Positive Results from Neurotech's NT-501 Phase 2 Dry AMD
(Geographic Atrophy) Study Demonstrate Proof of Concept
-- Validate Company's ECT Technology and Support Initiation of Pivotal
Lincoln, RI (March 26, 2009) –
Neurotech Pharmaceuticals, Inc., today announced that the Company's
lead product candidate, NT-501, substantially slowed the loss of vision in
a Phase 2 clinical trial in subjects with dry age-related macular
degeneration (AMD) involving geographic atrophy (GA). GA is a
condition that destroys sharp central vision, often resulting in serious
vision loss to one or both eyes. There are currently no approved
treatments for dry AMD.
In the study, the high dose of NT-501 stabilized best corrected visual
acuity (BCVA) at 12-months, with 96.3% (p=0.078) of treated-patients
losing fewer than three lines of vision, or 15 letters, versus 75% of the
patients in the sham-treatment group.
NT-501 is an intraocular implant that consists of human cells that have
been genetically modified to secrete ciliary neurotrophic factor (CNTF).
CNTF is delivered directly to the back of the eye in a controlled,
continuous basis by means of the Company's proprietary Encapsulated
Cell Technology (ECT) platform, thereby bypassing the blood-retinal
barrier and overcoming a major obstacle in the treatment of retinal
The Phase 2 study is a multi-centered, randomized, double-masked,
sham-controlled study of 51 subjects with GA. Patients received either a
high or low dose NT-501 implant or a sham treatment in one eye only
and were assessed for changes in BCVA. BCVA was measured by an
Electronic Visual Acuity Tester (EVA) using the Early Treatment Diabetic
Retinopathy Study (ETDRS) protocol. Patients were also evaluated for
an increase in BCVA. However, no increase was observed, likely due to
existing photoreceptor damage. There were no NT-501 associated
serious adverse events reported and both NT-501 and the surgical
procedure were well-tolerated.
"The favorable functional visual acuity results for patients at this
advanced stage of dry AMD are particularly promising due to the
significant prevalence of the condition, its serious impact on quality of life
and the current unmet medical need for effective therapy," stated Dr.
George A. Williams, a study investigator and Professor and Chair of the
Department of Ophthalmology at William Beaumont Hospital and the
Oakland University William Beaumont School of Medicine in Royal Oak,
The strong trend in visual acuity stabilization at 12 months was preceded
by a dose-dependent, statistically significant (p<0.001 and p=0.013 for
high and low dose, respectively) increase in retinal thickness as
measured by optical coherence tomography (OCT) that was observed as
early as 4 months post-implantation.
The observed structural change is consistent with preclinical studies of
NT-501 in which CNTF was shown to increase the thickness of the retina
and the outer nuclear layer of photoreceptors responsible for vision. This
increase in retinal thickness may be responsible for photoreceptor
rescue and protection as observed in numerous animal models of retinal
"Based on the increase in retinal thickness observed in this study it
appears that CNTF may be exhibiting a biological effect on retinal
photoreceptors as has been observed previously in animal studies," said
Dr. Paul Sieving, Director of the National Eye Institute and Principal
Investigator of Neurotech's Phase 1 study of NT-501 in retinitis
"We believe the anatomical changes observed in patients treated with
NT-501 have led to the emergence of a clinically meaningful visual acuity
benefit for patients with geographic atrophy," commented Ted Danse,
President and Chief Executive Officer of Neurotech. "NT-501 may
provide a much needed treatment option for these patients and we
intend to discuss these data and a pivotal trial design with the FDA."
"We are very pleased that the outcome of this trial has shown such
promise for patients with dry AMD involving geographic atrophy and are
proud of our long-term support for this unique, breakthrough technology,"
stated Stephen Rose, PhD, Chief Research Officer, Foundation Fighting
Five devices from this trial have been explanted 12 months following
implantation and all have been found to have uniformly healthy, viable
cells that continue to produce therapeutic levels of CNTF. This is
consistent with data from multiple trials of NT-501 in which, to date, 23
devices have been explanted between 12 and 18 months following
implantation and all devices have contained healthy, viable CNTF-
"We also believe the positive results of this study and long-term cell
viability validate our ECT platform and support a breakthrough
opportunity to advance long-term, well-tolerated treatments for patients
facing chronic sight-stealing retinal diseases. As such, we are developing
our second product utilizing the ECT platform to address a well-validated
target, anti-VEGF therapy for wet AMD, that has the potential to provide
a one-time administration for a 12 to 18 month period versus the current
wet AMD treatment regimen that requires monthly injections with routine
patient monitoring," concluded Danse.
Data from the Phase 2 dry AMD/GA trial will be presented at the Retinal
Physician Symposium on March 27, 2009 in the Bahamas, at IBC's 5th
International Ocular Angiogenesis & Retinal Degeneration conference on
March 31, 2009 in Las Vegas, at the Advanced Vitreoretinal Techniques
and Technologies meeting on April 24, 2009 in Las Vegas and at the
Association for Research in Vision and Ophthalmology (ARVO) meeting
on May 6, 2009 in Ft. Lauderdale.
About Dry AMD/Geographic Atrophy
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Neurotech's lead product, NT-501, consists of encapsulated human cells
genetically modified to secrete ciliary neurotrophic factor (CNTF). CNTF
is a growth factor capable of rescuing dying photoreceptors and
protecting them from degeneration. NT-501 is designed to continually
deliver a therapeutic dose of CNTF into the back of the eye.
About Encapsulated Cell Technology
Neurotech's core technology platform is Encapsulated Cell Technology
(ECT), a unique technology that allows for the long-term, sustained
delivery of therapeutic factors to the back of the eye. ECT implants
consist of cells that have been genetically modified to produce a specific
therapeutic protein and are encapsulated in a semi-permeable hollow
fiber membrane. The diffusive characteristics of the hollow fiber
membrane are designed to promote long-term cell survival by allowing
the influx of oxygen and nutrients while simultaneously preventing direct
contact of the encapsulated cells with the cellular and molecular
elements of the immune system. The cells continuously produce the
therapeutic protein which diffuses out of the implant at the target site.
ECT thereby enables the controlled, continuous delivery of therapeutic
factors directly to the retina, bypassing the blood-retina barrier.
About Neurotech Pharmaceuticals, Inc.
Neurotech is developing sight-saving therapeutics for the treatment of
chronic retinal diseases. The Company's lead product candidate, NT-
501, is currently in late-stage clinical development for advanced dry age-
related macular degeneration (dry AMD) and retinitis pigmentosa (RP).
The Company's portfolio of product candidates also includes treatments
for wet AMD. All of Neurotech's development programs are based on the
Company's proprietary Encapsulated Cell Technology (ECT). ECT
uniquely enables the controlled, continuous delivery of biologics directly
to the back of the eye, thereby overcoming a major obstacle in the
treatment of retinal disease. To learn more, please visit our web site at