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Acorda Therapeutics

acor · NASDAQ Healthcare
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Industry Drug Manufacturers - Specialty & Generic
Employees 201-500
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FY2012 Annual Report · Acorda Therapeutics
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2 0 1 2   A N N U A L   R E P O R T

 
 
 
 
 
DEAR MEMBERS OF THE 
ACORDA COMMUNITY:

2012 marked a critical transition for Acorda, as we achieved several milestones that positioned the Company for 
long-term growth and leadership in neurology product development and commercialization. These included: 

v  $266.1 million in net sales of AMPYRA® (dalfampridine) Extended Release Tablets, 10 mg, a 26% increase over 2011; 
v  acquisition of a novel product, Diazepam Nasal Spray for cluster seizures, for which an NDA filing is expected in 2013;
v  conducting two proof-of-concept Phase 2 clinical trials (completed in April 2013) exploring the potential of dalfampridine  
extended release tablets in the treatment of post-stroke deficits and cerebral palsy; study found dalfampridine improved  
walking in people with post-stroke deficits;

v  completing the first Phase 1 clinical trial of GGF2 in heart failure with positive efficacy signals; and
v  successfully filing an IND for rHIgM22 for remyelination in multiple sclerosis (MS).

Based on these milestones, in 2013 we expect to have at least five product opportunities in various stages of clinical  
development and/or NDA review. 

CONTINUED GROWTH FOR AMPYRA 
More than 75,000 people with MS have tried AMPYRA in  
the U.S. since its launch in March 2010. This breakthrough 
therapy has helped tens of thousands of people to improve  
their walking; it represents a unique contribution by Acorda  
to the treatment of MS, one that is highly valued by people  
with this challenging disease.

AMPYRA has shown consistent year-over-year growth since 
approval. In 2012, we closed the year with $266.1 million in net 
sales, a 26% increase over 2011. Our 2013 net sales guidance 
of $285-315 million reflects our confidence that we can 
continue to build AMPYRA through a combination of organic 
growth and pricing adjustments. 

To support future AMPYRA sales growth, we will continue  
to develop or expand innovative commercial strategies, such  
as our highly successful First Step and patient outreach 
programs. We are also piloting initiatives that encourage people 
who are benefiting from therapy to continue to take AMPYRA  
as prescribed. At the same time, we continue to focus  
on the cost-effectiveness of these initiatives, and project  
that commercial AMPYRA spend will be essentially the  

AMPYRA GROWTH

$285-3152

$266.1

$210.5

$133.11

$M

350

300

250

200

150

100

50

0

2010

2011

2012

2013

1. Ten months, Mar – Dec 2010 
2. 2013 Guidance

same in 2013 as in 2012, and significantly lower as a 
percentage of sales.

We are also exploring the potential of dalfampridine extended 
release tablets to help people with other neurological conditions 
and other indications within MS. These programs are discussed 
in more detail in the pipeline section of this letter.

MARKET STATUS OF ZANAFLEX® FRANCHISE 
AND FAMPYRA® 
In 2005, we launched our first commercial product, ZANAFLEX 
CAPSULES® (tizanidine hydrochloride). At the time, we  
viewed this product as a strategic bridge to enable us to build 
a commercial infrastructure that would ultimately support the 
launch of AMPYRA®. This strategy not only succeeded in that 
purpose, but the Zanaflex franchise ultimately generated far 
more revenue than our original projections. Market exclusivity for 
the Zanaflex franchise ended in February 2012. However, despite 
generic competition, we have continued to generate modest 
incremental revenue through both branded sales and royalties 
that we receive from Actavis, Inc. on sales of an authorized 
generic version of ZANAFLEX CAPSULES. 

AMPYRA has been launched in several ex-U.S. markets  
by our partner, Biogen Idec, under the brand name FAMPYRA® 
(prolonged-release fampridine tablets), and Biogen is 
continuing to roll out additional launches. FAMPYRA has been 
enthusiastically received by physicians and people living with 
MS, with strong product demand – through the end of 2012, 
more than 45,000 people with MS had tried FAMPYRA. 

