New Findings Show Moleculin's Annamycin Overcomes Resistance to Venetoclax in AML
Moleculin Biotech (MBRX) announced new findings showing Annamycin's effectiveness in treating Venetoclax-resistant acute myeloid leukemia (AML). Preliminary clinical results demonstrate that Annamycin plus Ara-C achieved a 60% CR/CRi rate in subjects previously treated with Venetoclax regimens, which is more than 4 times higher than historical rates. The MB-106 trial showed median overall survival of 11.6 months in subjects receiving AnnAraC as second-line therapy. The company is advancing to a Phase 3 'MIRACLE' trial, with patient treatment expected to begin in Q1 2025. Annamycin has received Fast Track Status and Orphan Drug Designation from both FDA and EMA.
Moleculin Biotech (MBRX) ha annunciato nuove scoperte che mostrano l'efficacia di Annamycin nel trattamento della leucemia mieloide acuta (AML) resistente a Venetoclax. I risultati clinici preliminari dimostrano che Annamycin più Ara-C ha ottenuto un tasso di CR/CRi del 60% nei soggetti precedentemente trattati con regimi a base di Venetoclax, che è più di 4 volte superiore ai tassi storici. Il trial MB-106 ha mostrato una sopravvivenza globale mediana di 11,6 mesi nei soggetti che hanno ricevuto AnnAraC come terapia di seconda linea. L'azienda sta avanzando verso un trial di Fase 3 denominato 'MIRACLE', con l'inizio del trattamento per i pazienti previsto nel primo trimestre del 2025. Annamycin ha ricevuto lo Statuto di Fast Track e la Designazione di Farmaco Orfano da sia FDA che EMA.
Moleculin Biotech (MBRX) anunció nuevos hallazgos que demuestran la efectividad de Annamycin en el tratamiento de leucemia mieloide aguda (LMA) resistente a Venetoclax. Los resultados clínicos preliminares demuestran que Annamycin más Ara-C lograron un tasa de CR/CRi del 60% en sujetos previamente tratados con regímenes de Venetoclax, lo que es más de 4 veces superior a las tasas históricas. El ensayo MB-106 mostró una supervivencia general media de 11,6 meses en los sujetos que recibieron AnnAraC como terapia de segunda línea. La empresa está avanzando hacia un ensayo de Fase 3 llamado 'MIRACLE', con el inicio del tratamiento de pacientes previsto para el primer trimestre de 2025. Annamycin ha recibido el estado de Fast Track y la designación de Medicamento Huérfano tanto de la FDA como de la EMA.
몰레큘린 바이오텍(MBRX)은 아나마이신이 베네토클락스 내성 급성 골수 백혈병(AML) 치료에 효과적이라는 새로운 발견을 발표했습니다. 초기 임상 결과에 따르면 아나마이신과 아라-C의 조합은 베네토클락스 요법으로 이전에 치료받던 환자에서 60% CR/CRi 비율을 달성했으며, 이는 역사적 비율보다 4배 이상 높은 수치입니다. MB-106 시험에서는 중간 전체 생존 기간이 11.6개월로 나타났습니다. 이 시험은 AnneAraC를 두 번째 치료제로 받은 환자에서 진행되었습니다. 회사는 'MIRACLE'라는 3상 시험으로 나아가고 있으며, 환자 치료는 2025년 1분기 중 시작될 예정입니다. 아나마이신은 FDA와 EMA 모두로부터 신속 심사(패스트 트랙) 및 오르팜 약물 지정 상태를 받았습니다.
Moleculin Biotech (MBRX) a annoncé de nouvelles découvertes montrant l'efficacité d'Annamycin dans le traitement de la leucémie myéloïde aiguë (LMA) résistante au Venetoclax. Les résultats cliniques préliminaires montrent qu'Annamycin associé à Ara-C a atteint un taux de CR/CRi de 60% chez des sujets ayant reçu des traitements à base de Venetoclax, ce qui est plus de 4 fois supérieur aux taux historiques. L'essai MB-106 a montré une survie globale médiane de 11,6 mois chez des sujets recevant AnnAraC comme traitement de deuxième ligne. L'entreprise progresse vers un essai de Phase 3 appelé 'MIRACLE', avec un début de traitement des patients prévu pour le premier trimestre de 2025. Annamycin a reçu le statut Fast Track et la désignation de médicament orphan par la FDA et l'EMA.
