行业研究丨专题报告丨医疗保健 [Table_Title] 药品产业链周度系列（四） 初探 Menin 抑制剂 报告要点 [Table_Summary] Menin 是一类关键表观遗传调控因子，在 KMT2A 重排与 NPM1 突变等急性白血病亚型中驱动 HOX/MEIS1 轴异常激活，是精准治疗 AML 的重要靶点。截至 2025 年 6 月，全球共有 9 款 Menin 抑制剂处于临床研发或已上市阶段，Revumenib 联合阿扎胞苷与维奈克拉治疗急性白血 病患者的 CRc 率为 81%，mOS 达 15.5 个月；Ziftomenib 联合"7+3"方案用于初诊 NPM1m 患者的 CRc 率高达 94%；BN104 在 KMT2A 重排复发难治人群中实现 CR/CRh 率为 60.9%， 安全性良好，具备 BIC 潜力。 分析师及联系人 [Table_Author] 彭英骐 徐晓欣 张楠 万梦蝶 刘长洪 SAC：S0490524030005 SAC：S0490522120001 SAC：S0490524070006 SAC：S0490525050001 SFC：BUZ392 [Table_Title 药 ...

– Encouraging clinical activity with deep responses demonstrated in the KOMET-007 trial with the combination of 600 mg ziftomenib with 7+3 in newly diagnosed patients with NPM1-m and KMT2A-r AML – – 93% (41/44) and 89% (24/27) CRc observed in NPM1-m and KMT2A-r AML response-evaluable patients, respectively – – 71% (24/34) and 88% (14/16) CR measurable residual disease (MRD)-negativity observed among responding NPM1-m and KMT2A-r AML patients, respectively – – 96% (47/49) of NPM1-m and 88% (29/33) KMT2A-r AM ...

Kura Oncology (KURA) FY Conference June 10, 2025 11:20 AM ET Speaker0 Thanks Speaker1 everyone for joining us. I'm Andrea Newkirk, one of the biotech analysts here at Goldman Sachs and I'm really pleased to be joined by Troy Wilson, President and CEO of Cura Oncology. Thanks so much Troy for joining us. Speaker0 My pleasure. Thank you for the chance to participate. Speaker1 Yeah, of course. Well maybe I will open it up to you for some high level remarks on the company, the overview of your clinical programs ...

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]

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]

Core Insights - Kura Oncology and Kyowa Kirin announced the acceptance of an abstract for the KOMET-001 trial of ziftomenib for R/R NPM1-m AML for oral presentation at the ASCO Annual Meeting in June 2025 [1][2] - Ziftomenib is the only investigational therapy with Breakthrough Therapy Designation from the FDA for treating R/R NPM1-m AML, which represents about 30% of AML cases [2][7] - The trial achieved its primary endpoint of complete remission and was well-tolerated with limited myelosuppression [2][3] Company Overview - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment, with ziftomenib being a key investigational therapy [7] - Kyowa Kirin is a Japan-based Global Specialty Pharmaceutical Company with over 70 years of experience in drug discovery and biotechnology innovation [8] Trial Details - The KOMET-001 trial is designed to assess the clinical activity, safety, and tolerability of ziftomenib in patients with R/R NPM1-m AML [2] - Positive topline results were previously announced, indicating a statistically significant primary endpoint achievement [2] - A trial-in-progress abstract for the KOMET-015 trial will also be presented at the ASCO Annual Meeting [3][4] Upcoming Events - Kura will host a virtual investor event on June 2, 2025, to discuss the trial results and provide insights from company management and investigators [5][6]

Core Insights - Kura Oncology and Kyowa Kirin announced that clinical data from the KOMET-007 trial of ziftomenib will be presented at the EHA2025 Congress, highlighting its potential as a treatment for acute myeloid leukemia (AML) patients with specific mutations [1][2][3] 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 relapsed or refractory NPM1-m AML [6][7] - Kyowa Kirin is a Japan-based global specialty pharmaceutical company with over 70 years of experience in drug discovery and biotechnology innovation, focusing on high unmet medical needs in various therapeutic areas [8] Clinical Trial Details - The KOMET-007 trial is a multicenter Phase 1 study evaluating ziftomenib in combination with standard care treatments for newly diagnosed AML patients with NPM1-m or KMT2A-r mutations [2][3] - The upcoming oral presentation will include updated data from the Phase 1a and Phase 1b portions of the trial, specifically focusing on the combination of ziftomenib with the 7+3 chemotherapy regimen [2][4] Future Developments - The results from the KOMET-007 trial are expected to support the initiation of a pivotal Phase 3 trial, KOMET-017, aimed at further evaluating the combination treatment for AML patients [3][5] - Kura and Kyowa Kirin are also conducting additional clinical trials to assess ziftomenib in various combinations for both newly diagnosed and relapsed/refractory AML patients [7]

