- Ongoing Clinical Trial Data Continue to Demonstrate a Single
Administration of OpRegen Can Provide Anatomical and Functional
Improvements in Patients with Dry Age-Related Macular Degeneration
with Geographic Atrophy
- All Three Cases of Retinal Restoration Have Shown Evidence
of Markedly Smaller Areas of Atrophy at 12 Months
Post-Treatment
- Detailed Imaging Analyses Showed Stability or Trends Towards
Improvement in Key Retinal Structures in Treated Eyes
- Statistically Significant Differences in Visual Acuity
Continue to be Observed in Cohort 4 Patients Between OpRegen
Treated and Fellow Untreated Eyes (N=12)
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing allogeneic cell
therapies for unmet medical needs, today reported updated interim
results from a Phase 1/2a clinical study of its lead product
candidate, OpRegen®, an investigational retinal pigment epithelium
(“RPE”) cell transplant therapy currently in development for the
treatment of dry age-related macular degeneration (“AMD”), were
presented at the 2021 American Academy of Ophthalmology (AAO) 125th
Annual Meeting. The presentation, “OpRegen Trial: Phase 1/2a Dose
Escalation Study of Human Embryonic Stem-Cell Derived Retinal
Pigment Epithelium Cells Transplanted Subretinally in Patients with
Advanced AMD, (Geographic Atrophy) (NCT02286089): Interim Results
and Further Insights from Imaging Analyses” was presented on
November 13, 2021 as part of the Gene and Cell-Based Therapies
Session, by Michael S. Ip, M.D., Professor, Department of
Ophthalmology at the David Geffen School of Medicine at the
University of California, Los Angeles. These updated results
include a minimum of 12 months of follow-up in all 12 patients
treated in Cohort 4, which as a group had better baseline vision
and smaller areas of geographic atrophy (“GA”) at baseline than
earlier cohorts. Overall, in the study (N=24), OpRegen has been
well tolerated to date and there have been no new, unexpected
ocular or systemic adverse events or serious adverse events not
previously reported.
Dr. Ip stated, “I am excited about several aspects of the
optical coherence tomography, or OCT, observations following our
team’s detailed image review. First, there appear to be resolution
of incomplete RPE and outer retinal atrophy or iRORA in several
patients. Additionally, there is evidence of resolution of areas
with features of cRORA, or complete loss of the RPE and outer
retinal tissue, in some eyes, which has not been previously
reported. Finally, there are statistically significant improvement,
or trends toward improvement, in the treated eyes versus the fellow
eyes as compared from baseline to month 12 post-treatment in
critical retinal tissue. I look forward to our group completing
these analyses in the remaining patients as data become
available.”
“We are encouraged by this additional supporting evidence
obtained from independent analyses of the OpRegen data in patients
with atrophic AMD,” added Brian M. Culley, Lineage CEO. “When
OpRegen is implanted in the transitional zones of the GA in
patients with less advanced disease, reversing damage, or even
simply arresting further progression for several years, has been
shown to be possible. Restoration of retinal tissue is a compelling
finding compared to conventional approaches which to date have only
shown an unexceptional slowing of progression. In addition to being
well tolerated to date, the durability of changes to areas of
atrophy and improvements in visual acuity observed point to the
urgency to further evaluate OpRegen in a larger, controlled trial.
We currently are preparing for multiple engagements with FDA to
discuss aspects of OpRegen’s designation, our manufacturing plans,
and the design of a later-stage clinical trial. We expect the first
of these engagements will begin in the fourth quarter of 2021 and
continue in the first quarter of 2022.”
OpRegen Phase 1/2a Interim Clinical Results
- Retinal restoration, reported in 3 patients to date, persists
and continues to be followed.
- Overall, using the Early Treatment Diabetic Retinopathy Study
(EDTRS) assessment of visual acuity, 7/12 (58%) of Cohort 4
patients' treated eye were at baseline or better at 12 months or
last time point, which extends beyond 3 years in some patients. In
comparison, at the same time points, 8/12 (67%) were below baseline
in those patients’ fellow untreated eyes.
