Omeros Announces the Omeros Oncology Clinical Steering Committee for AML to Help Guide Omeros’ Proprietary OncotoX-AML Clinical Program
Omeros (OMER) has established an Oncology Clinical Steering Committee to advance its OncotoX biologics program targeting acute myeloid leukemia (AML). The committee comprises distinguished clinical experts from leading National Comprehensive Cancer Network Centers, chaired by Dr. Naval Daver from MD Anderson Cancer Center.
The OncotoX-AML program features engineered molecules that deliver toxic payloads to cancer cells. In animal studies, the therapeutic has demonstrated superior efficacy to current standard treatments, showing effectiveness against tumors with mutations found in nearly 90% of AML patients. The treatment targets both AML blasts and leukemia stem cells, while preliminary studies indicate good tolerability at doses well above efficacious levels.
AML, affecting over 20,000 U.S. patients annually with 11,000+ deaths, represents a significant market opportunity. The global AML therapeutic market is projected to reach over $6 billion by 2030, within a broader leukemia therapeutic market forecast of $29 billion.
Omeros (OMER) ha istituito un Comitato Direttivo Clinico Oncologico per avanzare il suo programma biologico OncotoX mirato alla leucemia mieloide acuta (LMA). Il comitato è composto da esperti clinici di spicco provenienti dai principali Centri Nazionali per la Rete Oncologica Completa, presieduto dal Dr. Naval Daver del MD Anderson Cancer Center.
Il programma OncotoX-AML presenta molecole ingegnerizzate che consegnano carichi tossici alle cellule tumorali. Negli studi sugli animali, il trattamento ha dimostrato un'efficacia superiore rispetto ai trattamenti standard attuali, mostrando efficacia contro tumori con mutazioni presenti in quasi il 90% dei pazienti con LMA. Il trattamento colpisce sia i blast della LMA che le cellule staminali leucemiche, mentre studi preliminari indicano una buona tollerabilità a dosi ben superiori ai livelli efficaci.
La LMA, che colpisce oltre 20.000 pazienti negli Stati Uniti ogni anno con oltre 11.000 decessi, rappresenta una significativa opportunità di mercato. Si prevede che il mercato globale dei trattamenti per la LMA raggiunga oltre 6 miliardi di dollari entro il 2030, all'interno di una previsione più ampia del mercato terapeutico per la leucemia di 29 miliardi di dollari.
Omeros (OMER) ha establecido un Comité de Dirección Clínica Oncológica para avanzar en su programa biológico OncotoX dirigido a la leucemia mieloide aguda (LMA). El comité está compuesto por distinguidos expertos clínicos de los principales Centros Nacionales de Red de Cáncer Integral, presidido por el Dr. Naval Daver del MD Anderson Cancer Center.
El programa OncotoX-AML presenta moléculas diseñadas que entregan cargas tóxicas a las células cancerosas. En estudios en animales, la terapia ha demostrado una eficacia superior a los tratamientos estándar actuales, mostrando efectividad contra tumores con mutaciones que se encuentran en casi el 90% de los pacientes con LMA. El tratamiento se dirige tanto a los blastos de LMA como a las células madre leucémicas, mientras que los estudios preliminares indican una buena tolerancia a dosis muy por encima de los niveles eficaces.
La LMA, que afecta a más de 20,000 pacientes en EE. UU. anualmente con más de 11,000 muertes, representa una oportunidad de mercado significativa. Se proyecta que el mercado terapéutico global para la LMA alcance más de $6 mil millones para 2030, dentro de una proyección más amplia del mercado terapéutico de leucemia de $29 mil millones.
오메로스 (OMER)는 급성 골수성 백혈병 (AML)을 목표로 하는 OncotoX 생물학적 프로그램을 발전시키기 위해 종양학 임상 자문 위원회를 설립했습니다. 이 위원회는 MD 앤더슨 암 센터의 Naval Daver 박사가 의장을 맡고 있으며, 주요 국가 종합 암 네트워크 센터의 저명한 임상 전문가들로 구성되어 있습니다.
OncotoX-AML 프로그램은 암세포에 독성 화물을 전달하는 엔지니어링된 분자를 특징으로 합니다. 동물 연구에서 이 치료법은 현재 표준 치료법보다 우수한 효능을 나타내었으며, 거의 90%의 AML 환자에게 발견되는 돌연변이를 가진 종양에 대해 효과를 보였습니다. 이 치료법은 AML 블라스트와 백혈병 줄기 세포를 모두 표적으로 하며, 초기 연구에서는 효과적인 수준을 훨씬 초과하는 용량에서도 좋은 내약성을 나타냈습니다.
