Azacitidine

First patients dosed in OV-AML-1231, a Phase 2 randomized controlled trial of cusatuzumab in newly diagnosed AML AURORA, CO, USA I November 21, 2024 I OncoVerity, a pioneer in applying advanced bioinformatics to drug development, announced today the closing of a Series A extension financing led by existing investors, argenx and RefinedScience. With this funding, OncoVerity aims to advance its mission to address the unmet medical need of cancer patients, leveraging a unique computational approach to inform a development strategy that seeks breadth and depth of response. The proceeds from the Series A extension will fund the Phase 2 OV-AML-1231 trial of cusatuzumab, a novel, first-in-class high-affinity monoclonal anti-CD70 antibody, in combination with venetoclax and azacitidine for the treatment of newly diagnosed acute myeloid leukemia (AML). The trial is enriched explicitly for patients with limited treatment options who show potential for responding to a cusatuzumab-based regimen, as informed by OncoVerity’s computational approach to guide its development. Patient enrollment is underway, and the first eight patients have been dosed. “This financing round is a critical step forward in accelerating the development of cusatuzumab,” said Max Colao, Chief Executive Officer at OncoVerity. “Cusatuzumab has the potential to treat a broad range of tumors, demonstrating a strong biologic rationale in AML. We are particularly excited about the potential of the Phase 2 trial, and look forward to sharing results from the interim analysis in the second half of 2025. These results will bring us closer to potentially offering a breakthrough new treatment option for AML patients. “This randomized phase 2 clinical trial was based on insights derived from machine learning-based approaches and will be analyzed using cutting-edge single cell omics technologies for depth and breadth of responses. This novel strategy was designed to optimize trial design and execution, address patient heterogeneity and other key patient specific needs and provide important insights into critical clinical and biologic areas. Together we hope this will help advance care for persons with AML and other cancers where there is urgent unmet medical need.” About OV-AML-1231 OV-AML-1231 is a randomized, open-label, multicenter, multinational, Phase 2 trial evaluating the safety and efficacy cusatuzumab combined with venetoclax, and azacitidine (VAC) to venetoclax and azacitidine (VA) in newly diagnosed AML patients not eligible for intensive chemotherapy. The planned enrollment is 120 newly diagnosed AML patients, randomized 2:1 to VAC (80 patients) and VA (40 patients). The trial population will be enriched for patients with adverse cytogenetic and molecular risk features. The primary end point is overall survival. Secondary endpoints include event-free survival, response rates including CR, CR+CRh, and rates of MRD negativity. About Cusatuzumab Cusatuzumab is a first-in-class high-affinity humanized monoclonal antibody targeting CD70, an immune checkpoint target involved in hematological malignancies, several solid tumors, and severe autoimmune diseases. Cusatuzumab is designed to: block CD70, kill cancer cells expressing CD70 through complement-dependent cytotoxicity, enhance antibody-dependent cell-mediated phagocytosis, enhance antibody-dependent cell-mediated cytotoxicity, and restore immune surveillance against solid tumors (Silence K. et al. mAbs 2014; 6 (2):523-532) . Cusatuzumab is currently being evaluated in patients with hematologic malignancies, including a Phase 2 trial in combination with azacitidine and a Phase 1b trial in combination with azacitidine and venetoclax in patients with newly diagnosed AML. About Acute Myeloid Leukemia Acute myeloid leukemia (AML) is a cancer of the bone marrow and the blood that progresses rapidly without treatment. It is a heterogeneous disease with variable prognosis for different patient subpopulations. Despite the advancements in medical research, there is still a significant need for new effective therapies to treat AML. The five-year survival rate for patients remains the lowest of all blood malignancies (5-20%), and relapse is common. Innovative therapies and targeted treatment approaches that can improve survival rates, reduce toxicity, and target specific subtypes of AML are urgently needed to address this unmet need. About OncoVerity OncoVerity is pioneering the use of computational tools applied to clinical and biological data to advance therapies that transform hope into reality for cancer patients. Through a strategic collaboration with Refined Science , OncoVerity’s approach leverages an advanced bioinformatics platform that applies single-cell multi-omics and machine learning technologies to a large AML patient database to identify biologic drivers to therapeutic resistance and insights on patient-specific risk factors. This approach not only holds promise for accelerating the development of cusatuzumab in AML, but also has the potential to be applied to its development in other hematologic malignancies, signifying a broader impact on the future of cancer treatment by bringing new hope to a wider range of patients. SOURCE: OncoVerity

TUSCANY study is open to enroll patients to receive TUS+VEN+AZA triplet at select US sitesFavorable safety and broad clinical activity make tuspetinib an ideal agent to combine with venetoclax and azacitidine to potentially address larger AML populationsStudy execution update is expected during ASH 2024 SAN DIEGO and TORONTO, Nov. 20, 2024 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral targeted agents to treat hematologic malignancies, today announced initiation of the TUSCANY study, tuspetinib (TUS) in combination therapy with azacitidine (AZA) and venetoclax (VEN) as a frontline triplet combination therapy for patients newly diagnosed with acute myeloid leukemia, or AML. The trial is being conducted at multiple U.S. clinical sites. Tuspetinib is being developed for frontline AML therapy as part of the TUS+VEN+AZA triplet for newly diagnosed AML patients ineligible to receive intensive chemotherapy. Tuspetinib, a convenient once daily oral agent that potently targets SYK, FLT3, mutated KIT, JAK1/2, and RSK2 kinases, avoids many typical toxicity concerns observed with other agents and has the potential to treat the larger AML populations, not just narrow subpopulations. In the recently conducted Phase 1/2 APTIVATE trial in a very ill and heavily pre-treated relapsed or refractory (R/R) AML population, tuspetinib as a single agent (TUS) and in combination with venetoclax (TUS+VEN) safely achieved broad activity across various difficult-to-treat AML subpopulations. This included patients with prior-VEN, prior-FLT3 inhibitor (FLT3i) and prior-hematopoietic stem cell transplantation (HSCT) therapies, those with highly adverse genetics - including mutations in TP53 and RAS genes, and those with mutated or unmutated (wildtype) FLT3 genes. “Initiation of the trial is a key milestone for Aptose. AML treatment has rapidly shifted to combination therapies, and we are pleased to include tuspetinib as part of TUS+VEN+AZA triplet combination therapy in patients with newly-diagnosed AML – representing a new patient population for TUS,” said William G. Rice, Chairman, President and Chief Executive Officer. “We thank our investigators for their enthusiasm and our clinical team for activating the TUSCANY triplet study. As one of our investigators noted, if TUS brings added efficacy to frontline treatment of a broad array of AML patients without the added toxicities that are plaguing some other agents, we may have a game changer in TUS.” TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 StudyThe triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard of care dosing of azacitidine and venetoclax. TUS will be administered in 28-day cycles, beginning with 40mg, with dose escalations planned after safety review of each dose level. A planned 12 sites in the US will enroll in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025. More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here). About AptoseAptose 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 has two clinical-stage oral kinase inhibitors under development for hematologic malignancies: tuspetinib (TUS), 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; and luxeptinib (CG-806), an oral, dual lymphoid and myeloid kinase inhibitor in Phase 1 a/b stage development for the treatment of patients with relapsed or refractory hematologic malignancies. For more information, please visit www.aptose.com. Forward Looking StatementsThis 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 therapeutic potential and safety profile of tuspetinib and its clinical development, expected study updates and milestones as well as 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”, “potential”, 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 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