Summary of Conference Call Company Overview - The conference call discusses **Cura**, focusing on its clinical programs involving **menin inhibitors** and their applications in treating various cancers, particularly acute leukemia and gastrointestinal stromal tumors (GIST) [1][2][3]. Key Points and Arguments Menin Inhibitors - **Mechanism of Action**: Menin inhibitors are epigenetic regulators that affect gene expression, relevant in treating up to half of leukemia cases [2]. - **Clinical Applications**: Current trials include Zifdomenon in combination with imatinib for GIST, targeting the KIT gene, and exploring effects in diabetes by regulating pancreatic beta cell growth [3][4]. Upcoming Data and Competitive Landscape - The **ASCO meeting** is highlighted as a critical event for CURA, where data on cifdomenon pitavutal in relapsed/refractory AML will be presented [5]. - CURA aims to differentiate itself from competitors through superior safety and tolerability profiles, which are crucial for patient adherence to treatment [6][7]. Safety and Tolerability - ZiftoMenib is noted for its lack of clinically meaningful drug-drug interactions and dose-limiting toxicities, making it suitable for elderly patients who often have multiple comorbidities [10][11]. - The term "cruise medicine" is introduced, emphasizing the importance of maintaining patients' quality of life during treatment [13][15]. Regulatory Interactions - CURA submitted its **NDA** on March 31, with a request for priority review due to its breakthrough therapy designation, which could lead to a six-month review period [16][17]. - Positive interactions with the FDA are reported, with no disruptions noted in the review process [18][19]. Clinical Trial Insights - The **Frontline seven expansion cohort** is expected to yield new data, focusing on MRD negativity and event-free survival as primary endpoints [22][25]. - The historical context of relapse rates in AML is discussed, with a focus on improving response rates and delaying relapse through combination therapies [24][27]. Commercial Strategy - CURA's partnership with **Kyowa Kirin** is aimed at maximizing the value of Zifdomenib across various indications, including AML, GIST, and diabetes [58]. - The company plans to maintain a strong financial position without returning to equity markets, ensuring continued investment in its clinical programs [59]. Future Directions - Upcoming data presentations are anticipated for both frontline and relapsed/refractory settings, with a focus on the combination of ZiftoMenib with other therapies like venetoclax [52][53]. - The potential for ZiftoMenib to resensitize patients to venetoclax is noted, which could lead to improved complete response rates [50][51]. Additional Important Content - The call emphasizes the importance of patient-centric approaches in treatment, aiming to minimize toxicity and enhance quality of life [15][30]. - The competitive landscape is acknowledged, with CURA positioning itself against both menin inhibitors and other classes of therapies [54][56]. This summary encapsulates the critical insights from the conference call, focusing on CURA's strategic direction, clinical advancements, and regulatory interactions.

Core Viewpoint - Kura Oncology has submitted its first New Drug Application (NDA) for ziftomenib to the FDA for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an NPM1 mutation, marking a significant milestone for the company [2][5]. Company Updates - The NDA submission for ziftomenib occurred on March 31, 2025, with a 60-day filing review period expected from the FDA, potentially leading to a six-month review if Priority Review is granted [5]. - Kura Oncology is preparing for two Phase 3 studies in the frontline setting and anticipates multiple clinical data readouts throughout the year [2][4]. - The first patients have been dosed in the Phase 1 trial of ziftomenib in combination with imatinib for gastrointestinal stromal tumors (GIST) [1][6]. Financial Highlights - Collaboration revenue from the partnership with Kyowa Kirin for Q1 2025 was $14.1 million, compared to no revenue in Q1 2024 [9]. - Research and development expenses for Q1 2025 were $56.0 million, up from $36.3 million in Q1 2024, while general and administrative expenses increased to $22.8 million from $18.2 million [9]. - The net loss for Q1 2025 was $57.4 million, compared to a net loss of $49.5 million in Q1 2024, with cash, cash equivalents, and short-term investments totaling $658.2 million as of March 31, 2025 [9][16]. Milestones and Future Plans - Kura expects to present data from the KOMET-001 Phase 1b/2 trial at the ASCO and EHA meetings in the second quarter of 2025 [9]. - The company plans to initiate two independent Phase 3 registration-enabling trials in the second half of 2025 [9][13]. - Kura anticipates receiving up to $375 million in additional milestone payments from Kyowa Kirin, following the $45 million milestone payment triggered by the NDA submission [4][5].

Core Insights - Kura Oncology has initiated the KOMET-015 Phase 1 clinical trial to evaluate ziftomenib in combination with imatinib for patients with advanced gastrointestinal stromal tumors (GIST) after imatinib failure [1][3] - The combination of ziftomenib and imatinib has shown robust and durable antitumor activity in both imatinib-sensitive and imatinib-resistant GIST preclinical models [1][2] Company Overview - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment, with ziftomenib being a key investigational menin inhibitor [8] - The company has received Breakthrough Therapy Designation from the FDA for ziftomenib in treating relapsed/refractory NPM1-mutant acute myeloid leukemia (AML) [7][8] Clinical Trial Details - The KOMET-015 trial is designed to assess the safety, tolerability, and preliminary antitumor activity of ziftomenib combined with imatinib in adults with GIST who have shown disease progression on imatinib [3][4] - The trial will evaluate primary objectives including safety and tolerability, and secondary endpoints such as overall response rate (ORR) and progression-free survival (PFS) [3] Market Context - Approximately 4,000 to 6,000 new cases of GIST are diagnosed annually in the U.S., with limited treatment options available for advanced cases [2][5] - Most patients develop resistance to imatinib within two years, highlighting the need for new therapeutic options [2][6] Preclinical Findings - Preclinical studies indicate that the combination of ziftomenib and imatinib exerts antitumor activity through a synthetic lethal mechanism, targeting vulnerabilities in GIST tumors [2][3] - The combination has the potential to delay or overcome resistance to imatinib in patients [2]