Mustang Bio Granted Orphan Drug Designation by U.S. FDA for MB-101 (IL13Ra2-targeted CAR T-cells) to Treat Astrocytomas and Glioblastoma
Mustang Bio (NASDAQ:MBIO) has received Orphan Drug Designation from the FDA for MB-101, its IL13Ra2-targeted CAR T-cell therapy, for treating astrocytomas and glioblastoma. In an ongoing Phase 1 trial, MB-101 demonstrated promising results with 50% of patients achieving stable disease or better, including two partial responses and two complete responses lasting 7.5 and 66+ months.
The company plans to combine MB-101 with MB-108 (HSV-1 oncolytic virus), which previously received Orphan Drug Designation for malignant glioma treatment. The combination therapy aims to improve efficacy by using MB-108 to transform "cold" tumors into "hot" tumors, potentially enhancing MB-101's effectiveness. Phase 1 trials for both therapies continue at City of Hope and The University of Alabama at Birmingham, though further development requires additional funding or strategic partnership.
Mustang Bio (NASDAQ:MBIO) ha ottenuto la Designazione di Farmaco Orfano dalla FDA per MB-101, la sua terapia CAR T-cell mirata a IL13Ra2, destinata al trattamento di astrocitomi e glioblastoma. In uno studio di Fase 1 in corso, MB-101 ha mostrato risultati promettenti con il 50% dei pazienti che ha raggiunto una malattia stabile o migliore, inclusi due risposte parziali e due risposte complete della durata di 7,5 e oltre 66 mesi.
L'azienda prevede di combinare MB-101 con MB-108 (virus oncolitico HSV-1), che in precedenza ha ricevuto la Designazione di Farmaco Orfano per il trattamento del glioma maligno. La terapia combinata mira a migliorare l'efficacia utilizzando MB-108 per trasformare i tumori "freddi" in tumori "caldi", potenzialmente aumentando l'efficacia di MB-101. Gli studi di Fase 1 per entrambe le terapie proseguono presso il City of Hope e l'Universit脿 dell'Alabama a Birmingham, anche se lo sviluppo ulteriore richiede finanziamenti aggiuntivi o una partnership strategica.
Mustang Bio (NASDAQ:MBIO) ha recibido la Designaci贸n de Medicamento Hu茅rfano por parte de la FDA para MB-101, su terapia CAR T dirigida a IL13Ra2, para el tratamiento de astrocitomas y glioblastomas. En un ensayo de Fase 1 en curso, MB-101 mostr贸 resultados prometedores con el 50% de los pacientes logrando enfermedad estable o mejor, incluyendo dos respuestas parciales y dos respuestas completas que duraron 7.5 y m谩s de 66 meses.
La compa帽铆a planea combinar MB-101 con MB-108 (virus oncol铆tico HSV-1), que previamente recibi贸 la Designaci贸n de Medicamento Hu茅rfano para el tratamiento de glioma maligno. La terapia combinada busca mejorar la eficacia usando MB-108 para transformar tumores "fr铆os" en tumores "calientes", potencialmente aumentando la efectividad de MB-101. Los ensayos de Fase 1 para ambas terapias contin煤an en City of Hope y la Universidad de Alabama en Birmingham, aunque el desarrollo adicional requiere financiamiento o una asociaci贸n estrat茅gica.
Mustang Bio (NASDAQ:MBIO)電� IL13Ra2毳� 響滌爜鞙茧 頃橂姅 CAR T 靹疙彫 旃橂鞝� MB-101鞐� 雽�頃� FDA搿滊秬韯� 頋穩鞚橃暯頀� 歆�鞝曥潉 氚涭晿鞀惦媹雼�. 鞚措姅 靹膘儊靹疙彫膦呹臣 甑愲靹疙彫膦� 旃橂毳� 鞙勴暅 瓴冹瀰雼堧嫟. 歆勴枆 欷戩澑 1靸� 鞛勳儊鞁滍棙鞐愳劀 MB-101鞚� 頇橃瀽鞚� 50%臧� 鞎堨爼霅� 歆堩櫂 靸來儨 鞚挫儊鞚� 雼劚頄堨溂氅�, 攵�攵� 氚橃潙 2瓯搓臣 7.5臧滌洈 氚� 66臧滌洈 鞚挫儊 歆�靻嶋悳 鞕勳爠 氚橃潙 2瓯挫潉 韽暔頃╇媹雼�.
