HOUSTON - Moleculin Biotech (NASDAQ:MBRX), Inc., a late-stage pharmaceutical company, has announced the receipt of Institutional Review Board (IRB) approval for a pivotal Phase 3 trial of its drug candidate Annamycin, to be used in combination with cytarabine for treating relapsed or refractory acute myeloid leukemia (R/R AML). This approval is a significant step towards the initiation of the trial, named the MIRACLE study, which is set to begin enrollment in the first quarter of 2025.
The MIRACLE trial will be conducted globally and aims to assess the efficacy and safety of Annamycin in combination with high-dose cytarabine (HiDAC). The study will initially randomize 75 to 90 subjects to receive HiDAC with either a placebo or one of two doses of Annamycin. Upon unblinding, the optimum dose of Annamycin will be selected for the subsequent part of the trial, which will involve approximately 240 additional subjects.
Annamycin has been granted Fast Track Status and Orphan Drug Designation by the FDA for the treatment of R/R AML, and it also holds Orphan Drug Designation from the European Medicines Agency (EMA) for the same indication. The drug is designed to avoid multidrug resistance and eliminate the cardiotoxicity associated with current anthracyclines.
The announcement was made by Walter Klemp, Chairman and CEO of Moleculin, who expressed confidence in the trial's design and the data generated thus far. The trial's design aligns with the FDA's Project Optimus initiative, which aims to optimize dosing for patient benefit.
Moleculin's broader portfolio includes WP1066, an immune/transcription modulator, and WP1122, an antimetabolite targeting viruses and certain cancers. The company's development strategy for Annamycin is based on feedback from the FDA and is subject to further regulatory filings and potential additional feedback.
The information in this article is based on a press release statement from Moleculin Biotech, Inc.
In other recent news, Moleculin Biotech released updates on its financial position and the progress of its lead drug candidate, Annamycin, during its quarterly earnings call. The company ended the quarter with $9.4 million in cash, anticipated to fund operations until Q1 2025. The Phase 3 MIRACLE trial for Annamycin, intended to treat acute myeloid leukemia (AML), is currently underway with recruitment efforts in progress at 60 sites, and 17 additional sites targeted globally. Annamycin has shown promising results, with a 50% complete remission rate in second-line AML patients. Moleculin Biotech plans to begin treating subjects in the MIRACLE trial in early 2025 and expects crucial data readouts by mid-2026. The company also aims for a rolling New Drug Application (NDA) submission by late 2028. These recent developments represent significant steps in Moleculin Biotech's ongoing efforts to advance Annamycin through the clinical trial process.
InvestingPro Insights
As Moleculin Biotech (MBRX) advances its pivotal Phase 3 trial for Annamycin, investors should consider some key financial metrics and insights from InvestingPro. The company's market capitalization stands at a modest $7.14 million, reflecting its current developmental stage in the pharmaceutical industry.
InvestingPro data reveals that Moleculin's stock has faced significant challenges, with a year-to-date price total return of -80.51% as of the latest available data. This performance aligns with an InvestingPro Tip indicating that the stock price has fallen significantly over the last year. Despite these setbacks, the company's recent IRB approval for the MIRACLE study could potentially serve as a catalyst for future growth.
Another relevant InvestingPro Tip highlights that Moleculin holds more cash than debt on its balance sheet, which could be crucial for funding the upcoming Phase 3 trial and other research and development efforts. However, investors should note that the company is quickly burning through cash, a common characteristic of biotech firms in the clinical trial phase.
For those seeking a deeper understanding of Moleculin's financial position and growth prospects, InvestingPro offers additional tips and insights. In fact, there are 11 more InvestingPro Tips available for MBRX, providing a comprehensive view of the company's financial health and market position.
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