However, the global reimbursement environment has become 
progressively more restrictive, particularly in Europe. For now, 
we expect European reimbursement for FAMPYRA to remain 
challenging, but with a foundation of strong fundamental 
demand for FAMPYRA.

KEY ADDITION: DIAZEPAM NASAL SPRAY 
In December 2012, we finalized our acquisition of Neuronex, 
Inc., adding Diazepam Nasal Spray to our development pipeline. 
This proprietary formulation of diazepam is a potential  
acute treatment for people with epilepsy who experience cluster 
seizures, also known as acute repetitive seizures, which are not 
controlled by their other anti-epileptic medications. Outpatient 
treatment for cluster seizures is limited to diazepam administered 
as a rectal gel, which significantly limits acceptance by adult  
and older pediatric patients; a nasal formulation has the potential 
to introduce a more convenient mode of delivery that would 
mark an important advance in patient care. 

We are currently preparing a New Drug Application (NDA) for 
this product. Pending completion of certain manufacturing tests, 
we anticipate filing the NDA later in 2013. If the FDA review 
proceeds successfully, Diazepam Nasal Spray may be on the 
market as early as 2014. This product would leverage our 
existing commercial infrastructure, and we do not anticipate that 
we would need to expand our sales force significantly, if at all,  
to support it. If launched, this product should quickly be 
accretive to our bottom line.

In April 2013, three-month data 
from the Phase 1 GGF2 clinical 
trial in heart failure patients were 
highlighted at the American 
College of Cardiology meeting, 
the premier medical conference 
for cardiovascular physicians. 
These data were presented 
by our academic collaborator, 
Vanderbilt Medical Center.

2013 CLINICAL AND REGULATORY MILESTONES

v  Top-line data from extended release  

v  Initiation of Phase 2 clinical trial for AC105 in 

dalfampridine post-stroke deficits and cerebral 
palsy studies announced April 2013; study found 
dalfampridine improved walking in people with 
post-stroke deficits

acute SCI (2H 2013, pending results of additional 
preclinical data)

v  Initiation of Phase 1 clinical trial for rHIgM22 in  

MS (April 2013)

v  NDA filing for Diazepam Nasal Spray (2H 2013)

v  Initiation of second GGF2 clinical trial in heart 

failure (2H 2013)

ADVANCING PIPELINE  
As a result of our patient and methodical investments in product 
development, we believe the Acorda pipeline is now one of 
the most promising in the neurology space, encompassing five 
separate products at clinical or pre-NDA stage. 

Drug development is an expensive, high-risk enterprise.  
To mitigate risk, our approach has been to design  
blinded preclinical trials that provide reproducible results  
in validated preclinical models, where available, and stepwise 
clinical trials that provide clear “go/no go” endpoints.  
This approach allows us to make graduated, controllable 
increases in investment, based entirely on how much risk 
has been eliminated at a given point. As a result, we have 
abandoned numerous product opportunities since the 
Company’s founding in 1995, and advanced only those  
that have met our rigorous criteria.

Our approach is well-illustrated by the results that we 
announced in April 2013 from two studies exploring the use  
of extended release dalfampridine in new disease areas:  
post-stroke deficits and cerebral palsy (CP). 

Consistent with preclinical data, dalfampridine extended release 
tablets improved walking in people with post-stroke deficits, 
and we are therefore planning to continue clinical development 
in this area. There are more than seven million stroke survivors 
in the U.S., and more than half have mobility impairment. There 
are no medications currently available for these patients, so 
new therapies are desperately needed.

Efficacy data from the CP study suggested potential treatment 
activity on measures of walking and hand strength; however, we 
are still analyzing these data to determine if they are sufficiently 
robust to warrant further clinical studies.

Importantly, safety findings from both the post-stroke deficits 
and CP studies were consistent with previous AMPYRA® clinical 
trials and post-marketing experience in MS.

Our GGF2 program, likewise, exemplifies our approach to 
prudent R&D management. After years of meticulous preclinical 
studies that informed clinical trial design, in late 2012 we 
completed the first clinical trial of GGF2 in patients with heart 
failure. This trial was primarily a safety and tolerability study,  
but echocardiography revealed a long-lasting, dose-dependent 
improvement in cardiac ejection fractions that mirrored our 
preclinical data. These data were impressive enough to be 
awarded a platform presentation at the American College of 
Cardiology Meeting in April 2013. We have discussed these 
data with the FDA and have reached agreement on the next 

clinical study. This next study will primarily investigate further 
the safety profile of GGF2 across a range of doses, and will 
continue to explore efficacy outcomes. The FDA has granted 
Fast Track designation for GGF2 for the treatment of  
heart failure.

