Financial Data and Key Metrics Changes - The company ended 2022 with approximately $47 million in cash, cash equivalents, and investments, a decrease of $4.8 million compared to the previous quarter [31] - The net loss for the fourth quarter was approximately $10 million, translating to a loss of approximately negative $0.11 per share, down from a loss of $24.3 million in the comparable period in 2021 [31] - Research and development expenses were $6.8 million for the quarter, down from $20.2 million in the same quarter of 2021, and for the full year, R&D expenses were $28.1 million compared to $46 million in the previous year [32] Business Line Data and Key Metrics Changes - The company made significant strides in the development of Tuspetinib for the treatment of acute myeloid leukemia (AML), demonstrating a highly differentiated profile with complete remissions in difficult-to-treat patients [7][10] - The new formulation of Luxceptinib (G3) achieved up to 18-fold greater absorption than the original formulation, with continuous dosing showing promising results [16] Market Data and Key Metrics Changes - The treatment paradigm for AML is shifting towards doublet and triplet therapies, with Tuspetinib positioned as a potential drug of choice due to its safety profile and efficacy [19][20] - The company is focusing on specific patient populations, including those with TP53 mutations and FLT3 mutations, to enhance the clinical development of Tuspetinib [24][27] Company Strategy and Development Direction - The company aims to position Tuspetinib as a preferred agent for combination therapy and in earlier lines of treatment, with plans for accelerated approval pathways [27][78] - The strategic focus includes expanding the clinical trials for Tuspetinib in combination with Venetoclax and exploring triplet combinations for frontline patients [12][20] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenging biotech market but emphasized the steady progress made in 2022, including extending the cash runway to the end of Q1 2024 [6] - The management expressed optimism about the potential of Tuspetinib and its role in addressing significant unmet medical needs in AML [19][27] Other Important Information - The company has engaged in productive discussions with several big pharma and biotech companies regarding potential partnerships for Tuspetinib [19] - The ACTIVATE trial for Tuspetinib has begun, with plans to report data at major medical meetings throughout 2023 [25][29] Q&A Session Summary Question: Is it too soon to guide on patient numbers for the ACTIVATE trial update? - Management indicated that enrollment has been brisk for the monotherapy arm, and they expect to provide more clarity on patient numbers later in the year [38][40] Question: Can you provide more details on the different dose cohort sizes being enrolled? - The company has completed the dose escalation and exploration trial, with 60 patients treated, and the starting dose for the ACTIVATE trial is 120 milligrams for monotherapy [44] Question: What are the expectations for the regulatory plan for the monotherapy arm in ACTIVATE? - Management stated that they will align with the FDA on parameters for accelerated approval, with a focus on specific patient populations [50][78] Question: How does the company plan to present data from the ACTIVATE trial? - The company plans to provide updates at major medical meetings, with a more comprehensive data package expected later in the year [46][47] Question: What are the expectations for the activity seen in the monotherapy arm of ACTIVATE? - Early signs of anti-leukemic activity have been observed, but it is still early in the trial [66][69] Question: How is the company approaching the potential for accelerated approval based on specific mutations? - The company aims to enrich for specific populations with high unmet needs, such as those with TP53 mutations and FLT3 mutations, to support regulatory discussions [77][78]

