Moleculin completes enrollment in Part A of its MIRACLE trial

Moleculin Biotech, Inc. (Nasdaq: MBRX) completed enrollment in Part A of its pivotal Phase 2/3 MIRACLE trial, the company said in a press release dated September 29, 2026. The release was filed as Exhibit 99.1 to a Form 8-K. The trial evaluates Annamycin in combination with cytarabine, called AnnAraC, for adults with relapsed or refractory acute myeloid leukemia (R/R AML). The release says 89 subjects are enrolled and two additional subjects are in screening.

Enrollment and timing

Because enrollment closed at 89 subjects, or potentially more, the company said it remains on track for a comprehensive data readout of Part A in the first quarter of 2027. The 8-K furnishes the release under Item 7.01 and says it is not filed for purposes of the Securities Exchange Act of 1934.

The company's annual report describes the trial as the MIRACLE trial, derived from Moleculin R/R AML AnnAraC Clinical Evaluation. It says a blinded preliminary data readout had already been released, and that the company expected an interim unblinding of data in mid-2026. The company describes that early visibility as unusual for a pivotal registration-enabling trial, because stakeholders receive preliminary safety and efficacy data long before the trial concludes.

The lead candidate

Since its inception, drug candidates from each of the company's three core portfolios have been in or have completed eighteen clinical trials, and each core technology is based substantially on discoveries made at, made in conjunction with, or licensed from the University of Texas MD Anderson Cancer Center in Houston, Texas. Annamycin, also called L-Annamycin and naxtarubicin, is the company's lead drug candidate. The annual report describes it as the first of its kind DNA binding agent and a next-generation anthracycline. The company says earlier anthracyclines are used to treat approximately half of all cancers but carry cardiotoxicity and multidrug resistance problems. It reports that five clinical trials showed Annamycin to be non-cardiotoxic in 90 patients, and that some patients were safely dosed at five times the typical lifetime maximum allowable anthracycline dose.

The annual report also states that Annamycin showed a complete remission rate of 50% and a composite complete remission rate of 60% in combination with cytarabine for R/R AML as second-line therapy, in 10 subjects. Among all comers, the 8 subjects who achieved complete remission showed durability of approximately 13 months, with median overall survival of 15 months at the time of analysis.

Financial position

The company reported no revenue for 2025 or 2024. Research and development expense was $15.9 million in 2025 and $17.7 million in 2024. The net loss was $33.6 million in 2025 and $26.0 million in 2024. Cash from operations was negative $22.7 million in 2025 and negative $23.9 million in 2024. Year-end cash and cash equivalents were $4.3 million at December 31, 2024. At June 30, 2026, cash and cash equivalents were $7.3 million, according to the company's XBRL data.

What enrollment does and does not mean

Finishing enrollment says nothing about whether the drug works. No unblinded efficacy result has been released from the trial in these sources, and the earlier "promising" description in the annual report applies to blinded data from the first 30 subjects. The annual report expected an interim unblinding in mid-2026 and a further one in the second half of 2026; the release now points to a Part A readout in the first quarter of 2027. The remission figures the company cites come from just 10 subjects, and the durability figure from 8, in earlier trials. Cash was $7.3 million at June 30, 2026, against $22.7 million of operating cash used in 2025.

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