Moleculin Biotech (MBRX) hat neue Erkenntnisse vorgestellt, die die Wirksamkeit von Annamycin bei der Behandlung von venetoclax-resistenter akuter myeloischer Leukämie (AML) zeigen. Vorläufige klinische Ergebnisse zeigen, dass Annamycin plus Ara-C eine CR/CRi-Rate von 60% bei Probanden mit zuvor durchgeführten Venetoclax-Regimen erreicht hat, was mehr als 4-mal höher als die historischen Raten ist. Die MB-106-Studie ergab eine mediane Gesamtüberlebenszeit von 11,6 Monaten bei Probanden, die AnnAraC als Zweitlinientherapie erhielten. Das Unternehmen geht in eine Phase-3-Studie mit dem Namen 'MIRACLE', wobei die Behandlung der Patienten im ersten Quartal 2025 beginnen soll. Annamycin hat von beiden, der FDA und der EMA, den Status 'Fast Track' und die Bezeichnung 'Orphan Drug' erhalten.
- 60% CR/CRi rate in Venetoclax-resistant patients, 4x higher than historical rates
- 11.6 months median overall survival in second-line therapy patients
- Fast Track Status and Orphan Drug Designation from FDA and EMA
- Phase 3 trial initiation on track for Q1 2025
- None.
Insights
The preclinical and clinical data for Annamycin shows remarkable promise in treating Venetoclax-resistant AML patients. The 60% CR/CRi rate in previously Venetoclax-treated patients is particularly significant, as it's over 4x higher than the historical
The upcoming Phase 3 MIRACLE trial could be transformative for MBRX, especially given Annamycin's Fast Track Status and Orphan Drug Designations. With treatment initiation planned for Q1 2025 and unblinded data expected for the first 45 subjects within 12 months, this represents a critical catalyst for the company's
Preclinical data accepted for online publication at ASH Annual Meeting reveal significant activity of Annamycin in Venetoclax resistant AML model
New preliminary clinical results show Annamycin plus Ara-C achieved
Annamycin demonstrates an even greater potential than previously reported to address a significant AML patient population for which treatment options are extremely limited
New data from MB-106 trial show median overall survival of 11.6 months in subjects receiving AnnAraC as 2nd line therapy
Jorge Cortes, MD, Director of the Georgia Cancer Center at Augusta University and a member of the Company's Scientific Advisory Board, commented, "Although Venetoclax has been an important improvement in first line therapy for AML patients who are unfit for intensive chemotherapy, the rate of relapse is high and overall survival post relapse is just a few months. This turns out to be a large percentage of AML patients in total and we clearly need a better treatment option."
Michael Andreeff, MD, PhD, Professor of Medicine, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center and a member of the Company's Scientific Advisory Board, added, "Many patients get Ven-Aza, not because they are 'unfit' but because of the high initial response rates. When they relapse, however, our options are very limited. Annamycin combined with Ara-C could significantly advance the standard of care and provide better outcomes for these high-risk patients. I am excited to be a part of the next step in the development of this important asset."
A prior publication, Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and venetoclax regimens1, reported that the CR/CRi2 rate for salvage therapy using available standard of care in AML subjects who relapsed from or were refractory to Venetoclax regimens was
An abstract titled, "Annamycin, a non-cardiotoxic anthracycline, demonstrates unique organotropism and activity against Ara-C and Venetoclax resistant AML," supporting the clinical activity of Annamycin was accepted for online publication at the ASH Annual Meeting being held December 7-10, 2024, in
Additionally, new preliminary data from the Company's Phase 1B/2 clinical trial evaluating AnnAraC for the treatment of subjects with AML as both first line therapy and for subjects who were refractory to or relapsed after induction therapy (MB-106) demonstrated median overall survival ("OS") of 9.1 months for subjects with a wide range of (0-6) prior lines of therapy (n=22) and 11.6 months (n=10) for subjects with 1 prior line of therapy (second line therapy).
"Moleculin is focusing on development of Annamycin to address the significant unmet need in R/R AML. The growing body of preliminary data continue to bolster our confidence in the safety and efficacy of Annamycin, and its potential to provide patients and physicians with a promising new treatment option in AML," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We believe the latest preliminary OS data we are seeing in our MB-106 trial can now be considered exceptional and we look forward to the initiation of our pivotal registration study, especially now that our recent protocol amendment allows for disclosing unblinded data for the first 45 subjects, which we expect within the next 12 months."
The Company is advancing the development of Annamycin in a Phase 3 pivotal trial evaluating AnnAraC for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US. The Company remains on track to initiate patient treatment in the first quarter of 2025.
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
1 A. Maiti, C. Rausch, J. Cortes, Et al, "Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and venetoclax regimens, Haematologica online, vol. 106 No.3 (2021)
2 CR = complete remission; CRi = complete remission with incomplete hematologic recovery
View original content to download multimedia:https://www.prnewswire.com/news-releases/new-findings-show-moleculins-annamycin-overcomes-resistance-to-venetoclax-in-aml-302307821.html
SOURCE Moleculin Biotech, Inc.
FAQ
What are the latest clinical results for Moleculin's (MBRX) Annamycin in AML treatment?
When will Moleculin (MBRX) begin its Phase 3 MIRACLE trial for Annamycin?
What regulatory designations has Moleculin's (MBRX) Annamycin received?
