OncoVerity, a leader in integrating bioinformatics with oncology drug development, has closed a Series A extension financing round, bolstered by its key investors, argenx and RefinedScience. The funds are directed toward the advancement of cusatuzumab, a first-in-class monoclonal anti-CD70 antibody, through the OV-AML-1231 Phase 2 clinical trial for newly diagnosed acute myeloid leukemia (AML) patients.

First patients dosed in Phase 2 trial

Patient enrolment is underway for OV-AML-1231, and eight patients have already received their initial doses. This randomized, open-label, multicenter trial aims to enrol 120 newly diagnosed AML patients ineligible for intensive chemotherapy. Participants will be randomized in a 2:1 ratio to receive a combination of cusatuzumab with venetoclax and azacitidine (VAC), or venetoclax and azacitidine alone (VA). The trial focuses on patients with adverse cytogenetic and molecular risk profiles, aiming to address unmet needs within this high-risk population.

The study’s primary endpoint is overall survival, while secondary metrics include event-free survival, complete remission (CR) rates, CR with partial hematologic recovery (CR+CRh), and minimal residual disease (MRD) negativity. These endpoints align with the trial’s ambition to provide meaningful clinical insights into the efficacy of cusatuzumab-based regimens.

Innovative bioinformatics driving personalized care

OncoVerity is leveraging a cutting-edge bioinformatics platform developed in collaboration with RefinedScience to optimize the clinical development of cusatuzumab. This computational approach integrates single-cell multi-omics and machine learning to analyze extensive AML patient datasets, uncovering insights into therapeutic resistance and patient-specific risk factors. By addressing the heterogeneity of AML, OncoVerity hopes to deliver more precise treatment strategies.

Cusatuzumab targets CD70, an immune checkpoint involved in hematologic malignancies and solid tumors. The antibody functions by inhibiting CD70 signaling, killing CD70-expressing cancer cells, and restoring immune surveillance. These mechanisms make it a promising candidate not only for AML but also for other cancers and autoimmune diseases.

Expanding the horizon for AML treatment

Max Colao, Chief Executive Officer at OncoVerity, emphasized the significance of the financing round in accelerating cusatuzumab’s development. He highlighted the biologic rationale of the therapy, which has shown promise in previous trials. Colao noted that interim results from the Phase 2 trial, expected in late 2025, could mark a transformative moment for AML treatment, bringing hope to patients with limited options.

The innovative trial design of OV-AML-1231, informed by machine learning, exemplifies a new era in oncology research. By addressing patient-specific challenges and exploring biologic drivers of therapeutic resistance, OncoVerity’s strategy aims to redefine standards of care for AML and potentially other cancers.

A hopeful outlook for patients

OncoVerity’s commitment to combining bioinformatics with clinical expertise represents a paradigm shift in how oncology drugs are developed. Cusatuzumab’s potential in AML underscores the broader application of these approaches, paving the way for future breakthroughs in hematologic malignancies. With the completion of the Series A extension, the company is well-positioned to bring this promising therapy closer to clinical practice, potentially transforming outcomes for patients worldwide.

  Acute myeloid leukemia, AML, argenx, azacitidine, bioinformatics, cancer research, CD70 antibody, clinical trials, cusatuzumab, hematologic malignancies, machine learning, Oncology, OncoVerity, Phase 2 trial, RefinedScience, Venetoclax