ORYZON Receives European Medicines Agency Approval to Initiate a Phase II Study of iadademstat in Essential Thrombocythemia

Core Viewpoint - Oryzon Genomics has received authorization from the European Medicines Agency (EMA) to initiate a Phase II clinical trial for iadademstat, a selective LSD1 inhibitor, aimed at treating essential thrombocythemia (ET) [1][2]. Group 1: Clinical Trial Details - The Phase II study, named IDEAL, will be a multicenter, single-arm trial conducted in Spain, targeting adult ET patients who are resistant or intolerant to hydroxyurea [2]. - Primary objectives include evaluating the safety and tolerability of iadademstat and assessing its efficacy in reducing abnormal platelet counts in ET patients [2]. - The treatment duration for iadademstat will be up to 24 weeks, with an optional 24-week extension for patients benefiting from the treatment [3]. Group 2: Disease Background - Essential thrombocythemia is the most common type of myeloproliferative neoplasm, affecting approximately 200,000 people in the United States, and is associated with serious complications such as stroke and heart attack [4]. - Current treatments primarily focus on reducing platelet counts and controlling symptoms, but many patients develop resistance or intolerance to first-line therapies like hydroxyurea, indicating a need for new treatment options [4]. Group 3: Mechanism and Efficacy - LSD1 inhibition has been shown to prevent the differentiation of megakaryocytes into platelets, leading to a reduction in circulating platelet counts, supporting the use of LSD1 inhibitors in treating ET [5]. - Positive results from another LSD1 inhibitor in a Phase II trial for high-risk ET patients further validate this therapeutic approach [5]. Group 4: Company Insights - Dr. Carlos Buesa, CEO of Oryzon, emphasized that the IDEAL study aligns with the company's strategy to expand the clinical applications of iadademstat beyond acute leukemia to other hematologic conditions with significant unmet medical needs [6]. - Iadademstat is noted to be the most potent LSD1 inhibitor in clinical development, with over 100-fold greater potency than other inhibitors [6]. - The company is also exploring iadademstat in various oncology trials, including a Phase Ib study in combination with venetoclax and azacitidine for first-line acute myeloid leukemia (AML), which has shown a 100% overall response rate [6][8].