Sparks - Oryzon Genomics

Oryzon Genomics has announced the enrolment of the first patient in the Phase II IDEAL study, evaluating iadademstat in patients with essential thrombocythemia (ET) who are resistant or intolerant to hydroxyurea, the current standard of care for preventing thrombotic events in high-risk patients. The multicenter, single-arm study will assess the safety, tolerability and efficacy of iadademstat, with a particular focus on reducing abnormal platelet counts. Treatment will be administered for up to 24 weeks, with an optional 24-week extension period for patients deriving clinical benefit.

The initiation of IDEAL further broadens iadademstat’s clinical development beyond  acute myeloid leukaemia (AML) and solid tumours into non-malignant haematological disorders, highlighting both the drug’s broad therapeutic potential and LSD1 inhibition as a promising approach across a range of haematologic diseases. Oryzon is also evaluating iadademstat in first-line AML in combination with venetoclax and azacitidine (ALICE-2) as well as in sickle cell disease (RESTORE). The company recently reported encouraging interim results from ALICE-2, with 14 evaluable patients achieving a 100% overall response rate, a 93% composite complete remission rate and a 79% complete remission rate. A Phase II/III ALICE-3 study is expected to commence in 2027.