oryzon-iadademstat-aml-alice2-frida-eha-2026

Oryzon Genomics has reported updated Phase 1b data from two clinical studies evaluating its LSD1 inhibitor iadademstat in acute myeloid leukemia (AML), showing high response rates in combination with current standard-of-care regimens and continued activity across genetically defined high-risk subgroups.

The findings were presented at the European Hematology Association (EHA) 2026 Annual Congress and include results from the ALICE-2 study in newly diagnosed unfit AML patients and the FRIDA study in relapsed or refractory FLT3-mutated disease.

In the ALICE-2 trial, iadademstat was evaluated in combination with azacitidine and venetoclax. Among evaluable patients, the study reported a 100% overall response rate (18/18), an 89% composite complete remission rate (16/18) and a 78% complete remission rate (14/18). The company said responses occurred early in treatment, with most complete remissions observed in cycle one.

Activity was observed across adverse-risk genetic subgroups, including patients with TP53 and RAS pathway mutations and those with complex karyotypes. All patients with TP53 mutations (2/2) achieved complete remission, with reported reductions in TP53 variant allele frequency. All patients with RAS pathway mutations (3/3) also achieved complete remission.

After a median follow-up of eight months, median overall survival and event-free survival were not reached. Estimated 12-month overall survival and event-free survival were 79% and 71% respectively. Nine patients proceeded to allogeneic hematopoietic cell transplantation.

The safety profile of the combination was described as favourable and consistent with previous experience of the agents involved.

In the FRIDA study, iadademstat was evaluated with gilteritinib in FLT3-mutated relapsed or refractory AML. In 18 evaluable patients at the pharmacologically active dose, the combination produced a composite complete remission rate of 67%, with a manageable safety profile and no additional toxicity attributed beyond standard therapy.

Commenting on the ALICE-2 results, Carlos Buesa, chief executive officer of Oryzon Genomics, said: “We are encouraged by the sustained strength and consistency of the data from both the ALICE-2 and FRIDA trials,” and added: “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.”

He added: “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 Genomics, said: “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.”

She added that the maturing data from both trials continue to support further clinical development of the LSD1 inhibitor approach in AML.

Iadademstat is an investigational selective LSD1 inhibitor designed to target epigenetic regulation of gene expression without altering DNA sequence. The ALICE-2 study is an investigator-initiated Phase 1b trial sponsored by Oregon Health & Science University, while the FRIDA study is a Phase 1b Oryzon-sponsored study in relapsed or refractory AML.

The data add to growing clinical interest in epigenetic therapies in oncology, particularly in combinations designed to enhance responses to existing backbone regimens in difficult-to-treat haematological malignancies.