A phase 3 trial designed to evaluate eprenetapopt plus azacytidine (Vidaza) in patients with TP53-mutant positive myelodysplastic syndrome (MDS) completed full enrollment, according to Aprea Therapeutics, Inc.

With topline data expected to be seen by the end of 2020, promising efficacy outcomes could be very important for this patient population that is difficult to treat, according to Guillermo Garcia-Manero, MD, from the Department of Leukemia in the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center.

“When you give high dose chemotherapy to a patient with (TP53-positive MDS), the body is getting all of the toxicity from the chemotherapy, yet the organ or tissue you want to target — these abnormal leukemia bone marrow cells – are basically not responding to it,” he explained in interview with 

Further, Garcia-Manero discussed why TP53-mutant MDS are difficult to treat, the importance of genomic testing, and what positive results from this study could mean.

First of all, if this study is positive, this will be a major improvement in our treatment armamentarium for this very difficult disease to treat. There are other compounds that are coming. There are antibodies that target CD47 that may have activity.

This is now creating interest and more emphasis on treating these patients. The important issue for patients with MDS and AML is that they need to ask their doctor about what their chromosomes are, why they are important, and what the results are from a next generation sequencing test. In my opinion, it is mandatory. There is no way we should be treating patients with MDS/AML without some type of genomic molecular test. Now there are 4 or 5 mutations we can target in different ways. This is starting to make a huge impact in terms of how we treat our patients, how we prognosticate our patients. In terms of education, there maybe a new drug in the next year, but the key point is realizing the importance of these genomic molecular tests when assessing patients with MDS/AML.

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A phase 3 trial designed to evaluate eprenetapopt plus azacytidine (Vidaza) in patients with TP53-mutant positive myelodysplastic syndrome (MDS) completed full enrollment, according to Aprea Therapeutics, Inc.
 

Guillermo Garcia-Manero, MD

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Trial To Evaluate Eprenetapopt-Azacytidine Combo in TP53+ MDS Completes Enrollment