- First Reported Case of Retinal Tissue Restoration Showed
Zero Growth of Atrophy at 33 Months
- Second Case of Retinal Tissue Restoration Exhibited a 10%
Reduction in Atrophy Size at 8 Months
- Third Case of Retinal Restoration is 18 Letters Above
Baseline at Last Available Time Point
- Average Difference in BCVA Between Treated and Untreated
Eyes Was More Than Two ETDRS Lines (10.8 Letters Read) in Cohort 4
Patients at 9-12 Months Post-Treatment
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 its ongoing, 24-patient Phase 1/2a clinical study of
its lead product candidate, OpRegen. OpRegen is an investigational
cell therapy consisting of allogeneic retinal pigment epithelium
(RPE) cells, administered in a single surgery to the subretinal
space, for the treatment of dry age-related macular degeneration
(AMD) with geographic atrophy (GA). These updated results include a
minimum of 9 months of follow-up in all 12 patients treated in
Cohort 4, which as a group had better baseline vision and smaller
areas of 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.
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“I am particularly encouraged by the OCT findings in the second
retinal restoration patient. Based on historical growth patterns,
we knew this patient was a slower progressor than many other
patients enrolled, and therefore less likely to benefit from
treatment. Despite this, we have been able to demonstrate a
reduction in the atrophic area as quickly as 2 months
post-treatment and a marked slowing of disease progression,” stated
Jordi Monés, M.D., Ph.D., Director of the Institut de la Màcula and
Barcelona Macula Foundation. “Further, even in patients with an
incomplete coverage of OpRegen over the primary area of atrophy, we
have observed resolution of not only lesions of iRORA (incomplete
retinal pigment epithelial and outer retinal atrophy), but also
resolution of areas with features of cRORA, which is a state of
complete loss of the RPE and outer retinal tissue. Additionally,
the structural benefits may help explain the improvement in visual
acuity. I eagerly look forward to new data as they are
collected.”
“While competing efforts are focused on reducing the growth rate
of geographic atrophy, Lineage has reported several patients whose
areas of atrophy have stabilized or reduced in size. These
observations, which are present across clinically-meaningful
periods, indicate a reversal of the degeneration of critical
retinal tissue layers which support vision, consistent with the
proposed mechanism of an RPE cell transplant. Importantly, all
three of the patients exhibiting restoration had confirmed historic
growth rate in these areas and these data have been collected using
multiple imaging modalities. The durability of the improvements to
visual acuity, when coupled with the clear structural improvements
we’ve seen in patients which received fuller coverage of OpRegen
across their GA, strongly suggest that cell therapy may be able to
achieve therapeutic benefits that are beyond the reach of targeted
drugs or antibodies," added Brian M. Culley, Lineage CEO. “We are
extremely pleased that our data is moving in a positive direction
with each interim update we provide. We will continue to collect
follow up data and work towards a meeting with FDA to discuss key
aspects of our program. Our objective with OpRegen is to
demonstrate the potential for allogeneic cell therapy to deliver
the best available clinical outcomes and apply our technology to
additional areas such as cancer, spinal cord injury, and other
attractive opportunities.”
OpRegen Phase 1/2a Interim Clinical Results
- Overall, 8/12 (67%) of the Cohort 4 patients’ treated eyes were
at or above baseline visual acuity at their last assessment, based
on per protocol scheduled visits ranging from 9 months to over 3
years post-transplant. Conversely, 9/12 (75%) of the patients’
untreated eyes were below baseline visual acuity at that
assessment.
- Improvement in best corrected visual acuity (BCVA) reached up
to +24 letters on the Early Treatment Diabetic Retinopathy Study
(ETDRS) chart for a Cohort 4 patient.
- Comparing all treated eyes to all fellow (untreated) eyes
showed an average difference of 10.8 letters read in Cohort 4
patients at their last assessment.
- In those Cohort 4 patients with a benefit in treated as
compared with fellow eye (10/12), the average difference between
treated and untreated eyes was 13.6 letters read at the last
assessment, which exceeded 3 years post-transplant for some
patients.
- Among the six Cohort 4 patients treated between September and
November 2020, three (50%) continue to exhibit marked improvements
in BCVA, ranging from +5 to +18 to +24 letters read at the
patient’s last scheduled assessment, which was at least 9 months
post-transplant.
- Among the other three Cohort 4 patients treated in the Fall of
2020, one patient showed a gain of +1 letter read and two patients
measured -2 and -6 letters below baseline at their last
assessment.
- 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 have continued 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.
Retinal Tissue Restoration Update
- Three patients with evidence of retinal restoration and
confirmed history of GA growth continue to demonstrate areas of
retinal restoration as of their last per protocol assessments,
ranging from 9 months to 33 months following treatment.
- The first Cohort 4 patient with evidence of retinal restoration
and confirmed history of GA growth, demonstrated zero growth in
atrophy (GA) 33 months following treatment with OpRegen.
- The second patient with evidence of restoration of critical
retinal structures showed a 10% reduction at approximately 8 months
after treatment, as assessed by square root transformation (SQRT).
- Based on historical images of the patient’s treated eye taken
~2 years prior to treatment, the area of GA had increased from
approximately 2.39 mm to approximately 2.81 mm at baseline.
- Following OpRegen transplantation, the atrophic lesion measured
approximately 2.28 mm at the patient’s month 2 per protocol
assessment visit, which was smaller than the historical size of 2.39 mm
from the image taken ~2 years prior to treatment.
- Additional follow-up showed the lesion size calculated as
approximately 2.53 mm at the patient’s month 8 per protocol
assessment visit, which continued to be smaller than baseline.
- The third case of restoration demonstrated clinically
meaningful improvements in visual acuity, having gained +18 letters
on the ETDRS chart since OpRegen transplantation, supporting the
view that the changes in retinal structure observable on Optical
Coherence Tomography (OCT) can result in functional benefit.
As previously described, outer retinal layer restoration was
observed using clinical high-resolution OCT. To be considered as
suggestive of retinal restoration, 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, had to be present post-treatment with OpRegen. These
findings, observed in 3 Cohort 4 patients, suggest integration of
the new RPE cells with functional photoreceptors in areas that
previously showed no presence of these cells. These effects were
most prominent in the transitional areas around the primary area of
atrophy. The use of OCT allows for a more precise determination of
changes in retinal thickness, organization, and overall health of
the retina in areas of potential atrophy, benefits which are
possible with cell transplant therapy.
The loss of RPE cells over time creates progressively larger
areas of atrophy in the adult retina, leading to impaired vision or
complete blindness, a condition known as atrophic AMD. Humans lack
the innate ability to regenerate retinal tissue and replace lost
retina cells, which led to a presumption that progression of GA may
someday be slowed or halted but could not be reversed. The unique
findings from the ongoing OpRegen clinical study support a
different view, in which an RPE cell transplant can potentially
replace or rescue retinal cells in patients who suffer from retinal
lesions or degeneration. The totality of these findings supports
the view that atrophic AMD is not an irreversible, degenerative
condition and that some portion of diseased retinal tissue may be
recoverable.
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 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 immuno-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 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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Lineage Cell Therapeutics, Inc. IR Ioana C. Hone
(ir@lineagecell.com) (442) 287-8963 Solebury Trout IR
Gitanjali Jain Ogawa (Gogawa@soleburytrout.com) (646) 378-2949
Russo Partners – Media Relations Nic Johnson or David Schull
Nic.johnson@russopartnersllc.com David.schull@russopartnersllc.com
(212) 845-4242
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