On May 8, 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 first quarter ended March 31, 2025, and provided a corporate update (Press release, Aptose Biosciences, MAY 8, 2025, View Source [SID1234652721]).

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"Our TUSCANY clinical trial of tuspetinib in combination with venetoclax (VEN) and azacitidine (AZA) for frontline treatment of newly diagnosed acute myeloid leukemia (AML) continues to deliver robust safety and response data, with support and enthusiasm from our clinical investigators," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. "We recently escalated the TUS dose to 80 mg from the initial dose of 40 mg in the TUS+VEN+AZA triplet therapy. Three patients receiving the initial dose of 40 mg and three patients receiving the 80 mg dose all have achieved complete remissions (CRs*) and continue on treatment. We are especially encouraged that two patients having highly adverse TP53 mutations have achieved objective responses and remain on treatment."

Key Corporate Highlights

Tuspetinib Data Reported from First Two Cohorts of TUSCANY Phase 1/2 Trial – Tuspetinib based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating a one-of-a-kind frontline therapy for newly diagnosed AML patients that is safe and active across diverse AML populations (mutation agnostic triplet frontline therapy), including patients without FLT3 mutations (wildtype FLT3). Data from the first two cohorts, with a 40 mg or 80 mg dose of tuspetinib in the TUS+VEN+AZA combination, reveal promising clinical safety and antileukemic activity (press release here).
In the first cohort, our newly diagnosed AML patients received the initial 40 mg dose of TUS as part of the TUS+VEN+AZA combination. No safety concerns or dose limiting toxicities (DLTs) were observed. Three patients experienced rapid bone marrow blast reductions to achieve CRs; one having biallelic mutated TP53, complex karyotype (CK) chromosomal abnormalities, and wildtype FLT3; one having an IDH2 mutation wildtype FLT3; and one having FLT3-ITD and mutated NPM1. Clinical sites or central labs reported all these patients also achieved MRD-negative status. The fourth patient at the 40 mg dose of TUS, did not achieve a complete remission and discontinued the study. The first three patients continue on treatment.

In the second cohort, three newly diagnosed AML patients having diverse mutation profiles have received the 80 mg dose of TUS, as part of the TUS+VEN+AZA combination. To date, no safety concerns or DLTs have been reported. All patients at the 80 mg dose of TUS rapidly achieved CRs; one patient with biallelic TP53 mutations and a complex karyotype and wildtype FLT3 status, a second patient having mutated DDX41 and wildtype FLT3 status, and a third patient having FLT3-ITD and NPM1 mutations. All three patients at the 80 mg dose of TUS are early in their course of treatment, are expected to achieve normal blood count recovery as their leukemia is resolved, and MRD status will be monitored as the patients move through their courses of therapy.

OTC Exchange – Aptose common shares ("the Common Shares") are now listed for trading on the OTC Markets (OTC) under the ticker "APTOF," in addition to the Company’s continued listing on the Toronto Stock Exchange (TSX) under the symbol "APS". This significantly expands Aptose accessibility for U.S.-based investors and enhances overall share liquidity by enabling trading through U.S. broker-dealers. Listing on the OTC Markets is part of Aptose’s strategy to align with TSX capital markets while increasing visibility and participation from the U.S. investment community. Aptose’s Common Shares were delisted from The Nasdaq Stock Market effective April 2, 2025 because of non-compliance with the Exchange’s equity requirements.
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)

Report maturing data from TUS+VEN+AZA triplet study
2025: American Society of Hematology (ASH) (Free ASH Whitepaper)

Report response rate and durability of TUS+VEN+AZA triplet
Select TUS dose for TUS+VEN+HMA triplet Ph 2/3 PIVOTAL trials
Prepare for initiation of Ph 2/3 PIVOTAL program

FINANCIAL RESULTS OF OPERATIONS
Aptose Biosciences Inc.
Statements of Operations Data
(unaudited)
($ in thousands, except for share and per share data)

Quarter ended
March 31,
2025 2024
Expenses:

Research and development $ 2,364 $ 6,445
General and administrative 3,097 3,315
Operating expenses 5,461 9,760
Other (loss) income, net (82 ) 120

Net loss $ (5,543 ) $ (9,640 )

Net loss per share, basic and diluted $ (2.61 ) $ (22.02 )
Weighted average number of
common shares outstanding used in
computing net loss per share, basic
and diluted
2,126,287 437,750

Net loss for the quarter ended March 31, 2025 decreased by $4.1 million to $5.5 million, as compared to $9.6 million for the comparable period in 2024.

Aptose Biosciences Inc.
Balance Sheet Data
(unaudited)
($ in thousands)

March 31,
2025 December 31,
2024
Cash, cash equivalents and restricted cash
equivalents $ 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 )

Total cash, cash equivalents and restricted cash equivalents as of March 31, 2025 were $4.7 million. Based on current operations, the Company expects that cash on hand and available capital provides the Company with sufficient resources to fund planned Company operations including research and development until the end of May 2025. The Company is proactively implementing financing and cost reduction efforts to extend its cash runway.
As of May 1, 2025, we had 2,552,429 Common Shares issued and outstanding. In addition, there were 38,736 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 months ended March 31, 2025 and 2024 were as follows:

Quarter ended
(in thousands) March 31,
2025 March 31,
2024
Program costs – Tuspetinib $ 1,479 $ 3,923
Program costs – Luxeptinib 98 208
Program costs – APTO-253 - 22
Personnel related expenses 646 1,924
Stock-based compensation 141 328
Depreciation of equipment - 10
Total $ 2,364 $ 6,445

Research and development expenses decreased by $4.1 million to $2.3 million for the quarter ended March 31, 2025, as compared to $6.4 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 $1.5 million for the quarter ended March 31, 2025, compared with $3.9 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 decreased by approximately $0.1 million primarily due to lower clinical trial and manufacturing activities.
Program costs for APTO-253 were zero. This was due to the Company’s decision to discontinue further development of APTO-253.
Personnel-related expenses decreased by $1.3 million due to lower headcount for research and development personnel in the current quarter.
Stock-based compensation decreased by approximately $0.2 million in the quarter ended March 31, 2025, primarily due stock options forfeited and/or vested in prior periods that are no longer being expensed resulting in lower expense in the current period.