ORYZON Receives European Medicines Agency Approval to Initiate a Phase Ib Study of Iadademstat in Sickle Cell Disease

Core Insights - Oryzon Genomics has received EMA approval to initiate a Phase Ib clinical trial of iadademstat for sickle cell disease (SCD), marking the first investigation of this drug in a non-malignant hematological indication [1][2] Company Overview - Oryzon Genomics is a clinical-stage biopharmaceutical company based in Barcelona, Spain, specializing in epigenetics and personalized medicine, particularly in CNS disorders and oncology [7] - The company has a robust clinical portfolio, including two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [7] Clinical Trial Details - The Phase Ib study, named RESTORE, will enroll 40 adult patients with SCD across multiple sites in Spain, focusing on safety, tolerability, and establishing the Recommended Phase 2 dose (RP2D) [2] - Secondary objectives of the trial include assessing the drug's ability to induce fetal hemoglobin (HbF) levels [2] Sickle Cell Disease Context - SCD is a chronic inherited blood disorder affecting approximately 7.7 million people globally, characterized by the production of hemoglobin S (HbS) leading to various complications [3][5] - The disease results in significant health issues, including vaso-occlusive crises, hemolytic anemia, and reduced quality of life, highlighting a substantial unmet medical need for effective treatments [4] Market Potential - The SCD treatment market is projected to grow from approximately USD 3 billion in 2025 to around USD 8 billion by 2032, indicating strong commercial potential for effective therapies [5] - Existing gene therapies face challenges due to technical complexity and high costs, limiting patient access [5] Iadademstat's Mechanism and Other Investigations - Iadademstat is a selective LSD1 inhibitor showing promise in increasing HbF levels, which may mitigate the disease's pathological effects [5][8] - The drug is also under investigation in various oncology trials, including combinations with gilteritinib and azacitidine for acute myeloid leukemia (AML) [6][8]