Part I [Business](index=3&type=section&id=ITEM%201.%20BUSINESS) Aptose Biosciences is a clinical-stage biotech developing precision oncology medicines for hematologic malignancies, with lead assets tuspetinib and luxeptinib in Phase 1/2 trials - Aptose is a clinical-stage biotechnology company focused on precision oncology medicines for hematology, with assets designed for single-agent and combination efficacy without overlapping toxicities[14](index=14&type=chunk) - The company's strategy focuses on advancing oral targeted agents for life-threatening cancers, primarily tuspetinib and luxeptinib[15](index=15&type=chunk) - The clinical program for APTO-253, a MYC oncogene inhibitor, was discontinued on December 20, 2021, to prioritize advanced pipeline candidates[20](index=20&type=chunk)[39](index=39&type=chunk) Product Candidate Pipeline | Product Candidate | Target Indication | Stage of Development | | :--- | :--- | :--- | | **Tuspetinib** | Relapsed/Refractory Acute Myeloid Leukemia (R/R AML) | Phase 1/2 | | **Luxeptinib** | R/R B-Cell Malignancies & R/R AML/High-Risk MDS | Phase 1a/b | | **APTO-253** | R/R Blood Cancers (AML/MDS) | Program Discontinued | [Tuspetinib Program](index=5&type=section&id=Tuspetinib%20Program) Tuspetinib, an oral myeloid kinase inhibitor, has shown complete responses in R/R AML Phase 1/2 trials and advanced to the APTIVATE expansion study - Aptose acquired exclusive worldwide rights for tuspetinib from Hanmi Pharmaceutical for an upfront payment of **$12.5 million** ($5 million cash, $7.5 million shares) and up to **$407.5 million** in future milestones plus royalties[23](index=23&type=chunk) - In its Phase 1/2 trial for R/R AML, tuspetinib demonstrated **complete responses (CRs)** at multiple dose levels (40mg, 80mg, 120mg, 160mg) with a favorable safety profile, showing no QTc prolongations, liver/kidney toxicities, or differentiation syndrome[16](index=16&type=chunk)[28](index=28&type=chunk) - The program advanced to the APTIVATE expansion trial, with **120 mg** selected for monotherapy and **80 mg** for combination therapy with venetoclax[28](index=28&type=chunk) [Luxeptinib Program](index=7&type=section&id=Luxeptinib%20Program) Luxeptinib, a dual kinase inhibitor, is in Phase 1a/b trials for B-cell malignancies and AML, with a new G3 formulation showing improved bioavailability - Aptose holds worldwide rights (excluding Korea) to luxeptinib, licensed from Crystal Genomics, with a total deal value of up to **$125 million** for China rights, plus milestones and royalties[31](index=31&type=chunk) - A new G3 formulation was developed to address limited absorption, with a **50 mg** G3 dose predicted to provide exposure comparable to a **900 mg** G1 dose in R/R AML patients[19](index=19&type=chunk)[36](index=36&type=chunk) - The original G1 formulation yielded a **complete response (CR)** in a DLBCL patient at a **900mg BID** dose and a **CR** in an R/R AML patient at a **450mg BID** dose[19](index=19&type=chunk)[36](index=36&type=chunk) [Competitive Conditions](index=9&type=section&id=Competitive%20Conditions) The company faces intense competition in AML and B-cell malignancies from established pharmaceutical companies with approved and developing therapies - In the AML market, Aptose competes with approved therapies from companies including Jazz (VYXEOS), Pfizer (MYLOTARG), Novartis (RYDAPT), Astellas (XOSPATA), and AbbVie (VENCLEXTA)[42](index=42&type=chunk) - For B-cell malignancies, competitors include companies with approved BTK inhibitors like AbbVie (IMBRUVICA) and AstraZeneca (CALQUENCE), and those developing inhibitors for mutant BTK forms, such as Merck and Eli Lilly[43](index=43&type=chunk) [Intellectual Property](index=10&type=section&id=Intellectual%20Property) Aptose holds intellectual property for tuspetinib (39 patents, protection until 2038-2039) and luxeptinib (46 patents, protection until 2033-2038) via licensing - Aptose holds exclusive worldwide rights to tuspetinib, with **39 issued patents** providing protection until **2038-2039**[48](index=48&type=chunk) - The company holds rights to **46 issued patents** for luxeptinib, expected to provide protection until **2033-2038**[51](index=51&type=chunk) [Risk Factors](index=19&type=section&id=ITEM%201A.