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Acorda Therapeutics

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Industry Drug Manufacturers - Specialty & Generic
Employees 201-500
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FY2015 Annual Report · Acorda Therapeutics
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2 0 1 5   A N N U A L   R E P O R T

DEAR MEMBERS OF THE 
ACORDA COMMUNITY:

We aim to grow Acorda into the 
preeminent biopharmaceutical 
company delivering innovative 
therapies for people with 
neurological disorders, and are 
building on three pillars to drive 
long-term value creation:  

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Advancing our diversified clinical pipeline 
to approval and commercialization;  

Acquiring assets that leverage our core 

competencies in neurology; and

Continuing to grow sales in multiple 
sclerosis of AMPYRA® (dalfampridine) 
Extended Release Tablets, 10 mg.

We have made progress in all three areas since our last annual 
report. We:

  Announced the acquisition of Biotie Therapies in January 

2016. Key to this transaction are global rights to two clinical-stage 
Parkinson’s disease (PD) programs, which would complement our 
late-stage PD program, CVT-301. 

  Continued to advance our leading innovative neurology pipeline. 
Pending the close of the Biotie transaction, we will have eight 
clinical-stage assets — including four in Phase 3, two in Phase 
2 and two in Phase 1. With positive clinical trial data, we 
potentially will be able to submit New Drug Applications for 
three of our pipeline therapies — PLUMIAZ™ (diazepam) Nasal 
Spray, CVT-301 and tozadenant — by the end of 2018. We 
project these products, if approved, could have combined U.S. 
peak sales of over $1 billion.

  Successfully completed our first early-stage clinical study for 
CVT-427 in migraine and continued to enroll participants in our 
second Phase 1 study for rHIgM22 in MS.

  Achieved AMPYRA net revenue of $437 million in 2015, a 19% 
increase over 2014. We are projecting continued growth in 2016, 
guiding to net sales of $475-$485 million.

TRANSFORMING OUR PIPELINE
Acorda’s early history was defined by our efforts to develop and 
commercialize AMPYRA, which has become a standard of care for 
people with multiple sclerosis (MS) who experience MS-related walking 
difficulty. Over the past 24 months, we have dramatically enhanced our 
company’s profile. 

Our acquisition of Civitas Therapeutics in late 2014 added CVT-301, a 
highly promising therapy currently in Phase 3 development for treating 
OFF periods in people with Parkinson’s disease. In addition, this 
transaction brought to Acorda the novel ARCUS® inhaled dry powder 
technology platform, and a commercial-scale manufacturing facility 
for ARCUS-based products. ARCUS has shown the ability to deliver 
medication rapidly into the bloodstream, in much larger doses than 
other inhalation technologies. The ARCUS platform already has yielded 
a second pipeline product, CVT-427, an inhaled zolmitriptan formulation 
aimed at acute treatment of migraine, and has the potential to deliver 
others. We plan to initiate additional CVT-427 studies later this year.  

Our acquisition of Biotie Therapies, which we expect to complete in 
the second half of 2016, adds two Parkinson’s disease therapies to our 
pipeline that are complementary to CVT-301. One of these, tozadenant, 
is in Phase 3 clinical development. 

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Our legal team achieved a significant victory in a case against Mylan 
Laboratories, which had sought to litigate our patent case in its home 
state of West Virginia. The Federal Circuit Court of Appeals upheld 
the lower court’s verdict permitting the case to proceed in the District 
Court of Delaware, along with the other ANDA cases. Mylan has 
petitioned the Federal Circuit for a rehearing en banc, which we are 
now opposing.

Four of our Orange Book patents are also being challenged in a 
separate, relatively new U.S. Patent and Trademark Office process 
called Inter Partes Review (IPR). In 2015, the Patent Trial and Appeal 
Board (PTAB) rejected IPR filings by a hedge fund against two of 
our AMPYRA patents. In March 2016, the PTAB instituted four IPRs 
that were filed subsequently by the same hedge fund. We will litigate 
vigorously before the PTAB to defend our patents and anticipate a 
ruling on these IPR petitions by March 2017. 

