Moleculin Announces World Health Organization Approval of “naxtarubicin” as International Non-Proprietary Name for Annamycin
The World Health Organization has approved "naxtarubicin" as the international non-proprietary name for Moleculin Biotech's (NASDAQ: MBRX) drug candidate Annamycin. The drug is currently in development for treating relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases.
The company is conducting a pivotal, adaptive Phase 3 MIRACLE trial evaluating Annamycin in combination with cytarabine (AnnAraC) for AML treatment, with initial data expected in H2 2025. The global trial includes sites across the US, Europe, and the Middle East.
Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for AML treatment, plus Orphan Drug Designation for soft tissue sarcoma. The EMA has also granted Orphan Drug Designation for AML treatment.
L'Organizzazione Mondiale della Sanità ha approvato "naxtarubicina" come nome internazionale non proprietario per il candidato farmaco Annamycin di Moleculin Biotech (NASDAQ: MBRX). Il farmaco è attualmente in fase di sviluppo per il trattamento della leucemia mieloide acuta (LMA) recidivante o refrattaria e delle metastasi polmonari da sarcoma dei tessuti molli.
L'azienda sta conducendo uno studio pivotale, adattativo, Fase 3 MIRACLE che valuta Annamycin in combinazione con citarabina (AnnAraC) per il trattamento della LMA, con i primi dati attesi nella seconda metà del 2025. Lo studio globale include centri negli Stati Uniti, in Europa e in Medio Oriente.
Annamycin ha ottenuto lo status di Fast Track e la designazione di farmaco orfano dalla FDA per il trattamento della LMA, oltre alla designazione di farmaco orfano per il sarcoma dei tessuti molli. Anche l'EMA ha concesso la designazione di farmaco orfano per il trattamento della LMA.
La Organización Mundial de la Salud ha aprobado "naxtarubicina" como nombre internacional no propietario para el candidato a medicamento Annamycin de Moleculin Biotech (NASDAQ: MBRX). El fármaco se encuentra en desarrollo para el tratamiento de la leucemia mieloide aguda (LMA) en recaída o refractaria y las metástasis pulmonares de sarcoma de tejidos blandos.
La compañía está llevando a cabo un ensayo pivotal, adaptativo, Fase 3 MIRACLE que evalúa Annamycin en combinación con citarabina (AnnAraC) para el tratamiento de LMA, con datos iniciales esperados en la segunda mitad de 2025. El ensayo global incluye centros en Estados Unidos, Europa y Medio Oriente.
Annamycin ha recibido el estatus de Fast Track y la designación de medicamento huérfano por parte de la FDA para el tratamiento de LMA, además de la designación de medicamento huérfano para el sarcoma de tejidos blandos. La EMA también ha otorgado la designación de medicamento huérfano para el tratamiento de LMA.
세계보건기구(WHO)가 Moleculin Biotech(NASDAQ: MBRX)의 약물 후보 Annamycin의 국제 비독점명으로 "나크사루비신(naxtarubicin)"을 승인했습니다. 이 약물은 재발성 또는 난치성 급성 골수성 백혈병(AML) 및 연부조직 육종 폐 전이 치료를 위해 개발 중입니다.
회사는 AML 치료를 위해 사이타라빈과 병용하는 Annamycin(AnnAraC)을 평가하는 중대한 적응형 3상 MIRACLE 임상시험을 진행 중이며, 초기 데이터는 2025년 하반기에 발표될 예정입니다. 이 글로벌 임상시험은 미국, 유럽, 중동의 여러 기관에서 진행됩니다.
Annamycin은 FDA로부터 AML 치료를 위한 패스트 트랙 지정과 희귀의약품 지정을 받았으며, 연부조직 육종에 대해서도 희귀의약품 지정을 받았습니다. EMA 역시 AML 치료를 위한 희귀의약품 지정을 승인했습니다.
L'Organisation mondiale de la santé a approuvé "naxtarubicine" comme nom international non propriétaire pour le candidat médicament Annamycin de Moleculin Biotech (NASDAQ : MBRX). Ce médicament est actuellement en développement pour le traitement de la leucémie myéloïde aiguë (LMA) récurrente ou réfractaire et des métastases pulmonaires du sarcome des tissus mous.
L'entreprise mène un essai pivot adaptatif de phase 3 MIRACLE évaluant Annamycin en combinaison avec la cytarabine (AnnAraC) pour le traitement de la LMA, avec des données initiales attendues au second semestre 2025. L'essai mondial inclut des sites aux États-Unis, en Europe et au Moyen-Orient.
Annamycin a reçu le statut Fast Track et la désignation de médicament orphelin de la FDA pour le traitement de la LMA, ainsi que la désignation de médicament orphelin pour le sarcome des tissus mous. L'EMA a également accordé la désignation de médicament orphelin pour le traitement de la LMA.
Die Weltgesundheitsorganisation hat "Naxtarubicin" als internationalen Freinamen für den Arzneimittelkandidaten Annamycin von Moleculin Biotech (NASDAQ: MBRX) genehmigt. Das Medikament befindet sich derzeit in der Entwicklung zur Behandlung von rezidivierender oder refraktärer akuter myeloischer Leukämie (AML) und Lungenmetastasen von Weichteilsarkomen.
