Q1 2025 Moleculin Biotech Inc Earnings Call

Operator: Good morning. Welcome to the Moleculin Biotech first quarter 2025 update conference call webcast. The question and answer session will follow the formal presentation.

Good morning, and welcome to the molecular and biotech first quarter 2025 update conference call webcast.

A question and answer session will follow the formal presentation.

Operator: As a reminder, this conference is being recorded. It is now my pleasure to turn the call over to your host, Jenene Thomas, Investor Relations.

As a reminder, this conference is being recorded.

It's now my pleasure to turn the call over to your host Janine Thomas Investor Relations. Please go ahead your name.

Jenene Thomas: Please go ahead, Jenene. Thank you, Robin.

Speaker Change: Thank you Ravi and good morning, everyone. At this time I would like to remind our listeners that remarks made during this webcast may state managements intentions beliefs expectations or future projections. These are forward looking statements that 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 molecular and.

Jenene Thomas: Good morning, everyone. At this time, I would like to remind our listeners that remarks made during this webcast may state management's intentions be leased to 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 security 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 statement. 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 Moleculin files with the Securities and Exchange Commission.

Speaker Change: 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 those contemplated by such forward looking statements are discussed in the periodic reports molecular and files with the Securities and Exchange Commission. These documents are available.

Jenene Thomas: These documents are available in the investor's section of the company's website and on the Securities and Exchange Commission's website. We encourage you to review these documents carefully.

Speaker Change: On 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 back half relates to Earth based on studies publications, Jeremy and other data obtained from third party sources and the company's current estimates from research while the company believes.

Jenene Thomas: 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 verified any of the information obtained from a third-party source.

Speaker Change: These third party sources to be reliable.

Speaker Change: The date of this presentation. It does not independently verify it makes no representation as to the adequacy fair enough the accuracy or completeness that or that any independent source verified anything information obtained from a third party source any day to discuss regarding clinical trials in progress are considered preliminary and subject to change so joining us on today's call.

Jenene Thomas: Any data discussed regarding clinical trials in progress are considered preliminary and subject to change.

Walter Klemp: So, joining us on today's call for Moleculin's leadership team are Walter Klempp, Chairman and Chief Executive Officer, Dr. John Paul Weymouth, 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 Klempp. Wally, please proceed. Thanks, Janine. Hello, and welcome to our first quarter earnings conference call. For most of our investors, the focus is on the Phase III miracle trial studying antamycin for the treatment of relapsed and refractory acute myeloluchemia, and with good reason. This trial has now officially started with the first patient already treated and more on the way.

Speaker Change: Call for molecular and his leadership team are Walter Chairman and Chief Executive Officer, Dr. John Paul <unk>, Senior Chief Medical Officer, and Jonathan Foster Executive Vice President and Chief Financial Officer, I'd now like to turn the call over to work well.

Speaker Change: Well Walid. Please proceed.

Speaker Change: Thanks, Janeen, Hello, and welcome to our first quarter earnings Conference call.

Speaker Change: For most of our investors. The focus is on the phase III Miracle trial studying <unk> for the treatment of relapsed and refractory acute myeloid leukemia.

Speaker Change: And with good reason. This trial is now officially started with the first patients already treated and more on the way.

Walter Klemp: In total, we now have 38 sites selected worldwide between the U.S., Europe, Middle East, and North Africa. We just announced this week that we also received complete sign-off from the European Medicines Agency for all nine countries that we wanted to open in the EU. That was one of the longest lead time items for getting the EU up and running. So it was a major milestone and a real show of support from the EMA for our trial design and our objective. Also, the World Health Organization has officially recognized a new generic drug name for anamycin, which may now be referred to in literature as naxtorubicin.

Speaker Change: In total we now have 38 sites selected worldwide between the U S Europe Middle East and North Africa.

Speaker Change: We just announced this week that we also received complete sign off from the European Medicines Agency for all nine countries that we wanted to open in the EU.

Speaker Change: That was one of the longest lead time items forgetting the EU up and running so it was a major milestone and a real show of support from the EIA for our trial design and our objectives.

Speaker Change: Also the World Health organization has officially recognized a new generic drug name for <unk>.

Speaker Change: Which may now be referred to in literature as Knox to Ruben.

Walter Klemp: This was an important first step in actually launching Anamycin once it receives new drug approval. And, you know, compared with some of the unpronounceable, crazy drug names out there, we're really pleased with Maxdarubicin, especially since it sounds a lot like the next Rubicin, which really plays into the next generation positioning of Anamycin. You should expect to see us using both names together until such time as we establish an FDA-approved brand name, which is the next step in positioning the drug for launch. And we also announced some additional patent protection for Enemite. Even though we already had Composition of Matter Protection extending into at least 2040, these additional patents just continue to build a wider fence of protection around our core assets.

Speaker Change: This was an important first step and actually launching Ana <unk> once it receives new drug approval.

Speaker Change: Compared with some of the Unpronounceable Crazy drug names out there, we're really pleased with maxtor rubis, especially since it sounds a lot like the next Ruben, which really plays into the next generation positioning of animation.

Speaker Change: You should expect to see us using both names together until such time as we establish an F. D. A approved brand name, which is the next step in positioning the drug for launch.

Speaker Change: And we also announced some additional patent protection for animals.

Speaker Change: Even though we already had composition of matter protection extending into at least 2040. These additional patents.

Speaker Change: Continue to build a wider firm.

Speaker Change: Of protection around our core asset.

