Tonix Pharmaceuticals stock halted ahead of FDA approval news
HOUSTON - The U.S. Food and Drug Administration has agreed to Moleculin Biotech, Inc.’s (NASDAQ:MBRX) proposal for a single pediatric approval study of its cancer drug Annamycin in combination with Cytarabine for children with relapsed/refractory acute myeloid leukemia (R/R AML). The clinical-stage biotech company, currently valued at $9.4 million, maintains a strong balance sheet with more cash than debt, according to InvestingPro data.
In its response to Moleculin’s Initial Pediatric Study Plan, the FDA recommended including patients as young as 6 months old, younger than the company’s proposed minimum age of 2 years. The agency also indicated that efficacy data from adults could be extrapolated to children if drug concentration exposure and safety profiles are comparable.
Annamycin, also known as naxtarubicin, is being developed as an alternative to traditional anthracyclines, which are used to treat approximately 60% of children with cancer but carry risks of heart damage. According to the company, an independent expert’s review found no cardiotoxicity in 84 adult patients treated with Annamycin to date.
Moleculin is currently conducting the MIRACLE trial, a pivotal Phase 3 study evaluating the Annamycin-Cytarabine combination (AnnAraC) in adult patients with R/R AML. Initial data from this trial is expected in the second half of 2025.
The company plans to submit a revised pediatric study plan to the FDA later this quarter and anticipates beginning the pediatric clinical study in the second half of 2027.
Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating R/R AML, and an additional Orphan Drug Designation for treating soft tissue sarcoma. The European Medicines Agency has also granted Orphan Drug Designation for R/R AML.
This article is based on information from a company press release statement.
In other recent news, Moleculin Biotech, Inc. has reported positive results from its U.S. Phase 1B/2 clinical trial of Annamycin for treating soft tissue sarcoma lung metastases. The trial demonstrated improved overall survival and progression-free survival rates, with a Clinical Benefit Rate of 59.4%. Additionally, Moleculin announced the initiation of the Phase 3 MIRACLE trial for Annamycin, which has received approval from the European Medicines Agency in nine EU countries. However, the company received a non-compliance notice from Nasdaq for not meeting the minimum stockholders’ equity requirement, giving them 45 days to submit a compliance plan.
Moleculin’s Q1 2025 earnings call revealed that the company ended the quarter with $8 million in cash, projected to fund operations into Q3 2025. The company highlighted the need to raise an additional $15 million to sustain operations through Q1 2026. Despite not disclosing specific earnings figures, Moleculin remains focused on advancing Annamycin and expanding its clinical trials globally. The company also emphasized the potential of its drug to become the first non-cardiotoxic anthracycline, a significant development in cancer treatment.
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