showing that ISIS-SMN
Rx
is distributed throughout the spinal cord and neurons with greater
amounts of full-length SMN2 mRNAand SMN protein in tissues from ISIS-SMN
Rx
-treated infants
compared to the amounts of full-length SMN2 mRNAand SMN protein in the tissues analyzed
from untreated SMA infants.
•
At theAmerican Diabetes Association Scientific Sessions, we reported Phase 2 data on ISIS-GCGR
Rx
demonstrating that patients with type 2 diabetes uncontrolled on stable metformin therapy experienced up to
a 2.25 percentage point mean reduction in HbA1c levels after 13 weeks of treatment with ISIS-GCGR
Rx
.
•
We reported top-line Phase 2 data on ISIS-PTP1B
Rx
demonstrating that patients with type 2 diabetes who
are uncontrolled on metformin with or without sulfonylurea experienced statistically significant mean
reductions in body weight and HbA1c (0.7 percentage point) at 36 weeks.
•
At theAmerican Society of Hematology annual meeting, we reported Phase 2 clinical results for ISIS-FXI
Rx
in patients undergoing total knee replacement. The results showed that ISIS-FXI
Rx
-treated patients
experienced a seven-fold lower incidence of venous thromboembolism and numerically fewer bleeding
events compared to patients treated with enoxaparin.
•
These data demonstrate that ISIS-FXI
Rx
can dissociate the antithrombotic effect from the bleeding
risk in patients. This is the first time an antithrombotic drug has demonstrated this profile. We
published the Phase 2 clinical data of ISIS-FXI
Rx
in the NewEngland Journal of Medicine
•
We reported Phase 2 results showing that ISIS-CRP
Rx
produced statistically significant mean reductions of
CRP protein of 65%with reductions as great as 84% in patients withAF. In addition, two patients who had
elevated levels of CRP (>5 mg/L) experienced a reduction of CRP that was associated with a decline to zero
in overall AF burden while on treatment.
•
ATL reported Phase 2 data onATL1103 in patients with acromegaly. In this study, ATL reported that
treatment withATL1103 produced a statistically significant average reduction in IGF-1, levels at the 400 mg
per week dose.
•
OncoGenex reported top-line Phase 2 data on apatorsen (OGX-427) in patients with metastatic bladder
cancer. In this study, the Borealis-1 study, OncoGenex reported that treatment with apatorsen in combination
with gemcitabine/cisplatin at the 600 mg dose showed a 14 percent reduction in risk of death and a
17 percent reduction in progressive disease and death.
•
AstraZeneca presented data from a Phase 1/2 clinical study of ISIS-STAT3-2.5
Rx
(AZD9150) at the 26
th
European Organization for Research and Treatment of Cancer. In this study, preliminary evidence of
antitumor activity was observed in patients with cancer, including advanced/metastatic hepatocellular
carcinoma. Additional data presented at the conference demonstrated that ISIS-STAT3-2.5
Rx
reduced STAT3
levels in multiple cell types relevant to cancer growth and survival, clinically and pre-clinically. AstraZeneca
also presented preclinical data on ISIS-AR-2.5
Rx
(AZD5312) showing that the drug is active in several
tumor models.
•
We reported Phase 1 results showing that ISIS-PKK
Rx
produced significant, dose-dependent reductions of
PKK of up to 95 percent in healthy volunteers.
•
Regulus reported results from a completed clinical study on RG-101, an anti-miR drug in development to
treat patients with hepatitis C virus, or HCV. In this study, a single dose of either 2 mg/kg or 4 mg/kg of
RG-101 demonstrated a substantial mean reduction in viral load in patients with varied HCV genotypes and
treatment history.
•
Together with our partners, we continued to advance our pipeline of drugs.
•
We initiated ENDEAR and CHERISH, Phase 3 studies evaluating ISIS-SMN
Rx
in infants and
children with SMA, respectively.
•
We initiatedAPPROACH, the Phase 3 study evaluating ISIS-APOCIII
Rx
in patients with familial
chylomicronemia syndrome.
•
Achaogen initiated a Phase 3 study of plazomicin in patients with serious multi-drug resistant,
gram-negative bacterial infections.
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