Moleculin Biotech, Inc. Q4 FY2023 earnings call

• Focus on AML second line strategy with Annamycin showing promising results. - Annamycin has a 60% CRC rate in second line AML, 50% CR rate, and no cardiotoxicity observed in 82 subjects. - Plan to begin a pivotal registration study in 2024 for AML with potential accelerated approval. - Preclinical work on stat 3 inhibitors and metabolic inhibitors continues. - High unmet need in AML with current therapies having low CR rates for most patients. - Cardiotoxicity issues with traditional anthracyclines highlighted, Annamycin avoids this. - Progress in STS study with median progression-free survival over 3 months for certain dosing. - Cash on hand of ~$24 million as of year-end. - Plans to form a science advisory board, present final data at a conference, announce market exclusivity news, and meet with FDA in summer for approval pathway.

The key product segment is Annamycin. In second line AML patients, Annamycin generated a 60% CRC rate, including a 50% CR rate and 10% CRi. 82 subjects treated showed no indication of cardiotoxicity. In STS, the MB-107 study for soft tissue sarcoma with pulmonary metastases in patients with at least one prior therapy had a median progression-free survival over 3 months for those dosed 330 mg per meter square or less.

• Intend to complete the MB-106 AML trial in 2024, including first and third line subjects. - Hold end of Phase 2 meeting with regulators regarding second line data. - Initiate a pivotal study on AML using Annamycin's second line data. - Close the STS trial and move to an investigator-led, funded trial. - Form a science advisory board to guide new drug approval pathway. - Present final data at a prestigious conference. - Announce market exclusivity news. - Meet with FDA in summer to discuss data and establish approval pathway.

• Dependence on clinical trial results and regulatory approval. - Uncertainty in market acceptance of Annamycin. - Potential competition from other therapies. - Short selling issues affecting trading volume.

Q: What is the final patient number for first-line patients in the new Phase 2 cohort and what's needed to go forward with a pivotal first-line trial?

A: Anticipate going to FDA with second-line therapy as initial indication, first-line would be a randomized trial vs. other anthracyclines. Patient numbers depend on data from the trial. Phase 2 first-line patients' data will drive the decision, with a likely CR bogey around 40%.

Q: Thoughts on Phase 3 dose, FDA feedback, and potential for higher dosing?

A: Clinical team stopped at current dose as results were great with no toxicity. FDA feedback on dose is awaited, but current dose is effective with no safety issues observed, and higher dosing isn't necessary as current results are far beyond expectations and toxicity is not an issue at current dose.