Acorda Announces Results from Phase 2b Clinical Trial of CVT-301 for Treatment of OFF Periods in Parkinson’s Disease Published in Movement Disorders

ARDSLEY, N.Y.–(BUSINESS WIRE)– Data from the Phase 2b clinical trial of CVT-301, published
this week in the peer-reviewed journal Movement Disorders,
showed that people with Parkinson’s disease (PD) experiencing OFF
periods and who were treated with CVT-301 had significantly greater
improvements in motor function than those treated with placebo. A
treatment effect was evident at 10 minutes after dosing and was
sustained for at least one hour, the longest time point at which
patients were assessed.

Acorda Therapeutics, Inc. (NASDAQ:ACOR) is developing CVT-301 for the
treatment of OFF periods in people with PD.

“Oral levodopa is a cornerstone of Parkinson’s disease treatment, but
after chronic therapy, approximately half of people with PD who are
treated with oral L-dopa experience OFF periods,” said Peter LeWitt,
M.D., M.Med.Sc., Director of the PD and Movement Disorders Program at
Henry Ford Hospital and lead author of the study. “OFF periods tend to
increase in frequency, severity and duration during the course of the
disease, and are among the most challenging experiences for people with
this disorder. Based on the data from this Phase 2b clinical study,
CVT-301 has the potential to restore motor function to people with
Parkinson’s disease when they experience OFF periods, and this is now
being studied in a Phase 3 clinical trial.”

OFF periods are characterized by a re-emergence of PD symptoms,
including motor symptoms such as the impaired ability to move, muscle
stiffness and tremor. This re-emergence can occur even when treatment
regimens of oral levodopa (L-dopa) and other standard of care
medications have been optimized.

The Phase 2b trial was a randomized, double blind, placebo-controlled,
multicenter study in 86 people with PD for the treatment of OFF periods.
Participants were randomized to self-administer CVT-301 or placebo to
the lung via an inhaler, as an adjunct to their established oral PD
medications. Participants received 35mg of CVT-301 or placebo in study
weeks 1 and 2, and 50mg of drug or placebo in weeks 3 and 4.

The primary endpoint was defined as the mean change from baseline OFF
state in Unified Parkinson’s Disease Rating Scale motor function (UPDRS
Part III) score, (measured at 10-60 minutes post dose) after 4 weeks of
treatment. The UPDRS is an established assessment method to monitor PD
motor impairment that has been used extensively in clinical research.

In this study, participants receiving CVT-301 showed a statistically
significant and clinically important reduction in average UPDRS III
score compared to placebo (p<0.01) and across all measured time points
beginning at 10 and up to 60 minutes post-administration (p<0.05). There
were no concerning safety signals observed in either CVT-301 dose group,
with no increase relative to placebo in troublesome or non-troublesome
dyskinesias during ON periods. There were no serious adverse events
reported in the drug group, and the incidence of drug-related adverse
events was similar between treatment groups (23% drug group; 21% placebo
group). The most common adverse events were dizziness (7% drug group; 5%
placebo), cough (7% drug group; 2% placebo) and nausea (7% drug group;
0% placebo); there were no adverse events related to cardiovascular or
lung function. PD patients were able to self-administer treatment while
in an OFF state.

Earlier this week, data included in this paper were featured at the 68^th Annual
Meeting of the American Academy of Neurology (AAN) during the Movement
Disorders Invited Science Session. Data from the Phase 2b study were
also previously presented at the International Congress of Parkinson’s
Disease and Movement Disorders (MDS) annual meetings in 2014 and 2015.
The 2015 presentation was recognized in the meeting’s Blue Ribbon
Highlights Session.

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 as-needed
treatment of OFF periods in Parkinson’s disease (PD). 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 levodopa to the lung. Oral medication can be associated with slow
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 results of the Phase 2b trial, Acorda has initiated a Phase
3 clinical trial that is expected to enroll 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.

More details about the study, including enrollment criteria, can be
found at https://cvt301.acordatrials.com/en/patient/
or http://clinicaltrials.gov/ct2/show/NCT02240030?term=CVT-301&rank=2.

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

For more information, please visit 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 or at all;
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 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, Plumiaz (diazepam) Nasal Spray,
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, Plumiaz, any other products under development, or the
products that we would acquire if 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
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the goals or plans described in our forward-looking statements, and
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forward-looking statements as a result of developments occurring after
the date of this release.

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