- 12-Month Primary Endpoint Data Suggest OpRegen is Well
Tolerated with an Acceptable Safety Profile
- Preliminary Evidence of Visual Function and Outer Retinal
Structure Improvements Observed in Cohort 4 Patients with GA and
Impaired Vision
- Data Reported at 2022 ARVO Meeting by Allen C. Ho, M.D.,
FACS
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing allogeneic cell
therapies for unmet medical needs, today announced that results
from the primary endpoint, safety and tolerability 1 year
post-transplant, in the ongoing Phase 1/2a clinical study of RG6501
(OpRegen), a retinal pigment epithelial cell therapy currently in
development for the treatment of geographic atrophy (GA) secondary
to age-related macular degeneration (AMD), were presented yesterday
at the 2022 Association for Research in Vision and Ophthalmology
Annual Meeting (ARVO 2022). The presentation, “Safety and Efficacy
of a Phase 1/2a Clinical Trial of Transplanted Allogeneic Retinal
Pigmented Epithelium (RPE, OpRegen) Cells in Advanced Dry
Age-Related Macular Degeneration (AMD)” was featured as part of the
Retinal Prostheses and Transplantation Session, by Allen C. Ho,
M.D., FACS, Wills Eye Hospital Attending Surgeon and Director of
Retina Research, Professor of Ophthalmology, Thomas Jefferson
University, Mid Atlantic Retina and President, The Retina Society
(abstract number 3714956). RG6501 (OpRegen) is currently being
developed under an exclusive worldwide collaboration between
Lineage, Roche and Genentech, a member of the Roche Group.
“These data, though uncontrolled, offer the promising
demonstration that OpRegen may be able to impact GA disease
progression in a clinically meaningful manner, particularly when
delivered on-target and in earlier disease, in patients afflicted
with what was previously thought to be an inevitably progressive
disease,” stated Dr. Ho. “Obviously, larger, controlled studies are
required, but spontaneous disease regression does not occur in GA
so these results suggest that OpRegen may be a potentially
transformational therapy and strongly support further
development.”
2022 ARVO Presentation
Highlights
Summary of Safety Results
- All 24 treated patients reported at least one adverse event
(AE) and at least one ocular AE
- The majority of AEs reported with OpRegen were mild (Cohort
1-3, 87%; Cohort 4, 93%), and the immunosuppressive regimen was
well tolerated
- Ocular AEs observed with OpRegen were mainly related to the
surgical procedures used for subretinal delivery, with the most
common being conjunctival hemorrhage/hyperemia (n=17) and
epiretinal membrane (n=16)
- One patient discontinued the study due to an AE that was
unrelated to treatment
- No cases of rejection, acute or delayed intraocular
inflammation, or sustained increases in intraocular pressure
following OpRegen subretinal delivery have been reported
Summary of Activity Results
- Preliminary evidence of improvement in visual function was
observed in patients with GA and impaired vision at baseline
(Cohort 4 [n=12])
- Patients in Cohort 4 had an average 7.6 letter gain in visual
acuity at 12 months in the study eye
- Three patients in Cohort 4 (25%) had a 15 letter or greater
gain in visual acuity at 12 months in the study eye
- Five patients in Cohort 4 with OpRegen delivered to most or all
of the GA area, including the fovea, had greater gains in visual
function (average 12.8 letter gain), with evidence for regions of
apparent improvement of outer retinal structure as assessed by
SD-OCT
- The SD-OCT imaging analysis is ongoing
These data support the potential for OpRegen to slow, stop or
reverse disease progression in GA. Further assessment of the
optimal disease stage for intervention, surgical procedure for
subretinal delivery and target delivery location of OpRegen in a
larger, controlled clinical study is needed to confirm these
findings.
Dr. Ho’s presentation is available on the Events and
Presentations section of Lineage’s website.
The Association for Research in Vision and Ophthalmology, Inc.
(ARVO) was founded in 1928 in Washington, DC by a group of 73
ophthalmologists. ARVO is the largest and most respected eye and
vision research organization in the world. ARVO members include
nearly 11,000 researchers from over 75 countries. ARVO advances
research worldwide into understanding the visual system and
preventing, treating and curing its disorders. For more
information, please visit https://www.arvo.org/ or follow the
association on Twitter @ARVOInfo.
About OpRegen
OpRegen® is a retinal pigment epithelial cell therapy in
development for the treatment of geographic atrophy (GA) secondary
to age-related macular degeneration. Following subretinal delivery,
OpRegen has the potential to counteract RPE cell loss in areas of
GA lesions by supporting retinal structure and function. OpRegen is
being developed under a worldwide collaboration between Lineage,
Roche and Genentech, a member of the Roche Group.
About the Phase 1/2a Study
The Phase 1/2a study is an open-label, single-arm, multi-center,
dose-escalation trial evaluating a single administration of OpRegen
delivered subretinally in patients with bilateral GA. Twenty-four
patients were enrolled into 4 cohorts. The first 3 cohorts enrolled
only legally blind patients with a best corrected visual acuity
(BCVA) of 20/200 or worse. The fourth cohort enrolled 12 patients
with impaired vision (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 was 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 activity of OpRegen
treatment by assessing the changes in ophthalmological parameters
measured by various methods of primary clinical relevance.
About Geographic Atrophy
Geographic atrophy (GA) is an advanced form of
age-related macular degeneration (AMD) characterized by severe loss
of visual function. GA is a leading cause of adult blindness in the
developed world, affecting at least 5 million people globally.
There are two forms of advanced AMD: neovascular AMD and GA. GA and
neovascular AMD can occur simultaneously in the same eye, and
patients treated for neovascular AMD may still go on to develop GA.
GA typically affects both eyes. There are currently no U.S. Food
and Drug Administration (FDA) or European Medicines Agency (EMA)
approved treatment options available for patients with GA.
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 five allogeneic (“off-the-shelf”) product candidates: (i)
OpRegen, a retinal pigment epithelial cell therapy in Phase 1/2a
development for the treatment of geographic atrophy secondary to
age-related macular degeneration; (ii) OPC1, an oligodendrocyte
progenitor cell therapy in Phase 1/2a development for the treatment
of acute spinal cord injuries; (iii) VAC2, a 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 (iv) ANP1, an
auditory neuronal progenitor cell therapy for the potential
treatment of auditory neuropathy, and (v) PNC1, a photoreceptor
neural cell therapy for the treatment of vision loss due to
photoreceptor dysfunction or damage. 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,” “aim,” “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 collaboration and license agreement with Roche and
Genentech and activities expected to occur thereunder; and the
potential benefits of treatment with OpRegen and that OpRegen may
be a potential transformational therapy. 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, but
not limited to, the risk that positive findings in early clinical
and/or nonclinical studies of a product candidate may not be
predictive of success in subsequent clinical and/or nonclinical
studies of that candidate; the risk that competing alternative
therapies may adversely impact the commercial potential of OpRegen;
the risk that Roche and Genentech may not be successful in
completing further clinical trials for OpRegen and/or obtaining
regulatory approval for OpRegen in any particular jurisdiction; the
risk that Lineage may not be able to manufacture sufficient
clinical quantities of its product candidates in accordance with
current good manufacturing practice; risks and uncertainties
inherent in Lineage’s business and other risks discussed 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/20220503005172/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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