Oryzon reported positive EHA 2026 data showing iadademstat plus azacitidine and venetoclax achieved a 100% response rate in newly diagnosed AML.
Written By: Shaik Yasmeen, PharmD
Reviewed By: Pharmacally Editorial Team
Oryzon Genomics has reported updated Phase Ib data showing that iadademstat combined with azacitidine and venetoclax achieved a 100% overall response rate (ORR) in patients with newly diagnosed acute myeloid leukemia (AML), while maintaining a favorable safety profile. Results presented at the 2026 European Hematology Association (EHA) Congress also demonstrated encouraging activity in genetically high-risk patients, supporting plans to advance the regimen toward a potentially registrational study.
Iadademstat Shows Broad Activity in Newly Diagnosed AML
Iadademstat is an investigational lysine-specific demethylase 1 (LSD1) inhibitor that targets epigenetic mechanisms involved in leukemia cell survival and differentiation. Combining LSD1 inhibition with standard AML therapies may enhance treatment responses and overcome resistance, particularly in patients with adverse genetic features who continue to have poor outcomes with existing therapies.
Updated findings from the ongoing Phase Ib ALICE-2 trial (NCT06357182) evaluated iadademstat in combination with azacitidine and venetoclax in newly diagnosed AML.
Among 18 evaluable patients, investigators reported a 100% overall response rate (18/18), an 89% composite complete remission (CRc) rate, and a 78% complete response (CR) rate. Most complete remissions occurred during the first treatment cycle.
The regimen also demonstrated encouraging activity across adverse-risk genomic subgroups. Both evaluable patients with TP53-mutated AML achieved complete remission, accompanied by marked reductions in TP53 variant allele frequency. Likewise, all three patients with RAS pathway mutations achieved complete remission.
After a median follow-up of eight months, median overall survival and event-free survival had not been reached. Estimated 12-month overall survival and event-free survival were 79% and 71%, respectively. Nine patients proceeded to allogeneic hematopoietic cell transplantation (HCT), with an estimated 12-month overall survival of 88% after transplant.
The triplet combination continued to demonstrate a favorable and manageable safety profile throughout the study.
Combination Therapy Maintains High Remission Rate in Relapsed FLT3-Mutated AML
Oryzon also presented updated results from the fully enrolled Phase Ib FRIDA trial (NCT05546580) evaluating iadademstat with gilteritinib in patients with relapsed or refractory FLT3-mutated AML.
Among 18 response-evaluable patients treated at the selected pharmacologically active dose, the combination achieved a 67% composite complete remission rate while maintaining a favorable safety profile despite the heavily pretreated population.
The company noted that these results compare favorably with published real-world outcomes for gilteritinib monotherapy, where composite complete remission rates of approximately 28% have been reported in similar patient populations.
Hematology Development Program Continues to Expand
Building on its AML program, Oryzon has initiated the Phase II IDEAL trial evaluating iadademstat in adults with essential thrombocythemia (ET) who are resistant or intolerant to hydroxyurea. The study will assess the drug’s safety, tolerability, clinical activity, and its ability to normalize elevated platelet counts over a 24-week treatment period, with an optional extension for patients benefiting from therapy.
Enrollment also continues across additional iadademstat studies under the company’s Cooperative Research and Development Agreement with the U.S. National Cancer Institute, including trials in sickle cell disease, myeloproliferative neoplasms, extensive-stage small cell lung cancer, and myelodysplastic syndrome.
Company Plans Registrational AML Study Following Final Data
Chief Executive Officer Dr. Carlos Buesa said the updated ALICE-2 findings reinforce iadademstat’s potential as a differentiated first-line AML therapy, citing the combination’s efficacy across adverse genetic subgroups, manageable safety profile, and the number of patients who successfully underwent potentially curative stem cell transplantation.
Oryzon expects to present final data from both the ALICE-2 and FRIDA studies before the end of 2026. If the efficacy and safety profile remain consistent, the company plans to meet with the U.S. Food and Drug Administration to discuss a potentially registrational first-line AML study, with trial initiation targeted for 2027.
Vafidemstat Pipeline Also Advances
Beyond its hematology programs, Oryzon continues preparations for the Phase III PORTICO-2 trial evaluating vafidemstat for aggression associated with borderline personality disorder (BPD) while enrollment continues in the Phase IIb EVOLUTION study in schizophrenia. The company also received a new U.S. patent covering methods of treating non-aggressive symptoms of BPD with LSD1 inhibitors, extending intellectual property protection for vafidemstat through 2043, excluding any additional patent term extension that may be granted following regulatory review.
Reference
ORYZON reports financial results and corporate update for half year ended June 30th, 2026 | Oryzon
About the Writer
Shaik Yasmeen (LinkedIn) is a Pharm.D graduate with interests in clinical pharmacy, pharmacovigilance, and medical writing. She has gained experience through hospital clinical postings, patient case reviews, case presentations, and literature evaluation. Passionate about evidence-based healthcare, she is committed to creating accurate and engaging medical content while continuously expanding her professional knowledge.