On November 13, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (TSX: APS and OTC: APTOF), a clinical-stage precision oncology company developing a tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed acute myeloid leukemia (AML), reported financial results for the third quarter ended September 30, 2025, and provided a corporate update.
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"Tuspetinib in combination with VEN+AZA standard treatment (TUS+VEN+AZA) has been highly active and so well tolerated in newly diagnosed AML patients with 40 mg, 80 mg, and 120 mg TUS, we dose escalated to the 160 mg TUS dose level in the triplet," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. "Patients evaluated at the higher dose levels of 80 mg and 120 mg TUS have all (6/6; 100%) achieved CR/CRh responses, exceeding the 66% rate expected from VEN+AZA alone. We now are dosing at 160 mg TUS, and we look forward to providing further updates next month at ASH (Free ASH Whitepaper)."
Key Corporate Highlights
Tuspetinib Data Reported at European School of Haematology (ESH) 7th International Conference Data from the ongoing TUSCANY trial of tuspetinib in combination with venetoclax and azacitidine (TUS+VEN+AZA) were presented in a poster presentation, "TUSCANY Study of Safety and Efficacy of Tuspetinib plus Standard of Care Venetoclax and Azacitidine in Study Participants with Newly Diagnosed AML Ineligible for Induction Chemotherapy," at the European School of Haematology (ESH) 7th International Conference on Acute Myeloid Leukemia "Molecular and Translational": Advances in Biology and Treatment, held in October in Estoril, Portugal. Data from 10 patients in the TUSCANY trial across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, reveal promising clinical safety and antileukemic activity and support the use of TUS with standard of care treatment across a broad range of AML populations, including those carrying adverse mutations regardless of FLT3 mutation status.
As reported, the addition of TUS to VEN+AZA achieved CR/CRh responses in 6/6 (100%) patients treated at the higher dose levels of 80 mg and 120 mg TUS, exceeding the 66% rate expected from VEN+AZA alone. Overall, TUS+VEN+AZA has delivered CR/CRh responses in 9/10 (90%) patients. CR/CRh responses were achieved across diverse mutational subtypes including unmutated FLT3, FLT3-ITD, NPM1c, biallelic TP53 with complex karyotype, RAS, and myelodysplasia related mutations. MRD-negativity with TUS+VEN+AZA was observed in 7/9 (78%) of responding patients by central flow cytometry, and hematopoietic stem cell transplants (HSCT) have been completed in 2 patients to date.
Aptose Clinical Data Accepted for Poster Presentation at ASH (Free ASH Whitepaper) – Aptose was notified that its abstract, "TUSCANY Study demonstrates safety and efficacy of tuspetinib plus standard of care venetoclax and azacitidine in patients with newly diagnosed AML ineligible for induction chemotherapy," has been selected for poster presentation at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The meeting is scheduled to take place December 6-9, 2025, in Orlando, Florida. The abstract accepted for presentation can be viewed online at the ASH (Free ASH Whitepaper) conference website here, and will appear in the November supplemental issue of Blood. The actual presentation will include more recent updates and additional data not found in the abstract.
Completed and Planned Value-Creating Milestones
2025: 1H
Reported safety and efficacy with 40mg TUS+VEN+AZA
Reported safety and efficacy with 80mg TUS+VEN+AZA
2025: European Hematology Association (EHA) (Free EHA Whitepaper)
Reported maturing data from TUS+VEN+AZA triplet study
2025: 2H
Reported safety and efficacy with 120 mg TUS+VEN+AZA
CSRC review of data; decision to dose escalate to 160 mg TUS+VEN+AZA
2025: European School of Haematology (ESH) 7th International Conference
Reported excellent safety across three TUS dose levels of TUS+VEN+AZA
Reported CR/CRh responses in patients with biallelic TP53 mutations
Reported evolving data from 120 mg TUS+VEN+AZA triplet
2025: American Society of Hematology (ASH) (Free ASH Whitepaper)
Report evolving response rate and durability data from four (4) dose levels of TUS+VEN+AZA triplet
Report safety and tolerability of TUS with VEN+AZA in combination with unadjusted dosing of VEN+AZA
FINANCIAL RESULTS OF OPERATIONS
Aptose Biosciences Inc.
