Moleculin MIRACLE Trial Delivers 40% Preliminary Blinded CRc Rate (n=30)

Core Insights - The preliminary blinded complete remission (CR) rate in the MIRACLE trial shows a 67% improvement over historical cytarabine response rates, indicating a significant advancement in the treatment of acute myeloid leukemia (AML) [1][3] - Approximately 35% of subjects treated so far are failures from the ventoclax regimen, highlighting the challenging nature of this patient population [1][3] - The company is on track to treat the first 45 subjects by Q1 2026, with unblinding of data expected thereafter [1][8] Trial Progress and Efficacy - The MIRACLE trial has reported a preliminary composite complete remission (CRc) rate of 40% among the first 30 subjects, consisting of a 30% complete remission (CR) rate and a 10% complete remission with partial hematological recovery (CRh) [2] - The trial is designed as a Phase 2B/3, global multi-center, randomized, double-blind, placebo-controlled study, combining data from both phases to measure primary efficacy endpoints [5] - The first unblinding of preliminary efficacy data is expected to yield results from approximately 30 subjects treated with Annamycin in combination with cytarabine and 15 subjects treated with cytarabine plus placebo [5][8] Recruitment and Future Steps - The company has encountered higher than expected disqualifications among applicants, indicating a significant unmet medical need among relapsed/refractory AML patients [6] - Recruitment efforts are ongoing, with a focus on improving participation in the US, while European recruitment has been robust [7] - The second group of 45 subjects in Part A is expected to be fully recruited by Q3 2026, with unblinding anticipated in the second half of 2026 [8] Regulatory Status and Market Potential - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory AML, along with patent protection extending to 2040, potentially to 2045 [10] - The company believes Annamycin could offer a new treatment avenue for patients battling AML, especially given its lack of cardiotoxicity compared to current therapies [3][11]