Efficacy

Venclyxto/Venclexta (Venetoclax) for Leukemia

Venclyxto, known as Venclexta in the United States, is a targeted therapy drug that contains the active substance venetoclax. It is primarily used in the treatment of chronic lymphocytic leukemia (CLL), a type of cancer that affects a group of white blood cells called lymphocytes. Venetoclax works by selectively inhibiting the B-cell lymphoma-2 (BCL-2) protein, which supports cancer cell survival. By blocking this protein, venetoclax helps to initiate the process of programmed cell death, known as apoptosis, in the leukemia cells.

The efficacy of venetoclax has been demonstrated in several clinical trials. For instance, in a pivotal phase III clinical trial known as the CLL14 study, venetoclax, in combination with obinutuzumab, showed a significant improvement in progression-free survival (PFS) compared to patients who received chlorambucil plus obinutuzumab, a standard treatment. The venetoclax combination reduced the risk of disease progression or death by 65% compared to the control group. Additionally, the overall response rate (ORR) was higher in the venetoclax-treated group, indicating a greater number of patients had a reduction in their leukemia.

Lumoxiti (Moxetumomab Pasudotox) for Leukemia

Lumoxiti, with the active ingredient moxetumomab pasudotox, is an innovative treatment for hairy cell leukemia (HCL), a rare subtype of chronic leukemia. Moxetumomab pasudotox is a recombinant immunotoxin that targets CD22, a protein commonly found on the surface of HCL cells. By binding to CD22, the drug is internalized by the cancer cells, and its toxin component is released, leading to cell death. This targeted approach allows for the selective destruction of HCL cells while sparing healthy cells.

The efficacy of Lumoxiti was evaluated in a pivotal multicenter, single-arm, open-label clinical trial involving patients with relapsed or refractory HCL. The results demonstrated a durable complete response (CR) in a significant proportion of patients. Specifically, 30% of patients achieved a CR with a duration of response lasting more than six months. Furthermore, the overall response rate, including partial responses, was observed to be 75%. These findings indicate that Lumoxiti is an effective treatment option for patients with HCL who have limited treatment options and require a new therapeutic approach.