Moleculin Biotech, Inc. (Nasdaq: MBRX) is advancing its lead drug candidate, Annamycin, towards a pivotal Phase 3 clinical trial for relapsed or refractory acute myeloid leukemia (R/R AML). The company anticipates beginning the "MIRACLE" trial (Moleculin R/R AML AnnAraC Clinical Evaluation) in Q1 2025, a study designed with the potential for accelerated approval of Annamycin in combination with cytarabine. This development follows positive discussions with the FDA regarding the advancement of Annamycin, combined with cytarabine (AnnAraC), into Phase 3 trials.
The MIRACLE trial will be a global study, including sites in the US, and will employ an adaptive design. Initially, approximately 75 to 90 subjects will be randomized to receive high-dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin. An unblinding will then occur to select the optimum dose for Annamycin based on safety, tolerability, pharmacokinetics, and efficacy, aligning with the FDA's Project Optimus initiative. The second part of the trial will involve approximately 240 additional subjects randomized to receive HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin.
Clinical Data and Expert Opinion
Data from the MB-106 Phase 1B/2 AML clinical trial of Annamycin plus cytarabine continues to show promise, with the median durability of complete response composite (CRc) now exceeding eight months. A recent virtual AML Key Opinion Leader (KOL) event underscored the potential of Annamycin to significantly change the AML treatment landscape.
Walter Klemp, Chairman and Chief Executive Officer of Moleculin, stated, "We are thrilled to have emerged as a late stage company. Our focus remains on the ramp up for and execution of our upcoming MIRACLE trial... We believe with our recent clinical and regulatory 'wins,' we have foundationally set the stage for a transformational year ahead and the opportunity to drive significant value for all stakeholders."
Annamycin for Soft Tissue Sarcoma Lung Metastases
In addition to its AML program, Moleculin Biotech has completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma (STS) lung metastases. The study database is locked, and the clinical study report is being written and should be completed in early 2025.
Financial Update
Moleculin Biotech reported its financial results for the quarter ended September 30, 2024. Research and development (R&D) expense was $4.9 million, compared to $3.3 million for the same period in 2023. This increase is primarily related to costs incurred for clinical trials and sponsored research. As of September 30, 2024, the Company had cash and cash equivalents of $9.4 million and believes that the existing cash and cash equivalents as of September 30, 2024, will be sufficient to fund planned operations into the first quarter of 2025.
About Annamycin
Annamycin is a next-generation anthracycline designed to avoid multidrug resistance mechanisms and eliminate the cardiotoxicity commonly associated with currently prescribed anthracyclines. It has received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML, as well as Orphan Drug Designation for STS. The European Medicines Agency (EMA) has also granted Orphan Drug Designation for Annamycin in R/R AML.
