Moleculin receives Georgian approval for pivotal AML treatment trial

Published 09/07/2025, 13:48
Moleculin receives Georgian approval for pivotal AML treatment trial

HOUSTON - Moleculin Biotech, Inc. (NASDAQ:MBRX), a clinical-stage biotech company with a market capitalization of $12.6 million, announced Wednesday that Georgia’s Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) has approved its Clinical Trial Application for the Phase 2B/3 MIRACLE trial testing Annamycin in combination with cytarabine for relapsed or refractory acute myeloid leukemia (AML). According to InvestingPro data, the company maintains a strong balance sheet with more cash than debt, though it’s currently experiencing rapid cash burn typical of clinical-stage biotechs.

The approval follows recent authorization from the European Medicines Agency, expanding the global reach of the pivotal study that includes sites across the US, Europe, and the Middle East. The company expects the first Georgian patient to be treated by the end of August. With a current ratio of 1.35, InvestingPro analysis suggests the company maintains adequate liquidity to support its near-term operations.

Moleculin reported that seven subjects have already been treated in Part A of the trial, with one additional subject in screening. The company plans to add 16 clinical sites in Europe and the US by the end of August, building toward more than 30 sites for Part A of the study by year-end.

"This early success in recruitment underscores the capability of our clinical sites," said Walter Klemp, Chairman and CEO of Moleculin, in a press release statement.

The MIRACLE trial employs an adaptive design with Part A randomizing patients to receive high-dose cytarabine combined with either placebo, 190 mg/m² of Annamycin, or 230 mg/m² of Annamycin. The protocol allows for unblinding of preliminary efficacy data at 45 subjects and again at the conclusion of Part A (75-90 subjects).

Moleculin anticipates releasing initial data from the first 45 subjects in the second half of 2025, with the second data unblinding expected in the first half of 2026.

Annamycin, also known as naxtarubicin, has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory AML, as well as Orphan Drug Designation from the EMA.

In other recent news, Moleculin Biotech has announced the pricing of a public offering intended to raise approximately $5.9 million. The proceeds from this offering will support the advancement of its drug candidate Annamycin, along with other drug portfolios and research initiatives. Additionally, the U.S. Food and Drug Administration has agreed to Moleculin’s proposal for a pediatric study of Annamycin for children with relapsed/refractory acute myeloid leukemia. In another development, positive results from a Phase 1B/2 clinical trial of Annamycin for treating soft tissue sarcoma lung metastases have been reported, showing improved survival rates without cardiotoxicity. Meanwhile, Moleculin has received a non-compliance notice from Nasdaq for failing to meet the minimum stockholders’ equity requirement, though this does not immediately affect its stock trading. The company concluded its Q1 2025 earnings call without disclosing specific earnings figures but noted a cash position of $8 million, expected to fund operations into Q3 2025. Moleculin is also expanding its clinical trials globally, with European Medicines Agency approval for Annamycin in nine EU countries.

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