Significant Enrollment Milestone for Moleculin’s AML Trial
First 45 patients expected to complete treatment by Q1 2026, showing strong momentum in recruitment
HOUSTON -- Moleculin Biotech, Inc., (Nasdaq: MBRX), has reported that 60% of its target number of subjects for the first planned interim unblinding of data have consented to participate in the pivotal Phase 2B/3 “MIRACLE” study. This clinical trial is evaluating the efficacy of Annamycin combined with cytarabine for the treatment of adults with acute myeloid leukemia (AML) who are refractory or have relapsed after induction therapy.
The MIRACLE study aims to enroll a total of 45 subjects for the initial unblind of the data. As of early November, the company has confirmed that recruitment efforts are ongoing, with additional subjects continually being identified by site investigators. The treatment for these first 45 subjects is expected to conclude in the first quarter of 2026, followed by the initial unblinding of results.
Exciting Potential Ahead in AML Treatment
Walter Klemp, Chairman and CEO of Moleculin, expressed positivity regarding the recruitment momentum and enthusiasm from investigators in the U.S. and Europe. He noted, “We see blinded response activity tracking within our expected range,” drawing on historical data comparable to trial arm equivalents. Despite challenges like bed shortages at certain European sites, the MIRACLE study is progressing according to its timeline.
As Moleculin aims for crucial milestones, the company believes Annamycin could represent a significant advancement in treatment options for AML patients. “We’re excited about Annamycin’s potential to improve outcomes and safety for those affected by this serious disease,” Klemp added.
Details of the MIRACLE Trial
The MIRACLE study stands as a Phase 2B/3, global multi-center, randomized, double-blind, placebo-controlled trial. This design allows the data from the 2B (Part A) portion to be combined with the Phase 3 (Part B) portion for a robust evaluation of its primary efficacy endpoint.
Currently, subjects enrolled in the trial are from sites across five countries, highlighting the diverse population under study. The company has strategically planned for the unblinding of preliminary primary efficacy data once the first 45 patients are treated. In addition to the first unblinding, a second is anticipated during the first half of the following year.
Anticipated Challenges but Continued Progress
As the company approaches the completion of treatment for these first subjects, it acknowledges potential slowdowns due to the holiday season and unexpected bed shortages in some European sites. However, Moleculin is optimistic that it will complete treatment and be ready to release findings related to the first 45 subjects in due time.
Being aligned with the FDA, Annamycin currently holds both Fast Track Status and Orphan Drug Designation for AML, alongside Orphan Drug Designation for soft tissue sarcoma. This regulatory backing, coupled with patent protections extending into 2040, positions Moleculin strongly within the competitive landscape of oncology therapeutics.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. focuses on developing new therapeutics for hard-to-treat cancers and viruses. The company’s flagship candidate, Annamycin, is engineered to circumvent multidrug resistance and reduce cardiotoxicity—it is currently under investigation for AML and soft tissue sarcoma with lung metastases.
The company’s ongoing MIRACLE trial aims to uncover the therapeutic benefits of Annamycin combined with cytarabine, due to its prior positive results experienced in the initial Phase 1B/2 study. Following regulatory input, Moleculin is confident in its path toward potential approval for AML treatment.
For more information about the company, please consider visiting www.moleculin.com or connecting with them on X, LinkedIn, and Facebook.
Frequently Asked Questions
What is the MIRACLE study about?
The MIRACLE study focuses on assessing the efficacy of Annamycin combined with cytarabine for adult patients with relapsed or refractory acute myeloid leukemia.
How many participants are required for the initial data unblinding?
A total of 45 participants are required for the first planned unblind of data in the MIRACLE study.
What potential advantages does Annamycin offer?
Annamycin is designed to avoid multidrug resistance and does not have the cardiotoxic effects typical of other anthracyclines.
When is the treatment for the first cohort expected to be completed?
The treatment for the initial cohort of 45 subjects is expected to be finalized by the first quarter of 2026.
Where can I find more information about the trial?
More details can be found on clinical trial registries or by visiting the company's website at www.moleculin.com.
