- Improved Visual Acuity Continues to be Observed in Cohort 4
Patients
- First Known Clinical Report of Retinal Tissue Regeneration
Persisted to 23 Months with Further Improvement in Visual
Acuity
- Patient Enrollment Recently Completed
- Therapeutic Expert Call with Principal Investigator
Christopher D. Riemann, M.D. Scheduled for November 17, 2020 at
4:00 pm Eastern Time
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing three novel cell
therapies for serious medical conditions, today announced positive
interim results from the ongoing 24-patient Phase 1/2a clinical
study of Lineage’s 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). At AAO, new data were presented on 20
patients, including 8 patients treated in Cohort 4, which feature
better baseline vision and smaller areas of GA. All 8 of these
patients were treated with a new “thaw-and-inject” formulation of
OpRegen and 4 were treated using the Gyroscope Orbit Subretinal
Delivery System (Gyroscope SDS). Data presented at AAO showed
improvements in visual acuity in Cohort 4 patients, with treated
versus fellow eye comparisons reaching statistical significance at
9 and 12 months following OpRegen administration. These
improvements were maintained for up to 24 months in some patients.
A trend towards slower GA growth was observed in the first 6 Cohort
4 patients, a trend maintained for as long as 24 months in patients
with 24-month data available. Previously reported structural
improvements in the retina and decreases in drusen density have
continued with evidence of durable engraftment of OpRegen cells in
treated patients, some more than 4 years following administration,
with no immunosuppression utilized beyond the perioperative period.
Overall, OpRegen appears to be well-tolerated in all patients
treated to date. The final four patients in the study were treated
during November and will provide additional visual acuity data in
the coming months.
“These new data increasingly suggest to us that treatment with
OpRegen can provide clinically meaningful outcomes in dry AMD
patients with GA, particularly for those with earlier-stage
disease,” stated Brian M. Culley, Lineage CEO. “According to a
recent survey published in Investigative Ophthalmology & Visual
Science, only 27% percent of retinal specialists believed patients
with visual acuity of 20/200 or worse could benefit from treatment
with an agent which slows the growth of GA, while 93-99% of them
believed patients with visual acuity of 20/200 or better could
benefit from this approach. This is consistent with our belief that
recent data from our Cohort 4 patients, which have less advanced
disease and better baseline vision, are more exciting and provide a
better surrogate for the potential clinical and commercial
opportunity for OpRegen.”
Mr. Culley added, “In addition to reporting the first known
finding of anatomical restoration of retinal tissue, which has
persisted below baseline for 23 months and counting, treatment with
OpRegen continues to demonstrate other benefits in some patients,
including increases in visual acuity, reductions in the growth rate
of GA and increases in reading speed. These are additive to the
improvements we previously reported in retinal architecture and
drusen reduction. Further, the multi-year durability of transplants
without rejection is notable for our allogeneic cell therapy
approach, especially as patients did not require long-term
immunosuppression. With enrollment recently completed, our focus
turns next toward collecting safety and efficacy data on the most
recently treated patients, advancing partnership and investor
discussions we’ve been having, exploring our options for
later-stage clinical development, and speaking with the FDA about
next steps. Our objective is to position the OpRegen program as a
front-runner in the race to address an unmet need in what is widely
expected to be a multi-billion-dollar dry AMD therapeutic market
and to drive Lineage forward as the pre-eminent allogeneic cell
therapy company.”
OpRegen Data Update & Highlights from the AAO
Presentation (data presented on 20 patients, dosed through October
5, 2020):
- Continued progressive functional improvement.
- In Cohort 4, 6 out of 7 (86%) of patients’ treated eyes
measured above their baseline vision (Best Corrected Visual Acuity,
or BCVA) at 12 months, a clinically relevant timeframe, or as of
the longest available timepoint less than 12 months (data
collection continues for more recently-treated patients).
- Data to date demonstrate a localized slowing of GA progression
in the treated areas with a trend towards slower GA growth in
treated versus fellow eyes in pooled analyses.
