Latest news with #KOMET-001
Business Insider
03-06-2025
- Business
- Business Insider
Kura Oncology says ‘positive' results from KOMET-001 Phase 2 trial of ziftomenib
Kura Oncology (KURA) announced the presentation of positive pivotal results from the KOMET-001 Phase 2 registration-directed trial of ziftomenib, a once-daily, oral investigational menin inhibitor, in patients with relapsed/refractory NPM1-mutant acute myeloid leukemia in an oral session at the 2025 American Society of Clinical Oncology Annual Meeting being held in Chicago, IL from May 30 – June 3, 2025. 'We are delighted to announce positive pivotal data from the KOMET-001 trial in R/R NPM1-mutated AML patients treated with ziftomenib,' said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. 'NPM1 mutations are among the most common in AML, representing approximately 30% of cases, and there are no FDA-approved therapies specifically for this patient population. With these encouraging results and a PDUFA target action date of November 30, 2025, we and our partners at Kyowa Kirin look forward to supporting FDA with its review of the ziftomenib New Drug Application and are well-positioned to meaningfully impact relapsed or refractory patients with NPM1 mutations.' The KOMET-001 Phase 2 population included 92 adult patients with R/R NPM1-m AML. The median age was 69. Patients were heavily pretreated, with 33% having received three or more prior lines of therapy (median prior lines: 2) and 59% having been previously treated with venetoclax. A complete remission plus CR with partial hematological recovery rate of 23% was observed among patients with R/R NPM1-m AML in the Phase 2 portion of the KOMET-001 trial. Among those 21 patients who achieved CR/CRh, 13 had a CR and 8 had a CRh. The median duration of CR/CRh responses was 3.7 months and the restricted mean duration of response was 4.3 months at the time of the data cutoff. MRD status was assessed in 19 of 21 patients who achieved CR/CRh, and 63% of these patients were MRD-negative. Comparable CR/CRh rates were observed across pre-specified subgroups, regardless of prior HSCT, prior venetoclax or FLT3/IDH co-mutations. Additional patient benefit beyond CR/CRh was observed with a rate of transfusion conversion of 21% and a rate of maintenance of transfusion independence of 20%. A median OS of 16.4 months was observed for responders and a median overall survival of 3.5 months was observed among non-responders. The safety population included 112 adult patients with R/R NPM1-m AML from the pooled Phase 1b and Phase 2 portions of the KOMET-001 trial. The safety profile observed with ziftomenib in this population was consistent with previously reported data. Treatment-related adverse events led to treatment discontinuations in 3% of patients. TRAEs of Grade greater than or equal to3 which occurred in more than 10% of patients were limited to differentiation syndrome, which was well managed by protocol-specified mitigation strategies and no Grade 4/5 treatment-related DS was observed. Although QTc prolongation was reported in three patients per investigator assessment, all three patients were on concomitant medications associated with QTc prolongation, two had electrolyte abnormalities and one had a prior diagnosis of atrial fibrillation.
Yahoo
02-06-2025
- Business
- Yahoo
Kura Oncology and Kyowa Kirin Report Positive Pivotal Ziftomenib Monotherapy Data at 2025 ASCO Annual Meeting
– CR/CRh rate of 23% in pivotal Ph 2 cohort of R/R NPM1-m AML patients – – Consistent efficacy with comparable CR/CRh rates and clinically meaningful MRD-negative responses across pre-specified subgroups, regardless of prior HSCT, prior venetoclax, or FLT3/IDH co-mutations – – Favorable safety and tolerability profile in heavily pre-treated patients: limited myelosuppression; no clinically meaningful QTc prolongation; 3% of patients discontinued due to treatment-related adverse events – – Potential first approval of a once-daily, oral menin inhibitor for treatment of adult patients with relapsed or refractory NPM1-mutated AML with Priority Review and a PDUFA target action date of November 30, 2025 – – Kura Oncology to host virtual investor event today at 7:30pm ET / 4:30pm PT – SAN DIEGO and TOKYO, June 02, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, 'Kura') and Kyowa Kirin Co., Ltd. (TSE: 4151, 'Kyowa Kirin') announced the presentation of positive pivotal results from the KOMET-001 Phase 2 registration-directed trial of ziftomenib, a once-daily, oral investigational menin inhibitor, in patients with relapsed/refractory (R/R) NPM1-mutant (NPM1-m) acute myeloid leukemia (AML) in an oral session today at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting being held in Chicago, IL from May 30 - June 3, 2025. "We are delighted to announce positive pivotal data from the KOMET-001 trial in R/R NPM1-mutated AML patients treated with ziftomenib," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "NPM1 mutations are among the most common in AML, representing approximately 30% of cases, and