Walter Klemp: We should also be announcing a date for the presentation of the final data from our MD107 clinical trial using antamycin to treat advanced soft tissue sarcoma. Although the preliminary numbers were impressive, we think the final data are really going to turn some heads. So, we look for an announcement on that. So, please look for an announcement on this in the coming. Now, this graphic gives you a better perspective on the status of our site selection and approval process for the MIRACLE trial. Compared with the version from our last earnings call, we now have the great majority of countries and sites approved from a regulatory standpoint, and now we're just wrapping up hospital contracts and local ethics approvals.

Speaker Change: We should also be announcing a date for the presentation of the final data from our M. B 107 clinical trial using <unk> to treat advanced soft tissue sarcoma.

Speaker Change: Although the preliminary numbers were impressive.

Speaker Change: We think the final data.

Speaker Change: Are really going to turn some heads so we look for an announcement on there. So please look for an announcement on this in the coming weeks.

Speaker Change: Now this graphic gives you a better perspective on the status of our site selection and approval process for the Miracle trial.

Speaker Change: Compared with the version from our last earnings call. We now have the great majority of countries and sites approved from a regulatory standpoint, and now we're just wrapping up hospital contracts and local ethics approvals.

Walter Klemp: Just to be clear, though, the most important milestone for us coming up will be the unblinding of the first 45 patients. A lot of eyes will be on us for this data point, as it should tell the world definitively. that we are likely headed for new drug approval. With that said, what you see here is what gives us so much confidence that we expect to have those first 45 patients treated before the end of this year. To drive this home, just averaging one patient per site for the remainder of the year gets us there. And given the enthusiasm that we've seen from these sites, we think we're gonna outpace that rate.

Speaker Change: Just to be clear, though.

Speaker Change: The most important milestone for us coming up will be the unblinded of the first 45 patients.

Speaker Change: A lot of eyes will be on us for this data point as it should tell the world definitively that we are likely headed for new drug approval.

Speaker Change: With that said what you see here.

Speaker Change: Is what gives us so much confidence that we expect to have those first 45 patients treated before the end of this year.

Speaker Change: To drive this home just averaging one patient per site for the remainder of the year gets us there and given the enthusiasm that we've seen from these sites, we think we're going to outpace that rate.

Speaker Change: Okay.

Walter Klemp: Now look, we know a lot of investors think that Anamycin is the entire ballgame for us. And to be sure, it's where most of the attention should be focused because it's so close to NDA submission. But we do have two other very exciting technologies. And one of them is WP1066, our lead STAT3 inhibitor. Don't forget that WP1066 has already shown activity in the treatment of brain tumors. And that was with a very inefficient form of drug delivery. Well, now WP1066 is in another investigator-sponsored clinical trial, this time in combination with radiation at Northwestern University. And since we announced this trial in September of last year, we've already recruited seven patients.

Speaker Change: Now look we.

Speaker Change: We know a lot of investors think that <unk> is the entire ballgame for us and to be sure. It's where most of the attention should be focus because it's so close to NDA submission.

Speaker Change: But we do have two other very exciting technologies and one of them is W. P 10, 66, our lead statutory inhibitor.

Speaker Change: Don't forget that W. P. 10, 66 has already shown activity in the treatment of brain tumors and that was with a very inefficient form of drug delivery.

Speaker Change: I'll now WP 10, 66 is in another investigator sponsored clinical trial. This time in combination with radiation at northwestern University and.

Speaker Change: And since we announced this trial in September of last year, we've already recruited seven patients that's nearly a patient per month for just one site. So this is moving quickly.

Walter Klemp: That's nearly a patient per month for just one site. So this is moving quickly. Now, this is still with the existing oral delivery, which we know isn't optimal. And we're collaborating with Emory University on the development of an IV delivery for 1066, which we think could significantly improve its activity. So we'll keep you updated as more developments occur.

Speaker Change: Now this is still with the existing oral delivery, which we know isn't optimal.

Speaker Change: And we're collaborating with Emory University on the development of an IV delivery for 10, 66, which we think could significantly improve its activity. So we'll keep you updated as more developments occur here.

Walter Klemp: Well, that gives you a high level overview of recent events.

Speaker Change: Well that gives you a high level overview of recent events. So now let me hand things over to Dr. Paul way Mac, our senior Chief Medical Officer to give you a few more insights into our clinical activity Paul.

John Waymack: So now let me hand things over to Dr. Paul Waymack, our senior chief medical officer, to give you a few more insights into our clinical activity. Paul. Thanks, Wally. Before I speak about the MIRACLE trial, let's catch up on our MB-106 Phase 2 trial. which had essentially the same inclusion and exclusion criteria plus the same treatment regimen for patients as Miracle. Except we treated some first line plus some third line and beyond subjects in MD-106. Our first patient who did not receive a bone marrow transplant but did achieve a complete remission finally relapsed after over 600 days.

Paul: Thanks Wally.

Speaker Change: Before I speak about the Miracle trial, let Scott jump on RMB 106 phase II trial.

Scott: Which had essentially the same inclusion and exclusion criteria plus the same treatment regimen.

Speaker Change: Patients as miracle.

Speaker Change: Except we treated some first line plus some third line and beyond subjects.

Speaker Change: One of the six.

Speaker Change: Our first patient.

Who did not receive a bone marrow transplant, but did achieve a complete remission.

Speaker Change: Finally, relapsed after over 600 days.

