During previous trading session, the stock price of Moleculin Biotech, Inc. (NASDAQ: MBRX) saw a notable uptick, rising 23.11% to close at $3.09. This encouraging development came after MBRX revealed a revised clinical trial design for its Phase 3 pivotal trial, which evaluates the use of Annamycin and Cytarabine to treat relapsed or refractory acute myeloid leukemia (AML).
Strategic Amendments to the “MIRACLE” Trial
The goal of Moleculin’s Phase 3 trial, appropriately called “MIRACLE,” is to determine if anamycin and cytarabine work well together for individuals who are not responding to induction treatment. The U.S. Food and Drug Administration (FDA) accepted the updated study protocol, which takes a more efficient approach and might speed up the trial’s duration.
MBRX’s dedication to expanding AML treatment choices is demonstrated by the worldwide study, which will include locations both inside and outside of the United States. The new protocol allows Moleculin to unblind data from the first 45 subjects earlier than originally planned.
This early analysis is expected to provide actionable insights into all three study arms, offering a clearer understanding of Annamycin’s potential and enabling strategic decisions, including accelerated regulatory pathways and advanced partnering discussions.
Enhanced Trial Design Aligns with Regulatory Standards
The trial’s second phase (Part B) will include 244 subjects, slightly increasing from the initial estimate of 240. This adjustment accommodates the statistical requirements of the interim unblinding process.
Participants will be randomized to receive either HiDAC (high-dose Cytarabine) with a placebo or HiDAC with the optimal dose of Annamycin. Dose selection will adhere to FDA’s Project Optimus guidelines, emphasizing a balance of safety, efficacy, and pharmacokinetics.
The updated protocol is under review by the Institutional Review Board (IRB) and will be filed with Moleculin’s amended Investigational New Drug (IND) application upon approval.
Regulatory Designations and Market Potential
The FDA has granted Annamycin Orphan Drug Designation and Fast Track Status for relapsed or refractory AML. It also has similar designations from the European Medicines Agency (EMA), extending its potential market reach. With these strategic developments, Moleculin (MBRX) is positioning itself as a significant contender in the fight against AML, supported by robust clinical and regulatory initiatives.
