Fate Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from FDA for FT819 to Treat Moderate to Severe Systemic Lupus Erythematosus (SLE)
Fate Therapeutics (NASDAQ: FATE) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for its FT819 therapy, an off-the-shelf CAR T-cell treatment for moderate to severe systemic lupus erythematosus (SLE).
The RMAT designation was granted based on initial clinical safety and activity data from an ongoing Phase 1 study. The trial evaluates a fludarabine-free conditioning regimen with either bendamustine or cyclophosphamide alone, followed by a single FT819 dose. The company is currently expanding dosing to up to 10 patients at 360 million cells and assessing safety at 900 million cells.
The development is supported by a $7.9 million grant from the California Institute of Regenerative Medicine. Additional Phase 1 clinical data will be presented at medical conferences in 2025. The RMAT designation enables expedited development and review, including potential accelerated approval pathways and priority review.
Fate Therapeutics (NASDAQ: FATE) ha ricevuto la designazione di Terapia Avanzata in Medicina Rigenerativa (RMAT) dalla FDA per la sua terapia FT819, un trattamento CAR T-cell pronto all'uso per il lupus eritematoso sistemico (LES) da moderato a grave.
La designazione RMAT è stata concessa sulla base dei dati iniziali di sicurezza e attività clinica provenienti da uno studio di Fase 1 in corso. Il trial valuta un regime di condizionamento senza fludarabina con bendamustina o ciclofosfamide da sola, seguito da una singola dose di FT819. L'azienda sta attualmente ampliando la somministrazione fino a 10 pazienti a 360 milioni di cellule e valutando la sicurezza a 900 milioni di cellule.
Lo sviluppo è supportato da un finanziamento di 7,9 milioni di dollari dall'Istituto della California per la Medicina Rigenerativa. Ulteriori dati clinici di Fase 1 saranno presentati in conferenze mediche nel 2025. La designazione RMAT consente uno sviluppo e una revisione accelerati, inclusi potenziali percorsi di approvazione accelerata e revisione prioritaria.
Fate Therapeutics (NASDAQ: FATE) ha recibido la designación de Terapia Avanzada en Medicina Regenerativa (RMAT) de la FDA para su terapia FT819, un tratamiento CAR T-cell listo para usar para el lupus eritematoso sistémico (LES) de moderado a grave.
La designación RMAT se otorgó en base a datos iniciales de seguridad y actividad clínica de un estudio de Fase 1 en curso. El ensayo evalúa un régimen de acondicionamiento sin fludarabina con bendamustina o ciclofosfamida sola, seguido de una sola dosis de FT819. La empresa está ampliando actualmente la dosificación a hasta 10 pacientes a 360 millones de células y evaluando la seguridad a 900 millones de células.
El desarrollo cuenta con un subsidio de 7,9 millones de dólares del Instituto de Medicina Regenerativa de California. Se presentarán datos clínicos adicionales de Fase 1 en conferencias médicas en 2025. La designación RMAT permite un desarrollo y revisión acelerados, incluidos posibles caminos de aprobación acelerada y revisión prioritaria.
Fate Therapeutics (NASDAQ: FATE)는 FDA로부터 FT819 요법에 대해 재생 의학 고급 치료(RMAT) 지정을 받았습니다. FT819는 중등도에서 중증의 전신성 홍반 루푸스(SLE)를 위한 즉시 사용 가능한 CAR T세포 치료제입니다.
RMAT 지명은 진행 중인 1상 연구의 초기 임상 안전성 및 활성 데이터에 기반하여 부여되었습니다. 이 시험은 플루다라빈이 없는 조건화 요법을 벤다무스틴 또는 사이클로포스파미드 단독으로 평가하고, 그 뒤에 FT819의 단일 용량을 투여합니다. 현재 회사는 360백만 세포로 최대 10명의 환자에게 투여를 확대하고 900백만 세포에서의 안전성을 평가하고 있습니다.
이 개발은 790만 달러의 보조금을 통해 캘리포니아 재생 의학 연구소의 지원을 받고 있습니다. 추가 1상 임상 데이터는 2025년 의료 회의에서 발표될 예정입니다. RMAT 지명은 신속한 개발 및 검토를 가능하게 하며, 잠재적인 가속 승인 경로와 우선 검토를 포함합니다.
Fate Therapeutics (NASDAQ: FATE) a reçu la désignation de Thérapie Avancée en Médecine Régénérative (RMAT) de la FDA pour sa thérapie FT819, un traitement CAR T-cell prêt à l'emploi pour le lupus érythémateux systémique (LES) modéré à sévère.
