- Seventy-Five Percent of All Cohort 4 Patients Have
Experienced BCVA Increases
- Visual Acuity Continues to Decline in the Majority of
Untreated Eyes
- No Acute or Delayed Inflammation or Rejection of OpRegen
Observed, Even in Patients Treated with a Reduced Immunosuppressive
Regimen
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing novel cell
transplants for serious medical conditions, today announced new
positive 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 to the subretinal space
for the treatment of dry age-related macular degeneration (AMD)
with geographic atrophy (GA). Additional interim data were
collected on all 24 patients enrolled in the study, including the
12 patients treated in Cohort 4, which feature less advanced
disease, better baseline visual acuity, and smaller areas of
GA.
Overall, 9/12 (75%) 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 3 months to > 2
years post- transplant. Improvements in best corrected visual
acuity (BCVA) reached up to +19 letters on an EDTRS chart. In
contrast, 9/12 (75%) of the patients’ untreated eyes were below
baseline entry values at the same time points. Among the newly
reported data, three (50%) of the more recently treated Cohort 4
patients have exhibited marked improvements in BCVA ranging from +7
to +16 letters at their last scheduled assessments of at least 4.5
months. Two additional Cohort 4 patients remained within 2 letters
of their baseline values (one each above and below). One patient
measured 7 letters below baseline.
Previously reported structural improvements in the retina and
decreases in drusen density have continued with evidence of durable
engraftment of OpRegen cells in some treated patients now extending
to more than 5 years in the earliest treated patients. A trend
towards slower GA progression in treated compared to fellow eyes
also continued, although significant changes in GA growth over a
3-month period following treatment are not expected. Overall,
OpRegen has been well tolerated with no unexpected adverse events
or serious adverse events.
“Data collected from the six additional Cohort 4 patients which
we treated last fall has reinforced our prior results and further
supports that treatment with OpRegen may provide clinically
meaningful outcomes in dry AMD patients with GA. Improvements in
BCVA have become apparent within a few months after dosing,
consistent with the predicted activity of an RPE cell transplant,”
stated Brian M. Culley, Lineage CEO. “If these early indications of
a treatment effect are maintained or improve further, it will be
another positive indicator for the potential of OpRegen to improve
outcomes in this condition. We continue to monitor all patients on
study and in the coming months we will be looking in particular for
indications of retinal restoration, reductions in the size and
growth of the areas of GA, and functional improvement in visual
acuity. Further, the multi-year stability of OpRegen transplants,
some in excess of 5 years without signs of rejection, is notable
for the durability of our allogeneic cell therapy approach,
especially as patients did not require long-term
immunosuppression.”
As part of an ongoing effort to administer the minimally
effective dose and duration of immunosuppression, reflecting the
COVID pandemic and age of typical AMD patients while ensuring the
survival of OpRegen cells, no immunosuppression was utilized beyond
the perioperative period of up to 3 months in Cohort 4 patients.
Notably, the one OpRegen patient who had received a modified
immunosuppressive regimen at baseline which included no tacrolimus
and only mycophenolate mofetil, does not show any signs of acute or
delayed inflammation or rejection of OpRegen cells. One other
patient was diagnosed with COVID shortly after treatment with
OpRegen and all immunosuppression was halted and then reinstated
once the patient was asymptomatic. This second patient similarly
showed no signs of acute or delayed inflammation or rejection of
OpRegen cells. Other than the reduced regiments described above,
immunosuppressants have been discontinued as scheduled, typically
within 90 days post-operatively, and no cases of acute or delayed
rejection or inflammation due to OpRegen have been reported.
Additional details regarding this data will be presented as part
of a corporate update by Mr. Culley at the Benzinga Global Biotech
Small Cap conference on March 24, 2021 at 11:50am Eastern Time /
8:50am Pacific Time. Mr. Culley will also be participating in a
panel entitled “Coming Together to Address Unmet Medical Needs,” on
March 24, 2021 at 12:50pm Eastern Time / 9:50am Pacific Time.
Interested investors are encouraged to register for the event in
advance: https://www.benzinga.com/events/small-cap/biotech/. The
live and archived webcasts from the event will be available on the
Events and Presentations section of Lineage’s website. Additional
videos are available on the Media page of the Lineage website.
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 (vision from 20/65
to 20/250 with smaller 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. Additional objectives include the
evaluation of the safety of delivery of OpRegen using the Orbit
SDS, manufactured by Gyroscope Therapeutics, Ltd.
OpRegen is a registered trademark of Cell Cure Neurosciences
Ltd., a majority-owned subsidiary of Lineage Cell Therapeutics,
Inc.
About Dry AMD
Dry age-related macular degeneration (AMD) is a leading cause of
adult blindness in the developed world. There are two forms of AMD:
wet AMD and dry AMD. Dry AMD is the more common of the two types,
accounting for approximately 85-90% of cases. Wet AMD is the less
common of the two types, accounting for approximately 10-15% of
cases. Global sales of the two leading wet AMD therapies were in
excess of $10 billion in 2019. Nearly all cases of wet AMD begin as
dry AMD. Dry AMD 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
dry 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) VAC, an allogeneic dendritic cell therapy platform for
immuno-oncology and infectious disease, currently in 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,” “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
expected clinical outcomes of dry AMD patients with GA and the
expected timing when indications of retinal and reductions in size
and expansion of the areas of GA may become apparent.
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 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/20210323005436/en/
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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