Efficacy

Besponsa (Inotuzumab Ozogamicin) Efficacy in Leukemia

Besponsa (inotuzumab ozogamicin) is a targeted therapy used in the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). This monoclonal antibody drug conjugate specifically targets the CD22 antigen expressed on the surface of B cells. Once bound to CD22, the drug is internalized by the cell, releasing a cytotoxic agent that induces cell death. Clinical trials have demonstrated the efficacy of Besponsa in inducing complete remission in a significant proportion of patients. These studies have shown that patients treated with Besponsa have higher remission rates and longer progression-free survival compared to those who received standard therapy.

The INO-VATE ALL trial, a key study that led to the approval of Besponsa, compared the drug to standard chemotherapy. The results indicated that a greater number of patients achieved complete remission with Besponsa than with chemotherapy. Additionally, the median duration of remission was notably longer for patients treated with Besponsa, highlighting its efficacy in this patient population. However, it is important to note that while Besponsa can be effective, it is not curative and is associated with a risk of severe side effects, which must be balanced against its potential benefits.

Onureg (Azacitidine) Efficacy in Leukemia

Onureg (azacitidine) is an oral hypomethylating agent approved 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. Azacitidine works by inhibiting DNA methyltransferases, leading to the reactivation of silenced genes that can suppress tumor growth. The efficacy of Onureg in AML was demonstrated in the QUAZAR AML-001 clinical trial, which showed a statistically significant improvement in overall survival for patients taking Onureg as maintenance therapy compared to those who received a placebo.

The QUAZAR AML-001 trial highlighted that patients treated with Onureg had a median overall survival of 24.7 months, compared to 14.8 months for patients on the placebo. Furthermore, the study also showed a delay in relapse for patients treated with azacitidine, suggesting a potential benefit in maintaining remission and extending survival. These results indicate that Onureg can be an important treatment option for AML patients who are in remission but are not candidates for intensive curative therapies, such as stem cell transplantation.