Moleculin Biotech, Inc. Unveils Promising Preclinical Data of Annamycin in Liver Cancer Treatment
Moleculin Biotech (NASDAQ:MBRX) has presented promising preclinical data for its lead drug candidate Annamycin in treating various liver cancers. The drug demonstrated significant efficacy against hepatocellular carcinoma (HCC), colorectal liver metastases, and pancreatic ductal adenocarcinoma (PDAC) liver metastases.
Key findings show that Annamycin achieves higher concentrations in the liver, spleen, lungs, and pancreas compared to doxorubicin. The drug exhibited strong anti-tumor activity in multiple cancer models while maintaining a favorable safety profile with low or no cardiotoxicity. Notably, 32 out of 42 subjects previously received above FDA's lifetime maximum allowable anthracycline level without cardiotoxicity evidence.
Annamycin is currently in late-stage clinical development for AML treatment, with preliminary data expected in 2H 2025.
Moleculin Biotech (NASDAQ:MBRX) ha presentato dati preclinici promettenti per il suo principale candidato farmaco Annamycin nel trattamento di diversi tumori del fegato. Il farmaco ha mostrato un'efficacia significativa contro il carcinoma epatocellulare (HCC), le metastasi epatiche da carcinoma colorettale e le metastasi epatiche da adenocarcinoma duttale pancreatico (PDAC).
I risultati chiave evidenziano che Annamycin raggiunge concentrazioni più elevate nel fegato, milza, polmoni e pancreas rispetto alla doxorubicina. Il farmaco ha dimostrato una forte attività antitumorale in vari modelli di cancro, mantenendo un profilo di sicurezza favorevole con bassa o assente cardiotossicità. In particolare, 32 su 42 soggetti avevano precedentemente ricevuto dosi superiori al limite massimo consentito dall'FDA per gli antraciclinici senza evidenza di cardiotossicità.
Annamycin è attualmente in fase avanzata di sviluppo clinico per il trattamento della leucemia mieloide acuta (AML), con dati preliminari attesi nella seconda metà del 2025.
Moleculin Biotech (NASDAQ:MBRX) ha presentado datos preclínicos prometedores para su principal candidato a fármaco Annamycin en el tratamiento de varios tipos de cáncer de hígado. El medicamento demostró una eficacia significativa contra el carcinoma hepatocelular (HCC), las metástasis hepáticas de cáncer colorrectal y las metástasis hepáticas del adenocarcinoma ductal pancreático (PDAC).
Los hallazgos clave muestran que Annamycin alcanza concentraciones más altas en el hígado, bazo, pulmones y páncreas en comparación con la doxorrubicina. El medicamento exhibió una fuerte actividad antitumoral en múltiples modelos de cáncer, manteniendo un perfil de seguridad favorable con baja o nula cardiotoxicidad. Cabe destacar que 32 de 42 sujetos habían recibido previamente dosis superiores al máximo permitido por la FDA para antraciclinas sin evidencia de cardiotoxicidad.
Annamycin se encuentra actualmente en desarrollo clínico avanzado para el tratamiento de la leucemia mieloide aguda (AML), con datos preliminares esperados en la segunda mitad de 2025.
Moleculin Biotech (NASDAQ:MBRX)는 다양한 간암 치료를 위한 주요 약물 후보인 Annamycin의 유망한 전임상 데이터를 발표했습니다. 이 약물은 간세포암(HCC), 대장암 간 전이 및 췌장관 선암(PDAC) 간 전이에 대해 상당한 효능을 보였습니다.
주요 결과에 따르면 Annamycin은 독소루비신에 비해 간, 비장, 폐, 췌장에서 더 높은 농도를 달성합니다. 이 약물은 여러 암 모델에서 강력한 항종양 활성을 나타냈으며, 심장 독성이 낮거나 없는 우수한 안전성 프로필을 유지했습니다. 특히, 42명 중 32명이 FDA가 정한 생애 최대 허용량 이상의 안트라사이클린을 이전에 투여받았음에도 심장 독성 증거가 없었습니다.
Annamycin은 현재 급성 골수성 백혈병(AML) 치료를 위한 후기 임상 개발 단계에 있으며, 2025년 하반기에 예비 데이터가 발표될 예정입니다.