John Waymack: While we are disappointed for the subject, it must be said that achieving an almost two-year complete remission for a 78-year-old following 17 cycles of a venetoclax regimen It's remarkable, given the recent literature, which documents the dismal median overall survival for venetoclax regimen failures, which have a median overall survival of less than three months, regardless of further treatment. Our durability for MD-106 is still developing as the final three subjects are maintaining their complete remission. You can see on this chart that receiving a bone marrow transplant definitely correlates with better durability. And if these three keep moving to the right.

While we are disappointed with the subject.

Speaker Change: It must be said that achieving an almost two year complete remission.

Speaker Change: 78 year old following 17 will need a class regimen.

Speaker Change: This remarkable given the recent literature, which documents the dismal median overall survival.

Speaker Change: We need a class regimen failure.

Speaker Change: You have a median overall survival of less.

Speaker Change: And three months, regardless of further treatments.

Speaker Change: Our durability for RMB 106 is still developing.

Speaker Change: As the final III subjects are maintaining their complete remission.

Speaker Change: You can see on this chart that receiving a bone marrow transplant definitely correlates with better durability.

Speaker Change: And if these three keep moving to the right.

John Waymack: The median durability for the trial should eventually be even with subject two's durability. That is roughly 400 days.

Speaker Change: The median durability for the trial should eventually be even with subject choose durability.

Speaker Change: That is roughly 400.

John Waymack: Now, moving on to the miracle trial. We will only treat initial refractory relapsed AML subtypes. That is, this is a second line therapy study. where anamycin in combination with cytarabine will be delivered on a 5 plus 3 days basis. compared to just citerabine plus placebo. As Wally mentioned, Recruitment and treatment of subjects is already underway in Ukraine, and we expect the initial readout of safety and efficacy data. on the first 45 subjects around the end of 2025. On May 12th, we announced that the European Medicines Agency, that is the EMA, They approved our clinical trial application to conduct their miracle trial in all nine countries we submitted to in the EU.

Speaker Change: Now moving on to the Miracle Toronto.

Speaker Change: We will only treat initial refractory relapsed AML subjects.

Speaker Change: That is just as a second line therapy study.

Speaker Change: We are analyzing in combination with cytarabine.

Speaker Change: We will be deliberate on a five plus three days basis.

Speaker Change: <unk>, two just cytarabine plus placebo.

Wally: As Wally mentioned.

Wally: Recruitment and treatment of subjects is already underway in Ukraine.

Wally: We expect the initial readout of <unk>.

Wally: Safety and efficacy data.

Wally: On the first 45 subjects around the end of 2025.

Wally: On May 12, we announced that the European Medicines agency the.

Wally: The EMEA.

Wally: They improved their clinical trial application to conduct a miracle trial in all nine countries, we submitted to the EU.

John Waymack: We received final reports of acceptable for Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain. These approvals were under the condition that we submit results of appropriate non-clinical GLP studies before initiating the phase three portion, that is the Part B of the MIRACLE study. Combined with individual country committees and ethics approval for these nine countries in the EU. allows us to proceed in rolling subjects in these countries.

Wally: We received final reports of acceptable for.

Wally: For Belgium.

Wally: France, Germany.

Wally: Italy, Lithuania, Poland, Romania, and Spain.

Wally: These approvals were under the condition that we submit result, the appropriate non clinical Gilles <unk> studies.

Wally: Before initiating the phase III portion of that is b.

Wally: Be well.

Wally: The Miracle study.

Combined with individual country committees and ethics approval for these nine countries in the EU.

Wally: Allows us to proceed enrolling subjects in these countries.

John Waymack: Also, in November 2024, we amended our existing USIND by submitting the miracle trial protocol, which allows for dosing of USAML patients above the lifetime maximum allowable dose for currently prescribed anthracyclines. since then. We received FDA feedback and guidance on that amendment. This feedback allowed a reduction in the number of subjects to be enrolled in Part B of the Phase III Pivotal Protocol to 222 patients. And obviously, any reduction in recruitment numbers helps to shorten the time to completion of the trial. FDA made a number of other requests related to our protocol. These requested changes focus mainly on safety and subject monitoring, clinical pharmacology, and inclusion-exclusion criteria.

Wally: Also.

Wally: In November 2024.

Wally: We amended our existing U S D.

Wally: By submitting the Miracle trial protocol.

Wally: Which allows for dosing of U S AML patients above.

Wally: The lifetime maximum allowable dose we're currently prescribed anthracycline.

Wally: Since then.

Wally: We received FDA feedback and guidance on amendments this feedback allow the reduction in the number of subjects to be enrolled in part b of the phase III pivotal protocol to 222 patients.

Wally: Obviously any reduction in recruitment numbers helps to shorten the time to completion of the trial.

Wally: <unk> made a number of other requests related to our protocol.

Wally: These requested changes focused mainly on safety and subject monitoring clinical pharmacology and inclusion exclusion criteria.

John Waymack: None of the requests resulted in significant alterations in the overall trial design or the dosing of analyzed. Our last response to FDA was submitted on April 18, 2025. And we have not yet received follow-up communications since that time. We are therefore now proceeding with this amended miracle trial protocol in the U.S. As is typical with large, pivotal phase three global clinical trials at their onset, There are minor differences between the U.S. and EU protocols due to FDA and EMA requests. However, we do not view these as a barrier to successfully completing the study, and we are now working to harmonize the protocols into a single global version.

Wally: None of the request resulted in significant alterations in the overall trial design or the dosing them analyzed.

