Moleculin Biotech, Inc. (MBRX) September 2026 Conference Transcript

Good afternoon, everyone, and thank you for joining the 2026 H.C. Wainwright 28th Annual Global Investment Conference. My name is Ahmed Mahmood, an Associate Research Analyst at H.C. Wainwright, and I'm happy today to introduce our presenter for this session, Walter Klimt, Chairman, President, and CEO of Moleculin Biotech, a clinical stage biopharmaceutical company developing therapies for hard-to-treat cancers. Walter?

Thank you, Ahmed. Before we start, a reminder that Moleculin is publicly listed, and I'll be making forward-looking statements. Our latest information and SEC filings are on our website at Moleculin.com. Let me start with the punchline because everything that follows supports this page. Anomycin is positioned to potentially become the first ever non-cardiotoxic anthracycline, now in a pivotal FDA-guided Phase III trial for relapsed or refractory AML. In June, we got our initial unblinded look at the first 45 subjects and the anomycin ARMS delivered a complete remission rate three times higher than control. And that was after just a single cycle of treatment. And that matters because almost every comparable trial allows multiple cycles, which yield better results, just like anomycin did in its phase two trial. On safety, zero cardiotoxicity detected across 90 independently reviewed patients, and anomycin is generally more tolerable than currently approved anthracyclines. We're roughly 80% recruited in Part A, and we see base case peak revenue of about $900 million in AML alone, and nearly $10 billion across the entire pipeline. That's the story. Now let me unpack it our c-suite brings over 200 years of combined drug development experience seven fda approvals and two big pharma exits and since our 2016 ipo we've supported 18 clinical trials including the pivotal trial we're running today we've also invested roughly two million dollars of our own after-tax money in animicin down to the vp level management now has over an 18 percent benefit beneficial interest for my part i exited my last startup to avvi for nearly 600 million then the largest pre-revenue valuation in its class dr don picker led the team that created and won approval for carboplatin and cisplatin the first metals-based drugs that the fda ever approved, both blockbusters, and then exited to Bristol-Myers Squibb. Dr. Paul Waymack, our senior CMO, is ex-FDA, and his own approvals include arsenic trioxide, now first-line treatment for acute promyelocytic leukemia. He also trained the FDA cardiology team leader that's now opining on our drug safety. And Adriano Treve, recently retired as head of Roche's Eastern European and Western Asian operation, he brings a big pharma perspective and expands our partner network. Moleculin is technically a portfolio company, three distinct technologies all discovered at MD Anderson, all with the potential to combine in future therapies. That said, we've focused our resources on anomycin. So WP 1066 and 1122 move at grant speed on investigator-initiated work at Northwestern, Emory, and UNC Chapel Hill. That's development that we get without spending our own capital. So today is all about anomycin. And the message here is that although our pivotal trial is in AML, Anomycin has broad potential indications, hence our activity in soft tissue sarcoma, pancreatic cancer, and pediatric AML. I firmly believe that very few companies are positioned for the market cap breakout that we are. Anomycin is a patent-protected disruptive technology, bringing superior safety and efficacy to a huge unmet need, with phase two efficacy better than any approved second-line AML therapy. And unlike a year ago, this isn't about what a phase three might show. We have data in hand with a second readout at the end of the year. This is a billion-dollar market where recent exits have shown strong revenue multiples, And where most AML drugs are limited to a narrow mutation-defined indication, anomycin's breadth represents 10 times the growth potential beyond AML alone. So let me set the stage for how big the AML opportunity is. The gold standard remains 7 plus 3. That's 7 infusions of cytarabine alongside 3 infusions of an anthracycline, usually donarulacin. Response rates are very high, usually within the first 21-day cycle, and durable responders typically go on to a curative bone marrow transplant. But this standard of care has some problems. First, it's highly toxic, so only about half of AML patients, usually those under 65, are considered fit enough to withstand it. Second, relapse rates are also very high. So little more than a third actually reached the potentially curative bone marrow transplant, and 20% of those transplants failed. So that leaves about 14 out of every 100 patients actually cured through 7 plus 3. Unfit patients get venetoclax plus a hypomethylating agent, usually azacitidine, hence venaza. outcomes are similar, but they take much longer. And venetoclax failures typically survive only about two and a half months. So most AML experts consider these some of the hardest cases in relapsed refractory AML. And that's why seven plus three is still preferred where you can use it, because if it fails, there's still time for something else. But that second attempt generally won't be more seven plus three. By then, the patient is likely resistant and cardiotoxicity comes into play. Then the targeted therapies. Well, the big promise for more than 10 years. Unfortunately, they just didn't deliver. Only about nine out of 100 patients reaches a