Genzyme Corporation (NASDAQ: GENZ) today announced three-year
follow-up data from patients enrolled in the phase 2 clinical trial
for its investigational oral therapy for Gaucher disease type 1
known as eliglustat tartrate. Sustained or further improvements
were observed across all endpoints, including bone disease, at the
three-year timepoint. The results were presented for the first time
this week at the Lysosomal Disease Network WORLD Symposium in Las
Vegas, Nevada.
Genzyme previously reported that the eliglustat tartrate phase 2
trial had met its primary endpoint at one year, and that data
demonstrated continued improvement through two years. The primary
composite endpoint was a clinically meaningful response in at least
two of three endpoints: improvements in spleen size, hemoglobin and
platelet levels. The study has continued with 19 patients through
three years. The extension phase of this trial is still
ongoing.
Eliglustat tartrate continued to show robust clinical response
through three years:
- Spleen volume decreased from baseline
by a mean of 61 percent and liver volume decreased from baseline by
29 percent.
- Hemoglobin level increased from
baseline by a mean of 2.6 grams per deciliter.
- Platelet count increased from baseline
by a mean of 91 percent.
The study also analyzed the clinical response of patients in the
phase 2 trial with respect to achieving therapeutic goals. Due to
the heterogeneity of Gaucher disease, therapeutic goals were
previously developed by experts involved in the treatment of
Gaucher patients to assess their response to enzyme replacement
therapy (ERT). Most patients dosed with eliglustat tartrate met
established therapeutic goals for hemoglobin, platelets, spleen
volume and liver volume, demonstrating progressive and clinically
meaningful responses in multiple organ systems. At three years, 100
percent of patients met at least 3 of the 4 therapeutic goals
developed for hematologic and organ volume parameters.
The three-year data also included analyses that suggest
eliglustat tartrate positively impacts indicators of bone disease
through three years of follow up. These indicators include bone
mineral density in the lumbar spine, as measured by dual energy
x-ray absorptiometry (DXA), and dark marrow signal in the femur, as
visualized by magnetic resonance imaging (MRI). Dark marrow
reflects the infiltration of lipid-laden Gaucher cells into bone
marrow. Specifically:
- In the 18 patients at baseline with
dark marrow in the femur visible by MRI, five improved by one year,
seven by two years and 10 by three years, with the other eight
patients remaining stable.
- In the 15 patients with results
available at all time points, bone mineral density in the lumbar
spine showed clinically and statistically significant improvements
after one year of treatment (T score = +0.4) which further improved
after 2 years (T score = +0.6) and were sustained after three years
of treatment.
Ravi S. Kamath, M.D., Ph.D., Staff Radiologist at Massachusetts
General Hospital and Instructor in Radiology at Harvard Medical
School, who is the central radiology reviewer for the phase 2
study, said, "These data suggest that eliglustat tartrate may have
a meaningful clinical impact on bone disease in Gaucher disease
type 1 patients."
The most common adverse events (AEs) reported in greater than 2
patients through three years included viral infections (six
patients), urinary tract infections and upper respiratory
infections (4 patients each), headache, increased blood pressure,
diarrhea and abdominal pain (three patients each). Eight
drug-related AEs, including one serious event, were reported in six
patients. Most AEs overall and all drug-related AEs were considered
mild. The largest number of AEs was reported during the first 3
months of treatment.
“For thirty years, Genzyme has pioneered treatments for patients
with lysosomal storage disorders, including the very first enzyme
replacement therapy for Gaucher disease,” said Genzyme’s President,
Personalized Genetic Health, John P. Butler. “Our momentum
continues through the phase 3 trials – the largest ever conducted
for Gaucher - as we build upon our foundation and commitment to
Gaucher and look to extend the therapeutic options available to
patients and physicians.”
Eliglustat tartrate, a capsule taken orally, is being developed
to provide a convenient treatment alternative for adult patients
with Gaucher disease type 1, and to offer a broader range of
treatment options for patients and physicians to achieve individual
therapeutic goals. Genzyme is currently enrolling patients in three
global, multi-center, phase 3 trials of eliglustat tartrate. This
is the largest clinical program ever focused on Gaucher disease,
with over 50 sites in more than 25 countries currently
participating. Genzyme’s Gaucher disease portfolio also offers
Cerezyme® (imiglucerase for injection), the standard of care for
patients with Gaucher disease type 1, which is administered through
intravenous infusions.
To learn more about the phase 3 trials of eliglustat tartrate,
contact Genzyme Medical Information at medinfo@genzyme.com or
1-800-745-4447. More information can also be found at
www.clinicaltrials.gov or www.explorerstudies.com.