LOOKING AHEAD
Our top priorities for 2016-2017 are 1) execution of our late-stage 
CVT-301 and PLUMIAZ clinical trials, and the subsequent preparation 
and filing of regulatory submissions; 2) planning for commercialization 
of these therapies; 3) conducting an unblinded analysis of the BID 
dalfampridine post-stroke walking difficulty trial and completion of 
ongoing QD formulation development for use in future planned studies; 
4) completion of the Biotie acquisition, integration of Biotie employees 
and execution of ongoing clinical programs; and 5) defending our 
intellectual property rights for AMPYRA.

Over the past two years, with the acquisitions of Civitas and Biotie, 
we have evolved Acorda into a company that is poised to make major 
contributions to the treatment of people with neurological disorders 
and deliver significant shareholder value. Diversification has been a key 
pillar of our growth strategy. All biopharma companies face repeated 
challenges and risks in their clinical, regulatory and intellectual property 
endeavors. Success ultimately depends on having enough high-quality 
“shots on goal,” in addition to outstanding personnel and supportive 
shareholders who are willing to take the long view. On behalf of our 
Management Team, Board of Directors and our passionate team of 
more than 500 Acordans, thank you, our shareholders, for your support 
in helping us build a great enterprise at Acorda.

I look forward to updating you on our progress.

Ron Cohen, M.D.
President and CEO

LATE-STAGE PIPELINE: MULTIPLE NEAR-TERM MILESTONES
We anticipate milestones in the next 6-12 months for three of our 
late-stage programs:

  CVT-301: We expect to achieve the “last patient out” milestone in 
the pivotal Phase 3 clinical trial by the end of 2016. If successful, 
we plan to file a New Drug Application (NDA) in the first quarter of 
2017.

  PLUMIAZ™ (diazepam): We are advancing the clinical work 

necessary to refile an NDA for PLUMIAZ and anticipate submitting 
the NDA in Q1 2017.

  Dalfampridine for Post-Stroke Walking Difficulty (PSWD): 
•  At the end of Q1 2016, we announced the completion of our 
single-dose pharmacokinetic (PK) studies of three different 
once-daily (QD) formulations of dalfampridine. Results for at least 
one of these formulations met our criteria. The multi-dose phase 
of PK testing will begin in the second quarter of 2016. 

•  Given our progress in the development of a QD formulation, we 
made the decision to stop enrollment of the Phase 3 clinical trial 
and conduct an unblinded analysis of the data, having reached 
50% of its target enrollment in the study, or 270 subjects. Data 
are expected by the fourth quarter of 2016 and will be used to 
inform the design of planned Phase 3 trials.

EARLIER-STAGE PROGRAM UPDATES
  rHIgM22: We began a second Phase 1 clinical trial for 

remyelination in MS, following successful completion of a Phase 1 
trial in 2014. We presented the results of the first Phase 1 trial at 
the 2015 American Academy of Neurology (AAN) and European 
Committee for Treatment and Research in Multiple Sclerosis 
(ECTRIMS) annual meetings.

  CVT-427: This program uses the ARCUS technology to deliver 
doses of zolmitriptan from the lungs directly to the bloodstream. 
Results of our Phase 1 PK trial met our specifications, and we plan 
to continue clinical development of CVT-427.

  Cimaglermin alfa: The program for heart failure was put on clinical 
hold following a safety report of hepatotoxicity in our second Phase 1 
clinical trial. There was a similar report in the first cimaglermin trial. 
We are working with outside experts to better understand this liver 
effect and to assess next steps for this program. 

AMPYRA® (DALFAMPRIDINE) INTELLECTUAL PROPERTY
As expected, generics companies have challenged our AMPYRA 
patents. We have five Orange Book-listed patents on AMPYRA that 
extend through 2027. Our outstanding in-house Legal team, together 
with experienced outside counsel, are vigorously defending our 
intellectual property. We have settled with four of the ten entities that 
submitted Abbreviated New Drug Applications (ANDA) for AMPYRA, 
granting them the right to launch generic dalfampridine tablets 
on a designated date in 2027, or potentially earlier under certain 
circumstances. A patent infringement litigation hearing with the remaining 
ANDA filers is scheduled to begin in federal district court in September. 