AML은 매년 20,000명 이상의 미국 환자에게 영향을 미치며 11,000명 이상의 사망자를 초래하여 상당한 시장 기회를 나타냅니다. 전 세계 AML 치료 시장은 2030년까지 60억 달러를 초과할 것으로 예상되며, 더 넓은 백혈병 치료 시장의 전망은 290억 달러입니다.
Omeros (OMER) a établi un Comité de Pilotage Clinique en Oncologie pour faire avancer son programme biologique OncotoX ciblant la leucémie myéloïde aiguë (LMA). Le comité est composé d'experts cliniques distingués des principaux Centres Nationaux de Réseau de Cancer Complet, présidé par le Dr. Naval Daver du MD Anderson Cancer Center.
Le programme OncotoX-AML présente des molécules ingénierées qui livrent des charges toxiques aux cellules cancéreuses. Dans des études animales, le traitement a démontré une efficacité supérieure à celle des traitements standards actuels, montrant son efficacité contre des tumeurs avec des mutations présentes chez près de 90 % des patients atteints de LMA. Le traitement cible à la fois les blasts de LMA et les cellules souches leucémiques, tandis que des études préliminaires indiquent une bonne tolérance à des doses bien supérieures aux niveaux efficaces.
La LMA, qui touche plus de 20 000 patients aux États-Unis chaque année avec plus de 11 000 décès, représente une opportunité de marché significative. Le marché thérapeutique mondial de la LMA devrait atteindre plus de 6 milliards de dollars d'ici 2030, dans le cadre d'une prévision plus large du marché thérapeutique de la leucémie de 29 milliards de dollars.
Omeros (OMER) hat einen Onkologischen Klinischen Lenkungsausschuss eingerichtet, um sein OncotoX-Biologika-Programm zur Bekämpfung von akuter myeloischer Leukämie (AML) voranzutreiben. Der Ausschuss besteht aus angesehenen klinischen Experten führender Nationaler Netzwerke für umfassende Krebsversorgung und wird von Dr. Naval Daver vom MD Anderson Cancer Center geleitet.
Das OncotoX-AML-Programm umfasst entwickelte Moleküle, die toxische Lasten an Krebszellen abgeben. In Tierversuchen hat die Therapie eine überlegene Wirksamkeit gegenüber den aktuellen Standardbehandlungen gezeigt und war gegen Tumore mit Mutationen wirksam, die bei fast 90 % der AML-Patienten vorkommen. Die Behandlung zielt sowohl auf AML-Blasten als auch auf Leukämie-Stammzellen ab, während vorläufige Studien eine gute Verträglichkeit bei Dosen zeigen, die weit über den wirksamen Niveaus liegen.
Die AML betrifft jährlich über 20.000 Patienten in den USA und führt zu über 11.000 Todesfällen, was eine bedeutende Marktchance darstellt. Der globale therapeutische Markt für AML wird bis 2030 voraussichtlich über 6 Milliarden Dollar erreichen, innerhalb eines breiteren Prognosemarktes für Leukämie von 29 Milliarden Dollar.
- Superior efficacy demonstrated in animal studies compared to current standard treatments
- Treatment effective against mutations found in 90% of AML patients
- Good tolerability at doses substantially above efficacious levels
- Large market opportunity with $6B projected AML market by 2030
- Strong endorsement from leading clinical experts joining steering committee
- Product still in pre-clinical phase, requiring IND-enabling work
- No human clinical trial data available yet
- Faces competition in growing but crowded leukemia market
Insights
Omeros' announcement represents a significant advancement in its oncology pipeline development. The formation of this steering committee with key opinion leaders from prestigious institutions like MD Anderson, Mayo Clinic, and Memorial Sloan Kettering signals the company is transitioning from preclinical to clinical development for its OncotoX-AML program. This is particularly notable as the company states it's "initiating IND-enabling work" – a critical milestone that typically precedes clinical trials by 9-12 months.
The preclinical data outlined shows several promising differentiation points: effectiveness against tumors with mutations found in nearly 90% of AML patients (suggesting a mutation-agnostic approach), ability to target both AML blasts and leukemia stem cells (addressing a key mechanism of resistance and relapse), and favorable tolerability profile. Particularly compelling is the reported superior efficacy to venetoclax/azacytidine combination, the current standard of care.
From a development perspective, assembling this caliber of clinical steering committee provides Omeros with valuable expertise for trial design and regulatory interactions, potentially accelerating development timelines and optimizing clinical protocol design. This strategic move positions the company to advance more efficiently through the clinical development process in a projected $6 billion AML market by 2030.
