Moleculin Expands Phase 3 MIRACLE Clinical Trial into the Country of Georgia, Enrollment Update and Future Plans
Moleculin Biotech (Nasdaq: MBRX) has received approval from Georgia's RAMPA to conduct its Phase 2B/3 MIRACLE clinical trial for Annamycin in combination with cytarabine (AnnAraC) for treating relapsed/refractory acute myeloid leukemia (R/R AML).
The trial has shown early progress with 7 subjects treated and 1 in screening. The company plans to expand to 16 additional clinical sites by August end, building to over 30 sites for Part A. Initial data from the first 45 subjects is expected in 2H 2025.
The MIRACLE study features an adaptive design with Part A randomizing 75-90 subjects across three arms, followed by Part B with approximately 220 additional subjects. Annamycin currently holds Fast Track Status and Orphan Drug Designation from both FDA and EMA.
Moleculin Biotech (Nasdaq: MBRX) ha ottenuto l'approvazione dalla RAMPA della Georgia per condurre la sperimentazione clinica MIRACLE di Fase 2B/3 sull'Annamicina in combinazione con la citarabina (AnnAraC) per il trattamento della leucemia mieloide acuta recidivante/refrattaria (R/R AML).
Lo studio ha mostrato progressi iniziali con 7 soggetti trattati e 1 in fase di screening. L'azienda prevede di espandersi a 16 siti clinici aggiuntivi entro la fine di agosto, arrivando a oltre 30 siti per la Parte A. I dati iniziali dai primi 45 soggetti sono attesi nella seconda met脿 del 2025.
Lo studio MIRACLE presenta un disegno adattativo con la Parte A che randomizza 75-90 soggetti in tre bracci, seguita dalla Parte B con circa 220 soggetti aggiuntivi. Attualmente, l'Annamicina detiene lo Status Fast Track e la Designazione di Farmaco Orfano sia dalla FDA che dall'EMA.
Moleculin Biotech (Nasdaq: MBRX) ha recibido la aprobaci贸n de la RAMPA de Georgia para llevar a cabo su ensayo cl铆nico MIRACLE de Fase 2B/3 con Annamicina en combinaci贸n con citarabina (AnnAraC) para tratar la leucemia mieloide aguda refractaria o en reca铆da (R/R AML).
El ensayo ha mostrado avances tempranos con 7 sujetos tratados y 1 en evaluaci贸n. La compa帽铆a planea expandirse a 16 sitios cl铆nicos adicionales para finales de agosto, alcanzando m谩s de 30 sitios para la Parte A. Se esperan los datos iniciales de los primeros 45 sujetos en la segunda mitad de 2025.
El estudio MIRACLE cuenta con un dise帽o adaptativo, con la Parte A que aleatoriza entre 75-90 sujetos en tres grupos, seguida por la Parte B con aproximadamente 220 sujetos adicionales. Annamicina actualmente posee estatus Fast Track y designaci贸n de medicamento hu茅rfano tanto de la FDA como de la EMA.
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MIRACLE 鞐瓣惮電� 鞝侅潙順� 靹り硠搿�, 韺岉姼 A鞐愳劀電� 75-90氇呾潣 頂柬棙鞛愲ゼ 靹� 攴鸽９鞙茧 氍挫瀾鞙� 氚办爼頃橂┌, 鞚错泟 韺岉姼 B鞐愳劀 鞎� 220氇呾潣 於旉皜 頂柬棙鞛愱皜 彀胳棳頃╇媹雼�. Annamycin鞚� 順勳灛 FDA鞕� EMA搿滊秬韯� 韺姢韸� 韸鸽灆 歆�鞙� 氚� 頋穩 鞚橃暯頀� 歆�鞝�鞚� 氚涭晿鞀惦媹雼�.
Moleculin Biotech (Nasdaq : MBRX) a re莽u l'approbation de la RAMPA de G茅orgie pour mener son essai clinique de phase 2B/3 MIRACLE portant sur l'Annamycine en combinaison avec la cytarabine (AnnAraC) pour le traitement de la leuc茅mie my茅lo茂de aigu毛 en rechute/r茅fractaire (R/R AML).
