Moleculin Accelerates Planned Unblinded Data Readout for MIRACLE Phase 3 R/R Acute Myeloid Leukemia (AML) Pivotal Trial to H2 2025
Moleculin Biotech (MBRX) has amended its Phase 3 MIRACLE trial protocol for Annamycin in combination with Cytarabine (AnnAraC) for treating relapsed/refractory AML. The amended protocol allows for earlier unblinding of preliminary efficacy data at 45 subjects in H2 2025, accelerating from the previous 2026 timeline. The trial will use an adaptive design with two parts: Part A will randomize 75-90 subjects across three arms, while Part B will include 244 additional subjects. The study maintains its planned first subject treatment start in Q1 2025. Annamycin holds Fast Track Status and Orphan Drug Designation from both FDA and EMA for various indications.
Moleculin Biotech (MBRX) ha modificato il protocollo della fase 3 dello studio MIRACLE per Annamycin in combinazione con Cytarabine (AnnAraC) nel trattamento dell'AML recidivante/refrattario. Il protocollo rivisto consente un'apertura dei dati preliminari di efficacia più precoce con 45 soggetti nel secondo semestre del 2025, accelerando rispetto alla tempistica precedente del 2026. Lo studio utilizzerà un design adattivo in due parti: Parte A randomizzerà 75-90 soggetti su tre bracci, mentre la Parte B includerà 244 soggetti aggiuntivi. Lo studio mantiene l'inizio del trattamento del primo soggetto programmato per il primo trimestre del 2025. Annamycin possiede lo status di Fast Track e la designazione di Farmaco Orfano sia dalla FDA che dall'EMA per diverse indicazioni.
Moleculin Biotech (MBRX) ha modificado su protocolo del ensayo de fase 3 MIRACLE para Annamycin en combinación con Citarabina (AnnAraC) para el tratamiento de la leucemia mieloide aguda (AML) en recaída/refractaria. El protocolo modificado permite una revelación anticipada de los datos preliminares de eficacia con 45 sujetos en la segunda mitad de 2025, acelerando respecto a la línea temporal anterior de 2026. El ensayo utilizará un diseño adaptativo en dos partes: la Parte A aleatorizará entre 75 y 90 sujetos en tres brazos, mientras que la Parte B incluirá 244 sujetos adicionales. El estudio mantiene programado el inicio del tratamiento del primer sujeto para el primer trimestre de 2025. Annamycin tiene el estatus de Fast Track y la designación de Medicamento Huérfano por parte de la FDA y la EMA para diversas indicaciones.
몰레큘린 바이오텍 (MBRX)이 아나마이신과 사이타라빈(AnnAraC)을 병용하여 재발/내성 급성 골수성 백혈병(AML) 치료를 위한 3상 MIRACLE 임상시험 프로토콜을 수정했습니다. 수정된 프로토콜은 2025년 하반기에 45명의 피험자에서 초기 유효성 데이터의 조기 공개를 허용하여 이전의 2026년 일정에서 앞당겨집니다. 이번 시험은 두 부분으로 구성된 적응형 디자인을 사용할 것이며, A 부분은 75-90명의 피험자를 세 개의 팔로 무작위 배정하고, B 부분은 추가로 244명의 피험자를 포함합니다. 연구는 2025년 1분기에 첫 번째 피험자의 치료 시작을 계획대로 진행합니다. 아나마이신은 FDA와 EMA로부터 다양한 적응증에 대해 신속 개발 절차(Fast Track) 및 희귀약품 지정(Orphan Drug Designation)을 받았습니다.
Moleculin Biotech (MBRX) a modifié le protocole de l'essai clinique de phase 3 MIRACLE pour Annamycin en combinaison avec la Cytarabine (AnnAraC) pour traiter la leucémie aiguë myéloïde (LAM) en rechute/réfractaire. Le protocole modifié permet un dévoilement précoce des données préliminaires d'efficacité avec 45 sujets au second semestre 2025, accélérant par rapport au calendrier précédent de 2026. L'essai utilisera une conception adaptative en deux parties : la Partie A randomisera 75-90 sujets sur trois bras, tandis que la Partie B inclura 244 sujets supplémentaires. L'étude maintient le début prévu du traitement du premier sujet au premier trimestre 2025. Annamycin possède un statut de Fast Track et une désignation de Médicament Orphelin de la part de la FDA et de l'EMA pour diverses indications.
