Moleculin at Roth Conference: Annamycin’s Market Potential

On Tuesday, 24 March 2026, Moleculin Biotech (NASDAQ:MBRX) presented at the 38th Annual Roth Conference, highlighting the progress and strategic direction of its MIRACLE phase III trial for Annamycin. The company shared promising updates, though it acknowledged initial recruitment challenges. CEO Walter Klemp emphasized Annamycin’s potential in the AML market and its unique benefits over traditional treatments.

The MIRACLE phase III trial is structured in two parts, A and B, with part A involving three arms: a control using HiDAC and two different doses of Annamycin combined with HiDAC. Recruitment faced initial delays due to the CTIS system in the EU but has since accelerated, with 45 patients now enrolled. The unblinded data for these patients is expected in a few months, with the primary endpoint being the CR rate for Annamycin versus the control.

Annamycin is highlighted for its reduced cardiotoxicity, having shown zero cardiotoxicity in over 100 patients, even those exceeding the FDA’s lifetime limit for anthracycline exposure. Its unique chemical structure allows it to avoid cross-resistance with other AML treatments. Moleculin anticipates a significant market opportunity, projecting a $500 million to $1 billion global market for Annamycin in AML. Additionally, Annamycin’s organotropism offers potential in treating other cancers like soft tissue sarcoma, colorectal cancer, and pancreatic cancer.

CEO Walter Klemp expressed concerns about the company’s current market cap, which he believes is disconnected from reality given the promising phase II data. The valuation is reportedly 30 times lower than the lowest for targeted therapies. Upcoming milestones include the unblinding of 45-patient data, a potential filing for breakthrough therapy status, and the unblinding of 90-patient data by year-end.

There is significant interest in investigator-sponsored trials for Annamycin, though these are limited by drug supply, with priority given to the phase III trial. Indications of interest include sarcomas, colorectal cancer, renal cell carcinoma, and pancreatic cancer, with a clinical trial already underway for the latter.

For further insights, refer to the full transcript below.

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: Welcome back. This is Jonathan Aschoff, senior biotechnology analyst at Roth Capital Partners. Who we have now is Walter Klemp, the CEO of Moleculin Biotech.

Walter Klemp, CEO, Moleculin Biotech: Hey, Jonathan.

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: Um-

Walter Klemp, CEO, Moleculin Biotech: Thanks for having me.

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: You’re very welcome, Wally. You know, last year you were just beginning the MIRACLE phase III trial in relapsed refractory AML. Can you update us on the enrollment progress? There was certainly news about that this week, and what you are seeing as the trial advances toward interim unblinded data?

Walter Klemp, CEO, Moleculin Biotech: You bet. Maybe just to set the stage, this is a registration-enabling phase III trial that has two parts, part A and part B. Part A has three arms, with one being a control arm using high-dose cytarabine or HiDAC, which is a current standard of care, by the way, and the other two arms are two different doses of Annamycin combined with HiDAC. This approach addresses FDA’s Project Optimus guidelines, where they want us to establish an optimum dose before completing part B. The data from both parts are combined for approval purposes, so it’s a very efficient trial design. Our recruiting was a bit delayed out of the blocks as we navigated the new CTIS system in the E.U. Once we cleared that, our rate of recruitment has been steadily growing.

We just announced yesterday, as you said, that we have recruited our 45th patient in the MIRACLE trial. Now, look, it was just a month ago that we announced the blinded data from the first 30 patients. You can see that the rate of recruitment is picking up big time. Even though the results we announced last month were blinded, they appear to be supportive of what we hope to see when we unblind the 45-patient data. Namely, the historical studies suggest that our control arm should deliver a CR rate somewhere in the low to high teens, and our phase II data suggest we might see something in the 40%-50% range from Annamycin. Now, the blinded data we’ve seen so far seem consistent with those historical numbers, but of course, that’s only speculation.

Look, we’re only a few months away now from unblinding the data for our first 45 patients, so we don’t need to speculate for much longer.

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: Yeah, this is a very, very important catalyst, the most important one since I’ve been covering you for about six years. Can you explain how the somewhat unique MIRACLE trial design helps accelerate development while still generating the data that regulators are gonna want?

