Myeloid malignancies, blood cell cancers arising in the bone marrow, have long been associated with disruptions of epigenetic regulation. There are many clinical varieties of myeloid diseases, all of which can evolve into life-threatening phases. As with other cancers, several different types of agents may be needed to bring myeloid diseases under control.
The first-in-patient study of bomedemstat was initiated in late 2016 in patients with high-risk acute myeloid leukemia and myelodysplastic syndrome. The multiple-ascending dose Phase 1 portion of that study was concluded in 2017. The Phase 2 portion of the study of bomedemstat in combination with all-trans retinoic acid (ATRA) was recently completed. A Phase 2b study of bomedemstat in patients with high or intermediate-2 risk myelofibrosis is currently enrolling patients globally.
Bomedemstat is a small molecule, orally available inhibitor of the enzyme, Lysine-Specific Demethylase (LSD1). This first-in-class drug for the treatment of myeloproliferative neoplasms was discovered and developed by Imago BioSciences. Currently bomedemstat is being tested in clinical studies as a potentially disease-modifying monotherapy for MF, essential thrombocythemia (ET) and related indications. Bomedemstat has also been studied clinically in AML and pre-clinically in various solid tumor models, where in combination with other agents it has shown efficacy (?)
Ongoing Phase 2b Study and the Path Forward
Bomedemstat has the potential to be a differentiated, first-in-class, disease-modifying therapy for a patient population with very limited therapeutic options. The FDA has granted Orphan Drug and Fast Track designations to bomedemstat, which is currently enrolling a 75 patient, 24 week, Phase 2b study in MF in US and EU (Clinicaltrials.gov NCT03136185).
Imago will initiate additional Phase 2 studies in other hematologic disorders, including essential thrombocythemia, in 2020.
Learn More About our Phase 2 Clinical Trials
Investigator Initiated Trials with Bomedemstat