Chronic myelogenous leukemia: New drug Scemblix overcomes drug resistance

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Chronic myelogenous leukemia welcomes new drug Scemblix

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Chronic myelogenous leukemia: New drug Scemblix overcomes drug resistance. Overcome the problem of targeted therapy drug resistance! 

Chronic myelogenous leukemia is one of the more common types of leukemia, mainly manifested as anemia, repeated infections, and splenomegaly. Current treatment drugs are resistant.

Recently, the US FDA approved Scemblix (Asciminib) for the treatment of chronic myeloid leukemia (CML) with two different indications:

1. Accelerate the approval of Scemblix for adult patients with Philadelphia chromosome-positive CML chronic phase (Ph+CML-CP) who have received two or more tyrosine kinase inhibitors (TKI) in the past;

2. Fully approved Scemblix Ph+CML-CP adult patients with T315I mutation.

The drug is the first FDA-approved therapy to combine with the ABL myristoyl pocket, bringing an important treatment option for patients who are resistant or intolerant to currently available TKI therapies.

Chronic Myeloid Leukemia (CML)

Chronic myelogenous leukemia (CML) is one of the more common types of leukemia, referred to as chronic myeloid, which is a malignant tumor that affects the blood and bone marrow.

The disease progresses slowly, mainly manifested as anemia, repeated infections, splenomegaly, and a certain tendency to bleeding.

The cause of CML is still unclear, but it is closely related to the Philadelphia chromosome. About 90-95% of patients have the Philadelphia chromosome.

At present, tyrosine kinase inhibitors (TKI) are the main drugs for the treatment of chronic particles and require long-term medication; other therapies include chemotherapy, interferon, and allogeneic hematopoietic stem cell transplantation.

However, with the deepening of TKI treatment, many patients are resistant or intolerant to currently available TKI therapies, and continuous use of available TKIs will also increase the failure rate of treatment.

In an analysis of CML patients who were previously treated with two TKIs, about 55% of patients reported that they were intolerant to the previous treatment.

In addition, a second-line joint analysis showed that up to 70% of patients failed to achieve a major molecular response (MMR) within two years of follow-up. Patients with T315I mutations are resistant to most available TKIs and have an increased risk of disease progression. These patients urgently need better treatments.

Innovative STAMP inhibitor: Scemblix

Scemblix is ​​an inhibitor therapy specifically targeting ABL myristoyl pocket (STAMP). This mechanism helps to solve the drug resistance of CML patients who have previously received two or more TKI treatments.

Moreover, the drug can overcome the defective BCR-ABL1 gene mutation, which is related to the overproduction of leukemia cells. In addition, in preclinical studies, Scemblix has also been shown to limit off-target activity.

In February of this year, the FDA granted the drug the title of breakthrough therapy.

At present, Scemblix is ​​in clinical trials in other patients who are resistant or intolerant to currently available TKI therapies. In a phase 3 clinical trial called ASC4FIRST, Scemblix’s potential as a first-line therapy is evaluated.

The new drug has significant advantages, and the adverse reaction rate is 1/3 lower!

This approval is based on the positive results of the Phase III ASCEMBL trial and a Phase I study. In the pivotal Phase III ASCEMBL trial.

The results show that:

• At the 24th week, compared with the control group Bosutinib (Bosutinib, Bosulif), the Scemblix group has a clinically significant advantage in the rate of major molecular response (MMR), almost doubled, the treatment group and the control group The MMR is 25.5% VS 13.2%;
• In addition, the number of patients in the Scemblix treatment group (n=156) who stopped treatment due to adverse reactions was 1/3 (7% VS 25%) of the control group (n=76).

At present, there are still many patients with chronic particles that do not respond adequately to at least two available treatments and cannot tolerate adverse reactions. The approval of Scemblix has the potential to change the standard therapy for slow granular particles, bringing new hope for the treatment of such patients.

Reference materials:

https://pipelinereview.com/index.php/2021103079562/Small-Molecules/FDA-approves-Novartis-Scemblix-asciminib-with-novel-mechanism-of-action-for-the-treatment-of-chronic-myeloid-leukemia.html

(source:internet, reference only)

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