Aprea Therapeutics (APRE.Q) today announced positive results from its Phase 2 trial evaluating eprenetapopt with azacitidine for post-transplant maintenance therapy in patients with TP53 mutant MDS and AML.

“The post-transplant RFS and OS data with eprenetapopt and azacitidine maintenance therapy in these very difficult-to-treat TP53 mutant MDS and AML patients are incredibly exciting,” said trial principal investigator Asmita Mishra, M.D., of the H. Lee Moffitt Cancer Center and Research Institute.

Who is Aprea Therapeutics?

There are a lot of terms to unpack here, but lets start by introducing Aprea Therapeutics. Aprea is a biopharmaceutical company focused on the development and commercialization of novel cancer therapeutics, some of which are intended to reactive the mutant tumor suppressor protein p53, the most commonly mutated gene in relation to cancer. For example, eprenetapopt (APR-246) is Aprea’s lead product candidate capable of restoring p53 function in TP53-mutant cells. For context, the TP53 gene provides instructions for making the protein p53, which acts as a tumor suppressor. With this in mind, individuals with a TP53 mutation may not be able to control the growth of their cells, which can lead to the development of cancer.

Phase 2 Study of APR-246 with Azacitidine

Aprea’s Phase 2 study is intended to evaluate the safety and efficacy of APR-246 with azacitidine for post-transplant maintenance therapy in patients with TP53 mutant MDS and AMLAzacitidine is an anti-cancer drug commonly used for the treatment of myelodysplastic syndrome (MDS), including a subset of patients with acute myelogenous leukemia (AML). MDS refers to a group of disorders that affect the bone marrow’s ability to produce mature red blood cells, white blood cells and platelets. Similarly, AML also impacts the bone marrow’s ability to produce healthy blood cells, however, AML can progress rapidly if not treated.


So what do TP53, MDS, and AML have in common? Well, mutations in TP53 confer the highest risk for disease progression and death among all known MDS-associated mutations. An analysis of the International Working Group for Prognosis in MDS indicated that TP53 mutations accounted for most of the adverse outcomes in patients with MDS or AML. As for treatments, the only proven cure for MDS is stem cell transplantation, however, the risk of relapse is quite high. With this in mind, APR-246 paired with azacitidine is intended to improve outcomes for patients.

“…Post-transplant maintenance therapy with eprenetapopt and azacitidine could, if approved, represent a new treatment paradigm that meaningfully improves outcomes for these patients with limited treatment options,” continued Asmita Mishra.

The Phase 2 study of APR-246 with azacitidine enrolled 33 patients. According to results, the relapse free survival (RFS) at 1 year post-transplant was 58% and the median RFS was 12.1 months. Furthermore, the overall survival (OS) at 1 year post-transplant was 79%, with a median OS of 19.3 months. To put things into perspective, prior clinical trials evaluating post-transplant outcomes have reported a 1 year post-transplant RFS of 30% and a median OS of 5-8 months. Additionally, APR-246 with azacitidine was well tolerated among patients during the clinical trial.


According to Aprea’s Q1 2021 financial results, the Company’s total assets were reduced to $80,394,665 in Q1 2021 from $92,805,219 in Q4 2020. However, Aprea’s total liabilities declined from $15,410,012 in Q4 2020 to $11,248,501 in Q1 2021. As of March 31, 2021, Aprea’s cash and cash equivalents were sitting at $77,616,074 compared to $89,017,686 on December 31, 2020. Although APR-246 has not yet been approved by the FDA, it has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS. With this in mind, Aprea would be worth keeping an eye on as the Company’s several clinical trials continue to develop.


Aprea’s share price opened at $5.35, up from a previous close of $5.07. The Company’s shares are down -2.36% and are currently trading at $4.93 as of 12:16PM ET.

Written By:

Kieran Robertson

Kieran Robertson is an experienced writer with a passion for technology, esports, and video games. He graduated from the University of Western Ontario with a Major in English Language and Literature. After graduating, Kieran worked as a freelance writer, managing his own blog and volunteering for La Maza Magazine, a magazine startup focused on collaborating with photographers, writers, models, and designers.

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Aprea Therapeutics
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