•
$36 million fromGSK related to the development of ISIS-TTR
Rx
, ISIS-HBV
Rx
, ISIS-GSK4-L
Rx
and ISIS-RHO-2.5
Rx,
formerly ISIS-GSK5-2.5
Rx
.
•
$35 million from Janssen related to our alliance to treat autoimmune disorders of the GI tract.
•
$23 million fromAstraZeneca related to the development of ISIS-AR-2.5
Rx
and ISIS-STAT3-2.5
Rx
.
•
$10 million fromAlnylam related toAlnylam’s license of our technology to its partners.
•
$4 million fromAchaogen for the initiation of a Phase 3 study of Plazomicin.
•
We received cash through the sale of stock we owned in our satellite company partners of more than
$25 million, including more than $20 million from the sale of a portion of our Regulus stock.
•
We and our partners were recognized by the drug development community for our innovative and
collaborative alliances and our commitment to developing drugs to treat patients with serious, unmet
medical needs.
•
We and Genzyme received the 2014 Partners in Progress CorporateAward from the National
Organization for Rare Disorders, or NORD, for the development and approval of KYNAMRO, a
drug selected for being a very important orphan therapy to reach the market in the United States.
This award honors companies that have brought important and innovative treatments to market for
patients with rare disorders.
•
Our innovative collaboration with Biogen Idec was voted breakthrough alliance of 2014 by
Thomson Reuters Recap.
•
We added Joseph Loscalzo, M.D., Ph.D. to our Board of Directors.
•
Our senior vice president of research, Frank Bennett, Ph.D., was awarded the Commitment to a CureAward
by theALSAssociation for his research and commitment to develop a treatment for ALS.
•
Our founder, CEO and chairman of the board of directors, Stanley T. Crooke, M.D., Ph.D., was recognized
with several awards.
•
The prestigious SCRIP LifetimeAchievement Award.
•
The SMABreakthroughAward by CURE SMA.
Drug Discovery and Development
Introduction to Drug Discovery
Proteins are essential working molecules in a cell. Almost all human diseases result from inappropriate
protein production or improper protein activity. Scientists use traditional drug discovery methods to design drugs
to interact with the proteins in the body that are supporting or causing a disease. Antisense drugs are different
from traditional small molecule drugs because typically antisense drugs interrupt the production of
disease-causing proteins by targeting RNAs. RNAs are naturally occurring molecules in the body that provide the
information the cell needs to produce proteins. When our antisense drugs bind to the specific RNAs of a
particular gene, they will ultimately inhibit or alter the expression of the protein encoded in the target gene or
degrade the RNA.
Our Development Projects
We are the leader in the discovery and development of an exciting class of RNA-targeted drugs called
antisense drugs. With our proprietary drug discovery platformwe can rapidly identify drugs from a wealth of
potential targets to treat a broad range of diseases. We focus our efforts in therapeutic areas where our drugs will
work best, efficiently screening many targets in parallel and carefully selecting the best drugs. When we combine
this efficiency with our rational approach to selecting disease targets, we can build a large and diverse portfolio
of drugs designed to treat a variety of health conditions, with an emphasis on cardiovascular, metabolic, severe
and rare diseases, including neurological disorders, and cancer. We and our partners are developing antisense
drugs for systemic, intrathecal and local delivery. We expect to continue to add new drugs to our pipeline,
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