Core Viewpoint - Moleculin Biotech is advancing its pivotal Phase 3 clinical trial, named the "MIRACLE" trial, for Annamycin in combination with Cytarabine for treating relapsed or refractory acute myeloid leukemia (R/R AML), with the first subject expected to be treated in Q1 2025 [1][8]. Company Developments - The FDA has provided feedback that allows Moleculin to reduce the size of Part B of the trial by approximately 10%, while major aspects of the trial remain unchanged [2]. - The trial will be global, with sites in the US, Europe, and the Middle East, facilitating an accelerated timeline for new drug approval [2][3]. - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML and soft tissue sarcoma, as well as Orphan Drug Designation from the European Medicines Agency [5]. Trial Design and Expectations - The MIRACLE trial will utilize 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 [3][4]. - Preliminary primary efficacy data will be unblinded at 45 subjects, with the first unblinding expected in the second half of 2025 and a second unblinding in the first half of 2026 [3]. - Part B of the trial will involve approximately 220 additional subjects randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin [4]. Product Overview - Annamycin is a next-generation anthracycline designed to avoid multidrug resistance mechanisms and eliminate cardiotoxicity associated with current anthracyclines, targeting R/R AML and soft tissue sarcoma [7][8].

First of many site approvals, including sites in the US, Europe and the Middle East, expected throughout Q1-Q3 2025 Company remains on track for first subject treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) in the first quarter of 2025 Recently amended protocol accelerates unblinding of preliminary primary efficacy data (CR) and safety/tolerability of the three arms at first 45 subjects, expected in the second half of 2025 HOUSTON, Feb. 11, 2025 /PRNewswire/ -- Moleculin Biotech, ...

Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses [4] - The company's lead program, Annamycin, is a next-generation anthracycline aimed at overcoming multidrug resistance and reducing cardiotoxicity associated with traditional anthracyclines [4] Clinical Trial Update - The company is conducting a Phase 3 pivotal trial named "MIRACLE" to evaluate Annamycin in combination with Cytarabine (AnnAraC) for treating relapsed or refractory acute myeloid leukemia (R/R AML) [2][5] - The MIRACLE trial will be a global study with sites in the US, Europe, and the Middle East [2] - Following a successful Phase 1B/2 study, the company believes it has significantly de-risked the development pathway for Annamycin's potential approval [5] Additional Developments - Moleculin is also developing WP1066, an immune/transcription modulator targeting various cancers, and a portfolio of antimetabolites, including WP1122 for treating pathogenic viruses and certain cancer indications [6]

Clinical Development Update - The company has received US Institutional Review Board (IRB) approval for the pivotal, adaptive Phase 3 clinical trial (MIRACLE trial) and engaged a leading contract research organization (CRO) [1] - The MIRACLE trial is on track to begin dosing of Annamycin in combination with cytarabine for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML) in Q1 2025 [1] - Expected timelines for recruitment updates and preliminary readouts of the MIRACLE trial have been accelerated to 2H 2025 (n=45) and 1H 2026 (n=~75-90) with potential for an accelerated NDA process beginning as early as 2027 [1] - Annamycin plus Ara-C achieved a 60% CR/CRi rate in subjects who were relapsed from or refractory to Venetoclax regimens, more than 4 times greater than published historical rates [1] - The MIRACLE trial will be global, including sites in the US, Europe, and the Middle East [3] - The trial utilizes an adaptive design with the first 75 to 90 subjects randomized in Part A to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin [4] - The amended protocol allows for unblinding of preliminary primary efficacy data (CR) and safety/tolerability at 45 subjects, with the first unblinding expected in 2H 2025 and the second in 1H 2026 [4] - Part B of the trial will randomize approximately 244 additional subjects to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin [5] Annamycin Performance and Potential - Annamycin has demonstrated a 50% CR rate and a 60% CRc rate for second-line subjects in the Phase 1B/2 study (n=10) [12] - Median durability was ~8 months and increasing, with median overall survival of 9.1 months for 0-6 prior lines of therapies (n=22) and 11.6 months for second-line subjects (n=10) [12] - Annamycin has shown strong efficacy even where prior venetoclax combination therapy has failed [12] - The company believes Annamycin is a potential game-changing asset for the treatment of AML, with significant value creation potential [2] - Annamycin has outperformed the response rates seen in billion-dollar assets in the AML space [2] - The drug has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of R/R AML and soft tissue sarcoma, as well as Orphan Drug Designation from the EMA for R/R AML [9] Expected Milestones - 1Q 2025: First subject enrolled and treated in the MIRACLE trial [12] - 2H 2025: Data readout (n=45) unblinded efficacy/safety review [12] - 1H 2026: Interim efficacy and safety data (n=~75-90) unblinded and optimum dose set for the MIRACLE trial [12] - 2027: Begin enrollment of third-line subjects in MIRACLE2 and end enrollment of second-line subjects [12] - 2028: Primary efficacy data for second-line subjects in MIRACLE and begin submission of a Rolling New Drug Application (NDA) for accelerated approval [12] Other Development Programs - The company completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases [8] - The clinical study report for the soft tissue sarcoma trial is expected to be completed in early 2025 [8] - The company is also developing WP1066, an Immune/Transcription Modulator targeting brain tumors, pancreatic and other cancers, and a portfolio of antimetabolites including WP1122 for the potential treatment of pathogenic viruses and certain cancer indications [15]