durable outcome that way. Added up across every approved pathway, only about 38 of 100 AML patients reach a durable outcome. That's an unmet need of roughly 60%. In our trials, anomycin with citerabine, or anorak, produced complete remission in about half of second-line patients. So that's the sweet spot for anomycin. Now, to understand why anomycin is potentially disruptive, remember how important anthracyclines still are. They're the most critical part of 7 plus 3, and roughly half of all cancers and about 60% of pediatric cancer treatments still use an anthracycline today. But they're extremely toxic, so toxic that the FDA sets a lifetime maximum on anthracycline exposure. Go over that limit and there's a 65% chance of heart damage. Even within the limits, 60% of childhood cancer survivors develop cardiac dysfunction. As you saw, 50 to 60% of adult AML patients can't be given an anthracycline at all. And yet, Jazz Pharma paid a billion and a half for the last incremental improvement in AML-based anthracyclines, a drug that did nothing for cardiotoxicity and added three and a half months survival. The message, anthracyclines are critical to patient care, and there's tremendous room for a true disruptor. Anomycin's next-generation status is both a tremendous opportunity and it's a real challenge. Many academicians roll their eyes the moment we say anthracycline until they see how different anomycin is. This is an entirely new chemical entity with significant structural changes. Among them is an added iodine atom, which radically changes both the structure and the lipophilic nature of the active ingredient. When we then position that API inside a unique multilamellar lipid delivery system, which further enhances lipophilicity and supports the lack of cardiotoxicity already inherent in the molecule. It also helps explain anomycin's accelerated cellular uptake and its organotropic ability to target organs that current anthracyclines just can't reach. Now, this slide puts anomycin side by side with the doxorubicin class of anthracyclines. A few rows deserve specific attention. Let's start with half-life. Anomycin clears in one to two hours. Conventional anthracyclines linger for one to two days. Net charge at physiologic pH is neutral for us, cationic for them. We believe those two properties alone drive much of the safety and tolerability story. Additionally, we use a 5 plus 3 regimen, even further lessening the toxicity that patients have to endure. On cardiotoxicity, none detected across 90 independently reviewed patients at cumulative doses up to almost 3,000 milligrams per square meter compare doxorubicin where congestive heart failure is estimated at about five percent and at 400 milligrams and nearly 50 percent at 700 milligrams per square meter anamycin also avoids mdr1 resistance mechanisms it has no vesicant activity no tissue necrosis on extravasation and no central line required. Alopecia runs about seven percent versus 60 to 100 percent. Severe mucositis was zero of 22 patients in our phase two against 16 to 26 percent for intensive seven plus three. And our shelf life is five years versus about two. Row after row, this is not incremental. It's a game changer. Now, convincing key opinion leaders that there can actually be a better anthracycline, it's a real challenge. Every AML thought leader now recognizes, however, that targeted therapies have failed to deliver on their promise, and we need a better solution. But when they first hear the word anthracycline, they assume anomycin is old technology and there is outright disbelief that any anthracycline can truly be this safe. Every physician on this slide started from that disbelief. But once they saw the science and the clinical performance, every one of them became a strong supporter. Dr. Martinelli in Bologna calls it a potential game changer. Dr. Andre of FED MD Anderson calls the data striking and notes that it holds up even in venetoclax-treated patients. Dr. Tallman and Dr. Cherry make similar points about the unmet need. There used to be one universal caveat, though, and that's could the phase two numbers we generated, could they be replicated or did we just get lucky? As you'll see, we've now begun to answer that question. So let me summarize what we believe makes anomycin a disruptor. First, anomycin is positioned to become the first ever non-cardiotoxic anthracycline. And we've treated more than 100 patients, most dosed over the lifetime maximum, and some as high as five times that limit with zero cardiotoxicity. Those data have been independently reviewed by the Cleveland Clinic and by the FDA. That alone changes who you can treat. The old definition of unfit no longer applies. And for the first time, it opens the door to anthracycline maintenance therapy. Second, for all its safety improvements, anamycin appears to give up nothing in terms of efficacy. In fact, just the opposite. In our phase two combination trial, we saw 60% CRC and 50% CR, 13-month durability, an overall survival of 15 months. We were mutation agnostic with high response in venetoclax failures. Third, it avoids the resistance mechanisms that typically defeat current anthracyclines, and it lacks cross-resistance with them, with venetoclax, and with cytarabine. And fourth, the lipid delivery system supports rapid cellular uptake and organotropism, specifically targeting certain organs. And all of it is protected by composition of matter patents that run through 2040 with the potential to extend to 2045. We call the phase three study miracle designed in consultation