Core Viewpoint - Moleculin Biotech, Inc. reported positive topline efficacy results from its Phase 1B/2 clinical trial of Annamycin for treating soft tissue sarcoma lung metastases, indicating significant potential for this treatment option in a challenging cancer market [1][3]. Company Summary - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viruses, with Annamycin as its lead program [13]. - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for both relapsed or refractory acute myeloid leukemia and soft tissue sarcoma [5]. - The company is also advancing other therapeutic candidates, including WP1066 and WP1122, targeting various cancers and pathogenic viruses [16]. Clinical Trial Results - The MB-107 trial demonstrated a Clinical Benefit Rate (CBR) of 59.4%, with 18 subjects showing stable disease and 1 subject achieving a partial response [6]. - Median Progression Free Survival (PFS) was approximately 4 months, and Overall Survival (OS) was around 20 months for optimized dose subjects [6]. - In Phase 2, subjects with fewer prior therapies (≤2) showed improved OS of 19.9 months and PFS of 127 days, indicating better outcomes with Annamycin [6]. Market Opportunity - The soft tissue sarcoma market is projected to grow to $2.6 billion by 2030, with approximately 13,500 new cases expected annually [1][10]. - The current market for soft tissue sarcoma was valued at $1.58 billion in 2024, with a compound annual growth rate (CAGR) of 8.43% anticipated [10]. Study Design - The MB-107 trial was a multi-center, open-label, single-arm study assessing the safety and efficacy of Annamycin as a monotherapy for STS lung metastases [2][11]. - Treatment involved intravenous infusion of Annamycin over 2 hours, followed by a 20-day rest period, with safety monitoring conducted at each cycle [11][12].

Financial Data and Key Metrics Changes - The company ended the quarter with approximately $8 million in cash, which is expected to sustain operations into the third quarter of 2025 [20] - A need to raise approximately $15 million to support operations into the first quarter of 2026 was highlighted [21] - The market capitalization has increased to over $14 million with 14.1 million shares outstanding [22] Business Line Data and Key Metrics Changes - The Phase III MIRACLE trial for Anamycin has officially started, with the first patient treated and 38 sites selected worldwide [5] - The company received complete sign-off from the European Medicines Agency (EMA) for all nine countries intended for the trial in the EU, marking a significant milestone [6] - The company is also working on WP1066, a lead STAT3 inhibitor, which is currently in an investigator-sponsored clinical trial [10] Market Data and Key Metrics Changes - The trading volume has been healthy, with a three-month average of nearly 6 million shares traded per day [22] - A spike in trading volume was noted with approximately 2.4 million shares traded following the EU news [22] Company Strategy and Development Direction - The company aims to position Anamycin as a potentially non-cardiotoxic anthracycline, which could disrupt the market for cancer treatments [24] - The focus is on achieving a complete remission rate of at least 17.5% in the MIRACLE trial to meet FDA approval requirements [18] - The company is also looking to harmonize US and EU trial protocols to streamline the study process [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming data readouts and the potential for significant market cap increases based on trial results [23] - The company believes it is well-positioned to achieve FDA approval based on its Phase II efficacy data, which outperforms existing treatments [25] - Management acknowledged the challenges of developing new formulations but remains optimistic about achieving targets by the end of the year [32] Other Important Information - The company announced additional patent protection extending into at least February 2040, enhancing the protection around its core asset [7] - The final data from the MD-107 clinical trial using Anamycin to treat advanced soft tissue sarcoma is expected to be announced soon [8] Q&A Session Summary Question: Does the statement about results being submitted as a substantial modification to the EMA have negative implications for the timeline of EU approval versus US? - Management does not expect significant delays, as the EMA requested additional GLP preclinical data, which can be produced in a timely manner [30] Question: How close is Emory to getting an optimum formulation for IV delivery? - Management believes they are in the implementation stage for a new formulation and expects to have updates by the end of the year [32] Question: Is the $3.5 million a fair run rate for R&D for the rest of the year? - Management indicated that R&D expenses will increase, especially as they head into 2026 with GLP and manufacturing expenses [34]

Moleculin Biotech (MBRX) Q1 2025 Earnings Call May 14, 2025 08:30 AM ET Speaker0 Good morning. Welcome to the Molecular and Biotech first quarter twenty twenty five update conference call webcast. Question and answer session will follow the formal presentation. As a reminder, this conference is being recorded. It is now my pleasure to turn the call over to your host, Janine Thomas, investor relations. Please go ahead, Janine. Speaker1 Thank you, Rob, and good morning, everyone. At this time, I would like to ...

Exhibit 99.1 Moleculin Reports First Quarter 2025 Financial Results and Provides Corporate Update Enrollment and dosing underway in Phase 3 clinical trial (the "MIRACLE" trial) evaluating Annamycin (naxtarubicin) for the treatment of R/R AML Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) The Company is currently evaluating Annamycin (naxtarubicin) in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") in a Phase 3 pi ...

