Moleculin Biotech, Inc. Q4 FY2024 earnings call

• The MIRACLE Phase 3 pivotal trial for Annamycin in combination with Cytarabine for second-line AML is ongoing, with 25 sites selected, regulatory and ethics approval in a European country, and patient screening begun. First patient treatment expected this quarter.
• The trial will have multiple unblindings, with first unblinding at 45 subjects by end of 2024 and second at 75-90 subjects in first half of 2026. Primary endpoint is complete remission after one cycle (approx. 30 days).
• Phase 2 data for Annamycin shows 50% complete remission rate, 11-month overall survival, and 9-month progression-free survival, better than existing therapies for second-line AML.
• Cash on hand at end of 2024 plus $9 million raised in Feb 2025 totals ~$13 million, lasting into third quarter 2025. Focus on operational execution and meeting milestones.
• Expect to publicly release data from MB107 clinical trial of Annamycin monotherapy for advanced soft tissue sarcoma metastases to lungs in April 2025, with plans to develop a pivotal investigator-initiated trial in Europe later in 2025.

No specific product segment financial performance discussed; focus is on the Annamycin trial for relapsed and refractory AML patients, with Phase 2 data showing 50% complete remission rate, 11-month overall survival, and 9-month progression-free survival.

• First subject enrolled and treated in MIRACLE trial expected in first quarter 2025, with data readout and unblinding of efficacy and safety by end of 2024.
• First half of 2026 will have unblinding of data from 75-90 subjects in MIRACLE trial, sharing safety and efficacy data and setting optimum dose for Part B.
• Plan to use data from MB107 to develop a pivotal investigator-initiated trial in Europe later in 2025.

• Uncertainty in clinical trial outcomes, including whether the control arm (placebo plus high-dose Cytarabine) will perform as expected, which could impact the trial's success.
• Regulatory approval process uncertainties, including potential delays in site approvals and IRB processes, particularly in the US.
• Risk that actual results may differ from forward-looking statements due to various factors like clinical trial data variability, regulatory requirements, and external market conditions.

Q: Rationale for choosing 190 dose in MIRACLE trial and dose for soft tissue sarcoma trial?

A: Chose 190 for MIRACLE trial as per FDA's Project Optimus, but both 190 and 230 showed efficacy in Phase 2. For soft tissue sarcoma, monotherapy used higher dose (~300 mg per meter square) with good efficacy and safety, and plan to request end of Phase 1/2 meeting with FDA for pivotal trial dosing.