Moleculin Biotech (NASDAQ:MBRX) a présenté des données précliniques prometteuses pour son principal candidat-médicament Annamycin dans le traitement de divers cancers du foie. Le médicament a démontré une efficacité significative contre le carcinome hépatocellulaire (CHC), les métastases hépatiques du cancer colorectal et les métastases hépatiques de l'adénocarcinome canalaire pancréatique (PDAC).
Les résultats clés montrent qu'Annamycin atteint des concentrations plus élevées dans le foie, la rate, les poumons et le pancréas par rapport à la doxorubicine. Le médicament a montré une forte activité antitumorale dans plusieurs modèles de cancer tout en maintenant un profil de sécurité favorable avec une cardiotoxicité faible ou inexistante. Notamment, 32 des 42 sujets avaient déjà reçu des doses supérieures au maximum à vie autorisé par la FDA pour les anthracyclines sans preuve de cardiotoxicité.
Annamycin est actuellement en phase avancée de développement clinique pour le traitement de la leucémie myéloïde aiguë (LMA), avec des données préliminaires attendues au second semestre 2025.
Moleculin Biotech (NASDAQ:MBRX) hat vielversprechende präklinische Daten für seinen führenden Wirkstoffkandidaten Annamycin bei der Behandlung verschiedener Leberkrebsarten vorgestellt. Das Medikament zeigte eine signifikante Wirksamkeit gegen hepatozelluläres Karzinom (HCC), kolorektale Lebermetastasen und Lebermetastasen des pankreatischen duktalen Adenokarzinoms (PDAC).
Wichtige Erkenntnisse zeigen, dass Annamycin höhere Konzentrationen in Leber, Milz, Lunge und Bauchspeicheldrüse erreicht als Doxorubicin. Das Medikament zeigte starke antitumorale Aktivität in mehreren Krebsmodellen und behielt ein günstiges Sicherheitsprofil mit geringer oder keiner Kardiotoxizität bei. Bemerkenswert ist, dass 32 von 42 Probanden zuvor eine Dosis oberhalb des von der FDA erlaubten lebenslangen Höchstwerts von Anthrazyklinen erhalten hatten, ohne Anzeichen von Kardiotoxizität.
Annamycin befindet sich derzeit in der späten klinischen Entwicklungsphase für die Behandlung von AML, mit vorläufigen Daten, die für die zweite Hälfte des Jahres 2025 erwartet werden.
- Strong efficacy demonstrated in multiple liver cancer models including HCC and metastatic cancers
- Higher drug concentration achieved in target organs compared to existing treatment (doxorubicin)
- Favorable safety profile with no cardiotoxicity, unlike traditional anthracyclines
- Potential to treat multiple types of cancers beyond current development programs
- Still in preclinical stage for liver cancer indications
- Clinical validation in humans for liver cancer applications yet to be demonstrated
Insights
Moleculin's Annamycin shows promising preclinical efficacy against liver cancers with targeted accumulation and favorable safety profile versus traditional anthracyclines.
Moleculin's preclinical data for Annamycin demonstrates a significant therapeutic opportunity in liver-based cancers through its unique organotropic properties. The drug shows preferential accumulation in the liver, spleen, lungs, and pancreas compared to standard doxorubicin – a critical advantage for treating liver-localized tumors.
The efficacy data across three distinct liver cancer models is particularly compelling. In orthotopic hepatocellular carcinoma models, Annamycin showed marked tumor reduction and improved survival. Similarly positive results were observed in colorectal liver metastasis models – significant given that nearly 50% of colorectal cancer patients develop liver metastases. The drug also demonstrated efficacy against pancreatic cancer liver metastases, a notoriously treatment-resistant condition.
What truly differentiates Annamycin is its favorable cardiotoxicity profile. Traditional anthracyclines like doxorubicin are limited by cumulative, dose-dependent cardiac damage. Annamycin's apparent lack of cardiotoxicity – even when administered above FDA-established lifetime maximum anthracycline levels – could position it as the first non-cardiotoxic anthracycline to reach approval, allowing for extended treatment cycles without the typical anthracycline-associated heart damage.
While these remain preclinical findings, they complement Moleculin's existing clinical programs in AML and soft tissue sarcoma lung metastases, potentially expanding Annamycin's therapeutic applications considerably if these results translate to humans.
