- Four Patients With Dry Age-Related Macular Degeneration
Observed to Have Areas of Geographic Atrophy Which Diminished or
Remained Unchanged Relative to Baseline for a Period of at Least 12
Months
- All Four Patients Exhibited Improvements in Visual Acuity at
12 Months
- Differences in Visual Acuity Between Treated and Untreated
Eyes in all Cohort 4 Patients Remained Statistically Significant At
9, 12, and 15 Months
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing allogeneic cell
therapies for unmet medical needs, announced today that restoration
of retinal tissue was observed in a fourth patient enrolled in the
Company’s Phase 1/2a clinical study of its lead product candidate,
OpRegen. Retinal tissue restoration and improved visual acuity has
now been observed in all four better vision patients treated in
Cohort 4, where surgeons successfully covered the majority of the
area of atrophy with a suspension of OpRegen cells. Outer retinal
layer restoration, which was observed using clinical
high-resolution Optical Coherence Tomography (OCT), was evidenced
by the presence of new areas of retinal pigment epithelium (RPE)
monolayer with overlying ellipsoid zone, external limiting
membrane, and outer nuclear layer, which were not present at the
time of baseline assessment. These new and additive findings
continue to support the Company’s view that atrophic AMD is not an
irreversible, degenerative condition and that some portion of
diseased retinal tissue may be recoverable. OpRegen is an
allogeneic RPE cell transplant in development for the treatment of
age-related macular degeneration (AMD) with geographic atrophy
(GA), or dry (atrophic) AMD.
“After reporting three previous cases of retinal restoration
over the course of the last two years, we have been carefully
examining the other Cohort 4 patients. We have put significant work
into reviewing images from the remaining patient who had the
majority of the area of atrophy covered by the OpRegen suspension
at the time of surgical implant. I am pleased to report that at the
scheduled Month 12 post-operative visit for this patient, although
less prominent than the 3 previously reported cases due to a
smaller area of atrophy at baseline, there is clear evidence of
retinal restoration in some areas, and the total area of atrophy as
calculated using square root transformation, or SQRT, is smaller
than the size calculated at baseline,” stated Jordi Monés, M.D.,
Ph.D., Director, Institut de la Màcula and Director, Principal
Investigator and Founder, Barcelona Macula Foundation. “The finding
of a fourth case of restoration further supports our goal of
showing that atrophic age-related macular degeneration can be
responsive to this type of cell therapy. Dry AMD is a progressive
disease and halting or reversing an area of atrophy does not occur
spontaneously, which we believe makes the durability and
reproducibility of these changes unprecedented within the field.
With 12 months of follow-up complete, I am eager to have these
cases submitted for peer-reviewed publication.”
“We have treated 24 patients with OpRegen, 12 of which were not
legally blind at baseline and represent our intended treatment
population. Among these 12 patients, four received thorough
coverage of OpRegen cells across the majority of the atrophic area
and experienced a cessation or reversal of their areas of atrophy
at 12 months and continue to be followed. These patients also
experienced increases in their visual acuity in their treated eye.
We believe these four patients represent the only examples of an
experimental treatment for dry AMD which can reduce an area of
atrophy in humans, rather than simply slow its growth,” added Brian
M. Culley, Lineage CEO. “Restoration of retinal tissue using cell
therapy represents a paradigm shift compared to conventional
approaches, which to date have only shown an unremarkable slowing
of progression. In addition to being well tolerated to date, the
durability of changes to areas of atrophy and improvements in
visual acuity support the continued and rapid clinical development
of OpRegen. Alongside our advisors, 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 to begin next year. We anticipate the
first of these engagements will begin next month and continue in
the first quarter of 2022.”
OpRegen Phase 1/2a Interim Clinical Trial Results
- Retinal restoration, reported in four patients to date,
persisted from over 12 months to greater than 3 years following
treatment and continues to be followed.
- Restoration was evidenced by the presence of new areas of RPE
monolayer with overlying ellipsoid zone, external limiting
membrane, and outer nuclear layer, which were not present at the
time of baseline assessment.
- Reductions, or no progression for at least 1 year, was observed
in the total area of GA in all four of these better vision Cohort 4
patients.
- Overall, using the Early Treatment Diabetic Retinopathy Study
(EDTRS) assessment of best corrected visual acuity (BCVA), 7/12
(58%) of each of Cohort 4 patients' treated eye were at baseline or
better at 15 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 same patients’ fellow
untreated eyes.
- All four retinal restoration patients reported improvements in
their visual acuity, which has been maintained for at least 12
months in all four of those patients.
- 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.
Overall, 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.
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 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 the
risk that treatment with OpRegen in dry AMD patients with GA may
not provide the benefits anticipated, the risk that interim results
from clinical trials may not be predictive of future results,
including later clinical trial results, and that interim data from
clinical trials may change as more patient data become available
and are subject to audit and verification procedures, and 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/20211130005471/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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