The biopharmaceutical company Oryzon Genomics, a member of Catalonia.health, has presented updated positive clinical data from two clinical trials with its selective LSD1 inhibitor, iadademstat, in acute myeloid leukemia (AML), at the 2026 Annual Congress of the European Association of Hematology (EHA), held on June 11.

ALICE-2 trial: 100% overall response in first line

The Phase Ib clinical trial ALICE-2 (NCT06357182), sponsored by Oregon Health & Science University (OHSU), is evaluating iadademstat in combination with azacitidine and venetoclax in patients with newly diagnosed AML not eligible for intensive treatment. Data presented at the EHA show an overall response rate (ORR) of 100% (18/18), a composite complete remission rate (CRc) of 89% (16/18) and a complete remission rate (CR) of 78% (14/18). Complete remissions occur early, most of them during the first cycle of treatment.

The trial has shown efficacy in several high-risk genomic risk groups: all patients with a TP53 mutation (2/2) achieved a complete remission and showed a reduction in the allelic frequency of the TP53 variant, and all patients with mutations in the RAS pathway (3/3) also achieved a complete remission. With a median follow-up of 8 months, the estimated overall survival (OS) and event-free survival (EFS) rates at 12 months were 79% and 71%, respectively. Nine patients were able to proceed to allogeneic hematopoietic cell transplantation (HSCT), with an estimated 12-month OS rate of 88%. The combination has continued to show a favorable safety profile.

FRIDA trial: CRc of 67% in relapsed or refractory patients

The Phase Ib FRIDA clinical trial (NCT05546580), sponsored by Oryzon, is evaluating iadademstat in combination with gilteritinib in patients with relapsed or refractory AML with FLT3 mutation. The data presented correspond to the expansion cohort at the selected pharmacologically active dose (75 μg of iadademstat), with 23 patients recruited, of which 18 were evaluable for response. Results show a CRc rate of 67% (12/18) in a heavily pretreated population, with a manageable safety profile that does not add toxicity to standard treatment.

Statements of those responsible

Carlos Buesa, CEO of Oryzon Genomics, highlighted the robustness and consistency of the data from the ALICE-2 and FRIDA trials, and emphasized that the favorable safety profile and efficacy signals of iadademstat reinforce confidence in this combination strategy, also in genomically defined adverse risk populations. Buesa said the company expects to report final data by the end of the year and move toward a potential first-line AML registration trial in 2027, focused on adverse-risk populations.

Ana Limón, Senior Vice President of Clinical Development and Global Medical Affairs at Oryzon, has pointed out that, historically, a third of first-line AML patients do not respond to standard treatment with azacitidine and venetoclax, which highlights the need for new strategies based on triple combinations. Limón emphasized that the increasingly mature data from both trials continue to reinforce LSD1 inhibition as a therapeutic strategy and that the high proportion of patients who have been able to proceed to allogeneic transplantation demonstrates the potential to improve long-term survival.