Presented data highlights the potential of Annamycin for treating broad range of human cancers, including high-need oncology indications
Annamycin currently in late-stage clinical development in combination with cytarabine for the treatment of AML; Anticipated preliminary data readout in 2H 2025
HOUSTON, Aug. 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 tumors and viruses, today announced the presentation of encouraging preclinical data for its lead drug candidate, Annamycin, also known by its non-proprietary name of naxtarubicin, which demonstrated significant efficacy against various primary and metastatic liver cancers, including hepatocellular carcinoma (HCC), colorectal liver metastases, and pancreatic ductal adenocarcinoma (PDAC) liver metastases. This is believed to be the result of targeted accumulation in the liver and other organs.
These findings were highlighted in a poster titled "Liposomal Annamycin (L-ANN) Efficacy Against Primary and Metastatic Liver Cancers," presented by Dr. Waldemar Priebe, Lead Author and Chairman of the Scientific Advisory Board at Moleculin at the recently held Shelby-Lavine Pancreatic Cancer Symposium at MD Anderson Cancer Center.
Key Highlights
- Targeted Accumulation in Organs: The preclinical studies confirmed that Annamycin exhibits distinct organotropic properties, leading to significantly higher concentrations in the liver, spleen, lungs, and pancreas when compared to doxorubicin (DOX). This targeted accumulation is critical for effectively treating liver-localized tumors.
- Efficacy in Orthotopic Hepatocellular Carcinoma (HCC) Models: Annamycin demonstrated excellent anti-tumor activity in orthotopic HCC models (Hepa 1-6 Luc), showing a marked reduction in tumor progression and improved survival rates in treated animals compared to vehicle controls.
- Potent Impact on Colorectal Liver Metastasis: In an experimental liver metastatic model of colorectal carcinoma (CT26 Luc), Annamycin significantly inhibited metastatic growth and extended survival, highlighting its potential for addressing liver metastases in colon cancer that affects close to
50% of patients. - Promising Results in Pancreatic Cancer Liver Metastasis: Annamycin also showed compelling efficacy in liver-implanted human pancreatic ductal adenocarcinoma (MIA PaCa-2), leading to inhibition of tumor growth, suggesting its potential role in managing advanced pancreatic cancer with liver involvement.
- Favorable Safety Profile: Consistent with previous preclinical and clinical findings, Annamycin continued to show low or no cardiotoxicity, a significant advantage over traditional anthracyclines like doxorubicin, which are often limited by dose-dependent cardiac side effects. This safety profile was previously observed in clinical trials, where 32 out of 42 subjects reviewed received more than the FDA-established lifetime maximum allowable level of anthracycline without evidence of cardiotoxicity.
“We are incredibly encouraged by these preclinical results, which further validate Annamycin’s potential as a powerful and differentiated therapeutic agent for liver cancers,” said Dr. Priebe. “The ability of Annamycin to concentrate effectively in the liver, combined with its demonstrated efficacy across multiple aggressive liver cancer models and its favorable cardiotoxicity profile, positions it as a highly promising candidate for patients facing these devastating diseases. We believe these data provide a strong foundation for advancing Annamycin in clinical development for these indications, offering new hope where treatment options are often limited.”
Walter Klemp, Chairman and CEO of Moleculin, added, “We continue to be strongly encouraged by the potential of Annamycin. Beyond our preliminary programs in AML and soft tissue sarcoma lung metastases (STS lung mets), we continue to advance and develop Annamycin through multiple investigator-initiated studies to further unlock its potential as a treatment option for many other types of cancers. We believe this preclinical data highlights that potential and provides additional validation of its unique pharmacological profile and importantly showcases the opportunity for its use across a wide range of cancers.”
Moleculin’s novel drug candidate is being positioned to become the first ever non-cardiotoxic anthracycline to be approved and is currently being developed for the treatment of AML and STS lung mets. For more information, please visit moleculin.com.
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 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 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 whether the preclinical results described above are able to be reproduced in clinical trials. 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
FAQ
What are the key findings from Moleculin's (MBRX) Annamycin preclinical study in liver cancer?
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What distinguishes Moleculin's (MBRX) Annamycin from traditional anthracyclines?
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