Moleculin Receives First Country Approval in Europe to Begin Recruiting for the MIRACLE Phase 3 R/R Acute Myeloid Leukemia (AML) Pivotal Trial
Moleculin Biotech (NASDAQ: MBRX) has received its first country regulatory approval in Europe (Ukraine) to begin recruiting for the MIRACLE Phase 3 pivotal trial, evaluating Annamycin in combination with Cytarabine (AnnAraC) for treating refractory or relapsed AML patients. The global trial will include sites across the US, Europe, and the Middle East.
The study features an adaptive design with two parts: Part A will randomize 75-90 subjects (1:1:1) to receive high-dose cytarabine combined with either placebo, 190 mg/m2, or 230 mg/m2 of Annamycin. The amended protocol allows for preliminary data unblinding at 45 subjects in H2 2025. Part B will enroll approximately 244 additional subjects randomized between HiDAC plus placebo or HiDAC plus optimum Annamycin dose.
The company remains on track for first subject treatment in Q1 2025, with initial site approvals expected throughout Q1-Q3 2025.
Moleculin Biotech (NASDAQ: MBRX) ha ricevuto la sua prima approvazione normativa in Europa (Ucraina) per iniziare il reclutamento per il trial pivotale MIRACLE di Fase 3, che valuta l'Annamycin in combinazione con il Citarabina (AnnAraC) per il trattamento di pazienti con AML refrattaria o recidivante. Lo studio globale includerà siti negli Stati Uniti, in Europa e in Medio Oriente.
Lo studio presenta un design adattivo con due parti: la Parte A randomizzerà 75-90 soggetti (1:1:1) per ricevere citarabina ad alta dose combinata con placebo, 190 mg/m2 o 230 mg/m2 di Annamycin. Il protocollo modificato consente un'apertura dei dati preliminari per 45 soggetti nella seconda metà del 2025. La Parte B prevede di arruolare circa 244 ulteriori soggetti randomizzati tra HiDAC più placebo o HiDAC più la dose ottimale di Annamycin.
L'azienda rimane in carreggiata per il trattamento del primo soggetto nel primo trimestre del 2025, con le approvazioni iniziali dei siti previste durante il primo e il terzo trimestre del 2025.
Moleculin Biotech (NASDAQ: MBRX) ha recibido su primera aprobación regulatoria en Europa (Ucrania) para comenzar el reclutamiento para el ensayo pivotal MIRACLE de Fase 3, que evalúa Annamycin en combinación con Citarabina (AnnAraC) para tratar a pacientes con AML refractaria o en recaída. El ensayo global incluirá sitios en Estados Unidos, Europa y Medio Oriente.
El estudio presenta un diseño adaptativo con dos partes: la Parte A randomizará de 75 a 90 sujetos (1:1:1) para recibir citarabina de alta dosis combinada con placebo, 190 mg/m2 o 230 mg/m2 de Annamycin. El protocolo modificado permite la apertura de datos preliminares para 45 sujetos en la segunda mitad de 2025. La Parte B inscribirá aproximadamente 244 sujetos adicionales randomizados entre HiDAC más placebo o HiDAC más la dosis óptima de Annamycin.
La empresa sigue en camino para el tratamiento del primer sujeto en el primer trimestre de 2025, con las aprobaciones iniciales de los sitios esperadas a lo largo del primer y tercer trimestre de 2025.
몰레쿨린 바이오텍 (NASDAQ: MBRX)는 유럽 (우크라이나)에서 규제 승인을 최초로 받아 MIRACLE 3상 주요 시험을 위해 환자 모집을 시작할 수 있게 되었습니다. 이 시험은 재발성 혹은 난치성 급성 골수성 백혈병(AML) 환자를 치료하기 위해 Annamycin과 사이타라빈(AnnAraC)의 병용 요법을 평가합니다. 전 세계 시험은 미국, 유럽 및 중동에 걸쳐 사이트를 포함할 것입니다.
이 연구는 2개의 부분으로 이루어진 적응형 디자인을 특징으로 하며, Part A는 75-90명의 피험자를 (1:1:1) 무작위 배정하여 고용량 사이타라빈과 위약, 190 mg/m2 또는 230 mg/m2의 Annamycin과 조합하여 치료합니다. 수정된 프로토콜은 H2 2025에서 45명의 피험자에 대한 예비 데이터 공개를 허용합니다. Part B에는 HiDAC와 위약 또는 HiDAC와 최적의 Annamycin 용량 간에 무작위로 배정된 약 244명의 추가 피험자가 등록될 것입니다.
회사는 2025년 1분기에 첫 번째 피험자 치료를 진행할 계획이며, 초기 사이트 승인은 2025년 1분기부터 3분기까지 예상됩니다.
Moleculin Biotech (NASDAQ: MBRX) a reçu sa première approbation réglementaire en Europe (Ukraine) pour commencer le recrutement du essai pivot MIRACLE de Phase 3, évaluant l'Annamycin en combinaison avec la Cytarabine (AnnAraC) pour le traitement des patients atteints de LAM réfractaire ou en rechute. L'essai mondial comprendra des sites aux États-Unis, en Europe et au Moyen-Orient.
L'étude présente un design adaptatif en deux parties : la Partie A randomisera 75 à 90 sujets (1:1:1) pour recevoir de la cytarabine à forte dose combinée soit avec un placebo, soit avec 190 mg/m2 ou 230 mg/m2 d'Annamycin. Le protocole modifié permet une première ouverture des données pour 45 sujets dans la seconde moitié de 2025. La Partie B recrutera environ 244 sujets supplémentaires randomisés entre HiDAC plus placebo ou HiDAC plus la dose optimale d'Annamycin.
La société reste sur la bonne voie pour le traitement du premier sujet au premier trimestre 2025, avec les premières approbations de sites attendues tout au long du premier au troisième trimestre 2025.
Moleculin Biotech (NASDAQ: MBRX) hat die erste regulatorische Genehmigung in Europa (Ukraine) erhalten, um mit dem Rekrutierungsprozess für die MIRACLE Phase 3 entscheidende Studie zu beginnen, die Annamycin in Kombination mit Cytarabin (AnnAraC) zur Behandlung von refraktären oder zurückgekehrten AML-Patienten bewertet. Die globale Studie wird Standorte in den USA, Europa und dem Nahen Osten umfassen.
Die Studie hat ein adaptives Design mit zwei Teilen: Teil A wird 75-90 Probanden (1:1:1) randomisieren, die eine hochdosierte Cytarabin-Behandlung zusammen mit entweder einem Placebo, 190 mg/m2 oder 230 mg/m2 Annamycin erhalten. Das geänderte Protokoll erlaubt eine vorzeitige Datenfreigabe für 45 Probanden in der zweiten Jahreshälfte 2025. Teil B wird etwa 244 weitere Probanden einschließen, die zwischen HiDAC plus Placebo oder HiDAC plus optimaler Annamycin-Dosis randomisiert werden.
Das Unternehmen bleibt auf Kurs für die Behandlung des ersten Probanden im ersten Quartal 2025, wobei die ersten Standortgenehmigungen im Laufe des ersten bis dritten Quartals 2025 erwartet werden.
- First country approval received for Phase 3 trial recruitment
- Fast Track Status and Orphan Drug Designation from FDA
- Orphan Drug Designation from EMA
- Accelerated timeline for preliminary data unblinding (H2 2025)
- 30 investigators across 11 countries already engaged
- Complete trial results not expected until after H1 2026
- Additional regulatory approvals still needed for other countries
- Increased subject count (244 vs 240) due to additional interim unblinding
Insights
The approval to begin recruiting for the MIRACLE Phase 3 trial in Ukraine represents a significant milestone in Moleculin's clinical development program. The trial's adaptive design is particularly noteworthy for several reasons:
The protocol's amendment for early unblinding at 45 subjects is strategically brilliant, as it will provide important efficacy and safety data in H2 2025, potentially months ahead of the original timeline. This accelerated data readout could be a major catalyst for the company, especially given the 2:1 ratio of treated vs. control subjects (30 Annamycin vs. 15 placebo).
The trial's design aligns with the FDA's Project Optimus initiative, demonstrating regulatory sophistication. The two-dose strategy (190 mg/m2 and 230 mg/m2) allows for optimal dose selection based on a comprehensive benefit-risk assessment, potentially strengthening the eventual regulatory submission.
The global scope of the trial, spanning sites across the US, Europe and Middle East, suggests robust recruitment potential. With over 30 investigators across 11 countries already engaged and the CEO's personal involvement in site selection, the company appears well-positioned to meet its enrollment targets.
For investors, the combination of Fast Track Status and Orphan Drug Designations from both FDA and EMA provides multiple regulatory advantages, including potential market exclusivity and accelerated review pathways. The R/R AML market represents a significant opportunity, as current treatment options are and outcomes remain poor.
First of many site approvals, including sites in the US,
Company remains on track for first subject treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) in the first quarter of 2025
Recently amended protocol accelerates unblinding of preliminary primary efficacy data (CR) and safety/tolerability of the three arms at first 45 subjects, expected in the second half of 2025
"We continue to make solid progress across our site initiation and enrollment efforts. Achieving this important milestone of receiving our first country's approval puts us another step closer to getting this study well underway and further bolsters our confidence that we're on track for unblinded preliminary data from the first 45 subjects in the second half of this year. Our team remains committed to operational execution and face-to-face meetings with investigators, over 30 investigators in 11 countries of which I personally have met," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Building on this momentum, we are focused on getting more of the initial 25 sites we have selected to date on board and enrolling for the study."
The MIRACLE study, 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 amended protocol 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). This early unblinding will yield 30 subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15 subjects with just HiDAC. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.
For Part B of the trial, approximately 244 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. This increase from 240 to 244 subjects represents the statistical "cost" of the additional interim unblinding.
For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756.
Annamycin 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. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (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 anthracycline designed to avoid multidrug resistance mechanisms and to eliminate 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 is initiating 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 is 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 achievements of each of the milestones in this press release. 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
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SOURCE Moleculin Biotech, Inc.
FAQ
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