jCyte Identifies Key Anatomical Biomarker Predictive of Substantial Restoration of Visual Function in Retinitis Pigmentosa Patients Treated With jCell Therapy

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–jCyte, Inc., today announced key findings presented at AAO 2021 which demonstrated that retinitis pigmentosa (RP) patients with a baseline central visual field diameter greater than 20 degrees had a profound response to jCell treatment in its Phase 2b study, the largest randomized clinical trial ever conducted in RP for a locally administered investigational treatment.

Baruch Kuppermann, MD, PhD, Chairman of the Department of Ophthalmology at the UCI Gavin Herbert Eye Institute, presented detailed analyses of the Phase 2b data that assessed the impact of a single 6 million cell intravitreal injection of jCell vs. Sham over a 12-month study period and how the restoration of visual function produced by jCell treatment depended on the patients’ central visual field (VF) at baseline. Dr. Kuppermann explained how jCell upregulates and restores the function of cone photoreceptors, and the mechanism by which this is achieved.

During his presentation, Dr. Kuppermann reviewed that cone photoreceptors are dependent for their metabolism upon a neurotrophic factor, Rod derived Cone Viability Factor (RdCVF), released by rod photoreceptors. Therefore, patients with more surviving adjacent rod photoreceptors have a far greater potential for restoration of cone photoreceptor function, while patients whose disease progression has reduced their central VF diameter to < 20 degrees have few remaining rod photoreceptors and, consequently, a low potential for restoration of their cone function. Since the maximum density of rods in the retina corresponds to a VF diameter of between 20 and 45 degrees, Dr. Kuppermann explained that patients with > 20 degrees of remaining central VF diameter should have the greatest potential for restoration of cone function and the strongest response to jCell.

Dr. Kupperman presented data comparing the response of a single 6 million cell intravitreal injection of jCell vs. Sham. The data showed that RP patients with a central VF diameter > 20 degrees had a superior and substantial restoration of visual function by jCell as measured by the primary endpoint (BCVA) and all three secondary endpoints, each of which measure distinct and important aspects of visual function. The BCVA change from baseline at 12 months in this group was 15.6 letters above Sham, with this difference being statistically significant (p=0.029). While the study was not powered to show statistical significance of jCell vs. Sham on the secondary endpoints, the differences observed at 12 months between the groups were large, consistent, and extremely meaningful (difference between jCell and Sham – Contrast Sensitivity (CS): +242%; Kinetic Visual Field (KVF): +317%; and the Low Luminance Mobility Test (LLMT): +1 Critical Illumination Level).

“The magnitude and duration of response demonstrated in patients with a baseline central visual field diameter greater than 20 degrees treated with a single 6 million cell injection of jCell was statistically significant and clinically meaningful,” said Dr. Kuppermann. “This work built on the research conducted by Dr. Sunil Srivastava presented at ARVO 2021, which also demonstrated a strong, statistically significant correlation between central subfield mean foveal thickness and restoration of visual function following jCell treatment. The analyses of response to jCell treatment based on these two objective anatomical biomarkers are consistent with the pathophysiology of RP and demonstrate that the patients expected to exhibit the greatest response to jCell should have both more surviving cone photoreceptors within their fovea and more surviving rod photoreceptors adjacent to the cones.”

“The size and scope of the jCell Phase 2b study has allowed us to effectively identify the patients that are likely to benefit most from jCell therapy,” said Dr. John Pollack, Chief Medical Officer, jCyte. “The application of these key findings will help the company maximize the probability of success of the pivotal Phase 3 RP trial program without significantly limiting the potential pool of study candidates, since most RP patients have greater than 20 degrees of central visual field and a central subfield mean thickness of greater than 130 microns. These data demonstrate that jCell has the potential to transform the lives of patients with RP.”

About the Phase 2b Study:

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 agnostic to genotype with BCVA between 20/80 and 20/800. Patients received a single injection of 3 or 6 million retinal progenitor cells, or a sham treatment. The primary endpoint was mean change in BCVA from baseline to month 12, and the key secondary endpoints consisted of assessment of mean change on Kinetic Visual Field, Contrast Sensitivity, and the Low Luminance Mobility Test (LLMT).

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 late-stage clinical development biotech company focused on developing its first-in-class regenerative cell therapy, jCell, for RP and other retinal degenerative disorders. The treatment is minimally invasive and given as an intravitreal injection. 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.

Contacts

jCyte, Inc.

Robert Beathard

Sr. Vice President, Corporate Development

[email protected]