Lineage Cell Therapeutics Provides Update on OPC1 Cell Therapy Program for Spinal Cord Injury
June 25 2020 - 8:00AM
Business Wire
- Improvements Implemented in OPC1 Production Process
- Patent Position Strengthened to Protect the Processes,
Product, and Methods of Use
- Company is Evaluating Novel Delivery Devices to Enhance and
Ease Surgical Procedure
- FDA RMAT Meeting Planned by Year End to Discuss
Manufacturing and Clinical Development
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing novel cell
therapies for unmet medical needs, today provided an update on
OPC1, the Company’s oligodendrocyte progenitor cell (OPC) therapy
for the treatment of acute spinal cord injury (SCI). The OPC1
program was obtained through Lineage’s acquisition of Asterias
Biopharmaceuticals in March 2019, and manufacturing has been
completely transferred to the Company’s current Good Manufacturing
Practice (cGMP) facility in Israel, where key process improvements
have been developed and implemented. Lineage also has strengthened
its patent position in order to protect the OPC1 processes, product
and composition, and methods of use. Lineage intends to meet with
the U.S. Food and Drug Administration (FDA) to discuss further
development of the OPC1 program by the end of 2020.
“We have worked diligently over the past year to transition all
manufacturing activities for the OPC1 program to our in-house cGMP
facility, where our experienced cell therapy production team could
develop and deploy much-needed improvements and modernization to
the production and analytical processes,” stated Brian M. Culley,
Lineage CEO. “This work has achieved significantly better
efficiency and improved quality control, which we expect will
enable a consistent supply of material to support a late-stage
clinical trial of OPC1. With these necessary steps now completed,
our focus turns to developing a “thaw-and-inject” formulation and
superior delivery tools, to enable an easier surgical procedure and
facilitate faster enrollment in the next clinical trial. We also
are evaluating ways to return OPC1 to the clinic sooner than
originally planned, reflecting our view of compelling clinical data
which continues to read out from the 25-patient phase 1/2a SCiStar
study. Our approach with OPC1 replicates our development strategy
for OpRegen, our cell therapy program for dry AMD, in which we seek
not only to provide cell-based regenerative benefits, but also
commercially relevant solutions with competitive advantages in
areas of scale-up, production costs, and delivery techniques. By
analyzing every piece of the overall therapeutic landscape, we seek
to position ourselves as the clear leader in the emerging field of
cell therapy transplant medicine.”
Key OPC1 Program Milestones Achieved Since the Asterias
Acquisition Include:
- Technology transfer has been completed and cell production
processes have been established at Lineage’s cGMP cell therapy
facility;
- The production process was improved to achieve greater
efficiency and higher quality control, becoming compatible with
larger-scale manufacturing, and Biologics Review Application (BLA)
readiness;
- Multiple OPC1 batches have been successfully manufactured; GMP
production is planned to begin in early 2021;
- New master and working pluripotent cell banks have been
produced to supply consistent clinical and commercial batches of
OPC1;
- New in-process controls and release testing have been
introduced to the production and release process and have been
updated to be more compatible with current and expected future FDA
BLA review processes;
- Issuance of two new patents in 2019 related to methods for
utilizing pluripotent stem cell-derived OPCs for the treatment of
SCI and reducing cavitation in patients with acute SCI. Additional
patent applications are pending which may further protect the
processes, product and composition, and methods of use of
OPC1.
Key 2020 Milestone and Development Plans for the OPC1 Program
Include:
- Meeting with the FDA to discuss manufacturing improvements and
the design of a late-stage comparative clinical trial;
- Introducing a new “thaw-and-inject” formulation to enable
easier preparation and administration of cells to the spinal cord,
avoiding the need for washing and other dose preparation steps one
day prior to treatment and improving commercial positioning;
- Identifying a novel delivery system, to make the surgical
procedure easier, faster, and more compatible with a thaw and
inject formulation of OPC1, improving overall end-user experience
for the surgeon.
About OPC1
OPC1 is an oligodendrocyte progenitor cell (OPC) transplant
therapy designed to provide clinically meaningful improvements to
motor recovery in individuals with acute spinal cord injuries
(SCI). OPCs are naturally occurring precursors to the cells which
provide electrical insulation for nerve axons in the form of a
myelin sheath. SCI occurs when the spinal cord is subjected to a
severe crush or contusion injury and typically results in severe
functional impairment, including limb paralysis, aberrant pain
signaling, and loss of bladder control and other body functions.
There are approximately 18,000 new spinal cord injuries annually in
the U.S. and there currently are no FDA-approved drugs specifically
for the treatment of SCI. The OPC1 program has been partially
funded by a $14.3 million grant from the California Institute for
Regenerative Medicine. OPC1 has received Regenerative Medicine
Advanced Therapy (RMAT) designation and Orphan Drug designation
from the U.S. Food and Drug Administration (FDA).
About the OPC1 Clinical Study
The SCiStar Study of OPC1 is an open-label, 25-patient,
single-arm trial testing three sequential escalating doses of OPC1
which was administered 21 to 42 days post-injury, at up to 20
million OPC1 cells in patients with subacute motor complete (AIS-A
or AIS-B) cervical (C-4 to C-7) acute spinal cord injuries (SCI).
These individuals had experienced severe paralysis of the upper and
lower limbs. The primary endpoint in the SCiStar study was safety
as assessed by the frequency and severity of adverse events related
to OPC1, the injection procedure, and immunosuppression with
short-term, low-dose tacrolimus. Secondary outcome measures
included neurological functions measured by upper extremity motor
scores (UEMS) and motor level on International Standards for
Neurological Classification of Spinal Cord Injury (ISNCSCI)
examinations through 365 days post-treatment. Enrollment is
complete in this study; patients will continue to be evaluated on a
long-term basis.
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 and in
preclinical development for additional cancers and as a vaccine
against infectious diseases, including SARS-CoV-2, the virus which
causes COVID-19. 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 Lineage’s
plans for OPC1 development and clinical studies. 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/20200625005225/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
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