with both FDA and EMA. Part A is a three-arm equal randomization of 90 patients comparing two doses of anomycin, 190 and 230 milligrams per square meter, each with high-dose citerabine against the control arm of citerabine plus placebo. The driver is FDA's Project Optimus, aimed at preventing sponsors from just defaulting to the maximum tolerable dose. And by the way, we had strong phase two results at both doses, so we're testing both. At the end of Part A, we select the optimum dose on efficacy, safety, and tolerability. We carry that into Part B. 222 more patients are randomized one-to-one against control, with 60 Part A patients pooling forward into that Part B. The primary endpoint is complete remission rate at about one month, importantly after just a single cycle. Now, we limited it to one cycle so that control arm patients could move to another standard of care if they weren't responding. Both citerabine alone and anorac did better in prior multi-cycle trials, and we fully expect anomycin, if approved, to be used across multiple cycles, especially for initial responders. So approval, though, turns on anomycin plus cytarabine outperforming cytarabine a low. And so far, we're beating control by 300% or more. That said, we expect the absolute CR and CRC numbers to run lower than they would with multiple cycles. And for investors, this design gives us two unblinded readouts comprising close to 20% of the total trial that roll into the final analysis. So here's how that plays out on a calendar. Our first unblinded readout on 45 patients came at the end of June. We expect Part A to be fully recruited at 90 patients in the third quarter and plan to file for breakthrough therapy designation in that same window. For reference, drugs carrying breakthrough status have an approval rate in the 70 percent range. Now the second unblinded readout at 90 patients comes in the fourth quarter of this year for the or the first quarter of next. We then select the optimum dose. Part B then runs about 18 months. Our fast track status allows a rolling NDA which we expect to begin in 2028 with the potential approval in 2030 this is a highly powered study we don't need to reach our phase two numbers for approval the miracle trial is a global adaptive phase three in adults with aml relapsed refractory after first line therapy stratified blinded, and randomized one-to-one-to-one. At the first interim, 17 patients in the control arm on high-dose citerabine plus placebo, 14 on anomycin 190, and 14 on anomycin 230. Primary endpoint for approval, complete remission rate. Secondary and exploratory endpoints include composite complete remission, durability, overall survival, transplant rate, MRD, and genotyping. And here are the results. The control arm, high-dose citerabine plus placebo, delivered 12% CR, that's 2 out of 17, and 29% CRC. Anomycin at 190 delivered 43% CR and 50% CRC. Anomycin at 230 delivered 36% and 57% respectively. Both anomycin arms outperformed control on the primary endpoint by 300% or more, and CRC was nearly double. And I want to underline this. After only one cycle, and recall prior trials showed that multiple cycles improved results on both arms. Significance was never expected at 45 patients, but the separation is exactly what we hoped for. Just as important, the Independent Data Monitoring Committee recommended that we continue Miracle without modification, so there were no safety signals warranting a protocol change. Now look at the population, because context matters. Over 75% of these patients are above age 60. More than half failed 7 plus 3 as first-line therapy, and another 31% failed a venetoclax HMA regimen. So this is not a cherry-picked population, and we're running it across 11 enrolling sites in seven countries. 18 months ago, we hadn't dosed our first patient, so remarkable speed for a pivotal trial. The other question was always whether the phase 2 numbers could be replicated. This slide begins to answer that. On the left, MB106, that's our phase 1B2 combination trial, 36% CR and 41% CRC across 22 patients from first through seventh line with multiple cycles allowed. In the middle, the Miracle Anomycin Arms pooled 39% CR and 54% CRC in 28 patients after just a single cycle. Combined, that's 50 patients on Anorak at 38% CR and 48% CRC. Now, that's two trials, different designs, different investigators, and the efficacy holds. That consistency gives us confidence heading into the 90-patient readout. One of the most exciting aspects of this trial is apparent efficacy we're having on patients who have failed first-line venetoclax regimens. The reason for this is that several published studies have confirmed that this is a very difficult to treat group, much more difficult than seven plus three failures. All 17 patients on this slide received venetoclax in first-line therapy. And in this group, we're seeing 29% CR and 47% CRC. To be precise, though, this cut is blinded. So those 17 include the placebo control arm. So the true anomycin number is almost certainly better than what you see here since a share of these patients received citerabine plus placebo. Either way, in a population where the field expects poor results, we're seeing meaningful responses. Now this slide reconciles our public numbers because we've released both blinded and unblinded figures and they behave differently. The blinded releases pool all three arms, including control, so they always understate what anomycin alone is doing. At 30 evaluable patients, we reported 30% CR, 40% CRC. At 62 evaluable, 24% and 37%. There's also