On May 2, 2023 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a growing pipeline, including Phase 2 clinical programs, for hard-to-treat tumors and viruses, reported the successful completion of the first cohort in the Phase 1b portion of its Phase 1b/2 clinical trial using Annamycin in combination with Cytarabine for the treatment of AML (MB-106) (Press release, Moleculin, MAY 2, 2023, https://moleculin.com/moleculin-successfully-completes-first-cohort-in-phase-1b-2-trial-evaluating-annamycin-in-combination-with-cytarabine-ara-c-for-the-treatment-of-acute-myeloid-leukemia-aml/ [SID1234630849]).
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In this first cohort three subjects were treated, all of whom are believed to be relapsed from multiple prior therapies. Annamycin was dosed at 190 mg/m2, along with Cytarabine at 2.0 g/m2/day for 5 days (total dose of 10 g/m2), consistent with the familiar 7+3 regimen, combining daunorubicin and Cytarabine, typically used as a first-line induction therapy. In the Company’s study therapy Annamycin replaces daunorubicin. The Company, at the recommendation of the data safety committee, deemed the first cohort dose as safe and opened the second cohort with the Annamycin dose being increased to 230 mg/m2.
The median of prior therapies for these three subjects were 5 (2 to 7). One of the subjects, who was 78 years of age at the time of study initiation with 2 prior multi-year therapies, was preliminarily recorded as a complete response at three weeks, or "CR", per the protocol. This was subsequently confirmed to be a durable CR after four weeks. The other two subjects were shown to have disease progression. Subjects have already been identified as potential candidates for the second cohort.
"We are pleased with the rapid progress of this trial and to be advancing to the second cohort of escalated dosing. These preliminary results demonstrated by Annamycin in combination with Ara-C, add to our confidence in Annamycin’s potential to be an important treatment for refractory or relapsed AML. Since it wasn’t until we exceeded 210 mg/m2 in our single agent trials that we saw a consistent response rate (80%), we are glad to see a CR at just 190 mg/m2 and are eager to see how subjects respond at 230 mg/m2, especially since our preclinical animal studies showed a significant increase in activity when Annamycin is combined with Cytarabine, as it is in this trial. We are also encouraged to see a full slate of candidates already identified for the second cohort and our clinical team is working to get subject screening and dosing for this next cohort. We look forward to providing additional updates in the near future," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.
The Phase 1b/2 MB-106 trial is an open label trial that builds on the safety and dosage data from the two successfully concluded single agent Annamycin AML Phase 1 trials, MB-104 and MB-105, conducted in the U.S. and Europe, respectively.
Annamycin is the Company’s next-generation anthracycline that has been designed to be non-cardiotoxic and has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin (a commonly prescribed anthracycline), as well as demonstrating the ability to avoid the multidrug resistance mechanisms that typically limit the efficacy of doxorubicin and other currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory AML and soft tissue sarcoma (STS) lung metastases and the Company believes it may have the potential to treat additional indications.
Annamycin currently has Fast Track Status and Orphan Drug Designation from the US Food and Drug Administration (FDA) for the treatment of STS lung metastases and the treatment of relapsed or refractory AML. For more information about the MB-106 Phase 1/2 trial, visit clinicaltrialsregister.eu and reference EudraCT 2020-005493-10 or clinicaltrials.gov and reference NCT05319587.