Statements of Operations Data
(unaudited)
($ in thousands, except for share and per share data)
Three months ended
September 30,
Nine months ended
September 30,
2025
2024
2025
2024
Operating expenses:
Research and development $ 2,205 $ 4,702 $ 7,867 $ 15,560
General and administrative 2,708 2,263 9,428 8,510
Total operating expenses 4,913 6,965 17,295 24,070
Other (expense) income, net (210 ) 12 (414 ) 225
Net loss $ (5,123 ) $ (6,953 ) $ (17,709 ) $ (23,845 )
Net loss per share, basic and diluted $ (2.01 ) $ (11.33 ) $ (7.34 ) $ (44.41 )
Weighted average number of common shares outstanding used in the calculation of basic and diluted loss per common share
2,552,429 613,604 2,411,943 536,891
Net loss for the quarter ended September 30, 2025 decreased by $1.8 million to $5.1 million, as compared to $7.0 million for the comparable period in 2024. Net loss for the nine months ended September 30, 2025 decreased by $6.1 million to $17.7 million, as compared to $23.8 million for the comparable period in 2024.
Aptose Biosciences Inc.
Balance Sheet Data
(unaudited)
($ in thousands)
September 30, December 31,
2025
2024
Cash, cash equivalents and restricted cash equivalents $ 1,637 $ 6,707
Working capital (3,302 ) 5,053
Total assets 6,341 10,127
Long-term liabilities 18,712 10,193
Accumulated deficit (558,676 ) (540,967 )
Shareholders’ deficit (19,450 ) (4,543 )
Total cash, cash equivalents and restricted cash equivalents as of September 30, 2025 were $1.6 million. The Company does not have sufficient cash to fund operations and relies on advances made by Hanmi to fund operations. The Company is actively deploying financing and cost reduction efforts to extend cash runway.
As of November 7, 2025, there were 2,552,429 common shares of the Company ("Common Shares") issued and outstanding. In addition, there were 37,370 Common Shares issuable upon the exercise of outstanding stock options and there were 1,267,585 Common Shares issuable upon the exercise of the outstanding warrants.
RESEARCH AND DEVELOPMENT EXPENSES
Research and development expenses for the three and nine months ended September 30, 2025 and 2024 were as follows:
Three months ended
Nine months ended
September 30,
September 30,
(in thousands) 2025
2024
2025
2024
Program costs – Tuspetinib $ 1,423 $ 4,067 $ 5,135 $ 10,656
Program costs – Luxeptinib 91 (225 ) 290 287
Program costs – APTO-253 - - - 13
Personnel related expenses 661 941 2,258 4,274
Stock-based compensation 30 (81 ) 184 317
Depreciation of equipment - - - 13
Total $ 2,205 $ 4,702 $ 7,867 $ 15,560
Research and development expenses decreased by $2.5 million to $2.2 million for the quarter ended September 30, 2025, as compared to $4.7 million for the comparable period in 2024. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:
Program costs for tuspetinib were $1.4 million for the quarter ended September 30, 2025, compared with $4.1 million for the comparable period in 2024. The lower program costs for tuspetinib in the current period are attributable to reduced activity in our APTIVATE clinical trial, reduced manufacturing activity, and related expenses.
Program costs for luxeptinib increased by approximately $0.3 million during the three months ended September 30, 2025 compared to the comparable period in 2024 due to a refund provided by one of our clinical vendors during the three months ended September 30, 2024.
The Company discontinued further development of APTO-253.
Personnel-related expenses decreased by $0.3 million due to lower headcount for research and development personnel in the current quarter.
Stock-based compensation increased by $0.1 million in the quarter ended September 30, 2025, compared to the comparable period in 2024, primarily due to forfeitures recognized during the three months ended September 30, 2024 in connection with employee terminations during the period.
Research and development expenses decreased by $7.7 million to $7.9 million for the nine months ended September 30, 2025, as compared to $15.6 million for the comparable period in 2024. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:
Program costs for tuspetinib were $5.1 million for the nine months ending September 30, 2025, compared to $10.7 million for the comparable period in 2024. The increased costs associated with the TUSCANY study were offset by a decrease in tuspetinib development expenses during the current period. This reduction is due to the conclusion of activities in our APTIVATE clinical trial during the current period, compared to higher APTIVATE activities during the nine months ended September 30, 2024, as well as lower manufacturing and related development costs.
Program costs for luxeptinib remained consistent during the nine months ended September 30, 2025 compared to the comparable period in 2024.
The Company discontinued further development of APTO-253.
Personnel-related expenses decreased by $2.0 million due to lower headcount for research and development personnel in the current quarter.
Stock-based compensation decreased by approximately $0.1 million in the nine months ended September 30, 2025, compared to the comparable period in 2024, primarily due to stock options forfeited and/or vested in prior periods that are no longer being expensed resulting in lower expense in the current period.
(Press release, Aptose Biosciences, NOV 13, 2025, View Source [SID1234659890])