a timing artifact. Failures declare themselves quickly, often within 21 days, while complete responses take 30 to 50 days to confirm. So any blinded snapshot is likely weighted toward failures that have already resolved. I'd also note that the proportion of venetoclax failures is nearly doubled since the 45 patient cut. A harder population, not an easier one. Bottom line, use the blinded data as a general indication of activity across all three arms, the unblinded 45 patient analysis shows what anomycin itself is doing. Let me address the competitive landscape also, because people assume that the shift to targeted therapy is a possible threat to anomycin. We think it's the opposite. Targeted agents like FLIT3, IDH, NPM1, menin inhibitors, they deliver only modest remission rates as a monotherapy. Their strongest results come when they're added to classic induction chemotherapy, and that pushes them toward first line. And layering targeted agent toxicity on top of cytotoxic chemotherapy in a triple combination just compounds the tolerability burden, which is exactly where anomycin fits. It's mutation agnostic. It avoids cross-resistance. It excels where venetoclax has failed. It's easier on patients, and it takes cardiotoxicity off the table. So the logical outcome in our view is twofold. Near term, anomycin should be the default salvage therapy for a high percentage of relapsed or refractory AML, regardless of mutation or targeted pathway, and especially for Venn HMA failures. Then, as the data mature, we believe anamycin becomes the preferred better tolerated cytotoxic partner to targeted therapies. It becomes the backbone of the first or second line triplet combination. Now, our numbers are only worth something if physicians will actually prescribe the drug. So this spring, we commissioned independent qualitative research, 19 oncologists, including KOLs, community hemonc, and pediatric oncologists. Only one of the 19 was highly satisfied with current second-line AML options. And they told us that targeted therapies are too narrow, the post-Venetoclax setting is a critical void, durability is the top unmet need, and the transplant gap exists and persists. They scored their likelihood to prescribe anomycin at 6 out of 7, praising the all-comer applicability and the lack of cardiotoxicity and the depth and durability of response. Now, one framing point from all of this work that's worth repeating, the backbone of the anthracycline made better. That's the story that physicians are responding to. Now, we ran the same exercise with payers, five insurers and five hospital administrators. Their concerns with the current standard of care aren't really about drug price, because total cost of care eclipses drug acquisition. Seven plus three drives inpatient stays, often not fully offset by DRG reimbursement. Coverage pathways are fragmented, and biomarker validation adds administrative friction. Anomycin addresses most of that. It's a five-day regimen shortening the hospital stay, has higher CR and MRD negative rates that get more patients to transplant faster, and that saves downstream cost. No cardiotoxicity means less cardiac monitoring and less follow-on cardiac care. So payers who gave us a number supported pricing between $27,000 and $35,000 per infusion, a modest premium over Vixios, with a ceiling set by the targeted agents. So what's all this worth? Well, let's start with AML. About 51,000 cases a year between U.S. and readily accessible ex-U.S. markets. Apply the 60% who become relapsed or refractory, add a 25% market share, four infusions per patient, a $30,000 U.S. price tag, so about half that ex-U.S. and you get over $600 million a year in second line peak revenue. Add first line, which expands the eligibility population by another 40%, and AML alone is roughly $900 million. But AML is just the first of nearly a dozen opportunities with compelling scientific data. And because we've eliminated cardiotoxicity and improved tolerability in general, we see for the first time the possibility of using an anthracycline as maintenance therapy. Anomycin's organotropism means potential against organ metastases and even primary tumors, pancreatic, hepatocellular, visceral mets in breast and colorectal, lung mets in renal cell and endometrial. Add those at conservative, single-digit market shares, and you get roughly $10 billion in peak revenue potential. That's the 10x I mentioned earlier in this presentation. So let me close where I started. We're at 75 of 90 patients enrolled in Part A with full recruitment expected in the third quarter, then dose selection, then Part B. and per protocol part a data from the selected dose and control arm carry forward into the pivotal analysis so none of this work is wasted with idmc clearance and a clear efficacy signal in both in both aml arms we believe miracle is proceeding toward a registration enabling readout now i began by saying molecular is positioned for a market cap breakout A year ago, that argument rested on phase two data and a promise. Today, it rests on a randomized, double-blind, placebo-controlled phase three that's separating from control on the primary approval endpoint in a hard population after just a single cycle. We think this is a multi-billion dollar asset and we are months not years from the next data point that further de-risks the opportunity. Thanks for your time.

Thank you Walter for that fantastic presentation. I'd also like to extend a thank you to all our presenters this year as well as everyone who took the time to watch this presentation.