On November 13, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported an enrollment update with 60% of the target number of subjects for the first planned interim unblinding of data having consented to its pivotal Phase 2B/3 "MIRACLE" study of Annamycin in combination with cytarabine for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML). The targeted number for the first unblinding of data is 45 subjects. Additional subjects continued to be identified by site investigators. This update is as of November 4, 2025, as identification and recruitment are ongoing. The Company expects to complete treatment of the first 45 subjects in the first quarter of 2026 with the initial unblinding of data thereafter.
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Walter Klemp, Chairman and CEO of Moleculin, commented, "We’re very encouraged by the strong momentum in recruitment and enthusiasm I’ve personally heard from investigators around Europe and the US. To date, we are seeing blinded response activity tracking within our expected range, based on historical responses of the trial arm equivalents. Although enrollment at some sites in Europe has been impacted by bed shortages, the MIRACLE study continues to progress as planned. As we move toward our first unblinding milestone, we are excited about Annamycin’s potential to fill a major gap in AML treatment. We believe we’re well on our way to determining if Annamycin has the potential to offer a much-needed, safer, and more effective option for patients facing this devastating disease."
MIRACLE Trial Progress and Next Steps
The MIRACLE study (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a Phase 2B/3, global multi-center, randomized, double-blind, placebo-controlled, adaptive designed clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) portion for purposes of measuring its primary efficacy endpoint. The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo.
The Company expects to reach the recruitment and treatment of the first 45 subjects in the first quarter of 2026 with unblinding thereafter, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.
The currently enrolled subjects are from sites across five countries, providing a diverse base of subjects. With the upcoming holidays in addition to unexpected bed shortages at certain EU sites, the Company expects treatment of enrolled subjects to slow, pushing the completion of the first 45 subjects into the first quarter of 2026. Such data will be audited, locked, and reviewed prior to release. The release of the unblinded data will be thereafter.
For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.
Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.
(Press release, Moleculin, NOV 13, 2025, View Source [SID1234659907])