Wally: Our last response to FDA was submitted on April 18 2025.

Wally: And we have not yet received follow up communications since that time.

Wally: We are therefore now proceeding with this amended Miracle trial protocol in the U S.

Wally: As is typical with large.

Wally: Pivotal phase III global clinical trials that they're onset.

Wally: There are minor differences between the U S and EU protocols due to FDA and EMA request. However.

Wally: However, we do not view these as a barrier to successfully completing the study.

Wally: And we are now working to harmonize the protocols into a single global version.

John Waymack: Regarding our newly initiated Phase 3 study, It was designed after an end-of-phase 1-2 meeting with FDA. It is to be a randomized study comparing the dosing regimen with which we had great success in our MD-106 study, that is anamycin plus high-dose cytarabine. with a control arm of placebo plus high-dose citarabine. We chose this design because the FDA encouraged it. And because two recent large, randomized clinical trials in refractory and relapsed AML patients, That is the MIROS and CLASSIC-1 clinical trials. used it, and both achieved approximately a 17.5% complete remission rate. among patients randomized to receive high-dose citerabine plus placebo.

Wally: Regarding our newly initiated phase III study.

Wally: It was designed after an end of phase <unk> meeting with FDA.

Wally: It has to be a randomized study comparing the dosing regimen with which we had great success in RMB one of those six study.

Wally: And that is <unk> plus high dose cytarabine.

Wally: With a control arm of placebo plus high dose cytarabine.

Wally: We chose this design because the FDA encouraged it.

Wally: And because.

Wally: Two recent large randomized clinical trials in refractory relapsed AML patients.

Wally: That is the mirrors and classic one clinical trials.

Wally: Used it and both achieved approximately <unk> <unk>.

Wally: 17, 5% complete remission rate.

Wally: Among patients randomized to receive high dose cytarabine plus.

Wally: Plus placebo.

John Waymack: To that end, we can expect we will need for our anamycin plus high-dose citerabine treatment arm to beat a 17.5% complete response rate to a statistically significant degree for our drug to be approved by FDA for market. But as a reminder, in our MD-106 study, this combination of antamycin plus high-dose citarabine achieved a 50% complete remission rate. The primary efficacy endpoint for this study will be the rate of complete remission of the leukemia at approximately day 35. During Part A of our study, we will have two different anamycin treatment arcs. a 190 milligram per meter square treatment arm, and a 230 milligram per meter square treatment arm.

Wally: To that end, we can expect we will need for our N a milestone plus high dose cytarabine treatment arm.

Wally: To beat a 17, 5% complete response rate.

Wally: Two a statistically significant degree.

Wally: For our drug to be approved by FDA for market.

But as a reminder.

Wally: Andy one of those six study.

Wally: This combination of <unk> plus high dose cytarabine achieved a 50% complete remission rate.

Wally: The primary efficacy endpoint for this study will be the rate of complete remission leukemia at approximately <unk> 35.

Wally: During part a of our study we will have two different analyzing treatment arms.

Wally: 190 milligram per meter square treatment arm.

Wally: 230 milligram per meter square treatment arm.

John Waymack: There will be an unblinding of the data in Part A after the first 45 patients have completed their efficacy analyses, and a second unblinding after between 75 and 90 patients have completed their efficacy analyses. These interim looks at the data are in part to determine which of the two antimicrobial dosing regimens will be taken to completion of the study. In Part B of the study, we will continue enrolling patients. But we will then randomize in a one-to-one ratio the placebo plus cytarabine treatment arm against whichever of the two dosing regimens of anamycin was found to be superior plus the cytarabine treatment arm.

Wally: There will be an unblinded the data in part a after the first 45 patients have completed their efficacy analysis.

Wally: And a second on blinding after between 75 and 90 patients have completed their efficacy analyses.

Wally: These interim looks at the data arent part to determine which of the two enemies and dosing regimens will be taken to completion of the study.

Wally: And part B of the study we will continue enrolling patients.

Wally: But we will then randomized in a one to one ratio the placebo plus <unk> plus cytarabine treatment arm against whichever of the two dosing regimens of <unk> was found to be superior plus cytarabine treatment arm.

Wally: Okay.

John Waymack: Finally, as Wally mentioned, we intend to release the final data readout on NV107, where we treated advanced soft tissue sarcomas. which had metastasized to the lung with anamycin as monotherapy. Most subjects were treated well above the lifetime maximum anthracycline dose. And yet, after reviewing all the cardiac safety data. Our expert noted that he saw no signs of study-related drug-induced cardiotoxicity. We are excited about the results of these trials.

Wally: Finally.

Speaker Change: As Wally mentioned, we intend to release the final data readout.

Speaker Change: And then B one of those seven where we treated advanced soft tissue sarcomas.

Speaker Change: Which had metastasized to the lung.

Speaker Change: With <unk> monotherapy.

Speaker Change: Most subjects were treated well above the lifetime maximum anthracycline dose.

Speaker Change: And yet after reviewing all of the cardiac safety data.

Speaker Change: Our expert and noted that he saw no signs of study related.

Speaker Change: Drug induced cardio toxicity.

Speaker Change: We are excited about the results of these trials obviously.

Jonathan Foster: Now I'll turn it over to John Foster, our Executive Vice President and CFO. John. Thanks, Paul. We ended the quarter with about $8 million with cash on hand that should run our operations into the third quarter of this year.

Jon Foster: Now I'll turn it over to Jon Foster.