Abstract published as part of the American Society of Hematology (ASH) Annual MeetingStudy shows Annamycin effectively targets both Cytarabine (Ara-C) and Venetoclax resistant acute myeloid leukemia (AML) cell lines from heavily pretreated relapsed/refractory primary AML patients in vitroLack of apparent cardiotoxicity, improved organotropism, synergy with Ara-C, and possible immune-memory reinforcing properties appear to contribute to the favorable performance of Annamycin in clinical settingsSuch preclini ...

Core Insights - Annamycin shows significant activity in overcoming resistance to Venetoclax in acute myeloid leukemia (AML), with a 60% complete remission (CR) rate in patients previously treated with Venetoclax regimens, which is over four times higher than historical rates [1][2][4] Company Developments - Moleculin Biotech, Inc. is advancing Annamycin in a Phase 3 pivotal trial named MIRACLE, targeting relapsed or refractory AML patients, with patient treatment expected to begin in Q1 2025 [6][9] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for relapsed or refractory AML, as well as Orphan Drug Designation from the European Medicines Agency [7] Clinical Trial Results - Preliminary data from the MB-106 trial indicate a median overall survival of 11.6 months for subjects receiving Annamycin plus Ara-C as second-line therapy, and 9.1 months for those with a wider range of prior therapies [4][6] - The CR/CRi rate for patients treated with Annamycin in the MB-106 trial was reported at 60%, significantly higher than the 12.5% rate for standard salvage therapy in similar patient populations [2][4] Expert Commentary - Experts emphasize the need for better treatment options for AML patients who relapse after Venetoclax, highlighting Annamycin's potential to improve outcomes for these high-risk patients [2][5]

Core Viewpoint - Moleculin Biotech, Inc. has amended its clinical trial protocol for the Phase 3 "MIRACLE" trial evaluating Annamycin in combination with Cytarabine for treating relapsed or refractory acute myeloid leukemia (AML), aiming for accelerated approval and earlier data unblinding [1][2][8] Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses, with its lead program Annamycin targeting relapsed or refractory AML and soft tissue sarcoma [7][8] Clinical Trial Details - The MIRACLE trial will utilize an adaptive design, initially randomizing 75 to 90 subjects to receive high dose cytarabine combined with either placebo or two different doses of Annamycin (190 mg/m2 and 230 mg/m2) as recommended by the FDA [3][4] - The amended protocol allows for the unblinding of preliminary primary efficacy data and safety/tolerability results at 45 subjects, with expectations to reach this number in the second half of 2025 [3][5] - Part B of the trial will involve approximately 244 additional subjects randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin, with the optimum dose determined based on safety, pharmacokinetics, and efficacy [4] Regulatory Status - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory AML, as well as Orphan Drug Designation from the European Medicines Agency for the same condition [6]

