Quizartinib Hydrochloride

Daiichi Sankyo’s Vanflyta (quizartinib) has been recommended by the National Institute for Health and Care Excellence (NICE) for certain cases of acute myeloid leukaemia (AML). The drug has been specifically recommended for routine NHS commissioning in England and Wales as an induction, consolidation and maintenance therapy for newly diagnosed FLT3-ITD-positive disease. Approximately 18,000 people are diagnosed with AML, a type of blood cancer that starts from young white blood cells in the bone marrow, every year in Europe. FLT3-ITD mutations occur in up to 30% of all AML cases and are associated with an increased risk of relapse and shorter overall survival. Vanflyta, taken orally once daily, is the first selective FLT3 inhibitor to be approved in the UK for newly diagnosed FLT3-ITD-positive AML. NICE’s decision on the drug follows an approval from the Medicines and Healthcare products Regulatory Agency in March and was supported by positive results from the late-stage QuANTUM-First trial. Results from the study showed that Vanflyta in combination with standard induction and consolidation chemotherapy, and then continued as a maintenance monotherapy, demonstrated a 22% reduction in the risk of death compared to standard chemotherapy alone. Median overall survival was 31.9 months for patients receiving Vanflyta versus 15.1 months for those in the control arm at a median follow-up of 39.2 months. Commenting on the recommendation, Haran Maheson, head of UK oncology and vice president, Daiichi Sankyo UK, said: “We are proud to have worked with NICE and NHS England to secure the first reimbursement for [Vanflyta] following health technology assessment in Europe. “UK patients with newly diagnosed FLT3-ITD positive AML can now receive a selective targeted therapy for their disease for the first time.” Sharing a similar sentiment, Colin Dyer, chief executive of UK charity Leukaemia Care, said: “We welcome the decision by NICE to approve this treatment… Patients with FLT3-ITD mutations have an increased risk of relapse with standard treatments, so another targeted treatment option for such patients is important for clinicians to tailor treatment plans to their patients effectively.”

The global market for AML therapies is expected to be worth $6.62bn in 2031, according to a GlobalData report. Image Credit: LALAKA / Shutterstock. Massachusetts-based company Edgewood Oncology has raised $20m in Series A to advance the clinical development of its lead cancer therapy BTX-A51. The Series A funding round was led by US-based venture fund Alta Partners. Edgewood Oncology expects the funds to finance multiple clinical trials into 2026. BTX-A51 is a multi-kinase inhibitor of casein kinase 1 alpha (CK1α) and cyclin-dependent kinases 7 and 9 (CDK7 and CDK9). Edgewood acquired the rights to the therapy from the Hebrew University of Jerusalem in 2023. Edgewood is evaluating BTX-A51 in an open-label Phase I/II trial (NCT04243785) as a monotherapy and in combination with a chemotherapeutic, azacitidine, in patients with relapsed/refractory acute myeloid leukaemia (r/r AML). In Phase Ia, the BTX-A51 monotherapy showed a favourable safety profile along with encouraging antileukemic activity. The combination cohort started enrolment in December 2023. The cohort is expected to enrol up to 30 participants. The cohort aims to evaluate the response rate, safety, toxicity, and pharmacokinetics of the combination therapy of BTX-A51 and azacytidine in patients with r/r AML. See Also: Oncology in 2024: The clinical trial trends reshaping the role of CROs Avenzo Therapeutics secures $150m to progress oncology pipeline The global market for AML therapies is expected to be worth $6.62bn in 2031, according to a GlobalData report . The AML therapy pipeline consists of more than 200 drugs in early development, with the majority of drugs in development being enzyme inhibitors, according to GlobalData Pharma Intelligence Center. GlobalData is the parent company of Pharmaceutical Technology . Edgewood is also investigating BTX-A51 as a treatment for solid tumours and non-Hodgkin lymphoma, open-label Phase I trial (NCT04872166). The study expects to enrol up to 116 participants and is estimated to be completed in May 2027. The company also plans to initiate a Phase II trial evaluating BTX-A51 in breast cancer patients with a genetically defined profile in Q2 2024. Advances in AML therapies include Daiichi Sankyo ’s Vanflyta (quizartinib). In November 2023, the European Commission (EC) approved Vanflyta as an add-on therapy for newly diagnosed fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) positive AML in adults. In September 2023, Mendus’s cancer vaccine, vividencel, received fast-track designation from the US Food and Drug Administration (FDA). A month later, Sellas Life Sciences Group’s CDK9 inhibitor, SLS009, received an orphan drug designation from the FDA. AML therapies in early development include Aptose Biosciences ’ tuspetinib. The company started the Phase I/II APTIVATE trial evaluating the drug’s pharmacodynamics, tolerability, safety, and pharmacokinetics in r/r AML patients in January. In September 2023, Foghorn Therapeutics dosed the first patient in the Phase I trial evaluating the combination therapy of FHD-286 and chemotherapy in patients with r/r AML.