there are no FDA-approved therapies specifically for this patient population. With these encouraging results and a PDUFA target action date of November 30, 2025, we and our partners at Kyowa Kirin look forward to supporting FDA with its review of the ziftomenib New Drug Application (NDA) and are well-positioned to meaningfully impact relapsed or refractory patients with NPM1 mutations.' "Relapsed or refractory NPM1-mutated AML is a highly challenging disease with a poor prognosis and an urgent need for new treatments," said Eunice Wang, M.D., Chief of Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center, Buffalo, NY. "The promising results for ziftomenib in this heavily pretreated population are highly encouraging. Notably, the clinically meaningful minimal residual disease (MRD)-negative responses observed as well as the similar response rates seen regardless of prior therapies, including hematopoietic stem cell transplantation (HSCT) and venetoclax, hold great promise for the potential use of ziftomenib in patients with relapsed and refractory NPM1-mutated AML.' The KOMET-001 Phase 2 population included 92 adult patients with R/R NPM1-m AML. The median age was 69 (range: 33 to 84). Patients were heavily pretreated, with 33% having received three or more prior lines of therapy (median prior lines: 2) and 59% having been previously treated with venetoclax. A complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% (21/92) was observed among patients with R/R NPM1-m AML in the Phase 2 portion of the KOMET-001 trial. Among those 21 patients who achieved CR/CRh, 13 had a CR and 8 had a CRh. The median duration of CR/CRh responses was 3.7 months (95% CI: 1.9, not estimable (NE)) and the restricted mean duration of response was 4.3 months (95% CI: 3.1, 5.6) at the time of the data cutoff. MRD status was assessed in 19 of 21 patients who achieved CR/CRh, and 63% (12/19) of these patients were MRD-negative. Comparable CR/CRh rates were observed across pre-specified subgroups, regardless of prior HSCT, prior venetoclax or FLT3/IDH co-mutations. Additional patient benefit beyond CR/CRh was observed with a rate of transfusion conversion of 21% (17/82; 95% CI: 13-31) and a rate of maintenance of transfusion independence of 20% (2/10; 95% CI: 3-56). A median OS of 16.4 months (95% CI, 9.6–20.4) was observed for responders (patients who achieved CR, CRh, CRi/CRp, MLFS or PR) and a median overall survival (OS) of 3.5 months (95% CI, 2.5–4.0) was observed among non-responders. The safety population included 112 adult patients with R/R NPM1-m AML from the pooled Phase 1b and Phase 2 portions of the KOMET-001 trial. The safety profile observed with ziftomenib in this population was consistent with previously reported data. Treatment-related adverse events (TRAEs) led to treatment discontinuations in 3% of patients. TRAEs of Grade ≥3 which occurred in more than 10% of patients were limited to differentiation syndrome (DS, 13%), which was well managed by protocol-specified mitigation strategies and no Grade 4/5 treatment-related DS was observed. Although QTc prolongation (1 Gr2; 2 Gr3) was reported in three patients per investigator assessment, all three patients were on concomitant medications associated with QTc prolongation, two had electrolyte abnormalities and one had a prior diagnosis of atrial fibrillation. 'Beyond ziftomenib's clinical activity, we are highly encouraged by its consistent safety and tolerability profile,' said Mollie Leoni, MD, Chief Medical Officer of Kura Oncology. 'Notably, the low rate of myelosuppression, low discontinuation rate, lack of clinically significant QTc prolongation, absence of drug-drug interactions, and effective management of differentiation syndrome underscore ziftomenib's potentially favorable benefit-risk profile for patients with relapsed or refractory NPM1-mutated AML.' 'The data presented at ASCO strengthen our conviction that ziftomenib has potential to become a meaningful treatment option for patients with relapsed or refractory AML with NPM1 mutations — patients who often face limited treatment options and significant uncertainty regarding their prognosis,' said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. 'Encouraged by the favorable safety, tolerability, and promising clinical activity observed thus far, Kyowa Kirin, in collaboration with Kura, is working with urgency and purpose to bring ziftomenib monotherapy to patients as swiftly and responsibly as possible.' Virtual Investor Event Kura will host a virtual investor event featuring company management and investigators from the KOMET-001 trial of ziftomenib in R/R NPM1-m AML at 7:30pm ET / 4:30pm PT on Monday, June 2, 2025. Those who would like to participate may access the live webcast here, or register in advance for the teleconference event can also be accessed on the Investors section of Kura's website at An archived replay will be available shortly after the conclusion of the live event. About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company's pipeline