Fate Therapeutics' FT819 Gains FDA RMAT Designation for Moderate to Severe SLE

Fate Therapeutics, Inc., a biopharmaceutical company focused on developing innovative stem cell-derived treatments, has received a significant regulatory boost for its investigational therapy, FT819. The U.S. Food and Drug Administration (FDA) has granted FT819 Regenerative Medicine Advanced Therapy (RMAT) designation, a nod that could expedite its development and review process. FT819, an off-the-shelf CAR T-cell therapy derived from induced pluripotent stem cells (iPSCs), is currently in Phase 1 clinical trials aimed at treating moderate to severe systemic lupus erythematosus (SLE), including lupus nephritis (LN).

Fate Therapeutics' CEO, Bob Valamehr, emphasized the recognition of the potential of FT819 to fill a crucial gap in the treatment of lupus patients. The company’s approach aims to make CAR T-cell therapy more accessible by reducing the need for rigorous conditioning treatments and prolonged hospital stays, thereby offering the possibility of community-based treatment. This could significantly enhance accessibility for patients, particularly in underserved areas.

The FDA's RMAT designation, which is similar to a breakthrough therapy designation, offers several advantages, including the opportunity for early and frequent interactions with the agency. This can facilitate discussions around potential surrogate or intermediate endpoints that might support accelerated approval. Additionally, treatments with RMAT designation may be eligible for priority review, potentially speeding up the time it takes to bring them to market.

In its application for RMAT designation, Fate Therapeutics included promising initial data from its ongoing Phase 1 clinical trial. This study is examining the safety and efficacy of FT819 when used following a conditioning regimen that does not include fludarabine. Instead, the regimen involves either bendamustine or cyclophosphamide, followed by a single dose of FT819. The company is also investigating the effects of administering FT819 at a higher cell dose in dose escalation studies. The initial development of FT819 has been supported by a $7.9 million grant from the California Institute of Regenerative Medicine (CIRM), underscoring the therapy’s innovative potential.

FT819 is designed using Fate Therapeutics' proprietary iPSC product platform. This platform capitalizes on the unique regenerative properties of human iPSCs, which can self-renew indefinitely and differentiate into any cell type. The company uses clonal master iPSC lines to produce engineered cell products that are consistent in composition and can be stored for off-the-shelf use, making them ready for immediate application in various therapeutic contexts. This method addresses many limitations faced by traditional patient- and donor-sourced cell therapies, such as variability and limited availability.

Beyond FT819, Fate Therapeutics is advancing a robust pipeline of iPSC-derived immunotherapies, including T-cell and natural killer (NK) cell product candidates. These are engineered to incorporate novel synthetic controls of cell function, aiming to deliver multifaceted therapeutic effects. The company, headquartered in San Diego, California, maintains an extensive intellectual property portfolio, with over 500 issued patents and 500 pending applications that support its pioneering work in cell therapy and regenerative medicine.

In summary, the FDA's RMAT designation for FT819 marks a promising step in the development of new therapeutic options for lupus patients. Fate Therapeutics' innovative approach and technological advancements hold the potential to transform the landscape of cellular immunotherapy, offering hope for more effective and accessible treatments for complex autoimmune conditions.