jCyte Announces Plans to Launch Phase 3 Clinical Trial of Cell Therapy for RP

The biotechnology company is preparing to launch a pivotal, Phase 3 clinical trial in the US for its emerging jCell therapy. The company’s announcement came after a successful planning meeting with the US Food & Drug Administration. Participant enrollment for the Phase 3 trial is expected to begin during the second half of 2024. More details of the trial are forthcoming.

jCells are similar to stem cells that haven't yet fully developed into mature photoreceptors. The cells are injected into the vitreous, the soft, gel-like substance in the middle of the eye. Intravitreal injections have a good record of safety and are commonly administered for other conditions in a doctor's office. jCells are designed to release proteins known as neurotrophic factors to preserve photoreceptors regardless of the mutated gene causing vision loss.

Previously, the company reported on 76 patients in the Phase 2b trial for jCells. In the Phase 2b trial, 39 percent of patients receiving 6 million cells, the high dose of the treatment, had improvement at 12 months post-treatment in best corrected visual acuity (BCVA) of 10 letters (two lines on an eye chart) or more, with 26 percent improving by at least 15 letters. In the sham group, 19 percent and 8 percent had BCVA improvement of 10 or more and 15 or more letters, respectively. Substantial improvements for eyes treated with 6 million jCells as compared to sham were also seen in contrast sensitivity, kinetic visual fields, and mobility-related visual function (as captured by the VFQ-48 questionnaire). The 3 million cell dose, the lower dose of treatment included in the study, was shown to be inferior to the 6 million cell dose.