Fate Therapeutics Selected for U.S. FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot Program to Support Manufacturing Readiness of FT819
FDA Chemistry, Manufacturing, and
Current Regenerative Medicine Advanced Therapy (RMAT) designation and CDRP participation provide opportunity for expedited regulatory pathway and advancement of FT819 Phase 2 potentially registrational clinical trial in patients with refractory moderate-to-severe systemic lupus erythematosus (SLE) with lupus nephritis
The FDA CDRP Program is a highly selective initiative, with no more than nine proposals accepted annually across the
FT819 is an off-the-shelf anti-CD19 chimeric antigen receptor (CAR) T-cell therapy candidate currently under investigation for patients with various B-cell related autoimmune disorders. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, a precisely engineered clonal master induced pluripotent stem cell (iPSC) line serves as the starting cell source to manufacture FT819, overcoming numerous limitations associated with patient- and donor-sourced CAR T-cell therapies. Under the FT819 Phase 1 autoimmune basket clinical protocol, broad patient accessibility is enabled, especially in underserved regions, with on-demand availability of drug product and outpatient treatment with same-day discharge. FT819 previously received RMAT designation from the FDA for the treatment of moderate-to-severe SLE. The RMAT designation was granted based on preliminary clinical evidence demonstrating the potential of FT819 to address significant unmet need in patients with SLE. The RMAT designation provides the Company with benefits including FDA guidance on efficient development, alignment on CMC readiness, and the potential for accelerated approval.
"The selection of FT819 into the
About
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 potential benefits of FT819 based on preliminary data, the anticipated benefits of RMAT designation and participation in the CDRP program, the ability of the Company to expedite commercial manufacturing readiness of FT819, the advancement of and plans related to the Company's product candidates and clinical studies, 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, 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, the Company’s clinical and product development strategy, and the Company’s progress and plans relating to, and the anticipated timing and outcome of, interactions with the FDA and other regulatory authorities, including its expectations relating to alignment with regulatory authorities on potential registrational pathways for FT819. 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, risks relating to regulatory interactions and the outcome of such interactions, and the risk that the Company may not obtain adequate financing to fund its planned clinical trials and other expenses. 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.
Contact:
IR@fatetherapeutics.com
Source: Fate Therapeutics, Inc.