Fate Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from FDA for FT819 to Treat Moderate to Severe Systemic Lupus Erythematosus (SLE)
– 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
“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
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.
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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
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Source: Fate Therapeutics, Inc.