consists of small molecule drug candidates designed to target cancer signaling pathways. Ziftomenib, a once-daily, oral menin inhibitor, is the first and only investigational therapy to receive Breakthrough Therapy Designation from the FDA for the treatment of R/R NPM1-m AML. In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib for AML and other hematologic malignancies. Enrollment in KOMET-001, a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-m AML, has been completed, and the companies announced submission of an NDA for ziftomenib for the treatment of adult patients with R/R NPM1-m AML in the first quarter of 2025. Kura and Kyowa Kirin are conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-m and KMT2A-rearranged AML. Ziftomenib is also being evaluated in a Phase1 dose-escalation trial (KOMET-015) in combination with imatinib for treatment of patients with advanced GIST. KO-2806, a next-generation farnesyl transferase inhibitor (FTI), is being evaluated in a Phase 1 dose-escalation trial (FIT-001) as a monotherapy and in combination with targeted therapies for patients with various solid tumors. Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial (KURRENT-HN) in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma. For additional information, please visit Kura's website at and follow us on X and LinkedIn. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin's values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at Kura Forward-Looking Statements This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of ziftomenib; interactions with the FDA relating to our NDA for ziftomenib; the anticipated timing of FDA approval of our NDA and the potential to benefit patients with R/R NPM1-m AML. Factors that may cause actual results to differ materially include the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Kura may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings, and other interactions with regulatory bodies, risks associated with reliance on third parties to successfully conduct clinical trials, the risks associated with reliance on outside financing to meet capital requirements, the risk that the collaboration with Kyowa Kirin is unsuccessful, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words 'may,' 'will,' 'would,' 'could,' 'should,' 'believes,' 'estimates,' 'projects,' 'promise,' 'potential,' 'expects,' 'plans,' 'anticipates,' 'intends,' 'continues,' 'designed,' 'goal,' or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the Securities and Exchange Commission, which are available at Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Kura Contacts Investors: Patti Bank Managing Director(415) Media:media@ Kyowa Kirin Contacts Investors: Ryohei Kawaiir@ Media, Global:Wataru Suzuki, media@ in to access your portfolio
Yahoo
02-06-2025
- Business
- Yahoo
Kura Oncology and Kyowa Kirin Announce FDA Acceptance and Priority Review of New Drug Application for Ziftomenib in Adults with Relapsed or Refractory NPM1-Mutant AML
– New Drug Application based on positive results from the Phase 2 KOMET-001 trial – – FDA assigns a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025 – – Potential first approval of a menin inhibitor for the treatment of adult patients with relapsed or refractory AML with an NPM1 mutation – SAN DIEGO and TOKYO, June 01, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, 'Kura') and Kyowa Kirin Co., Ltd. (TSE: 4151, 'Kyowa Kirin') today announced the U.S. Food and Drug Administration (FDA) has accepted Kura's New Drug Application (NDA) seeking full approval for ziftomenib as a treatment for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025. 'The FDA's acceptance of our New Drug Application marks a significant milestone for Kura and Kyowa Kirin and, more importantly, for patients living with this genetic subset of AML, who face an aggressive form of the disease with few treatment options,' said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. 'This achievement reflects the strength of the clinical data for ziftomenib as well as the incredible commitment of our teams. Along with our partners at Kyowa Kirin, we look forward to continuing to work closely with the FDA throughout the review process and to prepare for the anticipated launch of this treatment, which holds potential to meaningfully impact the lives of patients and their families.' The NDA is based on results from the Phase 2 KOMET-001 registrational trial in R/R NPM1-mutant (NPM1-m) AML (NCT #04067336). The KOMET-001 trial achieved its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. Ziftomenib was well‑tolerated with limited myelosuppression and 3% ziftomenib-related discontinuations. The safety and tolerability of ziftomenib were consistent with previous reports, and the benefit-risk profile for ziftomenib is highly encouraging. 'Adult R/R NPM1-m AML patients face a significantly poor prognosis, highlighting the urgent need for innovative treatment options that can improve their outcomes,' said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. 