Moleculin Receives Institutional Review Board Approval for MIRACLE Phase 3 Pivotal Trial of Annamycin in Combination with Cytarabine for the Treatment of R/R Acute Myeloid Leukemia (AML)
Moleculin Biotech (NASDAQ: MBRX) has received Institutional Review Board (IRB) approval for its MIRACLE Phase 3 pivotal trial evaluating Annamycin in combination with Cytarabine for treating relapsed or refractory AML. The global trial is designed for possible accelerated approval and is expected to begin enrollment in Q1 2025. The study will use an adaptive design with initial enrollment of 75-90 subjects in Part A, followed by approximately 240 additional subjects in Part B. Annamycin holds Fast Track Status and Orphan Drug Designation from both FDA and EMA for various indications.
Moleculin Biotech (NASDAQ: MBRX) ha ricevuto l'approvazione del Comitato Etico (IRB) per il suo trial pivotale MIRACLE di Fase 3 che valuta l'Annamycin in combinazione con il Citrarabina nel trattamento dell'AML recidivante o resistente. Lo studio globale è progettato per una possibile approvazione accelerata e ci si aspetta che inizi l'arruolamento nel primo trimestre del 2025. Lo studio utilizzerà un design adattivo con un arruolamento iniziale di 75-90 soggetti nella Parte A, seguito da circa 240 soggetti aggiuntivi nella Parte B. L'Annamycin ha lo Status di Fast Track e la Designazione di Farmaco Orfano sia dalla FDA che dall'EMA per varie indicazioni.
Moleculin Biotech (NASDAQ: MBRX) ha recibido la aprobación de la Junta de Revisión Institucional (IRB) para su ensayo pivotal MIRACLE de Fase 3 que evalúa Annamycin en combinación con Citarabina para tratar AML recidivante o refractaria. El ensayo global está diseñado para una posible aprobación acelerada y se espera que comience el reclutamiento en el primer trimestre de 2025. El estudio utilizará un diseño adaptativo con un reclutamiento inicial de 75-90 sujetos en la Parte A, seguido de aproximadamente 240 sujetos adicionales en la Parte B. Annamycin tiene el estatus de Fast Track y designación de Medicamento Huérfano tanto de la FDA como de la EMA para diversas indicaciones.
몰레큘린 바이오텍 (NASDAQ: MBRX)는 재발 또는 불응성 AML 치료를 위한 Annamycin과 Cytarabine의 조합을 평가하는 MIRACLE 3상 중대 시험에 대해 기관심사위원회(IRB)의 승인을 받았습니다. 이 글로벌 시험은 가속 승인을 위해 설계되었으며, 2025년 1분기에 피험자 모집을 시작할 것으로 예상됩니다. 본 연구는 적응형 설계를 사용하여 A파트에서 초기 75-90명의 피험자를 모집한 후 B파트에서 약 240명의 추가 피험자를 모집할 예정입니다. Annamycin은 FDA와 EMA로부터 여러 적응증에 대해 패스트트랙 및 오르팬 약물 지정을 받았습니다.
Moleculin Biotech (NASDAQ: MBRX) a obtenu l'approbation du Comité d'Éthique Institutionnel (IRB) pour son essai pivot MIRACLE de Phase 3 évaluant l'Annamycin en combinaison avec la Cytarabine pour le traitement des AML récidivantes ou réfractaires. L'essai mondial est conçu pour une approbation accélérée potentielle et devrait commencer le recrutement au premier trimestre de 2025. L'étude utilisera un design adaptatif avec un recrutement initial de 75 à 90 sujets dans la Partie A, suivi d'environ 240 sujets supplémentaires dans la Partie B. L'Annamycin bénéficie du statut Fast Track et de la désignation de médicament orphelin de la part de la FDA et de l'EMA pour diverses indications.
Moleculin Biotech (NASDAQ: MBRX) hat die Genehmigung der Ethikkommission (IRB) für seine MIRACLE Phase 3 pivotal Studie erhalten, die Annamycin in Kombination mit Cytarabin zur Behandlung von wiederkehrender oder refraktärer AML bewertet. Die globale Studie ist so konzipiert, dass sie eine mögliche beschleunigte Genehmigung erhält, und die Rekrutierung wird voraussichtlich im ersten Quartal 2025 beginnen. Die Studie verwendet ein adaptives Design mit einer initialen Rekrutierung von 75-90 Probanden in Teil A, gefolgt von etwa 240 zusätzlichen Probanden in Teil B. Annamycin hat den Status eines Fast Track und die Orphan Drug Designation sowohl von der FDA als auch von der EMA für verschiedene Indikationen.
- Received IRB approval for Phase 3 pivotal trial
- Trial designed for possible accelerated approval pathway
- Drug has Fast Track Status and Orphan Drug Designations from FDA and EMA
- First patient enrollment not expected until Q1 2025
- Large trial size requiring over 300 total subjects may extend completion timeline
Insights
The IRB approval for Moleculin's Phase 3 MIRACLE trial represents a significant milestone in the development of Annamycin for R/R AML treatment. The adaptive trial design is particularly noteworthy, incorporating FDA's Project Optimus initiative for optimal dosing strategy. With 315-330 total subjects planned across two parts, this pivotal study could potentially lead to accelerated approval.
The trial's design demonstrates strong regulatory alignment, supported by existing Fast Track Status and Orphan Drug Designations from both FDA and EMA. The combination therapy approach with cytarabine (AnnAraC) builds upon established treatment protocols while potentially offering improved outcomes for this difficult-to-treat patient population.
However, investors should note that with first patient enrollment targeted for Q1 2025, meaningful clinical data and potential commercialization remain distant milestones. The
Company on track for first subject treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) in the first quarter of 2025
Trial designed for possible accelerated approval of Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML
"IRB approval marks an important milestone towards the launch of our MIRACLE pivotal Phase 3 trial of Annamycin in AML patients. Our team is focused on getting clinical trial sites up and running, and we believe we will be in a position to commence enrollment in the first quarter of 2025," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We have been extremely busy meeting with potential investigators for this study and are highly encouraged by these conversations. The positive feedback from the clinicians on the strength of our data generated to date, along with our alignment with FDA on the strategic design of the MIRACLE study, gives us a high level of confidence as we take these next steps toward potentially bringing a much needed solution to AML patients."
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 our end of Phase 1B/2 meeting. At that point, the trial will be unblinded to select the optimum dose for Annamycin. For Part B of the trial, approximately 240 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.
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. 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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FAQ
When will Moleculin (MBRX) begin enrollment for the MIRACLE Phase 3 trial?
How many patients will be enrolled in Moleculin's (MBRX) MIRACLE Phase 3 trial?
What regulatory designations does Moleculin's (MBRX) Annamycin currently have?
