This study is being done to evaluate the safety, tolerability and antitumor activity of oral CG-806 (luxeptinib) for the treatment of patients with Acute Myeloid Leukemia (except APML), secondary AML, therapy-related AML, or higher-risk MDS, whose disease has relapsed, is refractory or who are ineligible for or intolerant of intensive chemotherapy or transplantation.

Start Date06 Oct 2020

Sponsor / Collaborator

Aptose Biosciences, Inc.

NCT03893682

/ TerminatedPhase 1

A Phase Ia/b Trial to Evaluate the Safety and Tolerability of CG-806 in Patients With CLL/SLL or Non-Hodgkin's Lymphomas

This study is being done to evaluate the safety, tolerability and effectiveness of Oral CG-806 for the treatment of patients with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), or Non-Hodgkin's Lymphomas who have failed or are intolerant to two or more lines of established therapy or for whom no other treatment options are available.

Start Date30 Apr 2019

Sponsor / Collaborator

Aptose Biosciences, Inc.

NCT03850574

/ RecruitingPhase 1/2

A Phase 1/2 Open Label, Multicenter, Dose Escalation and Expansion Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of HM43239 in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)

The main purpose of this study is to identify a safe and potentially effective dose of tuspetinib to be used in future studies in study participants diagnosed with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) that is relapsed or refractory after at least one line of prior therapy, or in study participants with newly diagnosed AML. Tuspetinib will be administered as a single agent or in combination with other drugs (venetoclax or venetoclax plus azacitidine), as specified for each part of the study.

Start Date11 Mar 2019

Sponsor / Collaborator

Aptose Biosciences, Inc.

100 Clinical Results associated with Aptose Biosciences, Inc.

0 Patents (Medical) associated with Aptose Biosciences, Inc.

Literatures (Medical) associated with Aptose Biosciences, Inc.

01 Jan 2025·Cancer research communications

Preclinical Development of Tuspetinib for the Treatment of Acute Myeloid Leukemia

Article

Author: Bejar, Raphael ; Jung, Seung Hyun ; Rice, William G. ; Sonowal, Himangshu ; Howell, Stephen B. ; Sinha, Ranjeet ; Byun, Joo-Yun

Abstract:

Tuspetinib (TUS) is a well-tolerated, once daily, oral kinase inhibitor in clinical development for treatment of acute myeloid leukemia (AML). Nonclinical studies show that TUS targets key prosurvival kinases with IC50 values in the low nmol/L range, including SYK, wild-type (WT) and mutant forms of FLT3, mutant but not WT forms of KIT, RSK2, and TAK1–TAB1 kinases, and indirectly suppresses expression of MCL1. Oral TUS markedly extended survival in subcutaneously and orthotopically inoculated xenograft models of FLT3-mutant human AML, was well tolerated, and delivered enhanced activity when combined with venetoclax (VEN) or 5-azacytidine. In vitro, TUS demonstrated potent killing of AML lines [concentration needed to reduce the growth of treated cells to half that of untreated cells (GI50) = 1.3–5.2 nmol/L] and Ba/F3 cells expressing WT (GI50 = 9.1 nmol/L) or various mutant forms of FLT3 (GI50 = 2.5–56 nmol/L). In AML lines, the multikinase targeting capacity of TUS suppressed phosphorylation of SYK, FLT3, STAT5, MEK, ERK, AKT, mTOR, 4E-BP1, and S6K kinases. Cells selected for stable acquired resistance to TUS exhibited increased BAX and hypersensitivity to VEN (1900 fold), navitoclax, and MCL1 inhibitors. MV-4-11 FLT3-ITD clones expressing NRASG12D revealed that high-level expression of NRASG12D generated modest resistance to TUS and greater resistance to VEN, yet the TUS/VEN combination exhibited synergy in the NRASG12D AML model. Favorable preclinical safety and pharmacology properties, the efficacy of the TUS/VEN combination in a murine model, and the synthetic lethal vulnerability to VEN that accompanies TUS resistance provide the basis for exploration of the TUS/VEN combination in patients with relapsed or refractory AML.

