Efficacy

Inqovi (decitabine and cedazuridine) for Leukemia

Inqovi, a combination of decitabine and cedazuridine, is an oral hypomethylating agent approved for the treatment of adults with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British (FAB) subtypes: refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia (CMML), as well as intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System (IPSS) groups. Clinical trials have demonstrated the efficacy of Inqovi in these conditions, which are considered a group of heterogeneous bone marrow disorders characterized by ineffective hematopoiesis leading to blood cell dysplasias and a risk of progression to acute myeloid leukemia (AML).

The efficacy of Inqovi was evaluated in two open-label, randomized, crossover trials in which the combination demonstrated similar drug exposure and safety profiles to intravenous decitabine, with the convenience of an oral administration. Patients treated with Inqovi showed hematologic improvement and transfusion independence in a subset of patients, which are important clinical endpoints in the treatment of MDS and CMML. These results support the use of Inqovi as an effective treatment option for patients with MDS and CMML, particularly for those who may not be candidates for more intensive therapies.

Vanflyta (quizartinib) for Leukemia

Vanflyta, also known as quizartinib, is a tyrosine kinase inhibitor specifically designed to target FLT3 (FMS-like tyrosine kinase 3), which is a common mutation found in acute myeloid leukemia (AML). FLT3 mutations are one of the most common genetic abnormalities in AML and are associated with a poor prognosis. Quizartinib has shown efficacy in clinical trials for the treatment of adult patients with relapsed or refractory AML who are FLT3-ITD positive. In these trials, quizartinib has been associated with a significant improvement in overall survival compared to chemotherapy.

The efficacy of Vanflyta was primarily demonstrated in a pivotal phase 3 trial, where patients with relapsed or refractory FLT3-ITD positive AML treated with quizartinib had a median overall survival that was significantly longer than those treated with salvage chemotherapy. Additionally, quizartinib has shown a higher rate of complete remission with full or partial hematologic recovery in this patient population. These results indicate that Vanflyta is an effective targeted therapy for AML patients with the FLT3-ITD mutation, offering a potential treatment option that could improve outcomes in this challenging disease state.