MBRX News & Events

Moleculin Biotech announced preliminary unblinded efficacy results from the first 45 patients enrolled in Part A of the company's pivotal Phase 2/3 MIRACLE trial, analyzed on a full intent-to-treat basis with no patient exclusions. Both Annamycin treatment arms demonstrated favorable efficacy trends compared with the control arm in patients with relapsed or refractory acute myeloid leukemia. The interim analysis demonstrated a clear efficacy advantage for both Annamycin treatment arms, 190 mg/m plus HiDAC and 230 mg/m plus HiDAC, over the HiDAC control arm. CR reached 43% and 36% in the respective Annamycin cohorts, compared with 12% for control, while CRc reached 50% and 57%, respectively, versus 29% for the control arm. The n=45 population contained 75.6% over 60 years of age, 55.6% 7+3 and 31.1% venetoclax regimens for first line therapies. Importantly, the remission rates for all three arms, including the control arm, reflect outcomes measured after only a single cycle of therapy, as specified by the MIRACLE protocol. The most commonly cited historical benchmarks in this setting, including the MIRROS and CLASSIC I studies, as well as Moleculin's own MB-106 study, permitted multiple cycles of treatment. The company therefore expected absolute remission rates for both the control and Annamycin arms in this single-cycle interim analysis to be lower than those reported in such multi-cycle datasets and believes the most meaningful comparison is the performance of the Annamycin arms relative to the concurrent, randomized control arm evaluated on the same single-cycle basis.

Moleculin Biotech announced it is approaching the first unblinding of data from its pivotal Phase 2B/3 "MIRACLE" trial evaluating Annamycin in combination with cytarabine for the treatment of subjects with relapsed or refractory acute myeloid leukemia. "The data coming from MIRACLE is encouraging," said Walter Klemp, chairman and CEO of Moleculin. "Against this backdrop, the data emerging from MIRACLE highlight what could be a substantial advancement, suggesting that we may finally be moving beyond the limitations that have defined standard-of-care outcomes". The company continues to expect this unblinding to occur prior to June 30. Preliminary blinded efficacy data for the first 45 subjects continues to approximate a composite complete remission rate exceeding 40% and a complete remission rate of approximately 30%.

Moleculin Biotech announced the publication of an abstract at the European Hematology Association 2026 Congress highlighting the cardiac safety profile of Annamycin, the Company's next-generation anthracycline currently in late-stage development for the treatment of relapsed/refractory acute myeloid leukemia. The abstract titled, "Cardiac Safety of L-Annamycin Across High Cumulative Anthracycline Exposure: Implications for Relapsed/Refractory AML," will highlight pooled cardiac safety findings from five completed clinical trials evaluating Annamycin in heavily pretreated patients, including those with substantial prior anthracycline exposure. Importantly, the analysis demonstrated no clinically significant treatment-related cardiotoxicity across cumulative anthracycline-equivalent doses that exceeded conventional lifetime exposure limits associated with traditional anthracyclines. Anthracyclines remain among the most effective agents in AML treatment, but their clinical utility is constrained by cumulative cardiac toxicity. This limitation is especially relevant in R/R AML, where patients often have prior anthracycline exposure and where currently available salvage therapies following venetoclax-based treatment have demonstrated limited efficacy. The independent cardiac review, conducted by a cardio-oncology laboratory at the Cleveland Clinic, analyzed comprehensive cardiac monitoring data from 90 patients treated with Annamycin. Among 78 patients with source-data verified pre- and post-treatment ejection fraction assessments, no patients met criteria for clinically significant left ventricular dysfunction. Mean ejection fraction remained stable, and no association was observed between cumulative dose and cardiac function decline. Additional analyses of serial ECGs, troponins, and global longitudinal strain assessments similarly demonstrated no evidence of drug-induced cardiotoxicity. Management believes these findings reinforce Annamycin's potential to address a major unmet need in R/R AML by enabling continued anthracycline-based treatment without the cumulative cardiac limitations commonly associated with conventional agents.

Moleculin Biotech announced a significant advancement in its intellectual property portfolio with the issuance of a Hong Kong patent covering its proprietary method of reconstituting liposomal Annamycin. The newly granted patent extends protection through June 25, 2040, further reinforcing Moleculin's long-term global exclusivity strategy. The invention is jointly owned with The University of Texas System.