Clinical Development Update - The company has received US Institutional Review Board (IRB) approval for the pivotal, adaptive Phase 3 clinical trial (MIRACLE trial) and engaged a leading contract research organization (CRO) [1] - The MIRACLE trial is on track to begin dosing of Annamycin in combination with cytarabine for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML) in Q1 2025 [1] - Expected timelines for recruitment updates and preliminary readouts of the MIRACLE trial have been accelerated to 2H 2025 (n=45) and 1H 2026 (n=~75-90) with potential for an accelerated NDA process beginning as early as 2027 [1] - Annamycin plus Ara-C achieved a 60% CR/CRi rate in subjects who were relapsed from or refractory to Venetoclax regimens, more than 4 times greater than published historical rates [1] - The MIRACLE trial will be global, including sites in the US, Europe, and the Middle East [3] - The trial utilizes an adaptive design with the first 75 to 90 subjects randomized in Part A to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin [4] - The amended protocol allows for unblinding of preliminary primary efficacy data (CR) and safety/tolerability at 45 subjects, with the first unblinding expected in 2H 2025 and the second in 1H 2026 [4] - Part B of the trial will randomize approximately 244 additional subjects to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin [5] Annamycin Performance and Potential - Annamycin has demonstrated a 50% CR rate and a 60% CRc rate for second-line subjects in the Phase 1B/2 study (n=10) [12] - Median durability was ~8 months and increasing, with median overall survival of 9.1 months for 0-6 prior lines of therapies (n=22) and 11.6 months for second-line subjects (n=10) [12] - Annamycin has shown strong efficacy even where prior venetoclax combination therapy has failed [12] - The company believes Annamycin is a potential game-changing asset for the treatment of AML, with significant value creation potential [2] - Annamycin has outperformed the response rates seen in billion-dollar assets in the AML space [2] - The drug has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of R/R AML and soft tissue sarcoma, as well as Orphan Drug Designation from the EMA for R/R AML [9] Expected Milestones - 1Q 2025: First subject enrolled and treated in the MIRACLE trial [12] - 2H 2025: Data readout (n=45) unblinded efficacy/safety review [12] - 1H 2026: Interim efficacy and safety data (n=~75-90) unblinded and optimum dose set for the MIRACLE trial [12] - 2027: Begin enrollment of third-line subjects in MIRACLE2 and end enrollment of second-line subjects [12] - 2028: Primary efficacy data for second-line subjects in MIRACLE and begin submission of a Rolling New Drug Application (NDA) for accelerated approval [12] Other Development Programs - The company 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 lung metastases [8] - The clinical study report for the soft tissue sarcoma trial is expected to be completed in early 2025 [8] - The company is also developing WP1066, an Immune/Transcription Modulator targeting brain tumors, pancreatic and other cancers, and a portfolio of antimetabolites including WP1122 for the potential treatment of pathogenic viruses and certain cancer indications [15]
Moleculin Highlights Development Progress of Annamycin, Phase 2 Data Outperforms Billion-Dollar Assets in AML, Phase 3 Data Readouts in 2025 & 2026