Moleculin Accelerates Recruitment in Phase 3 Acute Myeloid Leukemia “MIRACLE” Clinical Trial
Moleculin Biotech (Nasdaq: MBRX) reported significant progress in its Phase 3 "MIRACLE" clinical trial for Annamycin in combination with cytarabine (AnnAraC) for treating acute myeloid leukemia (AML). The company has recruited 13 subjects (10 already dosed) and expects to reach 20 subjects by September 2025.
The trial is expanding globally across multiple European countries and the US, with plans to increase from 12 to 20 active sites in September, and over 30 sites by year-end. The company aims to recruit the 45th subject in Q4 2025 for initial data unblinding, with Part A completion expected in H1 2026.
The MIRACLE study employs an adaptive design with 75-90 subjects in Part A and approximately 220 additional subjects in Part B. Annamycin currently holds Fast Track Status and Orphan Drug Designation from both FDA and EMA.
Moleculin Biotech (Nasdaq: MBRX) ha annunciato progressi rilevanti nella sua sperimentazione di Fase 3 «MIRACLE» per Annamycin in combinazione con citarabina (AnnAraC) nel trattamento della leucemia mieloide acuta (LMA). La società ha arruolato 13 soggetti (10 già trattati) e prevede di raggiungere 20 soggetti entro settembre 2025.
Lo studio si sta espandendo a livello globale in diversi paesi europei e negli Stati Uniti, con l'obiettivo di passare da 12 a 20 siti attivi a settembre e di superare i 30 siti entro fine anno. L'azienda punta ad arruolare il 45° soggetto nel quarto trimestre 2025 per il primo unblinding dei dati, mentre il completamento della Parte A è previsto nel primo semestre 2026.
Lo studio MIRACLE utilizza un disegno adattivo con una stima di 75-90 soggetti nella Parte A e circa 220 soggetti aggiuntivi nella Parte B. Annamycin beneficia attualmente dello status di Fast Track e della designazione di farmaco orfano sia dalla FDA sia dall'EMA.
Moleculin Biotech (Nasdaq: MBRX) informó avances importantes en su ensayo de Fase 3 «MIRACLE» para Annamycin en combinación con citarabina (AnnAraC) en el tratamiento de la leucemia mieloide aguda (LMA). La compañía ha reclutado 13 sujetos (10 ya dosificados) y espera alcanzar 20 sujetos para septiembre de 2025.
El ensayo se está ampliando a nivel mundial en varios países europeos y en EE. UU., con planes de aumentar de 12 a 20 centros activos en septiembre y superar los 30 centros a finales de año. La empresa pretende reclutar al 45.º sujeto en el cuarto trimestre de 2025 para el primer desenmascaramiento de datos, y la finalización de la Parte A está prevista para el primer semestre de 2026.
El estudio MIRACLE usa un diseño adaptativo con entre 75 y 90 sujetos en la Parte A y aproximadamente 220 sujetos adicionales en la Parte B. Annamycin cuenta actualmente con la condición de Fast Track y la designación de medicamento huérfano por parte tanto de la FDA como de la EMA.
Moleculin Biotech (Nasdaq: MBRX)는 급성 골수성 백혈병(AML) 치료를 위한 시타라빈과 병용한 Annamycin(AnnAraC)의 3상 임상시험 'MIRACLE'에서 의미 있는 진전을 보고했습니다. 회사는 피험자 13명 모집(이미 투여 완료 10명)을 완료했으며, 2025년 9월까지 20명에 도달할 것으로 예상합니다.
임상은 유럽 여러 국가와 미국으로 글로벌 확장을 진행 중이며, 9월에 활성 사이트를 12개에서 20개로 늘리고 연말까지 30개 이상을 목표로 하고 있습니다. 회사는 2025년 4분기에 45번째 피험자를 등록해 초기 데이터 블라인드 해제를 진행할 계획이며, 파트 A 완료는 2026년 상반기로 예상됩니다.
MIRACLE 연구는 적응형 설계를 사용하며 파트 A에 75–90명, 파트 B에 추가로 약 220명을 계획하고 있습니다. Annamycin은 현재 FDA와 EMA로부터 모두 패스트트랙 지정과 희귀의약품 지정을 받고 있습니다.
