Analyst Conference Summary

biotechnology

release date: March 16, 2021 (press release only)
for quarter ending: December 31, 2020 (fourth quarter, Q4)

Forward-looking statements

Overview: Clinical-stage company's leading trial had poor results.

Basic data (GAAP):

No revenue.

Net income was negative $13.1 milion, down sequentially from negative $12.5 million, and down from negative $10.9 year-earlier.

EPS (earnings per share) were negative $0.73, down sequentially from negative $0.58, and down from negative $0.64 year-earlier.

Guidance:

Cash use in 2021 Cash should be adequate into 2023.

Conference Highlights:

Christian S. Schade, CEO of Aprea, said "Though [we are] disappointed the topline complete remission rate from the Phase 3 clinical trial narrowly missed its primary endpoint, we continue to analyze the totality of the data from the study to understand those differences from our prior Phase 2 experience in frontline MDS patients and expect to present these findings in the second quarter of 2021. Our dedicated team remains committed to the clinical development of eprenetapopt and our next generation, oral p53 reactivator, APR-548, in hematological and solid tumor malignancies. In 2021, we look forward to sharing data from our current clinical studies as well as our plans to expand the clinical pipeline to include new indications."

At the end of the quarter Aprea had cash and equivalents of $89 million, down sequentially from $101 million.

The pivotal Phase 3 randomized, controlled trial evaluating eprenetapopt with azacitidine as frontline therapy in HMA-naive TP53 mutant myelodysplastic syndromes (MDS) patients failed to meet its prespecified complete response rate. Active arm CR was 53% higher than control arm, but that did not reach statistical significance. More detailed data will be released in Q2 2021.

Also completed enrollment, in Q3 2020, in the single-arm, open-label Phase 2 trial evaluating eprenetapopt 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. Should have results of the primary endpoint of relapse-free survival at 12 months in Q2 2021

A Phase 1/2 clinical trial of eprenetapopt therapy in TP53 mutant AML patients is currently enrolling. 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. So expanded the trial to treat 33 additional frontline TP53 mutant AML patients with the combination of eprenetapopt, venetoclax and azacitidine. In the 19 frontline AML patients who are evaluable for efficacy with the triplet regimen, achieved 63% CR + CRi composite response rate and a 31% CR rate. In Q2 should complete enrollment and report data from the expansion cohort.

A Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy is enrolling. The dose-escalation phase of the trial enrolled 6 patients with advanced solid tumors and no dose-limiting toxicities were observed. Now enrolling expansion cohorts for patients with advanced gastric, bladder and non-small cell lung cancers and has currently enrolled 8 patients across these expansion arms.

The Phase 1 CLL (chrnoic lymphoid leukemia) and mantle cell lymphoma trial is currently enrolling.

APR-548 is a next-generation p53 reactivator that is being developed in an oral dosage form. A Phase 1 dose-escalation clinical trial evaluating the safety, tolerability, and preliminary efficacy of APR-548 with azacitidine in frontline and relapsed/refractory MDS patients will begin enrollment in Q2 2021.

Total operating expenses were $ million, consisting of $ million for R&D and $ million for SG&A.

Q&A summary:

No question and answer session.

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