須岇偓電� 鞚挫爠鞐� 鞎呾劚 甑愲靹疙彫膦� 旃橂毳� 鞙勴暣 頋穩鞚橃暯頀� 歆�鞝曥潉 氚涭潃 MB-108(HSV-1 膦呾枒鞖╉暣 氚旍澊霟姢)瓿� MB-101鞚� 氤戩毄頃� 瓿勴殟鞛呺媹雼�. 鞚� 氤戩毄 鞖旊矔鞚� MB-108鞚� 靷毄頃� '雰夒嫶頃�' 膦呾枒鞚� '頇滌劚頇旊悳' 膦呾枒鞙茧 鞝勴櫂鞁滌紲 MB-101鞚� 須臣毳� 雴掛澊電� 瓴冹潉 氇╉憸搿� 頃╇媹雼�. 霊� 旃橂鞝滌棎 雽�頃� 1靸� 鞛勳儊鞁滍棙鞚� City of Hope鞕� 鞎澕氚半 雽�頃欔祼 氩勲皪頄勳棎靹� 瓿勳啀 歆勴枆 欷戩澊雮�, 於旉皜 臧滊皽鞚� 鞙勴暣靹滊姅 於旉皜 鞛愱笀 臁半嫭鞚措倶 鞝勲灥鞝� 韺岉姼雱堨嫮鞚� 頃勳殧頃╇媹雼�.
Mustang Bio (NASDAQ:MBIO) a obtenu la d茅signation de m茅dicament orphelin de la FDA pour MB-101, sa th茅rapie CAR T ciblant IL13Ra2, destin茅e au traitement des astrocytomes et glioblastomes. Lors d'un essai de phase 1 en cours, MB-101 a montr茅 des r茅sultats prometteurs avec 50 % des patients ayant atteint une maladie stable ou mieux, incluant deux r茅ponses partielles et deux r茅ponses compl猫tes durant 7,5 et plus de 66 mois.
L'entreprise pr茅voit de combiner MB-101 avec MB-108 (virus oncolytique HSV-1), qui avait d茅j脿 re莽u la d茅signation de m茅dicament orphelin pour le traitement du gliome malin. Cette th茅rapie combin茅e vise 脿 am茅liorer l'efficacit茅 en utilisant MB-108 pour transformer les tumeurs "froides" en tumeurs "chaudes", ce qui pourrait renforcer l'efficacit茅 de MB-101. Les essais de phase 1 pour les deux th茅rapies se poursuivent au City of Hope et 脿 l'Universit茅 de l'Alabama 脿 Birmingham, bien que le d茅veloppement ult茅rieur n茅cessite un financement suppl茅mentaire ou un partenariat strat茅gique.
Mustang Bio (NASDAQ:MBIO) hat von der FDA die Orphan-Drug-Designation f眉r MB-101 erhalten, seine IL13Ra2-gerichtete CAR-T-Zelltherapie zur Behandlung von Astrozytomen und Glioblastomen. In einer laufenden Phase-1-Studie zeigte MB-101 vielversprechende Ergebnisse mit 50 % der Patienten, die eine stabile Erkrankung oder besser erreichten, darunter zwei partielle und zwei komplette Remissionen, die 7,5 bzw. 眉ber 66 Monate anhielten.
Das Unternehmen plant, MB-101 mit MB-108 (HSV-1 onkolytischer Virus) zu kombinieren, das zuvor die Orphan-Drug-Designation f眉r die Behandlung von malignen Gliomen erhalten hat. Die Kombinationstherapie soll die Wirksamkeit verbessern, indem MB-108 "kalte" Tumoren in "hei脽e" Tumoren verwandelt und somit die Effektivit盲t von MB-101 potenziell steigert. Die Phase-1-Studien f眉r beide Therapien laufen weiterhin am City of Hope und der University of Alabama in Birmingham, wobei eine weitere Entwicklung zus盲tzliche Finanzierung oder eine strategische Partnerschaft erfordert.
- None.
- Additional funding or strategic partnership required for further development
- Complete responses only observed in small subset (3 patients) with 'hot' tumors
- Still in early Phase 1 trials with limited patient data
Insights
FDA's Orphan Drug Designation for MB-101 strengthens Mustang Bio's position in developing treatments for rare brain cancers with promising early clinical results.
