Next we'll hear from Moleculin Biotech. Moleculin Biotech is a Phase 3 clinical stage pharmaceutical company advancing the pipeline of therapeutic candidates, addressing hard-to-treat tumors and viruses. Now I'd like to introduce the founder, president, CEO, and chairman Walter Klemp. Walter, please go ahead and share your screen.
Thank you, Kevin.
Make sure we're.
Yep, we're sharing our screen. Good.
Thank you.
Well, hello everyone. I'm Wally Klemp, and I'd like to thank you for the opportunity to provide some insight into Moleculin Biotech. Since we are a publicly listed company, it's important to note that I will be making forward-looking statements as a part of the presentation. You can access all the latest information and our SEC filings on our website at moleculin.com. The Moleculin team is exceptionally qualified for what we're doing. The C-suite is responsible for a total of 7 FDA approvals, 2 Big Pharma exits, and we now have been responsible for 14 clinical trials just at Moleculin alone.
We've got well over 200 years of combined drug development experience and have recently added an ex-Roche executive as a strategic advisor to expand our capabilities in the area of outlicensing and partnering since we're now at a point as a company where such transactions we think are likely within the next 12 months. Perhaps one of the more telling signs of confidence on the part of Moleculin's management is the fact that we've collectively now invested roughly $1 million after tax of our own money in Moleculin stock. Although Moleculin is best known for Annamycin, we're actually a portfolio company. In fact, we have three distinctly different technologies. They were all discovered at MD Anderson Cancer Center, largest cancer research center in the world, and they've all shown activity in humans in Phase 2 settings.
The reality, though, is that we do have limited resources, and our lead technology, Annamycin, is now so close to a Phase 3 data inflection point that nearly all of our capital is being focused solely on Annamycin. Our other two technologies do continue to move forward with the help of NIH and other grant funding, primarily driven by investigator-initiated trials. So the rest of today's discussion will be focused just on Annamycin, and with good reason. I mean, Annamycin is positioned to make history as a truly next-generation anthracycline. Let me start by just giving some context here. Anthracyclines are one of the largest classes of cancer drugs, and they treat more cancers than any other. In fact, half of all cancers and 60% of all childhood cancers are treated with an anthracycline. The reason for that is simple: they work. Their key drawback is cardiotoxicity.
Once a patient reaches the FDA-mandated lifetime limit of 550 milligrams per square meter, there is a 65% chance of permanent heart damage. This is where Annamycin comes in. Annamycin was designed to be completely non-cardiotoxic, and in over 100 patients treated, even at doses well above that lifetime limit, we've seen no evidence of cardiotoxicity. Now, this is groundbreaking. Annamycin also avoids cross-resistance with existing anthracyclines and, in the case of acute leukemia, with other standard-of-care treatments. So this really changes everything. It means that we can increase dose levels, use Annamycin where existing anthracyclines have stopped working, and even for the first time ever consider using an anthracycline as a maintenance therapy. Annamycin is also more potent than existing anthracyclines in most tumor models and generally has fewer and less severe toxic side effects.
Perhaps just as important to those investing in Moleculin, it has composition and matter patent protection through 2040 and the potential to extend beyond that. In fact, we recently announced two additional patents adding to Annamycin's exclusivity along with its status as a new chemical entity with orphan drug and fast-track status. Now, as I mentioned, about half of all cancers are treated with an anthracycline, but we've chosen to focus the bulk of our effort on one indication, acute myeloid leukemia, to pursue our first new drug approval. We estimate that up to 60% of AML patients are treated with an anthracycline, and for those who cannot tolerate the side effects, another chemotherapy regimen using venetoclax can generate similar results if patients have sufficient time to respond. For those who respond adequately, it can lead them to being completely cured of their disease.
But if they don't, and most patients don't, they are left with unacceptable options. Even the targeted therapies that have been approved only work in about 20% of these second-line patients. This means that almost 60% of the entire AML patient population are desperate for a better option. We believe Annamycin is a part of that answer. And we have the Phase 2 clinical data to back this statement up. In our last Phase 2 trial, testing the combination of Annamycin and Ara-C, that's a common combination drug for the treatment of AML, we saw complete remission in 50% of second-line patients. That is significantly better than any second-line therapy ever approved for AML and more than double the performance of the average targeted therapy. What's more, the durability of those responses and the overall survival increase for responders was higher than anyone would expect for these patients.
These data were so good, in fact, that a number of experts thought we must have been cherry-picking patients. That was until they dug into the details and realized that these responses came from 7 different sites in 2 different countries. We saw similar responses regardless of prior therapy. Also, 89% of responders had one or more gene mutations that are generally considered negative prognostic indicators. These were very tough patients to treat. Oh, and 75% of our responders tested negative for measurable residual disease, and 44% went on to a curative bone marrow transplant. This kind of performance isn't just approvable. We think it's industry-changing. It's because of these data that a veritable who's who of AML thought leadership has now jumped onto the bandwagon about where the next big change in AML treatment will come from.