Core Viewpoint - Moleculin Biotech, Inc. is advancing its pivotal Phase 3 clinical trial, named the "MIRACLE" trial, for Annamycin in combination with cytarabine for treating relapsed or refractory acute myeloid leukemia (AML), with the first subject expected to be treated in Q1 2025 [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, with Annamycin as its lead program targeting relapsed or refractory AML and soft tissue sarcoma [4][5]. Clinical Trial Details - The MIRACLE trial has received Institutional Review Board (IRB) approval and will include global sites, with an initial adaptive design involving 75 to 90 subjects randomized to receive high-dose cytarabine combined with either placebo or two different doses of Annamycin [1][2]. - The trial will consist of two parts: Part A will determine the optimum dose of Annamycin, while Part B will randomize approximately 240 additional subjects to receive either high-dose cytarabine plus placebo or the optimum dose of Annamycin [2][5]. Regulatory Status - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory AML, as well as Orphan Drug Designation from the European Medicines Agency (EMA) [3]. Future Development - The company is also developing WP1066, an immune/transcription modulator targeting various cancers, and WP1122, an antimetabolite for potential treatment of pathogenic viruses and certain cancer indications [6].

Financial Performance - The net loss for the three months ended September 2024 was $10,592,000, compared to a net loss of $5,609,000 for the same period in 2023, indicating an increase in losses of 88.5%[20] - The net loss per common share for the three months ended September 2024 was $(2.85), slightly higher than $(2.82) for the same period in 2023[20] - Total operating expenses for the three months ended September 2024 were $7,135,000, compared to $5,947,000 for the same period in 2023, reflecting a 19.9% increase[20] - Interest income for the three months ended September 2024 was $102,000, a decrease from $324,000 for the same period in 2023, representing a decline of 68.5%[20] - Other income, net for the three months ended September 2024 was $9,000, down from $13,000 for the same period in 2023, a decrease of 30.8%[20] - The total loss from operations for the nine months ended September 2024 was $19,998,000, compared to $20,712,000 for the same period in 2023, showing a slight improvement of 3.4%[20] Research and Development - Research and development (R&D) expense increased to $4.9 million for Q3 2024, up from $3.3 million in Q3 2023, primarily due to clinical trial costs[9] - Research and development expenses for the three months ended September 2024 were $4,932,000, an increase from $3,280,000 for the same period in 2023, representing a 50.3% increase[20] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML treatment[4] - The company plans to present final data from the Phase 2 study of Annamycin for soft tissue sarcoma lung metastases in 2025[8] Clinical Trials - The company is on track to start dosing in the pivotal Phase 3 MIRACLE trial in Q1 2025, aimed at treating relapsed or refractory acute myeloid leukemia (R/R AML) with Annamycin and cytarabine[1] - The median durability of complete response composite (CRc) in the MB-106 trial has exceeded 8 months[2] - The MIRACLE trial will initially randomize approximately 75 to 90 subjects to receive high dose cytarabine combined with either placebo or Annamycin[6] - Expected milestones for the MIRACLE trial include first subject treated in Q1 2025 and interim efficacy data unblinded in 2H 2026[7] Financial Position - As of September 30, 2024, the company had cash and cash equivalents of $9.4 million, expected to fund operations into Q1 2025[10] - The company closed a financing of $5.5 million, with potential additional gross proceeds of up to $11.0 million upon the exercise of milestone-linked warrants[3] Administrative Expenses - General and administrative expense decreased to $2.2 million in Q3 2024 from $2.6 million in Q3 2023, reflecting a reduction in regulatory and legal fees[10]

- On track to start dosing in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) designed for possible accelerated approval of Annamycin in combination with cytarabine for the treatment of R/R AML in Q1 2025- Median durability of CRc in MB-106 Annamycin+ Cytarabine AML clinical trial continues to climb - now past 8 months - Recent virtual AML KOL event underscores how Annamycin could significantly change the AML treatment landscape; Replay available here - Company to host conference call and web ...