Under our alliance, Genzyme is responsible for the continued development and commercialization of
KYNAMRO. Genzyme is marketing KYNAMRO in the United States for patients with HoFH and has also
obtained marketing approval in other countries, includingMexico, Argentina, South Korea and Peru, and is
pursuing marketing approval in multiple additional markets. As part of the agreement, we contributed the first
$125 million in funding for the development costs of KYNAMRO. In 2011, we satisfied our development
funding obligation. As such, we and Genzyme are sharing development expenses equally until KYNAMRO is
profitable.
The license and co-development agreement for KYNAMROwill continue in perpetuity unless we or
Genzyme terminate it earlier under the following situations:
•
Genzyme may terminate the license and co-development agreement at any time by providing written
notice to Isis;
•
We may terminate the license and co-development agreement on a country-by-country basis or in its
entirety upon Genzyme’s uncured failure to use commercially reasonable efforts to develop and
commercialize KYNAMRO in the United States, France, Germany, Italy, Spain, the United Kingdom,
Japan and Canada; and
•
Either we or Genzyme may terminate the license and co-development agreement upon the other party’s
uncured failure to perform a material obligation under the agreement.
Upon termination of the license and co-development agreement, the license we granted to Genzyme for
KYNAMROwill terminate and Genzyme will stop selling the product. In addition, if Genzyme voluntarily
terminates the agreement or we terminate the agreement in a country or countries for Genzyme’s failure to
develop and commercialize KYNAMRO, then the rights to KYNAMROwill revert back to us and we may
develop and commercialize KYNAMRO in the countries that are the subject of the termination, subject to a
royalty payable to Genzyme.
If we are the subject of an acquisition, then within 180 days following the acquisition, Genzyme may elect
to purchase all of our rights to receive payments under the KYNAMRO license and co-development agreement
for a purchase price to be mutually agreed to by us and Genzyme, or, if we cannot agree, a fair market value
price determined by an independent investment banking firm.
During 2013 and 2012, we earned revenue of $32.5 million, and $67.6 million, respectively, from our
relationship with Genzyme, which represented 22 percent and 66 percent, respectively, of our total revenue for
those years. During 2014, we did not earn any revenue from our relationship with Genzyme.
GSK
InMarch 2010, we entered into a strategic alliance with GSK using our antisense drug discovery platform
to seek out and develop new drugs against targets for rare and serious diseases, including infectious diseases and
some conditions causing blindness. Our strategic alliance currently includes five drugs in development. GSK has
the exclusive option to license drugs resulting from this alliance at Phase 2 proof-of-concept for a license fee. If
GSK exercises its exclusive option for any drugs resulting from this alliance, it will be responsible for all further
global development, regulatory and commercialization activities for such drug. Under the terms of the agreement,
we received a $35 million upfront payment and inMay 2011 we received a $3 million payment when we and
GSK expanded the collaboration.
In October 2012, we and GSK amended the original agreement to reflect an accelerated clinical
development plan for ISIS-TTR
Rx
. From inception through February 2015, we have received $45 million,
primarily in milestone payments, fromGSK related to the development of ISIS-TTR
Rx
. We are also eligible to
earn an additional $25 million in pre-licensing milestone payments associated with the ISIS-TTR
Rx
Phase 2/3
study. In addition, under the amended agreement, we and GSK increased the regulatory and commercial
milestone payments we can earn should ISIS-TTR
Rx
receive marketing approval and meet pre-agreed sales
targets.
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