Aprea Therapeutics Completes Full Enrollment of Phase 3 Clinical Trial in TP53 Mutant Myelodysplastic Syndromes (MDS)
- Topline data expected by year-end 2020
- Applications for US and EU regulatory approval planned for 2021
“Completion of enrollment in this Phase 3 trial is an important milestone for Aprea and our first-in-class p53 reactivator, eprenetapopt,” said
The randomized, controlled pivotal Phase 3 trial is designed to evaluate eprenetapopt with azacitidine compared with azacitidine alone as front-line therapy in intermediate, high, and very high risk TP53 mutant MDS patients. The multi-center trial enrolled 154 patients, randomized 1:1 to the two arms with a primary endpoint of CR rate. The trial has 90% power with P-value < 0.05 to detect a difference in CR rates of 50% in the eprenetapopt-containing arm versus 25% in the azacitidine-only control arm.
About Aprea Therapeutics, Inc.
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About p53 and APR-246 (eprenetapopt)
The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.
APR-246 (eprenetapopt) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – and thereby induce programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with APR-246 in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with APR-246 has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.
Myelodysplastic syndromes (MDS) represent a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in up to 20% of MDS and AML patients and are associated with poor overall prognosis. There are no currently approved therapies specifically for TP53 mutant MDS or AML patients.
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Source: Aprea Therapeutics, Inc.
Scott M. CoianteSr. Vice President and Chief Financial Officer 617-463-9385 Gregory A. KorbelVice President of Business Development 617-463-9385
Source: Aprea Therapeutics