The establishment of this steering committee represents a meaningful step forward for Omeros' AML program, addressing a critical unmet need in hematologic malignancies. AML remains one of the most challenging leukemias to treat, with over 11,000 annual deaths in the US alone. The current standard of care – venetoclax plus azacytidine – marked an improvement but still leaves substantial room for therapeutic advancement.
What's scientifically promising about Omeros' approach is the dual-targeting of both AML blasts and leukemia stem cells (LSCs). This is particularly significant as LSCs are often refractory to chemotherapy and represent a primary cause of relapse. The targeted toxin delivery approach with molecules about half the size of antibodies potentially offers better tissue penetration while maintaining specificity.
The preclinical efficacy across multiple genetic backgrounds (TP53, NPM1, KMT2a, and FLT3) is noteworthy since these mutations collectively represent the genetic landscape of approximately 90% of AML patients. A truly mutation-agnostic approach would address a significant clinical challenge in AML treatment, where therapeutic options are often dictated by specific genetic alterations.
The involvement of Dr. Naval Daver as committee chair is particularly significant given his extensive experience in novel AML therapies. The composition of this committee, representing leading NCCN centers, suggests strong interest in the scientific approach and potential clinical utility of Omeros' technology.
– The Steering Committee brings together distinguished clinical experts in AML from leading National Comprehensive Cancer Network Centers –
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
About Omeros’ Oncology Programs
Omeros’ oncology programs are informed by a deep understanding of both the innate (complement-mediated) and adaptive (CD4 & CD8 T-cells) immune systems to derive highly differentiated, first-in-class therapeutic programs to treat a wide range of cancers. While significant progress has been made within oncology through targeted therapies, advanced biologics and immuno-therapies, toxicity and efficacy shortcomings remain significant. Omeros is focused on moving beyond these approaches with a family of wholly novel molecular and cellular platforms designed to be more effective, targeting both cell-surface and intracellular cancer antigens, with less toxicity. Our oncology franchise involves three distinct platforms – OncotoX, immunomodulators, and immunostimulators in combination with adoptive T-cell therapy. IND-enabling work has been initiated to advance to clinic our OncotoX-AML program comprised of engineered molecules designed to target and kill cancer cells with precision. Our immunomodulator platform is based on our identifying and evidencing the importance of certain molecules that selectively target T cells to regulate and potentiate immune responses. Our adoptive T-cell technology, unlike other cell therapy approaches, does not require cellular engineering and is being developed in concert with our immunostimulator platform to advance beyond the challenges of current vaccine approaches, which induce only transient and ineffective immune responses. By combining tumor antigens with a potent adjuvant, these novel, engineered biologics are designed specifically to target and activate antigen-presenting cells, leading to amplification of cancer-specific T and B cells to eliminate existing tumor cells and, importantly, to generate immune memory against future cancer relapse. Collectively, our portfolio of oncology platforms has the potential to deliver substantially more effective and safer treatments broadly across hematological malignancies and solid tumors.
About Omeros Corporation
Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing first-in-class small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders, including complement-mediated diseases and cancers, as well as addictive and compulsive disorders. Omeros’ lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application pending before FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy. Omeros’ long-acting MASP-2 inhibitor OMS1029 has successfully completed Phase 1 single- and multiple-ascending dose clinical studies. Zaltenibart, Omeros’ inhibitor of MASP-3, the key activator of the alternative pathway of complement, is in Phase 3 clinical development for paroxysmal nocturnal hemoglobinuria and is being evaluated in an ongoing Phase 2 clinical trial for complement 3 glomerulopathy. Funded by the National Institute on Drug Abuse, Omeros’ lead phosphodiesterase 7 inhibitor OMS527 is in clinical development for the treatment of cocaine use disorder. Omeros also is advancing a broad portfolio of novel cellular and molecular immuno-oncology programs. For more information about Omeros and its programs, visit www.omeros.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the “safe harbor” created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “likely,” “look forward to,” “may,” “objective,” “plan,” “potential,” “predict,” “project,” “should,” “slate,” “target,” “will,” “would” and similar expressions and variations thereof. Forward-looking statements, including statements regarding the anticipated safety and therapeutic benefits of Omeros’ drug candidates and plans for future development thereof are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. Omeros’ actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, our financial condition and results of operations, inability to raise capital when required to fund our operations and development plans, unfavorable, unexpected or inconclusive results of our preclinical and clinical development activities, unexpected outcomes of regulatory processes in relevant jurisdictions, including those associated with other Omeros’ development programs, challenges associated with manufacture or supply of our drug candidates or other materials needed to support preclinical and clinical development, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading “Risk Factors” in our Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 31, 2025. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law.
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Jennifer Cook Williams
Cook Williams Communications, Inc.
Investor and Media Relations
IR@omeros.com
Source: Omeros Corporation
FAQ
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