Key Highlights

● Moleculin completed enrollment of Part A of its MIRACLE trial, with 89 subjects enrolled and one more in screening.

● MIRACLE is a Phase 2/3 study of Annamycin plus cytarabine in adults with relapsed or refractory acute myeloid leukemia.

● The company expects to report Part A results in the first quarter of 2027, covering efficacy and safety.

● Moleculin says it requires significant additional financing and has no current commitments for it, and its milestones assume timely funding.

Moleculin Biotech, Inc. (NASDAQ:MBRX) said it has completed enrollment of Part A of its MIRACLE trial, a Phase 2/3 study of its drug candidate Annamycin combined with cytarabine in adults with relapsed or refractory acute myeloid leukemia (AML). According to a press release furnished with a Form 8-K on September 29, 2026, Part A enrolled 89 subjects, with one more in screening, and the company expects to report Part A results in the first quarter of 2027.

How the Trial Is Built

The release describes MIRACLE, also identified as MB-108, as a pivotal, adaptive-design, multicenter, randomized, double-blind, placebo-controlled study. In Part A, two dose levels of Annamycin plus cytarabine, a combination the company calls AnnAraC, are compared with a control arm of cytarabine plus placebo. Efficacy in Part A is measured after a single treatment cycle. The dataset is meant to help identify the optimal dose, which would then advance into Part B of the study.

The enrollment count is notably larger than the earlier interim analysis. The company had previously reported on a 45-subject cohort, and the 89 subjects now enrolled are about double that number. The release says the mix of patients has shifted as well. The initial 45-subject group was roughly 70 percent "fit" and 30 percent "unfit," while the final Part A population is expected to be approximately half fit and half unfit. In the release, fit subjects are those previously treated with intensive 7+3 chemotherapy, and unfit subjects are those previously treated with venetoclax-based regimens.

About Annamycin

Annamycin, also called naxtarubicin, is the company's lead program. The release describes it as an anthracycline designed to avoid multidrug resistance and to lack the cardiotoxicity associated with conventional anthracyclines. The company states that it "continues to see a lack of cardiotoxicity in MIRACLE's blinded data." That statement refers to blinded data, meaning the treatment assignments have not been revealed, and the release notes that cardiotoxicity assessments rely on expert reports. The release also says earlier Phase 1B/2 work, MB-106, gave positive signals that the company believes reduced risk in the development path, with input from the FDA.

Walter Klemp, Chairman and Chief Executive Officer, is quoted as saying: "Completing enrollment in Part A of MIRACLE is an important milestone for Moleculin and a strong demonstration of the operational execution behind this pivotal program." He also said the shift in patient composition provides "valuable insight into Annamycin's ability to Fill critical unmet needs."

Risks the Company Identifies

The release carries substantial cautionary language. It says Moleculin requires significant additional financing and has no current commitments for it, and that its milestones assume financing is available on a timely basis. It also cautions that the data remain subject to further subject review and long-term follow-up, and that trends seen in the interim analysis of 45 subjects may not be confirmed in the full population. The company also develops WP1066, an immune and transcription modulator aimed at p-STAT3 for brain tumors and pancreatic cancer, and WP1122, an antimetabolite for viruses and cancer indications, according to the release.

The filing was made under Item 7.01, Regulation FD Disclosure, meaning the information is furnished rather than filed, and Item 9.01. Moleculin is based in Houston, Texas.

Annamycin Part A Readout Due First Quarter 2027

The date the company gives is the first quarter of 2027 for Part A results. That readout is to cover efficacy and safety across the full randomized population and to inform the dose chosen for Part B. Because the trial is blinded, results by treatment arm have not been reported. The other item to follow is financing: the company states it has no commitments for the additional Capital it says it needs, so future filings may report how the trial and the Part B plan are funded.