Moleculin Biotech (MBRX) hat das Protokoll der Phase-3-Studie MIRACLE für Annamycin in Kombination mit Cytarabin (AnnAraC) zur Behandlung von rezidivierenden/refraktären AML geändert. Das geänderte Protokoll erlaubt eine frühere Aufhebung der Verblindung der vorläufigen Wirksamkeitsdaten bei 45 Probanden im zweiten Halbjahr 2025, was eine Beschleunigung im Vergleich zur vorherigen Zeitplanung 2026 darstellt. Die Studie wird ein adaptives Design in zwei Teilen verwenden: Teil A randomisiert 75-90 Probanden auf drei Arme, während Teil B 244 zusätzliche Probanden umfasst. Die Studie behält den geplanten Beginn der Behandlung des ersten Probanden im ersten Quartal 2025 bei. Annamycin hat den Status eines beschleunigten Verfahrens (Fast Track) und die Bezeichnung als Orphan Drug von der FDA und der EMA für verschiedene Indikationen.
- Accelerated timeline for preliminary data readout to H2 2025
- FDA Fast Track Status and Orphan Drug Designation maintained
- Protocol amendment could facilitate earlier strategic partnering opportunities
- Early unblinding may help de-risk trial financing
- Increased total subject count from 240 to 244 due to additional unblinding
- Protocol amendment still pending IRB review and FDA filing
Insights
The amended protocol for Moleculin's Phase 3 MIRACLE trial represents a significant strategic development in their AML treatment program. The key modification allowing early unblinding at 45 subjects in H2 2025 could provide important efficacy signals for AnnAraC much sooner than originally planned. The trial's adaptive design, incorporating FDA-recommended dosing levels of 190 mg/m2 and 230 mg/m2 of Annamycin, demonstrates strong regulatory alignment.
The study's structure, with 75-90 subjects in Part A and 244 additional subjects in Part B, is statistically robust. The early data readout could be particularly valuable for both financing and partnership discussions, potentially accelerating the path to market. The multiple regulatory designations (Fast Track Status and Orphan Drug Designation) suggest favorable development conditions and market potential.
Amended protocol accelerates unblinding of preliminary primary efficacy data (CR) and safety/tolerability of the three arms at first 45 subjects
Company remains on track for first subject treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) in the first quarter of 2025
Company releases Virtual Investor "What This Means" segment discussing the amended protocol; Available here
"Our team has been thoughtful and strategic with the design of the MIRACLE trial, which may allow for possible accelerated approval of Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML. This amended protocol enables us to share definitive data earlier, which helps to partially de-risk financing the trial and potentially accelerates the timeline for strategic partnering. We believe that the unblinding of data at 45 subjects will enable us to begin assessing all three arms of the study and provide us with a clear path forward in understanding the potential of Annamycin for AML patients. This change now puts us potentially less than 12 months away from definitive unblinded data that could be a strong indicator of our likelihood of approval, and the kind of data that is likely to drive advanced partnering discussions," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.
The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, is expected to initially utilize an adaptive design whereby the first 75 to 90 subjects will be randomized 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, such doses were specifically recommended by the FDA in the Company's end of Phase 1B/2 meeting. The amended protocol will allow for the unblinding of preliminary primary efficacy data (CR) and safety/tolerability of the three arms at 45 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 45 subjects in the second half of 2025, in addition to the planned unblinding expected in 2026 of the next 30-45 subjects.
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. 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 unblinding.
The amended protocol is currently being reviewed by the Institutional Review Board (IRB). Once approved, the amended protocol will be filed with the amendment for the Company's Initial New Drug (IND) application in the US with the FDA.
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 commencement of enrollment of the MIRACLE trial and the timing of the release of the unblinded data. 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
When will Moleculin (MBRX) release the first unblinded data from the MIRACLE Phase 3 trial?
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