- Eighty-Three Percent of All Cohort 4 Patients Exhibited
Stable or Improved BCVA
- Visual Acuity Declined in the Majority of Untreated
Eyes
- Positive Interim Outcomes on Patient-Reported Visual
Function Questionnaire
- No Acute or Delayed Inflammation or Rejection Events, Even
in Patients Receiving Reduced Immunosuppressive Regimen
- Previously Reported Evidence of Retinal Restoration Has
Persisted to Month 35
- Data Reported at 2021 ARVO Meeting
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing novel cell
therapies for unmet medical needs, announced today that updated
interim results from its ongoing, 24-patient Phase 1/2a clinical
study of its lead product candidate, OpRegen, were reported at the
2021 Association for Research in Vision and Ophthalmology Annual
Meeting (ARVO 2021). 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). At ARVO 2021, additional data were presented on 24 patients
enrolled in the study, including all 12 patients treated in Cohort
4, which have better baseline vision and smaller areas of GA than
earlier cohorts.
“I continue to be very excited about this work and the clinical
data generated to date with OpRegen, especially in the better
vision Cohort 4 patients,” stated Principal Investigator
Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship
Director, Cincinnati Eye Institute and University of Cincinnati
School of Medicine. “There seems to be a significant visual acuity
signal in Cohort 4 patients, with most treated eyes having stable
or improved vision over time when compared to the contralateral
eyes having stable or worsening vision over time. When looking at
reading speed progression, treated eyes also seemed to improve
while untreated eyes declined. Notably, some individual responders
had impressive visual acuity improvements and reductions in GA
progression compared to their contralateral eyes. Most importantly,
we believe that earlier intervention in less severely affected
patients along with a more central placement of transplanted
OpRegen cells may increase the likelihood of a clinically
beneficial effect. Overall, these results are encouraging and are
of a magnitude that could be clinically very important if confirmed
in further clinical studies.”
“These new data continue to indicate to us that treatment with
OpRegen can generate clinically meaningful outcomes in dry AMD
patients with GA, particularly in those with earlier-stage
disease,” stated Brian M. Culley, Lineage CEO. “It also appears
that earlier intervention in less severely affected patients and
more central placement of the transplanted cells may increase the
likelihood of observing a benefit. Additionally, among the newly
reported data, it was notable that Cohort 4 patients reported
improvements in a majority of the vision parameters measured by a
validated quality of life questionnaire. The magnitude of these
improvements was higher overall in Cohort 4 than in Cohorts 1
through 3, which is consistent with the larger clinical benefit
observed among those patients. As this data set matures, our
efforts turn next to evaluating the six most recently treated
Cohort 4 patients for indications of retinal restoration and
reductions in the size and growth of the areas of GA. Our overall
objective is to position OpRegen RPE transplants as the clear
leader 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.”
Updated results presented at ARVO 2021 included a minimum of 4.5
months of follow-up in all 24 patients treated with OpRegen. Nine
of twenty-four patients were treated with the “thaw and inject”
formulation of OpRegen, two via a standard pars plana vitrectomy
(PPV) and seven utilizing the Orbit™ Subretinal Delivery System
(Orbit SDS).
Overall, 10/12 (83%) 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 4.5 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 newly reported data, three (50%) of the more
recently treated Cohort 4 patients 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 experienced a gain of 2 letters from their baseline
values. One Cohort 4 patient measured 7 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.
Overall, OpRegen has been well tolerated with no unexpected adverse
events or serious adverse events, and evidence of durable
engraftment of OpRegen RPE cells have extended to more than 5 years
in earliest treated patients, supporting the potential for OpRegen
to be a one-time treatment.
A Cohort 4 patient with evidence of retinal restoration and
confirmed history of GA growth, which was first reported 9 months
following treatment, continues to demonstrate areas of retinal
restoration as of their last assessment, approximately 3 years
after treatment.
2021 ARVO Presentation OpRegen Data
Highlights (As of April 16, 2021):
- In Cohort 1-3 patients (all legally blind at baseline), visual
acuity reductions occurred as expected due to progressive GA;
- In Cohort 4 patients, which collectively had smaller areas of
GA and higher baseline BCVA as compared to Cohort 1-3 patients,
improved or sustained BCVA has been observed in 10/12 (83%)
patients as of their last visit prior to this update (range of -7
to +19 letters on the ETDRS chart);
- OpRegen continues to be well-tolerated in all treated patients
(N = 24);
- The majority of adverse events were mild (87%);
- Sustained subretinal pigmentation continues to suggest
multi-year durability of OpRegen transplants;
- Improved anatomy and function continue to be observed in some
patients, including:
- Reduction in drusen;
- Photoreceptor and RPE layer restoration;
- Localized slowing of GA progression in treated areas;
- Better visual acuity via ETDRS scores and reading speed;
and
- Improved National Eye Institute Visual Function Questionnaire
(VFQ-25) scores.
- Post-treatment surgical interventions occurred in four cases (5
events in 4 patients):
- Three epiretinal membranes (ERM) were surgically peeled. Mild
to moderate ERM were observed in an additional 12 out of 17 PPV
operated patients. Most ERMs were clinically insignificant.
- Retinal detachment (RD) was observed in 2 out of 17 patients,
neither of which appears to be attributable to OpRegen or any study
related medications:
- The first case of RD, which occurred in a Cohort 3 patient, was
an unsuccessful repair of a post-surgical retinal tear; visual
acuity did not regain baseline levels; and
- The second case of RD, which occurred in a Cohort 4 patient,
was successfully repaired; post-surgical visual acuity has remained
higher than baseline.
- Choroidal neovascularization (CNV) was observed in 3 out of 7
patients receiving OpRegen via the Orbit SDS, all of whom received
treatment with an approved anti-VEGF;
- As previously reported, one PPV operated patient developed CNV,
which was identified more than two years following treatment.
As part of an ongoing effort to administer the minimally
effective dose and duration of immunosuppressive therapy,
immunosuppression was utilized only during the perioperative period
of approximately 3 months in Cohort 4 patients. One patient
received a modified immunosuppressive regimen at baseline, which
included no tacrolimus and only mycophenolate mofetil. One patient
was diagnosed with COVID-19 shortly after treatment for whom all
immunosuppression was halted and reinstated once the patient was
asymptomatic. Both patients showed no signs of acute or delayed
inflammation or rejection of OpRegen cells with 4.5 months of
post-transplant follow up. Other than the reduced regimens
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 in any patients treated with OpRegen.
The presentation, “Phase I/IIa Clinical Trial of Transplanted
Allogeneic Retinal Pigmented Epithelium (RPE, OpRegen) Cells in
Advanced Dry Age-Related Macular Degeneration (AMD): Interim
Results” is being featured as part of the Stem Cells/Gene
Therapy/Transplantation Session, on May 6, 2021 between 5:15 pm and
6:45 pm EDT by Christopher D. Riemann, M.D.(abstract number
3538173).
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 (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. OpRegen is a registered
trademark of Cell Cure Neurosciences Ltd., a majority-owned
subsidiary of Lineage Cell Therapeutics, Inc. The Orbit subretinal
delivery system is used under agreement with Gyroscope Therapeutics
Limited. Orbit and Orbit SDS are trademarks of Gyroscope
Therapeutics Limited.
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 or European Medicines Agency
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 treatment with OpRegen in dry AMD
patients with GA. 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/20210503005289/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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