Das Unternehmen führt eine entscheidende, adaptive Phase-3-MIRACLE-Studie durch, in der Annamycin in Kombination mit Cytarabin (AnnAraC) zur AML-Behandlung untersucht wird. Erste Daten werden für die zweite Hälfte des Jahres 2025 erwartet. Die globale Studie umfasst Standorte in den USA, Europa und dem Nahen Osten.
Annamycin erhielt von der FDA den Fast Track Status und die Orphan Drug Designation für die AML-Behandlung sowie die Orphan Drug Designation für Weichteilsarkome. Auch die EMA hat die Orphan Drug Designation für die AML-Behandlung erteilt.
- Drug has received multiple regulatory designations including Fast Track Status and Orphan Drug Designation from FDA and EMA
- Phase 3 MIRACLE trial is progressing with global reach across US, Europe, and Middle East
- Initial data readout from Phase 3 trial expected in H2 2025
- Drug shows potential for multiple indications including AML and soft tissue sarcoma
- None.
Insights
WHO's approval of "naxtarubicin" as Annamycin's international name marks regulatory progress as Moleculin advances its Phase 3 AML trial toward H2 2025 data readout.
The World Health Organization's approval of "naxtarubicin" as the International Non-Proprietary Name (INN) for Moleculin's Annamycin represents a standard but necessary procedural milestone in drug development. This INN designation ensures the drug will have a unique, globally recognized name for regulatory filings, scientific literature, and eventual prescribing—a prerequisite for any pharmaceutical product approaching potential commercialization.
More significant than the naming approval is confirmation that Moleculin's pivotal Phase 3 MIRACLE trial is actively progressing. This adaptive trial design, evaluating Annamycin in combination with cytarabine (called AnnAraC) for relapsed/refractory acute myeloid leukemia (AML), has commenced patient dosing with an interim data readout expected in H2 2025. The adaptive design offers flexibility to modify certain trial parameters based on interim results, potentially enabling more efficient development.
Annamycin's regulatory designations are particularly noteworthy: Fast Track Status and Orphan Drug Designation from the FDA for AML treatment, additional Orphan Drug Designation for soft tissue sarcoma from the FDA, and Orphan Drug Designation for AML from the European Medicines Agency. These designations can provide tangible benefits including reduced development costs, expedited review processes, and market exclusivity periods if eventually approved.
Moleculin positions Annamycin as a "next-generation anthracycline" with "potential to be safer and more effective than current prescribed anthracyclines." Anthracyclines are a well-established class of chemotherapy drugs, but their clinical utility is often limited by cumulative dose-related cardiotoxicity. The company's global trial footprint—spanning sites across the US, Europe, and Middle East—suggests positioning for potential worldwide commercialization if trials prove successful.
For investors tracking Moleculin's progress, this announcement primarily serves to confirm that development remains on schedule, with the Phase 3 trial proceeding as planned toward the anticipated H2 2025 data milestone.
Annamycin has the potential to be safer and more effective than current prescribed anthracyclines to treat serious, hard-to-treat cancers
Ongoing pivotal, adaptive Phase 3 clinical trial (the “MIRACLE” trial) evaluating Annamycin for the treatment of acute myeloid leukemia (AML); Interim data readout expected in the second half of 2025
HOUSTON, May 06, 2025 (GLOBE NEWSWIRE) -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company”), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, today announced that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization approved “naxtarubicin1” for the non-proprietary name of the Company’s next-generation anthracycline in development, Annamycin. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases and the Company believes it may have the potential to treat additional indications.
“The assignment of the non-proprietary name represents an important step in the development and potential future commercialization of Annamycin. The INN naming process meticulously evaluates proposed drug names for adherence to nomenclature guidelines and potential conflicts, followed by expert consensus and public review. With this INN now given and prior approval by the United States Adopted Names (USAN), we have the ability to establish a universally recognized and conflict-free nonproprietary drug name for Annamycin,” said Walter Klemp, Chairman and CEO of Moleculin. “Looking ahead, our team remains focused on the successful execution of our ongoing pivotal, adaptive design Phase 3 MIRACLE trial of Annamycin for the treatment R/R AML and look forward to reporting initial data in the second half of 2025.”
Commonly referred to as a generic name, each INN is a unique name used to identify pharmaceutical substances or active pharmaceutical ingredients. Each active substance that is to be marketed as a pharmaceutical must be granted a unique name of worldwide acceptability to ensure the clear identification, safe prescription and dispensing of medicines to patients. Nonproprietary names are intended for wide use ranging from labelling and product information to drug regulation and scientific literature. Moleculin expects to transition to the use of naxtarubicin in the near-term.
The Company is currently evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia in the pivotal, adaptive design Phase 3 MIRACLE trial (MB-108). This Phase 3 “MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global trial, including sites in the US, Europe and the Middle East. 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 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 highly efficacious and well tolerated 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.
In March of this year, the Company initiated recruitment of patients into its 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 initial data readout in the MIRACLE trial. 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
1 WHO Drug Information, Vol. 39, No. 1, 2025, pg. 217
FAQ
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