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AbbVie, Inc.
We have a collaboration agreement with AbbVie
for the development and commercialization of
ZINBRYTA in MS. Under this agreement, we and
AbbVie conduct ZINBRYTA co-promotion activities in
the U.S., E.U. and Canadian territories, and we are
responsible for all manufacturing and research and
development activities.
For information on the Article 20 Procedure of
ZINBRYTA and resulting impairment of ZINBRYTA
related assets, please read Note 20, Collaborative
and Other Relationships, to our consolidated financial
statements included in this report.
Acorda Therapeutics, Inc.
We have a collaboration and license agreement
with Acorda to develop and commercialize products
containing fampridine, such as FAMPYRA, in markets
outside the U.S. We are responsible
for all regulatory
activities and the future clinical development of
related products in those markets.
Alkermes
We have an exclusive license and collaboration
agreement with Alkermes to develop and
commercialize BIIB098, an oral MMF prodrug in Phase
3 development for the treatment of relapsing forms of
MS.
Applied Genetic Technologies Corporation
We have a collaboration agreement with Applied
Genetic Technologies Corporation (AGTC) to develop
gene-based therapies for multiple ophthalmic
diseases. This collaboration is focused on the
development of a clinical-stage candidate for X-linked
Retinoschisis (XLRS) and a preclinical candidate for
the treatment of X-linked Retinitis Pigmentosa (XLRP),
for which we were granted worldwide
commercialization rights. This agreement also
provides us with options to early stage discovery
programs in two ophthalmic diseases and one non-
ophthalmic condition.
Bristol-Myers
Squibb Company
We have an exclusive license agreement with
BMS for the development and commercialization of
BIIB092. Under this agreement, we received
worldwide rights to BIIB092 and are responsible for
the full development and global commercialization of
BIIB092 in AD and PSP.
Eisai Co., Ltd.
We have a collaboration agreement with Eisai to
jointly develop and commercialize E2609 and
BAN2401, two Eisai product candidates for the
treatment of AD. Eisai serves as the global
operational and regulatory lead for both E2609 and
BAN2401 and all costs, including research,
development, sales and marketing expenses, are
shared equally between us and Eisai. Following
marketing approval in major markets, we will co-
promote E2609 and BAN2401 with Eisai and share
profits equally.
We also have
the Aducanumab Collaboration
Agreement with Eisai for the joint development and
commercialization of aducanumab. Under the
Aducanumab Collaboration Agreement, the two
companies will co-promote aducanumab with a region-
based profit split and we will continue to lead the
ongoing Phase 3 development of aducanumab.
We and Eisai will co-promote AVONEX, TYSABRI
and TECFIDERA in Japan in certain settings and Eisai
will distribute AVONEX, TYSABRI, TECFIDERA and
PLEGRIDY in India and other Asia-Pacific markets,
excluding China.
Genentech (Roche Group)
We have a collaboration agreement with
Genentech which entitles us to certain financial and
other rights with respect to RITUXAN, GAZYVA,
OCREVUS and other anti-CD20 product candidates.
Ionis Pharmaceuticals, Inc.
We have
an exclusive, worldwide option and
collaboration agreement with Ionis relating to the
development and commercialization of up to three
gene targets, and an exclusive, worldwide option and
collaboration agreement with Ionis under which both
companies are responsible for the development and
commercialization of SPINRAZA for the treatment of
SMA.
We also have research collaboration agreements
with Ionis, under which both companies perform
discovery level research and will develop and
commercialize new ASO drug candidates for the
treatment of SMA and additional antisense and other
therapeutics for the treatment of neurological
disorders.
Neurimmune
We have a collaboration and license agreement
with Neurimmune for the development and
commercialization of antibodies for the treatment of
AD, including aducanumab. Under this agreement, we
are responsible for the development, manufacturing
and commercialization of all licensed products.
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Samsung Bioepis
We and Samsung Biologics
established a joint
venture, Samsung Bioepis, to develop, manufacture
and market biosimilar pharmaceuticals. We also have
an agreement with Samsung Bioepis to
commercialize, over a 10-year term, three anti-TNF
biosimilar product candidates in specified E.U.
countries and, in the case of BENEPALI, Japan. Under
this agreement, we are manufacturing and
commercializing BENEPALI, an etanercept biosimilar
referencing ENBREL, and FLIXABI, an infliximab
biosimilar referencing REMICADE.
In addition to our joint venture and
commercialization agreement with Samsung Bioepis,
we license certain of our proprietary technology to
Samsung Bioepis in connection with Samsung
Bioepis' development, manufacture and
commercialization of its biosimilar products. We also
provide technical development and technology
transfer
services to Samsung Bioepis, and
manufacture clinical and commercial quantities of
bulk drug substance of Samsung Bioepis' biosimilar
products.
University of Pennsylvania
We have a collaboration and alliance with the
University of Pennsylvania (UPenn) to advance gene
therapy and gene editing technologies. The
collaboration is primarily focused on the development
of therapeutic approaches that target the eye, skeletal
muscle and the central nervous system. The alliance
is also focused on the research and validation of next-
generation gene transfer technology using adeno-
associated virus gene delivery vectors and exploring
the expanded use of genome editing technology as a
potential therapeutic platform.
Regulatory
Our current and contemplated activities and the
products, technologies and processes that result from
such activities are subject to substantial government
regulation.
Regulation of Pharmaceuticals
Product Approval and
Post-Approval Regulation in
the U.S.
APPROVAL PROCESS
Before new pharmaceutical products may be
sold in the U.S., preclinical studies and clinical trials
of the products must be conducted and the results
submitted to the FDA for approval. With limited
exceptions, the FDA requires companies to register
both pre-approval and post-approval clinical trials and
disclose clinical trial results in public databases.
Failure to register a trial or disclose study results
within the required time periods could result in
penalties, including civil monetary penalties. Clinical
trial programs must establish efficacy, determine an
appropriate dose and dosing regimen, and define the
conditions for safe use. This is a high-risk process
that requires stepwise clinical studies in which the
candidate product must successfully meet
predetermined endpoints.
The results of the
preclinical and clinical testing of a product are then
submitted to the FDA in the form of a Biologics
License Application (BLA) or a New Drug Application
(NDA). In response to a BLA or NDA, the FDA may
grant marketing approval, request additional
information or deny the application if it determines
the application does not provide an adequate basis
for approval.
Product development and receipt of regulatory
approval takes a number of years, involves the
expenditure of substantial resources and depends on
a number of factors, including the severity of the
disease in question, the availability of alternative
treatments, potential
safety signals observed in
preclinical or clinical tests and the risks and benefits
of the product as demonstrated in clinical trials. The
FDA has substantial discretion in the product approval
process, and it is impossible to predict with any
certainty whether and when the FDA will grant
marketing approval. The agency may require the
sponsor of a BLA or NDA to conduct additional clinical
studies or to provide other scientific or technical
information about the product, and these additional
requirements may lead to unanticipated delay or
expense. Furthermore, even if a product is approved,
the approval may be subject to limitations based on
the FDA's interpretation of the existing pre-clinical or
clinical data.