La désignation RMAT a été accordée sur la base des données initiales de sécurité et d'activité clinique d'une étude de Phase 1 en cours. L'essai évalue un schéma de conditionnement sans fludarabine avec soit de la bendamustine soit de la cyclophosphamide seule, suivi d'une dose unique de FT819. L'entreprise élargit actuellement l'administration à jusqu'à 10 patients à 360 millions de cellules et évalue la sécurité à 900 millions de cellules.
Le développement est soutenu par une subvention de 7,9 millions de dollars de l'Institut de Médecine Régénérative de Californie. Des données cliniques supplémentaires de Phase 1 seront présentées lors de conférences médicales en 2025. La désignation RMAT permet un développement et une révision accélérés, y compris des voies d'approbation accélérée potentielles et une révision prioritaire.
Fate Therapeutics (NASDAQ: FATE) hat von der FDA die Bezeichnung für Fortschrittliche Therapie in der Regenerativen Medizin (RMAT) für seine FT819-Therapie erhalten, eine sofort einsatzbereite CAR-T-Zellbehandlung für mittelschweren bis schweren systemischen Lupus erythematodes (SLE).
Die RMAT-Bezeichnung wurde auf der Grundlage erster klinischer Sicherheits- und Aktivitätsdaten aus einer laufenden Phase-1-Studie vergeben. Die Studie bewertet ein fludarabinfreies Konditionierungsregime mit entweder Bendamustin oder Cyclophosphamid allein, gefolgt von einer einzelnen Dosis FT819. Das Unternehmen erweitert derzeit die Dosierung auf bis zu 10 Patienten mit 360 Millionen Zellen und bewertet die Sicherheit bei 900 Millionen Zellen.
Die Entwicklung wird durch einen Zuschuss von 7,9 Millionen Dollar des California Institute for Regenerative Medicine unterstützt. Weitere klinische Phase-1-Daten werden 2025 auf medizinischen Konferenzen vorgestellt. Die RMAT-Bezeichnung ermöglicht eine beschleunigte Entwicklung und Prüfung, einschließlich potenzieller beschleunigter Genehmigungswege und Prioritätsprüfungen.
- Received RMAT designation from FDA, potentially accelerating approval process
- Ongoing Phase 1 trial showing promising initial safety and activity data
- Secured $7.9 million grant from CIRM for clinical development
- Treatment designed for community setting access and reduced hospitalization
- Still in early Phase 1 stage with patient data
- Additional clinical data not available until later in 2025
Insights
The FDA's RMAT designation for FT819 represents a significant regulatory milestone for Fate Therapeutics that substantially enhances the development pathway for their lupus treatment. This designation is not granted lightly - the FDA reviews preliminary clinical data and only awards RMAT status when a therapy shows meaningful potential to address serious conditions with unmet needs.
What's particularly notable is that the FDA reviewed actual clinical safety and activity data from the ongoing Phase 1 trial before granting this designation, suggesting promising early results. RMAT designation provides Fate with tangible benefits including accelerated development timelines, increased FDA interaction, potential surrogate endpoints for faster approval, and possible priority review - essentially unlocking all Breakthrough Therapy designation advantages.
The development approach for FT819 addresses several critical challenges in autoimmune disease treatment. As an off-the-shelf CAR T-cell therapy, it eliminates the manufacturing delays and logistical complexities associated with autologous therapies. Furthermore, their strategy to use a fludarabine-free conditioning regimen could significantly reduce patient burden and hospitalization requirements, potentially enabling community-based administration.
The
The application of CAR T-cell therapy to lupus represents a potentially groundbreaking approach in autoimmune disease management. Unlike conventional lupus treatments that broadly suppress the immune system, CAR T-cells can precisely target disease-causing B cells while preserving other immune functions.
What makes Fate's approach technically innovative is their induced pluripotent stem cell (iPSC) platform, which could solve several challenges in traditional CAR T manufacturing. By creating these cells from a renewable stem cell source rather than individual patients, FT819 can potentially deliver consistent quality while eliminating the 3-4 week manufacturing delay typical with autologous CAR T products.
The clinical strategy also appears well-conceived. They're evaluating FT819 with single-agent conditioning regimens (bendamustine or cyclophosphamide alone), which could substantially reduce treatment-associated toxicity compared to standard CAR T protocols that use fludarabine combinations. This approach may enable outpatient administration - a significant advancement that would drastically improve accessibility.
