Lineage Cell Therapeutics Provides Update on SCiStar Clinical Study and OPC1 Spinal Cord Injury Program
November 15 2019 - 8:00AM
Business Wire
- OPC1 Overall Safety Profile Excellent with Motor Recovery
Gains in Upper Extremities Maintained Through Year 2 Follow-Ups
Available to Date
- OPC1 Manufacturing Fully Transferred to cGMP Facility;
Improvements to Manufacturing Process Planned During 2020
- FDA Meeting to Discuss OPC1 Manufacturing and Clinical
Development Planned for Mid-2020
Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX),
a clinical-stage biotechnology company developing novel cellular
therapies for unmet medical needs, today provided an update on
OPC1, the Company’s oligodendrocyte progenitor cell (OPC) therapy
currently being tested in a Phase I/IIa clinical trial, the SCiStar
Study, for the treatment of acute spinal cord injury (SCI). Lineage
reported positive results from the ongoing SCiStar study of OPC1,
where the overall safety profile of OPC1 has remained excellent
with robust motor recovery in upper extremities maintained through
Year 2 patient follow-ups available to date. Additionally, OPC1
manufacturing has been completely transferred to the Company’s cGMP
manufacturing facility in Israel and manufacturing process
improvements are planned to continue throughout 2020. Moreover,
Lineage intends to meet with the U.S. Food and Drug Administration
(FDA) to discuss further development of the OPC1 program around the
middle of 2020.
“We remain extremely excited about the potential for OPC1 to
provide enhanced motor recovery to patients with spinal cord
injuries. We are not aware of any other investigative therapy for
SCI which has reported as encouraging clinical outcomes as OPC1,
particularly with continued improvement beyond 1 year,” stated
Brian M. Culley, CEO of Lineage Cell Therapeutics. “Overall gains
in motor function for the population assessed to date have
continued, with Year 2 assessments measuring the same or higher
than at Year 1. For example, 5 out of 6 Cohort 2 patients have
recovered two or more motor levels on at least one side as of their
Year 2 visit whereas 4 of 6 patients in this group had recovered
two motor levels as of their Year 1 visit. To put these
improvements into perspective, a one motor level gain means the
ability to move one’s arm, which contributes to the ability to feed
and clothe oneself or lift and transfer oneself from a wheelchair.
These are tremendously meaningful improvements to quality of life
and independence. Just as importantly, the overall safety of OPC1
has remained excellent and has been maintained 2 years following
administration, as measured by MRI’s in patients who have had their
Year 2 follow-up visits to date. We look forward to providing
further updates on clinical data from SCiStar as patients continue
to come in for their scheduled follow up visits.”
SCiStar Study Clinical Update
- Overall safety profile of OPC1 to date is excellent for
Year 2 follow-ups available to date (21 patients)
- Magnetic resonance imaging (MRI) scans at 24 months
post-injection of OPC1 have shown no evidence of adverse changes in
any of the 21 SCiStar study patients treated with OPC1 who have had
their Year 2 follow-up visits.
- To date, there have been no unexpected serious adverse events
(SAEs) related to the OPC1 cells in these patients, with no
concerning safety issues noted.
- No SCiStar patient had a decline in their motor function from
their Year 1 to Year 2 visits.
- No adverse findings were observed on follow-up MRI scans.
- Motor level improvements
- For Cohort 1, even at a low dose of OPC1, patients continue to
be stable 2-4 years out post treatment.
- Five of six Cohort 2 patients achieved at least two motor
levels of improvement over baseline on at least one side as of
their 24-month follow-up visit.
- In addition, one Cohort 2 patient achieved three motor levels
of improvement on one side over baseline as of the patient’s
24-month follow-up visit; improvement has been maintained through
the patient’s 36-month follow-up visit.
- Improvements in upper extremity motor function are being
measured using the International Standards for Neurological
Classification of Spinal Cord Injury (ISNCSCI) scale, widely used
to quantify functional status of patients with spinal cord
injuries. Improvements in upper extremity motor function are the
most desirable functional improvement target in the quadriplegic
population, since even relatively modest changes can potentially
have a significant impact on functional independence, quality of
life, and cost of care.
- Upper Extremity Motor Score (UEMS)
- Impressive improvements in UEMS scores for Cohort 2 patients
was observed at 24 months, with three patients who have recovered
back to a UEMS in the mid-to-high 40’s. The maximum total UEMS is
50.
- The UEMS is a linear scale used to quantify motor function at
each of five upper extremity muscle groups driving arm and hand
function; these scores are also used to determine "motor levels",
which define the level within the cord above which a patient has
normal function.
OPC1 Clinical Program Update
- OPC1 manufacturing has now completely been transferred to the
Company’s cGMP manufacturing facility in Israel.
- Continued improvements to the manufacturing process are planned
during 2020 and include enhancements to the production process to
ensure robust, controlled and commercially viable scale, purity,
and reproducibility of OPC1.
- A meeting with the FDA is planned around the middle of 2020 to
discuss the Company’s proposed manufacturing improvements and the
further development of OPC1 to best set the program up for success
moving forward.
- Concurrently, Lineage will work to expand its partnerships with
spinal cord injury advocacy and support organizations to support
their mission to accelerate stem cell treatments to patients with
unmet medical needs and fast-track the development of the most
promising stem cell technologies.
About the SCiStar Clinical Study
The SCiStar Study is an open-label, 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 25
patients with subacute motor complete (AIS-A or AIS-B) cervical
(C-4 to C-7) acute spinal cord injuries (SCI). These individuals
had essentially lost all movement below their injury site and
experienced severe paralysis of the upper and lower limbs. AIS-A
patients had lost all motor and sensory function below their injury
site, while AIS-B patients had lost all motor function but may have
retained some minimal sensory function below their injury site. 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 as measured by upper extremity motor scores
and motor level on International Standards for Neurological
Classification of Spinal Cord Injury (ISNCSCI) examinations at 30,
60, 90, 180, 270, and 365 days after injection of OPC1.
About OPC1
OPC1 is an oligodendrocyte progenitor cell (OPC) therapy
currently being tested in a Phase I/IIa clinical trial known as
SCiStar for the treatment of acute spinal cord injuries. 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.
The clinical development of 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 for the treatment of acute SCI
and has been granted Orphan Drug designation from the U.S. Food and
Drug Administration (FDA).
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 proprietary cell-based therapy
platform and associated 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 either to 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 assets include (i)
OpRegen®, a retinal pigment epithelium transplant therapy in Phase
I/IIa 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
I/IIa development for the treatment of acute spinal cord injuries;
and (iii) VAC2, an allogeneic cancer immunotherapy of
antigen-presenting dendritic cells currently in Phase I development
for the treatment of non-small cell lung cancer. Lineage is also
evaluating potential partnership opportunities for Renevia®, a
facial aesthetics product that was recently granted a Conformité
Européenne (CE) Mark. 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 planned
manufacturing process improvements and meetings with regulatory
agencies. 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 14, 2019 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/20191115005079/en/
Lineage Cell Therapeutics, Inc. IR Ioana C. Hone
(ir@lineagecell.com) (510) 871-4188
Solebury Trout IR Gitanjali Jain Ogawa
(Gogawa@troutgroup.com) (646) 378-2949
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