Significance::

This article reports preclinical development of TUS, an oral kinase inhibitor currently in clinical development for treatment of AML. The article covers the studies of TUS activities on cellular targets and the nonclinical studies that supported the advancement of TUS to a phase I/II trial of TUS/VEN in refractory AML and a phase I/II trial of TUS/VEN/5-azacytidine in newly diagnosed patients with AML (NCT03850574).

05 Jul 2022·MOLECULAR CANCER THERAPEUTICSQ2 · MEDICINE

Luxeptinib (CG-806) Targets FLT3 and Clusters of Kinases Operative in Acute Myeloid Leukemia

Q2 · MEDICINE

Article

Author: Tyner, Jeffrey W. ; Lo, Pierrette ; Rastgoo, Nasrin ; Druker, Brian J. ; Howell, Stephen B. ; Local, Andrea ; Rice, William G. ; Kurtz, Stephen E. ; Bottomly, Daniel ; McWeeney, Shannon K. ; Wilmot, Beth ; Zhang, Hongying

Abstract:

Luxeptinib (CG-806) simultaneously targets FLT3 and select other kinase pathways operative in myeloid malignancies. We investigated the range of kinases it inhibits, its cytotoxicity landscape ex vivo with acute myeloid leukemia (AML) patient samples, and its efficacy in xenograft models. Luxeptinib inhibits wild-type (WT) and many of the clinically relevant mutant forms of FLT3 at low nanomolar concentrations. It is a more potent inhibitor of the activity of FLT3—internal tandem duplication, FLT3 kinase domain and gatekeeper mutants than against WT FLT3. Broad kinase screens disclosed that it also inhibits other kinases that can drive oncogenic signaling and rescue pathways, but spares kinases known to be associated with clinical toxicity. In vitro profiling of luxeptinib against 186 AML fresh patient samples demonstrated greater potency relative to other FLT3 inhibitors, including cases with mutations in FLT3, isocitrate dehydrogenase-1/2, ASXL1, NPM1, SRSF2, TP53, or RAS, and activity was documented in a xenograft AML model. Luxeptinib administered continuously orally every 12 hours at a dose that yielded a mean Cmin plasma concentration of 1.0 ± 0.3 μmol/L (SEM) demonstrated strong antitumor activity but no myelosuppression or evidence of tissue damage in mice or dogs in acute toxicology studies. On the basis of these studies, luxeptinib was advanced into a phase I trial for patients with AML and myelodysplastic/myeloproliferative neoplasms.

01 Jan 2022·BIOCHEMICAL PHARMACOLOGYQ2 · MEDICINE

Luxeptinib disables NLRP3 inflammasome-mediated IL-1β release and pathways required for secretion of inflammatory cytokines IL-6 and TNFα

Q2 · MEDICINE

Article

Author: Howell, Stephen B ; Rice, William ; Sonowal, Himangshu ; Zhang, Hongying

Luxeptinib (CG-806) is an orally bioavailable multikinase inhibitor with nanomolar potency against select clusters of kinases including the BTK, FLT3, TRK, STE/MAPK and aurora kinase clusters. It is cytotoxic to primary malignant cells obtained from patients with AML, ALL, and CLL at lower concentrations than other BTK and FLT3 inhibitors, and has activity in AML and lymphoma xenografts at concentrations attainable in patients. Exposure of macrophages and monocytes to endotoxin triggers the release of IL-1β through activation of the NLRP3 inflammasome and IL-6 and TNFα through transcriptional up-regulation. These cytokines are key components of the innate immune signaling network that plays a central role in the pathogenesis of multiple human diseases including cancer. Drugs that concurrently inhibit proliferation and inflammatory signaling pathways may provide better therapeutic efficacy. The aim of this study was to determine the extent to which luxeptinib interferes with the release of IL-1β, IL-6 and TNFα from THP-1 monocytes and bone marrow-derived macrophages following endotoxin exposure and priming of the NLRP3 inflammasome. Luxeptinib inhibited the release of all 3 cytokines from THP-1 monocytes and macrophages at concentrations of 0.1 µM and above. Investigation of the mechanism disclosed that luxeptinib does not inhibit the assembly of the NLRP3 inflammasome but disables its ability to cleave and activate caspase-1 that is required for IL-1β release. It also inhibits the kinases p38MAPK, ERK1/2, SAPK/JNK and activation of transcription factor NF-κBp65 with a concentration profile similar to its inhibition of cytokine release. IMPLICATIONS: The ability of luxeptinib to inhibit the NLRP3-mediated release of IL-1β and pathways involved in the release of IL-6 and TNFα at concentrations which are well-tolerated in patients makes it a candidate for the treatment of inflammatory diseases and inflammation-associated resistance in cancer.