L'essai a montr茅 des progr猫s pr茅coces avec 7 sujets trait茅s et 1 en phase de s茅lection. La soci茅t茅 pr茅voit d'茅tendre l'茅tude 脿 16 sites cliniques suppl茅mentaires d'ici fin ao没t, pour atteindre plus de 30 sites pour la Partie A. Les premi猫res donn茅es des 45 premiers sujets sont attendues au second semestre 2025.
L'茅tude MIRACLE pr茅sente un design adaptatif avec une Partie A randomisant 75-90 sujets r茅partis en trois groupes, suivie d'une Partie B avec environ 220 sujets suppl茅mentaires. L'Annamycine b茅n茅ficie actuellement du statut Fast Track et de la d茅signation de m茅dicament orphelin d茅livr茅s par la FDA et l'EMA.
Moleculin Biotech (Nasdaq: MBRX) hat von Georgiens RAMPA die Zulassung erhalten, die Phase 2B/3 MIRACLE-Studie mit Annamycin in Kombination mit Cytarabin (AnnAraC) zur Behandlung von rezidivierender/refrakt盲rer akuter myeloischer Leuk盲mie (R/R AML) durchzuf眉hren.
Die Studie zeigt erste Fortschritte mit 7 behandelten Probanden und 1 in der Screening-Phase. Das Unternehmen plant, bis Ende August auf 16 zus盲tzliche Studienzentren zu erweitern und somit 眉ber 30 Zentren f眉r Teil A zu erreichen. Erste Daten von den ersten 45 Probanden werden f眉r die zweite H盲lfte 2025 erwartet.
Die MIRACLE-Studie verf眉gt 眉ber ein adaptives Design, wobei Teil A 75-90 Probanden auf drei Arme randomisiert, gefolgt von Teil B mit etwa 220 weiteren Probanden. Annamycin besitzt derzeit den Fast Track-Status und die Orphan-Drug-Zulassung sowohl von der FDA als auch von der EMA.
- Strong enrollment progress with 7 subjects treated and 1 in screening within 3 months of first site activation
- Expansion to 16 additional clinical sites by August end, scaling to 30+ sites by year-end
- Recent EU approval from EMA strengthens trial's global presence
- Fast Track Status and Orphan Drug Designation from both FDA and EMA
- Additional non-clinical GLP studies required before initiating Phase 3 portion in EU
- Complex trial design with multiple parts and interim analyses may extend timeline
Insights
Moleculin's Phase 3 AML trial expands internationally with solid enrollment progress, positioned for key data readouts in H2 2025.
Moleculin's expansion of its pivotal Phase 2B/3 MIRACLE trial into Georgia represents strategic geographic diversification of its clinical program for Annamycin (naxtarubicin). The approval from Georgia's RAMPA follows recent European Medicines Agency authorization, creating a truly global footprint spanning the US, Europe, and Middle East for this late-stage study.
The trial's current enrollment metrics are particularly encouraging - seven subjects already treated and one in screening from just a single Ukrainian site demonstrates remarkable recruitment efficiency that bodes well for meeting timelines. The planned expansion to 16 additional clinical sites by August and over 30 sites by year-end significantly strengthens the enrollment infrastructure.
The MIRACLE trial employs a sophisticated adaptive design, a modern approach that maximizes statistical power while potentially reducing total required patient numbers. The design includes two critical unblinding points - first at 45 subjects (expected H2 2025) and again at 75-90 subjects (H1 2026). This approach allows for mid-trial dose optimization between the 190 mg/m虏 and 230 mg/m虏 Annamycin arms, aligning with FDA's Project Optimus initiative for improved dose selection.
Annamycin's triple regulatory designations (Fast Track Status and Orphan Drug Designation from FDA, plus EMA Orphan status) highlight the significant unmet need in relapsed/refractory AML, particularly for venetoclax regimen failures where current treatment options yield poor outcomes. These designations provide development advantages including reduced costs and potential market exclusivity.
