OncoVerity Expands Series A to Progress Cusatuzumab for New AML Cases

OncoVerity, a company known for its innovative use of bioinformatics in drug development, has announced the completion of a Series A extension financing. This funding round was led by current investors, argenx and RefinedScience. The funds will be used to support the Phase 2 OV-AML-1231 trial, which is testing cusatuzumab, a high-affinity monoclonal antibody, in combination with venetoclax and azacitidine for newly diagnosed acute myeloid leukemia (AML) patients. This trial specifically includes patients who have limited treatment options and are likely to respond to this new regimen based on OncoVerity’s computational analysis. Patient enrollment is progressing, with the first eight patients already dosed.

Max Colao, CEO of OncoVerity, emphasized that this financing is vital for advancing cusatuzumab's development. He expressed optimism about the potential of this therapy to treat various tumors, particularly highlighting its applicability in AML. Colao also mentioned that interim analysis results from the Phase 2 trial are expected in the second half of 2025, which could bring a significant new treatment option closer to reality for AML patients.

The OV-AML-1231 trial is a randomized, open-label, multicenter, multinational Phase 2 study. It aims to evaluate the safety and efficacy of the combination of cusatuzumab, venetoclax, and azacitidine (VAC) compared to venetoclax and azacitidine (VA) alone in newly diagnosed AML patients who are not eligible for intensive chemotherapy. The trial plans to enroll 120 patients, with a 2:1 randomization—80 patients receiving the VAC combination and 40 receiving the VA regimen. The trial is particularly focused on patients with adverse cytogenetic and molecular risk features. The primary endpoint of the trial is overall survival, with secondary endpoints including event-free survival, response rates, and rates of minimal residual disease (MRD) negativity.

Cusatuzumab is a first-in-class, high-affinity humanized monoclonal antibody that targets CD70, an immune checkpoint involved in various hematological malignancies, solid tumors, and autoimmune diseases. The antibody is designed to block CD70, kill cancer cells expressing CD70 through multiple mechanisms, and restore immune surveillance against solid tumors. Current evaluations of cusatuzumab include a Phase 2 trial in combination with azacitidine and a Phase 1b trial in combination with azacitidine and venetoclax for newly diagnosed AML patients.

Acute myeloid leukemia (AML) is a rapidly progressing cancer of the bone marrow and blood. It is a heterogeneous disease with a variable prognosis among different patient populations. Despite medical advancements, AML remains a significant challenge to treat effectively. The five-year survival rate for AML patients is the lowest among blood cancers, ranging from 5% to 20%, and relapse is common. There is a pressing need for innovative therapies that can improve survival rates, reduce toxicity, and target specific AML subtypes more effectively.

OncoVerity leverages computational tools to advance cancer therapies. By using single-cell multi-omics and machine learning technologies, OncoVerity aims to identify the biological drivers of therapeutic resistance and patient-specific risk factors. This approach not only promises to accelerate the development of cusatuzumab for AML but also holds potential for broader applications in other hematologic malignancies, offering hope to a wider range of cancer patients.