Moleculin Biotech, Inc. Q3 FY2023 earnings call

• Walter Klemp highlighted that Annamycin is the lead program, a next-generation anthracycline designed to be non-cardiotoxic and effective for AML and advanced STS. Phase 2 data for AML and STS were discussed, with preliminary results showing promise. - Paul Waymack provided specifics on AML Phase 2 data: first eight patients in a trial with Annamycin and Cytarabine had a 38% complete response rate among those who completed dosing. For STS, Phase 2 enrollment is complete with preliminary data showing favorable response rates at optimal dosing. - Jonathan Foster mentioned the company had ~$25 million in cash on hand, with a runway into Q3 2024, and discussed ongoing and planned clinical trials utilizing non-dilutive funding.

No detailed financial performance by product segment provided in the transcript.

• AML: Recruitment in Phase 2 trial expected to be fulfilled early 2024, end of Phase 2 meetings with FDA/EMA anticipated, potential pivotal trial start in second half of 2024. - STS: Final readout of Phase 2 data expected in Q2 2024, potential investigator-funded pivotal trials in first half of 2024 with initiation in second half.

• Reliance on forward-looking statements with actual results potentially differing materially. - Clinical trial data discussed are preliminary and subject to change. - Regulatory challenges in obtaining marketing approval. - Uncertainties in funding for certain programs, particularly for 1122.

Q: Can you really exclude from the percent CR rate analysis in AML the Cytarabine, SAA, SAE, as well as the stroke patient?

A: Paul Waymack stated that in a Phase 2 study, they do things differently than in a pivotal trial; in Phase 2, they present patients who had finished treatment with documented data, and in a pivotal trial, all would be included.

Q: Is it fair to say that for the STS LM data set in today's press release that it is no new data compared to the update a week ago?

A: Paul Waymack disagreed, stating they added a dose and got substantially better data when looking at second and third line therapies with the optimal dose.

Q: How many patients do you expect to have to enroll in a pivotal trial for both AML and STS-LM?

A: Paul Waymack said for AML, a single-arm trial would take about 100 patients, and a randomized trial about 300; for STS, the ultimate number depends on FDA demands but would be in the few hundreds.

Q: What kind of progress have you made in finding funding or getting an investigative sponsored trial going with the other two products, 1066 and 1112?

A: Walter Klemp discussed progress on 1066, with ongoing discussions and a trial listed on clinicaltrials.gov, and slow progress on 1112 due to slow-moving U.S. government grant funding.

Q: In the STS data, with the median PFS, it looks like its 3.4 months for patients who are less than 330 mg per meter squared. What was the trabectadine OS before it was approved?

A: Walter Klemp mentioned that the referenced paper didn't focus on OS data, but they are excited about the median OS received in STS.

Q: Following on from what's been discussed already, my understanding of your path forward in STS lung mets is that you're looking at, similar to Trabectedin, a PFS endpoint for approval. Is that correct?

A: Paul Waymack stated they would measure both PFS and OS in clinical trials, preferring PFS for quicker approval but seeing impressive OS data.

Q: On the AML side, you're in sort of your timelines. You're talking about an end of Phase 2, the first half of 24, and a potential pivotal start second half of the year. And as you were talking about, that is depending on single arm or otherwise in a randomized study, 100 to 300 patients, what did the costs look like for pivotal study?

A: Paul Waymack mentioned a Phase 2/3 pivotal study for AML would cost roughly $18 million, drug included, and external funding was likely for STS pivotal trials.