•
We initiated a Phase 2 study of ISIS-APO(a)
Rx
in patients with high levels of lipoprotein(a), an
independent risk factor for cardiovascular disease.
•
We initiated a Phase 2 study for ISIS-GCCR
Rx
in patients with type 2 diabetes.
•
AstraZeneca initiated a Phase 2 study for ISIS-STAT3-2.5
Rx
in patients with hepatocellular
carcinoma.
•
OncoGenex initiated a Phase 2 study for apatorsen in patients with non-small cell lung cancer.
•
We initiated a Phase 1/2 study of ISIS-DMPK-2.5
Rx
in healthy volunteers and in patients with
myotonic dystrophy type 1.
•
Regulus initiated a Phase 1/2 study for RG-101 in healthy volunteers and in patients with HCV.
•
We initiated a Phase 1 study of ISIS-ANGPTL3
Rx
and ISIS-PKK
Rx
in healthy volunteers.
•
We added twelve drugs to our pipeline.
•
We received European Orphan Drug Designation for ISIS-APOCIII
Rx
for the treatment of patients with
familial chylomicronemia syndrome and for ISIS-TTR
Rx
for the treatment of patients with TTR amyloidosis.
We received FDAOrphan Drug Designation for ISIS-DMPK-2.5
Rx
for the treatment of patients with DM1.
Corporate Highlights in 2014 and early 2015
•
We formed a wholly owned subsidiary, Akcea, to develop and commercialize our lipid drugs,
ISIS-APOCIII
Rx
, ISIS-APO(a)
Rx
, ISIS-ANGPTL3
Rx
and any follow on drugs for these programs.
•
We appointed Paula Soteropoulos as president and chief executive officer of Akcea.
Ms. Soteropoulos will utilize her expertise in commercializing drugs for severe, rare and
cardiovascular diseases in global markets to advanceAkcea’s novel lipid franchise through
development and commercialization.
•
We formed an alliance with Janssen to discover and develop antisense drugs to treat autoimmune disorders
of the GI tract. We received $35 million in upfront payments and are eligible to receive nearly $800 million
in development, regulatory and sales milestone payments and license fees for the programs under this
alliance. We will also receive tiered royalties up to the low double-digits on sales of drugs successfully
commercialized.
•
We formed an alliance withAstraZeneca to discover and develop novel delivery methods for antisense
oligonucleotides. The agreement builds on an existing collaboration between us andAstraZeneca, and
supports AstraZeneca’s research and development capabilities in the area of antisense oligonucleotide-based
therapeutics and RNAbiology.
•
We strengthened our management teamwith the addition of Sarah Boyce as chief business officer.
Ms. Boyce will provide strategic marketing and business expertise from a commercial background to our
management team.
•
Abbott obtained CEMark and launched the Ibis Biosciences diagnostic platform, now called IRIDICA, in
Europe. IRIDICA is available in Europe and other CE-Mark recognized countries. IRIDICAwas developed
from technology discovered by us and transferred to Ibis Biosciences.
•
We andAlnylam formed a new agreement that included a cross-license of intellectual property on four
disease targets, providing each company with exclusive RNA therapeutic license rights for two programs.
•
We successfully completed an offering of $500 million aggregate principal amount of 1 percent convertible
senior notes due in 2021 in a private placement. We used a significant amount of the net proceeds from the
offering to repurchase a large portion of our 2¾ percent convertible senior notes due 2019.
•
We generated more than $250 million in payments from our partners, including the following:
•
$118 million fromBiogen Idec, including payments related to advancing ISIS-SMN
Rx
, initiating a
Phase 1 study of ISIS-DMPK-2.5
Rx
, validating two undisclosed targets to treat neurological
disorders, and selecting two development candidates, ISIS-BIIB3
Rx
and ISIS-BIIB4
Rx
, to move
into our pipeline.
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