- 74% ORR, 66% CR rate in 24 evaluable MDS patients
- Median duration of follow-up is 6.4 months
BOSTON, Dec. 09, 2019 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein p53, presented preliminary results at the 2019 ASH Annual Meeting from its French Phase Ib/II clinical trial in MDS and AML. The trial is evaluating the safety and efficacy of APR-246 in combination with azacitidine for the treatment of patients with TP53 mutated MDS and AML. The clinical trial is sponsored by the Groupe Francophone des Myélodysplasies.
As of the data cutoff, the overall response rate (ORR) in 24 evaluable MDS patients was 74%, with a 66% complete remission (CR) rate, based on International Working Group criteria. With a median duration of follow-up of 6.4 months, the median overall survival (OS) for all enrolled patients (n=53) had not been reached. In addition, all responding patients were alive at data cutoff. Relative to baseline, mutant TP53 variant allele frequency (VAF) was significantly decreased in responding patients and undetectable in all patients who achieved a CR.
About the Clinical Trial
Eligible patients in the Phase Ib/II clinical trial include HMA naïve, TP53 mutated MDS and acute myeloid leukemia (AML). All enrolled patients were to receive APR-246 as a 4,500 mg fixed dose IV daily (days 1-4) and AZA over 7 days (days 4-10 or 4-5 and 8-12) in 28-day cycles. The primary endpoint of the trial is CR rate.
About Myelodysplastic Syndrome
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
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 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 I/II 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.
A pivotal Phase 3 clinical trial of APR-246 and azacitidine for frontline treatment of TP53 mutant MDS is ongoing. APR-246 has received Orphan Drug and Fast Track designations from the FDA for MDS, and Orphan Drug designation from the EMA for MDS, AML and ovarian cancer.
About Aprea Therapeutics
Aprea Therapeutics Inc., (NASDAQ: APRE) is a biopharmaceutical company headquartered in Boston, Massachusetts with research facilities in Stockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein p53. The Company’s lead product candidate is APR-246, a small molecule in clinical development for hematologic malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). For more information, please visit the company website at www.aprea.com.
The Company may use, and intends to use, its investor relations website at www.ir.aprea.com as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.
The Company will host a 2019 ASH Clinical Update meeting and webcast as follows:
Time and Date: Monday, December 9 at 12:00 pm ET
Location: Hyatt Regency Orlando, Rainbow Spring 2 Room, 9801 International Drive, Orlando, Florida
Webcast: The Clinical Update meeting will be webcast live and can be accessed from "Events Calendar" in the News and Events section of the company's website at Link
Presentation: The presentation will be available as a PDF on the Company’s website at Link
Certain information contained in this press release includes “forward-looking statements”, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our clinical trials and regulatory submissions. We may, in some cases use terms such as “predicts,” “believes,” “potential,” “continue,” “anticipates,” “estimates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “likely,” “will,” “should” or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward-looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies and the other risks set forth in our filings with the U.S. Securities and Exchange Commission, including our Quarterly Report on Form 10-Q. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.
Christian S. Schade
President and Chief Executive Officer
Gregory A. Korbel
Vice President of Business Development
Source: Aprea Therapeutics, Inc.