2 0 1 6 A N N U A L R E P O R T
LET TER FROM T HE CEO
Dear Shareholder:
In 2014, we began charting a course for the next stage of Acorda’s growth, seeking
to identify and acquire promising late-stage therapeutic candidates outside the
company to bolster our internal development activities. These actions were important
to accelerate the pursuit of our mission while improving the opportunity to generate
long-term value for our shareholders.
Ron Cohen, M.D.
President and CEO
As a result of this new strategy, Acorda successfully
option for people with Parkinson’s experiencing highly
We anticipate the following milestones in the next
Our top priorities are:
acquired two companies with late-stage programs in
disruptive and sometimes unexpected OFF periods in their
12 months:
Parkinson’s disease: Civitas Therapeutics in the fall of
daily lives.
2014 and Biotie Therapies in early 2016. If successful,
the key programs from these acquisitions, CVT-301 and
As we evolve toward Acorda’s future in Parkinson’s
tozadenant, will serve as the foundation for Acorda’s
disease, we acknowledge the challenges we are experi-
future value and position the Company as a leader in the
encing: the failures of two of our development programs
treatment of Parkinson’s disease. Parkinson’s affects
(PLUMIAZ and dalfampridine-SR for post-stroke walking
more than one million Americans and between 7 million
diffi culties) and a signifi cant setback for AMPYRA, which
and 10 million people around the world. We are excited
has enabled the Company’s historical growth. A U.S.
by the potential for CVT-301 and tozadenant to address
District Court invalidated four patents that would have
unmet needs in the Parkinson’s community.
preserved market exclusivity for AMPYRA into the next
decade. While we strongly disagree with the Court’s ruling
In 2016, we focused on advancing these newly acquired
and will appeal the decision, we are prepared should we
late-stage Parkinson’s disease programs. Our acquisition
ultimately lose market exclusivity for AMPYRA in July
strategy has begun to deliver results, most recently with
2018. We already have acted decisively to reduce expenses,
the positive effi cacy and safety data from Phase 3 pivotal
downsizing the Company within days of the Court’s ruling.
and long-term safety trials of CVT-301 that will support
Restructuring was painful, yet a necessary step to ensure
our upcoming New Drug Application (NDA) submission to
that we focus resources on bringing our Parkinson’s
the U.S. Food and Drug Administration (FDA). If approved,
disease candidates to the marketplace.
we believe CVT-301 likely will become a key therapeutic
• CVT-301: We plan to submit an NDA to the FDA in the
second quarter of 2017. We believe CVT-301 has the
potential to achieve at least $500 million in annual net
sales in the U.S. We also plan to submit a Marketing
Authorization Application (MAA) to the European
Medicines Agency (EMA) by year-end.
• Tozadenant: An oral adenosine A2a antagonist,
tozadenant represents a potential fi rst-in-class
treatment for Parkinson’s disease in the U.S. Phase 2b
data showed its potential to lower average daily OFF
time by more than an hour relative to placebo, in people
already being treated with multiple other medications.
We expect results from our pivotal Phase 3 clinical trial
in Q1 2018. As part of the Phase 3 program, we also
initiated an open-label, long-term safety study in the
second quarter of 2017. We believe that tozadenant,
if approved, represents a commercial opportunity that
could exceed that for CVT-301.
1) submitting an NDA for CVT-301 in Q2 2017;
2) submitting an MAA to the EMA for CVT-301
by the end of 2017;
3) planning for commercialization and launch of
CVT-301 in the U.S.; and
4) executing the ongoing tozadenant clinical program,
with pivotal effi cacy data expected in Q1 2018.
On behalf of our Management Team, Board of Directors
and our associates: thank you, our shareholders, for your
continued support. Our focus to deliver both promising
therapies and shareholder value has only sharpened over
the past few months and we look forward to sharing with
you the signifi cant opportunities that lie ahead.
Ron Cohen, M.D.
Ron Cohen, M.D.
Ron Cohen, M.D.
President and CEO
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2
ACORDA BUSINESS HIGHLIGHTS
IN 2016 AND EARLY 2017
AMPYRA
BUSINESS DEVELOPMENT
• U.S. District Court in Delaware ruled in March
2017 that four of Acorda’s patents (8,663,685,
8,440,703, 8,354,437, and 8,007,826) were
invalid, while upholding a fifth patent (5,540,938)
expiring July 30, 2018. Acorda is appealing
the District Court decision. If this appeal is
unsuccessful, AMPYRA will lose market exclusivity
after July 30, 2018.
• AMPYRA net revenue was $493 million in 2016,
a 13% increase over 2015. We are projecting
continued growth in 2017, guiding to net sales of
$535-$545 million.
CORPORATE RESTRUCTURING
• As a result of the District Court’s decision on our
AMPYRA patents, we implemented a headcount
reduction of approximately 20% in April 2017.
We expect to realize estimated annualized cost
savings of approximately $21 million beginning in
the second quarter of 2017.
• In addition, R&D and SG&A expenses will be
reduced. Further details will be provided in our Q1
investor update.
• We completed the acquisition of Biotie
Therapies, which included global rights to two
clinical-stage Parkinson’s disease programs.
Tozadenant, a selective adenosine A2a antagonist,
is being developed to reduce daily average OFF
time in people with Parkinson’s.
