Press Releases
May 1, 2005
Positive Phase I Data for Neurotech’s NT- 501 Encapsulated
Cell Delivery of Ciliary Neurotrophic Factor to Eyes of Patients
with Retinitis Pigmentosa
Data Presented at the 77th Annual Meeting of the Association for
Research in Vision and Ophthalmology (ARVO) Conference in Fort Lauderdale,
Florida
Fort Lauderdale, Florida, May 1, 2005 ….
Neurotech SA, a specialist in the development of novel therapies
for retinal diseases, today announced positive results from an open-label
Phase I clinical trial (03-EI-0234) of its lead product, NT-501.
NT-501 uses Neurotech’s patented Encapsulated Cell Technology
(ECT) as a device to deliver ciliary neurotrophic factor (CNTF)
to eyes of visually impaired patients with retinitis pigmentosa
(RP). Results confirm that CNTF can be safely delivered into the
vitreous of patients with RP and that the ECT device was well tolerated
by all patients. Futhermore, some patients experienced more than
one-line of improvement in their visual acuity score. These Phase
I results were presented at the ARVO annual meeting and the trial
was conducted at the National Eye Institute (NEI), Bethesda, USA.
Neurotech has confirmed that it will now progress to a multi-center
Phase II trial.
ECT, a technique developed and patented by Neurotech, allows for
genetically-engineered specific protein delivery without manipulating
the patient’s genetic information or transferring new genetic
information into the target tissue. The Phase I study of NT-501
involved 10 patients with late-stage RP. The study was designed
as an open-label safety and tolerability evaluation. Two doses of
CNTF (5-fold difference in dose) were evaluated. Phase Ia treated
5 patients with a lower dose; Phase Ib treated 5 patients with a
higher dose. The ECT device was implanted in one eye per patient
and removed after six months. All explanted devices contained viable
cells that continued to produce CNTF.
Commenting on the positive results, Weng Tao, MD, PhD, CSO and
VP of R&D with Neurotech said:
“These safety and tolerability results are extremely encouraging
and strengthen our confidence in pursuing NT-501 for treating RP
and other retinal degenerative diseases. I would like to thank the
National Eye Institute for conducting this milestone study and for
its continued involvement with this technology.”
Al Reaves, PhD, VP of Clinical Development with Neurotech confirmed:
“This study represents the first use of ECT in human eyes
and it is reassuring that the devices were safe and well-tolerated.
We are planning Phase II development with well-designed and controlled
multi-center clinical studies to help understand the role that NT-501
will play in treating patients with retinitis pigmentosa and other
retinal degenerative conditions.”
In this trial, the small ECT device was surgically-implanted into
the vitreous cavity through the pars plana in one eye per patient.
The primary inclusion criteria for the study eye included visual
acuity of 20/100 or worse, central visual field diameter of 40 degrees
or less, and flicker ERG amplitude of 2 µV or less. The first
2 patients were also required to have visual acuity of 20/400 or
worse. After surgical implantation of the device, each patient was
followed for six months after which the device was explanted. Safety
and tolerability was monitored by an independent Data and Safety
Monitoring Committee. All 10 patients completed the study as planned
and the devices have been explanted. The ECT devices were well tolerated
during the 6 months of implantation and the surgical procedure resulted
in minimal or no observed inflammatory reaction. No serious adverse
events were reported and in the untreated fellow-eye, there was
little change from baseline in the visual acuity score during follow-up.
In the treated study-eye, however, the visual acuity score was more
variable during follow-up: while some treated eyes showed little
change from baseline, some patients experienced more than one-line
of improvement in their visual acuity score.
Bernard Davitian, President of Neurotech, said: “We are extremely
pleased that the Phase I trial has validated the safe use of the
Encapsulated Cell Technology to deliver CNTF to the vitreous. In
addition, the Phase I trial has validated ECT as a delivery platform
for the back of the eye and the visual acuity outcomes observed
in some patients are encouraging enough to pursue the clinical evaluation
of NT-501 in Phase II trials. In addition to further studies with
NT-501, we are evaluating other neurotrophic factors and agents
that can be used with ECT for treating other retinal diseases.”
In addition to NT-501 for the treatment of Retinitis Pigmentosa,
Neurotech is applying ECT technology to deliver other protein factors
for the treatment of other ophthalmic diseases, including anti-angiogenic
factors for the treatment of the wet form of age-related macular
degeneration (AMD) and diabetic macular edema (DME), and anti-inflammatory
factors for the treatment of posterior uveitis.
Notes to Editors
1. About Retinitis Pigmentosa (RP)
RP is a group of inherited retinal diseases that affects about 100,000
Americans and 1.5 million people worldwide. It causes the progressive
deterioration of specialized, light-absorbing cells in the retina,
the paper-thin tissue that lines the back of the eye like film in
a camera. As these cells slowly degenerate, people with RP develop
night blindness and a gradual loss of peripheral vision. By about
age 40, most have tunnel vision, although many may retain good central
vision. Between the ages of 50 and 80, however, they typically lose
their remaining sight. The extent of vision loss in people of the
same age with RP may be different.
2. About Encapsulated Cell Technology (ECT)
ECT is a unique technology to overcome drug delivery problems into
the back of the eye. ECT enables the controlled, continuous, long-term
delivery of therapeutic proteins directly to the retina. In addition,
the implants can be retrieved, providing an added level of safety
as well as the ability to reverse or adjust therapy, if needed.
ECT relies on the use of an immunoisolatory hollow fiber membrane
technology to allow the implantation of genetically engineered cells
that continuously produce the therapeutic protein at the site of
implantation. The cells are loaded into the interior volume of the
hollow fiber membrane and attach to a proprietary supportive matrix
within this space. The genetically modified cells remain captive
within the ECT device thus avoiding the risks associated with traditional
gene therapy. Long term protein delivery (18 months) in the vitreous
cavity of the eye has consistently been achieved when ECT devices
containing human retinal pigmented epithelial (RPE) cells genetically
engineered to secrete CNTF have been implanted in a highly disparate
mammalian species (rabbits).
3. About Neurotech
Neurotech is a biotechnology company specializing in the development
of novel therapeutics to treat diseases of the eye. The company’s
initial focus is on chronic diseases affecting the back of the eye,
especially the retina, because retinal diseases represent the greatest
unmet medical need and therefore offer the largest market opportunities
in ophthalmology. The Company has one product (NT-501) in development
for the treatment of retinitis pigmentosa and other degenerative
diseases of the retina, and is evaluating other neurotrophic factors
and agents that can be used with ECT for treating other retinal
diseases. The company is headquartered in Paris with an American
subsidiary, Neurotech USA, Inc., located in Lincoln, RI, south of
Boston. Neurotech is supported in its scientific and business strategies
by world experts in ophthalmology and by a group of international
investors led by Apax Partners and Merlin Biosciences.
To learn more about Neurotech, please visit our web site at www.neurotechusa.com
For further information please contact :
At Neurotech: +1 401 333-3880 x3116
Ted Danse, CEO
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