achieve milestones in accordance with our or our investors’ expectations, including milestones for additional
approvals or sales expectations of KYNAMRO or milestones related to the Phase 3 programs for
ISIS-APOCIII
Rx
, ISIS-SMN
Rx
and ISIS-TTR
Rx
, the price of our securities could decrease.
For example, inMarch 2013 the CHMP of the EuropeanMedicines Agency maintained a negative opinion
for Genzyme’s marketing authorization application for KYNAMRO as a treatment for patients with HoFH.
If we cannot protect our patents or our other proprietary rights, others may compete more effectively
against us.
Our success depends to a significant degree upon whether we can continue to develop and secure
intellectual property rights to proprietary products and services. However, we may not receive issued patents on
any of our pending patent applications in the United States or in other countries. In addition, the scope of any of
our issued patents may not be sufficiently broad to provide us with a competitive advantage. Furthermore, our
issued patents or patents licensed to us may be successfully challenged, invalidated or circumvented so that our
patent rights would not create an effective competitive barrier or revenue source.
Intellectual property litigation could be expensive and prevent us frompursuing our programs.
From time to time we have to defend our intellectual property rights. In the event of an intellectual property
dispute, we sometimes need to litigate to defend our rights or assert them against others. Disputes can involve
arbitration, litigation or proceedings declared by the United States Patent and Trademark Office or the
International Trade Commission or foreign patent authorities. Intellectual property litigation can be extremely
expensive, and this expense, as well as the consequences should we not prevail, could seriously harm our
business. For example, in September 2011 we filed a patent infringement lawsuit against Santaris PharmaA/S
and Santaris PharmaA/S Corp. in the United States District Court of the Southern District of California, and in
November 2013 we filed a patent infringement lawsuit against Gilead Sciences Inc. in the United States District
Court of the Northern District of California. These lawsuits may be costly and may not be resolved in our favor.
If a third party claims that our drugs or technology infringe its patents or other intellectual property rights,
we may have to discontinue an important product or product line, alter our products and processes, pay license
fees or cease certain activities. We may not be able to obtain a license to needed intellectual property on
favorable terms, if at all. There are many patents issued or applied for in the biotechnology industry, and we may
not be aware of patents or patent applications held by others that relate to our business. This is especially true
since patent applications in the United States are filed confidentially for the first 18 months. Moreover, the
validity and breadth of biotechnology patents involve complex legal and factual questions for which important
legal issues remain.
If we fail to obtain timely funding, we may need to curtail or abandon some of our programs.
Many of our drugs are undergoing clinical studies or are in the early stages of research and development.
All of our drug programs will require significant additional research, development, preclinical and/or clinical
testing, regulatory approval and/or commitment of significant additional resources prior to their successful
commercialization. As of December 31, 2014, we had cash, cash equivalents and short-term investments equal to
$728.8 million. If we do not meet our goals to successfully commercialize KYNAMRO and our other drugs,
including ISIS-APOCIII
Rx
, ISIS-SMN
Rx
and ISIS-TTR
Rx
, or to license our drugs and proprietary technologies,
we will need additional funding in the future. Our future capital requirements will depend on many factors, such
as the following:
•
additional marketing approvals and successful commercial launch of KYNAMRO;
•
changes in existing collaborative relationships and our ability to establish and maintain additional
collaborative arrangements;
•
continued scientific progress in our research, drug discovery and development programs;
•
the size of our programs and progress with preclinical and clinical studies;
•
the time and costs involved in obtaining regulatory approvals;
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