Moleculin Biotech, Inc. Q1 FY2023 earnings call

Management Statement and Operational Highlights

• Clinical Results Focus: Emphasis on AML and soft tissue sarcoma clinical data. In AML, final cohort of MB-105 trial had notable responses with corrected response classifications. In soft tissue sarcoma, ongoing trial in lung metastases patients showing better-than-expected PFS without cardiotoxicity.
• Annamycin Progress: Phase 1b in STS completed with recommended dose, Phase 2 enrollment over halfway; AML Phase 1 monotherapy trials completed, moved to combination therapy with Cytarabine, first cohort in combination trial had durable complete response.
• Other Technologies: WP1066 in Phase 1 trials for brain cancer, potential strategic partnerships; WP1122 Phase 1 done, ready for Phase 2, NIH animal testing on viral diseases upcoming.
• Financials: R&D expense $5.7M (+$1M), G&A expense up 8% (legal/consulting), cash >$37M to Q3 2024.

Segment Performance

• Annamycin (AML): Final cohort of MB-105 trial had 5 patients at 240 mg per square meter. 4 of the last 8 patients treated had responses, with 2 previously labeled partial responses upgraded to complete responses, resulting in a 50% complete response rate in heavily pre-treated AML patients.
• Annamycin (Soft Tissue Sarcoma): MB-107 trial nearly half enrolled, preliminary median Progression-Free Survival (PFS) approaching 3 months in patients with lung metastases, showing significant improvement over historical norms for untreated patients with worst prognoses, and no cardiotoxicity. Phase 1b completed with 330 mg per meter squared as recommended dose for Phase 2, 60% of subjects showed stable disease through two or more cycles.
• WP1066 (Immune Transcription Modulator): Phase 1 clinical trials in brain cancer (glioma, medulloblastoma) at prestigious medical universities, established 8 mg per kilogram as safe dose, clinical and radiological efficacy (limited data), orphan drug designations for brain tumors and rare pediatric disease.
• WP1122 (Metabolism Glycosylation Inhibitors): Phase 1 dose escalation study in normal volunteers completed, established safety and pharmacokinetics, ready for Phase 2 efficacy studies. Orphan drug designation for glioblastomas, NIH funded animal testing on arenaviruses upcoming.

Guidance

• AML: Expect to announce safety and efficacy in second cohort of combination therapy soon, aiming for Phase 2 expansion.
• Soft Tissue Sarcoma: STS trial recruiting quickly, potential to beat early 2024 timing estimate, may meet with FDA before full enrollment.
• Other Technologies: Top line results for WP1066 pediatric Phase 1 trial later, Phase 2 imminent for WP1122, NIH study on WP1122 portfolio upcoming.

Risks

• Clinical Data Uncertainty: Clinical trial data preliminary and subject to change.
• Screening Failures: Potential screening failures in patient enrollment.
• Market Stress: Small biotech market stress affecting share price, though strong Phase 2 data and FDA dialogue may mitigate.
• Historical Cardiotoxicity: Historical data shows 65% cardiotoxicity above lifetime max, but Moleculin's trials show 100% no cardiotoxicity, but reliance on third-party data.

Question and Answer

Q: Are the second cohort Phase 1b AML combo therapy patients being dosed yet and update frequency?

A: Just begun dosing first patient in second cohort, expect cohort to fill quickly; will announce cohort end and efficacy whether good or bad, possibly before next quarterly call.

Q: Number of patients above lifetime max in AML and STS, and FDA dialogue for AML?

A: Substantial number of patients above lifetime max (over 550 mg/m²), none showing cardiotoxicity; plan to dialogue with FDA using mountain of data on cardiotoxicity absence to explore U.S. expansion, will meet with FDA when enough efficacy data in expansion cohort.