Moleculin Biotech (Nasdaq: MBRX) a annoncé des progrès significatifs dans son essai de phase 3 « MIRACLE » pour Annamycin en association avec la cytarabine (AnnAraC) dans le traitement de la leucémie myéloïde aiguë (LMA). La société a recruté 13 sujets (10 déjà traités) et prévoit d'atteindre 20 sujets d'ici septembre 2025.
L'essai s'étend à l'échelle mondiale dans plusieurs pays européens et aux États-Unis, avec des plans pour passer de 12 à 20 sites actifs en septembre et à plus de 30 sites d'ici la fin de l'année. La société vise à recruter le 45e sujet au T4 2025 pour le premier déblindage des données, la fin de la Partie A étant attendue au 1er semestre 2026.
L'étude MIRACLE utilise un schéma adaptatif avec 75 à 90 sujets dans la Partie A et environ 220 sujets supplémentaires dans la Partie B. Annamycin bénéficie actuellement du statut Fast Track et de la désignation de médicament orphelin auprès de la FDA et de l'EMA.
Moleculin Biotech (Nasdaq: MBRX) meldete bedeutende Fortschritte in seiner Phase-3-Studie „MIRACLE“ für Annamycin in Kombination mit Cytarabin (AnnAraC) zur Behandlung der akuten myeloischen Leukämie (AML). Das Unternehmen hat 13 Probanden rekrutiert (10 bereits dosiert) und erwartet, bis September 2025 auf 20 Probanden zu kommen.
Die Studie wird global auf mehrere europäische Länder und die USA ausgeweitet, mit Plänen, die Zahl der aktiven Studienzentren von 12 auf 20 im September zu erhöhen und bis Jahresende über 30 Zentren zu erreichen. Das Unternehmen plant, im 4. Quartal 2025 den 45. Probanden für das erste Unblinding der Daten zu rekrutieren; der Abschluss von Teil A wird für das 1. Halbjahr 2026 erwartet.
Die MIRACLE-Studie verwendet ein adaptives Design mit geschätzten 75–90 Probanden in Teil A und etwa 220 zusätzlichen Probanden in Teil B. Annamycin besitzt derzeit Fast-Track-Status und die Orphan-Drug-Designation sowohl von der FDA als auch von der EMA.
- Fast recruitment progress with 13 subjects already recruited and 10 dosed
- Significant site expansion from 12 to 20 sites in September, and over 30 by year-end
- Early data unblinding opportunity at 45 subjects in Q4 2025
- Strong patent protection through 2040 with potential extension to 2045
- Fast Track Status and Orphan Drug Designation from both FDA and EMA
- EMA requires additional nonclinical GLP studies before initiating Phase 3 Part B
- Complex trial design requiring multiple unblinding stages and regulatory approvals
Insights
Moleculin's accelerated MIRACLE trial recruitment shows operational efficiency with pivotal data expected in H2 2025.
Moleculin's announcement represents a significant operational milestone in their pivotal Phase 2B/3 MIRACLE trial for Annamycin in treating relapsed/refractory acute myeloid leukemia (AML). With 13 subjects already recruited (10 dosed) and expansion to 7 additional countries beyond Ukraine, the company is demonstrating impressive recruitment acceleration for a serious indication with limited treatment options.
The trial's adaptive design offers two crucial early data readouts - first at 45 subjects in Q4 2025 and another at completion of Part A (75-90 subjects) in H1 2026. This provides unusually early visibility into efficacy signals before progressing to the larger Phase 3 portion. Such staged unblinding represents sophisticated trial design that can potentially save resources if signals aren't promising or accelerate development if they are.
From a competitive standpoint, Annamycin's regulatory advantages are substantial with Fast Track Status and Orphan Drug Designation from FDA and EMA, plus composition of matter patent protection through 2040 (potentially extendable to 2045). These provide market exclusivity and development pathway advantages.
The AML relapsed/refractory setting remains a significant unmet need with dismal outcomes. If Annamycin shows meaningful complete remission improvements over cytarabine alone, it could represent a meaningful advance in this difficult-to-treat population. The company's planned expansion to 30+ active sites by year-end should further accelerate enrollment, though investor expectations should account for the inherent risk in any Phase 2/3 oncology program.
