New Potential Combination Therapy for Acute Myelogenous Leukemia has Received Breakthrough Therapy Designation

Acute Myelogenous Leukemia

Acute myelogenous leukemia (AML) is a rare blood cancer whose prevalence increases with age. There are still extremely limited treatment options for AML and the five-year survival rate is just 27%. While chemotherapy has a small response rate, some don’t even have that option as they are ineligible for the treatment due to comorbidities. Patients with specific mutations in AML such as IDH1 also have no approved treatment options.

Argios Pharmaceuticals has been working to develop a treatment for those unable to receive chemotherapy and those who have an IDH1 mutation. They have just announced that the FDA has granted their investigational therapy TIBSOVO (an IDH1 inhibitor) Breakthrough Therapy Designation when combined with azacitidine for both of these indications of AML.

Specifically this designation is for TIBSOVO plus azacitidne for those who are at least 75 years old and have newly diagnosed AML in one of those two indications.

The Trial

The trial which led to this new designation was a Phase 1/2 study. The TIBSOVO arm was the Phase 1b section of the study. 23 patients with a median age of 76 were enrolled in the trial. They each received 500mg of the drug each day in addition to azacitidine. 78% of the participants responded to the treatment and 57% had a complete response. Additionally, the safety of the combined therapy was consistent with the safety of each drug on its own.

This designation is so exciting because it should help to expedite the development of this new potential therapy. It indicates that the FDA sees value in its potential to treat this patient population which currently has a high unmet need.

Stay tuned to hear further updates on TIBSOVO plus azacitidine. Hopefully, we will see this therapy become a new treatment option for AML patients in the near future.

You can read more about this potential therapy here.


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