The FDA's grant of Orphan Drug Designation (ODD) to Mustang Bio's MB-101 for treating astrocytomas and glioblastoma represents a significant regulatory milestone with strategic advantages. This designation provides critical benefits including tax credits, user fee waivers, and鈥攎ost importantly鈥�seven years of market exclusivity upon approval, independent of patent protection.
The clinical data supporting MB-101 is particularly noteworthy. In a Phase 1 trial published in Nature Medicine, the therapy demonstrated promising efficacy with 50% of patients achieving stable disease or better. Most impressive are the two complete responses with durations of 7.5 and 66+ months鈥攅xtraordinarily long for these aggressive brain tumors that typically show median survival of 12-15 months. The correlation between response and pre-existing "hot" tumors (high CD3+ T cell infiltration) provides crucial mechanistic insights for patient selection.
Mustang's combination strategy of MB-101 (CAR T-cell therapy) with MB-108 (oncolytic virus) represents an innovative approach to the well-known challenge of immunologically "cold" tumors. By using MB-108 to potentially convert "cold" tumors to "hot" ones, they're addressing a fundamental limitation of CAR T-cell therapies in solid tumors. The fact that both components now have ODD status enhances the combined program's regulatory position.
However, investors should note the explicit statement regarding the need for additional funding or strategic partnership to advance the MB-109 program, indicating near-term capital constraints that could impact development timelines for this promising therapeutic approach.
In an ongoing Phase 1 trial published in Nature Medicine, MB-101 was well-tolerated and
Preclinical data support a novel combination of MB-101 (IL13R伪2鈥恡argeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus) to optimize clinical results
FDA previously granted Orphan Drug Designation to Mustang for MB-108 for the treatment of malignant glioma
WORCESTER, Mass., July 07, 2025 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (鈥淢ustang鈥� or the 鈥淐ompany鈥�) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today鈥檚 medical breakthroughs in cell therapies into potential cures for difficult-to-treat cancers, today announced that the U.S. Food and Drug Administration (鈥淔DA鈥�) has granted Orphan Drug Designation to Mustang for MB-101 (IL13Ra2-targeted CAR T-cells) for the treatment of recurrent diffuse and anaplastic astrocytoma (astrocytomas) and glioblastoma (GBM).
The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. If a product receives Orphan Drug Status from the FDA, that product is entitled to seven years of market exclusivity for the disease in which it has Orphan Drug designation, which is independent from intellectual property protection.
Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, 鈥淲e are thrilled that MB-101 received Orphan Drug Designation on time and with a designation that is broader than the indication proposed. The Orphan Drug Designation for MB-101, coupled with the Orphan Drug Designation granted previously for MB-108, is strong validation for our science, as we hope to advance MB-101, in combination with MB-108, as a potential treatment option for patients living with malignant glioma, including patients with recurrent glioblastoma (鈥淕BM鈥�) and high-grade astrocytomas. Our novel therapeutic strategy, combining our MB-101 CAR-T cell therapy with our MB-108 oncolytic virus, leverages MB-108 to reshape the tumor microenvironment (鈥淭ME鈥�) to make cold tumors 鈥渉ot,鈥� thereby potentially improving the efficacy of MB-101 CAR-T cell therapy. This progress demonstrates our dedication to exploring new possibilities for improving outcomes in patients with challenging-to-treat cancers.鈥�
As previously reported, preclinical data presented at the American Association for Cancer Research (鈥淎ACR鈥�) Annual Meeting in 2022 supported a combination therapy to potentially optimize results to treat recurrent GBM. The combination leverages MB-108 to reshape the TME and make cold tumors 鈥渉ot,鈥� thereby potentially improving the efficacy of MB-101 CAR-T cell therapy. Data presented separately on MB-101 and MB-108 showed that administration of these therapies was well tolerated in recurrent GBM patients. As reported in City of Hope鈥檚 2024 Nature Medicine paper, 2 patients treated solely with MB-101 who had high levels of intratumoral CD3+ T cells pre-therapy (i.e., 鈥渉ot鈥� tumors) achieved complete responses lasting 7.5 and 66+ months, respectively. Importantly, of the 57 City of Hope Phase 1 patients evaluable for survival in that paper, these 2 complete responses were observed in the cohort of 3 patients with the 鈥渉ottest鈥� tumors prior to treatment with MB-101. Phase 1 clinical trials of MB-101 at City of Hope and of MB-108 at The University of Alabama at Birmingham continue to enroll patients.
