Drug Development Highlights in 2014 and early 2015
•
We and Genzyme reported data from a Phase 3 long-term extension study in patients treated with
KYNAMRO (mipomersen sodium) injection. In the study, a retrospective analysis of 104 patients with
familial hypercholesterolemia treated with KYNAMRO for a mean of one or two years had a significant
reduction in major adverse cardiovascular events, or MACE, compared to two years prior to therapy.
•
At the European Society of Cardiology Congress, our KYNAMRO advisor, John Kastelein, M.D.,
Ph.D., presented an analysis of patients treated with KYNAMRO for one year. These patients
experienced a reduction inMACE of 4.85/1000 compared to 25.72/1000 months (in the two years
prior to KYNAMRO treatment).
•
At theAmerican Heart AssociationMeeting, a KYNAMRO principal investigator, P. Bart Duell,
M.D., presented an analysis of patients treated with KYNAMRO for two years. These patients
experienced a seven-fold reduction inMACE of 3.6/1000 compared to 25.72/1000 months (in the
two years prior to KYNAMRO treatment).
•
We reported Phase 2 data on ISIS-APOCIII
Rx
in patients with high to extremely high triglyceride levels, in
patients with type 2 diabetes and high triglycerides and as a single agent as well as in combination with
fibrates.
•
At theAmerican College of Cardiology meeting, we presented Phase 2 data on ISIS-APOCIII
Rx
in
combination with fibrates in patients with high triglycerides. In this study, patients achieved
statistically significant reductions in triglycerides, apoC-III protein and statistically significant
increases in HDL-cholesterol, on top of improvements achieved with each patient’s existing
therapeutic regimen of triglyceride-lowering drugs.
•
At theArteriosclerosis, Thrombosis and Vascular Biology meeting, we presented Phase 2 data on
ISIS-APOCIII
Rx
in patients with type 2 diabetes and high triglycerides. In this study, patients with
diabetes experienced statistically significant decreases in triglyceride levels and statistically
significant improvements in glucose control with trends toward enhanced insulin sensitivity.
•
At the National LipidAssociation meeting, we presented Phase 2 data on ISIS-APOCIII
Rx
in
patients with FCS. FCS is a rare genetic disorder characterized by severely elevated levels of
triglycerides. Current treatment options are inadequate and, as a result, patients with FCS have an
increased risk of recurrent and potentially fatal pancreatitis and other complications. In this study,
patients with extremely high triglycerides experienced substantial reductions of triglycerides that
correlated with substantial reductions in triglyceride-rich chylomicrons. We published these data in
the NewEngland Journal of Medicine.
•
At the 2014 European Society of Cardiology Congress, our collaborator, John Kastelein, M.D.,
Ph.D. presented an overview of the ISIS-APOCIII
Rx
Phase 2 program in which treatment with
ISIS-APOCIII
Rx
produced consistent, robust and statistically significant reductions in triglycerides,
apoC-III and non-HDL-cholesterol and increases in HDL-cholesterol in all patient populations
evaluated.
•
At the European Society of Cardiology Congress, Dr. Sotirios Tsimikas, M.D. presented data from the
Phase 1 study of ISIS-APO(a)
Rx
in healthy volunteers. In this study, ISIS-APO(a)
Rx
treatment produced
dose-dependent and significant reductions in Lp(a) levels in these subjects.
•
We reported positive clinical results for ISIS-SMN
Rx
from two open-label Phase 2 studies in infants and
children with SMA, which were consistent with data reported earlier in the year at theAmericanAcademy
of Neurology meeting. SMA is a severe genetic disease that is the leading genetic cause of infant mortality.
•
At theWorldMuscle Society Congress, we reported the median event-free age of infants with
SMAas of September 2, 2014, which compared favorably to that of infants with SMA in the
PNCR natural history study. Time- and dose-dependent increases in muscle function scores were
observed in both infants and children with SMA in the on-going study. We presented clinical data
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