117

News (Medical) associated with Aptose Biosciences, Inc.

15 Jul 2025

·www.aptose.com

Aptose Receives Second Advance under the Loan Agreement with Hanmi Pharmaceutical to Continue Development of Tuspetinib in Triplet Therapy for AML

SAN DIEGO and TORONTO, July 15, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. ("Aptose" or the "Company") (TSX: APS; 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), today announced that it has received an additional advance of US$2.0M from Hanmi Pharmaceutical Co. Ltd. (“Hanmi”), as part of a US$8.5M loan facility agreement with Hanmi (the “Loan Agreement”) announced prior on June 20, 2025 (press release here). To date, Aptose has received an aggregate of US$4.5M under the Loan Agreement. “Tuspetinib in combination with venetoclax and azacitidine (the TUS+VEN+AZA triplet) continues to demonstrate exciting antileukemic activity and safety across genetically diverse populations of newly diagnosed AML patients – including TP53-mutated AML and wildtype AML, representing large AML populations for which there are few treatment options,” said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. “We are very grateful to Hanmi for its continued support to help advance tuspetinib clinical development and further extend Aptose's ability to fund this important study.” Tuspetinib is a convenient once daily oral agent that potently inhibits targets that drive excessive proliferation and anti-apoptotic mechanisms, including SYK, mutated and wild type forms of FLT3, mutated KIT, JAK1/2, and RSK2 kinases. Yet, TUS maintains a favorable safety profile by avoiding typical toxicity concerns observed with other agents. The ongoing TUSCANY triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of azacitidine and venetoclax in newly diagnosed patients with AML who are ineligible to receive induction chemotherapy. At the European Hematology Association Congress in June, Aptose reported early data from the first two dose cohorts that have demonstrated safety, CRs and minimal residual disease (MRD) negativity across patients with diverse mutations (press release here). About Aptose Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology. The Company's small molecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies and regimens without overlapping toxicities. The Company’s lead clinical-stage compound tuspetinib (TUS), is an oral kinase inhibitor that has demonstrated activity as a monotherapy and in combination therapy in patients with relapsed or refractory acute myeloid leukemia (AML) and is being developed as a frontline triplet therapy in newly diagnosed AML. For more information, please visit www.aptose.com. Forward Looking Statements This press release may contain forward-looking statements within the meaning of Canadian and U.S. securities laws, including statements relating but not limited to, the use of proceeds of the Loan Facility Agreement, the development of tuspetinib, and statements relating to the Company’s plans, objectives, expectations and intentions and other statements including words such as “continue”, “expect”, “intend”, “will”, “should”, “would”, “may”, and other similar expressions. Such statements reflect our current views with respect to future events and are subject to risks and uncertainties and are necessarily based upon a number of estimates and assumptions that, while considered reasonable by us are inherently subject to significant business, economic, competitive, political and social uncertainties and contingencies. Many factors could cause our actual results, performance or achievements to be materially different from any future results, performance or achievements described in this press release. Such factors could include, among others: our ability to negotiate a collaboration agreement to jointly develop tuspetinib with Hanmi, our ability to remain compliant with Nasdaq listing requirements and other risks detailed from time-to-time in our ongoing quarterly filings, annual information forms, annual reports and annual filings with Canadian securities regulators and the United States Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should the assumptions set out in the section entitled "Risk Factors" in our filings with Canadian securities regulators and the United States Securities and Exchange Commission underlying those forward-looking statements prove incorrect, actual results may vary materially from those described herein. These forward-looking statements are made as of the date of this press release and we do not intend, and do not assume any obligation, to update these forward-looking statements, except as required by law. We cannot assure you that such statements will prove to be accurate as actual results and future events could differ materially from those anticipated in such statements. Investors are cautioned that forward-looking statements are not guarantees of future performance and accordingly investors are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty therein. For further information, please contact: Aptose Biosciences Inc. Susan Pietropaolo Corporate Communications & Investor Relations 201-923-2049 spietropaolo@aptose.com Released July 15, 2025