The Company鈥檚 ability to further develop the MB-109 program for recurrent GBM and high-grade astrocytomas is contingent upon raising additional funding and / or consummating a strategic partnership.
About MB-109 (MB-101听(IL13R伪2 targeted CAR-T cells)听+ MB-108 oncolytic virus)听
MB-109 is Mustang鈥檚 designation for the treatment regimen combining MB-101 (IL13R伪2鈥恡argeted CAR-T cell therapy licensed from City of Hope) with MB-108 (HSV-1 oncolytic virus licensed from Nationwide Children鈥檚 Hospital). The combination is designed to leverage MB-108 to make cold tumors 鈥渉ot鈥� and potentially improve the efficacy of MB-101 CAR-T cell therapy. MB-108 oncolytic virus is first injected to infect tumor cells which, in turn, leads to reshaping of the TME through recruitment of endogenous CD8- and CD3-positive effector T-cells. This inflamed TME potentially permits MB-101 CAR-T cells injected into and around the tumor to better infiltrate into and throughout the tumor mass, undergo activation and, ideally, effect tumor cell killing.
About Mustang Bio
Mustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating today鈥檚 medical breakthroughs in cell therapies into potential cures for difficult-to-treat cancers. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR-T therapies. Mustang鈥檚 common stock is registered under the Securities Exchange Act of 1934, as amended, and Mustang files periodic reports with the U.S. Securities and Exchange Commission (鈥淪EC鈥�). Mustang was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit .
Forward-Looking Statements
This press release contains 鈥渇orward-looking statements鈥� within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渃ould,鈥� 鈥渆stimate,鈥� 鈥渆xpect,鈥� 鈥済oal,鈥� 鈥渋ntend,鈥� 鈥渓ook forward to,鈥� 鈥渕ay,鈥� 鈥減lan,鈥� 鈥減otential,鈥� 鈥減redict,鈥� 鈥減roject,鈥� 鈥渟hould,鈥� 鈥渨ill,鈥� 鈥渨ould鈥� and similar expressions. The Company鈥檚 forward-looking statements, include, but are not limited to, any statements relating to our growth strategy and product development programs, including the timing of and our ability to make regulatory filings such as INDs and other applications and to obtain regulatory approvals for our product candidates, statements concerning the potential of therapies and product candidates and any other statements that are not historical facts. Actual events or results may differ materially from those described in this press release due to a number of risks and uncertainties. Risks and uncertainties include, among other things, our need for substantial additional funds in the immediate future, risks that any actual or potential clinical trials described herein may not initiate or complete in sufficient timeframes to advance the Company鈥檚 corporate objectives, or at all, or that promising early results obtained therefrom may not be replicable; whether the purchaser of the Company鈥檚 manufacturing facility is able to successfully perform its obligation to produce the Company鈥檚 products under the manufacturing services agreement on a timely basis and to acceptable standards; disruption from the sale of the Company鈥檚 manufacturing facility making it more difficult to maintain business and operational relationships; negative effects of the announcement or the consummation of the transaction on the market price of the Company鈥檚 common stock; significant transaction costs; the development stage of the Company鈥檚 primary product candidates, our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; government regulation; patent and intellectual property matters; competition; as well as other risks described in Part I, Item 1A, 鈥淩isk Factors,鈥� in our Annual Report on Form 10-K filed on March 28, 2025, subsequent Reports on Form 10-Q, and our other filings we make with the SEC. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.
Company Contacts:
Jaclyn Jaffe and Nicole McCloskey
Mustang Bio,听Inc.
(781) 652-4500
[email protected]
FAQ
What is the significance of Mustang Bio's (MBIO) Orphan Drug Designation for MB-101?
What are the clinical results of Mustang Bio's MB-101 therapy?
How does Mustang Bio's combination therapy of MB-101 and MB-108 work?
What are the current development stages of MB-101 and MB-108?
What challenges does Mustang Bio face in developing MB-101?