'The acceptance of this NDA is a crucial step in our ongoing efforts to explore and evaluate various therapeutic strategies for AML through our comprehensive clinical trials. Our dedicated teams at Kyowa Kirin and Kura are fully committed to working tirelessly to ensure that, once approved, ziftomenib is made available to AML patients as quickly as possible. We recognize the importance of this endeavor and are excited about the possibility of making a meaningful impact on the lives of those affected by this challenging disease.' The KOMET-001 registration-directed trial is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib, the only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the FDA for treatment of R/R NPM1-mutant AML. In addition to BTD, ziftomenib has received Fast Track and Orphan Drug Designations. The full data analyses from the KOMET-001 trial of ziftomenib in R/R NPM1-m AML patients have been selected for oral presentation on Monday, June 2nd at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, and an encore presentation is planned at the 2025 European Hematology Association (EHA) Congress. About -Mutant AML AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately 30% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A, and IDH1/2, with prognosis heavily influenced by the presence of such co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line, and 3.5 months following the 4th line1. There are currently no FDA-approved therapies targeting NPM1-m AML. About Ziftomenib Ziftomenib is a potent and selective, oral, investigational menin inhibitor currently in development for the treatment of genetically defined AML patients with high unmet need. In April 2024, ziftomenib received BTD from the FDA for the treatment of adult patients with R/R AML with an NPM1 mutation based on data from Kura's KOMET-001 clinical trial. Additional information about clinical trials for ziftomenib can be found at About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company's pipeline consists of small molecule drug candidates designed to target cancer signaling pathways. In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib, a menin inhibitor, for AML and other hematologic malignancies. Enrollment in KOMET-001, a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-m AML, has been completed, and in the second quarter of 2025, the companies announced submission of an NDA for ziftomenib for the treatment of adult patients with R/R NPM1-m AML. Kura and Kyowa Kirin are conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-m and KMT2A-rearranged AML. KO-2806, a next-generation farnesyl transferase inhibitor (FTI), is being evaluated in a Phase 1 dose-escalation trial (FIT-001) as a monotherapy and in combination with targeted therapies for patients with various solid tumors. Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial (KURRENT-HN) in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma. For additional information, please visit Kura's website at and follow us on X and LinkedIn. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin's values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at Kura Forward-Looking Statements This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of ziftomenib; the potential for ziftomenib to obtain FDA approval for the treatment of patients with NPM1-m AML, and the anticipated timing of such FDA approval; and the potential launch of ziftomenib. Factors that may cause actual results to differ materially from those indicated by these forward-looking statements include the risk that Kura may not be able to successfully demonstrate the safety and/or efficacy of its product candidates, including ziftomenib; the risk that Kura may not obtain approval to market its product candidates, including ziftomenib, or that such approval may be delayed; the risk that the collaboration with Kyowa Kirin is unsuccessful; and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words 'may,' 'will,' 'would,' 'could,' 'should,' 'believes,' 'estimates,' 'projects,' 'promise,' 'potential,' 'expects,' 'plans,' 'anticipates,' 'intends,' 'continues,' 'designed,' 'goal,' or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the Securities and Exchange Commission, which are available at Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Kura Contacts Investors: Patti Bank Managing Director(415) Media:media@ Kyowa Kirin Contacts Investors: Ryohei Kawaiir@ Media, Global:Wataru Suzuki media@ in to access your portfolio
Yahoo
14-05-2025
- Business
- Yahoo
Kura Oncology and Kyowa Kirin Announce Combination Data for Ziftomenib in Oral Presentation at the 2025 European Hematology Association (EHA) Congress
