Moleculin Biotech, Inc. reports developments as a clinical-stage pharmaceutical company advancing drug candidates for hard-to-treat tumors and viruses. Its lead program is Annamycin, also known as naxtarubicin, a next-generation anthracycline designed to avoid cardiotoxicity and multidrug resistance, with development focused on relapsed or refractory acute myeloid leukemia and soft tissue sarcoma lung metastases.
Recurring news covers Annamycin clinical and preclinical data, the MIRACLE study of Annamycin in combination with cytarabine, oncology conference presentations, intellectual-property protection for liposomal Annamycin, and corporate financial updates. Additional pipeline coverage includes WP1066, an immune/transcription modulator targeting p-STAT3 and other oncogenic transcription factors, and WP1122, an antimetabolite program for pathogenic viruses and cancer indications.
Moleculin Biotech (NASDAQ: MBRX) has received its first country regulatory approval in Europe (Ukraine) to begin recruiting for the MIRACLE Phase 3 pivotal trial, evaluating Annamycin in combination with Cytarabine (AnnAraC) for treating refractory or relapsed AML patients. The global trial will include sites across the US, Europe, and the Middle East.
The study features an adaptive design with two parts: Part A will randomize 75-90 subjects (1:1:1) to receive high-dose cytarabine combined with either placebo, 190 mg/m2, or 230 mg/m2 of Annamycin. The amended protocol allows for preliminary data unblinding at 45 subjects in H2 2025. Part B will enroll approximately 244 additional subjects randomized between HiDAC plus placebo or HiDAC plus optimum Annamycin dose.
The company remains on track for first subject treatment in Q1 2025, with initial site approvals expected throughout Q1-Q3 2025.
Moleculin Biotech (NASDAQ: MBRX) announced CEO Walter Klemp's participation in a Virtual Investor 'What This Means' segment, focusing on updates regarding the company's Phase 3 MIRACLE trial. The pivotal trial evaluates Annamycin in combination with Cytarabine (AnnAraC) for treating acute myeloid leukemia (AML) patients who are refractory to or relapsed after induction therapy.
The global MIRACLE trial (Moleculin R/R AML AnnAraC Clinical Evaluation) will include sites across the US, Europe, and the Middle East. During the segment, Klemp discussed recent developments in the AML space and their implications for Moleculin's Annamycin development program.
Moleculin Biotech (MBRX) has received IRB approval for its pivotal Phase 3 'MIRACLE' trial of Annamycin in combination with cytarabine for R/R AML treatment. The trial's preliminary readouts are expected in H2 2025 (n=45) and 1H 2026 (n=~75-90), with potential NDA process starting in 2027.
Recent preliminary clinical results show Annamycin plus Ara-C achieved 60% CR/CRi in subjects relapsed/refractory to Venetoclax regimens, exceeding historical rates by more than 4 times. The Phase 1B/2 trial demonstrated a 50% Complete Remission rate and 60% CRc rate for 2nd line subjects, with median survival of 11.6 months for 2nd line subjects.
The MIRACLE trial will be conducted globally, utilizing an adaptive design with two parts. Part A will randomize 75-90 subjects, while Part B will include approximately 244 additional subjects. The company has engaged Catalyst Clinical Research as CRO for the trial.
Moleculin Biotech announced the publication of preclinical data demonstrating the significant activity of Annamycin in treating venetoclax-resistant acute myeloid leukemia (AML) cell lines. The study, presented at the American Society of Hematology Annual Meeting, revealed that Annamycin effectively targets both Cytarabine (Ara-C) and Venetoclax resistant AML cell lines from heavily pretreated relapsed/refractory primary AML patients in vitro.
The data showed Annamycin's synergy with Ara-C and Venetoclax in reducing cell viability, a lack of cardiotoxicity, improved organotropism, and potential immune-memory reinforcing properties. These findings correlate with preliminary clinical data showing a 60% composite complete remission (CRc) rate in subjects using Annamycin and Ara-C (AnnAraC) as a second-line treatment.