GGF2 has also shown promise in improving motor function 
in preclinical models of acute stroke, and in restoring erectile 
function in preclinical models of cavernous nerve injury, 
the same nerve that is routinely injured in men undergoing 
prostate surgeries. If GGF2 continues to show promise in our 
clinical program in heart failure, we plan also to develop it for 
neurological indications in the future.

We also initiated a new clinical program in April 2013, enrolling 
the first patient in a Phase 1 clinical trial for rHIgM22 to evaluate 
myelin repair in MS. rHIgM22 has shown the ability to repair 
myelin in several preclinical models of MS. 

Another clinical stage product, AC105, has shown promise in 
preclinical models of acute spinal cord injury (SCI), brain trauma 
and stroke. We are planning to initiate a Phase 2 clinical trial  
in AC105 later this year, pending results of additional  
preclinical data.

PROFITABILITY AND GROWTH 
We believe that the Company is now positioned advantageously 
to leverage its assets for future growth. These significant assets 
include: a seasoned team of executives with proven records 
of identifying and successfully developing opportunities that 
have not been obvious to others; a highly skilled, accomplished 
commercial organization in the specialty neurology space; and 
a disciplined approach to business and drug development that 
has yielded a promising and diversified clinical stage pipeline. 

Our priorities are to maximize our commercial opportunities 
with AMPYRA® and expand them where possible, to acquire 
new commercial or near-commercial stage opportunities 
that leverage our existing commercial organization, and to 
invest in the potential of our pipeline to fuel future growth. We 
also seek to achieve a reasonable balance between current 
profitability and future growth. Where we set this balance will be 
determined at any given time by the quality, stage and size of 
our pipeline opportunities.

On behalf of my associates at Acorda and our Board of 
Directors, I thank you for your continuing support of our  
mission to bring novel therapies to people with neurological 
diseases, and look forward to keeping you informed of our 
progress in 2013.

Ron Cohen

President and Chief Executive Officer

MANAGEMENT TEAM 

BOARD OF DIRECTORS

Ron Cohen, M.D.

Barry Greene

Peder K. Jensen, M.D.

John P. Kelley

Sandra Panem, Ph.D.

Lorin J. Randall

Steven M. Rauscher

Ian F. Smith

CONTACT

Jeff Macdonald 
Sr. Director, Corporate Communications 
jmacdonald@acorda.com 
914-326-5232

Ron Cohen, M.D. 
President and Chief Executive Officer 

Thomas C. Aquilina, M.D. 
Vice President, Drug Safety & Risk Management

Andrew R. Blight, Ph.D. 
Chief Scientific Officer 

Jennifer Burstein, M.B.A., CPA  
Vice President, Finance 

Anthony O. Caggiano, M.D., Ph.D. 
Vice President, Research & Development 

Enrique Carrazana, M.D. 
Chief Medical Officer 

Kerry Clem 
Vice President, Sales 

Denise Duca, Ed.M. 
Sr. Vice President, Human Resources 

Herbert R. Henney III, Pharm.D. 
Vice President, Medical Affairs 

Elizabeth Keating 
Vice President, Marketing 

Ruhi Khan, M.B.A. 
Vice President, Business Development 

David Lawrence, M.B.A. 
Chief Financial Officer 

Bonnie M. Pappacena 
Vice President, Quality 

Adrian L. Rabinowicz, M.D., FAAN 
Sr. Vice President, Medical Affairs 

Kent Rogers, M.B.A. 
Vice President, Managed Markets 

Lauren Sabella 
Executive Vice President, Commercial Development 

Tierney Saccavino 
Sr. Vice President, Corporate Communications 

Brian Walter, Ph.D. 
Vice President, Regulatory Affairs 

Jane Wasman, J.D. 
President, International and General Counsel

 
 
www.acorda.com