Ziftomenib Development and Clinical Trials - Ziftomenib is a targeted menin inhibitor being developed for relapsed/refractory and newly diagnosed acute myeloid leukemia (AML)[7] - An NDA for ziftomenib has been granted Priority Review, with a PDUFA target action date of November 30, 2025[7] - Kura Oncology and Kyowa Kirin are collaborating to investigate ziftomenib across the AML continuum, targeting up to 50% of patients for whom the Menin-KMT2A pathway is a disease driver[59] - KOMET-017 are Phase 3 clinical trials evaluating ziftomenib in combination with intensive chemotherapy (7+3) or non-intensive therapy (Venetoclax + Azacitidine) in newly diagnosed AML patients[62] KOMET-007 Clinical Trial Results - In the KOMET-007 study, the combination of ziftomenib 600 mg QD with 7+3 chemotherapy showed a safety profile consistent with previous reports for newly diagnosed AML patients treated with 7+3 alone[56] - The KOMET-007 study demonstrated robust clinical activity in newly diagnosed NPM1-m and KMT2A-r AML patients, with a composite complete remission (CRc) rate of 93% for NPM1-m and 89% for KMT2A-r patients[56] - Composite complete remission with measurable residual disease (CRc MRD) negativity was achieved in 68% of NPM1-m patients at a median of 4.7 weeks and 83% of KMT2A-r patients at a median of 4.1 weeks[56] - 96% (47/49) of NPM1-m and 88% (29/33) of KMT2A-r patients remained alive and continued on-study, with median follow-up times of 25 and 16 weeks, respectively[56] AML Market and Opportunity - An estimated 22,000 new cases of AML are diagnosed each year in the United States[16] - Up to 50% of AML cases may be menin-dependent, including those driven by NPM1m and KMT2Ar mutations[19] - The potential peak sales for menin inhibitors in first-line AML is estimated to be greater than $7 billion per year in the U.S[73]

Kura Oncology (KURA) EHA Virtual Investor Event Summary Company Overview - **Company**: Kura Oncology (KURA) - **Event Date**: June 18, 2025 - **Focus**: Update on ZIFTOMENED, an investigational Menin inhibitor for acute myeloid leukemia (AML) Key Points Industry Context - **Acute Myeloid Leukemia (AML)**: - Approximately 22,000 new cases annually in the U.S. - Median age of diagnosis is 69 years, with most patients diagnosed above this age [9][10] - Current therapies have limited long-term success, especially for older patients, with a 5-year survival rate of 60% to 50% for favorable AML [11] ZIFTOMENED Overview - **Investigational Drug**: ZIFTOMENED is under FDA review for relapsed and refractory NPM1 mutant AML, with a PDUFA target action date of November 30, 2025 [5] - **Partnership**: Collaboration with Kewa Kirin to advance the drug's development and commercialization [5] Clinical Data and Trials - **Phase 1 Study**: - Focused on the combination of ZIFTOMENED with intensive chemotherapy (7+3) for newly diagnosed AML patients with specific mutations (NPM1 and KMT2A) [15][16] - 82 patients enrolled, with a median age of 56; 93% remained on study at the data cutoff [22][18] - Safety profile similar to standard chemotherapy, with common adverse events including febrile neutropenia [22][23] - **Efficacy Results**: - Composite complete remission (CR) rates: 93% for NPM1 mutated patients and 89% for KMT2A rearranged patients [24] - Measurable residual disease (MRD) negativity rates: 68% for NPM1 mutated and 83% for KMT2A rearranged patients [25][32] - Median duration of CR not reached for both patient groups, indicating promising long-term outcomes [25][27] Market Opportunity - **Addressable Market**: - Estimated at over $7 billion annually for AML treatment, based on the potential to treat approximately 10,000 patients with Menin inhibitors for 12-24 months [47] - High unmet medical need, with 70% of patients relapsing within three years post-CR [47] Future Directions - **Clinical Trials**: - Plans to initiate two Phase 3 trials (COMMENT 17) to evaluate ZIFTOMENED in combination with high-intensity chemotherapy and in the maintenance setting post-transplant [38][50] - Focus on achieving MRD negativity as a primary endpoint, which is crucial for improving overall survival [40] Additional Insights - **Patient Management**: - Discussion on the potential to reduce the need for stem cell transplants by achieving deeper remissions with Menin inhibitors [61][62] - Importance of MRD negativity in guiding treatment decisions and improving patient outcomes [40][62] Conclusion - Kura Oncology is positioned to make significant advancements in the treatment of AML with ZIFTOMENED, addressing a critical unmet need in the market and aiming for improved patient outcomes through innovative therapies and clinical strategies [49][50]

