Moleculin Biotech, Inc. (NASDAQ:MBRX) Q4 2024 Earnings Conference Call March 24, 2025 8:30 AM ET Company Participants Walter Klemp - Chairman and CEO John Paul Waymack - Senior CMO Jonathan Foster - EVP and CFO Conference Call Participants Jonathan Aschoff - Roth Capital Partners Jason McCarthy - Maxim Group Vernon Bernardino - H.C. Wainwright Operator Hello, and welcome to the Moleculin Biotech Fourth Quarter and Full Year 2024 Update Conference Call and Webcast. A question-and-answer session will follow t ...

Financial Performance - The company reported a net loss of $21.8 million for the year ended December 31, 2024, compared to a net loss of $29.8 million in 2023[26]. - The weighted average common shares outstanding increased to 3,442,997 in 2024 from 1,975,610 in 2023[26]. Expenses - Research and development (R&D) expenses for the year ended December 31, 2024, were $17.7 million, a decrease of $1.8 million from $19.5 million in 2023[14]. - General and administrative (G&A) expenses decreased to $8.8 million in 2024 from $10.0 million in 2023, primarily due to reduced regulatory and legal services[15]. Cash Position - As of December 31, 2024, the company had cash and cash equivalents of $4.3 million, which, along with $9.3 million from February 2025 financing, is expected to fund operations into Q3 2025[16]. Clinical Trials - The company expects to unblind preliminary data from the first 45 subjects in the MIRACLE trial in the second half of 2025[2]. - The MIRACLE trial will randomize 75 to 90 subjects to receive either high dose cytarabine combined with placebo or Annamycin, with the first unblinding expected in the second half of 2025[5]. - The company completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial for Annamycin as monotherapy for soft tissue sarcoma lung metastases, with a clinical study report expected by the end of April 2025[10]. Regulatory Status - The company received positive FDA guidance for the MIRACLE trial, allowing for a smaller number of subjects in the study[6]. - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia[11].

– Continued progress in pivotal, adaptive Phase 3 clinical trial (the “MIRACLE” trial) toward first subject treated, on track to take place in the first quarter of 2025 – Multiple subjects currently being screened in first MIRACLE site activated – Initial data readout of the Phase 3 MIRACLE trial expected in the second half of 2025 – Company to host conference call and webcast today, Monday, March 24th at 8:30 AM ET HOUSTON, March 24, 2025 (GLOBE NEWSWIRE) -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Molecu ...

