Efficacy

Inqovi (decitabine and cedazuridine) Efficacy in Leukemia

Inqovi, a combination of decitabine and cedazuridine, is a medication approved for the treatment of adult patients 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. MDS can be considered a type of leukemia, and Inqovi is used to treat these specific subtypes that are characterized by abnormal cells in the bone marrow that can lead to acute myeloid leukemia (AML).

The efficacy of Inqovi for the treatment of these conditions was demonstrated in two Phase 3 trials, where patients treated with Inqovi experienced a higher rate of complete remission or complete remission with partial hematologic recovery compared to historical controls. The combination of decitabine and cedazuridine allows for oral administration, which maintains the efficacy of decitabine similar to its intravenous form, providing a more convenient treatment option for patients.

Lumoxiti (moxetumomab pasudotox) Efficacy in Leukemia

Lumoxiti, which contains the active ingredient moxetumomab pasudotox, is a CD22-directed cytotoxin indicated for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. Hairy cell leukemia is a rare, slow-growing cancer of the blood where the bone marrow makes too many B cells (lymphocytes), a type of white blood cell that fights infection. These excess B cells are abnormal and look "hairy" under a microscope.

The efficacy of Lumoxiti was established in a single-arm, open-label, multicenter clinical trial of adult patients with relapsed or refractory HCL. The trial's primary endpoint was durable complete response (CR), defined as the maintenance of hematologic remission for more than 180 days after the achievement of CR. The results showed a significant proportion of patients achieving durable CR, indicating that Lumoxiti is an effective treatment option for this patient population. The study provided evidence that Lumoxiti can induce deep and long-lasting remissions in patients with HCL who have limited treatment options.