Living Cell Technologies Advancing Regenerative Cell Therapy
into Larger Phase IIb Study to Evaluate Its Potential as a
Disease-modifying Treatment
Company to Host Conference Call on Wednesday, June 17, at 3:30
p.m. Pacific Daylight Time
Living Cell Technologies Limited (LCT) today announced results
from a Phase I/IIa clinical study of NTCELL®, an experimental
regenerative cell therapy being studied as a disease-modifying
agent in Parkinson’s disease. The study, conducted in four patients
in New Zealand, met its primary endpoint of safety, showing NTCELL
implantation was well tolerated, with no adverse events considered
to be related to NTCELL. NTCELL implantation also improved clinical
features of Parkinson’s disease in the four patients studied, as
measured by validated neurological rating scales and
questionnaires, with the improvement sustained at 26 weeks
post-implant.
The study results will be presented at the 19th International
Congress of Parkinson’s Disease and Movement Disorders in San Diego
on Wednesday, June 17, by principal investigator Barry J. Snow,
MBChB, FRACP, in a Guided Poster Tour presentation. The poster will
be available for viewing starting Monday, June 15, at 9:00 a.m.
Pacific Daylight Time.
“Currently, clinicians are able to manage only symptoms in
patients with Parkinson’s disease as there are no disease-modifying
treatments available that can reverse the underlying progressive
degeneration of neurons in the brain,” said Dr. Snow, a neurologist
in the Department of Neurology at Auckland City Hospital and
medical director of Adult Medical Services at the Auckland District
Health Board. “The positive clinical response observed in this
small study of four patients is encouraging and I look forward to
evaluating efficacy in a larger study.”
Ken Taylor, Ph.D., chief executive officer of LCT, said, “NTCELL
is the most advanced and only cell-based therapy currently in a
clinical trial to target regeneration of brain cells in patients
whose symptoms cannot be controlled by current therapies for
Parkinson’s disease. While this regulatory-enabling study is small
in scale, the secondary endpoint efficacy results are sufficiently
encouraging to warrant further studies. We are advancing the
clinical development of NTCELL and will use the results of this
study to design a larger registration-enabling Phase IIb study to
evaluate its potential as a disease-modifying treatment for
patients with Parkinson’s disease.”
LCT plans to initiate the confirmatory Phase IIb study in the
fourth quarter of 2015. The study will be led by Dr. Snow at
Auckland City Hospital. The efficacy and safety endpoints will be
the same as those evaluated in the Phase I/IIa study.
Phase I/IIa study design and results
The open-label Phase I/IIa clinical study evaluated the safety
and clinical effects of implantation of NTCELL, which contains
specialized brain cells that produce cerebrospinal fluid (CSF) and
neuroactive growth factors, into patients who had been diagnosed
with Parkinson’s disease for at least five years and who no longer
responded to current therapy. The study was conducted at Auckland
City Hospital in four patients aged 59 to 68 years at the time of
consent. NTCELL was injected under guidance by neuroimaging into
the affected area of the brain where neural activity was
substantially diminished or degenerated. No immunosuppressive drugs
were used.
The primary endpoint of the study was the safety of NTCELL
implantation, which was assessed by the occurrence of adverse
events and serious adverse events, as well as clinical and
laboratory evidence of porcine endogenous retrovirus (PERV) in
study participants and their partners. The secondary endpoint was
efficacy, which was measured by validated neurological rating
scales and questionnaires, including the Unified Parkinson’s
Disease Rating Scale (UPDRS), the Unified Dyskinesia Rating Scale
(UDysRS) and the Parkinson’s Disease Quality of Life Questionnaire
(PDQ-39) score. These scales assessed improvements in patients’
movement abnormalities, other physical symptoms, well-being and
ability to perform everyday tasks. PET scans were conducted to
measure the effects of NTCELL on dopamine brain metabolism. The
results at week 26 following implantation were compared with those
at baseline.
In the study, NTCELL was well tolerated. There were no adverse
events or serious adverse events related to NTCELL in any of the
four patients. Eight adverse events occurred, all of which were
considered to be related to the implant procedure and none to
NTCELL. There was no clinical or laboratory evidence of PERV
transmission in patients or their partners. MRIs showed no evidence
of inflammation.
All four patients experienced sustained improvement in clinical
features as seen in UPDRS, UDysRs and PDQ-39 scores at week 26
post-implant. The first patient treated continued to show
improvement in neurological scores at 74 weeks
post-implantation.
In this study, PET scan results did not show any consistent
changes in the uptake of fluorodopa and tetrabenazine in the four
patients, suggesting that the mechanism of NTCELL is not likely due
to a direct change in dopaminergic neurons.
Poster presentation details
The abstract is available at www.mdsabstracts.com. The Guided
Poster Tour presentation includes additional data not currently
available in the abstract.
Title: Safety and clinical effects of NTCELL® [immunoprotected
(alginate-encapsulated) porcine choroid plexus cells for
xenotransplantation] in patients with Parkinson's disease (PD): 26
weeks follow-up (abstract #550114, poster #321)
Presenter: Dr. Barry Snow
Session Name: Parkinson’s disease: Clinical trials
Session Date and Time: Wednesday, June 17, 12-1:30 p.m. Pacific
Daylight Time
Session Location: Seaport F, Manchester Grand Hyatt San
Diego
Investor Conference Call
Living Cell Technologies will host a conference call on
Wednesday, June 17, 2015, at 3:30 p.m. Pacific Daylight Time.
Telephone numbers for the conference call are 1-855-293-1544
(U.S.), 1-855-561-6766 (Canada) and +61 2 8038 5221
(international).
