Adaptimmune Announces U.S. FDA Breakthrough Therapy Designation Granted to Letetresgene Autoleucel (lete-cel) for Treatment of Myxoid/Round Cell Liposarcoma (MRCLS)
Adaptimmune Therapeutics (NASDAQ: ADAP) announced that its therapy letetresgene autoleucel (lete-cel) has received FDA breakthrough therapy designation for treating myxoid/round cell liposarcoma (MRCLS) in specific patient conditions. The designation was based on Phase II IGNYTE-ESO trial results, where 42% of patients (27/64) with synovial sarcoma or MRCLS showed responses, including 6 complete and 21 partial responses.
The median duration of response was 12.2 months overall, with 18.3 months for synovial sarcoma and 12.2 months for MRCLS patients. Safety findings showed manageable toxicities, with common adverse events including cytopenias, cytokine release syndrome, and rash.
The breakthrough designation will expedite lete-cel's development and review processes, with Adaptimmune planning to initiate a rolling Biologics License Application later in 2025. The company aims to bring lete-cel to market in 2026 for both synovial sarcoma and MRCLS treatment.
Adaptimmune Therapeutics (NASDAQ: ADAP) ha annunciato che la sua terapia letetresgene autoleucel (lete-cel) ha ricevuto la designazione di terapia innovativa dalla FDA per il trattamento del liposarcoma myxoide/cellule rotonde (MRCLS) in condizioni specifiche del paziente. La designazione si basa sui risultati della fase II dello studio IGNYTE-ESO, dove il 42% dei pazienti (27/64) con sarcoma sinoviale o MRCLS ha mostrato risposte, comprese 6 risposte complete e 21 risposte parziali.
La durata mediana della risposta è stata di 12,2 mesi complessivamente, con 18,3 mesi per i pazienti con sarcoma sinoviale e 12,2 mesi per i pazienti con MRCLS. I risultati sulla sicurezza hanno mostrato tossicità gestibili, con eventi avversi comuni che includono citopenie, sindrome da rilascio di citochine e rash.
La designazione di terapia innovativa accelererà i processi di sviluppo e revisione di lete-cel, con l'intenzione di Adaptimmune di avviare una Biologics License Application a rotazione entro la fine del 2025. L'azienda punta a portare lete-cel sul mercato nel 2026 sia per il trattamento del sarcoma sinoviale che del MRCLS.
Adaptimmune Therapeutics (NASDAQ: ADAP) anunció que su terapia letetresgene autoleucel (lete-cel) ha recibido la designación de terapia innovadora por parte de la FDA para el tratamiento del liposarcoma mixoide/células redondas (MRCLS) en condiciones específicas del paciente. La designación se basó en los resultados del estudio de fase II IGNYTE-ESO, donde el 42% de los pacientes (27/64) con sarcoma sinovial o MRCLS mostraron respuestas, incluyendo 6 respuestas completas y 21 parciales.
La duración mediana de la respuesta fue de 12,2 meses en general, con 18,3 meses para los pacientes con sarcoma sinovial y 12,2 meses para los pacientes con MRCLS. Los hallazgos sobre la seguridad mostraron toxicidades manejables, con eventos adversos comunes que incluyen citopenias, síndrome de liberación de citoquinas y erupción cutánea.
La designación de terapia innovadora acelerará los procesos de desarrollo y revisión de lete-cel, con la planificación de Adaptimmune para iniciar una solicitud de licencia biológica de forma continua a finales de 2025. La compañía tiene como objetivo llevar lete-cel al mercado en 2026 para el tratamiento tanto del sarcoma sinovial como del MRCLS.
Adaptimmune Therapeutics (NASDAQ: ADAP)는 자사의 치료제 letetresgene autoleucel (lete-cel)이 특정 환자 조건에 대한 점액종양/원형세포 지방육종(MRCLS) 치료를 위해 FDA의 혁신 치료제 지정을 받았다고 발표했습니다. 이 지정은 2상 IGNYTE-ESO 시험 결과를 바탕으로 하며, 이 시험에서 42%의 환자(27/64)가 활막육종 또는 MRCLS에 대해 반응을 보였고, 이에는 6명의 완전 반응과 21명의 부분 반응이 포함되었습니다.