License out/in

01 Jul 2025

·www.globenewswire.com

Aptose Upgraded to Trade on OTCQB Market

Aptose will trade under the ticker symbol APTOF and begin trading on OTCQB today, July 1stSAN DIEGO and TORONTO, July 01, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (TSX: APS; 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), announced today that the Company has been upgraded to list for trading on the OTCQB Market under the ticker “APTOF,” in addition to the Company’s continued listing on the Toronto Stock Exchange (TSX) under the symbol “APS”. Trading on OTCQB begins today, July 1, 2025. The OTCQB Venture Market is for early stage and developing U.S. and international companies. Companies are current in their reporting and undergo an annual verification and management certification process. Investors can find Real-Time quotes and market information for the company on www.otcmarkets.com. About Aptose Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology. The Company’s lead clinical-stage, oral kinase inhibitor tuspetinib (TUS) has demonstrated activity as a monotherapy and in combination therapy in patients with relapsed or refractory acute myeloid leukemia (AML) and is being developed as a frontline triplet therapy in newly diagnosed AML. For more information, please visit www.aptose.com. Forward Looking Statements This press release may contain forward-looking statements within the meaning of Canadian and U.S. securities laws, including, but not limited to, statements relating to the Company’s plans, objectives, expectations and intentions and other statements including words such as “continue”, “expect”, “intend”, “will”, “should”, “would”, “may”, and other similar expressions. Such statements reflect our current views with respect to future events and are subject to risks and uncertainties and are necessarily based upon a number of estimates and assumptions that, while considered reasonable by us are inherently subject to significant business, economic, competitive, political and social uncertainties and contingencies. These risks and uncertainties include, among others: our ability to obtain the capital required for research and operations and to continue as a going concern; the inherent risks in early stage drug development including demonstrating efficacy; development time/cost and the regulatory approval process; the progress of our clinical trials; our ability to find and enter into agreements with potential partners; our ability to attract and retain key personnel; changing market conditions; inability of new manufacturers to produce acceptable batches of GMP in sufficient quantities; unexpected manufacturing defects; and other risks detailed from time-to-time in our ongoing quarterly filings, annual information forms, annual reports and annual filings with Canadian securities regulators and the United States Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should the assumptions set out in the section entitled “Risk Factors” in our filings with Canadian securities regulators and the United States Securities and Exchange Commission underlying those forward-looking statements prove incorrect, actual results may vary materially from those described herein. These forward-looking statements are made as of the date of this press release and we do not intend, and do not assume any obligation, to update these forward-looking statements, except as required by law. We cannot assure you that such statements will prove to be accurate as actual results and future events could differ materially from those anticipated in such statements. Investors are cautioned that forward-looking statements are not guarantees of future performance and accordingly investors are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty therein. For further information, please contact Aptose Biosciences Inc.Susan PietropaoloCorporate Communications & Investor Relations201-923-2049spietropaolo@aptose.com

