Moleculin expands EU trial for leukemia treatment

Published 12/05/2025, 13:50
Moleculin expands EU trial for leukemia treatment

HOUSTON - Moleculin Biotech, Inc. (NASDAQ:MBRX), a late-stage pharmaceutical company with a market capitalization of $14.7 million, has received approval from the European Medicines Agency (EMA) to include nine additional countries in its Phase 3 clinical trial for Annamycin, the company’s drug candidate for treating relapsed or refractory acute myeloid leukemia (R/R AML). This expansion is part of the MIRACLE trial, a global study also taking place in the US and the Middle East. According to InvestingPro data, the company maintains a strong balance sheet with more cash than debt, positioning it well for this expansion phase.

The trial, which targets patients who have not responded to venetoclax regimens, aims to evaluate the efficacy of Annamycin in combination with cytarabine, a standard chemotherapy drug. The approval covers all nine EU countries where Moleculin sought authorization, reflecting the urgent need for new treatments for R/R AML patients. With analyst price targets ranging from $4 to $20, significantly above the current trading price, market expectations for this trial appear optimistic.

Enrollment and dosing have already begun, with the first interim data readout anticipated in the second half of 2025. The MIRACLE trial utilizes an adaptive design, initially randomizing 75 to 90 subjects to receive high-dose cytarabine with either placebo or two different doses of Annamycin. The trial will unblind preliminary efficacy and safety data after the first 45 subjects have been treated.

Moleculin’s CEO, Walter Klemp, expressed optimism about the trial’s potential to meet enrollment and data milestones, thanks to the EMA approval and the international collaboration it represents. The company is committed to addressing the significant unmet need in R/R AML treatment and believes Annamycin could provide a critical second-line treatment option.

The trial’s design aligns with the FDA’s Project Optimus initiative, which emphasizes the balance of safety, pharmacokinetics, and efficacy in determining the optimum drug dose. The Phase 3 portion of the trial will proceed once nonclinical GLP studies are presented to the EMA, as required by the conditional approval.

Annamycin has already received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML treatment, as well as Orphan Drug Designation for soft tissue sarcoma. Similarly, the EMA has granted Orphan Drug Designation for Annamycin in treating R/R AML.

This news is based on a press release statement. For further information about the MIRACLE trial, reference identifiers are available on clinicaltrials.gov (NCT06788756) and clinicaltrials.eu (2024-518359-47-00). Moleculin continues to advance its pipeline of drugs targeting difficult-to-treat cancers and viruses, with Annamycin at the forefront of its clinical stage developments. With the company’s next earnings report due on May 13, 2025, investors can access comprehensive analysis and 13 additional ProTips through InvestingPro’s detailed research reports, which provide deeper insights into the company’s financial health and growth prospects.

In other recent news, Moleculin Biotech announced the issuance of two new U.S. patents for its cancer drug Annamycin, extending its intellectual property protection until June 2040. These patents cover the preparation methods for the drug, which is designed to reduce cardiotoxicity, a common issue with similar chemotherapy drugs. In related developments, Moleculin disclosed findings at the American Association for Cancer Research meeting, highlighting Annamycin’s potential synergy with other FDA-approved anticancer therapies. This could expand its use in treating various cancers, including leukemia and pancreatic cancer. Additionally, H.C. Wainwright maintained its Buy rating on Moleculin Biotech, with a price target of $8, citing confidence in the company’s ongoing Phase 3 MIRACLE trial for Annamycin. The trial aims to evaluate the drug in combination with high-dose cytarabine for acute myeloid leukemia. Meanwhile, Moleculin faced procedural challenges, canceling a Special Meeting of Stockholders due to a lack of quorum, which had been adjourned twice before. This meeting was intended to address undisclosed shareholder matters. These developments collectively highlight Moleculin’s ongoing efforts to advance its drug pipeline and address corporate governance challenges.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Latest comments

Risk Disclosure: Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers
© 2007-2025 - Fusion Media Limited. All Rights Reserved.