The trial's continued progress, particularly in challenging regions like Ukraine, demonstrates operational resilience and investigator commitment that enhances confidence in the company's ability to deliver on projected timelines for this potentially important therapy in a difficult-to-treat cancer population.
Secured approval from the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in Georgia for Phase 2B/3 MIRACLE trial聽
Recent EU approval from the European Medicines Agency (EMA) boosts trial profile
Enrollment in Part A of MIRACLE reaches seven subjects treated and one additional subject in screening
16 additional clinical sites in Europe and the US expected to begin recruitment by the end of August; building to an expected 30 plus sites for Part A of MIRACLE
Anticipated data readout of Part A remains in 2H 2025
HOUSTON, July 09, 2025 (GLOBE NEWSWIRE) -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (鈥淢oleculin鈥� or the 鈥淐ompany鈥�), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, today announced that the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in the country of Georgia has approved its Clinical Trial Application (CTA) to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine (also known as 鈥淎ra-C鈥� and for which the combination of Annamycin and Ara-C is referred to as 鈥淎nnAraC鈥�) 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 鈥淢IRACLE鈥� 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. Additionally, the Company expects the first patient to be treated in Georgia to occur by the end of August, if not sooner.
鈥淏uilding upon our recently announced CTA approval in the European Union (EU) from the European Medicines Agency (EMA), RAMPA approval of the MIRACLE trial protocol represents another important milestone and bolsters our ongoing enrollment efforts,鈥� said Walter Klemp, Chairman and CEO of Moleculin. 鈥淲e expect to add 16 additional sites in Europe and the US to MIRACLE by the end of August. This will build to more than 30 sites by year-end for Part A of the MIRACLE. Adding to this progress, our recruitment in Part A of MIRACLE has hit seven subjects treated and one in screening with just our first site in Ukraine. All of this, importantly, supports our goal to report initial data on the first 45 subjects from Part A in the second half of 2025.鈥�
Mr. Klemp continued, 鈥淭his early success in recruitment underscores the capability of our clinical sites, with the first ten screened subjects within just three months of site activation, and it showcases the commitment of ARENSIA Exploratory Medicine鈥檚 research clinic in Kyiv, Ukraine (ARENSIA). We are grateful for the continued international regulatory collaboration for this trial and believe it reflects the potential demand for Annamycin and the significant unmet need for better treatment options for R/R AML patients, especially venetoclax regimen failures where the outcomes from currently available therapies are considered dismal. Our team remains focused on driving enrollment and advancing this important program forward.鈥�
The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 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鈥檚 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 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 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鈥檚 new Project Optimus initiative.
Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on and the reference identifier there is 2024-518359-47-00.聽
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. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.
About ARENSIA Exploratory Medicine GmbH
ARENSIA Exploratory Medicine GmbH is a German operator of proprietary hospital-based research clinics, dedicated to performing complex clinical trials with novel molecules, involving patients across numerous disease areas. ARENSIA aims to speed up patients鈥� access to innovative therapies by collaborating with cutting-edge pharmaceutical companies and currently operates 13 state-of-the-art research clinics across the United States of America and Eastern Europe.
For more information about ARENSIA and its clinical operations, please visit聽.
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鈥檚 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 and connect on , and .
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 and the Company鈥檚 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鈥檚 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 鈥榖elieves,鈥� 鈥榚stimates,鈥� 鈥榓nticipates,鈥� 鈥榚xpects,鈥� 鈥榩lans,鈥� 鈥榩rojects,鈥� 鈥榠ntends,鈥� 鈥榩otential,鈥� 鈥榤ay,鈥� 鈥榗ould,鈥� 鈥榤ight,鈥� 鈥榳ill,鈥� 鈥榮hould,鈥� 鈥榓pproximately鈥� 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. 鈥淩isk Factors鈥� 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
FAQ
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