Walter Klemp, CEO, Moleculin Biotech: Well, several things are important about this study. First, we’re talking about AML, a rare disease with a significant unmet need, so we’re allowed to use a surrogate endpoint, specifically CR or complete remission. Since our drug is capable of achieving a CR with just one treatment cycle, we know the outcome for any given patient within roughly a month. Next, we have fast track status, which means we can submit a rolling NDA as the study is finishing up. The most unique aspect of the study is our ability to unblind in order to support the dose optimization that I mentioned earlier as part of FDA’s Project Optimus. As you know, Jonathan, it’s unusual to have such an early read in a phase III trial, so understandably, there’s a lot of anticipation here.

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: You know, although you can’t provide data right now, can you set investor expectations on what type of data you’ll be announcing with the 45 patient interim look?

Walter Klemp, CEO, Moleculin Biotech: Sure. To start with, we expect about 15 subjects will have been treated in the control arm, and 30 subjects will have been treated with Annamycin, with about 15 of those at 190 milligrams and 15 at 230 milligrams. The numbers are approximate because the randomization process is such that there could be some variation between the three arms. We expect to report CR rates for each arm and to provide overall numbers for median age and what percentage of the overall population came into the trial as what we call venetoclax regimen failures. Now, that last point is important because a lot of AML trials are explicitly excluding ven regimen failures because those patients are really hard to treat, as you know. In our case, we are actually welcoming these patients because our phase II data for these folks was surprisingly strong.

At the end of the day, though, it’s the primary endpoint that really matters. Specifically, it’s the CR rate for patients receiving Annamycin versus the CR rate for control. That’s what’s gonna translate into approval here.

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: Definitely. You know, you often describe your drug, Annamycin, or its generic name, annamycin, as a next generation anthracycline. What differentiates it from traditional anthracyclines, particularly in terms of the cardiotoxicity you’ve not shown and potential efficacy?

Walter Klemp, CEO, Moleculin Biotech: Yeah. Well, to start with, Annamycin is truly a new chemical entity. By the way, it’s got patent protection through 2040 and probably extendable into 2045. Now, although it’s technically within the class of anthracyclines, its structure and unique lipid-based delivery system are so different that it behaves differently than existing anthracyclines. Look, today’s anthracyclines are referred to as pleiotropic, which basically means that they’re dirty drugs. They hit a lot of targets that are unintended and undesirable. Most notably, they attack cardiomyocytes, which translates into cardiotoxicity. Even though half of all cancers are treated with an anthracycline, they are so toxic that if a patient is treated over the FDA’s lifetime limit, there’s a 65% chance of heart damage. Even with those limits in place, 60% of all childhood cancer survivors will develop cardiac dysfunction because of their treatment.

Annamycin is much more targeted than today’s anthracyclines, so if it doesn’t have the same effect, it doesn’t have the same effect on cardiomyocytes, and the result has been zero cardiotoxicity in over 100 patients treated to date, and most of those patients were taken over the FDA’s lifetime limit. It’s also just generally more tolerable, with almost no alopecia, and mucositis can now be completely avoided. Its structure is so different that it not only is not recognized by typical multidrug resistance mechanisms, it avoids cross-resistance with current anthracyclines, with venetoclax, and with cytarabine. Because of this and the fact that it’s more targeted, we’ve seen better efficacy in our last phase II trial than any drug ever approved for relapsed or refractory AML.

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: What would be the Mucositis rate for Anthracyclines as a class?

Walter Klemp, CEO, Moleculin Biotech: You know, typically, you would expect to see mucositis in the 20%+ range. It would be even worse if not prophylaxed for. There are prophylactic-

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: Right

Walter Klemp, CEO, Moleculin Biotech: treatments, primarily cryotherapy, but it’s pretty common to see, and it’s pretty difficult to deal with when it happens.

Jonathan Aschoff, Senior Biotechnology Analyst, Roth Capital Partners: Yeah. It sounds nasty. How do you view the current treatment landscape in relapsed/refractory AML, and where c