Aprea Therapeutics, Inc. — Q3 2021 Form 10-Q
Business context and reporting period. The clinical-stage biopharmaceutical company develops cancer therapies intended to reactivate mutant p53. This filing covers the quarter and nine months ended September 30, 2021. It has no approved products and has not generated product revenue.
Financial performance and position
| Metric | Q3 2021 / Sep. 30, 2021 | Comparable period / prior year-end |
|---|---|---|
| Revenue | None | None reported |
| Operating expenses, quarter | $9.43 million | $12.23 million in Q3 2020 |
| Research and development, quarter | $6.02 million | $8.76 million in Q3 2020 |
| General and administrative, quarter | $3.41 million | $3.47 million in Q3 2020 |
| Net loss, quarter | $9.45 million; $0.45 per share | $12.32 million; $0.58 per share in Q3 2020 |
| Net loss, nine months | $29.37 million; $1.39 per share | $38.09 million; $1.80 per share for 2020 |
| Cash and cash equivalents | $61.43 million | $89.02 million at Dec. 31, 2020 |
| Operating cash used, nine months | $27.51 million | $28.60 million for 2020 |
| Total assets / liabilities | $62.48 million / $9.07 million | $92.81 million / $15.41 million at Dec. 31, 2020 |
| Accumulated deficit | $173.38 million | $144.01 million at Dec. 31, 2020 |
Gross profit and margins are not applicable because the company reports no product revenue. No borrowings or material financial debt are disclosed; liabilities consist mainly of accounts payable, accrued expenses, and operating lease obligations. Current liabilities were $9.04 million. Cash used in investing activities was zero and financing activities provided $0.09 million in the first nine months of 2021.
Material changes versus comparable periods
- Quarterly net loss narrowed by $2.87 million year over year; nine-month net loss narrowed by $8.71 million.
- Quarterly R&D expense fell $2.75 million, and nine-month R&D fell $9.12 million. The company attributed the decreases primarily to completion of enrollment in the pivotal MDS Phase 3 trial, lower post-transplant trial and companion-diagnostic costs, and paused commercial-scale manufacturing work. Spending on other early-stage programs increased.
- Cash declined $27.59 million from year-end 2020, primarily reflecting operating cash use. Stock-based compensation was $5.72 million for the nine months, versus $3.55 million in 2020.
Outlook, significant developments, and risks
- Management said the $61.4 million cash balance was expected to fund operating expenses and capital needs into 2023, based on assumptions that may prove incorrect. The company expects continued losses and says it will need additional financing; no external funding source is committed. It may need to reduce or stop development if financing is unavailable.
- On August 4, 2021, the FDA placed a partial clinical hold on eprenetapopt plus azacitidine trials in myeloid malignancies, citing questions about the risk-benefit profile and safety data from the Phase 3 frontline MDS study. No new patients may enroll until resolved; patients benefiting from treatment may continue. On August 11, the FDA placed a hold on a lymphoid-malignancy trial of eprenetapopt combinations; no patients were then receiving treatment. Management intends to address FDA questions.
- The pivotal Phase 3 frontline MDS trial had failed its predefined complete-remission primary endpoint in December 2020 and, following FDA feedback, management said it believed the trial had no registrational pathway. The company withdrew its Breakthrough Therapy designation.
- Management reported positive Phase 2 post-transplant results: among 33 patients, one-year relapse-free survival was 58% and one-year overall survival was 79%. In an AML study, 30 evaluable patients had a 37% complete-remission rate and a 53% composite CR/CRi rate; the trial met its primary efficacy endpoint under its Simon two-stage design. These are company-reported clinical findings, not approval outcomes.
- APR-548 Phase 1 dose escalation was enrolling its first cohort. Eprenetapopt plus anti-PD-1 therapy was being evaluated in solid tumors. Clinical trial outcomes, regulatory decisions, patient enrollment, COVID-19 effects, financing availability, and foreign-exchange movements remain risks. The company reported no material COVID-19 impact on results through the period and no clinical supply-chain disruption to date.
- The company reported no material legal proceedings, no provision for contingent losses, and no off-balance-sheet arrangements. Its $350 million shelf registration included a $50 million at-the-market program; no ATM shares had been sold by September 30, 2021.
Most important facts for investors to verify
- Current status and FDA requirements for both clinical holds, and whether either hold has been resolved.
- Full clinical datasets, follow-up, and durability of results for the post-transplant and AML studies, alongside the failed Phase 3 MDS endpoint.
- Actual cash burn, updated runway assumptions, and the timing and terms of any financing or collaboration.
- Development priorities and spending changes given paused manufacturing scale-up and reduced eprenetapopt R&D expense.
- Any subsequent changes in share count, ATM usage, commitments, or contingent liabilities.