- Eighty-Three Percent of All Cohort 4 Patients Continued to
Exhibit Stable or Improved BCVA At Least 6 Months Post-Treatment;
Visual Acuity Declined in the Majority of Untreated Eyes
- Three Patients with Evidence of Retinal Tissue Restoration
Continue to Demonstrate Areas of Restoration and Sustained Visual
Acuity Improvements as of Their Last Clinical Visit
- One Patient with Confirmed Atrophy Growth at Baseline has
had Zero Progression for Almost Three Full Years
- Company Intends to Meet with FDA in Q4 2021 to Discuss
Clinical Development Plan for OpRegen
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 one time 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
6 months of follow-up in all 24 patients treated with OpRegen,
including all 12 patients treated in Cohort 4, which had better
baseline vision and smaller areas of GA at baseline than earlier
cohorts.
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Improvements in Visual Acuity Observed
with OpRegen RPE Transplant (Graphic: Business Wire)
“I am excited about the observed changes in visual acuity that
show strong trends towards sustained vision improvement in almost
all of the treated eyes of Cohort 4 patients as compared to their
untreated fellow eyes, which continue to show progression of
atrophic AMD and decline of vision,” stated Principal Investigator
Allen C. Ho, M.D. FACS, Wills Eye Hospital Attending Surgeon and
Director of Retina Research, Professor of Ophthalmology, Thomas
Jefferson University. “Based on the observed retinal changes
suggestive of restoration of critical tissue in the area of
atrophy, the beneficial functional visual improvements may be
explained by structural changes as a result of OpRegen therapy and
these changes may be durable in effect. We look forward to
continuing to follow all study patients and reporting our efficacy
and safety observations over time.”
“This update further reinforces our view that a suspension of
OpRegen RPE cells can generate clinically meaningful anatomical and
functional outcomes in patients with dry AMD with GA, particularly
in those with earlier-stage atrophic disease,” stated Brian M.
Culley, Lineage CEO. “Not only has OpRegen generated the only known
cases of retinal tissue restoration in previously confirmed
atrophic areas in humans, but it also has provided a durable
functional benefit of improved visual acuity in the majority of the
twelve better vision, earlier-stage patients treated in the ongoing
clinical study. As these data continue to mature, we will work with
our advisors in preparation for a meeting with the FDA this year,
where we intend to discuss our proposed next steps for further
clinical development of OpRegen. Our belief is that RPE cell
transplants can provide outcomes beyond the reach of traditional
pharmaceutical approaches, which are limited to a subset of
biological pathways, and which may fail to provide the maximal
restorative benefit available to patients. We aim to position
OpRegen RPE transplants as the best available option in the race to
address the large unmet need in dry AMD with GA and establish
Lineage as the pre-eminent allogeneic cell therapy company.”
Overall, 10/12 (83%) of the Cohort 4 patients’ treated eyes
continued to be at or above baseline visual acuity at their last
assessment, based on per protocol scheduled visits ranging from 6
months to approximately 3 years post-transplant. Improvements in
best corrected visual acuity (BCVA) for Cohort 4 patients reached
up to +19 letters on the Early Treatment Diabetic Retinopathy Study
(ETDRS) chart. In contrast, 10/12 (83%) of the patients’ untreated
eyes were below pre-treatment baseline values at the same time
points. Among the six Cohort 4 patients treated between September
and November 2020, three (50%) continue to exhibit marked
improvements in BCVA ranging from +8 to +18 letters at their last
scheduled assessments of at least 6 months. Two additional Cohort 4
patients experienced a gain between +2 and +4 letters from their
baseline values. One Cohort 4 patient measured 6 letters below
baseline. Previously reported structural improvements in the
retina, decreases in drusen density, and a trend toward slower GA
progression in treated compared to untreated eyes continued to be
present. Overall, OpRegen has been well tolerated with no
unexpected adverse events or serious adverse events. 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.
Three patients with evidence of retinal restoration and
confirmed history of GA growth continue to demonstrate areas of
retinal restoration as of their last assessment, ranging from 6
months to approximately 3 years after treatment. Notably, on
Optical Coherence Tomography (OCT) analyses, the first Cohort 4
patient with evidence of retinal restoration and confirmed history
of GA growth, has demonstrated zero growth in the area of atrophy
(GA) almost 3 years following treatment with OpRegen. This is
unprecedented due to the progressive nature of the disease.
Applying the expected rate of progression based on changes from
historical images to the baseline assessment, the size of the
atrophic lesion at the visit would have been 4.58 mm using square
root transformation (SQRT). Instead, the lesion measured at 2.8 mm
SQRT, which was the same as it was at baseline, meaning there was
no GA growth over almost 3 years, representing a 63.6% (1.78 mm
SQRT) smaller area of GA than was expected. The area of GA in this
patient’s untreated fellow eye, which was less severely impacted at
baseline, progressed as expected from 3.63 mm to 4.01 mm using
SQRT, an increase of 10.5% or 0.38 mm. Notably, microperimetry data
collected at the Year 2 and Year 3 post-treatment study visits
indicated improvements in the patient’s ability to discern
different intensities of light and the patient has experienced
clinically significant improvement in visual acuity for more than 2
years, at one point gaining 12 letters on an ETDRS scale. In
addition to positive anatomical changes, all three patients with
evidence of retinal tissue restoration had visual acuity increases
above baseline levels within 9 months post-treatment and visual
acuity has remained relatively stable over time compared to their
fellow eyes.
Outer retinal layer restoration, which was observed using
clinical high-resolution OCT, 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. These findings 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 GA.
These findings of retinal restoration have been confirmed
utilizing multiple imaging technologies. The use of multiple
imaging modalities differs from traditional assessment of GA
progression, which employs only fundus autofluorescence (FAF) to
assess changes in the total surface area of the apparent GA over
time. Using only FAF may fail to identify structural changes that
can be observed only with the addition of OCT imaging. 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.
Improvements in Visual Acuity Observed with OpRegen RPE
Transplant. The graph represents the mean changes in Best Corrected
Visual Acuity (BCVA) for Cohort 4 patients’ treated eye when
compared to their fellow eye over a period of 12 months.
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, plans to meet with the FDA
in 2021 to discuss OpRegen’s clinical development, and Lineage’s
potential to become the pre-eminent allogeneic cell therapy
company. 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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