42
measured, all patients continued to progress in their disease. If diflunisal is successful in receivingmarketing approval for TTR
amyloidosis, it could competewith ISIS-TTR
Rx
. In addition, Alnylam is developingRNAimolecules designed to inhibit the
productionof TTR and recently started a Phase 3program for its drug candidate, patisiran, inpatientswithFAP. IfAlnylam’s drug
candidates are successful in clinical studies and receivemarketing approval, they could competewith ISIS-TTR
Rx
.
ISIS-APOCIII
Rx
We have completed a broadPhase 2programon our novel triglyceride-loweringdrug, ISIS-APOCIII
Rx
, andwe plan to
initiate a Phase 3programon this drug in2014. ISIS-APOCIII
Rx
is indevelopment to treat patientswith severely high to extremely
high triglyceride levels. Current triglyceride-lowering therapies, includingniacin, fibrates and fishoils, are often inadequate in these
patients. Our plan is todevelop ISIS-APOCIII
Rx
to treat patientswith severely high triglycerides, includingFCSpatients, who are
unable to adequately reduce their triglyceride levels to acceptable levelswith current therapies. Based onour Phase 2data, we believe
that ISIS-APOCIII
Rx
willwork equallywell as a single agent or in combinationwithother triglyceride-loweringdrugs on themarket.
As such, we do not intend todisplace any existing therapywith ISIS-APOCIII
Rx
.
There are several drugs indevelopment to reduce triglyceride levels inpatientswith severely high triglycerides. Themost
advancedof these drugs is pradigastat, a drug inPhase 3development in patientswithFCS. Although the Phase 2data for pradigastat
showed effective loweringof triglycerides inpatients, the high incidence of gastrointestinal side effects observed could limit the
drugs’ tolerability. If pradigastat is successful in clinical studies and receivesmarketing approval this oral drug could competewith
ISIS-APOCIII
Rx
. CAT-2003 is anoral drug inPhase 2development to treat patientswithFCS, or extremely high triglycerides. Based
on themechanismof actionofCAT-2003, we believeCAT-2003 is likely to have an effect only in a small subset of FCS patients. As
such, if CAT-2003 is successful in clinical studies and receivesmarketing approval, we believe it could competewith ISIS-APOCIII
Rx
only in patientswith severely elevated triglyceride levels, which is the second indicationwe plan topursue for ISIS-APOCIII
Rx
.
ISIS-SMN
Rx
In2014, we plan tobegin a Phase 3programon ISIS-SMN
Rx
inboth infantswith type 1SMA and in childrenwith type 2 and
type 3SMA. SMA is a rare genetic disease forwhich there is no approved therapyon themarket. Current treatment for patientswith
SMA is palliative and focuses onhelping tomaintain respiratoryhealth. We plan todevelop ISIS-SMN
Rx
to treat all forms of SMA.
There are a small number of therapeutic programs designed to treat SMA that are in earlier stages of development compared to ISIS-
SMN
Rx.
To competewith ISIS-SMN
Rx
these therapeutic programswouldneed to advance intoPhase 3 studies, eventually achieve
marketing approval, and show a better product profile than ISIS-SMN
Rx
. If those programs progress through clinical studies and the
resultingdrugs receivemarketing approval, those drugs could competewith ISIS-SMN
Rx
as a treatment for patientswithSMA.
Employees
As of February17, 2014, we employed 304people in all of our functions, excludingmanufacturing and relateddepartments,
which employed61people. A significant number of ourmanagement andprofessional employees have hadprior experiencewith
pharmaceutical, biotechnology ormedical product companies. Collective bargaining agreements donot cover anyof our employees,
andmanagement considers relationswithour employees tobe good.
ExecutiveOfficers of Isis
The following sets forth certain information regardingour executive officers as of February17, 2014:
Name
Age
Position
StanleyT. Crooke,M.D., Ph.D.........
68 Chairman, Chief ExecutiveOfficer andPresident
B. Lynne Parshall, J.D. .....................
58 Director, ChiefOperatingOfficer andSecretary
C. FrankBennett, Ph.D.....................
57 SeniorVice President, AntisenseResearch
RichardS. Geary, Ph.D.....................
56 SeniorVice President, Development
ElizabethL. Hougen .........................
52 SeniorVice President, Finance andChief FinancialOfficer
Brett P.Monia, Ph.D.........................
52 SeniorVice President, DrugDiscovery andCorporateDevelopment
PatrickR. O’Neil, Esq. .....................
40 SeniorVice President, Legal andGeneral Counsel
