potential competitor to ISIS-APOCIII
Rx
is pradigastat, a drug in Phase 3 development in patients with FCS. If
pradigastat is successful in clinical studies and receives marketing approval this oral drug could compete with
ISIS-APOCIII
Rx
. Data from a Phase 2 study of pradigastat showed effective lowering of triglycerides in patients,
but the high incidence of gastrointestinal side effects observed could limit the drugs’ tolerability. Another
potential oral drug competitor is CAT-2003, which is in Phase 2 development to treat patients with FCS, or
extremely high triglycerides. Based on the mechanism of action of CAT-2003, we believe CAT-2003 is likely to
have an effect only in a small subset of FCS patients.
ISIS-SMN
Rx
In 2014, we initiated two Phase 3 programs on ISIS-SMN
Rx
in infants and in children with SMA. SMA is a
rare genetic disease for which there is no approved therapy on the market. Patients with SMAare treated with
palliative care that focuses on helping to maintain respiratory health. We plan to develop ISIS-SMN
Rx
to treat all
forms of SMA. We believe that of the drugs that are in development, ISIS-SMN
Rx
’s closest competitor is
RG7800. RG7800 is a small molecule gene splicing modifier that PTCTherapeutics is co-developing with Roche
and the SMAFoundation. In November 2014, PTCTherapeutics announced that it initiated a Phase 1b/2a study
of RG7800 in adults and pediatric patients with SMA. Another oral drug, olesoxime, has shown beneficial effect
on the maintenance of motor function in SMApatients. Roche announced in January 2015 that it plans to acquire
Trophos, the company developing olesoxime, which could allowRoche to utilize a single marketing and
commercial salesforce for both RG7800 and olesoxime, should both drugs reach the market. Trophos reported
that SMApatients treated with olesoxime had less severe loss of motor function compared to patients treated
with placebo. AveXis is developing ChariSMA, a gene therapy in Phase 1 development to treat infants with
SMA. The first patient was dosed in December 2014. Because chariSMA is the first gene therapy trial for the
treatment of patients with SMAand it has just initiated clinical trials, the therapeutic potential of this treatment is
difficult to assess. To compete with ISIS-SMN
Rx
these therapeutic programs would need to advance into Phase 3
studies, eventually achieve marketing approval, and show a better product profile than ISIS-SMN
Rx
. If these
programs progress through clinical studies and the resulting drugs receive marketing approval, these drugs could
compete with ISIS-SMN
Rx
as a treatment for patients with SMA.
Employees
As of February 17, 2015, we employed 390 people. A significant number of our management and
professional employees have had prior experience with pharmaceutical, biotechnology or medical product
companies. Collective bargaining agreements do not cover any of our employees, and management considers
relations with our employees to be good.
Executive Officers of Isis
The following sets forth certain information regarding our executive officers as of February 17, 2015:
Name
Age
Position
Stanley T. Crooke, M.D., Ph.D.
69 Chairman, Chief Executive Officer and President
B. Lynne Parshall, J.D.
60 Director, Chief Operating Officer and Secretary
C. Frank Bennett, Ph.D.
58 Senior Vice President, Antisense Research
Sarah Boyce
43 Chief Business Officer
Richard S. Geary, Ph.D.
57 Senior Vice President, Development
Elizabeth L. Hougen
53 Senior Vice President, Finance and Chief Financial Officer
Brett P. Monia, Ph.D.
53 Senior Vice President, Drug Discovery and Corporate Development
Patrick R. O’Neil, Esq.
41 Senior Vice President, Legal and General Counsel
STANLEYT. CROOKE, M.D., Ph.D.
Chairman, Chief Executive Officer and President
Dr. Crooke is a founder of Isis and has been Chief Executive Officer and a Director since January 1989. He
was elected Chairman of the Board in February 1991. Prior to founding Isis, from 1980 until January 1989,
Dr. Crooke was employed by SmithKline Beckman Corporation, a pharmaceutical company, where his titles
included President of Research and Development of SmithKline and French Laboratories.
52
