AOP Health has launched the phase 1 clinical trial SERHEMA-1 to evaluate the first-in-class investigative drug AOP208 in patients with acute myeloid leukaemia (AML) and high-risk myelodysplastic syndrome (MDS), cancers with a dismal prognosis. This key milestone marks the progression of the clinical development programme 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 a first-in-class1, orally administered antagonist of serotonin receptor 1B (HTR1B). This target is implicated in tumour 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.
First clinical experience with AOP208 prompts expanded research
The initial AOP208 trial, SERONCO-1 (EU CT No: 2023-509532-25-00) in patients with solid tumors or lymphoma is being conducted in a collaboration between AOP Health and Leukos Biotech and the Vall d’Hebron Institute of Oncology, in Barcelona, with partial funding from the Spanish Ministry of Science and Innovation public-private partnership program (CPP2021-008715). The study has delivered promising early safety results supporting escalation to higher doses.2
Christoph Klade, Chief Scientific Officer of AOP Health explains: “After successfully advancing our phase I trial in solid tumours, we are now expanding development in blood cancers. AOP208 has a novel mechanism of action with potential for targeting leukemic stem cells and the tumour microenvironment in the bone marrow niche. This next step reflects the encouraging data generated so far and builds on our long-standing expertise in hemato-oncology.”
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 specialised centres 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 and exploring its anti-tumour activity.
Zoltan Magyarics, Vice-President of Research & Development at AOP Health emphasized: “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.”
