Acute Myeloid Leukemia with FLT3/ITD Mutation

Daiichi Sankyo’s quizartinib is indicated for use as a first-line treatment in adults with acute myeloid leukaemia. Credit: Nemes Laszlo / Shutterstock.com. The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation to Daiichi Sankyo for quizartinib (Vanflyta) for use as a first-line treatment in adults with acute myeloid leukaemia (AML) with a FLT3-ITD mutation. The decision allows for the oral administration of quizartinib in combination with chemotherapy – a significant development in the treatment of AML. Taken once daily, quizartinib targets tyrosine kinases to hinder the growth of abnormal bone marrow cells. The treatment duration and dosage are determined by the prescribing physician and may continue post-chemotherapy. The approval is backed by a double-blind, randomised, placebo-controlled Phase III clinical trial in 539 adult subjects aged between 18 and 75 years with newly diagnosed FLT3-ITD positive AML. See Also: MHRA approves Marinus’ ganaxolone for CDKL5 deficiency disorder Innovent’s ROS1 TKI is one step closer to winning the Chinese NSCLC market The study demonstrated a statistically significant improvement in overall survival (time from randomisation to death from any cause) for patients who received quizartinib. Patients receiving quizartinib had a median overall survival of 31.9 months compared to 15.1 months for those receiving a placebo. Abnormal liver enzymes, low blood platelet and white blood cell levels, anaemia, diarrhoea, nausea, stomach pain and headache were the most common side effects linked to the trial. MHRA Healthcare Quality and Access interim executive director Julian Beach stated: “ Our key priorities are keeping patients safe and enabling their access to high quality, safe and effective medical products. “We’re assured that the appropriate regulatory standards for the approval of this medicine have been met. As with all medicines, we will keep its safety under close review.” In another development, the MHRA also gave marketing authorisation to Pfizer for etrasimod (Velsipity) for the treatment of moderately to severely active ulcerative colitis. Etrasimod is prescribed as a long-term treatment and should be initiated only under a doctor’s supervision. The approval is supported by two clinical studies, ELEVATE UC 52 and ELEVATE UC 12, indicating its efficacy for patients where standard treatments were inadequate. The UK National Institute for Health and Care Excellence (NICE) has also published a final guidance recommending etrasimod for ulcerative colitis. With the NICE recommendation, more than 25,000 people in England will be eligible to receive the treatment. The latest development comes after the MHRA approved Marinus Pharmaceuticals ’ ganaxolone, marketed as Ztalmy, an anti-seizure medication for cyclin-dependent kinase-like 5 (CDLK5) deficiency disorder .

SAN MATEO, Calif. and CAMBRIDGE, Mass., Dec. 04, 2023 (GLOBE NEWSWIRE) -- Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer, today announced that Jorge DiMartino M.D., Ph.D., chief medical officer and executive vice president of clinical development, and Charles Lin, Ph.D., senior vice president of TRN mapping will participate in a fireside chat at the H.C. Wainwright 4th Annual Precision Oncology Virtual Conference on Wednesday, December 6, 2023 at 11:00 a.m. ET. A live audio webcast of the event will be available on the Investors and Media section of the Kronos Bio website at www.kronosbio.com. A replay of the webcast will be available for 30 days following the event. About Kronos Bio, Inc. Kronos Bio is a biopharmaceutical company that is advancing two investigational compounds in clinical trials for patients with cancer. The Company is developing the CDK9 inhibitor KB-0742 as a treatment for MYC-amplified solid tumors and other transcriptionally addicted solid tumors and lanraplenib, a next-generation SYK inhibitor, for patients with FLT3-mutated acute myeloid leukemia. The Company also has a maturing discovery pipeline from which they are on track to announce a development candidate in early 2024 for their IRF4 modulator program for use in multiple myeloma. The Company’s scientific focus is on developing medicines that target the deregulated transcription that is the hallmark of cancer and other serious diseases. Kronos Bio is based in San Mateo, Calif., and has a research facility in Cambridge, Mass. For more information, visit https://www.kronosbio.com/ or follow the Company on LinkedIn. Media Contact:Sarah ConnorsVice President of Corporate Affairs, Kronos Bio857-290-7305sconnors@kronosbio.com Investor Contact:Margaux BennettExecutive Director, Business Development and Investor Relations, Kronos Bio650-781-5026mbennett@kronosbio.com