%20RISK%20FACTORS) The company faces significant risks including going concern doubt, early-stage product development, reliance on third parties, regulatory uncertainties, intense competition, and Nasdaq delisting concerns - There is substantial doubt about the company's ability to continue as a going concern, requiring additional financing to fund operations over the next twelve months[94](index=94&type=chunk) - The company has a history of operating losses, reporting a net loss of **$41.8 million** for FY 2022 and an accumulated deficit of **$464.3 million** as of December 31, 2022[104](index=104&type=chunk) - On July 18, 2022, the company received a Nasdaq notice for its stock price falling below the **$1.00** minimum, with a **180-day** extension until July 18, 2023, to regain compliance[203](index=203&type=chunk)[204](index=204&type=chunk) - The company believes it is classified as a Passive Foreign Investment Company (PFIC), potentially leading to adverse U.S. federal income tax consequences for its U.S. shareholders[212](index=212&type=chunk) - Aptose relies heavily on contract manufacturing organizations (CMOs) for product manufacturing and contract research organizations (CROs) for clinical development, creating dependence on third parties[132](index=132&type=chunk)[143](index=143&type=chunk) [Properties](index=40&type=section&id=ITEM%202.%20PROPERTIES) Aptose leases office spaces in San Diego (7,556 sq. ft.) and Toronto (2,078 sq. ft.), with a San Diego lab lease expiring in February 2023 - The company leases office space in San Diego, CA (**7,556 sq. ft.**) and Toronto, Canada (**2,078 sq. ft.**)[221](index=221&type=chunk) [Legal Proceedings](index=40&type=section&id=ITEM%203.%20LEGAL%20PROCEEDINGS) The company reports no material pending legal proceedings as of the report date - As of the report date, the company is not aware of any material pending legal proceedings[222](index=222&type=chunk) Part II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=41&type=section&id=ITEM%205.%20MARKET%20FOR%20REGISTRANT%27S%20COMMON%20EQUITY%2C%20RELATED%20STOCKHOLDER%20MATTERS%20AND%20ISSUER%20PURCHASES%20OF%20EQUITY%20SECURITIES) The company's common shares trade on Nasdaq (APTO) and TSX (APS), with no anticipated cash dividends or Q4 2022 equity repurchases - Common shares are traded on Nasdaq (**APTO**) and the Toronto Stock Exchange (**APS**)[224](index=224&type=chunk) - The company does not expect to pay cash dividends in the foreseeable future[224](index=224&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=42&type=section&id=ITEM%207%20-%20MANAGEMENT%27S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) MD&A details a reduced net loss in 2022 due to lower R&D, but weakened liquidity and going concern doubt persist, while tuspetinib and luxeptinib programs advanced [Program Updates](index=42&type=section&id=PROGRAM%20UPDATES) In 2022, tuspetinib completed dose escalation and initiated the APTIVATE trial, while luxeptinib advanced with a new, more bioavailable G3 formulation - Tuspetinib completed dose escalation and exploration, delivering single-agent responses in heavily pretreated R/R AML patients, and initiated the APTIVATE expansion trial for monotherapy (**120mg**) and combination (**80mg**) with venetoclax[236](index=236&type=chunk) - For luxeptinib, a new G3 formulation was developed, with a **50mg** G3 dose predicted to achieve plasma exposure comparable to **900mg** of the original G1 formulation, with AML patient dosing beginning in November 2022[240](index=240&type=chunk) [Liquidity and Capital Resources](index=47&type=section&id=LIQUIDITY%20AND%20CAPITAL%20RESOURCES) The company's liquidity significantly declined in 2022, with cash and investments at **$47.0 million**, raising substantial doubt about its going concern status and necessitating additional financing - Management states that additional financing is necessary to continue operations, raising substantial doubt about the company's ability to continue as a going concern[255](index=255&type=chunk) Liquidity Position (in thousands) | Metric | Dec 31, 2022 | Dec 31, 2021 | | :--- | :--- | :--- | | Cash and cash equivalents | $36,970 | $39,114 | | Investments | $9,989 | $40,014 | | **Total Cash & Investments** | **$46,959** | **$79,128** | | Working capital | $37,235 | $73,563 | Cash Flow Summary (in thousands) | Activity | Year Ended Dec 31, 2022 | Year Ended Dec 31, 2021 | | :--- | :--- | :--- | | Net cash used in Operating activities | $(32,322) | $(43,304) | | Net cash from (used in) Investing activities | $30,066 | $(35,208) | | Net cash from Financing activities | $116 | $226 | [Results of Operations](index=50&type=section&id=RESULTS%20OF%20OPERATIONS) Aptose reported a reduced net loss of **$41.8 million** in 2022, down from **$65.4 million** in 2021, primarily due to lower R&D and G&A expenses Consolidated Results of Operations (in thousands, except per share data) | Metric | 2022 | 2021 | | :--- | :--- | :--- | | Research and development expenses | $28,088 | $45,985 | | General and administrative expenses | $14,514 | $19,462 | | **Net loss** | **$(41,823)** | **$(65,354)** | | **Basic and diluted loss per Common Share** | **$(0.45)** | **$(0.73)** | Research & Development Expense Breakdown (in thousands) | Expense Category | 2022 | 2021 | | :--- | :--- | :--- | | License fee – Tuspetinib | $— | $12,500 | | Program costs – Tuspetinib | $10,083 | $57 | | Program costs – Luxeptinib | $8,426 | $18,490 | | Program costs – APTO-253 | $141 | $3,543 | | Personnel & Stock-based comp. | $9,399 | $11,383 | | **Total R&D Expenses** | **$28,088** | **$45,985** | - The **$17.9 million** decrease in R&D expenses from 2021 to 2022 was primarily due to the **$12.5 million** tuspetinib license fee in 2021, a **$10.1 million** decrease in luxeptinib program costs, and a **$3.4 million** decrease in APTO-253 costs, partially offset by a **$10.0 million** increase in tuspetinib program costs[279](index=279&type=chunk) [Controls and Procedures](index=54&type=section&id=ITEM%209A.%20CONTROLS%20AND%20PROCEDURES) Management concluded that disclosure controls and internal control over financial reporting were effective as of December 31, 2022, with no material changes in Q4 2022 - Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2022[299](index=299&type=chunk) - Based on the COSO 2013 framework, management concluded that internal control over financial reporting was effective as of December 31, 2022[303](index=303&type=chunk) Part III [Directors, Compensation, Ownership, and Fees](index=56&type=section&id=ITEM%2010%2C%2011%2C%2012%2C%2013%2C%2014) Information for Items 10-14, covering directors, compensation, ownership, and fees, is incorporated by reference from the 2023 Proxy Statement - The information required for Items 10 through 14 is incorporated by reference from the definitive Proxy Statement for the 2023 Annual Meeting of Shareholders[306](index=306&type=chunk) Part IV [Exhibits and Financial Statement Schedules](index=57&type=section&id=ITEM%2015.%20EXHIBITS%2C%20FINANCIAL%20STATEMENT%20SCHEDULES) This section includes consolidated financial statements for 2022 and 2021, and KPMG LLP's auditor report, which highlights a "Going Concern" uncertainty - The Report of Independent Registered Public Accounting Firm (KPMG LLP) includes a "Going Concern" paragraph, citing recurring losses that raise substantial doubt about the company's ability to continue as a going concern[330](index=330&type=chunk) - The auditor's report identifies "Research and Development Prepaid and Accrued Costs" as a Critical Audit Matter due to subjective and complex estimation judgments[336](index=336&type=chunk)[337](index=337&type=chunk) Consolidated Statements of Financial Position (in thousands) | | Dec 31, 2022 | Dec 31, 2021 | | :--- | :--- | :--- | | **Total current assets** | **$49,519** | **$81,737** | | **Total assets** | **$51,027** | **$82,525** | | **Total current liabilities** | **$12,284** | **$8,174** | | **Total liabilities** | **$13,286** | **$8,289** | | **Total shareholders' equity** | **$37,741** | **$74,236** | Consolidated Statements of Loss and Comprehensive Loss (in thousands) | | Year ended Dec 31, 2022 | Year ended Dec 31, 2021 | | :--- | :--- | :--- | | Research and development | $28,088 | $45,985 | | General and administrative | $14,514 | $19,462 | | **Net loss** | **$(41,823)** | **$(65,354)** |

Aptose Biosciences Inc. (NASDAQ:APTO) Q3 2022 Results Conference Call November 1, 2022 5:00 PM ET Company Participants Susan Pietropaolo - Managing Director William Rice - Chairman, President and CEO Fletcher Payne - SVP and Chief Financial Officer Rafael Bejar - SVP and Chief Medical Officer Conference Call Participants Li Watsek - Cantor Fitzgerald Soumit Roy - Jones Trading John Newman - Canaccord Genuity Matthew Biegler - Oppenheimer Joe Pantginis - H.C. Wainwright Edward Tenthoff - Piper Sandler Operat ...

Aptose Biosciences Inc. (NASDAQ:APTO) Q2 2022 Earnings Conference Call August 2, 2022 5:00 PM ET Company Participants Susan Pietropaolo - Managing Director William Rice - Chairman, President and CEO Fletcher Payne - SVP and Chief Financial Officer Rafael Bejar - SVP and Chief Medical Officer Conference Call Participants Matthew Biegler - Oppenheimer Li Watsek - Cantor Fitzgerald Edward Tenthoff - Piper Sandler Soumit Roy - Jones Trading John Newman - Canaccord Genuity Operator Good day, and thank you for st ...

