Genentech, Inc. (NYSE: DNA) announced today the publication of data from
the two randomized, controlled pivotal Phase III clinical trials of
LUCENTIS(TM) (ranibizumab injection) in the New England Journal of
Medicine. The published findings include two-year efficacy and safety data
from the MARINA trial and one-year efficacy and safety data from the
ongoing ANCHOR trial. Based on these studies, LUCENTIS was granted U.S.
Food and Drug Administration (FDA) approval on June 30, 2006 for the
treatment of patients with the neovascular (wet) form of age-related
macular degeneration (AMD), a leading cause of blindness in people over 55.
The MARINA and ANCHOR clinical trials met the primary efficacy endpoint
of maintaining vision (defined as a loss of less than 15 letters in visual
acuity) at one year in patients with wet AMD. In these studies, nearly all
patients (approximately 95 percent) treated with LUCENTIS maintained or
improved vision at one year compared with 62 percent of patients in the
MARINA control group and 64 percent of patients in the ANCHOR control
group. Importantly, up to 40 percent of patients experienced an improvement
in vision of three lines (15 letters) or more on the study eye chart
compared with 5 percent and 6 percent of patients in the MARINA and ANCHOR
control groups, respectively. The improvement in visual acuity endpoints
among patients treated with LUCENTIS in the MARINA study was maintained at
year two, while patients in the control group continued to decline.
“The results of these Lucentis studies have changed the way we approach
the treatment of wet AMD by demonstrating, for the first time, improvements
in vision in more than one-third of patients treated,” said David Brown,
M.D., lead author for the ANCHOR study and retina specialist at
Vitreoretinal Consultants, The Methodist Hospital in Houston, Texas.
“What makes this publication particularly significant is that the
visual acuity benefits after one year of treatment in the MARINA study were
maintained through two years and associated with anatomic improvements
consistent with the changes in visual acuity observed,” said Philip J.
Rosenfeld, M.D., Ph.D., professor of ophthalmology, Bascom Palmer Eye
Institute in Miami and lead author for the MARINA study. “In this study,
Lucentis had a favorable safety profile and did not appear to put patients
at an additional risk for systemic adverse events and the ocular adverse
event rates were similar to what we would expect among people in this age
group who receive an injection in the eye.”
Patients treated with LUCENTIS in these studies, on average,
experienced an improvement over the control groups of three lines or more
on the study eye chart. In both studies, up to 40 percent of patients
treated with LUCENTIS achieved vision of 20/40 or better at one year
compared with 11 percent of patients in the MARINA control group and 3
percent of patients in the ANCHOR control group. At two years, 42 percent
of patients treated with LUCENTIS in the MARINA study achieved vision of
20/40 or better compared with 6 percent of patients in the control group.
Visual acuity was measured using the Early Treatment Diabetic Retinopathy
Study (ETDRS) chart, the standard method of quantifying visual acuity.
“The publication of these two studies represents nearly a decade of
rigorous clinical study of Lucentis that has led to a new era in the
treatment of wet AMD,” said Hal Barron, M.D., Genentech senior vice
president, Development and chief medical officer. “We have already begun to
expand on the findings of these studies to better understand optimal
treatment regimens for patients with wet AMD, as well as explore the
potential for Lucentis to meet other unmet medical needs in ophthalmology.”
LUCENTIS was specifically developed for intraocular use in the eye to
treat the underlying cause of wet AMD by targeting the molecular pathway
that controls the formation of new blood vessels. LUCENTIS is designed to
bind and inhibit VEGF-A, a protein that is believed to play a critical role
in angiogenesis (the formation of new blood vessels).
LUCENTIS 0.5 mg is recommended for intravitreal injection once a month.
If monthly injections are not feasible, treatments can be reduced to one
injection every three months after the first four monthly injections.
Compared to continued monthly dosing, dosing every three months will lead
to an approximate five-letter (one-line) loss of visual acuity benefit, on
average, over the following nine months. Patients should be evaluated
About the Pivotal Studies
MARINA (Minimally classic/occult trial of the Anti-VEGF antibody
Ranibizumab In the treatment of Neovascular AMD) was a Phase III
randomized, multi-center, double-masked, sham-controlled study of 716
patients in the United States with minimally classic or occult wet AMD who
were randomized 1:1:1 to receive intravitreal LUCENTIS injections (0.3 mg
or 0.5 mg) or a control regimen once a month for two years. The control
regimen consisted of a sham injection, meaning the treating physician
prepares and anesthetizes the patient’s eye but does not perform an
ANCHOR (ANti-VEGF Antibody for the Treatment of Predominantly Classic
CHORoidal Neovascularization in AMD) is a Phase III randomized,
multi-center, double-masked, active-treatment controlled study comparing
two different doses of LUCENTIS to verteporfin (Visudyne(R)) photodynamic
therapy (PDT) in 423 patients with predominantly classic wet AMD in the
United States, Europe and Australia. Patients were randomized 1:1:1 to
receive intravitreal LUCENTIS injections (0.3 mg or 0.5 mg) once a month or
PDT every three months for two years.