Speaker Change: Our executive Vice President and CFO John.

Speaker Change: Thanks, Paul.

Speaker Change: We ended the quarter with about $8 million with cash on hand that should run our operations into the third quarter of this year.

Jonathan Foster: To get us well into the first quarter of 2026, we will need to raise approximately $15 million. Now, we expect this amount will get us beyond the initial 45-subject data readout, supporting our efficacy rates for the additional 30 subjects receiving anamycin or Nexter-Rubison, and also by then we should have a total of 75 to 90 subjects recruited. moving us closer to the second data readout and the first half of 2026. Our market cap is up to over $14 million with 14.1 million shares outstanding. Our trading volume is healthy with a three month trading volume of almost 6 million shares per day.

Speaker Change: To get us well into the first quarter of 2026, we will need to raise approximately $15 million.

Speaker Change: Now we expect this amount will get us beyond the initial 45 subject data readout supporting our efficacy rates with additional 30 subjects receiving animation on extra Richardson and also by then we should have a total of 75% to 90 subjects recruited.

Speaker Change: Moving us closer to the second data readout in the first half of 2026.

Speaker Change: Our market cap is October $14 million with $14 1 million shares outstanding.

Speaker Change: Our trading volume is healthy with a three month trading volume of almost 6 million shares per day.

Jonathan Foster: It is spiky. We had a healthy volume of about 2.4 million shares traded this past Monday with the EU news that Wally and Paul just mentioned. Now, we've been busy by looking at this chart. We've delivered on contracting sites, site selections, presenting presentations, medical posters on NV106, and also on our sponsored research at MD Anderson.

Speaker Change: Is spiky we.

Speaker Change: We had a healthy volume of about $2 4 million shares traded this past Monday with the EU news at poly that Wally and Paul just mentioned.

Speaker Change: Now we've been busy but looking at this chart, we have delivered on contracting sites site selections.

Speaker Change: Presenting presentations medical posters on <unk> six and also on our sponsored research at MD Anderson.

Jonathan Foster: Most importantly, it's what we announced Monday, the approval of the EU member Obtaining all of these approvals with just 17 employees supported by a great team of consultants on the timeline which we expected and we told the public is something of which we're very, very This sets the stage for announcing, outside of the EU and the US, additional country regulatory approvals, along with first by country hospital site initiation visits, which sets them up to be open to recruitment. We'll update you on recruitment on our path to get to the initial 45 subjects. 15 of the control arm and 30 with the two different doses of animal.

Most importantly, it's what we announced on Monday, the approval in the EU member countries.

Speaker Change: Painting all of these approvals. So just 17 employees supported by a great team of consultants on the timeline, which we expected and we told the public is something of which we're very very proud.

Speaker Change: This sets the stage for announcing outside of the EU and the U S. Additional country regulatory approvals along with first by country Hospital site initiation visits.

Speaker Change: <unk> set them up to be.

Speaker Change: Open to recruitment.

Speaker Change: We will update you on recruitment on our path to get to the initial 45 subjects.

Speaker Change: <unk> on the control arm and 30 with two different doses of animation.

Walter Klemp: Now, this safety and efficacy readout, in addition to the 1MD106 data, should provide the market enough data to support a substantial increase in our market cap.

Speaker Change: This safety and efficacy readout in addition to the one and the 106 data.

Speaker Change: <unk> provided the market enough data to support a substantial increase in our market cap.

Walter Klemp: Wally? Thanks, John. You know, from a big picture perspective, we are all intensely focused on pushing Moleculin's market cap into the range that it truly deserves. And we think the building blocks for that market cap breakout are well placed. Anamycin is a truly disruptive technology in a space where exit valuations are often measured in the billion. The fact is we are positioned to possibly become the first ever non-cardiotoxic anthracycline. And don't forget, anthracyclines are used to treat not just AML, but nearly half of all cancers and 60% of all childhood cancers. And Moleculin doesn't just avoid cross resistance with other anthracyclines and chemotherapies like Benidoclax, it actually outperforms current anthracyclines in most preclinical tumor models.

Speaker Change: Wally.

Wally: Thanks, John.

Speaker Change: From a big picture perspective, we are all intensely focused on pushing molecular market cap into the range that is truly deserves.

Speaker Change: And we think the building blocks for that market cap breakout.

Speaker Change: Well placed.

Speaker Change: <unk> is a truly disruptive technology in a space where exit valuations are often measured in the billions.

Speaker Change: The fact is we are positioned to possibly become the first ever non cardio toxic anthracycline and don't forget anthracycline or used to treat not just AML, but nearly half of all cancers and 60% of all childhood cancers.

Speaker Change: Yeah.

Speaker Change: And molecular doesn't just avoid cross resistance with other anthracycline and Chemotherapies like Benita clacks, It actually outperforms current anthracycline in most preclinical tumor models.

Walter Klemp: It's not just that we believe we have a better drug here, it's far more clinically advanced than we think the market is giving us credit for. are phase two efficacy data. better than any drug ever approved for second line AML therapy, and we should have phase three data to share publicly by the second half of this year. All of this is supported by a diverse pipeline of follow-on technologies. and it's managed by a truly veteran drug development team with multiple FDA approvals and big pharma exits to our name.

Speaker Change: It's not just that we believe we have a better drug here.

Speaker Change: It's far more clinically advanced then we think the market is giving us credit for.

Speaker Change: Our phase II efficacy data.

