First patients dosed in OV-AML-1231, a Phase 2 randomized controlled trial of cusatuzumab in newly diagnosed AML

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.

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