Fate Therapeutics (FATE) Jefferies 2024 Global Healthcare Conference summary

Industry landscape and therapeutic rationale

• CAR T-cell therapy in autoimmunity has shown remarkable results, with some patients achieving drug-free remission and profound disease reset after a single intervention.

• Current regimens require intense conditioning and hospitalization, similar to oncology, presenting challenges for patient access and safety.

• Off-the-shelf, iPSC-derived CAR T and CAR NK cell programs are being developed to improve accessibility and patient-friendliness.

Product construct and clinical experience

• The iPSC-derived CD19 CAR T construct, previously used in oncology, is now being tested in autoimmunity at the same dose and cell line.

• In oncology, the construct showed a 50% response rate, 25% CR rate, and strong safety, even in heavily pretreated patients.

• Translational data indicate deep B-cell depletion in blood and tissue, with reconstitution of naive B cells, supporting the immune reset hypothesis.

Study design and operational strategy

• The lupus phase I study leverages existing oncology sites and partnerships between oncologists and rheumatologists to facilitate enrollment.

• Two sites (University of Minnesota and University of Nebraska Medical Center) are open, with more planned.

• The study offers flexible conditioning regimens: Cy/Flu, bendamustine, or cyclophosphamide, aiming for patient-friendly protocols.

• Only three days of mandated hospitalization are required, compared to 10-14 days for other protocols.