Release Details
Aprea Therapeutics Presents Results From French Phase Ib/II Clinical Trial of APR-246 (Eprenetapopt) and Azacitidine in Patients with TP53 Mutant Myelodysplastic Syndromes and Acute Myeloid Leukemia at the 25th European Hematology Association Annual Meeti
- 57% CR and 75% ORR rate by IWG criteria in all evaluable MDS patients
- 12.1 months median OS in intention-to-treat MDS patients with 9.7 months median duration of follow-up
- 13.7 months median OS in patients receiving 3 or more cycles of therapy with 9.7 months duration of follow-up
As of the
“The data from this ongoing trial of eprenetapopt with azacitidine continue to be very encouraging in these most difficult-to-treat TP53 mutant MDS and AML patients, who not only have at least one TP53 mutation but the majority of whom also have high risk cytogenetic abnormalities,” said Thomas Cluzeau, M.D., co-lead investigator for the GFM trial. “We continue to observe ORR and CR rates in these patients that are substantially higher than the GFM’s experience with azacitidine monotherapy. Furthermore, with increased duration of follow-up, we now also see the emergence of highly encouraging overall survival that appears to be better than azacitidine alone or in combination with others agents in this very high-risk molecular group of patients with a TP53 mutation.”
Details of the on-demand oral presentation are as follows:
Title: APR-246 Combined with Azacitidine in TP53 Mutated Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia. A Phase 2 Study by the Groupe Francophone des Myélodysplasies (GFM)
Oral Abstract Session: Novel treatments for MDS I
Abstract: S181
About the Clinical Trial
Eligible patients in the Phase Ib/II clinical trial include hypomethylating agent (HMA) naïve, TP53 mutated MDS and AML. All enrolled patients were to receive APR-246 as a 4,500 mg fixed dose IV daily for 4 days and AZA over 7 days in 28-day cycles. The primary endpoint of the trial is CR rate.
About Aprea Therapeutics, Inc.
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About Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) represents 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.
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.
Forward-Looking Statement
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Source: Aprea Therapeutics, Inc.
Corporate Contacts:Scott M. Coiante Sr. Vice President and Chief Financial Officer 617-463-9385Gregory A. Korbel Vice President of Business Development 617-463-9385
Source: Aprea Therapeutics