Omeros Corporation (Nasdaq: OMER) announced today the formation of a specialized Oncology Clinical Steering Committee to advance its OncotoX biologics program targeting acute myeloid leukemia (AML), a devastating blood cancer with limited treatment options.
The committee brings together eight distinguished clinical experts in AML from leading National Comprehensive Cancer Network Centers, including specialists from MD Anderson Cancer Center, Mayo Clinic, Northwestern University, City of Hope, Memorial Sloan Kettering Cancer Center, and Fred Hutchinson Cancer Center.
Elite Clinical Leadership Team
Naval Daver, M.D., Professor and Director of the Leukemia Research Alliance Program at MD Anderson Cancer Center, will chair the committee. The steering committee will play a crucial role in the development of the OncotoX-AML program, providing expertise on clinical trial design, institutional review board interactions, and direct participation in upcoming clinical trials.
"AML is a devastating disease, and these patients have difficulty tolerating the side effects of chemotherapeutic agents and current antibody-drug conjugates, which generally have a narrow therapeutic index," stated Dr. Daver. "The OncotoX platform is designed to kill only dividing cancer cells while sparing normal cells. In animal models using cell lines derived from AML patients, the OncotoX-AML molecule has shown superior efficacy to current standard of care in AML treatment."
Promising Preclinical Results
The OncotoX program for AML consists of proprietary targeted, engineered molecules approximately half the size of an antibody that deliver a toxic payload directly to cancer cells. Preclinical studies have demonstrated several key advantages:
- High efficacy even at very low doses
- Significant survival benefit over the current standard combination therapy (venetoclax and azacytidine)
- Effectiveness against tumors with mutations found in nearly 90% of AML patients (TP53, NPM1, KMT2a, and FLT3)
- Ability to target both AML blasts (abnormal myeloid cells) and leukemia stem cells (LSCs), which are often responsible for treatment resistance and relapse
- Excellent tolerability at doses substantially higher than therapeutic levels, without causing neutropenia or significant changes in blood chemistry
The "mutation-agnostic" nature of the therapy addresses a major unmet need in AML treatment, as current therapies often work only for specific genetic subtypes of the disease.
Addressing a Significant Market Need
AML represents a substantial unmet medical need, accounting for approximately 80 percent of acute leukemias in adults and one-third of all blood and bone marrow cancers. In the United States alone, over 20,000 patients are diagnosed with AML annually, resulting in more than 11,000 deaths each year.
The global AML therapeutic market is projected to exceed $6 billion by 2030, with the broader leukemia therapeutic market forecast to reach $29 billion.
Gregory A. Demopulos, M.D., Omeros' Chairman and Chief Executive Officer, emphasized the significance of the committee's formation: "Each is an internationally recognized leader in the fields of leukemia and AML and has been integrally involved in the development of new treatments for their patients. Their willingness to invest time and effort in our OncotoX-AML program is a strong endorsement of our molecule's potential."
Advancing to Clinical Development
Omeros is now initiating IND-enabling work for its OncotoX-AML therapeutic, preparing for the transition to clinical trials. Success in treating AML, considered one of the most aggressive leukemias, could indicate broader applicability across various leukemia types.
The OncotoX-AML program is part of Omeros' comprehensive oncology portfolio, which includes three distinct platforms: OncotoX, immunomodulators, and immunostimulators combined with adoptive T-cell therapy. Unlike some competing approaches, Omeros' adoptive T-cell technology does not require cellular engineering, potentially offering advantages in manufacturing and implementation.
Broader Oncology Strategy
Omeros' approach to oncology is informed by a deep understanding of both innate and adaptive immune systems. While acknowledging the significant progress made in oncology through targeted therapies, advanced biologics, and immunotherapies, the company aims to address remaining challenges in toxicity and efficacy.
The company's oncology platforms are designed to deliver more effective treatments with reduced toxicity by targeting both cell-surface and intracellular cancer antigens. By combining tumor antigens with potent adjuvants, these novel biologics specifically target and activate antigen-presenting cells, potentially creating lasting immune responses against cancer cells.
This comprehensive approach positions Omeros to potentially deliver more effective and safer treatments across both hematological malignancies and solid tumors, addressing significant unmet needs in cancer care.
