Lineage Cell Therapeutics Announces Vitelliform Maculopathy Patient Treated With OpRegen® Under Named Patient Compassionate ...
March 29 2021 - 8:00AM
Business Wire
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing novel cell
transplants for serious medical conditions, today announced that a
patient suffering from adult-onset vitelliform macular dystrophy
(AVMD) had recently been treated with its lead product candidate,
OpRegen, at Hadassah-Hebrew University Medical Center in Jerusalem,
using a named patient compassionate use approval granted by the
Israeli Ministry of Health. OpRegen is an investigational cell
therapy consisting of allogeneic retinal pigment epithelium (RPE)
cells administered to the subretinal space and is currently being
investigated in a 24-patient phase 1/2a clinical trial for the
treatment of dry age-related macular degeneration (AMD) with
geographic atrophy (GA).
“Lineage is pioneering a new branch of medicine, consisting of
the directed differentiation and transplant of specific cell types
to replace damaged or dying cells with the goal of restoring or
improving function lost to injury or disease,” stated Brian M.
Culley, Lineage CEO. “With OpRegen, we are transplanting new retina
cells to replace old cells that were lost or damaged to disease,
with a goal of providing stability or functional improvements to
vision. As outlined more fully on our recent earnings call, we
believe there are many potential applications of Lineage’s core
technology and intend this year to demonstrate that although we
currently have three clinical-stage product candidates, those
assets and our underlying platform may have utility in additional
settings. For example, our RPE cells may be useful for treating
additional retinal diseases, such as AVMD or Stargardt’s Disease.
Similarly, our spinal cord program may be applicable to other
conditions characterized by demyelination, and our oncology
platform may have application across many different tumor types,
depending on which antigen we elect to present to the patient’s
immune system.”
Mr. Culley continued, “In this first instance, we treated a
patient with AVMD, because it closely resembles dry AMD and
similarly involves impaired RPE function and progressive vision
loss. When the team at Hadassah approached us about treating their
existing AVMD patient with OpRegen on a compassionate use basis, we
were supportive of the request and saw it as an opportunity to
investigate a new application for our OpRegen product
candidate.”
This patient presented to the Department of Ophthalmology at
Hadassah-Hebrew University Medical Center in late December 2020
with sudden and severe visual acuity decreases in one eye. BCVA in
the worse vision eye was measured at 20/200, compared to 20/40 in
the patient’s contralateral eye. After an onset of blurred vision
in 2018, evaluation and imaging diagnosed the patient as suffering
from AVMD. Because AVMD is a disease of impaired RPE function
leading to atrophy and shares similar characteristics to dry AMD,
the team at Hadassah approached Lineage about the potential to
treat this patient on a compassionate use basis. Lineage submitted
a request on behalf of Hadassah-Hebrew University Medical Center
which was approved by the Israeli Ministry of Health. Following
approval from the University’s Ethics Committee, the patient was
treated in February 2021. The delivery of OpRegen RPE cells via
pars plana vitrectomy was successful, with no complications arising
during the procedure and the patient remains in follow-up.
About Adult-onset Vitelliform Maculopathy (AVMD)
AVMD is an eye disorder that can cause progressive vision loss
and usually begins after age 40. AVMD affects an area of the retina
called the macula, which is responsible for sharp central vision.
The condition causes a fatty yellow pigment to accumulate in cells
underlying the macula, eventually damaging the cells. Some people
remain without symptoms throughout their life while others may
slowly develop blurred and/or distorted vision, that can progress
to central vision loss over time. There is currently no effective
treatment for vitelliform macular dystrophy.
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. OpRegen is a registered trademark of
Cell Cure Neurosciences Ltd., a majority-owned subsidiary of
Lineage Cell Therapeutics, Inc.
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
potential conditions and diseases applicable to Lineage’s
clinical-stage product candidates. 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/20210329005210/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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