Genzyme
Corporation (NASDAQ: GENZ) announced today two-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 (formerly Genz-112638). Continued improvements were
observed across all endpoints, including bone disease, at the two-year
timepoint, compared with baseline. The two-year results were presented
for the first time today at the Lysosomal Disease Network WORLD
Symposium in Miami, Fla.
“We have set a high threshold for success for eliglustat
tartrate, and are very encouraged by the results we continue to observe.”
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’s Cerezyme® (imiglucerase for injection), the
standard of care for patients with Gaucher disease type 1, is
administered through intravenous infusions.
Genzyme reported last year that the phase 2 trial had met its primary
composite endpoint: a clinically meaningful response in at least two of
three endpoints (improvements in spleen size, hemoglobin and platelet
levels) in individual patients after the 52-week study period.
Twenty-two of 26 study participants completed at least one year of
treatment, and 20 patients completed two years of treatment. The study
is continuing with 19 patients in their third year. Medical centers in
North America, South America, Europe and the Middle East participated in
this study.
Continued improvement was observed through two years for patients who
received eliglustat tartrate:
-
Spleen volumes decreased from baseline by a mean of 52 percent and
liver volumes decreased from baseline by 24 percent.
-
Hemoglobin levels increased from baseline by a mean of 2.1 grams per
deciliter.
-
Platelet counts increased from baseline by a mean of 81 percent.
-
Chitotriosidase levels decreased from baseline by a median of 63
percent (only 18 month data are available to date), among the 17
patients with chitotriosidase. Chitotriosidase is commonly monitored
by physicians as a biomarker of Gaucher disease burden and response to
treatment.
“We remain committed to building our Gaucher health portfolio by
developing clinically meaningful solutions to support individualized
care for this community,” said Genzyme Senior Vice President Geoff
McDonough, MD. “We have set a high threshold for success for eliglustat
tartrate, and are very encouraged by the results we continue to observe.”
According to the ICGG Gaucher Registry, an international multi-center
program sponsored by Genzyme that tracks the routine clinical outcomes
for patients with Gaucher disease, irrespective of treatment status,
over 80 percent of Gaucher patients have radiologic evidence of bone
disease. The data presented today also included pre-planned analyses
that suggest eliglustat tartrate may positively impact some indicators
of bone disease through two years of follow up. These indicators include
bone mineral density, as measured by dual energy x-ray absorptiometry
(DXA), and the proportion of dark marrow on magnetic resonance imaging
(MRI). Dark marrow reflects the infiltration of diseased cells into the
bone marrow. Specifically: