APR-548 / Aprea - LARVOL DELTA

[VIRTUAL] Targeting p53 in MDS (SOHO 2021) - P1, P1b, P1b/2, P2, P3 | "Lastly, a 2nd generation oral agent (APR-548) will enter the clinic in 2021...These data support the planned Phase 3 open-label study of azacitidine + magrolimab versus azacitidine + venetoclax in unfit AML patients and versus induction chemotherapy in fit patients with a primary endpoint of OS in the non-intensive group (NCT04778397). Other novel HMA combinations have recently had high ORR/CR rates as frontline therapy, which include sabatolimab and pevonedistat, although unclear molecular subset data in TP53 mutant patients...Specifically, PDL1 has been shown to be upregulated on TP53 mutant hematopoietic stem cells and bone marrow blasts.17,18 Importantly, TP53 mutant AML patients have been strongly associated with an adverse immune microenvironment and potentially may be an optimal molecular subset for novel immunomodulatory agents.19 In this regard the bispecific agent flotetuzumab targeting CD123 has had activity in TP53 mutant relpased..."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • CD123 • TP53

Aprea Therapeutics Reports Third Quarter 2022 Financial Results and Provides Update on Business Operations (GlobeNewswire) - "We initiated a Phase 1 clinical trial testing APR-548 in relapsed/refractory MDS and AML and enrollment in the first dosing cohort was completed. There are currently no patients receiving APR-548 in this trial and enrollment into the trial has been closed."

Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - P1 | N=4 | Terminated | Sponsor: Aprea Therapeutics | N=46 ➔ 4 | Suspended ➔ Terminated; Sponsor decision

Combination therapy • Enrollment change • Trial termination • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • TP53

APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - P1 | N=46 | Suspended | Sponsor: Aprea Therapeutics | Recruiting ➔ Suspended

Combination therapy • Trial suspension • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • TP53

APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - P1; N=46; Recruiting; Sponsor: Aprea Therapeutics; Trial primary completion date: Dec 2021 ➔ Apr 2022

Clinical • Combination therapy • Trial primary completion date • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • TP53

APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - P1; N=46; Recruiting; Sponsor: Aprea Therapeutics; Initiation date: Apr 2021 ➔ Sep 2021

Clinical • Combination therapy • Trial initiation date • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • TP53

APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - P1; N=46; Recruiting; Sponsor: Aprea Therapeutics; Not yet recruiting ➔ Recruiting

Clinical • Combination therapy • Enrollment open • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • TP53

Aprea Therapeutics Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Update on Business Operations (GlobeNewswire) - "Business Operations Update: Pivotal Phase 3 MDS Trial - The Company is completing analysis from this Phase 3 clinical trial and expects to present additional information in the second quarter of 2021; Phase 2 MDS/AML Post-Transplant Trial – The Company anticipates initial results from the primary endpoint of relapse-free survival at 12 months in the second quarter of 2021; Phase 1/2 AML Trial – The Company anticipates completion of enrollment in the triplet regimen expansion cohort during the second quarter of 2021 with availability of preliminary response rate data from the cohort also in the second quarter of 2021; APR-548: The Company anticipates the first patient to be enrolled early in the second quarter of 2021."

Enrollment status • P1/2 data • P2 data • P3 data • Acute Myelogenous Leukemia • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Aprea Therapeutics Receives FDA Fast Track Designation for Eprenetapopt in the Treatment of TP53 Mutant Acute Myeloid Leukemia (AML) (GlobeNewswire) - "Aprea Therapeutics, Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for eprenetapopt in the treatment of patients with TP53 mutant acute myeloid leukemia (AML)...'Emerging data from our AML trials evaluating eprenetapopt with azacitidine, and with eprenetapopt, azacitidine and venetoclax, are promising and we continue to enroll patients to identify the best treatment regimen. As these data mature in 2021, we look forward to continued interaction with FDA as we map out opportunities for an accelerated pathway to potential approval.'...Enrollment in a Phase 1 clinical trial of APR-548 is anticipated to begin in the first quarter of 2021"

Clinical data • Enrollment status • Fast track designation • Acute Myelogenous Leukemia • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • TP53

APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - P1; N=46; Not yet recruiting; Sponsor: Aprea Therapeutics

Clinical • Combination therapy • New P1 trial • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • TP53

Aprea Therapeutics to Highlight Changing Treatment Paradigm in MDS as well as Development Pipeline Progress at Virtual R&D Day Today (GlobeNewswire) - "IR&D Day Webinar Agenda:...Introduction and discussion with Drs. David Sallman (Moffitt Cancer Center), Guillermo Garcia-Manero (MD Anderson Cancer Center), and Eyal Attar (Aprea’s Chief Medical Officer) to review current clinical therapy options for TP53 mutant MDS/AML patients and the potential role of eprenetapopt. Discussion with review of Aprea’s Phase 3 Clinical program in MDS to be followed by Q&A....A Phase 1 clinical trial of APR-548 in TP53 mutant MDS is planned."

Clinical • New P1 trial • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Aprea Therapeutics Receives OK from FDA to Initiate Phase 1 Clinical Studies for Next-Generation Mutant p53 Reactivator, APR-548 (GlobeNewswire) - “Aprea Therapeutics Inc…announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug (IND) application for APR-548 to treat TP53 mutant myelodysplastic syndromes (MDS). APR-548 is a next-generation small molecule reactivator of mutant p53 that is being developed for oral administration…‘We look forward to initiating the Phase 1 clinical trial of APR-548 in MDS…’”

IND • Hematological Malignancies • Myelodysplastic Syndrome • Oncology