– Ziftomenib combined with 7+3 in 1L NPM1-m or KMT2A-r AML patients selected for oral presentation on Thursday, June 12th – – Updated dataset to be presented in oral presentation at EHA2025 Congress – SAN DIEGO and TOKYO, May 14, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, 'Kura') and Kyowa Kirin Co., Ltd. (TSE: 4151, 'Kyowa Kirin') today announced that an abstract highlighting clinical data from the KOMET-007 combination trial of ziftomenib, a once-daily, oral investigational menin inhibitor, has been accepted for presentation at the upcoming 2025 European Hematology Association (EHA) Congress, to be held in Milan, Italy, from June 12-15, 2025. KOMET-007 is a multicenter Phase 1 trial of ziftomenib in combination with standards of care, including cytarabine plus daunorubicin (7+3) and venetoclax/azacitidine (ven/aza), in patients with NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML). The data presented at EHA will be from the Phase 1a dose-escalation and Phase 1b dose-expansion portions of the trial, in the cohort evaluating ziftomenib in combination with 7+3 in newly diagnosed patients with AML. 'The latest findings from the KOMET-007 trial underscore the potential of the combination of ziftomenib with intensive chemotherapy for newly diagnosed patients, strengthening our confidence in its role as a potential treatment option for a broad segment of the AML community,' said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. 'The Phase 1a/b KOMET-007 trial positions us to further evaluate this combination, and its potential to expand treatment options for AML patients, in the upcoming pivotal Phase 3 KOMET-017 trial.' In addition to the oral presentation, two abstracts for the KOMET-001 and KOMET-017 trials have been accepted for an encore presentation and publication, respectively. Session titles and information for all three abstracts are listed below and are now available on the website. Updated data from the published abstract for KOMET-007 will be disclosed during the oral presentation. Ziftomenib Combined with Intensive Induction (7+3) in Newly Diagnosed -m or -r Acute Myeloid Leukemia (AML): Updated Phase 1a/b Results from KOMET-007Session: s411. Menin inhibitors and venetoclax-based regimens in AML treatmentDate and Session Time: Thursday, June 12, 2025; 5:00PM - 6:15PM CESTLocation: Allianz MiCo, Milano Convention Centre, AuditoriumPublication Number: S136 Ziftomenib in Relapsed/Refractory -Mutant Acute Myeloid Leukemia: Phase 1b/2 Clinical Activity and Safety Results from the Pivotal KOMET-001 Study Session: Poster Session 1Date and Time: Friday, June 13, 2025; 6:30 PM - 7:30 PM CESTLocation: Allianz MiCo, Milano Convention Centre, Poster HallPublication Number: PF473 Registrational Phase 3 Study of Ziftomenib in Combination with Non-Intensive or Intensive Chemotherapy for Newly Diagnosed -m and/or -r Acute Myeloid Leukemia (AML): The KOMET-017 Trial Online Publication OnlyPublication Number: PB2573 Copies of the presentations will be available on Kura's website at following presentation at the meeting. About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company's pipeline consists of small molecule drug candidates designed to target cancer signaling pathways. Ziftomenib, a once-daily, oral menin inhibitor, is the first and only investigational therapy to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory (R/R) NPM1-m AML. In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib for AML and other hematologic malignancies. Enrollment in KOMET-001, a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-m AML, has been completed, and in the second quarter of 2025, the companies announced submission of a New Drug Application for ziftomenib for the treatment of adult patients with R/R NPM1-m AML. Kura and Kyowa Kirin are conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-m and KMT2A-r AML. KO-2806, a next-generation farnesyl transferase inhibitor (FTI), is being evaluated in a Phase 1 dose-escalation trial (FIT-001) as a monotherapy and in combination with targeted therapies for patients with various solid tumors. Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial (KURRENT-HN) in combination with alpelisib for patients with PIK3CA-dependent HNSCC. For additional information, please visit Kura's website at and follow us on X and LinkedIn. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin's values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at Kura Forward-Looking Statements This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of ziftomenib; potential benefits of combining ziftomenib with intensive chemotherapy and the expected timing and presentation of results and data from clinical trials. Factors that may cause actual results to differ materially include the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Kura may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings, and other interactions with regulatory bodies, risks associated with reliance on third parties to successfully conduct clinical trials, the risks associated with reliance on outside financing to meet capital requirements, the risk that the collaboration with Kyowa Kirin is unsuccessful, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words 'may,' 'will,' 'would,' 'could,' 'should,' 'believes,' 'estimates,' 'projects,' 'promise,' 'potential,' 'expects,' 'plans,' 'anticipates,' 'intends,' 'continues,' 'designed,' 'goal,' or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the Securities and Exchange Commission, which are available at Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Kura Contacts Investors: Patti Bank Managing Director(415) Media:media@ Kyowa Kirin Contacts Investors: Ryohei Kawaiir@ Media, Global:Wataru Suzuki, media@ in to access your portfolio