반응의 중앙 지속기간은 전체적으로 12.2개월이었으며, 활막육종 환자는 18.3개월, MRCLS 환자는 12.2개월이었습니다. 안전성 결과는 관리 가능한 독성을 보였고, 일반적인 부작용으로는 세포감소증, 사이토카인 방출 증후군 및 발진이 포함됩니다.
혁신 치료제 지정을 통해 lete-cel의 개발 및 검토 과정이 가속화될 것이며, Adaptimmune는 2025년 말에 순차적 생물학적 라이센스 신청을 시작할 계획입니다. 회사는 2026년에 활막육종 및 MRCLS 치료를 위해 lete-cel을 시장에 출시할 목표를 가지고 있습니다.
Adaptimmune Therapeutics (NASDAQ: ADAP) a annoncé que sa thérapie letetresgene autoleucel (lete-cel) a reçu la désignation de thérapie innovante par la FDA pour le traitement du liposarcome myxoïde/cellules rondes (MRCLS) dans des conditions patient spécifiques. La désignation est basée sur les résultats de l'essai de phase II IGNYTE-ESO, où 42 % des patients (27/64) atteints de sarcome synovial ou de MRCLS ont montré des réponses, y compris 6 réponses complètes et 21 réponses partielles.
La durée médiane de la réponse était de 12,2 mois dans l'ensemble, avec 18,3 mois pour les patients atteints de sarcome synovial et 12,2 mois pour les patients atteints de MRCLS. Les résultats de sécurité ont montré des toxicités gérables, les événements indésirables courants incluant des cytopénies, le syndrome de libération de cytokines et des éruptions cutanées.
La désignation de thérapie innovante accélérera les processus de développement et de révision de lete-cel, Adaptimmune prévoyant d'initier une demande de licence biologique par roulement plus tard en 2025. L'entreprise vise à commercialiser lete-cel en 2026 pour le traitement à la fois du sarcome synovial et du MRCLS.
Adaptimmune Therapeutics (NASDAQ: ADAP) gab bekannt, dass ihre Therapie letetresgene autoleucel (lete-cel) von der FDA die Auflagen für eine bahnbrechende Therapie zur Behandlung von Myxoiden/runden Zell-Liposarkom (MRCLS) unter bestimmten Patientenbedingungen erhalten hat. Die Zulassung wurde auf Grundlage der Phase-II-Studie IGNYTE-ESO erteilt, in der 42 % der Patienten (27/64) mit synovialem Sarkom oder MRCLS auf eine Therapie ansprachen, einschließlich 6 vollständiger und 21 teilweiser Ansprechen.
Die mediane Ansprechdauer betrug insgesamt 12,2 Monate, wobei sie für Patienten mit synovialem Sarkom 18,3 Monate und für MRCLS-Patienten 12,2 Monate betrug. Die Sicherheitsdaten zeigten, dass handhabbare Toxizitäten vorliegen, wobei häufige unerwünschte Ereignisse Zytopenien, Zytokinfreisetzungssyndrom und Hautausschlag umfassten.
Die bahnbrechende Zulassung wird die Entwicklung und Überprüfung von lete-cel beschleunigen, wobei Adaptimmune plant, Ende 2025 eine kontinuierliche Biologics License Application einzureichen. Das Unternehmen zielt darauf ab, lete-cel 2026 sowohl zur Behandlung von synovialem Sarkom als auch von MRCLS auf den Markt zu bringen.