- Across the study, in patients with previously reported
structural improvements in the retina, decreases in drusen density,
and a trend toward slower GA progression in treated compared to
untreated eyes continue to be present.
- Evidence of durable engraftment of OpRegen RPE cells has
extended to more than 5 years in the earliest treated patients,
supporting the potential for OpRegen to be a one-time
treatment.
Doheny OCT Sub Study Results
- The Doheny Image Reading and Research Lab (DIRRL) is leading a
sub study that is further analyzing OCT images collected as part of
the study protocol. The images are being independently assessed for
the presence or absence of retinal layers and thickness and volume
of those layers at baseline and at months 6 and 12 following
OpRegen treatment using a proprietary program known as “3D-OCTOR”.
- 3D-OCTOR is a validated, part 11-compliant, image-grading
software tool originally developed by the Doheny Imaging
Exploration and Software Engineering Laboratory (DIESEL).
- Boundaries of various retinal and subretinal spaces are
manually drawn on each SDOCT scan collected and then
comprehensively analyzed.
- After the grader draws the required layers in each of the
B-scans, the software calculates the distance in pixels between the
manually drawn boundary lines and calculates a thickness map for
the nine ETDRS macular subfields.
- In OCT images analyzed from the first 4 better vision patients
treated in Cohort 4, significant improvements or stabilization of
key retinal structures such as the thickness and volume of the
ellipsoid zone (EZ) from baseline to 12 months post-treatment have
been observed.
- These areas and structures continued to progressively worsen in
the untreated fellow eyes.
- Complete resolution of both iRORA and cRORA have been noted in
some patients.
- Further analysis and review are ongoing of OCT images from the
remaining 8 better vision patients treated in Cohort 4.
About OpRegen
OpRegen is currently being evaluated in a Phase 1/2a open-label,
dose escalation safety and efficacy study of a single injection of
human retinal pigment epithelium cells derived from an established
pluripotent cell line and transplanted subretinally in patients
with advanced dry AMD with GA. The study enrolled 24 patients into
4 cohorts. The first 3 cohorts enrolled only legally blind patients
with best corrected visual acuity (BCVA) of 20/200 or worse. The
fourth cohort enrolled 12 better vision patients (BCVA from 20/65
to 20/250 with smaller mean areas of GA). Cohort 4 also included
patients treated with a new “thaw-and-inject” formulation of
OpRegen, which can be shipped directly to sites and used
immediately upon thawing, removing the complications and logistics
of having to use a dose preparation facility. The primary objective
of the study is to evaluate the safety and tolerability of OpRegen
as assessed by the incidence and frequency of treatment emergent
adverse events. Secondary objectives are to evaluate the
preliminary efficacy of OpRegen treatment by assessing the changes
in ophthalmological parameters measured by various methods of
primary clinical relevance. OpRegen has been well tolerated to date
and there have been no new, unexpected ocular or systemic adverse
events or serious adverse events that have not been previously
reported. OpRegen is a registered trademark of Cell Cure
Neurosciences Ltd., a majority-owned subsidiary of Lineage Cell
Therapeutics, Inc.
About Age-Related Macular Degeneration
Age-related macular degeneration (AMD) is an eye disease that
can blur the sharp, central vision in patients and is the leading
cause of vision loss in people over the age of 60. There are two
forms of AMD: dry (atrophic) AMD and wet (neovascular) AMD. Dry
(atrophic) AMD is the more common of the two forms, accounting for
approximately 85-90% of all cases. In atrophic AMD, parts of the
macula get thinner with age and accumulations of extracellular
material between Bruch’s membrane and the RPE, known as drusen,
increase in number and volume, leading to a progressive loss of
central vision, typically in both eyes. Global sales of the two
leading wet AMD therapies were in excess of $10 billion in 2019.