Particularly noteworthy is FT819's inclusion of lupus nephritis patients, as kidney involvement represents one of the most severe and treatment-resistant lupus manifestations. The ongoing dose-finding work (testing both 360 million and 900 million cells) is essential to determine the optimal therapeutic window. If successful, this therapy could fundamentally change treatment paradigms not just for lupus but potentially for numerous B-cell mediated autoimmune conditions.
– Recognizes potential of off-the-shelf CAR T-cell therapy to address significant unmet need and enables increased dialogue with FDA throughout the development process
– RMAT review by FDA included initial clinical safety and activity data from ongoing Phase 1 study of FT819 in SLE
– Additional Phase 1 clinical data of FT819 to be presented at medical conferences in 2025
SAN DIEGO, April 14, 2025 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to FT819, an investigational, off-the-shelf, iPSC-derived CAR T-cell therapy in Phase 1 clinical development for the treatment of active moderate to severe systemic lupus erythematosus (SLE), including lupus nephritis (LN).
“RMAT designation recognizes the unique therapeutic potential of our off-the-shelf CAR T-cell therapy to address the unmet need of a wide range of lupus patients,” said Bob Valamehr, Ph.D., MBA, President and Chief Executive Officer of Fate Therapeutics. “We believe our current development strategy for FT819, which is designed to provide CAR T-cell therapy on-demand in a cost-effective manner and alleviate patient burden associated with intense conditioning chemotherapy and extended hospitalization, may enable treatment in the community setting and access to patients in underserved areas. With this designation, we look forward to working closely with the FDA as we seek to accelerate development of FT819 to bring this unique treatment to patients across the continuum of care.”
The Company’s RMAT application included initial clinical safety and activity data from patients treated with FT819 in its ongoing multi-center Phase 1 clinical trial. The Phase 1 study is designed to evaluate the safety and efficacy of a fludarabine (flu)-free conditioning regimen, consisting of either bendamustine alone or cyclophosphamide alone, followed by a single dose of FT819. The Company is currently conducting dose expansion in up to 10 patients at 360 million cells, and is also assessing the safety, pharmacokinetics, and anti-B cell activity of FT819 at 900 million cells in dose escalation. The Company will report additional clinical data from the Phase 1 study of FT819 at scientific meetings later in 2025. The clinical development of FT819 is supported by a
The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases or conditions. RMAT designation includes all Breakthrough Therapy designation features, such as early interactions with the FDA, including discussions on potential surrogate or intermediate endpoints that may support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application.
About Fate Therapeutics’ iPSC Product Platform
Human induced pluripotent stem cells (iPSCs) possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s proprietary iPSC product platform combines multiplexed-engineering of human iPSCs with single-cell selection to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a starting cell source to manufacture engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be administered in combination with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with patient- and donor-sourced cell therapies. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 500 issued patents and 500 pending patent applications.
About Fate Therapeutics, Inc.
Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients. Using its proprietary iPSC product platform, the Company has established a leadership position in creating multiplexed-engineered master iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products. The Company’s pipeline includes iPSC-derived T-cell and natural killer (NK) cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit www.fatetherapeutics.com.
Forward-Looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the advancement of and plans related to the Company's product candidates, clinical studies and preclinical research and development programs, the Company’s progress, plans and timelines for the clinical investigation of its product candidates, the initiation and continuation of enrollment in the Company’s clinical trials, the initiation of additional clinical trials, including in new indications, and additional dose cohorts in ongoing clinical trials of the Company’s product candidates, the availability of data from the Company’s clinical trials and the Company’s plans to provide updates on its clinical trials, the therapeutic and market potential of the Company’s research and development programs and product candidates, and the Company’s clinical and product development strategy. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company’s research and development programs and product candidates, including those product candidates in clinical investigation, may not demonstrate the requisite safety, efficacy, or other attributes to warrant further development or to achieve regulatory approval, the risk that results observed in prior studies of the Company’s product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Company’s product candidates or in the initiation and conduct of, or enrollment of patients in, any clinical trials, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, changes in the therapeutic, regulatory, or competitive landscape for which the Company’s product candidates are being developed, the amount and type of data to be generated or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Company’s ongoing and planned clinical trials, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, failure to demonstrate that a product candidate has the requisite safety, efficacy, or other attributes to warrant further development, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects, and the risk that research funding and milestone payments received by the Company from CIRM may be less than expected. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the Securities and Exchange Commission, including but not limited to the Company’s most recently filed periodic report, and from time to time in the Company’s press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.
Contact:
Christina Tartaglia
Precision AQ
212.362.1200
christina.tartaglia@precisionaq.com
FAQ
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