Custirsen
Issued patent claims have been obtained from an application jointly filed by Isis and OncoGenex to protect
the specific chemical composition of custirsen in the United States. The issued U.S. claims should protect
custirsen from generic competition in the United States until at least 2021. The table below lists the U.S. issued
patent:
Jurisdiction
Patent No.
Title
Expiration
Description of Claims
United States
6,900,187
TRPM-2ANTISENSE THERAPY
USINGANOLIOGNUCLEOTIDE
HAVING 2’-O-(2-METHOXY)
ETHYLMODIFICATIONS
2021
Antisense sequence and composition of
custirsen
Manufacturing Patents
We also own patents claiming methods of manufacturing and purifying oligonucleotides. These patents
claimmethods for improving oligonucleotide drug manufacturing, including processes for large-scale
oligonucleotide synthesis and purification. These methods allow us to manufacture oligonucleotides at lower cost
by, for example, eliminating expensive manufacturing steps.
We also rely on trade secrets, proprietary know-how and continuing technological innovation to develop and
maintain a competitive position in antisense therapeutics.
Government Regulation
Regulation by government authorities in the United States and other countries is a significant component in
the development, manufacture and commercialization of pharmaceutical products and services. In addition to
regulations enforced by the FDAand relevant foreign regulatory authorities, we are also subject to regulation
under the Occupational Safety and HealthAct, the Environmental ProtectionAct, the Toxic Substances Control
Act, the Resource Conservation and RecoveryAct and other present and potential future federal, state and local
regulations.
Extensive regulation by United States and foreign governmental authorities governs our manufacture,
development and potential sale of therapeutics. In particular, pharmaceutical products are subject to nonclinical
and clinical testing, as well as other approval requirements, by the FDA in the United States under the Federal
Food, Drug and CosmeticAct and other laws and by comparable agencies in those foreign countries in which we
conduct business. Various federal, state and foreign statutes also govern or influence the manufacture, safety,
labeling, storage, record keeping, marketing and quality of our products. State, local, and other authorities also
regulate pharmaceutical manufacturing facilities and procedures.
In January 2013, the FDAapproved the marketing application for KYNAMRO for patients with HoFH. Our
facility is subject to periodic inspection by the FDA to ensure that it is operating in compliance with cGMP
requirements. Approval of each new drug will require a rigorous manufacturing pre-approval inspection by
regulatory authorities.
Our operations may be directly, or indirectly through our customers, distributors, or other business partners,
subject to various federal and state fraud and abuse laws, including, without limitation, anti-kickback statutes and
false claims statutes. These laws may impact, among other things, our commercialization partners’ proposed
sales, marketing and education programs.
The U.S. Foreign Corrupt Practices Act, or FCPA, prohibits certain individuals and entities, including us,
from promising, paying, offering to pay, or authorizing the payment of anything of value to any foreign
government official, directly or indirectly, to obtain or retain business or an improper advantage. Violations of the
FCPAmay result in large civil and criminal penalties and could result in an adverse effect on a company’s
reputation, operations, and financial condition. Acompany may also face collateral consequences such as
debarment and the loss of export privileges.
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