Company Overview - Kura Oncology, Inc. (KURA) is currently experiencing significant activity in the options market, particularly with the July 18, 2025 $2.50 Put option showing high implied volatility, indicating potential for a major price movement [1][3] Analyst Sentiment - Kura Oncology holds a Zacks Rank 3 (Hold) within the Medical - Biomedical and Genetics industry, which is in the top 30% of the Zacks Industry Rank [3] - Over the past 60 days, four analysts have raised their earnings estimates for the current quarter, while one has lowered theirs, resulting in a shift in the Zacks Consensus Estimate from a loss of $0.62 per share to a profit of $0.21 per share [3] Options Market Insights - The high implied volatility surrounding Kura Oncology suggests that options traders anticipate a significant price movement, which could be indicative of an upcoming event that may lead to either a rally or a sell-off [2][4] - Seasoned options traders often seek out high implied volatility options to sell premium, aiming to benefit from the decay of the option's value if the underlying stock does not move as much as expected by expiration [4]

Core Insights - The KOMET-007 trial demonstrated encouraging clinical activity for ziftomenib in combination with 7+3 for newly diagnosed NPM1-m and KMT2A-r AML patients, showing high rates of complete remission and minimal residual disease negativity [1][2][4] Group 1: Clinical Data - In the KOMET-007 trial, 93% (41/44) of NPM1-m patients and 89% (24/27) of KMT2A-r patients achieved complete remission composite (CRc) [1] - Among responding patients, 71% (24/34) of NPM1-m and 88% (14/16) of KMT2A-r patients achieved measurable residual disease (MRD) negativity [1] - The median follow-up times were 24.9 weeks for NPM1-m patients and 15.7 weeks for KMT2A-r patients, with 96% (47/49) of NPM1-m and 88% (29/33) of KMT2A-r patients remaining alive [3] Group 2: Safety and Tolerability - The safety profile of ziftomenib was consistent with previous data, with Grade 3 adverse events occurring in over 10% of patients, including febrile neutropenia (15%) and decreased platelet count (15%) [4] - No dose-limiting toxicities or additive myelosuppression were observed, indicating a favorable safety profile for ziftomenib [4] Group 3: Future Developments - Kura Oncology plans to initiate the KOMET-017-IC and NIC Phase 3 studies in the second half of 2025 to further evaluate ziftomenib's efficacy in AML treatment [1][5] - A virtual investor event is scheduled for June 18, 2025, to discuss the results and broader development plans for ziftomenib [6] Group 4: Company Background - Kura Oncology is focused on developing precision medicines for cancer, with ziftomenib being the first investigational therapy to receive Breakthrough Therapy Designation from the FDA for R/R NPM1-m AML [7] - Kyowa Kirin, a partner in the development of ziftomenib, has a long history in drug discovery and biotechnology innovation, aiming to address high unmet medical needs [9]

Summary of Cura Oncology Conference Call Company Overview - **Company**: Cura Oncology - **Lead Program**: ZIFTOMENIB, a menin inhibitor for acute leukemia [3][4] Clinical Programs and Data - **ZIFTOMENIB**: - Data presented at ASCO supports NDA submission, currently under FDA review [4] - Expected launch later this year for relapsed/refractory population [4] - Upcoming data presentation at EHA regarding frontline opportunities [5] - Two Phase III trials planned to support registration in both intensive and non-intensive settings [6] - Market opportunity for menin inhibitors estimated at $7 billion, with ZIFTOMENIB potentially capturing half [6] - **GIST Program**: - Initiated dosing in GIST, aiming to reverse resistance to KIT inhibitors [7][12] - Potential market opportunity comparable to AML [14] - **Farnesyltransferase Programs**: - Data expected at ESMO, targeting solid tumor indications [8][17] Financial Position - Strong cash position with $73 million as of the last quarterly update [8] - Anticipated significant milestones related to ZIFTOMENIB commercialization [9] Safety and Efficacy - ZIFTOMENIB shows promising safety profile with no myelosuppression or significant drug-drug interactions [25][31] - Reported a complete response (CR) rate of 23% in relapsed/refractory NPM1 mutant population [23] - Median overall survival of 16.4 months in responders [24] - Focus on making AML treatment more chronic and manageable [27][29] Market Dynamics - Physicians show interest in using menin inhibitors earlier and in combination therapies [39][46] - Cura's commercial strategy includes educating physicians on safe usage and potential off-label opportunities [42][44] - Anticipated competition with Syndax's Rebuminib, with Cura's ZIFTOMENIB offering advantages in safety and convenience [39][75] Future Expectations - Upcoming data disclosures expected to provide insights into durability and MRD negativity rates [56][60] - Focus on maintaining patients on therapy for extended periods to improve outcomes [52][73] - Potential for ZIFTOMENIB to become a backbone therapy in AML treatment [81] Conclusion - Cura Oncology is positioned strongly with ZIFTOMENIB, targeting significant market opportunities in acute leukemia and GIST, while maintaining a robust financial position and a focus on safety and efficacy in treatment. The company is preparing for upcoming data releases and commercialization efforts, aiming to establish ZIFTOMENIB as a leading therapy in its class.