Speaker Change: <unk> then any drug ever approved for second line AML therapy, and we should have phase three data to share publicly by the second half of this year.

Speaker Change: All of this is supported by a diverse pipeline of follow on technologies.

Speaker Change: And it's managed by a truly veteran drug development team with multiple FDA approvals and big.

Speaker Change: <unk> pharma exits to our names.

Walter Klemp: The key milestones are coming quickly this year. So stay tuned for a wild ride at Molex.

Speaker Change: The key milestones are coming quickly this year.

Speaker Change: So stay tuned for a wild ride at molecular.

Operator: Operator, we're now ready to open up for Q&A. Thank you. We'll now be conducting a question and answer session. If you would like to ask a question at this time, you may press star 1 from your telephone keypad and a confirmation tone will indicate your line is in the question queue. Let me press star two if you'd like to withdraw your question from the queue. For participants that are using speaker equipment, it may be necessary to pick up your handset before pressing the star key. One moment please while we poll for questions. Thank you.

Speaker Change: Operator, we're now ready to open up for Q&A.

Speaker Change: Thank you, we'll now be conducting a question and answer session.

Speaker Change: I'd like to ask a question at this time you May press Star one from your telephone keypad and a confirmation tone will indicate your line is in the question queue.

Speaker Change: You May press star two if you'd like to withdraw your question from the queue.

Speaker Change: For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.

Speaker Change: One moment. Please we poll for questions. Thank you.

Speaker Change: Okay.

Jonathan Aschoff: The first question is from the line of Jonathan Aschoff with Roth Capital. Please receive your question. Thanks.

Speaker Change: Thank you. The first question is from the line of Jonathan Aschoff with Roth Capital. Please proceed with your questions.

Walter Klemp: Good morning and congrats on getting Miracle enrolling. I was curious, the statement about the results will be submitted as a substantial modification to the EMA, does that have any negative implications for the timeline of EU approval versus U.S. Well, we... We don't think so. The issue here is that EMA requested some additional GLP preclinical data that we know we can produce. It's just a matter of timing and budgeting. So the issue here will be how quickly we can complete that GLP testing before the EU countries are allowed to move on to Part B. That said, Jonathan, the timelines that we've built, we would continue to be recruiting in the US sites and the non-EU sites even during that interim period in theory.

Jonathan Aschoff: Good morning, and congrats on getting Miracle enrolling I was curious the statement about the results will be submitted as a substantial modification.

Jonathan Aschoff: <unk> does that have any negative implications for the timeline of EU approval versus U S.

Jonathan Aschoff: Yeah well.

Jonathan Aschoff: Sure.

Jonathan Aschoff: We don't think so.

Jonathan Aschoff: The issue here is M a.

Jonathan Aschoff: <unk> some additional.

Jonathan Aschoff: G L Pic preclinical data.

That we know we can produce.

Jonathan Aschoff: It's just a matter of timing and budgeting.

Jonathan Aschoff: So.

Jonathan Aschoff: The.

Jonathan Aschoff: The issue here will be how quickly we can complete that GOP testing.

Jonathan Aschoff: Before the EU countries are allowed to move on to part D.

Jonathan Aschoff: That said, Jonathan the timelines that we've built we would continue to be recruiting in the U S sites and the non EU sites, even during that interim period in theory. So the trial really doesn't have to slow down and it and we will.

Walter Klemp: So the trial really doesn't have to slow down. And we believe we will get to the requisite number of EU patients to satisfy EU approval requirements. So the answer is, in theory, it shouldn't change the timeline for approval for EMA. But we have to acknowledge that depending on the time it takes to get those GLP studies done, that it could, but that's not our expectation.

Jonathan Aschoff: We believe we will get to the requisite number of EU patients to satisfy EU.

Jonathan Aschoff: Approval requirements so.

Jonathan Aschoff: The answer is in theory, it shouldn't change the timeline for approval for EMEA, but we have to acknowledge that depending on the timing.

Jonathan Aschoff: To get those GOP studies done that it could but that's not our expectation.

Walter Klemp: Okay, you know, how close is Emory, you think, to getting an optimal formulation? Like, you know, in the bag by the end of this year, or is it something that could drag out a little? You know, the thing, every time you ask a scientist to give you a timeline for discovering something new, they'll give you a lecture on what it takes to discover something new, right? We're, we think we're beyond the having to discover something new stage and that we, where we are now is, is actually implementing a strategy for a new formulation that, that satisfies the needs for IV delivery.

Jonathan Aschoff: Okay.

Jonathan Aschoff: How close is Ann Marie you think to getting.

Jonathan Aschoff: From a formulation like.

Jonathan Aschoff: In the bag by the end of this year or is it something that could drag out a little.

Jonathan Aschoff: Yeah.

Jonathan Aschoff: Yeah.

Jonathan Aschoff: Every time you ask the scientists to to give you a timeline for discovering something new that they'll give you a lecture.

Jonathan Aschoff: What it takes to discover something new right.

Jonathan Aschoff: Where we think we're beyond the having to discover something new stage and that we where we are now is.

Jonathan Aschoff: Is actually implementing a strategy for a new formulation that that satisfies the needs for IV delivery. So we feel like we're now in blocking blocking and tackling and just getting the preclinical work done so that we can move this into clinic.

