Statistically Significant Evidence of a
Treatment Effect with OpRegen Observed in Cohort 4 Patients
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing allogeneic cell
therapies for unmet medical needs, reported today that updated
interim results from a Phase 1/2a clinical study of its lead
product candidate, OpRegen®, an investigational retinal pigment
epithelium cell transplant therapy currently in development for the
treatment of dry age-related macular degeneration (AMD), were
featured in a podium presentation at the 54th Annual Scientific
Meeting of the Retina Society. The presentation, “Phase 1/2a
Clinical Trial of Transplanted Allogeneic Retinal Pigmented
Epithelium (RPE, OpRegen) Cells in Advanced Dry Age-Related Macular
Degeneration (AMD): Interim Results,” was presented by Christopher
D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director,
Cincinnati Eye Institute (CEI) and University of Cincinnati School
of Medicine.
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Statistically Significant Evidence of
Treatment Effect with OpRegen RPE Transplant Over Time (Graphic:
Lineage Cell Therapeutics)
Patients enrolled into the clinical study all had bilateral,
advanced, atrophic AMD. OpRegen was transplanted into the
subretinal space, near or across the area of geographic atrophy
(GA) of their worse seeing eyes, and the patients were routinely
followed as scheduled per protocol. Data presented today showed
that as patients continued to progress into post-operative
follow-up, eyes receiving OpRegen trended toward improvement in
visual acuity, a secondary objective under the study, while their
untreated eyes typically lost visual acuity, as expected with this
progressive disease. As additional patients have reached longer
periods post-treatment, differences in visual acuity between
treated and untreated eyes across Cohort 4 patients became
statistically significant beginning at month 9 (P = 0.0085), as
well as months 12 (P = 0.0220) and 15 (P = 0.0273) as determined
via 2-sided Wilcoxon Signed Rank (using NCSS, LLC statistical
software). These results, when combined with the previously
reported data from detailed analyses of multimodal imaging,
including optical coherence tomography (OCT), which showed evidence
of retinal restoration in areas previously considered to be
atrophic, suggest that both a structural and functional benefit is
possible with OpRegen therapy. Additionally, OpRegen continues to
be well tolerated and there have been no new, unexpected ocular or
systemic adverse events or serious adverse events.
“This is an exciting time for the OpRegen program, the
participating investigative sites, and for patients suffering from
dry AMD. The efficacy findings presented today are both
statistically significant as well as clinically important,” stated
Christopher D. Riemann, M.D. “Prior clinical data readouts were
mostly descriptive and lacked enough patients and post-treatment
follow-up to allow for detailed statistical analyses. A significant
number of better vision Cohort 4 patients are now 9, 12, and even
15 months post-treatment with OpRegen. As a result, we were able to
analyze the available results to ascertain if a detectable efficacy
signal could be observed in a relatively small number of patients.
Though this early phase trial is open label, non-randomized and not
placebo controlled, the significant differences in visual acuity of
patients’ OpRegen treated eyes compared to their fellow, untreated
eyes over time is very important. Though larger, controlled studies
are needed, OpRegen is a potentially transformational therapy for
these patients without any approved treatment options. These
results strongly support further late-stage development, and we are
eager to begin those studies.”
“As more patients in the OpRegen trial reach clinically relevant
observation periods, our data set grows larger and permits us to
conduct additional analyses like the one reported today. These new
data support our view that our cell transplant approach can deliver
not only anatomical changes detectable by imaging studies, but also
durable functional benefits to visual acuity,” stated Brian M.
Culley, Lineage CEO. “OpRegen already is well-positioned among
product candidates in development for dry AMD as the only one that
has demonstrated an ability to halt or reverse the expansion of
geographic atrophy and we are additionally reporting statistically
significant differences in visual acuity over time between treated
and untreated eyes in our intended patient population. We are
excited to continue developing OpRegen as a potential treatment for
dry AMD with GA.”
Dr. Riemann’s presentation will be available on the Events and
Presentations section of Lineage’s website.
The Retina Society was founded in 1968 exclusively for
educational and scientific purposes concerning the diagnosis, care
and treatment of diseases and injuries to the retina. For more
information on the Retina Society or its annual scientific meeting,
please visit https://www.retinasociety.org/ or follow the
association on Twitter @RetinaSociety.
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’s 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 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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