About Gaucher disease
Gaucher disease is an inherited condition affecting fewer than
10,000 people worldwide. People with Gaucher disease do not have
enough of an enzyme, acid β-glucosidase (glucocerebrosidase) that
breaks down a certain type of fat molecule. As a result, lipid
engorged cells (called Gaucher cells) amass in different parts of
the body, primarily the spleen, liver and bone marrow. Accumulation
of Gaucher cells may cause spleen and liver enlargement, anemia,
excessive bleeding and bruising, bone disease and a number of other
signs and symptoms. The most common form of Gaucher disease, type
1, does not typically affect the nervous system and brain.
About eliglustat tartrate
Eliglustat tartrate, a novel glucosylceramide analog given
orally, is designed to partially inhibit the enzyme
glucosylceramide synthase, which results in reduced production of
glucosylceramide. Glucosylceramide is the substance that builds up
in the cells and tissues of people with Gaucher disease. In
preclinical studies, the molecule, developed with James A. Shayman,
M.D. from the University of Michigan, has shown high potency and
specificity. Based on its mechanism of action, which is independent
of genotype, eliglustat tartrate may be a potential therapy for
patients with Gaucher disease type 1. Initiation of the phase 2 and
3 studies of eliglustat tartrate in Gaucher disease followed
completion of an extensive pre-clinical research effort and a phase
1 program. Over 300 subjects have now been treated in nine separate
studies.
The data from the phase 2 trials with eliglustat tartrate were
previously published in the journal Blood and the results can be
found at the below references:
- Phase 2 data at the 1 year time point:
Lukina et al. Blood, Aug 2010; Vol. 116: 893 - 899
- Phase 2 data at the 2 year time point:
Lukina et al. Blood, Nov 2010; Vol 116: 4095 - 4098
Cerezyme important safety information
Approximately 15 percent of patients have developed IgG
antibodies, and these patients have a higher risk of
hypersensitivity reaction. Therefore periodic monitoring is
suggested; caution should be exercised in patients with antibodies
or prior symptoms of hypersensitivity. Symptoms suggestive of
hypersensitivity occurred in 6.6 percent of patients, and include
anaphylactoid reaction, pruritus, flushing,
urticaria, angioedema, chest discomfort, dyspnea, coughing,
cyanosis and hypotension. Reactions related to Cerezyme
administration have been reported in less than 15 percent of
patients. Each of the following events occurred in less than 2
percent of the total patient population. Reported adverse events
include nausea, vomiting, abdominal pain, diarrhea, rash, fatigue,
headache, fever, dizziness, chills, backache, and tachycardia.
Adverse events associated with the route of administration include
discomfort, pruritus, burning, swelling or sterile abscess at the
site of venipuncture. For full prescribing information, please
visit www.genzyme.com.
About Genzyme
One of the world's leading biotechnology companies, Genzyme is
dedicated to making a major positive impact on the lives of people
with serious diseases. Since 1981, the company has grown from a
small start-up to a diversified enterprise with approximately
10,000 employees in locations spanning the globe.
With many established products and services helping patients in
100 countries, Genzyme is a leader in the effort to develop and
apply the most advanced technologies in the life sciences. The
company's products and services are focused on rare inherited
disorders, kidney disease, orthopaedics, cancer, transplant, and
immune disease. Genzyme's commitment to innovation continues today
with a substantial development program focused on these fields, as
well as cardiovascular disease, neurodegenerative diseases, and
other areas of unmet medical need. Genzyme’s press releases and
other company information are available at www.genzyme.com and by
calling Genzyme’s investor information line at 1-800-905-4369
within the United States or 1-678-999-4572 outside the United
States.
Important Information
Genzyme has filed with the Securities and Exchange Commission a
Solicitation/Recommendation Statement on Schedule 14D-9 relating to
the tender offer by Sanofi-Aventis. Genzyme shareholders are
advised to read the company's Solicitation/Recommendation Statement
on Schedule 14D-9 because it contains important information.
Shareholders may obtain a free copy of the
Solicitation/Recommendation Statement on Schedule 14D-9, as well as
any other documents filed by Genzyme in connection with the tender
offer, free of charge at the SEC's website at http://www.sec.gov.
In addition, investors can obtain free copies of these documents
from Genzyme by directing a request to Genzyme at 500 Kendall
Street, Cambridge, MA 02142, Attention: Shareholder Relations
Department, or by calling 617-252-7500 and asking for the
Shareholder Relations Department.
Genzyme® and Cerezyme® are registered trademarks of Genzyme
Corporation. All rights reserved.
Genzyme (NASDAQ:GENZ)
Historical Stock Chart
Von Nov 2024 bis Dez 2024
Genzyme (NASDAQ:GENZ)
Historical Stock Chart
Von Dez 2023 bis Dez 2024