About NTCELL
NTCELL, a unique cell therapy, is an alginate coated capsule
containing clusters of neonatal porcine choroid plexus cells that
are sourced from a unique herd of designated pathogen-free pigs
bred from stock originally discovered in the remote sub-Antarctic
Auckland Islands. Choroid plexus cells are naturally occurring
“support” cells for the brain and secrete CSF, which contains a
range of factors that support nerve cell functions and protective
enzymes that are crucial for nerve growth and healthy functioning.
In NTCELL, the porcine choroid plexus cells are coated with LCT’s
propriety technology IMMUPEL™ to protect them from attack by the
immune system. Therefore, no immunosuppressive regimen is required
for treatment.
Following implantation into a damaged site within the brain,
NTCELL functions as a neurochemical factory producing CSF and
secreting multiple nerve growth factors that promote new central
nervous system (CNS) growth and repair disease-induced nerve
degeneration while potentially removing waste products such as
amyloids and proteins.
NTCELL has the potential to treat neurodegenerative diseases
because choroid plexus cells help produce CSF as well as a range of
neurotrophins (nerve growth factors) that have been shown to
protect against neuron (nerve) cell death in animal models of
disease. NTCELL has been shown in preclinical studies to regenerate
damaged tissue and restore function in animal models of Parkinson’s
disease, stroke, Huntington’s disease, hearing loss and other
non-neurological conditions, such as wound healing. In addition to
Parkinson’s disease, NTCELL has the potential to be used in a
number of other CNS indications, including Huntington’s,
Alzheimer’s and motor neuron diseases including amyotrophic lateral
sclerosis (ALS).
About Parkinson’s disease
Parkinson’s disease is a progressive neurological condition
characterized by a loss of brain cells that produce dopamine (a
neurotransmitter that conveys messages between brain cells to
ensure effective movement and planning of movement) and many other
types of neurons. People with Parkinson’s disease experience
reduced and slow movement (hypokinesia and bradykinesia), rigidity
and tremors.
Parkinson’s disease is the second most common neurodegenerative
disorder after Alzheimer’s disease, affecting approximately 4
million people worldwide. The average age of onset is 60 years, and
the incidence increases with age. Men are one and a half times more
likely to have Parkinson's disease than women.
Current treatments for Parkinson’s disease are symptomatic and
do not reverse or slow the degeneration of neurons in the brain.
Most existing pharmaceutical treatment options focus on restoring
the balance of dopamine and other neurotransmitters. The
effectiveness of dopamine replacement therapy declines as the
disease progresses. When dopamine treatments are no longer useful,
some patients are treated with Deep Brain Stimulation (DBS), in
which a medical device is surgically implanted in the brain in
order to send electrical impulses to regions of the brain involved
in the control of movement. While DBS leads to short-term
symptomatic improvement, it does not impact disease progression and
is not curative or neuroprotective.
About Living Cell Technologies
Living Cell Technologies (LCT) is an Australasian biotechnology
company improving the wellbeing of people with serious diseases
worldwide by discovering, developing and commercializing
regenerative treatments that restore function using naturally
occurring cells. LCT’s unique proprietary encapsulation delivery
technology, IMMUPEL™, coats cells with protective capsules that
prevent them from attack by the immune system. This allows cell
therapies to be used without the need for immunosuppressive drugs.
LCT’s lead product, NTCELL®, is being developed for
neurodegenerative diseases. LCT recently completed a Phase I/IIa
clinical trial of NTCELL in New Zealand for the treatment of
Parkinson’s disease and plans to initiate a Phase IIb study in the
fourth quarter of 2015. LCT holds a 50 percent interest in Diatranz
Otsuka Limited, which is developing DIABECELL®, a cell therapy in
late-stage clinical trials for the treatment of type 1 diabetes.
LCT is listed on the Australian (ASX: LCT) and United States
(OTCQX: LVCLY) stock exchanges. The company is incorporated in
Australia, with research and development and operations based in
New Zealand. For more information, visit www.lctglobal.com or
follow @lctglobal on Twitter.
Forward-looking statement
This document contains certain forward-looking statements,
relating to LCT’s business, which can be identified by the use of
forward-looking terminology such as “promising,” “plans,”
“anticipated,” “will,” “project,” “believe,” “forecast,”
“expected,” “estimated,” “targeting,” “aiming,” “set to,”
“potential,” “seeking to,” “goal,” “could provide,” “intends,” “is
being developed,” “could be,” “on track,” or similar expressions,
or by express or implied discussions regarding potential filings or
marketing approvals, or potential future sales of product
candidates. Such forward-looking statements involve known and
unknown risks, uncertainties and other factors that may cause
actual results to be materially different from any future results,
performance or achievements expressed or implied by such
statements. There can be no assurance that any existing or future
regulatory filings will satisfy the FDA’s and other health
authorities’ requirements regarding any one or more product
candidates nor can there be any assurance that such product
candidates will be approved by any health authorities for sale in
any market or that they will reach any particular level of sales.
In particular, management’s expectations regarding the approval and
commercialization of the product candidates could be affected by,
among other things, unexpected clinical trial results, including
additional analysis of existing clinical data, and new clinical
data; unexpected regulatory actions or delays, or government
regulation generally; our ability to obtain or maintain patent or
other proprietary intellectual property protection; competition in
general; government, industry, and general public pricing
pressures; and additional factors that involve significant risks
and uncertainties about our products, product candidates, financial
results and business prospects. Should one or more of these risks
or uncertainties materialize, or should underlying assumptions
prove incorrect, actual results may vary materially from those
described herein as anticipated, believed, estimated or expected.
LCT is providing this information and does not assume any
obligation to update any forward-looking statements contained in
this document as a result of new information, future events or
developments or otherwise.
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