Nearly all cases of wet AMD eventually will develop the underlying
atrophic AMD if the newly formed blood vessels are treated
correctly. There are currently no U.S. Food and Drug Administration
(FDA), or European Medicines Agency, approved treatment options
available for patients with atrophic AMD.
About Lineage Cell Therapeutics, Inc.
Lineage Cell Therapeutics is a clinical-stage biotechnology
company developing novel cell therapies for unmet medical needs.
Lineage’s programs are based on its robust proprietary cell-based
therapy platform and associated in-house development and
manufacturing capabilities. With this platform Lineage develops and
manufactures specialized, terminally differentiated human cells
from its pluripotent and progenitor cell starting materials. These
differentiated cells are developed to either replace or support
cells that are dysfunctional or absent due to degenerative disease
or traumatic injury or administered as a means of helping the body
mount an effective immune response to cancer. Lineage’s clinical
programs are in markets with billion dollar opportunities and
include three allogeneic (“off-the-shelf”) product candidates: (i)
OpRegen®, a retinal pigment epithelium transplant therapy in Phase
1/2a development for the treatment of dry age-related macular
degeneration, a leading cause of blindness in the developed world;
(ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a
development for the treatment of acute spinal cord injuries; and
(iii) VAC2, an allogeneic dendritic cell therapy produced from
Lineage’s VAC technology platform for immune-oncology and
infectious disease, currently in Phase 1 clinical development for
the treatment of non-small cell lung cancer. For more information,
please visit www.lineagecell.com or follow the Company on Twitter
@LineageCell.
Forward-Looking Statements
Lineage cautions you that all statements, other than statements
of historical facts, contained in this press release, are
forward-looking statements. Forward-looking statements, in some
cases, can be identified by terms such as “believe,” “may,” “will,”
“estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,”
“could,” “can,” “plan,” “potential,” “predict,” “seek,” “should,”
“would,” “contemplate,” "project,” “target,” “tend to,” or the
negative version of these words and similar expressions. Such
statements include, but are not limited to, statements relating to
the potential benefits of treatment with OpRegen in dry AMD
patients with GA, the significance of clinical data reported to
date from the ongoing Phase 1/2a study of OpRegen, including the
findings of retinal tissue restoration, Lineage's plans to meet
with the FDA to discuss OpRegen’s clinical development, the
potential utilization of OCT imaging to measure efficacy in a
pivotal clinical trial of OpRegen for the treatment of dry AMD with
GA, and the potential for Lineage’s investigational allogeneic cell
therapies to provide safe and effective treatment for multiple,
diverse serious or life threatening conditions. Forward-looking
statements involve known and unknown risks, uncertainties and other
factors that may cause Lineage’s actual results, performance or
achievements to be materially different from future results,
performance or achievements expressed or implied by the
forward-looking statements in this press release, including risks
and uncertainties inherent in Lineage’s business and other risks in
Lineage’s filings with the Securities and Exchange Commission
(SEC). Lineage’s forward-looking statements are based upon its
current expectations and involve assumptions that may never
materialize or may prove to be incorrect. All forward-looking
statements are expressly qualified in their entirety by these
cautionary statements. Further information regarding these and
other risks is included under the heading “Risk Factors” in
Lineage’s periodic reports with the SEC, including Lineage’s most
recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q
filed with the SEC and its other reports, which are available from
the SEC’s website. You are cautioned not to place undue reliance on
forward-looking statements, which speak only as of the date on
which they were made. Lineage undertakes no obligation to update
such statements to reflect events that occur or circumstances that
exist after the date on which they were made, except as required by
law.
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version on businesswire.com: https://www.businesswire.com/news/home/20211115005451/en/
Lineage Cell Therapeutics, Inc. IR Ioana C. Hone
(ir@lineagecell.com) (442) 287-8963
Solebury Trout IR Mike Biega (Mbiega@soleburytrout.com)
(617) 221-9660
Russo Partners – Media Relations Nic Johnson or David
Schull Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com (212) 845-4242
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