jCyte, Inc. today announced presentations at the prestigious Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting. Following the Phase 2b headline results of jCell therapy in retinitis pigmentosa (RP) first presented at the American Society of Retinal Specialists (ASRS) Annual Meeting, further detailed analyses from this study, the largest randomized clincal trial ever conducted in RP for a locally administered investigational treatment, were presented at ARVO by Dr. David Liao from Retina Vitreous Associates in Los Angeles, and Dr. Sunil Srivastava from the Cleveland Clinic Cole Eye Institute.
David Liao, M.D., a principal investigator of the Phase 2b study of jCell, presented data that demonstrated patients in the target population (which excludes patients with highly advanced disease who could not be reliably measured), treated with a single 6 million cell dose, had an early, sustained, and significant mean improvement in the primary endpoint of best corrected visual acuity (BCVA) (+16.27 letters vs. Sham +1.85 letters between baseline and the final 12 month study visit, p=0.003). All key secondary visual function endpoints, which evaluate important and different aspects of functional vision in RP patients, were aligned with the significant results in BCVA, with meaningful improvements in peripheral visual field area, contrast sensitivity, and the ability to ambulate better in substantially lower light settings as measured by the Low Luminance Mobility Test (LLMT).
Sunil Srivastava, M.D., presented a detailed analysis by the Cleveland Clinic Cole Eye Institute which identified central foveal thickness (CFT), measured by spectral-domain optical coherence tomography (SD-OCT), as an anatomical marker for response to jCell, which jCyte believes will be an important factor in the inclusion criteria for the Phase 3 pivotal study. A strong, statistically significant correlation was seen between CFT and all 5 visual function clinical trial endpoints in the target patient population treated with a single 6 million cell dose, with higher CFT values corresponding to greater improvements in each endpoint. A moderate to strong correlation was also seen in the same target group between all trial endpoints and mid-subfield mean ellipsoid zone (EZ) thickness.
“The identification of these important structural predictors of response aligns scientifically with the RP patient population expected to respond to jCell treatment based upon its paracrine mechanism of action,” said Dr. Sunil Srivastava from the Cleveland Clinic Cole Eye Institute, lead investigator for the OCT analysis. “These results are very encouraging as we know that jCell releases an array of well-established neurotrophic factors that have been shown to support photoreceptor function and survival in key preclinical models of RP and other retinal degenerative disorders when administered early in the disease process, prior to the significant loss of key retinal cell layers.”
“These findings are very promising given the magnitude and breadth of visual function improvements seen 12-months post treatment in the target population across all of the clinical trial endpoints included within our Phase 2b trial, four of which measure important, distinct and objective attributes of functional vision in RP patients,” said Dr. Shannon Blalock, Chief Executive Officer, jCyte. “We look forward to working closely with our scientific advisory board (SAB) and principal investigators to apply these key learnings to our upcoming pivotal trial of jCell to optimize its probability of success in an effort to advance the clinical development program of our RMAT designated therapy for RP patients who currently have no treatment options.”
About the Studies
The multicenter, randomized Phase 2b study was conducted to evaluate the safety and efficacy of a single intravitreal injection of jCell therapy across a 12-month study period in a broad set of RP patients with BCVA between 20/80 and 20/800. Patients received a single dose of 3 or 6 million human retinal progenitor cells, or a sham treatment. The primary endpoint was mean change in BCVA from baseline to month 12, and the secondary endpoints consisted of assessment of mean change on the Low Luminance Mobility Test (LLMT), kinetic visual fields, contrast sensitivity, and a visual function quality of life questionnaire. These endpoints were assessed in a target subgroup of patients which excluded those with highly advanced RP who could not be reliably measured based on the following criteria: 1) patient could not maintain fixation 2) patient had remaining visual field <12° central diameter and 3) patient study eye was significantly worse than the fellow eye (BCVA difference >15 letters).
Investigators also examined the baseline SD-OCT imaging characteristics to identify objective, anatomical predictors of efficacy in this target population. The team explored the relationship between each baseline SD-OCT parameter and the change in each of the visual function outcomes in the study.
About Retinitis Pigmentosa (RP)
Retinitis pigmentosa (RP) is a rare, genetic condition that progressively destroys the rod and cone photoreceptors in the retina. It often strikes people in their teens, with many patients rendered legally blind by middle age. Worldwide, over 2 million individuals are estimated to suffer from the disease, including approximately 100,000 people in the U.S., making it the leading cause of inherited blindness.
About jCyte, Inc.
jCyte, Inc. is a clinical-stage biotech company focused on the development of its first-in-class regenerative cell therapy, jCell, for retinitis pigmentosa (RP) and other retinal degenerative disorders. The treatment is minimally invasive and given as an intravitreal injection. There are currently no FDA approved therapies for RP. The Company is pioneering a new era of regenerative therapies to address the significant unmet medical needs of patients suffering from a broad set of retinal degenerative diseases. For more information, visit www.jcyte.com.