Before the Annamycin data, the focus was still on targeted therapies, even though those therapies have delivered relatively poor efficacy results. Now, though, they all agree that Annamycin could change how we treat AML in a major way. The FDA saw this opportunity as well, and they worked with us to design a very creative, adaptive trial built to support accelerated approval of Annamycin. And we call this the MIRACLE trial, and it has a Part A and a Part B. Part A is designed to optimize the dosing regimen for Annamycin using three arms. The two test arms treat patients with Ara-C plus Annamycin at either 190 or 230 milligrams per square meter, while the control arm treats patients with Ara-C alone, actually plus placebo, which, by the way, is one of the established standards of care. That is Ara-C monotherapy, a standard of care for second-line AML patients.
A single primary efficacy point, complete remission or CR, determines the success or failure roughly 35 days after a single cycle. So this trial will be relatively fast to execute. Once safety and efficacy are determined for Part A, we'll choose the optimum dose and reduce the trial to two arms in Part B. One of the most important near-term attributes of this trial from an investor point of view is that in order to assess safety and efficacy during Part A, there will be two unblindings of the data. The first will be after the treatment of 45 patients, and the second will be at the end of Part A, which will be between 75 and 90 patients, depending on the 45-patient results. This means investors should have visibility on our likelihood of success within the first half of this year.
This kind of early visibility is very rare in Phase 3 trials, and we believe it could represent a massive inflection point for the company's stock. Beyond that, if the completed Part A dataset is close to our Phase 2 performance, there's also an opportunity to request a Type A Meeting that could amend Part B to become an open-label single-arm safety study, allowing us to start a rolling NDA as early as next year. Now, we don't need the data to be that good in order to gain approval, but it could be a pathway to an even shorter approval process. So the MIRACLE trial is off to a great start. Our recruitment in Europe has been steady and climbing. And although U.S.
Sites have been slower to open, largely due to the requirement of a control arm in the trial design. The response rate in Europe has started to, frankly, excite the U.S. sites, and we're glad to say that a number of additional U.S. sites are now coming on board within the next month. While I can't talk specifics at this time, we expect to be providing a major update on recruitment and interim results very soon. With that said, we're still signaling that we expect to reach recruitment of the first 45 patients yet this quarter. We also recently announced the completion of our MB107 clinical trial of Annamycin to treat soft tissue sarcoma lung metastases. More detail on this is available on our website, and we were able to deliver overall survival of 13.5 months in patients who were very far gone.
We're talking average seventh line of treatment here. That actually compares favorably to standard-of-care and even experimental treatments in just second-line patients. So these results have now gotten independent investigators excited and proposing to run a grant-funded trial for possible approval of Annamycin in sarcoma. And that brings us back to one of the early points I made about anthracyclines. They're used in roughly half of all cancers, not just AML or sarcoma. If you just look at the indications for which our preclinical testing shows superiority of Annamycin over existing anthracyclines, we believe the revenue potential for Annamycin is 10 times greater than just AML alone. Now, with the 45-patient enrollment milestone within just the next few months, we think things are going to get very exciting this year. That should be followed by the completion of Part A in 2026 as well.
Now, I've made no secret of our intentions toward partnering with Big Pharma for the development of Annamycin. That's why we brought in Adriano Treve, an ex-high-ranking officer of Roche, as a strategic advisor. We are building towards something big here, which brings me to the most important message that I can leave listeners with. We believe Moleculin is positioned for a market cap breakout in the very near future. It's great that we have a diverse pipeline of technologies, and yes, I'm very proud of the deep management bench that we have at Moleculin. But what really sets things up here is that we have a potentially highly disruptive technology in Annamycin with a very strong patent position in a very promising safety and efficacy profile. What's more, we're now so clinically advanced that we're measuring our time to early Phase 3 data readout in mere months.
So all of this in a competitive space where far lesser assets are going for price tags in the $ billions. I challenge anyone listening to this today to find a Phase 3 asset with more near-term visibility and more upside potential than Annamycin. Well, thanks, everyone, for taking time to learn about Moleculin and Annamycin, and I'd be happy to take any questions in the time that we have left.
Thank you, Walter. Very exciting updates. Justin can help you with the Q&A section. You can ask your questions at the bottom of your screen.
Great. Thank you. Wally, our first question is, can you help us understand how meaningful the non-cardiotoxic profile of Annamycin could be in the real-world AML setting? What has feedback been from clinicians?
So that's a very interesting way to focus that question. And candidly, I would say many hematology oncologists that are treating AML patients will say, "I'm not initially all that worried about cardiotoxicity because these patients have a very short while to live, and I'm willing to risk their cardiac health, their long-term cardiac health, to possibly give them extra life here against their AML." Now, that said, a couple of things. One, first of all, the FDA won't allow treatment beyond 550 milligrams. And so there are situations we know there are several situations where the clinician would like to treat that patient a second, maybe a third, even a fourth cycle, but they can't. And they won't because they know at some point they're going to do so much cardiac damage that they could lose the patient just by treating them.