Clinical Trials and Drug 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 data expected by the end of 2025[22]. - The company has completed or is currently conducting fourteen clinical trials for its drug candidates, with three candidates showing human activity in clinical trials[23]. - The Phase 2B/3 trial for Annamycin is expected to be pivotal, with the first site initiated for the global trial[34]. - The FDA's EOP2 meeting in July 2024 resulted in a positive discussion and the design of a Phase 2B/3 pivotal trial (MIRACLE) for R/R AML patients, which will be a global trial including sites in the US, Europe, Western Asia, and the Middle East[39]. - The MIRACLE trial will utilize a double-blind, placebo-controlled design, comparing Annamycin to high-dose cytarabine (HiDAC) with complete remission (CR) as the primary endpoint[39]. - The company completed recruitment and treatment in the MB-107 Phase 1B/2 clinical trial using Annamycin for STS lung mets in 2023, with follow-up for progression-free survival (PFS) and overall survival (OS) during 2024[38]. - The company plans to initiate a follow-on MIRACLE2 trial for third-line patients once the optimum dose is established in the MIRACLE trial[39]. - The company completed recruitment of 22 subjects for the MB-106 clinical trial, focusing on Annamycin in combination with Cytarabine for AML treatment[84]. - The Phase 1 clinical trial of Annamycin (MB-104) successfully demonstrated safety at the lifetime maximum allowable dose of anthracycline, with no signs of cardiotoxicity observed[67]. - The Phase 1B/2 clinical trial for Annamycin in STS lung metastases has completed enrollment with 36 subjects, and results are anticipated by the end of April 2025[100]. Drug Efficacy and Safety - Annamycin has shown a complete response (CR) rate of 50% and a composite complete response (CRc) rate of 60% in combination with Cytarabine for the treatment of R/R AML, which is higher than other approved second-line therapies[25]. - Annamycin has demonstrated non-cardiotoxicity in clinical trials, with some patients safely dosed at five times the typical lifetime maximum allowed anthracycline dose[25]. - Annamycin's overall survival (OS) rate is approximately 11 months, which is climbing, in combination with Cytarabine for the treatment of R/R AML[25]. - Annamycin has shown no signs of cardiotoxicity in clinical trials, allowing for higher dosing and potential treatment of traditionally "Unfit" patients[64]. - In the MB-106 trial, 41% of subjects achieved a complete response (CR) or CR with incomplete recovery (CRi) when treated with Annamycin and Cytarabine[76]. - The combination of Annamycin and Cytarabine (AnnAraC) demonstrated a 68% improvement in median overall survival (OS) compared to Annamycin alone and a 241% increase compared to Cytarabine alone in animal models[72]. - The company has received independent assessments covering 84 subjects treated with Annamycin, showing no evidence of cardiotoxicity, even with doses exceeding the FDA's lifetime maximum anthracycline limit[56]. Regulatory Designations and Market Potential - The company has received orphan drug and fast track status for Annamycin, with patent protection extending through 2040[25]. - The FDA and EMA have granted Orphan Drug Designation (ODD) to Annamycin for AML and soft tissue sarcoma, indicating a medically plausible basis for its use[48]. - The company has obtained orphan drug designations (ODD) from the FDA for Annamycin, WP1066, and WP1122 for various cancer treatments[147]. - The FDA granted orphan drug designation (ODD) for WP1066 for glioblastoma treatment, indicating a medically plausible basis for its use[105]. - The FDA granted "Rare Pediatric Disease" designation for WP1066, which may lead to a Priority Review Voucher valued at up to $100 million[170]. - The US market for anthracyclines, including Annamycin, was estimated at approximately $1.3 billion in 2023 and is expected to grow to $2 billion by 2032[158][159]. - The global market size for soft tissue sarcoma (STS) is projected to grow from $1.58 billion in 2024 to $2.57 billion by 2030[164]. - The American Cancer Society projects 2.0 million new cancer cases and 618,120 cancer deaths in the US in 2025, highlighting the significant market potential for oncology drugs[156]. Competitive Landscape and Challenges - The company faces significant competition in the oncology market from larger pharmaceutical and biotechnology firms, as well as academic institutions[185]. - FDA approved targeted therapies for AML patients with specific gene mutations, but they only serve subsets of the population[189]. - Only 20% of patients with soft-tissue sarcomas (STS) lung metastases respond to current treatments, indicating a significant unmet need[190]. - There are currently 551 ongoing clinical trials for pancreatic cancer, leading to intense competition for patient enrollment[191]. Financial and Operational Aspects - The company has a workforce of 17 full and part-time employees as of year-end 2024, operating primarily in a virtual environment[139]. - The company does not have manufacturing facilities and contracts all manufacturing activities to third parties[139]. - Total expenses under license agreements for the years ended December 31, 2024, and 2023 were $0.2 million and $0.3 million, respectively[175]. - The company has a sponsored research agreement with MD Anderson, with expenses of $2.0 million and $0.8 million for the years ended December 31, 2024, and 2023, respectively[176]. - The reverse stock split on March 22, 2024, reduced the number of authorized shares from approximately 33 million to 2 million[182]. Drug Mechanism and Research - Annamycin is designed to avoid multidrug resistance and has shown no cardiotoxicity in subjects treated in five clinical trials to date[28]. - WP1066 has shown significant anti-tumor activity in various animal models, with the ability to inhibit key transcription factors such as c-Myc and HIF-1α, and boost immune response by reducing TRegs[107]. - WP1066 has demonstrated potential for combination use with checkpoint inhibitors, with preclinical evidence suggesting it may reverse immune tolerance in brain tumor patients[109]. - The company believes WP1122 has the potential to enhance the effectiveness of checkpoint inhibitors and impact hard-to-treat viruses reliant on glycolysis[131].

Core Viewpoint - Moleculin Biotech, Inc. is set to report its financial results for the year ended December 31, 2024, on March 21, 2025, with a conference call scheduled for March 24, 2025, to discuss these results [1][2] Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses [1][3] - The company's lead program, Annamycin, is a next-generation anthracycline aimed at overcoming multidrug resistance and reducing cardiotoxicity associated with current treatments [3] Clinical Trials and Development - The company is initiating the MIRACLE Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for relapsed or refractory acute myeloid leukemia (AML) [4] - Following a successful Phase 1B/2 study, the company believes it has de-risked the development pathway for Annamycin's potential approval for AML treatment [4] - Additionally, the company is developing WP1066, an immune/transcription modulator targeting various cancers, and a portfolio of antimetabolites, including WP1122 for treating pathogenic viruses and certain cancer indications [5]

