On March 26, 2020 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53, reported financial results for the three months and year ended December 31, 2019 and provided a corporate update (Press release, Aprea, MAR 26, 2020, View Source [SID1234555875]).
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Corporate Update:
The Company is conducting, supporting and planning multiple clinical trials of APR-246:
·Pivotal Phase 3 MDS Trial—The Company is currently enrolling a pivotal Phase 3 randomized, controlled trial evaluating APR-246 with azacitidine as frontline therapy in HMA-naïve TP53 mutant myelodysplastic syndromes (MDS) patients. The trial has a target enrollment of 154 patients randomized in a 1:1 ratio to either the azacitidine control arm or to the APR-246 + azacitidine experimental arm, with a primary endpoint of CR rate. The Company had anticipated full enrollment in its Phase 3 trial in the first quarter of 2020 and as of March 25, 2020, the Company had enrolled 133 patients. The Company has observed a recent decrease in both patient screening and patient enrollment as a result of the recent coronavirus (COVID 19) pandemic. Together with its investigators and clinical sites, the Company is assessing the potential impact of the coronavirus pandemic on enrollment and the ability to maintain patients enrolled in the trial and the corresponding impact on the timing of the completion of the trial and subsequent availability of top-line data. The Company remains confident that it can complete the trial and have top-line data available before year end 2020.
·Phase 2 MDS/AML Post-Transplant Trial—The Company is currently enrolling its single-arm, open-label Phase 2 trial evaluating APR-246 with azacitidine as post-transplant maintenance therapy in TP53 mutant MDS and acute myeloid leukemia (AML) patients who have received an allogeneic stem cell transplant. The primary endpoint is relapse-free survival at 12 months. As of March 25, 2020, the Company had enrolled 11 patients in this trial. Target enrollment is 31 patients and the Company had anticipated full enrollment in the first half of 2020. Together with its investigators and clinical sites, the Company is assessing the potential impact of the coronavirus pandemic on the enrollment and the ability to maintain patients enrolled in this trial.
·Phase 1 AML Trial—Based on in vitro data evidencing synergistic activity between APR-246 and a Bcl-2 inhibitor, the Company is conducting a Phase 1 clinical trial in frontline and relapsed/refractory TP53 mutant AML assessing APR-246 with venetoclax with or without azacitidine. The primary endpoint is the composite rate of CR and CR with incomplete hematologic recovery, or CRi. The first patient was enrolled in 1Q 2020 and the Company completed enrollment of the first two safety cohorts of three patients each. Together with its investigators and clinical sites, the Company is assessing the potential impact of the coronavirus pandemic on the enrollment and the ability to maintain patients enrolled in this trial.
·Phase 1 NHL Trial—As further assessment of APR-246 in hematological malignancies, the Company has designed and plans to conduct a Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) and mantle cell lymphoma (MCL) assessing APR-246 with venetoclax and rituximab, and APR-246 with ibrutinib. The Company is targeting the first patient to be enrolled in the second half of 2020.
·Phase 1/2 Solid Tumor Trial—Based on in vitro data evidencing synergistic activity between APR-246 and immuno-therapy agents including anti-PD-1 antibody, the Company has designed and plans to conduct Phase 1/2 clinical trials in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing APR-246 with anti-PD-1 therapy. The Company is targeting the first patient to be enrolled in the second half of 2020.
·APR-548 — The Company’s second product candidate, APR-548, is a next-generation p53 reactivator with the potential for oral administration. APR-548 is a unique analog of APR-246 and therefore a pro-drug of MQ. APR-548 exhibits high oral bioavailability in preclinical testing and is being developed in an oral dosage form. The Company has completed Investigational New Drug, or IND, enabling preclinical studies of APR-548. Final reports from these studies are pending and the Company is targeting the submission of an IND in the first half of 2020.
·APR-246 INN — The Company has secured eprenetapopt as the international nonproprietary name (INN) for APR-246.
Fourth Quarter Financial Results
·Cash and cash equivalents: As of December 31, 2019, Aprea had $130.1 million of cash and cash equivalents compared to $65.7 million of cash and cash equivalents as of December 31, 2018. In October 2019, the Company completed the sale of 6,516,667 shares of common stock in an initial public offering resulting in net proceeds of approximately $86.9 million. The Company expects cash burn for 2020 to be between $35.0 million $40.0 million. The Company believes its cash and cash equivalents as of December 31, 2019 will be sufficient to meet its current projected operating requirements into 2023.
·Research and Development (R&D) expenses: R&D expenses were $8.0 million for the quarter ended December 31, 2019, compared to $4.4 million for the comparable period in 2018. The increase in R&D expenses was primarily related to the advancement of the Company’s lead product candidate, APR-246. In Q1 2019 the Company commenced a pivotal Phase 3 clinical trial of APR-246 with azacytidine for frontline treatment of TP53 mutant MDS which is supported by two ongoing Phase 1b/2 investigator initiated trials, one in the U.S. and one in France, testing APR-246 with azacitidine as frontline treatment in TP53 mutant MDS and AML patients.
·General and Administrative (G&A) expenses: G&A expenses were $3.9 million for the quarter ended December 31, 2019, compared to $0.5 million for the comparable period in 2018. The increase in G&A expenses was primarily due to increased professional fees associated with operating as a public company, as well as increased personnel costs.
·Net loss: Net loss was $13.1 million, or $0.64 per share for the quarter ended December 31, 2019, compared to a net loss of $4.7 million, or $4.05 per share for the quarter ended December 31, 2018. Net loss for the year ended December 31, 2019 was $28.1 million, or $4.67 per share, compared to a net loss of $15.5 million, or $13.45 per share for the year ended December 31, 2018.