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Ziftomenib: A Promising New Drug For NPM1 AML
Posted: Jun 12, 2024
Ziftomenib: A Promising New Drug For NPM1 AML image

There are different subtypes of AML, and one specific type is defined by a mutation in a gene called NPM1. NPM1-mutated AML is actually the most common subtype, affecting about a third of adult AML patients, and it can lead to a poor prognosis, with only 50% of patients alive after 5 years since diagnosis.  

While there are treatment options available for AML, these can be intensive and may not be effective for everyone. Relapsed/refractory NPM1-mutated AML, where the cancer comes back or doesn't respond well to initial treatments, is a particular challenge. This is why there's a strong need for new targeted therapies specifically designed for this type of leukemia. A biopharmaceutical company named Kura Oncology is developing a new targeted therapy called ziftomenib specifically for this type of AML.

What is Ziftomenib?

Ziftomenib is a new, once-daily oral medication designed to target a specific pathway important for the survival of AML cells with the NPM1 mutation. Early data suggests good tolerability, with manageable side effects.

Promising initial results

In a preliminary study, ziftomenib showed a 35% complete remission rate, meaning the cancer cells were no longer detectable in the bone marrow. The first patient responded to treatment within 51 days, with an average follow-up of nearly 9 months.

Manageable Side Effects

The most common side effects reported in the study were diarrhea (45%), potassium deficiency (40%), nausea (30%), anemia (25%), and low platelet count (20%). These side effects are typically manageable with medication or supportive care.

Breakthrough Therapy Designation and Clinical Trials

The U.S. Food and Drug Administration (FDA) recently granted "Breakthrough Therapy Designation" to ziftomenib. This designation highlights the drug's potential to significantly improve treatment outcomes for patients with relapsed/refractory NPM1-mutated AML.

Kura Oncology has successfully completed enrollment for a clinical trial designed to evaluate the safety and efficacy of ziftomenib in patients with this specific type of AML. This late-stage trial will provide valuable data on how well the drug works and its potential side effects.

What this means for you:

While ziftomenib is still under development, these recent advancements suggest it could be a promising new treatment option for patients with NPM1-mutated AML. It's important to speak with your doctor to learn more about your specific treatment options and if participating in a clinical trial might be right for you.  

Find Recruiting AML Clinical Trials with HealthTree Cure Hub

With HealthTree's Clinical Trial Finder, you can search for open clinical trials for ziftomenib or any other new treatment for AML. You can filter by treatment, location, or keyword. Create a HealthTree Cure Hub account to start exploring!



The author Lisa Foster

about the author
Lisa Foster

Lisa Foster is a mom of 3 daughters, a puzzle lover, writer and HealthTree advocate. She believes in the mission of the foundation and the team that builds it forward. She calls Houston, Texas home. 

Thanks to our HealthTree Community for Acute Myeloid Leukemia Sponsors:

Astellas Pharma

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