LUCENTIS Safety Profile
In clinical trials, the most common adverse reactions among patients
treated with LUCENTIS (reported in at least 6 percent more patients than in
the control groups in at least one study) included conjunctival hemorrhage,
eye pain, vitreous floaters, increased intraocular pressure and intraocular
inflammation. Although there was a low rate (less than 4 percent) of
arterial thromboembolic events (ATEs) observed in the LUCENTIS clinical
trials that was not statistically different between the LUCENTIS and
control groups, there is a theoretical risk of ATEs following intravitreal
use of inhibitors of VEGF. Serious adverse events related to the injection
procedure occurred in less than 0.1 percent of intravitreal injections,
including endophthalmitis, retinal detachments and traumatic cataracts.
Other serious ocular adverse events observed among LUCENTIS-treated
patients (that occurred in less than 2 percent of patients) included
intraocular inflammation and increased intraocular pressure. LUCENTIS is
contraindicated in patients with hypersensitivity and ocular or periocular
LUCENTIS(TM) (ranibizumab injection) (0.5 mg) is approved for the
treatment of patients with neovascular (wet) AMD. LUCENTIS is a recombinant
humanized IgG1 kappa isotype therapeutic antibody fragment developed for
LUCENTIS binds to and inhibits the biologic activity of human vascular
endothelial growth factor A (VEGF-A), a protein that is believed to play a
critical role in angiogenesis (the formation of new blood vessels). VEGF-A
has been shown to lead to wet AMD disease progression and central vision
loss. LUCENTIS was developed by Genentech and the Novartis Ophthalmics
Business Unit for diseases or disorders of the eye. Genentech retains
commercial rights in the United States and Novartis has exclusive
commercial rights for the rest of the world. For LUCENTIS prescribing
information, please call 1-866-LUCENTIS or visit lucentis.
AMD is a major cause of painless central vision loss and is a leading
cause of blindness in people over 55. The National Eye Institute estimates
that there are 1.7 million people with the advanced form of AMD in the
United States alone and that this prevalence will grow to 2.95 million by
2020. AMD occurs in two forms: dry and wet.
The dry form is associated with atrophic cell death of the central
retina or macula, which is required for fine vision used for activities
such as reading, driving or recognizing faces. The wet form is caused by
growth of abnormal blood vessels, also known as choroidal
neovascularization (CNV) or ocular angiogenesis, under the macula. These
vessels leak fluid and blood and cause scar tissue that destroys the
central retina. This process results in a deterioration of sight over a
period of months to years.
Genentech is a leader in research and product development in the area
of angiogenesis, the process by which new blood vessels are formed. In
1989, Napoleone Ferrara, M.D., and a team of scientists at Genentech
conducted seminal work in the field, which resulted in the identification
and cloning of a gene termed Vascular Endothelial Growth Factor (VEGF), now
known as VEGF-A. The VEGF-A protein is believed to play a critical role in
angiogenesis and serves as one of the key contributors to physiological or
pathological conditions that can stimulate the formation of new blood
vessels. The process of angiogenesis is normally regulated throughout
development and adult life, and the uncontrolled growth of new blood
vessels is an important contributor to a number of pathologic conditions,
including wet AMD.
Genentech’s Commitment to Patient Access
Genentech is committed to assisting eligible patients in accessing our
therapies for approved indications, regardless of their ability to pay.
Although Genentech’s products are covered by most government and private
insurance, Genentech established the Genentech(R) Access to Care Foundation
(GATCF) in 1990 for its marketed products, and donates free product to
eligible uninsured patients in the United States, except for Pulmozyme(R)
(dornase alfa, recombinant), which is covered by the Genentech Endowment
for Cystic Fibrosis. In 2005 alone, GATCF supported over 18,000 patients by
providing approximately $200 million of free product. Genentech recently
donated more than $25 million to several independent public charities that
provide financial assistance to eligible patients who cannot access needed
medical treatment due to co-pay costs. To learn more about these
independent, public charities and potential financial assistance options,
patients can speak with an Alternative Funding Specialist from Genentech’s
Single Point of Contact (SPOC) group by calling 866-724-9394 or visiting
Founded 30 years ago, Genentech is a leading biotechnology company that
discovers, develops, manufactures and commercializes biotherapeutics for
significant unmet medical needs. A considerable number of the currently
approved biotechnology products originated from or are based on Genentech
science. Genentech manufactures and commercializes multiple biotechnology
products and licenses several additional products to other companies. The
company has headquarters in South San Francisco, California and is listed
on the New York Stock Exchange under the symbol DNA. For additional
information about the company, please visit gene.
This press release contains a forward-looking statement regarding the
potential for Lucentis to meet other unmet medical needs in ophthalmology.
Such statement is a prediction and involves risks and uncertainties such
that the actual result may differ materially. Among other things, Lucentis’
potential could be affected by unexpected safety, efficacy or manufacturing
issues, need for additional clinical studies, discussions with the FDA or
FDA actions, failure to receive or maintain FDA approval, competition,
reimbursement or coverage, pricing, the ability to supply product, product
withdrawal, new product approvals and launches, intellectual property or
contract rights, and achieving sales revenue consistent with internal
forecasts. Please also refer to Genentech’s periodic reports filed with the
Securities and Exchange Commission. Genentech disclaims, and does not
undertake, any obligation to update or revise the forward-looking statement
in this press release.
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