Efficacy

Efficacy of Onureg (azacitidine) in Leukemia

Onureg (azacitidine) is a hypomethylating agent approved by the FDA for the continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy. The efficacy of Onureg was demonstrated in a randomized, controlled trial where patients treated with Onureg had a statistically significant improvement in overall survival compared to those receiving a placebo. The median overall survival was 24.7 months for patients on Onureg versus 14.8 months for the placebo group, indicating a clear benefit in extending life expectancy for these AML patients.

Furthermore, the trial showed that Onureg also extended the duration of remission. The median relapse-free survival was 10.2 months for patients treated with Onureg compared to 4.8 months for those on placebo. This suggests that Onureg can effectively maintain remission in AML patients who are in a post-remission state but are not candidates for intensive curative therapy, such as a stem cell transplant.

Efficacy of Lumoxiti (moxetumomab pasudotox) in Leukemia

Lumoxiti (moxetumomab pasudotox) is an anti-CD22 recombinant immunotoxin 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. The efficacy of Lumoxiti was evaluated in a single-arm, multicenter clinical trial. The trial's results showed that 75% of patients achieved a durable complete response, which is a complete remission that lasts for more than 180 days. This high rate of durable complete response highlights Lumoxiti's effectiveness in treating HCL, a rare form of leukemia.

Additionally, the overall response rate (ORR), which includes both complete and partial responses, was observed to be 80%. This indicates that the majority of patients treated with Lumoxiti experienced a reduction in their disease burden. The median duration of response had not been reached at the time of the data cut-off, suggesting that the responses induced by Lumoxiti can be long-lasting. This is particularly significant for patients with HCL who have limited treatment options after failure of initial therapies.