Aptose Biosciences(APTO) - 2025 Q1 - Quarterly Results

Report Overview & Highlights TUSCANY Clinical Trial Progress The TUSCANY trial for frontline AML with tuspetinib, venetoclax, and azacitidine shows robust safety and response, with all six patients achieving complete remissions across escalated doses up to 80 mg, including those with adverse mutations - The TUSCANY trial is evaluating a tuspetinib (TUS)-based triple drug frontline therapy (TUS+VEN+AZA) for newly diagnosed AML patients[2] - The tuspetinib dose was escalated from an initial 40 mg to 80 mg in the triplet therapy[3] - All six patients treated in the first two cohorts (three at 40 mg and three at 80 mg) achieved Complete Remissions (CRs) and remain on treatment[3] - In the 40 mg cohort, three of four patients achieved CRs, and all three were reported as MRD-negative[4] - In the 80 mg cohort, all three patients achieved CRs, with no safety concerns or dose-limiting toxicities (DLTs) reported to date[5] - The therapy has shown effectiveness in patients with diverse and adverse mutation profiles, including TP53, FLT3-ITD, IDH2, and DDX41 mutations[3][4][5] Corporate Updates Aptose common shares were delisted from Nasdaq due to non-compliance and now trade on OTC Markets under "APTOF" to enhance U.S. investor accessibility and liquidity, while maintaining TSX listing - Aptose common shares now trade on the OTC Markets under the ticker "APTOF" in addition to the Toronto Stock Exchange (TSX) under "APS"[5] - The company's common shares were delisted from The Nasdaq Stock Market effective April 2, 2025, because of non-compliance with the exchange's equity requirements[5] - The move to OTC is part of a strategy to increase visibility and participation from the U.S. investment community and enhance overall share liquidity[5] Completed and Planned Value-Creating Milestones Aptose reported initial safety and efficacy data for TUS+VEN+AZA cohorts, with plans to present maturing data at EHA and ASH in 2025, aiming to select the optimal tuspetinib dose for pivotal Phase 2/3 trials - Reported initial safety and efficacy data for both the 40mg and 80mg TUS+VEN+AZA cohorts[12] - Plan to report maturing data from the triplet study at the European Hematology Association (EHA) in 1H 2025[7] - Plan to report response rate and durability data at the American Society of Hematology (ASH) in 2025[8][12] - Future goals include selecting the tuspetinib dose for pivotal Phase 2/3 trials and preparing for their initiation[12] Financial Results of Operations Financial Summary (Statements of Operations) Aptose reported a Q1 2025 net loss of $5.5 million, a significant improvement from $9.6 million in Q1 2024, driven by reduced operating expenses, with net loss per share improving to $2.61 from $22.02 Q1 2025 vs Q1 2024 Statement of Operations ($ in thousands) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Research and development | $2,364 | $6,445 | | General and administrative | $3,097 | $3,315 | | Operating expenses | $5,461 | $9,760 | | Net loss | $(5,543) | $(9,640) | | Net loss per share | $(2.61) | $(22.02) | | Weighted average shares | 2,126,287 | 437,750 | Balance Sheet Summary As of March 31, 2025, Aptose reported $4.7 million in cash, $7.5 million in total assets, and a shareholders' deficit of $7.4 million, reflecting a decrease in cash and an increased deficit from year-end 2024 Balance Sheet Data ($ in thousands) | Metric | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and restricted cash | $4,743 | $6,707 | | Working capital | $651 | $5,053 | | Total assets | $7,467 | $10,127 | | Long-term liabilities | $8,542 | $10,193 | | Accumulated deficit | $(546,510) | $(540,967) | | Shareholders' deficit | $(7,393) | $(4,543) | Research and Development Expenses Q1 2025 R&D expenses significantly decreased to $2.4 million from $6.4 million in Q1 2024, primarily due to reduced tuspetinib program costs, lower personnel expenses, and the discontinuation of the APTO-253 program R&D Expense Breakdown - Q1 2025 vs Q1 2024 ($ in thousands) | Program | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Tuspetinib | $1,479 | $3,923 | | Luxeptinib | $98 | $208 | | APTO-253 | $- | $22 | | Personnel related expenses | $646 | $1,924 | | Stock-based compensation | $141 | $328 | | Total R&D Expenses | $2,364 | $6,445 | - Tuspetinib program costs decreased by $2.4 million due to reduced activity in the APTIVATE clinical trial and lower manufacturing expenses[16] - Personnel-related expenses decreased by $1.3 million due to lower R&D headcount[16] - The APTO-253 program was discontinued, eliminating its associated costs[16] Liquidity and Capital Resources As of March 31, 2025, Aptose held $4.7 million in cash, projecting funding only until May 2025, and is actively pursuing financing and cost-reduction strategies to extend its cash runway - Total cash, cash equivalents and restricted cash equivalents were $4.7 million as of March 31, 2025[15] - The company expects its current cash on hand to fund operations only until the end of May 2025[15] - Aptose is proactively implementing financing and cost reduction efforts to extend its cash runway[15] - As of May 1, 2025, the company had 2,552,429 common shares issued and outstanding[15] Corporate Information & Disclosures About Aptose Aptose Biosciences is a clinical-stage biotechnology company developing precision oncology medicines, with its lead compound, tuspetinib, an oral kinase inhibitor, targeting frontline acute myeloid leukemia - Aptose is a clinical-stage biotechnology company developing precision medicines for oncology, with a focus on hematology[15] - The company's lead clinical-stage compound is tuspetinib (TUS), an oral kinase inhibitor for acute myeloid leukemia (AML)[17] Forward-Looking Statements This section serves as a legal disclaimer for forward-looking statements, highlighting risks like capital acquisition, drug development efficacy, and market conditions, with no obligation to update unless legally required - The press release contains forward-looking statements that are subject to significant risks, uncertainties, and assumptions[18] - Key risks include the ability to obtain required capital, drug development efficacy, regulatory approval processes, clinical trial progress, and changing economic conditions[18] - Aptose does not assume any obligation to update these forward-looking statements, except as required by law[19]