Walter Klemp: So we feel like we're now in blocking, blocking and tackling and just getting the preclinical work done so that we can move this into clinic. And I, I feel like you're, you're sort of estimate there for by the end of the year is, is a, is a decent target, but I do have to acknowledge. things can always, you know, you can always get speed bumps when you're developing a new formulation. So we don't want to over promise there. But I think I think you should expect a year something from us before the end of the year.

Jonathan Aschoff: And I.

Jonathan Aschoff: I feel like your your sort of estimate therefore by the end of the year is it a decent target, but I do have to acknowledge.

Jonathan Aschoff: Things can always you can always get speed bumps when you're developing a new formulation. So we don't want to over promise there, but I think I think you should expect to hear something from us before the end of the year.

unknown: Okay, that's very fair.

Jonathan Aschoff: Okay.

Jonathan Foster: One for John, please. Is the is that three and a half million sort of a fair run rate for R&D for the rest of the year's quarters? Or is that I mean, that should go up, shouldn't it? Yeah, it'll go up especially as we head into 2026 when we bring on the GLP and some manufacturing. Right now we have enough drug for Part A.

Speaker Change: Very fair one for John Please is the is that three and a half million sort of a fair run rate for R&D.

Jonathan Aschoff: The year's quarters or is that I mean that should go up shouldn't it.

Jonathan Aschoff: It will go up, especially as we head into 2026, when we bring on the DLP and some manufacturing expenses.

Jonathan Aschoff: Right now we have one drug for part right now we have enough drug for part a west start manufacturing drug for pardon me.

unknown: We have to start manufacturing drug for Part Great, thank you very much, guys.

Jonathan Aschoff: Great. Thank you very much guys.

unknown: Thank you.

Jonathan Aschoff: Thank you.

Jason Mccarthy: The next question is in the line of Jason McCarthy with Maxim Group. Please review with your questions. Hey, guys. Thank you for taking the questions. Good morning. You mentioned the primary is the 35-day CRA, call it one month, just for simplicity. Do you need any durability data for potential approval, or is one month enough? And what are really the expectations for cytarabine alone, and if CRs are achieved with cytarabine, how long do they typically last? I'm trying to get a sense of... You know, what the bar is going to be here when you get to that unblinded interim data and say, like, you know, this looks like this should carry through all the way to the end for an approvable drug.

Speaker Change: The next question is from the line of Jason Mccarthy with Maxim Group. Please proceed with your questions.

Jason McCarty: Hey, guys. Thank you for taking the questions good morning.

Speaker Change: You didn't mention the prime areas of the 35 day CRA at call. It one month just for simplicity do you need any durability data for potential approval or is one month enough.

Jason McCarty: And.

Jason McCarty: What is really the expectations for cytarabine alone.

Jason McCarty: And then alright.

Jason McCarty: <unk> with cytarabine, how long do they typically last I'm trying to get a sense of.

Jason McCarty: You know what what the bar is going to be here when you get to that unblinded interim data and say like you.

Jason McCarty: This looks like this should carry through all the way to the end and for an approvable drug.

Walter Klemp: Well, I'm going to I'm going to ask Paul to just sort of give you a more thorough answer, Jason. But but the good news, just to be really clear, the good news is, durability is not a primary endpoint for approval here. It's a secondary objective. So our approval doesn't depend on that. But it's still an interesting topic. And I think it's important to talk about.

Speaker Change: Well I'm going to I'm going to ask Paul to just sort of give you a more thorough answer Jason but the good news just to be really clear. The good news is durability is not a primary endpoint for approval here, it's a secondary.

Speaker Change: <unk>. So are our approval doesn't depend on that but it's still an interesting topic and I think it's important to talk about so Paul do you want to maybe give a more thorough response to Jason here.

John Waymack: So Paul, do you want to maybe give a more thorough response to Jason here? That's a good question. As Wally said, FDA said that's their primary endpoint and These studies I quoted that had the 17 to 18% CR rate with high-dose cytarabine, these were not small studies. These were studies where the control arm of cytarabine plus placebo had hundreds of patients. So we're therefore quite confident that in our trial, the CR rate with cytarabine is gonna be in the teens. It will, as far as durability, it'll last for a few months, but that's a secondary endpoint.

Paul: That's a good question.

Paul: While he said FDA just said, that's our primary endpoint and <unk>.

Paul: These studies I quoted that had the 17% to 18% CR rate with high dose Cytarabine. These were not small studies. These were studies, where the control arm of cytarabine plus placebo had hundreds of patients.

Paul: So we are therefore quite confident.

Paul: In our trial.

Paul: CR rate with cytarabine is going to be in the teens it will as far as durability to last for a few months, but that's a secondary endpoint.

John Waymack: Of course, the ultimate arbitrator is FDA, but at our end of Phase 1-2 meeting, they said that's our end point, and they understand that we are not powered to show statistical significance for, we're not reasonably powered to show statistical significance for these other ones. There should be a strong trend. This is because that's what the FDA asked for, and when you look at the recent approvals for AML drugs, The secondaries were overall survival durability. Those were trends. They never reached statistical significance for these drugs, so we think we're in good shape there. And certainly, if we are anywhere near the 50% CR rate, we're in great shape.

Paul: Of course, the ultimate arbitrator is FTA, but at our end of phase two meeting they said, that's our endpoint and they understand that we're not powered to show statistical significance for.

Paul: Reasonably powered to show statistical significance. These other means there should be a strong trend.

Paul: This is because that's what the FDA asked for and when you look at the recent approvals for AML drugs.

