Acute myeloid leukemia is haematological neoplasia characterized by the accumulation of transformed immature myeloid cell in bone marrow. The course of the disease is marked by poor prognosis, frequent relapse, and high disease-related mortality. In spite of improvements in its therapy, above 60% of AML patients will eventually succumb to the disease, a fact stressing the need for new therapeutic approaches for remission induction and prevention of relapse. The difficulty in treating AML is thought to arise from a chemoresistant subpopulation of leukaemia stem cells (LSCs) that are capable of maintaining and reinitiating the disease.
Since the establishment of the Josep Carreras Leukaemia Research Institute, the Leukemia Stem Cell research group led by Dr. Risueño develops a research program focused on the development of therapeutic strategies for targeting LSCs. An in silico screening was performed to search for already human-approved drugs that may differentially kill LSCs while sparing healthy hematopoietic stem cells (HSCs).
Leukos Biotech holds a license from Josep Carreras Leukaemia Research Institute covering proprietary use of a family of drugs and antibodies with the potencial for diagnosing, prognosing and treating AML patients developed by Dr. Risueño's team at IJC. After 3 years of comprehensive research on the mechanism of action of known serotonin antagonist drugs on AML cells, and recognizing the strong potential of the target in the oncological setting, the company started a medicinal chemistry program aiming to deliver new compounds with high selectivity, high specificity, oral bioavailability and no BBB crossing. As a result, Leukos Biotech has defined a first-in-class broad chemistry space with blockbuster potential. Its first candidate for AML/MDS started IND-enabling work in August 2020 and will be IND-ready by 1Q2023, as the result of the co-development agreement signed in July 2022 between Leukos Biotech and AOP Health.
