Moleculin Gains EU Clearance to Expand Leukemia Drug Trial Across Nine EU Countries

Moleculin Biotech (NASDAQ: MBRX) has taken a major step forward in its development of Annamycin, a next-generation cancer therapy, by receiving authorization from the European Medicines Agency to expand its late-stage clinical trial across nine EU countries.

• EMA approval granted for Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain
• Trial is focused on patients with relapsed or treatment-resistant acute myeloid leukemia (AML)
• Initial data from the first 45 patients expected in the second half of 2025

The clinical trial, known as the MIRACLE study, is evaluating a combination of Annamycin and an existing chemotherapy drug (cytarabine) in patients with a form of blood cancer called acute myeloid leukemia. These patients have either relapsed after initial treatment or failed to respond to prior therapies, including newer treatments like venetoclax.

The recent EMA decision allows Moleculin to open trial sites in nine European countries, joining existing locations in the U.S. and Middle East. The company is already enrolling and treating patients, with early results expected later this year.

Annamycin is a newer type of cancer drug designed to address some of the key limitations of older chemotherapy treatments. Specifically, it aims to avoid damage to the heart and overcome resistance that can make standard drugs less effective in certain patients. Annamycin has received “Orphan Drug” and “Fast Track” designations in the U.S., which are special statuses granted by the FDA to support the development of treatments for rare and serious diseases.

The MIRACLE trial will ultimately involve 265 patients, starting with a smaller group to assess safety and response. Moleculin will use this early data to guide dosing and other trial decisions before advancing to the broader Phase 3 portion of the study.

The company emphasized that it continues to work with both U.S. and European regulators to keep the trial aligned across regions, and does not expect any regulatory differences to delay progress.