Core Insights - The company is advancing its Phase 3 clinical trial, known as the "MIRACLE" trial, evaluating Annamycin (naxtarubicin) for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML) with interim data readout expected in the second half of 2025 [1][11] - The European Medicines Agency (EMA) has granted approval for the trial in nine additional countries, enhancing the company's operational scope [1][6] - The company reported a decrease in research and development expenses for Q1 2025 compared to the same period in 2024, indicating a focus on clinical trial activities [18] Clinical Development Update - The MIRACLE trial is a pivotal, adaptive Phase 3 study evaluating Annamycin in combination with Cytarabine for R/R AML patients, with 38 sites selected globally, including 5 in the US as of April 2025 [4][23] - The trial design allows for unblinding of preliminary efficacy data at 45 subjects, with the first unblinding expected in the second half of 2025 [8][11] - The company is also seeing advancements in its pipeline, including the MB-107 trial for soft tissue sarcoma lung metastases, with final data readouts expected by the end of June 2025 [3][13] Financial Results - For Q1 2025, the company reported research and development expenses of $3.4 million, down from $4.3 million in Q1 2024, reflecting reduced clinical trial activity levels [18] - General and administrative expenses increased slightly to $2.5 million in Q1 2025 from $2.4 million in Q1 2024, primarily due to higher regulatory and legal fees [19] - As of March 31, 2025, the company had cash and cash equivalents of $7.7 million, which is expected to fund operations into Q3 2025 [19] Regulatory and Intellectual Property Developments - The company received EMA approval for its Clinical Trial Application to conduct the MIRACLE trial in all nine EU countries submitted [6] - Two new U.S. patents were granted, enhancing the intellectual property portfolio for Annamycin [6] - Annamycin has received Fast Track Status and Orphan Drug Designation from both the FDA and EMA for the treatment of R/R AML and soft tissue sarcoma [14]

Clinical Trials and Development - The company is conducting a pivotal Phase 3 trial for Annamycin in combination with Cytarabine for relapsed/refractory acute myeloid leukemia (AML), with interim unblinding expected by the end of 2025[74]. - Annamycin has shown no evidence of cardiotoxicity in 84 subjects treated across five clinical trials, with some subjects receiving doses significantly above the FDA's lifetime maximum anthracycline limit of 550 mg/m²[81]. - The MIRACLE trial has received approval from the European Medicines Agency (EMA) to expand into nine EU countries, contingent on presenting results of appropriate nonclinical GLP studies before initiating Part B[91]. - The FDA has allowed a reduction in the size of Part B of the Phase 3 trial protocol to 222 subjects, which is approximately a 10% decrease from the initial proposal[89]. - The company has completed a Phase 1B/2 clinical trial for Annamycin treating Acute Myeloid Leukemia (AML) and is currently in follow-up[85]. - The company has selected 38 sites for the MIRACLE trial across the US, Europe, and the Middle East/Africa, with one site in Ukraine actively treating subjects[88]. - Annamycin is designed to avoid multidrug resistance and has shown no cardiotoxicity in clinical trials, which could disrupt the competitive landscape for AML and Advanced STS treatments[76]. - The company has multiple active INDs/CTAs, comprising a total of fourteen clinical trials, both internally and externally funded[85]. - The company plans to host a Key Opinion Leader (KOL) call to announce and review final top-line data from the MB-107 trial in May or early June[92]. - The company is focusing on the development of Annamycin due to its potential to benefit a majority of patients with AML and Advanced STS[76]. Financial Performance - Revenues for the three months ended March 31, 2025, were $0, consistent with the same period in 2024[105]. - Research and development expenses decreased to $3.4 million in Q1 2025 from $4.3 million in Q1 2024, a reduction of approximately 21%[106]. - General and administrative expenses increased slightly to $2.5 million in Q1 2025 from $2.4 million in Q1 2024, reflecting a 4% increase[107]. - The net loss for the three months ended March 31, 2025, was $6.4 million, compared to a net loss of $5.0 million in the same period of 2024, representing a 29% increase in losses[105]. - The company recorded a net gain of $9.1 million from the change in fair value of warrant liability in Q1 2025, compared to a gain of $1.5 million in Q1 2024[108]. - Cash used in operating activities decreased to $4.6 million in Q1 2025 from $6.7 million in Q1 2024, a decrease of approximately 31%[112]. - The company raised gross proceeds of $9.3 million in February 2025 through various securities offerings[114]. - As of March 31, 2025, the company had $0.6 million in cash on hand and projected sufficient funds to support operations into Q3 2025[116]. - The company anticipates needing approximately $15 million in additional funding to support operations into Q1 2026[116]. Regulatory and Naming Approvals - The World Health Organization approved "naxtarubicin" as the International Non-Proprietary Name for Annamycin, facilitating its recognition in clinical and public documents[99].