ACORDA’S MISSION IS TO DEVELOP THERAPIES THAT 
RESTORE FUNCTION AND IMPROVE THE LIVES OF 
PEOPLE WITH NEUROLOGICAL DISORDERS.

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PIPELINE

Acorda has a leading pipeline of novel neurological therapies addressing 
a range of disorders, including Parkinson’s disease, epilepsy, post-stroke 
walking difficulty, migraine, and multiple sclerosis. 

Pending the completion of our acquisition of Biotie, we will add three 
additional clinical-stage programs — including Phase 3 and Phase 2 
compounds for the treatment of Parkinson’s disease. Combined with our 
CVT-301 program, we will be positioned to become a leader in Parkinson’s 
disease therapeutic development and commercialization.

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* pending completion of Biotie acquisition

ARCUS® TECHNOLOGY

We secured global rights to the proprietary ARCUS 
technology platform as part of our acquisition of Civitas 
Therapeutics in 2014. We are excited about the potential 
application of this technology to a broad range of therapies. 
Most importantly, the Civitas team has been a tremendous 
addition to our company, providing expertise on ARCUS 
and the clinical trial programs utilizing this technology. Many 
of these Acordans have been working with the ARCUS 
technology for over twenty years.

ARCUS is an investigational dry powder pulmonary delivery 
system that has potential applications in multiple disease 
areas. This platform is being studied to deliver significantly 
larger doses of medication than are possible with 
conventional dry powder formulations. Currently, we have 
two clinical-stage programs utilizing the ARCUS platform: 
CVT-301 and CVT-427. 

The ARCUS inhaler is breath-actuated, operated by the 
user putting their lips to the device and simply breathing in. 

Based on research and discoveries at the Massachusetts 
Institute of Technology (MIT) and Pennsylvania State 
University, the ARCUS technology has been used to 
successfully deliver more than one million doses to patients 
in clinical trials of various therapeutic agents. Our Chief 
Technology Officer, Dr. Rick Batycky, was one of the original 
members of the team that developed this technology. 

Construction of our laboratory and commercial 
manufacturing facility in Chelsea, MA was completed in 
2003. Many of our current colleagues contributed to the 
planning and design of the facility.

Investigational products CVT-301 and CVT-427 use the 
ARCUS technology as a delivery method for established, 
standard-of-care oral medications. We are also exploring 
additional development opportunities that may leverage the 
ARCUS technology. 

In 2015, the Bill & Melinda Gates Foundation awarded 
Acorda a $1.4 million grant to explore development 
of a formulation and delivery system for a dry powder 
version of lung surfactant, a drug used to treat 
respiratory distress syndrome (RDS) in newborns. 
The formulation, based on the ARCUS technology, is 
being developed in collaboration with MIT. RDS can 
be fatal, and the program objective is to increase 
access to appropriate care in developing countries.   

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BIOTIE ACQUISITION

2016 GUIDANCE

In January 2016, we announced an agreement to acquire 
Biotie Therapies for approximately $363 million in cash. 
This transaction, when completed, will add promising 
assets to our clinical pipeline and represents a significant 
opportunity to create shareholder value. The acquisition, 
which positions us to become a leader in developing 
novel therapies for Parkinson’s disease, includes:

  Worldwide rights to tozadenant, an oral adenosine 

A2a receptor antagonist currently in Phase 3 
development to reduce daily OFF time for people 
with Parkinson’s disease. If approved, adenosine A2a 
receptor antagonists would be the first new class of 
therapy for the treatment of Parkinson’s disease in 
the U.S. in more than 20 years. We are targeting an 
NDA filing for tozadenant by the end of 2018.

  Further expanding our Parkinson’s disease 

therapeutics pipeline, we will also obtain global 
rights to SYN120, an oral, 5-HT6/5-HT2A dual 
receptor antagonist for treatment of Parkinson’s-
related dementia, in Phase 2 development with 
support from the Michael J. Fox Foundation.

  In addition, we will receive rights to two other assets:  

1.  Selincro® (nalmefene), an opioid receptor 

antagonist marketed by H. Lundbeck A/S in the 
EU to reduce alcohol consumption in patients with 
alcohol dependence. We will receive a low double 
digit royalty on net sales.

