Moleculin’s Phase 3 AML trial gets FDA nod for size reduction

Published 13/02/2025, 15:30
Moleculin’s Phase 3 AML trial gets FDA nod for size reduction

HOUSTON - Moleculin Biotech, Inc. (NASDAQ:MBRX), a late-stage pharmaceutical company with a current market capitalization of $1.26 million, announced today that it has received guidance from the U.S. Food and Drug Administration (FDA) for its Phase 3 clinical trial protocol, allowing a reduction in trial size. According to InvestingPro data, the company maintains a healthy liquidity position with a current ratio of 2.08, indicating its ability to meet short-term obligations. The trial, known as the MIRACLE trial, is focused on evaluating the drug Annamycin in combination with Cytarabine for the treatment of acute myeloid leukemia (AML) in patients unresponsive to initial therapy.

The FDA’s feedback on the company’s Investigational New Drug (IND) amendment has led to a 10% reduction in Part B of the trial, without altering the major aspects of the study. This development supports Moleculin’s aim for an accelerated new drug approval timeline. InvestingPro analysis reveals that while the company holds more cash than debt, it is quickly burning through its cash reserves, with a net loss of $30.15 million in the last twelve months.

The MIRACLE trial will be conducted globally, with sites in the U.S., Europe, and the Middle East. The study employs an adaptive design, initially enrolling 75 to 90 subjects in Part A to determine the efficacy and safety of two different doses of Annamycin with high-dose cytarabine (HiDAC). An early unblinding after 45 subjects will provide preliminary data, with first results expected in the second half of 2025 and further unblinding in early 2026.

Part B will involve approximately 220 additional subjects, randomized to receive either HiDAC with a placebo or HiDAC with the optimal dose of Annamycin. This dose will be selected based on safety, pharmacokinetics, and efficacy data, aligning with the FDA’s Project Optimus initiative.

Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory AML, as well as Orphan Drug Designation for soft tissue sarcoma treatment. The European Medicines Agency (EMA) has also granted Orphan Drug Designation for Annamycin in AML.

Moleculin’s Annamycin is a next-generation anthracycline designed to avoid multidrug resistance and eliminate the cardiotoxicity associated with current treatments. The company’s pipeline also includes WP1066, an immune/transcription modulator, and a portfolio of antimetabolites for potential treatment of viruses and certain cancers.

This announcement is based on a press release statement from Moleculin Biotech, Inc. For further details on the MIRACLE trial, clinicaltrials.gov lists the study under identifier NCT06788756. The stock currently trades at $0.42, near its 52-week low of $0.40. Investors seeking deeper insights into Moleculin’s financial health and growth prospects can access comprehensive analysis through InvestingPro, which offers exclusive access to 17 additional ProTips and detailed financial metrics for informed decision-making.

In other recent news, Moleculin Biotech, Inc. has made significant advances in their clinical trials and investor relations. The company received the first European regulatory approval to begin recruiting for its Phase 3 trial of Annamycin, a treatment for acute myeloid leukemia (AML). This approval, granted by Ukraine’s Ministry of Health, is a crucial step for the global "MIRACLE" trial, which includes multiple sites worldwide.

Furthermore, Moleculin Biotech has amended the clinical trial protocol for the MIRACLE study, facilitating an earlier unblinding of preliminary primary efficacy data and safety/tolerability for the first 45 subjects in the trial. The amendment, agreed upon with the U.S. Food and Drug Administration (FDA), is expected to expedite the trial process.

In other developments, Moleculin Biotech has reached an agreement with an investor to modify certain terms of previously issued warrants. This agreement enables the pharmaceutical company to proceed with a proposed offering under specific conditions, with Moleculin agreeing to pay the investor a cash fee of $750,000 upon the closing of the proposed offering.

These recent developments reflect Moleculin Biotech’s commitment to operational execution and its ongoing efforts in initiating and enrolling study sites. The company continues to work towards addressing hard-to-treat tumors and viruses, as it advances its clinical trials and investor relations.

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

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