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The Company will present at the Annual J.P. Morgan Healthcare Conference, providing business updates on its sarcoma franchise and other cell therapy pipeline assets
Adaptimmune Allo-T program to be featured at the Biotech ShowCase(TM) and the
Wuxi Global Forum 2025
Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - January 13, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine the treatment of solid tumor cancers with cell therapy, today announced that letetresgene autoleucel (lete-cel), has been granted breakthrough therapy designation by the U.S. FDA for the treatment of patients with unresectable or metastatic myxoid/round cell liposarcoma (MRCLS) who have received prior anthracycline-based chemotherapy, are positive for HLA-A*02:01, HLA-A*02:05, or
HLA-A*02:06, and whose tumor expresses the NY-ESO-1 antigen.
More details about the Company's sarcoma franchise, including the lete-cel clinical program and launch progress for TECELRA® (afamitresgene autoleucel), the Company's first commercial product and the first FDA approved engineered cell therapy for a solid tumor, will be provided during the Company's presentation at the Annual J.P. Morgan Healthcare Conference, taking place in San Francisco, California, on Tuesday, January 14th, 2025, 4:30-5:10 PM PST (Webcast access here).
Breakthrough therapy designation for lete-cel in MRCLS was based on the results in this indication from the Phase II IGNYTE-ESO trial. The Company previously received breakthrough therapy designation for lete-cel for the treatment of synovial sarcoma in 2016. In the Phase II analysis, 27/64 (
Adrian Rawcliffe, Adaptimmune's Chief Executive Office: "This designation by the FDA highlights the potential of lete-cel to address a critical need for new treatment options for patients with MRCLS. This is another important milestone in building out our sarcoma franchise, as we aim to bring lete-cel to market in 2026 for the treatment of synovial sarcoma and MRCLS. We look forward to initiating a rolling Biologics License Application for lete-cel later this year for the treatment of both sarcoma indications."
The breakthrough therapy designation is designed to expedite drug development and review processes. The criteria for this designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. With the designation, lete-cel will receive incentives, such as additional interaction and guidance from the FDA, the potential for a rolling submission, and potential priority review of the biologics license application, as well as other opportunities to expedite the development.
In addition to the Company's presentation at J.P. Morgan, Adaptimmune will present the Company's Allo-T program as a spin-out opportunity at the Biotech ShowCase™ and the Wuxi Global Forum 2025 Investor Roundtable. Details can be found below:
Biotech Showcase™ | Investor conference | Co-produced by Demy-Colton and EBD Group
Presentation at 2 p.m. PST on Tuesday, January 14th, Yosemite C, Hilton Hotel, Union Square.
WuXi Global Forum 2025 - WXPress: for WuXi news and R&D insights
Round Table Presentation at 5 p.m. PST on Tuesday, January 14th, Tower 3, Hilton Hotel, Union Square
About Adaptimmune
Adaptimmune is a fully integrated cell therapy company working to redefine how cancer is treated. With its unique engineered T cell receptor (TCR) platform, the Company is developing personalized medicines designed to target and destroy difficult-to-treat solid tumor cancers and to radically improve the patient's cancer treatment experience.
Forward-Looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 (PSLRA). These forward-looking statements involve certain risks and uncertainties. Such risks and uncertainties could cause our actual results to differ materially from those indicated by such forward-looking statements, and include, without limitation: the success, cost and timing of our product development activities and clinical trials and our ability to successfully advance our TCR therapeutic candidates through the regulatory and commercialization processes. For a further description of the risks and uncertainties that could cause our actual results to differ materially from those expressed in these forward-looking statements, as well as risks relating to our business in general, we refer you to our Annual Report on Form 10-K filed with the Securities and Exchange Commission for the year ended 31 December, 2023, our Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, and other filings with the Securities and Exchange Commission. The forward-looking statements contained in this press release speak only as of the date the statements were made and we do not undertake any obligation to update such forward-looking statements to reflect subsequent events or circumstances.
Investor Relations
Juli P. Miller, Ph.D. - VP, Corporate Affairs and Investor Relations
T : +1 215 825 9310
M : +1 215 460 8920
Juli.Miller@adaptimmune.com
Media Relations
Dana Lynch, Senior Director of Corporate Communications
M: +1 267 990 1217
Dana.Lynch@adaptimmune.com
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/236918
FAQ
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