2.  BTT1023, an early stage antibody candidate 

against VAP-1, a promising target for treatment of 
primary sclerosing cholangitis (PSC). An open-
label, proof-of-concept study is currently enrolling. 
As liver disease is not a core focus, we expect to 
explore partnership opportunities for this asset.

The Biotie team has done a remarkable job in 
developing these clinical programs. Following the 
close of the acquisition, we plan to maintain the Biotie 
operation in San Francisco and retain staff at that site. 
We are considering the long-term future of the Biotie 
office in Turku, Finland.

We believe tozadenant and SYN120 are highly 
complementary to CVT-301, our existing Phase 3 
Parkinson’s disease program. CVT-301 is being 
studied to restore motor function when OFF periods 
occur, whereas tozadenant is being developed to 
reduce overall OFF time during the day in combination 
with current oral treatment regimens. SYN120, for 
Parkinson’s-related dementia, is a novel compound that 
could benefit people with more advanced disease. 

If approved, tozadenant would offer significant 
synergies with our outstanding neurology commercial 
organization. We anticipate an incremental expansion 
of our existing commercial infrastructure and sales 
force if CVT-301 is approved; no further significant 
expansion would then be necessary to accommodate 
commercialization of tozadenant. We estimate U.S. 
peak annual net revenue of tozadenant of more than 
$400 million, and patent protection through 2025 with a 
potential patent term extension to 2030.

Combined with our other late-stage assets (CVT-301 and 
PLUMIAZ™ (diazepam)), we have the potential to launch 
three therapies in the U.S. by 2019 with projected U.S. 
peak sales of more than $1 billion.

We anticipate that this transaction will be completed in the 
second half of 2016. 

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2016 GUIDANCE

  AMPYRA has shown steady growth since launch in 2010; 
2016 guidance represents ~10% increase over 2015 sales.

  We are conducting registrational clinical trials for CVT-
301 and PLUMIAZ™ (diazepam) and plan to submit 
U.S. regulatory filings for both compounds in 2017. An 
unblinded analysis of data from the Phase 3 twice-daily 
(BID) dalfampridine post-stroke walking difficulty trial, 
along with results from our QD formulation work, will 
inform next steps for this program.

  We are advancing earlier-stage clinical programs  

CVT-427 for acute treatment of migraine and rHIgM22 
for remyelination in MS.

2015 HIGHLIGHTS

  Our infrastructure currently supports three commercial 
products, five clinical programs (including registration 
trials for CVT-301, PLUMIAZ and dalfampridine), and 
ARCUS® manufacturing capability in our Chelsea facility.  

* excludes share-based compensation

WE FOUNDED ACORDA TO BRING LIFE-CHANGING 
THERAPIES TO PEOPLE LIVING WITH A WIDE 
RANGE OF NERVOUS SYSTEM DISORDERS. 

OUR SUCCESS IS ROOTED IN THE CLOSE 
COLLABORATION OF A HIGHLY-SKILLED, 
DIVERSE AND MOTIVATED TEAM. 

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MANAGEMENT 
TEAM

Ron Cohen, M.D.

David Lawrence, M.B.A.

President & Chief Executive Officer

Chief of Business Operations

Richard P. Batycky, Ph.D.

Michael Rogers

Chief Technology Officer 

Chief Financial Officer

and Site Head

Andrew R. Blight, Ph.D.

Chief Scientific Officer

Denise Duca, Ed. M.

Executive Vice President, 

Human Resources

Andrew A. Hindman

Lauren Sabella

Chief Commercial Officer

Tierney Saccavino

Executive Vice President, 

Corporate Communications

Jane Wasman, J.D.

President, International & 

Chief Business Development Officer

General Counsel

BOARD OF 
DIRECTORS

Ron Cohen, M.D.

Founder

Barry Greene

Board Member since 2007

Peder K. Jensen, M.D.

John P. Kelley

Board Member since 2011

Board Member since 2008

Sandra Panem, Ph.D.

Lorin J. Randall 

Board Member since 1998

Board Member since 2006

Steven M. Rauscher 

Ian F. Smith

Board Member since 2005

Board Member since 2007

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Contact:

Jeff Macdonald
Senior Director, Corporate Communications

jmacdonald@acorda.com
914-326-5232