Paul: <unk> rate was the primary end point the secondary is where overall survival durability those were trends. They never reached statistical significance for these drugs. So we think we're in good shape, there and certainly if we are anywhere near.

Paul: The 50% CR rate, we're in great shape.

Walter Klemp: There's a nuance here too, Jason, that I think is important to feed in, and that is the prior studies that Paul mentioned. They're two, two specifically, the MIROS trial and the CLASSIC-1 trial. Both of those studies allowed multiple cycles of cytarabine before measuring for CR. So that's a difference between their trial designs and ours. In our trial, you only get one cycle, you only get that roughly 35 days, and that's when we measure. So if the patient can't get a CR off of a single cycle of citerabine plus placebo, then that counts as not a CR.

Jason McCarty: There's a nuance here too Jason that I think is important to two.

Paul: Seed in and.

Jason McCarty: And that is the.

Jason McCarty: Prior studies that Paul mentioned.

Jason McCarty: Choose to specifically the mirrorless try them trial and the classic one trials both of those studies.

Jason McCarty: Allowed multiple cycles of cytarabine.

Jason McCarty: Before measuring for CR so.

Jason McCarty: So that's that's the difference between that their trial designs and hours in our trial.

Jason McCarty: Only get one cycle only get that roughly 35 days and that's when we measure.

Jason McCarty: If the patient can't get a CR off of a single cycle of Cytarabine plus placebo then they are out there.

Jason McCarty: There that counts is not a CR.

Walter Klemp: So. what partly why why Paul said we expect it to be in the teens. What he didn't say was... Our instincts say it's probably going to be lower than that 17.5% because those patients were allowed more than one cycle of citerabine. In ours, they won't get more than one cycle. So logic says we should expect that number to be lower. Now, we're not statistically planning on that, but it's what we expect.

Jason McCarty: No.

Speaker Change: Well, partly why why Paul said, we expect it to be in the teens.

Speaker Change: What he didn't say was our instincts say, it's probably going to be lower than that 17, 5% because those patients were allowed more than one cycle of cytarabine and ours, they won't get more than one cycle. So logic says.

Speaker Change: Should expect that number to be lower now were not statistically planning on that but it's what we expect.

Walter Klemp: And I'd imagine that some of your results, or could they be impacted by. Underlying factors like age and mutational and genomic alterations are those things that will be considered when you announce the data Well, for sure, we will, you know, we'll stratify by age and and by genetic mutations. But our primary endpoint isn't dependent upon it. And interestingly, when you look at the phase two data that we that we've talked about, that Paul's talked about, We're pretty agnostic to what genetic mutations are and to what prior therapies were. Our view is, you know, we're glad for all comers.

Speaker Change: And I'd imagine that some of your results or did they would be impacted by.

Speaker Change: Underlying factors like age and mutational genomic alterations to those things that will be considered when you announce the data.

Speaker Change: Well for sure we will.

Speaker Change: We will stratify by age and and a buy.

Speaker Change: Kinetic mutations.

Speaker Change: But our primary endpoint isn't dependent upon it and interestingly when you look at the phase II data that we've talked about that Paul has talked about.

Speaker Change: We're pretty agnostic to what genetic mutations are and and two what prior therapies were so.

Speaker Change: Yes.

Speaker Change: Our view is you know.

Speaker Change: We're glad for all comers, it's a funny just an anecdotal stories.

Walter Klemp: It's a funny, just an anecdotal story. When Paul and I presented this protocol to a number of practitioners, a common theme from the practitioner was, well, we assume you're going to exclude venetoclax failures because they're so difficult to treat. And our answer is no, absolutely not. Bring them on. We are not excluding venetoclax failures. In fact, we're hoping for them because the results are so dismal for those patients. And our, you know, our phase two data says we deliver essentially the same CR rate for venetoclax failures as we do for anybody else. So we are not sensitive to those those differentiations in terms of because, you know, obviously, fitness for intensive chemotherapy is largely determined by age.

Speaker Change: When Paul Hi, presented this protocol to a number of.

Speaker Change: Practitioners.

Speaker Change: A common theme from the practitioner was what we assume you're going to exclude the need of clocks failures because they are so difficult to treat and our answer is no absolutely not bring them on.

Speaker Change: We are not excluding the need to cause failures in fact, we're hoping for them because the results are so dismal for those patients and.

Speaker Change: R.

Speaker Change: Our phase II data says we deliver.

Speaker Change: Essentially the same CR rate for vanilla clocks failures as we do for anybody else. So we are we are not sensitive to those those differentiations in terms of because you know obviously fitness for intensive chemotherapy is largely determined by age. So there's also the assumption.

Walter Klemp: So there's also the assumption that we are adverse to elderly patients. And that's just not the case. Our median age in the phase two data set was in the 60s. And so we're happy to have elderly patients and we're happy to have mutational abnormalities and venetoclax failures.

Speaker Change: We are adverse to elderly patients and that's just not the case with our median age in the phase II data set was in the sixties and so we're happy to have elderly patients and we're happy to have mutation or lab abnormalities and.

Speaker Change: They need a class failures.

unknown: Thank you, Wally. Thank you.

Wally: Thank you Wally.

Speaker Change: Thank you.

Speaker Change: Thank you.

Operator: This will conclude today's question and answer session and will also conclude today's call. Thank you for your participation. You may now disconnect your lines at this time and have a wonderful day.

Speaker Change: This will conclude todays question and answer session and also conclude today's call. Thank you for your participation. You may now disconnect. Your lines at this time and have a wonderful day.

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