Moleculin Biotech advances Annamycin AML trial protocol

HOUSTON - Moleculin Biotech, Inc. (NASDAQ:), a late-stage pharmaceutical company, has announced an amendment to its clinical trial protocol for the Phase 3 "MIRACLE" study of Annamycin in treating acute myeloid leukemia (AML). This amendment, agreed upon with the U.S. Food and Drug Administration (FDA), will expedite the unblinding of preliminary primary efficacy data and safety/tolerability for the first 45 subjects in the trial.

The MIRACLE trial is assessing the efficacy of Annamycin in combination with Cytarabine for AML patients who have not responded to initial therapy. The amended protocol will allow for an earlier assessment of the three arms of the study, with an expected unblinding of data in the second half of 2025. This early data release is anticipated to facilitate financing and potential strategic partnerships for the trial.

The trial's adaptive design will initially include 75 to 90 subjects randomized to receive either a high dose of Cytarabine with a placebo or with Annamycin at doses of 190 mg/m2 or 230 mg/m2. Subsequent to the initial unblinding, an additional 244 subjects will be randomized to receive either the placebo or the optimum dose of Annamycin, determined by the balance of safety, efficacy, and pharmacokinetics.

Annamycin holds Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory AML, as well as Orphan Drug Designation from the European Medicines Agency (EMA). This designation is indicative of the significant need for new treatments in this area and the potential benefits Annamycin may offer.

The Institutional Review Board (IRB) is currently reviewing the amended protocol, which, upon approval, will be filed with the FDA as part of the Company's Initial New Drug (IND) application. Moleculin's commitment to addressing hard-to-treat tumors and viruses is further exemplified by its development of other therapeutic candidates, including WP1066 for brain tumors and WP1122 for pathogenic viruses.

This announcement is based on a press release statement from Moleculin Biotech, Inc. and is intended to provide factual information regarding the company's clinical trial developments.

In other recent news, Moleculin Biotech, Inc. has made significant strides in its clinical trials and financial position. The company received Institutional Review Board approval for a pivotal Phase 3 trial of its drug candidate Annamycin, set to begin enrollment in Q1 2025. This drug, aimed at treating relapsed or refractory acute myeloid leukemia, has been granted Fast Track Status and Orphan Drug Designation by the FDA and EMA.

In financial developments, Moleculin ended the quarter with $9.4 million in cash, anticipated to fund operations until Q1 2025. The company is also planning a rolling New Drug Application submission by late 2028.

These developments come alongside the ongoing Phase 3 MIRACLE trial for Annamycin, with recruitment efforts in progress at 60 sites and 17 additional sites targeted globally. Moleculin Biotech expects crucial data readouts from this trial by mid-2026. The company's leadership, including CEO Walter Klemp, has expressed confidence in the drug's market potential and the trial's design. These recent developments represent critical steps in Moleculin Biotech's ongoing efforts to advance Annamycin through the clinical trial process.

InvestingPro Insights

Moleculin Biotech's recent protocol amendment for its Phase 3 MIRACLE study comes at a critical time for the company, as reflected in its financial metrics and market performance. According to InvestingPro data, Moleculin's market capitalization stands at a modest $7.14 million, indicating its current small-cap status in the biotech sector.

The company's financial health presents a mixed picture. An InvestingPro Tip highlights that Moleculin "holds more cash than debt on its balance sheet," which could be crucial for funding its ongoing clinical trials, including the MIRACLE study. This financial cushion may provide some stability as the company progresses through its research and development phases.

However, investors should note that Moleculin is "quickly burning through cash," another InvestingPro Tip that underscores the capital-intensive nature of biotech research and the importance of the expedited unblinding of preliminary data from the MIRACLE study. This early data release could indeed be critical for securing additional financing or partnerships, as mentioned in the article.

The company's stock performance has been challenging, with InvestingPro data showing a significant year-to-date price total return of -80.51% as of the latest available data. This decline aligns with the InvestingPro Tip indicating that the "price has fallen significantly over the last year." The upcoming data release from the MIRACLE study could potentially serve as a catalyst for the stock, depending on the results.

It's worth noting that analysts do not anticipate the company to be profitable this year, according to another InvestingPro Tip. This expectation is consistent with the company's focus on clinical development rather than immediate commercialization.

For investors seeking a more comprehensive analysis, InvestingPro offers additional tips and insights, with 11 more tips available for Moleculin Biotech. These additional insights could provide valuable context for understanding the company's position as it advances its clinical programs.

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