Yahoo
27-02-2025
- Business
- Yahoo
Kura Oncology Inc (KURA) Q4 2024 Earnings Call Highlights: Strategic Advances and Financial Growth
Collaboration Revenue: $53.9 million for Q4 2024, compared to none for Q4 2023. Research and Development Expenses: $52.3 million for Q4 2024, up from $32.5 million for Q4 2023. General and Administrative Expenses: $24.1 million for Q4 2024, compared to $14.2 million for Q4 2023. Net Loss: $19.2 million for Q4 2024, compared to a net loss of $42.8 million for Q4 2023. Non-Cash Share-Based Compensation Expense: $8.6 million for Q4 2024, compared to $7.2 million for Q4 2023. Cash, Cash Equivalents, and Short-Term Investments: $727.4 million as of December 31, 2024, including a $330 million upfront payment from Kyowa Kirin, compared to $424 million as of December 31, 2023. Warning! GuruFocus has detected 2 Warning Sign with KURA. Release Date: February 26, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Kura Oncology Inc (NASDAQ:KURA) is poised to submit its first NDA for ziftomenib, a potentially best-in-class menin inhibitor for relapsed/refractory NPM1-mutant AML. The company has reached alignment with both the FDA and EMA on potential accelerated approval pathways for ziftomenib in frontline AML. Kura Oncology Inc (NASDAQ:KURA) reported positive top-line results from the KOMET-001 Phase II trial, achieving its primary endpoint with a 20% to 30% CR/CRh rate. The company is well-financed, with cash, cash equivalents, and short-term investments totaling $727.4 million, sufficient to fund operations into 2027. Kura Oncology Inc (NASDAQ:KURA) has a strong pipeline with multiple clinical data updates expected throughout the year, including the initiation of the KOMET-015 trial in GIST and the KOMET-017 trials in AML. The anticipated top-line results for the MRD-negative CR accelerated endpoint in the intensive chemotherapy setting are not expected until 2028, which may be seen as a long timeline for investors. Research and development expenses increased significantly to $52.3 million in Q4 2024 from $32.5 million in Q4 2023, indicating rising costs. General and administrative expenses also rose to $24.1 million in Q4 2024 from $14.2 million in Q4 2023, reflecting higher operational costs. Despite positive developments, the net loss for Q4 2024 was $19.2 million, although this was an improvement from a $42.8 million loss in Q4 2023. There is potential uncertainty regarding the acceptance of MRD-negative CR as a surrogate endpoint by global health authorities, which could impact regulatory approval timelines. Q: Troy, you guided to the Phase III top-line in frontline intensive AML setting in 2028. Just curious what assumptions went into that guidance? And anything you can share on the trial size, that plan or enrollment timeline? A: Yes, Li, thanks for the question. We thought it was important because we've been getting questions from both analysts and investors to give people some idea of the timeline, particularly now that we've gained alignment with both FDA and EMA, but specifically with FDA on pathways to accelerated approval in the U.S. and we feel comfortable. We think we're being conservative, but we feel comfortable saying that we think we can have top-line results for that accelerated approval endpoint in 2028. We'll give you more detail on the trial size, the powering, et cetera, as we get a little bit closer to the trials actually going live and being posted on Q: And then just wondering what the remaining items that you may need to work through before the NDA submission. Any notable feedback from the pre-NDA meeting? A: No. I mean, that's -- thank you for the question. I don't know if at the time we made that announcement a few weeks ago, if that -- if people sort of read over that. We were deliberate actually in not saying anything until after we've had the pre-NDA meeting. And the team -- Mollie and the team did a phenomenal job of again gaining alignment with the agency on all the aspects of the submission. Clearly, the clinical data is always the long pole in the tent, and that's what is driving the timing of the submission at this point. But given that we have BTD and given that we've had pretty constructive engagements not only on the monotherapy, but now in the combination setting with FDA, we feel like we're in good shape. And again, we're guiding to a second quarter submission. The team is doing everything in its power to accelerate those timelines. But so far, I think we have everything we need and we've gotten very -- it's been a very constructive series of interactions with the agency. Q: So the first question I have is that when we look