Core Insights - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focusing on drug candidates for hard-to-treat tumors and viruses [1][3] - The company participated in the Virtual Investor "Top 5 for '25" On-Demand Conference, where CEO Walter Klemp highlighted five reasons for investment interest in Moleculin [2] Company Overview - Moleculin is advancing a pipeline of therapeutic candidates, with its lead program Annamycin targeting relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases [3] - Annamycin is designed to avoid multidrug resistance and eliminate cardiotoxicity associated with current anthracyclines [3] Clinical Trials - The company is initiating the MIRACLE Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for AML treatment [4] - Following a successful Phase 1B/2 study, the company believes it has de-risked the development pathway for Annamycin's potential approval [4] Additional Developments - Moleculin is also developing WP1066, an Immune/Transcription Modulator targeting brain tumors and pancreatic cancers [5] - The company is working on a portfolio of antimetabolites, including WP1122 for treating pathogenic viruses and certain cancer indications [5]

Core Insights - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses [3] - The company will participate in the 37th Annual ROTH Conference on March 18, 2025, with a live webcast of the fireside chat available [1][2] Company Overview - Moleculin Biotech, Inc. is advancing a pipeline of therapeutic candidates, with its lead program Annamycin targeting relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases [3] - Annamycin is designed to avoid multidrug resistance and eliminate cardiotoxicity associated with current anthracyclines [3] Clinical Trials - The company is initiating the MIRACLE Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML [4] - Following a successful Phase 1B/2 study, the company believes it has de-risked the development pathway for Annamycin towards potential approval [4] Additional Developments - Moleculin is also developing WP1066, an immune/transcription modulator targeting brain tumors and pancreatic cancers [5] - The company is working on a portfolio of antimetabolites, including WP1122, for potential treatment of pathogenic viruses and certain cancer indications [5]

Core Insights - Moleculin Biotech, Inc. has received a Notice of Intent to Grant for a European patent for its drug candidate Annamycin, which is aimed at treating certain cancers and is expected to enhance global exclusivity [1][2][3] - The company anticipates an initial data readout from the phase 3 MIRACLE trial in the second half of 2025, which evaluates Annamycin in combination with cytarabine for relapsed or refractory acute myeloid leukemia (AML) [1][3] Patent and Market Expansion - The new patent will cover methods for making liposomal Annamycin suspension and its compositions for cancer treatment, with a base patent term extending until June 2040, potentially longer due to regulatory approval processes [2] - Annamycin is positioned to be the first non-cardiotoxic anthracycline approved for use, with ongoing studies suggesting its potential for treating various cancer types [2] Clinical Development - The MIRACLE trial (MB-108) is a pivotal phase 3 study that follows a successful phase 1B/2 study, indicating a de-risked development pathway for Annamycin's approval for AML treatment [3] - The company is also developing WP1066, an immune/transcription modulator targeting brain tumors and other cancers, along with a portfolio of antimetabolites for treating pathogenic viruses and certain cancer indications [4]

Core Insights - Moleculin Biotech, Inc. has announced that its abstract has been accepted for poster presentation at the Acute Leukemias XIX International Symposium, scheduled for March 16-19, 2025, in Munich, Germany [1][2]. Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses. Its lead program, Annamycin, is a next-generation anthracycline aimed at overcoming multidrug resistance and reducing cardiotoxicity associated with traditional anthracyclines [3]. - The company is currently advancing a pipeline that includes Annamycin for the treatment of 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. This follows a successful Phase 1B/2 study, which has provided confidence in the development pathway towards potential approval [4]. - The trial design is adaptive, and the company is preparing for future regulatory filings with the FDA and other international regulatory bodies [4]. Additional Developments - Moleculin is also developing WP1066, an immune/transcription modulator targeting various cancers, including brain tumors and pancreatic cancer. Additionally, the company is working on a portfolio of antimetabolites, including WP1122, aimed at treating pathogenic viruses and certain cancer indications [5].

Core Viewpoint - Moleculin Biotech, Inc. has announced a securities purchase agreement for a registered direct offering and concurrent private placement, aiming to raise approximately $3.5 million for working capital and corporate purposes [1][2]. Group 1: Offering Details - The company will sell 3,271,029 shares of common stock (or pre-funded warrants) at a price of $1.07 per share, along with warrants to purchase up to 6,542,058 additional shares [1]. - The warrants will have an exercise price of $1.07 per share, exercisable upon shareholder approval, and will expire five years from the initial exercise date [1]. - Roth Capital Partners is acting as the exclusive placement agent for the offering, which is expected to close on or about February 26, 2025 [2]. Group 2: Use of Proceeds - The net proceeds from the offering will be utilized for working capital and general corporate purposes [2]. Group 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 [6]. - The lead program, Annamycin, is designed to avoid multidrug resistance and cardiotoxicity, currently in development for relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases [6][7]. - The company is initiating the MIRACLE trial, a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for AML treatment [7]. Group 4: Additional Developments - Moleculin is also developing WP1066, an immune/transcription modulator targeting various cancers, and a portfolio of antimetabolites including WP1122 for potential treatment of pathogenic viruses [8].