CLINICAL DEVELOPMENT
• Received Phase 3 data for CVT-301. Based on
positive efficacy data announced in February 2017,
coupled with data from two long-term safety
studies, we are planning to file an NDA for CVT-301
by the end of Q2 2017. Pending FDA review and
approval, we are planning for a potential commercial
launch of this product in 2018.
• CVT-427, an inhaled formulation of zolmitriptan
for acute migraine, showed favorable results in a
pharmacokinetic Phase I study. Some asthmatic
subjects showed evidence of acute, reversible
bronchoconstriction in a subsequent special
population study, which we are evaluating.
• Discontinued two late-stage clinical programs.
Results from clinical trials of PLUMIAZ for
cluster seizures in epilepsy and dalfampridine-SR
for treatment of post-stroke walking difficulties did
not show the pharmacokinetic profile and sufficient
efficacy needed to advance these programs.
• Exploring partnerships for early stage programs:
MANUFACTURING
SYN120, a 5-HT6/2A dual antagonist, is being
studied in a Phase 2 clinical trial in Parkinson’s
disease-related dementia. We expect this study
to be completed in the first half of 2018.
rHIgM22 is currently in a Phase 1 clinical trial
for remyelination in multiple sclerosis, which
we expect to complete in the second half of 2017.
• Converted Acorda’s Chelsea location to a
dedicated manufacturing site in preparation
for potential regulatory approval of CVT-301.
Non-manufacturing activities were moved out of
the Chelsea facility to minimize GMP compliance
risks and support launch readiness. Non-301
activities were moved to a small satellite lab space
located in Waltham, MA.
Cimaglermin alfa for the treatment of heart
failure was put on clinical hold following a
safety report of hepatotoxicity in our second
Phase 1 clinical trial.
• Manufacturing scale up continuing: 2016 marked
the shift to 24/7 manufacturing capabilities for
CVT-301.
33
4
PIP ELINE
Acorda has a leading pipeline of novel neurological
therapies addressing a range of disorders, including
Parkinson’s disease (PD), migraine and multiple sclerosis.
With two late-stage programs exploring different treatment
approaches to Parkinson’s, we are positioned to become a
leader in PD therapeutic development and commercialization.
We plan to submit a New Drug Application (NDA) to the U.S.
Food and Drug Administration (FDA) for CVT-301 by June
2017, followed by a regulatory fi ling in Europe by the end of
the year. We expect results from our pivotal Phase 3 clinical
trial of tozadenant in Q1 2018.
Parkinson’s
Disease
Parkinson’s
Disease
Parkinson’s
Disease
(timolumab)
Primary Sclerosing
Cholangitis (PSC)
Migraine
5
6
HI GHLI GHTS
CVT-301
7
AMPYRA NET SALES
$493 M
13% Growth from 2015
SUCCESSFUL COMPLETION OF
Women
Millennials
Baby Boomers
Medium Companies
GREAT
PLACE
TO
WORK®
2016
Best Workplaces
GREAT
PLACE
TO
WORK®
2016
Best Workplaces
GREAT
PLACE
TO
WORK®
2016
Best Workplaces
GREAT
PLACE
TO
WORK®
2016
Best Workplaces
CVT-301
PHASE 3 PROGRAM
FDA SUBMISSION TARGETED IN Q2 2017
ACQUIRED BIOTIE THERAPIES
ADDED INVESTIGATIONAL THERAPIES
TOZADENANT, SYN120
AND BTT1023 TO ACORDA PIPELINE
THE WORKPLACE ENVIRONMENT CREATED BY OUR ASSOCIATES HAS RECEIVED
NATIONAL AND LOCAL RECOGNITION, INCLUDING BEING NAMED ONE OF THE
100 BEST MEDIUM WORKPLACES IN THE U.S. BY FORTUNE MAGAZINE. OUR
CULTURE OF TRUST, COLLABORATION AND OPEN COMMUNICATION IS CRITICAL
TO ADVANCING OUR MISSION TO DEVELOP THERAPIES THAT RESTORE FUNCTION
AND IMPROVE THE LIVES OF PEOPLE WITH NEUROLOGICAL DISORDERS.
M AN AGEMENT
MANAGEMENT
TEAM
Ron Cohen, M.D.
President and Chief Executive Offi cer
David Lawrence, M.B.A.
Chief, Business Operations and
Principal Accounting Offi cer
Richard P. Batycky. Ph.D.
Chief Technology Offi cer and Site Head
Burkhard Blank, M.D.
Chief Medical Offi cer
Andrew R. Blight, Ph.D.
Chief Scientifi c Offi cer
Denise Duca, Ed.M.
Executive Vice President,
Human Resources
Andrew A. Hindman
Chief Business Development Offi cer
Lauren Sabella
Chief Commercial Offi cer
Tierney Saccavino
Executive Vice President,
Corporate Communications
Jane Wasman, J.D.
President, International &
General Counsel
BOARD OF
DORECTORS
Ron Cohen, M.D.
Founder
Lorin J. Randall
Board Member since 2006
Barry Greene
Steven M. Rauscher
Board Member since 2007
Board Member since 2005
Peder K. Jensen, M.D.
Ian F. Smith
Board Member since 2011
Board Member since 2007
John P. Kelley
Board Member since 2008
Catherine D. Strader, Ph.D.
Board Member since 2017
Sandra Panem, Ph.D.
Board Member since 1998
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Contact:
Felicia Vonella
Executive Director, Investor Relations
fvonella@acorda.com
914-326-5146