European expansion in September across Georgia, Italy, Lithuania, Poland, Romania, Spain, and US
13 subjects recruited (treated, enrolled, or screened)
Forecasts total recruitment of 20 or more subjects by end of September
On track to recruit to 45 patients in Q4 25 for initial data unblinding
HOUSTON, Sept. 09, 2025 (GLOBE NEWSWIRE) -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company”), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, today announced updates to its active site status and recruitment for its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine (also known as “Ara-C” and for which the combination of Annamycin and Ara-C is referred to as “AnnAraC”) for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML). This Phase 3 “MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East.
To advance this trial to recruiting the 45th subject in Q4 2025 for its first unblinding for early data insights into the trial’s efficacy and safety endpoints, the Company has the following expectations for September:
- Global Expansion: Expanding recruiting beyond Ukraine to include Spain, Georgia, Poland, Romania, Italy, Lithuania, and the US;
- Site Expansion: Adding 8 new active sites by the end of September, increasing the total to 20 sites recruiting;
- Recruitment Goals: Treating, enrolling, or screening a total of 20 subjects by the end of September; and
- Data Insights: Setting a solid footprint to recruit the 45th subject in Q4 2025, allowing for unblinded preliminary data assessment regarding efficacy and safety upon data lock.
Walter Klemp, Chairman and CEO of Moleculin, commented, “Our team is diligently expanding our active site footprint across the US, EU, and neighboring regions. We are confident in our target to recruit the 45th subject by Q4 2025 and achieve our first data unblinding shortly thereafter, especially since the consistent message we are receiving from investigators is that they are very eager to enroll patients in the MIRACLE trial. Moving from our end of Phase 2 meeting with the FDA to screening 13 subjects in just over a year exemplifies our operational efficiency. Of these 13, 10 have been dosed, further supporting our confidence in the speed of recruitment for MIRACLE.”
Mr. Klemp continued, “We expect to increase to over 30 active sites by year-end, as we move toward a second data unblinding for the conclusion of Part A of the trial. That will only require an additional 30 to 45 subjects, so we expect to conclude Part A in the first half of 2026, before commencing Part B of the trial. This level of early visibility is, we believe, exceptionally rare for a Phase 2B/3 trial and highly advantageous for investors.”
The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.
The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the recruitment of the first 45 subjects in the second half of 2025 with unblinding shortly thereafter, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.
As previously announced with regard to the EU, the clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the study. Results will be submitted as a substantial modification to the existing approved CTA.
For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.
For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.
Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company’s lead program, Annamycin, is a next-generation highly efficacious and well tolerated anthracycline designed to avoid multidrug resistance mechanisms and to lack the cardiotoxicity common with currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases.
The Company has begun the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway towards a potential approval for Annamycin for the treatment of AML. This study remains subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents.
Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of pathogenic viruses, as well as certain cancer indications.
For more information about the Company, please visit www.moleculin.com and connect on X, LinkedIn and Facebook.
Forward-Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the timing of the release of the initial data on the first 45 subjects in the trial, the timing of the completion of Part A of the trial, and the Company’s ability to reconcile the US and EU protocols with the FDA and EMA, respectively. Moleculin will require significant additional financing, for which the Company has no commitments, in order to conduct its clinical trials as described in this press release, and the milestones described in this press release assume the Company’s ability to secure such financing on a timely basis. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including ‘believes,’ ‘estimates,’ ‘anticipates,’ ‘expects,’ ‘plans,’ ‘projects,’ ‘intends,’ ‘potential,’ ‘may,’ ‘could,’ ‘might,’ ‘will,’ ‘should,’ ‘approximately’ or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. “Risk Factors” in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
Investor Contact:
JTC Team, LLC
Jenene Thomas
(908) 824-0775
MBRX@jtcir.com
FAQ
What is the current status of Moleculin's (MBRX) MIRACLE Phase 3 trial?
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What regulatory designations does Moleculin's Annamycin have?
How is the MIRACLE trial structured?
