Page 1

aprea therapeutics Aprea Therapeutics Reports First Quarter 2021 Financial Results and Provides Update on Business Operations May 6 , 2021 BOSTON , May 06 , 2021 ( GLOBE NEWSWIRE ) -- Aprea Therapeutics , Inc. ( Nasdaq : APRE ) , a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein , p53 , today reported financial results for the three months ended March 31 , 2021 and provided a business update . " During our recent R & D Day presentation , we reported an analysis of available data from its Phase 3 MDS trial . We identified an imbalance in dose modifications in the experimental arm which we believe negatively impacted efficacy , particularly the primary CR endpoint , " said Christian S. Schade , Chairman and Chief Executive Officer of Aprea . " We remain confident that eprenetapopt and our next - generation oral agent , APR - 548 , represent important potential therapeutic options for cancer patients and are encouraged by emerging data from our ongoing clinical trials . We look forward to sharing data updates from these clinical trials as well as our continued progress in expanding the opportunity for our therapies in new indications . " Business Operations Update : The Company is conducting , supporting , and planning multiple clinical trials of eprenetapopt ( APR - 246 ) and APR - 548 : • Phase 3 Frontline MDS Trial -- In June 2020 , the Company completed full enrollment of 154 patients in a pivotal Phase 3 trial of eprenetapopt with azacitidine for frontline treatment of patients with TP53 mutant MDS . The pivotal Phase 3 trial is supported by data from two Phase 1b / 2 investigator - initiated trials , one in the U.S. and one in France , testing eprenetapopt with azacitidine as frontline treatment in TP53 mutant MDS and AML patients . The data from the U.S. and French Phase 1b / 2 trials were published in The Journal of Clinical Oncology in January 2021 and February 2021 , respectively . In December 2020 , the Company announced that its pivotal Phase 3 trial failed to meet its predefined primary endpoint of complete remission ( CR ) rate . Analysis of the primary endpoint at this data cut demonstrated a higher CR rate ( 53 % more patients achieving a CR ) in the experimental arm receiving eprenetapopt with azacitidine versus the control arm receiving azacitidine alone but did not reach statistical significance . Based on a thorough analysis of the current Phase 3 trial data and comparisons to the U.S. and French Phase 1b / 2 trials the Company believes that despite similar types and frequency of adverse events observed in the Phase 3 experimental arm and the Phase 1b / 2 trials , patients in the Phase 3 experimental arm experienced substantially more study treatment dose modifications compared to the experience in the U.S. and French Phase 1b / 2 trials . The Company believes that dose modifications of eprenetapopt and azacitidine led to undertreatment in the Phase 3 experimental arm that negatively impacted efficacy , particularly the primary endpoint of CR rate . The Company continues to follow patients who remain on - study and anticipates discussing with FDA the Phase 3 data and future possible regulatory pathways in the second half of 2021 . • Phase 2 MDS / AML Post - Transplant Trial – The Company has completed enrollment of 33 patients in a single - arm , open - label Phase 2 clinical trial evaluating eprenetapopt with azacitidine as post - transplant maintenance therapy in TP53 mutant MDS and AML patients who have received an allogeneic stem cell transplant . The primary endpoint of the trial is the rate of relapse - free survival ( RFS ) at 12 months , with a published benchmark of ~ 30 % . An interim analysis in April 2021 showed a 62 % rate of RFS at 12 months , with a median RFS of 462 days . An interim analysis of overall survival ( OS ) showed a 77 % OS at 1 year , with a median number of events not yet reached . The Company anticipates initial results from the primary endpoint of RFS at 12 months in the second quarter of 2021 . • Phase 1/2 AML Trial - The Company is currently enrolling a Phase 1/2 clinical trial evaluating the safety , tolerability , and preliminary efficacy of eprenetapopt therapy in TP53 mutant AML patients . The lead - in portion of the trial evaluated the tolerability of eprenetapopt with venetoclax , with or without azacitidine , and no dose - limiting toxicities were observed in 12 patients receiving either regimen . Based on these results , the Company has expanded the trial to treat 33 additional frontline TP53 mutant AML patients with the combination of eprenetapopt , venetoclax and azacitidine . In the 19 frontline