AOP Health has launched the phase 1 clinical trial SERHEMA-1 to evaluate the investigative drug candidate AOP208 in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS), which are severe forms of cancer. This “first-in-class” approach1 marks a key milestone in the progression of the clinical development program for AOP208, which began with the first-in-human trial SERONCO-1 and builds on AOP Health’s expertise in delivering cancer stem-cell targeting agents.
AOP208 is an innovative, orally administered, potential new therapy which inhibits the serotonin receptor 1B (HTR1B). This target plays a role in tumor cell survival and treatment resistance but has not previously been exploited in cancer therapy. Preclinical data and early clinical safety data demonstrate the potential for further development of AOP208 in patients with relapsed/refractory AML and high-risk MDS, who have limited treatment options.
The initial AOP208 trial, SERONCO-1 (EU CT No: 2023-509532-25-00) in patients with solid tumors or lymphoma is being conducted in the Vall d’Hebron Institute of Oncology in Barcelona in a collaboration between AOP Health and Leukos Biotech with partial funding from Public-Private Collaboration initiative (CPP2021-008715) from the Spanish Ministry of Science and Innovation.
Dr. Christoph Klade, Chief Scientific Officer of AOP Health:
"After successfully advancing our phase I trial in solid tumors, we are now expanding development in blood cancers. AOP208 has a novel mechanism of action with potential for targeting leukemic stem cells and the tumor microenvironment in the bone marrow niche. Now we are thrilled to start a clinical trial and further investigate our compound’s potential."
Phase 1 Clinical Trial SERHEMA-1 Treats First Patient
The first patient has begun treatment with AOP208 in SERHEMA-1 (EU CT No: 2025-523396-38-00), a phase I/II trial in relapsed or refractory AML and high-risk MDS. This study is taking place in specialized centers in Spain and Austria and is sponsored by AOP Health. Initiation of the SERHEMA-1 trial represents a significant step towards understanding the safety and tolerability of AOP208.
Dr. Zoltan Magyarics, Vice-President of Research & Development at AOP Health:
“Advancing research in rare diseases is central to our mission. We are pleased to contribute our scientific expertise to a clinical program that seeks to expand knowledge and offer therapeutic options in an area of high unmet medical need in hemato-oncology.”
About AOP208
AOP208 is an innovative, orally administered serotonin receptor 1B antagonist. AOP Health has licensed AOP208 from Leukos Biotech (a spin-off company from the Josep Carreras Leukaemia Research Institute in Barcelona, Spain) as well as the entire compound library generated to identify the drug candidate. AOP Health holds further development rights and exclusive global commercial rights including USA, Europe, Korea and Taiwan with the exclusion of mainland China.
References:
1 https://pubmed.ncbi.nlm.nih.gov/28193998/
2 https://pubmed.ncbi.nlm.nih.gov/30224768/
About AOP Health
AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions. (aop-health.com)