On August 14, 2026 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported financial results for the second quarter ended June 30, 2026, and highlighted continued progress in its pivotal Phase 2/3 MIRACLE trial evaluating Annamycin in relapsed or refractory acute myeloid leukemia (AML).
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Moleculin continues to advance the pivotal Phase 2/3 MIRACLE trial following positive preliminary unblinded efficacy results from the first 45 patients enrolled in Part A, which showed complete remission rates at least three times greater than the control arm. Enrollment remains on track to reach the 90-patient milestone in September with the data readout in the December 2026 to February 2027 timeframe. These data are expected to support selection of the optimal Annamycin dose and the planned initiation of Part B in the first half of 2027.
Walter Klemp, Chairman and Chief Executive Officer of Moleculin, commented, "In addition to the positive interim MIRACLE results, we are just as excited by the feedback we are receiving from investigators about Annamycin and their enthusiasm for participating in the study. Their response reflects both the significant unmet need in relapsed or refractory AML and growing recognition of Annamycin’s potential, with its encouraging data generated to date and differentiated cardiac safety profile, to play an important role in the treatment landscape. As we advance toward the 90-patient milestone and next unblinded efficacy readout, this strong investigator engagement adds to our confidence in the program and the potential for Annamycin to meaningfully improve outcomes for patients."
Recent Highlights
Reported positive interim results from the Phase 2/3 MIRACLE trial, with both Annamycin treatment arms demonstrating complete remission (CR) rates at least three times higher than the control arm in patients with relapsed or refractory AML. The interim analysis demonstrated a clear efficacy advantage for both Annamycin treatment arms, 190 mg/m² plus cytarabine and 230 mg/m² plus cytarabine, over the cytarabine control arm. CR reached 43% and 36% in the respective Annamycin cohorts, compared with 12% for control, while composite complete remission (CRc) reached 50% and 57%, respectively, versus 29% for the control arm. The n=45 population contained 75.6% over 60 years of age, 55.6% 7+3 and 31.1% venetoclax regimens for first line (1L) therapies.
Announced independent market research indicating strong physician intent to prescribe Annamycin based on its potential efficacy, transplant-bridging potential and favorable cardiac safety profile.
Presented data at the 2026 ASCO (Free ASCO Whitepaper) Annual Meeting demonstrating no detectable cardiotoxicity with Annamycin despite cumulative exposure levels exceeding conventional anthracycline limits, further supporting its differentiated safety profile.
Chairman and Chief Executive Officer Walter Klemp discussed the positive preliminary MIRACLE interim results during a Virtual Investor "What This Means" segment, providing additional context on the Company’s clinical progress and anticipated next milestones.
Enrollment continued in Part A of the MIRACLE trial, with more than 80% of the planned 90 patients enrolled as of the interim analysis.
Cash on hand and cash equivalents as of June 30, 2026, together with $9.3 million in financing proceeds raised subsequent to the quarter, expected to support planned operations into the first quarter of 2027.
Clinical Development Update
Annamycin – MIRACLE Trial
Moleculin continues to advance the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial, a pivotal adaptive-design Phase 2/3 study evaluating Annamycin in combination with cytarabine (AnnAraC) for the treatment of adults with relapsed or refractory acute myeloid leukemia.
During the second quarter, the Company reported positive preliminary unblinded efficacy results from the first 45 patients enrolled in Part A of the trial. Both Annamycin treatment arms demonstrated substantially higher complete remission rates than the control arm, while maintaining the encouraging safety profile previously observed in clinical studies. Activity in the MIRACLE trial continues to demonstrate no evidence of cardiotoxicity. The Company continues to enroll patients in Part A and expects to use these data to select the optimal dose for advancement into Part B.
Expected Milestones for the Annamycin Development Program
September 2026: Completion of enrollment in Part A of MIRACLE
December 2026 to February 2027 timeframe: MIRACLE – data unblinding for Part A 90 subjects completed
2H 2026: Atlantic Health pancreatic cancer clinical trial begins
1H 2027: MIRACLE – Start of Part B
2027: Begin 3rd line R/R AML subject trial
2027: Begin pediatric AML clinical study
2028: End recruitment of Part B
2028: Primary efficacy data for MIRACLE 2nd line subjects
2028: Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE
2029: NDA submission complete
Additional Pipeline Programs
Moleculin continues to support development activities for WP1066 through investigator-sponsored and externally funded studies, while maintaining its focus on advancing Annamycin as the Company’s lead clinical program.
Second Quarter 2026 Financial Results
Research and development expenses were $5.5 million and $3.6 million for the three months ended June 30, 2026 and 2025, respectively. The increase of $1.9 million is mainly related to the MIRACLE clinical trial of $1.1 million, additional nonclinical studies of $0.4 million, and $0.4 million in other research costs during the current quarter as compared to the prior year quarter.
General and administrative expenses for the quarter ended June 30, 2026 were approximately $2.1 million, compared with approximately $2.1 million for the same period in 2025.
As of June 30, 2026, the Company had cash and cash equivalents of approximately $7.3 million. Management believes that its cash on hand and cash equivalents as of June 30, 2026, together with $9.3 million in financing proceeds raised subsequent to the quarter, will support planned operations into the first quarter of 2027.
(Press release, Moleculin, AUG 14, 2026, View Source [SID1234670128])