Moleculin Completes FDA Phase 2 Meeting for Annamycin in AML

HOUSTON, July 10, 2024 — Moleculin Biotech, Inc. (Nasdaq: MBRX), a clinical-stage pharmaceutical company focused on developing treatments for challenging tumors and viruses, has successfully concluded its End of Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA). This critical meeting pertains to the company's Phase 1B/2 clinical trial, which is evaluating the efficacy of Annamycin in conjunction with Cytarabine (Ara-C) for treating acute myeloid leukemia (AML). This combination therapy is known as AnnAraC and is being tested as both a first-line treatment and for patients who have not responded to or have relapsed after initial therapy.

The outcomes from the EOP2 meeting will be disclosed upon the receipt of official minutes from the FDA, expected by the end of the third quarter of 2024. Walter Klemp, the Chairman and CEO of Moleculin, expressed optimism regarding the productive discussions with the FDA and anticipates providing a detailed report on the meeting's outcomes before the end of August.

This EOP2 meeting was further substantiated by recent data from the ongoing MB-106 clinical trial. As of June 14, 2024, 22 subjects had been enrolled in the trial, with 20 completing efficacy evaluations. The results are promising: 45% achieved a composite complete remission (CRc or CR/CRi), with 40% reaching complete remission and one subject experiencing complete remission with an incomplete recovery of peripheral blood counts (CRi). Results for two additional subjects are still pending.

In the subset of 10 subjects who received AnnAraC as a second-line treatment, half achieved complete remission, and 60% achieved a composite complete remission. Among the 13 subjects who were treated either as a first or second-line therapy, 54% achieved complete remission, and 62% had a composite complete remission. The median duration of response (mDOR) for those who achieved composite complete remission is around seven months and is expected to increase.

Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory AML. It also holds Orphan Drug Designation for soft tissue sarcoma treatment and from the European Medicines Agency for relapsed or refractory AML. For more details about the MB-106 Phase 1B/2 trial, visit clinicaltrialsregister.eu (reference EudraCT 2020-005493-10) or clinicaltrials.gov (reference NCT05319587).

Moleculin Biotech, Inc. is dedicated to developing innovative treatments for difficult-to-treat tumors and viruses. Its leading program, Annamycin, is an advanced anthracycline designed to bypass multidrug resistance mechanisms and eliminate the cardiotoxicity seen with existing anthracyclines. Annamycin is in development for treating AML and lung metastases from soft tissue sarcoma.

Additionally, Moleculin is advancing WP1066, an Immune/Transcription Modulator that inhibits oncogenic transcription factors and stimulates a natural immune response, targeting cancers like brain tumors and pancreatic cancer. The company is also developing other antimetabolites, including WP1122, for potential viral treatments and certain cancer types.

Moleculin Biotech is committed to its mission of developing effective therapies for some of the most challenging illnesses, continually expanding its pipeline and advancing its clinical programs.