Core Insights - Kura Oncology's shares have increased by 15.5% over the past week, contrasting with a 0.1% decline in the industry [1] - The FDA has accepted Kura's new drug application (NDA) for ziftomenib, aimed at treating adult patients with relapsed or refractory acute myeloid leukemia (AML) with an NPM1 mutation [1][9] - A decision from the FDA regarding the NDA is anticipated on November 30, 2025, following a priority review [1] Company Developments - Ziftomenib is positioned to be the first menin inhibitor approved for treating R/R NPM1-mutant AML if the FDA grants approval [2] - The NDA submission was based on the phase II KOMET-001 study, which achieved its primary endpoint of complete remission and demonstrated statistically significant results [7] - Kura has entered into a partnership with Kyowa Kirin for the development and commercialization of ziftomenib, which triggered a $45 million milestone payment upon NDA submission [6][9] Clinical Pipeline - Ziftomenib is also being explored in combination with imatinib for advanced gastrointestinal stromal tumors after imatinib failure [10] - Kura is developing KO-2806, a next-generation farnesyl transferase inhibitor, for various solid tumors, and another FTI, tipifarnib, in combination with alpelisib for head and neck squamous cell carcinoma [11] - Data from these studies are expected to be presented later in 2025, which may further influence Kura's stock performance [12]

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 - The FDA has accepted the new drug application for Ziftamenib based on positive results from the COMET-one trial, with a PDUFA target action date of November 30, 2025 [5][39]. - The company reported having over $700 million on the balance sheet as of the end of the first quarter, with potential to earn an additional $375 million in near-term milestones [46]. Business Line Data and Key Metrics Changes - The COMET-one trial demonstrated a 35% overall response rate and a 25% complete remission with and without hematologic recovery (CRCRH) rate in patients treated with Ziftamenib [14][22]. - Among the 92 patients in the phase two portion of the study, the CRCRH rate was 23%, significantly superior to the historical 12% rate with conventional chemotherapy [14][22]. Market Data and Key Metrics Changes - The company is targeting the relapsed refractory acute myeloid leukemia (AML) patient population, where there is a high unmet medical need, with 20% being primary refractory and 50% relapsing within one year [39]. - Physicians have expressed positive feedback regarding Ziftamenib's overall survival among responders, which is longer than expected in this patient population [36]. Company Strategy and Development Direction - The company is pursuing an aggressive development and commercialization strategy for Ziftamenib, aiming to treat up to 50% of AML patients who may benefit from menin inhibitor therapy [5][25]. - A comprehensive development strategy is in place to address patients in both frontline and relapsed refractory settings, utilizing both company-sponsored and investigator-initiated studies [25][28]. Management's Comments on Operating Environment and Future Outlook - Management emphasized the importance of Ziftamenib's favorable safety profile, which positions it as a potentially best-in-class menin inhibitor for AML [44]. - The company is focused on supporting the FDA review process and preparing for a potential launch in the fourth quarter of 2025 [44][46]. Other Important Information - The COMET-one study showed that Ziftamenib was well tolerated, with low rates of myelosuppression and no clinically significant QTc prolongation [19][22]. - The differentiation syndrome was observed in 23% of patients, but with effective management strategies, the majority of patients continued therapy [21][80]. Q&A Session Summary Question: Can you talk about predicting patient responses to Ziftamenib? - Management indicated that currently, there is no clear way to predict which patients will respond to Ziftamenib, although some patients with IDH1 co-mutations appeared to respond better [50][53]. Question: What are the plans for commercial launch preparation? - The company is building out its capabilities to ensure product availability upon approval, learning from the market experience of other menin inhibitors [56][60]. Question: How are physicians managing differentiation syndrome? - Experienced investigators have implemented early recognition and mitigation strategies for differentiation syndrome, leading to better management and patient retention in studies [78][80]. Question: What differentiates Ziftamenib from other products in the market? - Convenience and safety are highlighted as key differentiators, with Ziftamenib being a once-daily oral medication that does not require extensive monitoring [86][90]. Question: What is the expected average duration of therapy for Ziftamenib? - The expected duration of therapy is approximately six months, based on the time needed for patients to achieve a response and the duration of that response [94][97]. Question: Why might FLT3 ITD patients have a lower response rate? - The lower response rate in FLT3 ITD patients may be due to their heavily pretreated status and the development of resistance to multiple therapies [100][101].