There are also patients who present with impaired cardiac function. In those cases, they're really relegated to the combination of, let's say, venetoclax and azacitidine. While treatment with venetoclax has encouraging results, it takes a long time to respond, and frankly, most people don't respond. So they're still left without an outcome there. Well, even a patient coming in with impaired cardiac function, all of a sudden, Annamycin is a much better choice because of its lack of cardiotoxicity. And look, once we're approved and there's a decent track record and safety profile, if you're a clinician and you've got a choice and you think you can get equal or better results with Annamycin and no cardiotoxicity, that starts to become an easy decision. So in the end, it will absolutely change things in AML.
I think it has more chance to change things for, let's say, pediatric AML. Now, that's a very rare disease. And frankly, second-line AML pediatric patients have virtually nothing available to them that's acceptable. And that's a situation where it would be a travesty to save their lives from the AML disease only to shorten their lifespan because of the cardiac damage. So that's a situation where the use of Annamycin becomes a no-brainer. And then when you start looking beyond AML in other indications where lifetime expectancy calls for it, then the cardio issue becomes a lot more black and white, so.
Great. Thank you. Our next question, with the upcoming unblinding readouts, what are you most focused on learning from those data, and what does success look like?
Success is pretty easy to define, in part because the primary endpoint is so clean and simple. It's complete remission after a single cycle of treatment. I think the best way to put that topic in perspective is to reflect on historical outcomes. So as it relates to Annamycin, our prior Phase 2 data suggests that we could expect as much as a 50% complete remission rate in this trial as well. Now, we don't have to get nearly all the way to 50%, but it's possible, certainly possible. Then when you look at the control arm, that's cytarabine alone, monotherapy. Now, because of the blinded trial nature, it's cytarabine plus a placebo, but it's reasonable to compare that arm to historical trials of cytarabine monotherapy. Those trials historically generate a CR rate of around 17%-18%. Now, those trials allowed multiple cycles of cytarabine.
We're only allowing one before the assessment of the primary endpoint. When we ask experienced hematology oncologists for their, by the way, there are no good published studies on the efficacy of a single cycle of cytarabine monotherapy. But when we ask experienced clinicians, "What do you expect from that arm?" they're saying maybe closer to 10%, certainly in the low teens, not the high teens. So back to your question, what are we looking for with this initial data? Well, remember, it's three arms. One is cytarabine alone. The other two are with Annamycin at two different dose levels. But let's just assume that the two Annamycin dose levels maybe are similar in performance. That's still a 2-to-1 ratio.
When you do the math and you say, "Well, two-thirds of the trial, let's say, delivers 50% and one-third of the trial delivers 15%," or pick a number, then all of a sudden, what you're looking for is probably a composite CR rate of around, let's say, 30% to get to those numbers. Now, there's potential for a lot of movement there. But the point being, the first thing we will be able to tell people in the near term is what the blinded data are. And specifically, we'll be able to say, "Here's the composite CR rate, the combined CR rate for the entire trial." Now, until we're unblinded, we won't know how that splits between the control arm and the two other arms. But we can infer from that overall CR rate that mathematically, we should be in the right zone, okay?
So that's kind of the thing one. Then when the data are unblinded, then seeing the exact CR rates for the 2 treatment arms versus the control arm, that's where the real rubber meets the road. That will be very we think that will be very indicative of the likelihood of clinical trial success. Now, you can't guarantee it, but it's very difficult once we get to 45 patients. And certainly, by the time we get to 90 patients, it would be very difficult to argue that that's not indicative of the trend we should expect to see for the balance of the trial. So now, we will be looking at other things. We'll be looking at genotyping, which are there genetic profiles that seem to respond better? We will be describing for people what their prior therapies were. Did we have a lot of patients?
For example, there are a lot in the AML community that are challenging us, saying, "Are you sure you're going to be effective against venetoclax failures?" That's people who either are relapsed or refractory from venetoclax plus azacitidine. And that's half the patient population, by the way. And they're very skeptical because guess what? The overall survival rate of a venetoclax failure is about 2.5 months, even with aggressive salvage therapy, 2.5 months. Well, remember, in our Phase 2 trial, we had several venetoclax failures who responded with a CR, and the overall survival there was high, was in the 10-month kind of range, completely beyond what they would expect. Well, we'll be able to hopefully see some of that information as well when we unblind these data.
Thank you, Wally. Definitely an exciting time for the company. That is all the time we have for Q&A.
Okay. Thank you, Wally.
Thank you, Justin. Thank you, Kevin.
Thank you. For more information on Moleculin Biotech, please visit their Corporate Connects page on the app. Lastly, we will hear from AIM ImmunoTech, an immunopharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including COVID-19. Now, I'd like to introduce the Chief Executive Officer, President, and Executive Vice Chairman, Thomas K. Equels.