- Long-term engraftment is supported with imaging
observations up to more than 4 years, even with a short
immunosuppression regimen.
- In all Cohort 4 patients receiving OpRegen TAI formulation, per
protocol, immunosuppressants have been discontinued as scheduled,
typically within 90 days post-operatively, and no cases of acute or
delayed rejection or inflammation have been reported.
- One Cohort 4 patient was treated only with mycophenolate
mofetil and received no tacrolimus for immunosuppression.
- Anatomical restoration of retinal tissue.
- A Cohort 4 patient with evidence of retinal restoration and
confirmed history of GA growth, which was first reported at 9
months, continues at month 23 to have an area of GA smaller than at
baseline.
- This patient also experienced additional improvement in BCVA
from 9 to 23 months post-treatment, while the untreated eye has
experienced further reduction in visual acuity.
- Long-term monitoring on this patient is expected to
continue.
- Treatment overview.
- As of October 5, 2020, 16 patients were treated via pars planar
vitrectomy (PPV), while 4 were treated with the Gyroscope SDS.
- As of November 10, 2020, 17 patients were treated via PPV,
while 7 were treated with the Gyroscope SDS.
- Enrollment in the phase 1/2a study is complete; follow-up
continues for safety and efficacy.
- Safety and tolerability.
- The primary objective of the study is to evaluate the safety
and tolerability of OpRegen at 12 months, and in patients which
have reached this time point OpRegen appears well tolerated.
- There have been no unexpected adverse events (AEs) or
treatment-related systemic serious AEs reported in enrolled
patients.
- The most common and expected ocular AEs were the formation or
exacerbation of mild to moderate epiretinal membranes (ERMs) and a
single report of a retinal detachment, with cause unknown (all
occurring in patients receiving OpRegen via the PPV route of
administration).
- The Gyroscope SDS is an alternative to the PPV route and is
designed to avoid ERM formation.
- Through October 2020, 16 patients were treated via PPV while 4
were treated with the Gyroscope SDS. ERMs were observed in 13 PPV
patients.
- One patient treated with the Gyroscope SDS developed a mild
choroidal neovascularization (CNV) at the site of needle
penetration 6 months post-treatment which was successfully treated
with a single dose of an approved anti-VEGF agent. The cause was
unknown.
- One patient treated via PPV developed a mild CNV at > 24
months post-treatment.
- Other changes observed following OpRegen treatment persisted
through the last time point examined (> 4 years in some
patients), including subretinal pigmentation and hyper-reflective
areas seen on optical coherence tomography (OCT).
The results were presented at the 2020 American Academy of
Ophthalmology Annual Meeting (AAO 2020). The presentation, “Phase
1/2a Study of Subretinally Transplanted hESC-Derived RPE Cells in
Advanced Dry-Form AMD Patients” was featured as part of the
Original Paper Session, OP02V Retina, Vitreous Original Papers on
November 15, 2020 and was presented by Christopher Riemann,
M.D.
KOL Call and Webcast
Lineage will host a therapeutic area expert call with
Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship
Director, Cincinnati Eye Institute and University of Cincinnati
School of Medicine, to discuss the interim results on November 17,
2020 at 4:00 pm Eastern Time / 1:00 p.m. Pacific Time. Interested
parties can access the event on the Events and Presentations
section of Lineage’s 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. In total, 17 patients
were treated via PPV, while 7 were treated with the Gyroscope SDS.
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. Additionally, for the
patients in Cohort 4 that receive subretinal delivery of OpRegen
utilizing the Gyroscope SDS, objectives will include the evaluation
of the safety of delivery of OpRegen using the Gyroscope SDS.
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
development plans for OpRegen and post-enrollment timing
expectations. 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 (the 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 Annual Report on Form 10-K filed with the SEC
on March 12, 2020 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/20201116005438/en/
Lineage Cell Therapeutics, Inc. IR Ioana C. Hone
(ir@lineagecell.com) (442) 287-8963
Solebury Trout IR Gitanjali Jain Ogawa
(Gogawa@troutgroup.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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