Core Insights - Kura Oncology and Kyowa Kirin announced positive pivotal results from the KOMET-001 Phase 2 trial of ziftomenib for relapsed/refractory NPM1-mutant AML patients, with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% [1][3][2] - The trial demonstrated consistent efficacy across various pre-specified subgroups, indicating the potential of ziftomenib as a treatment option for heavily pre-treated patients [1][4][2] - Ziftomenib has a favorable safety profile, with limited myelosuppression and a low discontinuation rate due to treatment-related adverse events [1][5][7] Company Overview - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment, with ziftomenib being the first investigational therapy to receive Breakthrough Therapy Designation from the FDA for R/R NPM1-m AML [9] - The company has a strategic collaboration with Kyowa Kirin to develop and commercialize ziftomenib, with ongoing clinical trials evaluating its efficacy in combination with current standards of care [9][10] Clinical Trial Details - The KOMET-001 Phase 2 trial included 92 adult patients with a median age of 69, with 33% having received three or more prior lines of therapy [2][3] - Among the patients achieving CR/CRh, 63% were minimal residual disease (MRD)-negative, highlighting the treatment's potential effectiveness [3][4] Safety and Tolerability - The safety profile of ziftomenib showed that treatment-related adverse events led to discontinuation in only 3% of patients, with differentiation syndrome being the most common adverse event [5][6][7] - The absence of clinically significant QTc prolongation and effective management of differentiation syndrome further support ziftomenib's favorable benefit-risk profile [7][6] Future Outlook - The FDA has set a PDUFA target action date of November 30, 2025, for the New Drug Application (NDA) of ziftomenib, indicating a potential first approval for a once-daily oral menin inhibitor for this patient population [1][2] - Kura Oncology is hosting a virtual investor event to discuss the trial results and future plans for ziftomenib [8]

Core Viewpoint - Kura Oncology and Kyowa Kirin have announced the acceptance of a New Drug Application (NDA) for ziftomenib by the FDA, targeting adult patients with relapsed or refractory acute myeloid leukemia (AML) with an NPM1 mutation, with a PDUFA target action date set for November 30, 2025 [1][2] Company Overview - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment, with a pipeline of small molecule drug candidates [7] - Kyowa Kirin is a Japan-based global specialty pharmaceutical company with over 70 years of experience in drug discovery and biotechnology innovation [8] Drug Development - Ziftomenib is an investigational menin inhibitor that has received Breakthrough Therapy Designation (BTD), Fast Track, and Orphan Drug Designations from the FDA for the treatment of adult patients with R/R AML with an NPM1 mutation [3][6] - The NDA is based on positive results from the Phase 2 KOMET-001 trial, which achieved its primary endpoint of complete remission and demonstrated a favorable safety profile with limited myelosuppression [2][3] Clinical Trial Insights - The KOMET-001 trial is designed to assess the clinical activity, safety, and tolerability of ziftomenib, and full data analyses will be presented at the 2025 ASCO Annual Meeting and the 2025 EHA Congress [3][4] - Adult patients with R/R NPM1-m AML have a poor prognosis, with only 30% overall survival at 12 months in the relapsed setting, highlighting the urgent need for innovative treatment options [4][5] Market Potential - There are currently no FDA-approved therapies specifically targeting NPM1-m AML, indicating a significant market opportunity for ziftomenib if approved [5][6]