Moleculin to Seek Accelerated FDA Approval and Plans for Pivotal Phase 2 AML Trial

About this update from Moleculin Biotech, Inc.

[{"type":"text","content":"Following Successful Completion of US Phase 1 Trial\n\n\nHOUSTON, Feb. 5, 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 it intends to discuss with the FDA and EMA (European Medicines Agency) plans to conduct a single arm Phase 2 trial that would serve as the basis for accelerated approval of Liposomal Annamycin (\"Annamycin\") to treat relapsed or refractory acute myeloid leukemia (\"AML\"). This will follow the establishment of a recommended Phase 2 dose (\"RP2D\") in the Company's ongoing Phase 1 dose escalation trial in Europe. The FDA has already granted Annamycin Fast Track status and Orphan Drug Designation for AML. FDA grants Fast Track designation to drugs intended to treat serious conditions that demonstrate the potential to address unmet medical needs, which can include providing efficacy comparable to available therapy while avoiding toxicity associated with the existing treatment. The benefits of Fast Track include FDA actions to expedite development and review, including \"rolling review,\" where the agency reviews portions of a marketing application before the complete application is submitted.\n\n \n \n\n \nThe announcement follows continuing positive results from Moleculin's open label, single arm Phase 1 trials in AML patients in Europe and the US. Most recently, the US Phase 1 study met its primary objective of demonstrating the safety of Annamycin at a dose that was cumulatively at or below the lifetime maximum anthracycline dose. Those results are consistent with results achieved with the parallel Phase 1 study being conducted in Europe, which has demonstrated the safety of escalating doses of Annamycin in AML patients, including doses that significantly exceed the maximum lifetime dose of anthracyclines imposed in the US. In both trials, the primary endpoints are aimed at demonstrating the product's safety, primarily the lack of cardiovascular risk. This is a key characteristic that, if borne out, could significantly differentiate Annamycin from other anthracyclines, which generally are well-known to have treatment-limiting cardiotoxicity.\nBased on these results, Moleculin will continue to focus the Company's efforts on the Eu...

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