at the commercial opportunities for ziftomenib, I guess, the most important variable is the treatment duration. Could you share the reason behind your confidence that frontline AML patients will be able to stay on the treatment for more than a year? A: Yes. Let me actually -- Yen-Der, thank you for the question. I'm actually going to do this in 2 parts. Maybe Mollie can speak to how we've informed the duration of treatment from our experience with KOMET-007. And then I'll ask -- after she answers, I'll ask Brian to comment on how does that translate into our assumptions around the commercial potential in the frontline. Mollie, if you would? Sure. As Troy alluded to, the 007 trial has been highly informative to how we handle designing and our expectations for the 017 trial and for use in the frontline in general. I think initially, we thought that, especially with the intensive chemo setting, the use would be some induction, some consolidation, then the patient will go off to transplant and potentially some proportion would come back after transplant, post consolidation maintenance. What we're seeing is incredibly different, especially for the NPM1 patients that make up such a large majority of the patient population. These patients are coming on doing induction, consolidation and post-continuation consolidation immediately with few trips to transplant, except for the KMT2As who get to transplant whenever possible and then almost universally either have returned or intend to return to post-consolidation, post-transplant maintenance. Similarly, with the ven/aza, we have patients that are staying on for very prolonged periods of time on treatment in the frontline even when -- or if the backbone drops away or is decreased in intensity or used a little bit differently from when it's used prior to gaining control of disease. So we're seeing good prolonged use in each of these that was probably more significant than what we initially thought. But with that, Brian, I'll turn it over to you to how you see that translating. Sure. And based on the assumptions that we are observing -- or what we've observed in the trial, like Mollie said, we do think there's a tremendous potential that menin inhibitors could transform the treatment of AML with patients being able to get on therapy, stay on therapy for a long period of time. When you think about our opportunities with -- and as we've stated that we think this could be -- as Troy said, it could be a $7 billion potential market. If you're able to get patients to receive menin inhibitor for 18 to 24 months, and there could be potential for longer than that, but if you think between that 18 to 24 months in the FIT intensive chemo population and then 18 months plus within the non-intensive, that gives you a significant duration that we think really could reflect that transformative potential and really kind of align with what we've seen in some other markets like multiple myeloma where long duration of treatments with IMiDs have really transformed the market and also the outcomes for patients. Q: Congrats on the progress. I was curious, as you look to the additional dose expansion data from the 007 study, what in your view would be encouraging from the 7+3 combo as you start to expand into the non-adverse risk group? Specifically in terms of MRD negativity, what gives you -- what would give you confidence that addition of zifto would ultimately give you a survival benefit? A: Yes. Jason, thanks for the question. I'm going to ask Mollie to speak to it. But Molly, maybe you can take a minute and help folks understand how we think about the MRD negativity and sort of what the standard is as you answer Jason's question. Absolutely. So what we understand, obviously, we're part of a consortium of a large amount of pharmaceutical companies that are really looking into this MRD negativity for this patient population. And we have access, therefore, to both published and unpublished data from those other pharmaceutical companies as well as from some of the key opinion leaders that are really advancing the MRD as an endpoint in AML. So we understand that a good outcome for these patients with the current treatments available would be to see about a 40% to 45% MRD negativity in the bone marrow. And that's for the better performing patients, so your NPM1s, for instance. So we would expect that any incremental increase in that would be due to the influence of having a targeted agent in the mix as well and maybe getting into the 50s and 60% MRD negativity rate would be encouraging for translation into the overall survival as well. So that's the way we're thinking about our data as we continue to look at these patients enrolling. Obviously, you've seen the patients do very well from a response perspective. So I wouldn't expect to see much difference in that. They're going to continue to do well. But it's -- as you're pointing to, the MRD that is what we're looking at to make sure we're headed in the right For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
