Moleculin Biotech (MBRX) investor relations material

Key clinical developments

• Annamycin is in a pivotal FDA-guided phase III trial for relapsed/refractory AML, showing a complete remission rate three times higher than control after a single cycle, with zero cardiotoxicity detected in over 90 patients.

• The trial is about 80% recruited in Part A, with full recruitment expected in Q3, and a second unblinded readout anticipated by year-end or early next year.

• Annamycin arms in the MIRACLE trial delivered 43% and 36% CR rates at two doses, both outperforming control by 300% or more, with consistent efficacy across phase II and III trials.

• The Independent Data Monitoring Committee recommended continuing the trial without modification, indicating no safety concerns.

• Fast Track status allows a rolling NDA, with potential approval targeted for 2030.

Market opportunity and financial outlook

• Peak revenue is projected at $900 million for AML alone and nearly $10 billion across the pipeline, driven by Annamycin's broad potential indications.

• Annamycin's mutation-agnostic profile and lack of cardiotoxicity position it as a disruptive therapy with 10x growth potential beyond AML.

• Payers support pricing between $27,000 and $35,000 per infusion, with a five-day regimen reducing hospital stays and downstream costs.

• The addressable AML market is about 51,000 cases annually in the U.S. and accessible ex-U.S. markets, with a 25% market share yielding over $600 million in second-line peak revenue.

• Expansion into first-line AML and other cancers could drive total peak revenue to $10 billion.

Competitive landscape and physician feedback

• Targeted therapies have not delivered durable outcomes in AML; Annamycin excels in post-venetoclax settings and is easier on patients.

• Independent research with 19 oncologists showed high dissatisfaction with current second-line AML options and strong interest in Annamycin due to its safety and efficacy.

• Payers and hospital administrators value Annamycin's potential to reduce total cost of care and administrative friction.

• Annamycin is seen as the likely default salvage therapy for relapsed/refractory AML and a future backbone for combination regimens.

• Key opinion leaders became strong supporters after reviewing clinical data, highlighting its game-changing potential.