Data from Clinical and Preclinical Trials of CVT-301 for Treatment of OFF Periods in Parkinson’s Disease Published in Science Translational Medicine

ARDSLEY, N.Y.–(BUSINESS WIRE)– Results from Phase 1, Phase 2a and preclinical studies of CVT-301, an
inhaled form of levodopa, have been featured in the current edition of Science
Translational Medicine. Acorda
Therapeutics, Inc. (NASDAQ:ACOR) is developing CVT-301 for the
treatment of OFF periods in people with Parkinson’s disease (PD).

As PD progresses, people with Parkinson’s experience OFF periods, which
are characterized by the re-emergence of PD symptoms. These include
motor symptoms such as impaired movement, muscle stiffness, and tremor,
as well as non-motor symptoms. This re-emergence can occur even when an
individual’s treatment regimen has been optimized. OFF periods typically
increase in frequency during the course of the disease.

“OFF periods can be hugely disruptive to the lives of people with
Parkinson’s and their families, and are considered one of the greatest
unmet medical needs in the treatment of Parkinson’s,” said Ron Cohen,
M.D., President and CEO of Acorda Therapeutics. “CVT-301 is an inhaled
powder form of levodopa that is being studied in combination with
standard of care Parkinson’s disease regimens. Following two successful
Phase 2 clinical trials^{1 ,2}, our Phase 3 program
is assessing the extent to which CVT-301, used when people with
Parkinson’s begin to experience OFF periods, can restore motor function.
If approved, CVT-301 may provide a valuable new treatment option for
these individuals.”

Acorda’s Phase 3 clinical program comprises a Phase 3 safety and
efficacy study as well as general and special population safety studies.
The program is designed to confirm the efficacy and safety profile of
CVT-301 and support global regulatory marketing authorization
applications. The Company expects to announce results from its
randomized, placebo-controlled Phase 3 trial in Q1 2017.

About Parkinson’s Disease and OFF Periods

There are approximately one million people in the U.S. and 1.2 million
people in Europe diagnosed with Parkinson’s disease (PD). PD is a
progressive neurodegenerative disorder resulting from the gradual loss
of certain neurons responsible for producing dopamine, which causes
impairment of motor function including impaired movement, muscle
stiffness and tremors. Non-motor symptoms are also common; they include
anxiety, depression, sleep difficulties and gastrointestinal (GI)
disorders among others.

In the United States, approximately 350,000 people with Parkinson’s
experience OFF periods.

About CVT-301 / Phase 3 Program

CVT-301 is an investigational agent being developed as a
self-administered, inhaled levodopa (L-dopa) therapy for the treatment
of OFF periods in Parkinson’s disease. It is intended for use as an
adjunctive therapy to a patient’s individually optimized oral
L-dopa/carbidopa regimen.

CVT-301 utilizes Acorda’s ARCUS^® platform for inhaled
therapeutics, which delivers a precise dose of a dry powder formulation
of L-dopa to the lung. Oral medication can be associated with slow and
variable onset of action, as the medicine is absorbed through the
gastrointestinal (digestive) tract before reaching the brain. Inhaled
treatments, such as those that utilize our ARCUS technology, enter the
body through the lungs and reach the brain shortly thereafter, bypassing
the digestive system.

Based on the successful results of two Phase 2 trials, Acorda initiated
a Phase 3 clinical program that includes a safety and efficacy study as
well as general and special population safety studies. The efficacy
trial has enrolled approximately 345 participants across three arms:
50mg, 35mg, or placebo. These are the same doses used in the Phase 2b
study. The primary outcome measure is improvement on the Unified
Parkinson’s Disease Rating Scale Part 3 (UPDRS III) after administration
of CVT-301 in patients experiencing an OFF period (30 minutes post
dose). UPDRS III is an established scale to monitor PD motor impairment,
and is considered a standard in the field. The Company expects to
announce results from this study in Q1 2017.

Early CVT-301 clinical studies were funded in part by grants from The
Michael J. Fox Foundation for Parkinson’s Research.

About Acorda Therapeutics

Founded in 1995, Acorda
Therapeutics is a biotechnology company focused on developing
therapies that restore function and improve the lives of people with
neurological disorders.

Acorda has a pipeline of novel neurological therapies addressing a range
of disorders, including Parkinson’s disease, post-stroke walking
difficulties (PSWD), migraine, and multiple sclerosis. Acorda markets
three FDA-approved therapies, including AMPYRA^® (dalfampridine)
Extended Release Tablets, 10 mg.

For more information, please visit the Company’s website at: www.acorda.com.

Forward-Looking Statement

This press release includes forward-looking statements. All statements,
other than statements of historical facts, regarding management’s
expectations, beliefs, goals, plans or prospects should be considered
forward-looking. These statements are subject to risks and uncertainties
that could cause actual results to differ materially, including: the
ability to complete the Biotie transaction on a timely basis; the
ability to realize the benefits anticipated from the Biotie and Civitas
transactions, among other reasons because acquired development programs
are generally subject to all the risks inherent in the drug development
process and our knowledge of the risks specifically relevant to acquired
programs generally improves over time; the ability to successfully
integrate Biotie’s operations and Civitas’ operations, respectively,
into our operations; we may need to raise additional funds to finance
our expanded operations and may not be able to do so on acceptable
terms; our ability to successfully market and sell
Ampyra (dalfampridine) Extended Release Tablets, 10 mg in the U.S.;
third party payers (including governmental agencies) may not reimburse
for the use of Ampyra or our other products at acceptable rates or at
all and may impose restrictive prior authorization requirements that
limit or block prescriptions; the risk of unfavorable results from
future studies of Ampyra or from our other research and development
programs, including CVT-301 or any other acquired or in-licensed
programs; we may not be able to complete development of, obtain
regulatory approval for, or successfully market CVT-301, any other
products under development, or the products that we will acquire when we
complete the Biotie transaction; the occurrence of adverse safety events
with our products; delays in obtaining or failure to obtain and maintain
regulatory approval of or to successfully market Fampyra outside of the
U.S. and our dependence on our collaborator Biogen in connection
therewith; competition; failure to protect our intellectual property, to
defend against the intellectual property claims of others or to obtain
third party intellectual property licenses needed for the
commercialization of our products; and failure to comply with regulatory
requirements could result in adverse action by regulatory agencies.

These and other risks are described in greater detail in our filings
with the Securities and Exchange Commission. We may not actually achieve
the goals or plans described in our forward-looking statements, and
investors should not place undue reliance on these statements.
Forward-looking statements made in this presentation are made only as of
the date hereof, and we disclaim any intent or obligation to update any
forward-looking statements as a result of developments occurring after
the date of this presentation.

¹ Lipp MM et al. Preclinical and clinical assessment of
inhaled levodopa for OFF episodes in Parkinson’s disease. Sci.
Transl. Med. 8, 360ra136 (2016) [Online version].

² LeWitt PA et al. Randomized Trial of Inhaled Levodopa
(CVT-301) for Motor Fluctuations in Parkinson’s Disease. Movement
Disorders 2016; 31(9): 1356-1365

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