Acute Myeloid Leukemia (AML):

The Most Common Type of Acute Leukemia in Adults

Commencing MIRACLE Trial: Pivotal Adaptive Phase 3 Clinical Study for the Treatment of Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a type of blood cancer that starts in the bone marrow. AML usually begins in cells that turn into white blood cells and can quickly spread to the lymph nodes and other organs. AML is currently treated with chemotherapy and targeted therapies with a 5-year survival rate of only 26%. While anthracyclines for AML can be effective, their use can be constrained due to their inherent cardiotoxicity1, which limits the cumulative amount of anthracycline that can be given to patients. In addition, many patients quickly develop resistance to currently prescribed anthracyclines due to multidrug resistance mechanisms. Given its lack of cardiotoxicity1 and demonstrated (in animal models) ability to avoid multidrug resistance mechanisms, we believe Annamycin could become an important treatment option for relapsed or refractory AML patients.

We are initiating the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial, a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory AML. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, we believe we have substantially de-risked the development pathway towards approval for Annamycin for the treatment of AML. The MIRACLE study is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents.

Learn More About the MIRACLE Trial

The Annamycin Opportunity in AML:

  • Annamycin in combination with Cytarabine (AnnAraC) has potential to fill unmet need in AML; 60% CRc (50% CR) in 2nd line patients reported in latest Phase 1B/2 study

  • All Annamycin subjects (in multiple studies) continue to show no signs of cardiotoxicity1 during study; lower toxicity profile than traditional intensive therapy

AML Patient Journey and the Annamycin Opportunity 

~160,000 cases worldwide2

~20,000 new cases annually (U.S.)2

5-Year survival rate of only 26%2

1: Moleculin Biotech, Inc. (2024). Form 10-K for the fiscal year ended December 31, 2024. https://moleculin.com/sec-filings/. For updates on Annamycin and its lack of cardiotoxicity, see subsequent disclosures in Forms 8-K, 10-Q, and 10-K.

2: Glabbeke M V, et al. Prognostic Factors for the Outcome of Chemotherapy in Advanced Soft Tissue Sarcoma: An Analysis of 2,185 Patients Treated With Anthracycline-Containing First-Line Regimens—A European Organization for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group Study. Journal of Clinical Oncology, Vol 17, No 1 (January), 1999: pp 150-157​

Annamycin

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