ORYZON Presents Updated Positive Clinical Data for Iadademstat in Acute Myeloid Leukemia (AML) at European Hematology Association (EHA) 2026 Annual Congress

Oryzon Genomics, S.A., a clinical-stage biopharmaceutical company and a global leader in epigenetics, today presented updated positive clinical data from two clinical trials of its selective LSD1 inhibitor iadademstat in acute myeloid leukemia (AML) at the 2026 European Hematology Association (EHA) Annual Congress.

“We are encouraged by the sustained strength and consistency of the data from both the ALICE-2 and FRIDA trials,” said Carlos Buesa, Chief Executive Officer of Oryzon Genomics. “With over 80% of patients now enrolled in ALICE-2, the favorable safety profile and strong efficacy signals of iadademstat in newly diagnosed, unfit AML patients reinforce our confidence in this combination approach, including within genomically defined adverse-risk populations such as TP53-mutated and RAS pathway–mutated AML. These results are consistent with prior findings in TP53-mutated patients in our ALICE trial, where the combination of iadademstat and azacitidine doubled median overall survival compared with historical rates. As enrollment continues, we anticipate reporting final data by year-end and advancing toward a potential registrational study in first-line AML by 2027, with a focus on adverse-risk populations”

Ana Limón, Senior Vice President of Clinical Development and Global Medical Affairs at Oryzon, added: “Historically, with azacitidine plus venetoclax, one third of first line AML patients do not respond, and the depth of response is variable, underscoring the need for novel triplet strategies, particularly for patients without targetable mutations. The maturing data from both trials continue to reinforce the strength of LSD1 inhibition as an add-on approach in AML. In addition to the high ORR and CR rates observed to date 1 in the ALICE-2 trial, treatment with the iadademstat-azacitidine-venetoclax triplet has enabled a high proportion of patients to transition to allogeneic hematopoietic cell transplantation (HCT), potentially improving long-term survival. Overall, the safety and efficacy observed across both trials support further clinical development.”

Data Summary

 ALICE-2 Phase Ib clinical trial (NCT06357182) investigating iadademstat in combination with azacitidine and venetoclax in newly diagnosed AML

• High rates of activity, with a 100% (18/18) overall response rate (ORR), 89% (16/18) composite complete remission (CRc) rate and 78% (14/18) complete response (CR) rate. 
• CRs occur early, most of them in cycle 1. 
• Efficacy was observed across different genomic risk groups, including TP53 and RAS pathway mutations and patients with complex karyotypes, all considered adverse risk. 
• Patients with TP53-mutated disease (2/2) attained CR and showed a reduction in TP53 variant allele frequency (14% to undetected and 22% to 1%, respectively). 
• All patients with RAS pathway mutations (3/3) achieved CR. 
• After a median follow-up of 8 months, median overall survival (OS) and event-free survival (EFS) were not reached; estimated 12-month OS and EFS were 79% and 71%, respectively. 
• 9 patients successfully transitioned to allogeneic HCT, with an estimated 12-month OS of 88%. 
• The iadademstat-azacitidine-venetoclax combination continues to show a favorable safety profile. 

FRIDA Phase Ib clinical trial (NCT05546580) investigating iadademstat in combination with gilteritinib in FLT3‑mutated relapsed/refractory AML 

• The poster reports data from the expansion cohort at the selected pharmacological active dose (PAD, 75 ug iadademstat); 23 patients have been enrolled at this dose, with 18 being evaluable for response. 
• High CRc rate of 67% (12/18) in a heavily pre-treated population. 
• Iadademstat plus standard of care (SoC) treatment gilteritinib demonstrated a manageable safety profile, without adding toxicity to the SoC.