ORYZON doses first patient in trial of iadademstat with venetoclax and azacitidine for AML

Oryzon Genomics, S.A., a biopharmaceutical company based in Madrid, Spain, and Cambridge, Massachusetts, United States, has announced a pivotal development in the treatment of acute myeloid leukemia (AML). On January 13th, 2025, the company reported that the first patient has been administered a dose in a Phase I clinical trial focusing on a promising therapy for newly diagnosed AML patients. This trial is sponsored by the National Cancer Institute (NCI), a part of the National Institutes of Health.

The study is officially titled "Phase 1 Trial of Iadademstat in Combination With Venetoclax and Azacitidine in Patients With Treatment Naive AML" and is registered under the identifier NCT06514261. The primary objective of this trial is to assess the safety, tolerability, and optimal dosage of iadademstat, Oryzon's selective LSD1 inhibitor, when used in conjunction with venetoclax and azacitidine. These two drugs are part of the current standard-of-care for AML. The trial also aims to evaluate the preliminary efficacy of this drug combination in patients who have not received prior treatment for AML.

Overseeing the study is Dr. Natalie Galanina, a prominent figure from the University of Pittsburgh Cancer Institute. The trial has set out to enroll a total of 45 participants and operates under a Cooperative Research and Development Agreement (CRADA) between Oryzon and the NCI.

Dr. Carlos Buesa, the CEO of Oryzon Genomics, expressed enthusiasm over the commencement of this new trial. According to Dr. Buesa, the study builds upon positive results from the earlier ALICE trial. In the ALICE trial, the combination of iadademstat with azacitidine was shown to produce significant antileukemic effects in the first-line treatment of AML. The combination led to deep and long-lasting responses while maintaining a manageable safety profile, even in patients with high-risk factors who typically respond poorly to venetoclax and azacitidine alone.

The exploration of iadademstat for AML treatment does not stop with this trial. It is also being studied in other clinical settings. At the Oregon Health & Science University (OHSU) Knight Cancer Institute, another investigator-initiated Phase I clinical trial is underway. This study examines the efficacy of iadademstat in combination with venetoclax and azacitidine for newly diagnosed AML patients, registered under NCT06357182.

Additionally, Oryzon is conducting its own company-sponsored Phase Ib clinical trial. This trial investigates the use of iadademstat in combination with gilteritinib for patients with relapsed or refractory AML harboring a specific genetic mutation known as FMS-like tyrosine kinase mutation (FLT3mut+). This study is registered under NCT05546580.

These trials collectively highlight Oryzon Genomics' commitment to advancing the treatment landscape for acute myeloid leukemia. By exploring various combinations of iadademstat with other drugs, Oryzon aims to provide more effective and safer treatment options for patients with this challenging and aggressive form of leukemia.