Financial Performance - Net loss for the three months ended March 31, 2022, was $11,481 thousand, compared to a net loss of $16,227 thousand for the same period in 2021, reflecting an improvement of approximately 29.5%[18] - Basic and diluted loss per common share improved from $(0.18) in Q1 2021 to $(0.12) in Q1 2022[18] - The net loss for the three-month period ended March 31, 2022, decreased by $4.7 million to $11.5 million compared to $16.2 million for the same period in 2021[126] - Cash used in operating activities for the three-month period ended March 31, 2022, was approximately $9.6 million, compared to $10.4 million for the same period in 2021[118] - General and administrative expenses for the three-month period ended March 31, 2022, were $4,107 thousand, compared to $8,024 thousand in the same period in 2021[128] Assets and Liabilities - Total assets decreased from $82,525 thousand on December 31, 2021, to $72,603 thousand on March 31, 2022, representing a decline of approximately 12.3%[14] - Current assets fell from $81,737 thousand to $71,874 thousand, a decrease of about 12.0%[14] - Total liabilities decreased from $8,289 thousand on December 31, 2021, to $7,319 thousand on March 31, 2022, a reduction of about 11.7%[15] - Shareholders' equity decreased from $74,236 thousand to $65,284 thousand, a decline of approximately 12.1%[15] - Cash and cash equivalents decreased from $39,114 thousand at the beginning of the period to $36,991 thousand at the end of the period, a decline of approximately 5.4%[24] Research and Development - The company is advancing two clinical-stage programs and a third program in discovery-stage, focusing on differentiated kinase inhibitors for oncology[28] - Research and development expenses decreased from $8,228 thousand in Q1 2021 to $7,393 thousand in Q1 2022, a reduction of about 10.1%[18] - Research and development expenses for the three-month period ended March 31, 2022, were $7.4 million, a decrease of $835 thousand from $8.2 million in the same period in 2021[132] - The ongoing COVID-19 pandemic may adversely affect the company's research and development activities, including clinical trial enrollment and data collection[29] - The company has ongoing Phase 1 clinical trials for product candidates HM43239 and luxeptinib, with increased costs expected for these trials[129] Stock and Compensation - The Company recorded total share-based payment expense of $2.514 million in Q1 2022, down 62.2% from $6.643 million in Q1 2021[77] - Stock-based compensation decreased by approximately $3.7 million to $1.6 million for the three months ended March 31, 2022, primarily due to lower grant date fair value of options[137] - The Company granted 3,870 thousand stock options in Q1 2022, compared to 2,962 thousand in Q1 2021, reflecting a 30.6% increase[75] - As of March 31, 2022, there was $6.99 million of total unrecognized compensation cost related to non-vested stock options, expected to be recognized over an estimated weighted-average period of 1.67 years[69] Clinical Trials and Product Development - Aptose obtained exclusive worldwide rights to the clinical-stage myeloid kinome inhibitor HM43239 from Hanmi Pharmaceutical on November 4, 2021[88] - An ongoing international Phase 1/2 clinical trial for HM43239 has shown robust clinical activity, including multiple complete remissions (CR) in relapsed or refractory AML patients[89] - The FDA granted orphan drug designation to HM43239 for AML treatment, providing seven additional years of marketing exclusivity and various development incentives[90] - Fast Track Designation was granted to HM43239 for relapsed or refractory AML with FLT3 mutation, facilitating expedited development through early communication with the FDA[91] - Luxeptinib is being developed for relapsed or refractory AML and B-cell malignancies, with ongoing Phase 1a/b trials showing promising safety and efficacy results[94][95] Future Outlook and Capital Needs - The company expects to need to raise additional capital or incur indebtedness to fund operations in the future, with a base shelf prospectus allowing for the distribution of up to $200 million of common shares[111] - The company anticipates that negative cash flows will continue until regulatory approval is received for any of its products under development[119] - The company’s cash needs for the next twelve months will depend on clinical trial enrollment rates and general corporate overhead costs[31] Miscellaneous - The company has not experienced material delays in the enrollment of patients or timelines for clinical trials due to COVID-19[138] - The company has not been party to any off-balance sheet arrangements as of March 31, 2022[144] - There were no material changes to the company's critical accounting policies and estimates during the three months ended March 31, 2022[146] - The report was signed by William G. Rice, Ph.D., President and Chief Executive Officer, on May 9, 2022, in San Diego, California[165].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended September 30, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period from to Commission File Number: 1-35447 APTOSE BIOSCIENCES INC. (Exact Name of Registrant as Specified in Its Charter) Canada (State or other jurisdiction of incorporatio ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended June 30, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period from to Commission File Number: 1-35447 APTOSE BIOSCIENCES INC. (Exact Name of Registrant as Specified in Its Charter) Canada (State or other jurisdiction of incorporation or ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended March 31, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period from to Commission File Number: 1-35447 APTOSE BIOSCIENCES INC. (Exact Name of Registrant as Specified in Its Charter) Canada (State or other jurisdiction of incorporation or ...