Annual R&D Day to provide overview of data and updated strategy across Daiichi Sankyo R&D pipeline BASKING RIDGE, N.J.--(BUSINESS WIRE)-- Daiichi Sankyo (TSE: 4568) will present new clinical research across its oncology portfolio in multiple types of solid and blood cancers at the 2023 ESMO Asia Congress (#ESMOAsia23), San Antonio Breast Cancer Symposium (#SABCS23) and American Society of Hematology (#ASH23) Annual Meeting prior to its annual R&D Day. Presentation highlights include the first presentation of results from three trials from Daiichi Sankyo’s DXd ADC and hematology portfolio, which include the DESTINY-Gastric06 phase 2 trial of ENHERTU® (trastuzumab deruxtecan) in Chinese patients with previously treated HER2 positive locally advanced/metastatic gastric cancer or gastroesophageal junction adenocarcinoma at ESMO Asia, the DESTINY-Breast08 phase 1b trial of ENHERTU in combination with anastrozole or fulvestrant in patients with HER2 low metastatic breast cancer at SABCS and the VALENTINE-PTCL01 phase 2 trial of valemetostat in patients with relapsed/refractory peripheral T-cell lymphoma (PTCL) at ASH. “Our goal is to push the boundaries of science to change the way cancer is treated,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “These data reinforce the breadth and depth of our oncology pipeline and the important progress we are making in developing innovative medicines and exploring combination strategies for patients across a wide range of cancers, including breast, lung, gastric, acute myeloid leukemia and peripheral T-cell lymphoma.” Subgroup analyses of the TROPION-Lung05 phase 2 trial of datopotamab deruxtecan (Dato-DXd) in Asian patients with non-small cell lung cancer (NSCLC) with actionable genomic alterations and the DESTINY-Lung02 phase 2 trial of ENHERTU in Asian patients with HER2 mutant NSCLC will be presented at ESMO Asia. Additional subgroup analyses of the QuANTUM-First phase 3 trial in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia (AML), which formed the basis of recent approvals of VANFLYTA® (quizartinib) in the EU, Japan and U.S. will be presented at ASH. Daiichi Sankyo will hold its annual R&D Day for investors, analysts and media on Monday, December 11 at 5:30 - 7:00 pm EST/Tuesday, December 12 at 7:30 - 9:00 am JST. Company executives will provide highlights of Daiichi Sankyo’s research data presented at SABCS and ASH, as well as updates on the company’s R&D strategy. ESMO Asia Data Encore presentations of the TROPION-Lung01 phase 3 trial and TROPION-Lung05 phase 2 trial of datopotamab deruxtecan (Dato-DXd) as well as the DESTINY-PanTumor02 phase 2 trial of ENHERTU will be presented at ESMO Asia from December 1 - 3 in Singapore, along with other data, including: Presentation Title Presenter Abstract Presentation ENHERTU (trastuzumab deruxtecan; T-DXd) Lung Trastuzumab deruxtecan (T-DXd) in Asian patients with human epidermal growth factor receptor 2 (HER2; ERBB2) mutant (HER2m) metastatic non–small cell lung cancer: subgroup analysis of DESTINY-Lung02 Y. Goto 510MO Mini-Oral Session December 2, 2023 9:00 – 10:30 am SGT Pan Tumor Trastuzumab deruxtecan (T-DXd) for pretreated patients with HER2 expressing solid tumors: primary analysis from the DESTINY-PanTumor02 study D. Oh 74MO Mini-Oral Session December 1, 2023 10:45 am – 12 :15 pm SGT Gastric Trastuzumab deruxtecan (T-DXd) in Chinese patients with previously treated HER2 positive locally advanced/ metastatic gastric cancer or gastroesophageal junction adenocarcinoma: primary efficacy and safety from the phase 2 single-arm DESTINY-Gastric06 trial L. Shen 172P Poster Presentation December 2, 2023 Datopotamab deruxtecan (Dato-DXd) Lung Datopotamab deruxtecan (Dato-DXd) vs docetaxel in previously treated advanced/metastatic non-small cell lung cancer: results of the randomized phase 3 study TROPION-Lung01 M. Ahn 509MO Mini-Oral Session December 2, 2023 9:00 – 10:30 SGT TROPION-Lung05: datopotamab deruxtecan (Dato-DXd) in previously treated non-small cell lung cancer with actionable genomic alterations S. Kitazono 518MO Mini-Oral Session Sunday, December 3 9:00 – 10:30 am SGT TROPION-Lung05: datopotamab deruxtecan (Dato-DXd) in Asian patients with previously treated non-small cell lung cancer with actionable genomic alterations Y. Goto 552P Poster Presentation December 2, 2023 SABCS Data Highlights of Daiichi Sankyo data across several subtypes of breast cancer to be presented at SABCS from December 5 - 9 in San Antonio, Texas, include: Presentation Title Author Abstract Presentation ENHERTU (trastuzumab deruxtecan; T-DXd) HER2 Low Breast Trastuzumab deruxtecan (T-DXd) in combination with anastrozole or fulvestrant in patients with HER2 low HR+ advanced/metastatic breast cancer: a phase 1b, open-label, multicenter, dose-expansion study (DESTINY-Breast08) K. Jhaveri RF02-03 Rapid-Fire Mini-Oral December 7, 2023 12:00 – 12:45 pm CST An open-label, interventional multicenter study of trastuzumab deruxtecan monotherapy in patients with unresectable and/or metastatic HER2 low or HER2 immunohistochemistry 0 breast cancer: DESTINY-Breast15 S. Modi PO2-19-06 Poster Presentation December 6, 2023 5:00 – 7:00 pm CST HER2 Positive Breast European