Moleculin Biotech, Inc. Q3 FY2024 earnings call

• Moleculin believes its current market cap undervalues it as a Phase 3 company, with reduced risk to approval pathway. Annamycin is a safer, more effective anthracycline for AML and other potential applications.
• MIRACLE trial updates: Phase 2 data showed 50% complete remission rate in second line patients, median OS now over 7 months, durability of responses, and adaptive trial design as per FDA input.
• Recruitment efforts: 60 sites interested, with management physically visiting sites globally, including in war-torn and rioting areas.
• Financials: Ended the quarter with $9.4 million in cash on hand, enough to reach Q1 2025, with market cap at ~$15.9 million.

No specific product segment financial performance detailed; focus is on Annamycin's potential in acute myeloid leukemia (AML) and beyond, with Annamycin being a safer, more effective anthracycline with patent protection through 2040.

• MIRACLE trial milestones: First subject treated in Q1 2025, interim primary efficacy and safety data in mid-2026, primary efficacy data for second line subjects in 2028, with rolling NDA submission in the second half of 2028.
• Potential to shorten approval timeline if data outperforms expectations, with consideration of Type A meetings with FDA if early unblinding shows strong efficacy.

• Biotech industry risk: Phase 3 trial success rate around 45%, with majority failing due to lack of efficacy.
• Recruitment and site risks: Complexities in global trial site setup, potential budget and timeline challenges due to varying per-patient costs in different regions.

Q: Update on median OS in second line patients?

A: Our overall median survival now is over seven months in this population, far greater than literature predictions.

Q: Reason for MIRACLE enrollment changes?

A: Refinement of trial design and statistics, with efforts to avoid misinterpretation of numbers, ensuring accurate representation of trial scope.

Q: Phase 3 trial cost?

A: A good run rate is ~$15 million over several quarters, influenced by trial site locations (lower costs in Eastern Europe/Western Asia vs. US).

Q: STS program and partnership?

A: Focus on MIRACLE trial, with interest in seeking a partner for the STS pivotal trial due to MIRACLE trial's resource focus.

Q: FDA special protocol assessment?

A: Decided not to pursue due to associated delay and potential constraints on protocol flexibility if an SPA were agreed upon.