jCyte Inc. Announces Promising Phase 2b Results of jCell Therapy in Retinitis Pigmentosa


  • Phase 2b results show that treatment with jCell was well-tolerated and demonstrated encouraging evidence
    of potential benefit in patients as an investigational treatment for retinitis pigmentosa agnostic to genetic
    subtype
  • Based on these results, jCyte plans to initiate a pivotal study in 2021
  • jCell is a first-in-class investigational treatment which has received Regenerative Medicine Advanced
    Therapy (RMAT) designation from the US Food and Drug Administration (FDA), and Orphan Drug
    designation from both the FDA and the European Medicines Agency (EMA)
  • Data presented at the American Society of Retina Specialists (ASRS) 2020 Virtual Annual Meeting on
    Saturday, July 25, 2020

NEWPORT BEACH, Calif.–(BUSINESS WIRE)– jCyte Inc., a biotech company dedicated to preserving and
restoring vision in patients with retinitis pigmentosa and other degenerative retinal disorders, announced today
promising results from a Phase 2b clinical trial of jCell in retinitis pigmentosa (RP), an inherited retinal disease,
irrespective of genetic subtype.

jCell is a first-in-class investigational treatment for RP which has received FDA Regenerative Medicine
Advanced Therapy (RMAT) designation. In addition to RMAT, jCell has received Orphan Drug designation
from the FDA and the European Medicines Agency (EMA).

The Phase 2b trial was a multicenter, randomized study that evaluated the safety and efficacy of intravitreal
injection of jCell therapy in adult subjects with RP. A large and broad set of patients with RP with best-corrected
visual acuity (BCVA) between 20/80 and 20/800 were randomized to treatment versus sham. Treatment
consisted of a single intravitreal injection of either 3 or 6 million human retinal progenitor cells (hRPC). The
study evaluated changes in visual function and functional vision in subjects with RP who received a single jCell
injection in comparison to a sham-treated control group.

The primary endpoint in the Phase 2b study was mean change in best corrected visual acuity (BCVA) from
baseline to month 12. Secondary endpoints included low light mobility, contrast sensitivity, kinetic visual fields,
and a visual function questionnaire. In a post hoc exploratory analysis, the primary and secondary endpoints
were assessed in a target subgroup of patients meeting the following criteria: 1) study eye with reliable fixation
(≥12° central diameter and/or steady central fixation) and 2) study eye did not have significantly worse BCVA
than the fellow eye (≤15 letters).

The Phase 2b clinical trial was specifically designed to learn more about the clinical effects of jCell therapy in a
broad set of patients suffering from RP to better understand three critical study variables: the optimal jCell dose,
the performance of clinical trial endpoints in the study population and the RP patient population likely to realize
the most pronounced improvement from a single jCell treatment during the 12-month pivotal trial study period.

Phase 2b Results

A total of 84 patients were randomized of which 74 met criteria for the per protocol analysis. The mean change
in BCVA from baseline to month 12 were +2.81, +2.96, and +7.43 letters in the sham (N=26), 3.0×106 hRPC
(N=25), and 6.0×106 hRPC (N=23) treatment arms, respectively. In a post hoc exploratory analysis of the target
subgroup (n=37), mean change in BCVA from baseline to month 12 were +1.85, -0.15, and +16.27 letters in the
sham (N=13), 3.0×106 hRPC (N=13), and 6.0×106 hRPC (N=11) treatment arms, respectively (p=0.003 for
6.0×106 hRPC vs sham). Supportive improvements in the 6.0×106 hRPC target subgroup compared to the
control (sham) group were also observed in all secondary endpoints which further support the BCVA findings in
this subpopulation. Adverse events were generally minor and transient; there was one serious adverse event in
the 3.0×106 hRPC arm of Grade 3 ocular hypertension that resolved with treatment, but no serious adverse
events nor any adverse events greater than Grade 1 (mild) in the higher, 6 million cell treatment group during the
12-month study period that were related to the study treatment.

Based on these results, the company plans to advance jCell into a pivotal trial utilizing the data generated, which
is supportive of both the efficacy and safety of jCell therapy in a broad set of RP patients. The Phase 2b study
also provides essential insights into the three critical study variables which are key to optimizing the probability
of technical success of the upcoming jCell pivotal trial.

“There is currently no effective treatment for RP, and once photoreceptors are lost, they do not regenerate. The
promising results from this Phase 2b study suggest that jCell therapy in patients with RP can result in significant
slowing of visual deterioration and an improvement in function of existing photoreceptors. The primary goal of
jCell therapy is to preserve, and potentially improve vision by intervening in the disease process at a time when
dystrophic photoreceptors can be protected and reactivated. We are excited to be continuing the development of
jCell investigational therapy, based on these promising Phase 2b results, for patients with this degenerative
retinal disease,” said Anthony Joseph, M.D., Ophthalmic Consultants of Boston, who presented the Phase 2b
data at the ASRS meeting.

“We are thrilled to report these encouraging Phase 2b results from one of the largest studies ever conducted in
RP, which indicate significant clinical activity of jCell in patients agnostic of genetic subtype,” said Paul Bresge,
Chief Executive Officer, jCyte, Inc. “The Phase 2b results reported that jCell therapy had promising efficacy and
was well tolerated in patients, and we plan to advance jCell into a pivotal clinical trial in 2021. There is currently
no FDA approved treatment option for RP patients, irrespective of genetic subtype. We believe jCell has the
potential to be the first approved therapy to address this critical unmet medical need, and dramatically improve
the lives of patients with this progressive, blinding disease which typically presents early in the lives of the
approximately 1:3500 people who are affected.”

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, an estimated 1.9 million people suffer from the disease, including approximately
100,000 people in the U.S., making it the leading cause of inheritable blindness.

About jCyte, Inc.

jCyte, Inc. is a clinical-stage biotech company focused on developing jCell therapy for retinitis pigmentosa (RP)
and other degenerative retinal disorders. jCell is a first-in-class investigational treatment for retinitis pigmentosa,
an inherited retinal disease. The treatment is minimally-invasive and given as an intravitreal injection. There are
currently no FDA approved therapies for RP agnostic of genetic subtype. The company is pioneering a new era
of regenerative therapies to treat patients with unmet medical needs. For more information, visit www.jcyte.com

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