Moleculin Reports More Positive Interim Data from AML Trial

Moleculin Biotech, Inc., a clinical-stage pharmaceutical company, has shared positive preliminary results from its ongoing Phase 1B/2 (MB-106) clinical trial. This trial evaluates the combination of Annamycin and Cytarabine, known as AnnAraC, for treating acute myeloid leukemia (AML). These results were presented at the European Hematology Association (EHA) 2024 Hybrid Congress, with additional insights shared at a KOL meeting in Madrid.

The trial has enrolled 22 subjects, with 20 having completed evaluations across various lines of treatment. Notably, 45% of these subjects achieved composite complete remission (CRc), with 40% achieving complete remission (CR) and one subject reaching complete remission with incomplete recovery of peripheral blood counts (CRi). The median durability of response (mDOR) for these 9 subjects stands at approximately six months and continues to improve.

A focused analysis on the 2nd line subjects reveals that out of 10 individuals, 50% achieved CR, and 60% reached CRc. For those in the first and second line treatment group (13 subjects), the CR rate was 54%, and the CRc rate was 62%. The median overall survival (mOS) in the 2nd line subjects is also around six months and increasing.

Importantly, 89% of the subjects achieving CRc had cytogenetic and mutation profiles typically associated with poor prognosis, including FLT3, IDH2, ASXL1, and KMT2A mutations. Although these findings are not yet statistically significant, they offer valuable insights for clinicians.

Walter Klemp, Chairman and CEO of Moleculin, expressed optimism about the preliminary data, highlighting the efficacy of AnnAraC in 2nd line AML patients as surpassing that of currently approved drugs. He emphasized the company's commitment to advancing its clinical program and preparing for an End of Phase 2 meeting with the FDA.

Reinforcing the potential of Annamycin, Mr. Klemp noted the significant unmet need for effective and safe treatments for relapsed/refractory AML. He believes the current data underscores the promise of Annamycin as a viable treatment option.

The median age of subjects in the MB-106 trial is 69 years. Among the 20 subjects who have completed treatment, 17 had relapsed/refractory AML, and 3 were undergoing first-line treatment. Two subjects discontinued early due to allergic reactions. All treated subjects underwent post-therapy bone marrow assessments, and no significant cardiotoxicity was observed, a common issue with traditional anthracyclines. The treatment was generally well-tolerated, with myelosuppression and infections being the primary adverse events.

Annamycin has garnered Fast Track Status and Orphan Drug Designation from the U.S. FDA for AML treatment and soft tissue sarcoma, and similar designations from the European Medicines Agency (EMA) for AML.

Moleculin Biotech continues to advance its pipeline, focusing on hard-to-treat tumors and viruses. Besides Annamycin, the company is developing WP1066, an immune/transcription modulator targeting brain tumors and pancreatic cancer, and WP1122 for potential viral treatments and certain cancers.

All clinical data from the MB-106 trial remains preliminary and subject to further analysis and updates.

The promising results from the ongoing MB-106 trial highlight Annamycin's potential to significantly improve treatment outcomes for patients with AML, particularly those with relapsed or refractory disease. These findings pave the way for further clinical developments and potential regulatory milestones.