Moleculin Biotech's 2024Q4: Discrepancies in MIRACLE Trial Decisions on Enrollment, Dosage, and Costs

These are the key contradictions discussed in Moleculin Biotech's latest 2024Q4 earnings call, specifically including: MIRACLE trial enrollment and dose selection, Annamycin's dose selection in the MIRACLE trial, interchangeability of Annamycin with Cytarabine, and the cost estimate for the MIRACLE trial:



MIRACLE Phase 3 Pivotal Trial:
- Moleculin Biotech's MIRACLE Phase 3 pivotal trial for Annamycin is beginning enrollment, with sites planned in the US, Europe, and the Middle East.
- The primary endpoint is the rate of complete remission of leukemia at approximately day 35, with interim unblinding of data at 45 and 75-90 subjects.
- The trial is designed to win approval for Annamycin in combination with Cytarabine for the second-line treatment of relapsed and refractory AML patients.

Annamycin Efficacy and Safety:
- Annamycin has shown a 50% complete remission rate in second-line AML patients, exceeding the historical 17%-18% CR rate.
- There is a complete absence of drug-related cardiotoxicity, making it non-cardiotoxic compared to traditional anthracyclines.
- Annamycin has been shown to overcome resistance to Venetoclax in AML, delivering durable complete remission in patients where Venetoclax has failed.

Soft Tissue Sarcoma (STS) Trial:
- Moleculin is preparing to publicly release data from its MB107 trial of Annamycin in monotherapy treating advanced soft tissue sarcoma metastases to the lungs in April.
- The data shows efficacy in challenging patients, with overall survival numbers comparable to first-line patients, indicating potential for a pivotal trial.

MIRACLE Trial Timeline and Dosing:
- Two Annamycin dosing regimens (190 and 230 mg/m2) are being tested in Part A of the MIRACLE trial, with one regimen to be selected based on efficacy and safety data.
- The primary efficacy endpoint is the rate of complete remission of leukemia at approximately day 35.
- An early unblinding of data at 45 subjects is expected before the end of 2025, with a second unblinding at 75-90 subjects in the first half of 2026.