Moleculin Biotech (MBRX) Corporate presentation summary

Pipeline and technology overview

• Lead asset Annamycin is a next-generation anthracycline with no observed cardiotoxicity and strong efficacy in relapsed/refractory AML and soft tissue sarcoma lung metastases, with additional preclinical work in pancreatic and other cancers.

• Phase 3 pivotal trial (MIRACLE) for R/R AML is underway, with adaptive design and FDA guidance; two major data readouts expected in 2026.

• Additional pipeline assets include WP1066 (STAT3 inhibitor) for glioblastoma and pediatric brain tumors, and WP1122 for oncology and virology indications.

• All technology is derived from or jointly developed with MD Anderson Cancer Center.

Clinical efficacy and safety

• Annamycin demonstrated a 50% complete remission rate in 2nd-line AML, more than double the rate of approved therapies, with durable responses and improved overall survival.

• Zero cardiotoxicity observed in 84 subjects, enabling repeated dosing beyond FDA lifetime limits for traditional anthracyclines.

• In soft tissue sarcoma lung metastases, Annamycin achieved median overall survival of 13.5 months, outperforming historical controls even in heavily pretreated patients.

• Efficacy is dose-dependent, with improved progression-free and overall survival in patients receiving higher doses and fewer prior therapies.

Market opportunity and competitive landscape

• Annamycin addresses a significant unmet need in AML, where ~60% of patients do not achieve durable remission with current therapies.

• Broad potential across multiple solid tumor indications, with a total addressable market estimated at $5–12 billion annually.

• Competitive therapies in AML show lower complete remission rates (15–37%) compared to Annamycin (50–60%).

• Management team has over 200 years of combined drug development experience and multiple FDA approvals.