for that patient population [114][116]