30 Jun 2025

·www.aptose.com

Aptose Announces Deferral of Interest Payment

SAN DIEGO and TORONTO, June 30, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. ("Aptose" or the "Company") (TSX: APS; 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), announced that it has entered into an Interest Deferral Agreement (the “Agreement”) with Hanmi Pharmaceutical Co., Ltd. (“Hanmi”), whereby Hanmi has agreed to further defer the interest payment due under the loan facility agreement dated August 27, 2024 (the “2024 Loan Agreement”) between Aptose and Hanmi for the period commencing December 21, 2024 and ending on March 31, 2025 from June 27, 2025 to December 31, 2025 and to defer the interest due under the 2024 Loan Agreement for the period commencing March 31, 2025 and ending on June 30, 2025 to December 31, 2025. About Aptose Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology. The Company's small molecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies and regimens without overlapping toxicities. The Company’s lead clinical-stage compound tuspetinib (TUS), is an oral kinase inhibitor that has demonstrated activity as a monotherapy and in combination therapy in patients with relapsed or refractory acute myeloid leukemia (AML) and is being developed as a frontline triplet therapy in newly diagnosed AML. For more information, please visit www.aptose.com. Forward Looking Statements This press release may contain forward-looking statements within the meaning of Canadian and U.S. securities laws, including statements relating but not limited to statements relating to the Company’s plans, objectives, expectations and intentions and other statements including words such as “continue”, “expect”, “intend”, “will”, “should”, “would”, “may”, and other similar expressions. Such statements reflect our current views with respect to future events and are subject to risks and uncertainties and are necessarily based upon a number of estimates and assumptions that, while considered reasonable by us are inherently subject to significant business, economic, competitive, political and social uncertainties and contingencies. Many factors could cause our actual results, performance or achievements to be materially different from any future results, performance or achievements described in this press release. Such factors could include, among others: our ability to negotiate a collaboration agreement to jointly develop tuspetinib with Hanmi, our ability to remain compliant with Nasdaq listing requirements and other risks detailed from time-to-time in our ongoing quarterly filings, annual information forms, annual reports and annual filings with Canadian securities regulators and the United States Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should the assumptions set out in the section entitled "Risk Factors" in our filings with Canadian securities regulators and the United States Securities and Exchange Commission underlying those forward-looking statements prove incorrect, actual results may vary materially from those described herein. These forward-looking statements are made as of the date of this press release and we do not intend, and do not assume any obligation, to update these forward-looking statements, except as required by law. We cannot assure you that such statements will prove to be accurate as actual results and future events could differ materially from those anticipated in such statements. Investors are cautioned that forward-looking statements are not guarantees of future performance and accordingly investors are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty therein. For further information, please contact: Aptose Biosciences Inc. Susan Pietropaolo Corporate Communications & Investor Relations 201-923-2049 spietropaolo@aptose.com Released June 30, 2025

100 Deals associated with Aptose Biosciences, Inc.

100 Translational Medicine associated with Aptose Biosciences, Inc.

Corporation Tree

Boost your research with our corporation tree data.

view full example data

Pipeline

Pipeline Snapshot as of 24 Jul 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Phase 2

Approved

Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

Tuspetinib ( FLT3 x JAK1 x JAK2 x Syk )	Adult Acute Myeloblastic Leukemia More	Phase 1/2
Luxeptinib ( BTK x FLT3 )	Chronic lymphocytic leukaemia refractory More	Discontinued
LOR-2501 ( RNR1 )	Solid tumor More	Discontinued
APL-581 ( BRD4 x JAK )	Hematologic Neoplasms More	Discontinued
APTO-253 ( c-Myc )	Solid tumor More	Pending

Deal

Boost your decision using our deal data.

view full example data

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Profit

Explore the financial positions of over 360K organizations with Synapse.

view full example data

Grant & Funding(NIH)

Access more than 2 million grant and funding information to elevate your research journey.

view full example data

Investment

Gain insights on the latest company investments from start-ups to established corporations.

view full example data

Financing

Unearth financing trends to validate and advance investment opportunities.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis