Moleculin Receives Positive FDA Guidance for Acceleration of its Registration-Enabling MIRACLE Trial for R/R Acute Myeloid Leukemia (AML)
Moleculin Biotech (NASDAQ: MBRX) has received positive FDA guidance for its Phase 3 MIRACLE trial, evaluating Annamycin in combination with Cytarabine (AnnAraC) for treating refractory or relapsed AML patients. The FDA's feedback allows for a 10% reduction in Part B trial size while maintaining major aspects unchanged.
The MIRACLE trial will be conducted globally across the US, Europe, and Middle East. The adaptive design includes Part A with 75-90 subjects randomized 1:1:1, and Part B with approximately 220 subjects randomized 1:1. The company expects to reach first unblinding (45 subjects) in H2 2025 and second unblinding in H1 2026.
Annamycin, positioned as the first-ever non-cardiotoxic anthracycline, holds Fast Track Status and Orphan Drug Designation from both FDA and EMA. The company remains on track for first subject treatment in Q1 2025.
Moleculin Biotech (NASDAQ: MBRX) ha ricevuto una guida positiva dalla FDA per il suo trial di Fase 3 MIRACLE, che valuta Annamycin in combinazione con Cytarabine (AnnAraC) per il trattamento di pazienti con AML refrattaria o recidivante. Il feedback della FDA consente una riduzione del 10% della dimensione del trial nella Parte B, mantenendo invariati gli aspetti principali.
Il trial MIRACLE sarà condotto a livello globale negli Stati Uniti, in Europa e in Medio Oriente. Il design adattivo include la Parte A con 75-90 soggetti randomizzati 1:1:1, e la Parte B con circa 220 soggetti randomizzati 1:1. L'azienda prevede di raggiungere il primo sblocco (45 soggetti) nel secondo semestre del 2025 e il secondo sblocco nel primo semestre del 2026.
Annamycin, posizionato come il primo anthracycline non cardiotossico, detiene lo Status di Fast Track e la Designazione di Farmaco Orfano sia dalla FDA che dall'EMA. L'azienda rimane in linea per il trattamento del primo soggetto nel primo trimestre del 2025.
Moleculin Biotech (NASDAQ: MBRX) ha recibido una guía positiva de la FDA para su ensayo de Fase 3 MIRACLE, que evalúa Annamycin en combinación con Cytarabine (AnnAraC) para tratar a pacientes con AML refractaria o en recaída. La retroalimentación de la FDA permite una reducción del 10% en el tamaño del ensayo de la Parte B, manteniendo inalterados los aspectos principales.
El ensayo MIRACLE se llevará a cabo a nivel global en los EE.UU., Europa y Medio Oriente. El diseño adaptativo incluye la Parte A con 75-90 sujetos aleatorizados 1:1:1, y la Parte B con aproximadamente 220 sujetos aleatorizados 1:1. La empresa espera alcanzar el primer desbloqueo (45 sujetos) en el segundo semestre de 2025 y el segundo desbloqueo en el primer semestre de 2026.
Annamycin, posicionado como el primer antraciclínico no cardiotóxico, tiene el estatus de Fast Track y la designación de Medicamento Huérfano tanto de la FDA como de la EMA. La empresa sigue en camino para el tratamiento del primer sujeto en el primer trimestre de 2025.
Moleculin Biotech (NASDAQ: MBRX)는 재발성 또는 불응성 AML 환자를 치료하기 위해 Cytarabine(AnnAraC)와 함께 Annamycin을 평가하는 3상 MIRACLE 시험에 대해 FDA로부터 긍정적인 가이드를 받았습니다. FDA의 피드백은 주요 측면을 유지하면서 Part B 시험 크기를 10% 줄일 수 있도록 허용합니다.
MIRACLE 시험은 미국, 유럽 및 중동 전역에서 진행됩니다. 적응형 설계에는 75-90명의 피험자가 1:1:1로 무작위 배정되는 A 부분과 약 220명의 피험자가 1:1로 무작위 배정되는 B 부분이 포함됩니다. 회사는 2025년 하반기에 첫 번째 블라인딩 해제(45명)를, 2026년 상반기에 두 번째 블라인딩 해제를 예상하고 있습니다.
Annamycin은 최초의 비심장독성 안트라사이클린으로 자리 잡고 있으며, FDA와 EMA 모두에서 신속 심사 및 희귀의약품 지정을 받고 있습니다. 회사는 2025년 1분기에 첫 번째 피험자 치료를 진행할 계획입니다.
Moleculin Biotech (NASDAQ: MBRX) a reçu des retours positifs de la FDA concernant son essai de Phase 3 MIRACLE, qui évalue l'Annamycin en combinaison avec la Cytarabine (AnnAraC) pour le traitement des patients atteints de LAM réfractaire ou en rechute. Les commentaires de la FDA permettent une réduction de 10% de la taille de l'essai dans la Partie B, tout en maintenant les principaux aspects inchangés.
L'essai MIRACLE sera mené à l'échelle mondiale aux États-Unis, en Europe et au Moyen-Orient. Le design adaptatif comprend la Partie A avec 75-90 sujets randomisés 1:1:1 et la Partie B avec environ 220 sujets randomisés 1:1. L'entreprise prévoit d'atteindre le premier déverrouillage (45 sujets) au second semestre 2025 et le second déverrouillage au premier semestre 2026.
L'Annamycin, positionné comme le premier anthracycline non cardiotoxique, bénéficie du statut Fast Track et de la désignation de médicament orphelin de la part de la FDA et de l'EMA. L'entreprise reste sur la bonne voie pour le traitement du premier sujet au premier trimestre 2025.
Moleculin Biotech (NASDAQ: MBRX) hat positives Feedback von der FDA für seine Phase-3-Studie MIRACLE erhalten, die Annamycin in Kombination mit Cytarabine (AnnAraC) zur Behandlung von refraktären oder rezidivierenden AML-Patienten bewertet. Das Feedback der FDA erlaubt eine Reduzierung der Studiengröße in Teil B um 10%, während die wesentlichen Aspekte unverändert bleiben.
Die MIRACLE-Studie wird weltweit in den USA, Europa und dem Nahen Osten durchgeführt. Das adaptive Design umfasst Teil A mit 75-90 Probanden, die im Verhältnis 1:1:1 randomisiert werden, und Teil B mit etwa 220 Probanden, die im Verhältnis 1:1 randomisiert werden. Das Unternehmen erwartet die erste Entblindung (45 Probanden) im zweiten Halbjahr 2025 und die zweite Entblindung im ersten Halbjahr 2026.
Annamycin, das als erster nicht-kardiotoxischer Anthrazyklinder gilt, hat den Status eines Fast Track und die Orphan Drug Designation von sowohl der FDA als auch der EMA erhalten. Das Unternehmen ist auf Kurs, um die Behandlung des ersten Probanden im ersten Quartal 2025 zu beginnen.
- FDA guidance enables 10% reduction in Phase 3 trial size, potentially reducing costs and accelerating timeline
- Annamycin has Fast Track Status and Orphan Drug Designations from both FDA and EMA
- Trial timeline acceleration with first unblinding expected in H2 2025
- Strong investigator interest indicating potential for efficient recruitment
- Phase 3 trial completion and potential approval still years away
- Large-scale trial requiring approximately 300 total subjects across multiple regions
Insights
The FDA's guidance on Moleculin's MIRACLE trial represents a significant optimization of the registration pathway for Annamycin. The 10% reduction in Part B trial size isn't merely about cost savings - it reflects the FDA's confidence in the trial design and could accelerate the path to market by several months. The adaptive trial design, with two strategic unblinding points (45 and 75-90 subjects), provides critical early efficacy signals while maintaining statistical rigor.
The trial's structure is particularly noteworthy:
- Part A's three-arm design (including two different Annamycin doses) aligns with FDA's Project Optimus initiative, demonstrating a modern approach to dose optimization
- Early unblinding at 45 subjects provides a important go/no-go decision point, potentially saving resources and accelerating development
- The streamlined Part B with approximately 220 subjects optimizes statistical power while reducing operational complexity
The non-cardiotoxic profile of Annamycin represents a potential paradigm shift in anthracycline therapy. With anthracyclines used in nearly
The projected timeline, with first unblinding in H2 2025 and second unblinding in H1 2026, suggests potential market entry by late 2026 or early 2027, assuming positive trial results. The global trial footprint across US, Europe, and Middle East optimizes recruitment potential and supports future commercial expansion strategies.
Company remains on track for first subject treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) in the first quarter of 2025
"Given the importance of our Miracle trial as a pivotal registration clinical trial, we were extremely pleased to receive detailed follow up from the FDA regarding our IND amendment to enable the trial. While all of the major aspects of the trial remain unchanged, guidance from FDA recommended an alteration to the statistical plan that will allow us to reduce the size of Part B of our trial by approximately
Mr. Klemp added, "The spirit of collaboration surrounding the Miracle trial is, frankly, inspiring. From investigating clinicians to regulatory authorities around the world, people are starting to realize the potential significance of approving the first-ever non-cardiotoxic anthracycline. Annamycin's approval in AML alone would bring the potential to save thousands of lives every year, and the opportunity to eliminate the threat of cardiotoxicity in a wide range of tumors equates to as much as 20 times that in long term potential. It's critical to remember that nearly half of all cancers are treated with an anthracycline…and
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 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.
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.
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