Annamycin's favorable toxicity profile, as compared to DOX, and its ability to extend survival in combination with Ara-C, underscore its potential as a breakthrough treatment. Moleculin is advancing Annamycin in a Phase 3 trial, 'MIRACLE,' set to begin patient treatment in Q1 2025. Annamycin has Fast Track Status and Orphan Drug Designation from the FDA and EMA.
Moleculin Biotech (MBRX) announced new findings showing Annamycin's effectiveness in treating Venetoclax-resistant acute myeloid leukemia (AML). Preliminary clinical results demonstrate that Annamycin plus Ara-C achieved a 60% CR/CRi rate in subjects previously treated with Venetoclax regimens, which is more than 4 times higher than historical rates. The MB-106 trial showed median overall survival of 11.6 months in subjects receiving AnnAraC as second-line therapy. The company is advancing to a Phase 3 'MIRACLE' trial, with patient treatment expected to begin in Q1 2025. Annamycin has received Fast Track Status and Orphan Drug Designation from both FDA and EMA.
Moleculin Biotech (MBRX) has amended its Phase 3 MIRACLE trial protocol for Annamycin in combination with Cytarabine (AnnAraC) for treating relapsed/refractory AML. The amended protocol allows for earlier unblinding of preliminary efficacy data at 45 subjects in H2 2025, accelerating from the previous 2026 timeline. The trial will use an adaptive design with two parts: Part A will randomize 75-90 subjects across three arms, while Part B will include 244 additional subjects. The study maintains its planned first subject treatment start in Q1 2025. Annamycin holds Fast Track Status and Orphan Drug Designation from both FDA and EMA for various indications.
Moleculin Biotech (NASDAQ: MBRX) has received Institutional Review Board (IRB) approval for its MIRACLE Phase 3 pivotal trial evaluating Annamycin in combination with Cytarabine for treating relapsed or refractory AML. The global trial is designed for possible accelerated approval and is expected to begin enrollment in Q1 2025. The study will use an adaptive design with initial enrollment of 75-90 subjects in Part A, followed by approximately 240 additional subjects in Part B. Annamycin holds Fast Track Status and Orphan Drug Designation from both FDA and EMA for various indications.
Moleculin Biotech reported its Q3 2024 financial results and provided a corporate update. Key highlights include the initiation of dosing in the Phase 3 MIRACLE trial for Annamycin in Q1 2025. The median durability of CRc in the MB-106 trial has exceeded 8 months. Moleculin hosted a virtual AML KOL event and appointed Dr. Daniel D. Von Hoff to its Scientific Advisory Board.
Financially, R&D expenses rose to $4.9 million from $3.3 million year-over-year, while G&A expenses decreased to $2.2 million from $2.6 million. The company closed a $5.5 million financing and has $9.4 million in cash reserves, expected to fund operations into Q1 2025.
Upcoming milestones include the first subject treated in the MIRACLE trial in Q1 2025 and final data readout for the STS lung metastases trial in 2025. Moleculin will host a conference call on November 11, 2024, at 8:30 AM ET to discuss these updates.
Moleculin Biotech (MBRX) has scheduled its third quarter 2024 financial results announcement for November 8, 2024. The company will host a conference call and webcast to discuss operational and financial results on November 11, 2024, at 8:30 AM ET. Participants can join via phone using the numbers (877) 407-0832 (domestic) or (201) 689-8433 (international). The webcast will be available on the company's website and archived for 90 days.
Moleculin Biotech (NASDAQ: MBRX) has appointed Dr. Daniel D. Von Hoff to its Annamycin Scientific Advisory Board. Dr. Von Hoff, a Distinguished Professor at the Translational Genomics Research Institute and expert in pancreatic cancer, joins following promising preclinical evidence of Annamycin's potential in treating pancreatic cancer presented at AACR. Annamycin is currently in clinical trials for treating relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases. The drug holds Fast Track Status and Orphan Drug Designation from FDA for AML treatment, plus Orphan Drug Designation for soft tissue sarcoma and from EMA for AML.