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Moleculin Announces Successful Completion of US Phase 1 AML Trial of Annamycin
No evidence of cardiotoxicity; preliminary assessment shows efficacy in 2 out of 6 patients; Company updates interim Phase 1 trial results in Europe HOUSTON,
About this update from Moleculin Biotech, Inc.
[{"type":"text","content":"No evidence of cardiotoxicity; preliminary assessment shows efficacy in 2 out of 6 patients; Company updates interim Phase 1 trial results in Europe\n\n\nHOUSTON, Feb. 3, 2020 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (\"Moleculin\" or the \"Company\"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, today announced that its open label, single arm US Phase 1 trial met its primary objective of demonstrating the safety of Liposomal Annamycin (\"Annamycin\") in treating relapsed or refractory acute myeloid leukemia (\"AML\"). The Company also announced an update on interim enrollment, safety and efficacy data in its parallel Phase 1 trial in Europe, which continues with dose escalation, thus far without safety concerns. \n\n \n \n\n \nSafety of Annamycin in AML Patients\nThe US Phase 1 trial met its primary endpoint, demonstrating the safety of Annamycin in treating AML when delivered to patients at or below the lifetime maximum anthracycline dose established by the FDA. The primary safety signal was the absence of cardiotoxicity (potential damage to the heart), a serious and often treatment-limiting issue prevalent with currently approved anthracyclines. This was determined by echocardiograms, as well as cardiac health biomarkers, principally blood troponin levels, which are considered an indicator of potential long-term heart damage. The data showed no cardiotoxicity in any of the 6 patients evaluated in the US Phase 1 trial. Additionally, there were no unexpected serious adverse events and no dose limiting toxicities at any dose tested. \nPreliminary Evidence of Effectiveness\nAlthough the primary objective of the Phase 1 trial was to evaluate safety, the study also gathered data to support a preliminary assessment of the product's potential efficacy. Among other things, the study recorded complete response (CR), partial response (PR), event-free survival, overall survival (Kaplan-Meier), and time to and duration of remission/response. Based on these criteria, efficacy was seen in 2 of the US patients, even though the drug was dosed at what was expected to be sub-therapeutic levels. The evidence of efficacy consisted of 1 patient who achieved a \"morphologically leukemia-free state,\" which the protocol defined as a CR with incomplete recovery of ...