Core Viewpoint - Moleculin Biotech, Inc. has received approval from the European Medicines Agency (EMA) for its pivotal Phase 2B/3 clinical trial of Annamycin in combination with cytarabine for treating relapsed or refractory acute myeloid leukemia (R/R AML) [1][2] Group 1: Clinical Trial Details - The "MIRACLE" trial is a multi-center, randomized, double-blind, placebo-controlled study aimed at evaluating Annamycin for R/R AML patients, particularly those who have failed venetoclax regimens [1][2] - The trial has been approved in all nine EU countries submitted, including Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain [2] - Patient enrollment and dosing have commenced, with initial data readout expected in the second half of 2025 [1][5] Group 2: Trial Design and Protocol - The trial utilizes an adaptive design, with the first 75 to 90 subjects randomized to receive high dose cytarabine combined with either placebo or two different doses of Annamycin (190 mg/m and 230 mg/m) [2][3] - The protocol allows for unblinding of preliminary primary efficacy data at 45 subjects, with the first unblinding expected in the second half of 2025 [3] - Part B of the trial will randomize approximately 220 additional subjects to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin [4] Group 3: Regulatory Status and Designation - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML and soft tissue sarcoma [6] - The EMA has also granted Orphan Drug Designation for Annamycin for the treatment of R/R AML [6] Group 4: Company Overview - Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company focused on developing therapeutic candidates for hard-to-treat tumors and viral infections [7] - The lead program, Annamycin, is designed to avoid multidrug resistance mechanisms and lacks the cardiotoxicity associated with current anthracyclines [7][8]

Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viruses [3] - The company's lead program, Annamycin, is a next-generation anthracycline designed to avoid multidrug resistance and cardiotoxicity, currently in development for relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases [3] Recent Developments - The company initiated patient recruitment for its pivotal Phase 3 MIRACLE trial (MB-108) evaluating Annamycin in combination with cytarabine for treating relapsed or refractory AML [4] - Following a successful Phase 1B/2 study (MB-106) and FDA input, the company believes it has de-risked the development pathway for Annamycin's potential approval for AML [4] Additional Pipeline - Moleculin is also developing WP1066, an Immune/Transcription Modulator targeting brain tumors and pancreatic cancers, and a portfolio of antimetabolites including WP1122 for treating pathogenic viruses and certain cancer indications [5] Upcoming Events - The company will report its financial results for Q1 2025 on May 13, 2025, followed by a conference call on May 14, 2025, at 8:30 AM ET [1][2]

Core Viewpoint - Annamycin, a next-generation anthracycline, is being developed by Moleculin Biotech, Inc. for the treatment of serious cancers, particularly relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases, with the potential to be safer and more effective than current treatments [1][4] Group 1: Drug Development and Trials - The ongoing pivotal Phase 3 clinical trial, named the "MIRACLE" trial, is evaluating Annamycin in combination with cytarabine (referred to as AnnAraC) for treating relapsed or refractory AML, with initial data expected in the second half of 2025 [1][3] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for AML treatment, as well as Orphan Drug Designation from the European Medicines Agency (EMA) [3] - The recruitment of patients for the MIRACLE trial commenced in March 2025, following a successful Phase 1B/2 study, which has de-risked the development pathway towards potential approval [5] Group 2: Regulatory Approvals and Naming - The World Health Organization's International Nonproprietary Names (INN) Expert Committee has approved "naxtarubicin" as the non-proprietary name for Annamycin, marking a significant step in its development and future commercialization [2] - The INN naming process ensures that drug names adhere to guidelines and are conflict-free, facilitating safe prescription and dispensing [2] Group 3: Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing therapeutic candidates for hard-to-treat tumors and viral infections, with Annamycin as its lead program [4] - The company is also developing other candidates, including WP1066, an immune/transcription modulator targeting various cancers, and WP1122, aimed at treating pathogenic viruses [6]

Core Viewpoint - Moleculin Biotech, Inc. has received two additional U.S. patents for Annamycin, enhancing its intellectual property portfolio and exclusivity for this novel drug candidate targeting hard-to-treat cancers and viruses [1][3]. Patent and Intellectual Property - The U.S. Patent and Trademark Office granted U.S. patent number 12,257,261 for the preparation of preliposomal Annamycin lyophilizate and U.S. patent 12,257,262 for the method of reconstituting liposomal Annamycin, both extending the patent term until June 2040 [1]. - With these new patents, the total number of U.S. patents related to Annamycin has increased to four, in addition to European patents granted [3]. Drug Development and Clinical Trials - Annamycin is being developed as a treatment for acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases (STS lung mets), with potential applications for other cancer types based on preclinical studies [2][4]. - The company is conducting the pivotal, adaptive Phase 3 MIRACLE trial evaluating Annamycin in combination with cytarabine for relapsed or refractory AML, with initial data expected in the second half of 2025 [3][5]. Regulatory Status - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for relapsed or refractory AML and STS lung mets, as well as Orphan Drug Designation from the European Medicines Agency [3]. Company Overview - Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company focused on developing therapeutic candidates for hard-to-treat tumors and viruses, with Annamycin as its lead program designed to avoid cardiotoxicity associated with current anthracyclines [4].