HOUSTON – Moleculin Biotech, Inc. (NASDAQ:), a clinical stage pharmaceutical company, announced today that the European Medicines Agency (EMA) has granted Orphan Drug Designation to its drug candidate Annamycin for the treatment of acute myeloid leukemia (AML).
The designation is a significant milestone for the company, providing benefits such as reduced regulatory fees and potential market exclusivity in the European Union for up to 10 years upon approval.
Annamycin is a next-generation anthracycline, a class of drug used in cancer treatment, that has been developed to avoid cardiotoxicity, a common and serious side effect of current anthracycline drugs. In clinical studies involving 82 subjects in the U.S. and Europe, Annamycin has shown no signs of cardiotoxicity. The drug, in combination with Cytarabine, has also demonstrated a preliminary composite complete response (CRc) rate of 60% in 2nd line AML subjects in a European trial.
The company’s Chairman and CEO, Walter Klemp, expressed optimism about the drug’s progress, citing the recent grant of a new patent extending commercial exclusivity through 2040 and the promising interim clinical data. Moleculin anticipates beginning a pivotal registration study for Annamycin as a 2nd line therapy in AML before the end of the year.
Orphan Drug Designation by the EMA is reserved for treatments aimed at rare, life-threatening, or chronically debilitating diseases affecting fewer than five in 10,000 people in the EU. This status comes in addition to Annamycin’s Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of relapsed or refractory AML and for the treatment of soft tissue sarcoma (STS) lung metastases.
Moleculin Biotech is focused on developing treatments for hard-to-treat tumors and viruses, including Annamycin for AML and STS lung metastases, as well as other drug candidates targeting brain tumors, pancreatic cancer, and cutaneous T-cell lymphoma. The interim and preliminary data mentioned is subject to change, and the company remains committed to advancing its clinical and regulatory strategies.
The information reported here is based on a press release statement from Moleculin Biotech, Inc.
InvestingPro Insights
As Moleculin Biotech, Inc. (NASDAQ:MBRX) garners attention with its recent Orphan Drug Designation for Annamycin, investors are closely monitoring the company’s financial health and stock performance. The InvestingPro data paints a detailed picture of the firm’s current market standing. With a market capitalization of just $9.53 million, Moleculin Biotech is considered a small-cap company, which often means it could have high growth potential but also comes with higher risk. Additionally, the stock’s price-to-book ratio as of the last twelve months ending Q4 2023 stands at 0.37, indicating that the stock may be undervalued compared to its book value.
However, the company’s financials show significant challenges. The latest metrics reveal an operating income, adjusted for the same period, at a loss of $29.63 million, which aligns with the InvestingPro Tip that Moleculin Biotech is quickly burning through cash. Furthermore, the stock has experienced a sharp decline over various timeframes, including a 1-week price total return of -10.27% and a staggering year-to-date price total return of -66.76%. This volatility is highlighted by another InvestingPro Tip, which notes that the stock price movements are quite volatile.
For investors seeking a more comprehensive analysis, InvestingPro offers additional insights, including tips on Moleculin’s cash position, which holds more cash than debt on its balance sheet, and the RSI indicator suggesting the stock is currently in oversold territory. To access these insights and more, investors can visit InvestingPro’s dedicated page for MBRX. For those ready to dive deeper, use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription. With a total of 14 InvestingPro Tips available, investors can make more informed decisions about Moleculin Biotech’s future in the pharmaceutical industry.
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