Moleculin Biotech, Inc.

Greetings. Welcome to the Moleculin Biotech 2023 year-end conference call and webcast. At this time, all participants are in a listen-only mode. The question-and-answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. Please note that this conference is being recorded. I will now turn the conference over to Jeannine Thomas, Investor Relations. Thank you. You may begin.

Thank you, Daryl, and good morning and welcome, everyone. At this time, I would like to remind our listeners that remarks made during this webcast may state management's intentions, beliefs, expectations, or future projections. These are forward-looking statements and involve risks and uncertainties. Forward-looking statements on this call are made pursuant to the safe harbor provisions of the federal securities laws and are based on Moleculin's current expectations and actual results could differ materially. As a result, you should not place undue reliance on any forward-looking statements. Some of the factors that could cause actual results to differ materially from these contemplated by such forward-looking statements are discussed in the periodic reports and molecular files with the Securities and Exchange Commission. These documents are available in the investor section of the company's website and on the Securities and Exchange Commission's website. We encourage you to review these documents carefully. Additionally, certain information contained in this webcast relates to or is based on studies, publications, surveys, and other data obtained from third-party sources and the company's own estimates and research. While the company believes these third-party sources to be reliable as of the date of this presentation, it does not independently verify and makes no representation as to the adequacy, fairness, accuracy, or completeness of or that any independent source of verified any information obtained from third-party source. Any data discussed regarding clinical trials and progress are considered preliminary and subject to change. Joining us on the call from Moleculin's leadership team are Walter Klemp, Chairman and Chief Executive Officer, Dr. John Paul Waymax, Senior Chief Medical Officer, and Jonathan Foster, Executive Vice President and Chief Financial Officer. I'd now like to turn the call over to Walter Klemp, Chairman and CEO. Wally, please proceed.

Thanks, Deneen. And welcome everyone to our year end recap and pipeline update. We have some very exciting progress to talk about today. And because of that, we will primarily focus on our AML second line strategy and the significant opportunity we believe this represents. As we review the activities of 2023 and the data that we are just now announcing, It becomes increasingly clear that anamycin, anthracyclines, that is, remain perhaps the most critical component of treatment for AML and STS. For too long, though, the limitations of anthracyclines have prevented a majority of patients from benefiting. Anamycin has changed that, and for the first time ever, we're showing a viable pathway to enable that underserved majority to benefit from anthracyclines. It's for this reason that we won't be focusing on the rest of our development pipeline today, other than to say that preclinical work continues to position both our STAT3 inhibitors and our metabolic inhibitors for continued outside investment that only helps molecular investors. The primary focus of this call and the updates we expect to provide over the coming months are squarely on anamycin. and even more specifically, on the treatment of relapsed or refractory AML, and for good reason. A lot of small biotechs, frankly, would give anything to have the data we just announced. Anamycin was able to generate a 60% CRC rate in second-line patients, and that includes a 50% CR rate in combination with another 10% CRI. A conservative estimate says that anamycin should be able to more than double the number of patients achieving complete remission as compared with all of the approved targeted therapies combined. Now, this is possible in part because of anamycin's complete lack of cardiotoxicity. We've treated 82 subjects so far across multiple studies and have yet to see any indication of cardiotoxicity. What is important here is that all of this puts us on course to begin a pivotal registration study this year with the potential for securing an accelerated approval pathway from regulators. Look, this is a fundamentally different company than most of you on this call invested in, candidly even compared with just a few short months ago. We are now phase three ready. We have data that outperforms every asset approved in AML and in a space where lesser assets have sold for billions. And we have established what we believe is a pathway to approval. Animicin is a remarkable next-generation treatment. Its lack of cardiotoxicity combined with its greater potency and lack of cross-resistance with currently prescribed anthracyclines truly puts it in a class by itself. And while this is true for a wide range of potential indications, it's especially meaningful in AML. The AML treatment landscape is complicated, and it's easy to lose sight of just how big the unmet need remains. We estimate that currently in the US, almost 60% of AML patients remain without a viable treatment option that could cure their disease or provide lasting remission. And that's despite all of the recent advances in targeted therapies. While we don't have time to explore this in detail today, it's very important for investors to understand why and how significant the unmet need still is. This is so important, in fact, that we produced a short video taking you through this analysis. The link to that video is shown here, and it's also right on the landing page of our website. If you are currently an investor or considering investment in molecular, you owe it to yourself to watch this short six-minute video. And because the unmet need is so great, a lot of recent new drug approvals have been allowed on the basis of some pretty low performance numbers. Of the five targeted therapies that have been approved, they've done so on the basis of an average 21% CR rate. What we just announced is more than double that number. But the difference is even greater than that. The average 21% CR rate only applies to the few patients that happen to have the required targeted gene mutation, and that leaves 53% of AML patients out in the cold. In comparison, we've been treating all comers with anamycin, regardless of gene mutation or prior therapy. And this is where anamycin really shines compared with all of the currently approved second-line therapies. The orange bars on this chart show the CR rates documented for each existing The blue bars weight those CR rates for the actual percentage of AML patients that can benefit because they happen to have the required gene mutation. And when you add them all together, it reveals that only 13% of all second-line patients will achieve a CR as a result of current targeted therapies. But since anamycin is an all-comers drug, there's no reduction from weighting. Instead, 100% of the CR rate should be expected for all second line patients. And that means our 50% CR rate is multiple times greater than the expected benefit from all targeted therapies combined. And if you're thinking that the relatively low performance bar set by targeted therapies means they don't have much market value, well, think again. In 2021, Servier, a French mid-pharma company, paid $2 billion for the combination of Idifa and Tibsobo, two drugs that together are expected to produce complete responses in just 4% of the entire population of second-line AML patients. We are currently showing a performance from anamycin that is more than 10 times that number. And if you think that's an outlier, Consider that our performance numbers in second line are better than Venetoclax's numbers in first line patients, where you would expect higher performance. Yet Venetoclax generates $2 billion a year in revenue for AbbVie. And let's not forget that Jazz Pharma paid $1.5 billion for Vixios, where, again, we believe our performance numbers are much greater. Any way you look at it, We believe the efficacy numbers we are sharing with you today support an eventual exit for molecular shareholders that will be measured in the billions. And again, this is just AML. As we've said before, our preclinical data suggests anamycin should also be beneficial in a wide range of other indications, potentially addressing 10 times as many patients as both AML and STS combined. Now keep in mind our opening slide. Anthracyclines remain a cornerstone of chemotherapy in many of the worst cancers. And an anthracycline that has demonstrated a lack of cardiotoxicity could be a game changer for these cancers, especially in children where the current anthracyclines may cure their disease only to limit their lives because of the damage done to their hearts. With that as an overview, let me now hand the call over to Dr. Paul Waymack, our Senior Chief Medical Officer, to dive a bit deeper into the data. Paul?