real-world experience of patients with HER2+ advanced/metastatic breast cancer accessing trastuzumab deruxtecan through a named patient program: first interim analysis of EUROPA T-DXd M. De Laurentiis PO3-16-12 Poster Presentation December 7, 2023 12:00 – 2:00 pm CST Real-world experience with trastuzumab deruxtecan in patients with breast cancer: 6 month interim analysis of an all patient post marketing surveillance in Japan J. Tsurutani PO3-17-07 Poster Presentation December 7, 2023 12:00 – 2:00 pm CST Population pharmacokinetics of trastuzumab deruxtecan (T-DXd) in HER2 positive breast cancer subjects: analyses across 12 Phase 1-3 studies C. Li PO3-04-01 Poster Presentation December 7, 2023 12:00 – 2:00 pm CST Exposure-efficacy and exposure-safety analysis of trastuzumab deruxtecan in patients with advanced metastatic HER2+ breast cancer: analyses from phase 3 studies DESTINY-Breast02 and DESTINY-Breast03 C. Li PO3-04-02 Poster Presentation December 7, 2023 12:00 – 2:00 pm CST Datopotamab deruxtecan (Dato-DXd) HR Positive Breast Randomized phase 3 study of datopotamab deruxtecan vs chemotherapy for patients with previously treated inoperable or metastatic hormone receptor positive, HER2 negative breast cancer: results from TROPION-Breast01 A. Bardia GS02-01 Oral Presentation December 7, 2023 8:15 - 8:30 am CST TNBC Durvalumab + datopotamab deruxtecan in patients with PD-L1 positive advanced/metastatic triple negative breast cancer: arm 8 of the phase 1b/2, open label, platform BEGONIA study P. Schmid PO1-19-10 Poster Presentation December 6, 2023 12:00 - 2:00 pm CST ASH Data Highlights of Daiichi Sankyo data from its hematology portfolio to be presented at ASH from December 9 - 12 in San Diego, California, include: Presentation Title Presenter Abstract Presentation Valemetostat (EZHARMIA in Japan only) T-cell Lymphoma Efficacy and safety of valemetostat monotherapy in patients with relapsed or refractory peripheral T-cell lymphomas: primary results of the phase 2 VALENTINE-PTCL01 study S. Horwitz 302 Oral Presentation December 9, 2023 4:15 pm PST Valemetostat for relapsed or refractory peripheral T- cell lymphomas: primary results from a phase 1 trial E. Jacobsen 303 Oral Presentation December 9, 2023 4:30 pm PST B-cell Lymphoma Valemetostat for relapsed or refractory B-cell lymphomas: primary results from a phase 1 trial K. Izutsu 1731 Poster Presentation December 9, 2023 5:30 - 7:30 pm PST VANFLYTA (quizartinib) FLT3-ITD AML QuANTUM-First: FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD)-specific measurable residual disease (MRD) clearance assessed through induction and consolidation is associated with improved overall survival in newly diagnosed FLT3-ITD+ AML patients A. Perl 832 Oral Presentation December 11, 2023 3:30 pm PST QuANTUM-First: safety by treatment phase and by age in newly diagnosed patients with FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) positive acute myeloid leukemia (AML) H. Erba 972 Oral Presentation December 11, 2023 5:45 pm PST Patient-reported outcomes in acute myeloid leukemia patients with FLT3-ITD mutation receiving quizartinib vs. standard chemotherapy: results from the QuANTUM-First trial E. Oliva 918 Oral Presentation December 11, 2023 4:00 pm PST QuANTUM-First: clinical bridging study for FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) companion diagnostic development J. Rohrbach 4260 Poster Presentation December 11, 2023 6:00 - 8:00 pm PST Kinetics of complete remission (CR) and CR duration and its impact on overall survival (OS) and event-free survival (EFS) in QuANTUM-First P. Montesinos 4254 Poster Presentation December 11, 2023 6:00 - 8:00 pm PST About the DXd ADC Portfolio of Daiichi Sankyo The DXd ADC portfolio of Daiichi Sankyo currently consists of six ADCs in clinical development across multiple types of cancer. ENHERTU, a HER2 directed ADC, and datopotamab deruxtecan (Dato-DXd), a TROP2 directed ADC, are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck & Co., Inc., Rahway, NJ. U.S.A. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo. Designed using Daiichi Sankyo’s proprietary DXd ADC technology to target and deliver a cytotoxic payload inside cancer cells that express a specific cell surface antigen, each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. Datopotamab deruxtecan, ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan and DS-3939 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established. About Daiichi Sankyo Daiichi Sankyo is an innovative global healthcare company contributing to the sustainable development of society that discovers, develops and delivers new standards of care to enrich the quality of life around the world. With more than 120 years of experience, Daiichi Sankyo leverages its world-class science and technology to create new modalities and innovative medicines for people with cancer, cardiovascular and other diseases with high unmet medical need. For more information, please visit .