On April 10, 2025 Omeros Corporation (Nasdaq: OMER) reported the establishment of the Omeros Oncology Clinical Steering Committee to advance Omeros’ OncotoX biologics program focused on acute myeloid leukemia (AML) (Press release, Omeros, APR 10, 2025, View Source [SID1234651874]). AML is the most fatal form of leukemia and accounts for approximately 80 percent of acute leukemias in adults and one-third of all cancers affecting the blood/bone marrow, representing a high unmet need. Omeros’ OncotoX program for AML consists of proprietary targeted, engineered molecules (about half the size of an antibody) that deliver a toxic payload within the cancer cells, thereby killing them.

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Members of the Omeros Oncology Clinical Steering Committee include:

Naval Daver, M.D., Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center (Chair of the Omeros Oncology Clinical Steering Committee)
Aref Al-Kali, M.D., Chair of the Acute Myeloid Group and Research Chair of the Acute Leukemia and Myeloid Neoplasms Disease Group at Mayo Clinic
Jessica K. Altman, M.D., Professor of Medicine in the Division of Hematology and Oncology and Director of the Leukemia Program of Robert H. Lurie Comprehensive Cancer Center at Northwestern University
Jayastu Senapati, M.B.B.S., M.D., D.M., Assistant Professor in the Department of Leukemia at MD Anderson Cancer Center
Mithun Shah, M.D., Ph.D., Assistant Professor of Medicine and Oncology, and Consultant in the Division of Hematology at Mayo Clinic
Anthony S. Stein, M.D., Co-director of the Gehr Family Center for Leukemia Research within the Hematologic Malignancies and Stem Cell Transplantation Institute at City of Hope
Eytan M. Stein, M.D., Chief of the Leukemia Service and Director of the Program for Drug Development in Leukemia in the Division of Hematologic Malignancies at Memorial Sloan Kettering Cancer Center
Roland B. Walter, M.D., Ph.D., M.S., Professor in the Translational Science and Therapeutics Division and José Carreras/E. Donnall Thomas Endowed Chair for Cancer Research at the Fred Hutchinson Cancer Center
The steering committee will, among other activities, assist Omeros with development of the OncotoX-AML program, clinical trial design and interactions with institutional review boards and will participate in OncotoX-AML 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 Naval Daver, M.D., Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center and Chair of the Omeros Oncology Clinical Steering Committee. "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, and its potential utility extends broadly beyond AML to other types of leukemia. Together with my esteemed physician-scientist colleagues on the steering committee, I look forward to advancing the Omeros therapeutic for AML and bringing this novel treatment to patients as quickly as possible."

Across extensive in vivo and ex vivo studies, the OncotoX-AML therapeutic has shown to be highly effective even at very low doses, providing a significant survival benefit over currently approved combination therapy (venetoclax and azacytidine). These studies, conducted with human cell lines in well-established animal models and considered predictive of clinical response, have targeted AML tumors with mutational backgrounds commonly found in nearly 90 percent of AML patients (TP53, NPM1, KMT2a, and FLT3) indicating that the OncotoX-AML therapeutic could be "mutation-agnostic," a currently large unmet need for oncologists and their patients. In studies using primary AML derived from patients, the OncotoX molecule preferentially and efficiently kills AML blasts (abnormal myeloid cells). OncotoX-AML also targets leukemia stem cells (LSC), which are often refractory to chemotherapy and represent another major challenge in the treatment of AML. The OncotoX-AML therapeutic is designed specifically to kill both AML blasts and LSCs that can lead to relapse. Preliminary in vivo tolerability studies demonstrate that the OncotoX therapeutic is well tolerated at doses substantially greater than one order of magnitude above efficacious doses without causing neutropenia or meaningful changes in blood chemistry values.

Omeros is initiating IND-enabling work for its OncotoX-AML therapeutic. Clinical efficacy in AML, considered one of the most aggressive leukemias, is thought to bode well for a therapeutic agent’s broad applicability across leukemias. In addition to OncotoX-AML, Omeros’ portfolio of oncology platforms include signaling and antigen-driven immunomodulators, immune memory-enhancing immunostimulators, and an adoptive T-cell technology that, unlike other cell therapy approaches, does not require cellular engineering.

"We are very pleased to be working with all the members of our oncology steering committee," said Gregory A. Demopulos, M.D., Omeros’ Chairman and Chief Executive Officer. "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 and, with their help, we look forward to assessing this next-generation therapeutic in AML patients."

In the U.S. alone, AML is diagnosed in over 20,000 patients annually and is responsible each year for more than 11,000 deaths. In 2030, the global AML therapeutic market size is projected to be over $6 billion with the leukemia therapeutic market forecast at $29 billion.