Efficacy

Overview of Besponsa (Inotuzumab Ozogamicin) for Leukemia

Besponsa (inotuzumab ozogamicin) is a targeted therapy approved for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). As a monoclonal antibody conjugated to a chemotherapy drug, inotuzumab ozogamicin specifically targets the CD22 antigen on B cells and delivers its cytotoxic agent directly to the leukemic cells. Clinical trials have demonstrated significant efficacy in this patient population. The INO-VATE ALL study, a pivotal phase 3 trial, showed that patients treated with Besponsa had a higher complete remission (CR) rate and a longer progression-free survival (PFS) compared to those who received standard chemotherapy.

Efficacy Results from Clinical Trials of Besponsa

In the INO-VATE ALL trial, 80.7% of patients who received Besponsa achieved a complete remission with or without complete hematologic recovery, compared to 29.4% of patients treated with standard therapy. Moreover, the median progression-free survival for patients treated with Besponsa was significantly longer at 5.0 months, versus 1.8 months in the standard therapy group. These results indicate a substantial improvement in response rates and disease control with Besponsa in the relapsed or refractory ALL setting.

Overview of Scemblix (Asciminib) for Leukemia

Scemblix (asciminib) is a newer medication that has been approved for the treatment of chronic myeloid leukemia (CML) in adults. It is specifically indicated for patients with Philadelphia chromosome-positive CML in chronic phase who have received two or more prior tyrosine kinase inhibitors (TKIs). Asciminib is a first-in-class STAMP inhibitor that uniquely targets the ABL myristoyl pocket, providing a novel mechanism of action against the BCR-ABL1 oncoprotein that drives CML. Clinical trials have shown promising efficacy in this patient population, particularly for those who have developed resistance to or are intolerant of previous therapies.

Efficacy Results from Clinical Trials of Scemblix

In a pivotal phase 3 trial, asciminib demonstrated superior efficacy in achieving and maintaining major molecular response (MMR) compared to bosutinib, another TKI, in patients with CML who had previously been treated with two or more TKIs. The trial reported that 25.5% of patients treated with asciminib achieved MMR at 24 weeks, compared to 13.2% of patients on bosutinib. Additionally, asciminib was associated with a lower discontinuation rate due to adverse events, suggesting a favorable safety and tolerability profile alongside its efficacy in treating CML.