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]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-37758 MOLECULIN BIOTECH, INC. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Inc ...

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]

Annamycin has the potential to be safer and more effective than current prescribed anthracyclines to treat serious, hard-to-treat cancers Ongoing pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) evaluating Annamycin for the treatment of acute myeloid leukemia (AML); Interim data readout expected in the second half of 2025 HOUSTON, May 06, 2025 (GLOBE NEWSWIRE) -- Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio o ...

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].

Core Viewpoint - Annamycin is a next-generation anthracycline that shows potential for synergistic effects with various FDA-approved anticancer therapies, indicating opportunities for expanded clinical applications in treating both hematological malignancies and solid tumors [1][2][4] Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viral infections, with Annamycin as its lead program [6][7] - The company is advancing its pipeline through pivotal clinical trials, including the MIRACLE trial for relapsed or refractory acute myeloid leukemia (AML) [8] Research Findings - Recent studies presented at the AACR Annual Meeting 2025 demonstrate that Annamycin can work effectively in combination with various FDA-approved drugs, both in vitro and in vivo [1][4] - The research aims to identify new clinical applications for Annamycin, particularly in combination therapies for treatment-resistant cancers [4][5] Clinical Trials - The company is initiating the MIRACLE trial, which evaluates Annamycin in combination with cytarabine for AML treatment, following a successful Phase 1B/2 study [8] - Annamycin has shown high activity against drug-resistant cell lines in previous experiments, suggesting its potential effectiveness in challenging cancer cases [3][4] Future Development - The company is exploring additional indications for Annamycin, including pancreatic cancer and soft tissue sarcomas, as part of its strategy to expand the drug's market potential [2][4]

Core Insights - Moleculin Biotech, Inc. announced that an abstract regarding its next-generation anthracycline, Annamycin, has been selected for poster presentation at the AACR Annual Meeting 2025 [1][2] 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 lead program, Annamycin, is designed to avoid multidrug resistance and eliminate cardiotoxicity associated with current anthracyclines, targeting relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases [3] Clinical Trials - The company is initiating the MIRACLE Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for relapsed or refractory AML [4] - Following a successful Phase 1B/2 study, the company believes it has de-risked the development pathway towards potential approval for Annamycin [4] Additional Developments - Moleculin is also developing WP1066, an immune/transcription modulator targeting brain tumors and other cancers, and a portfolio of antimetabolites including WP1122 for treating pathogenic viruses and certain cancer indications [5]