ARDSLEY, N.Y.--(BUSINESS WIRE)--Acorda Therapeutics, Inc. (Nasdaq:ACOR)
today announced that the MILESTONE clinical study did not show
sufficient efficacy to support further development of dalfampridine to
improve post-stroke walking difficulties (PSWD).
“We are disappointed by this outcome. The study indicated there was
activity related to walking in people with PSWD, as suggested by the
prior Phase 2 study, but overall this was not sufficiently clinically
meaningful. I want to express our gratitude to the study participants,
their care partners and clinicians, who gave their time and commitment
to this research,” said Ron Cohen, M.D., President and CEO of Acorda.
“This outcome underscores the risks that companies in the
biopharmaceutical industry must take in order to develop innovative
medicines. Over the past three years, we have successfully diversified
our pipeline portfolio to account for this risk. We plan to focus R&D
resources on developing our promising late-stage Parkinson’s disease
therapies, CVT-301 and tozadenant, as well as advancing our earlier
stage assets, CVT-427 in migraine, SYN120 in Parkinson’s disease
dementia, and rHIgM22 in MS.”
As part of the PSWD development program, a multi-dose pharmacokinetic
(PK) study confirmed the Company has developed a potentially viable
once-daily (QD) formulation of dalfampridine.
MILESTONE Efficacy and Safety Findings
The Company elected to stop enrollment and to conduct an unblinded
analysis of the MILESTONE trial after reaching enrollment of 377
participants. This analysis included 368 participants who received
either 10 mg or 7.5 mg of dalfampridine twice daily (BID) or placebo.
The primary outcome measure of the study was the proportion of
participants who showed at least a 20% improvement on the Two Minute
Walk Test (2MinWT) at Week 12 as compared to baseline. The 2MinWT
measures the distance a subject can walk in 2 minutes, and is a
validated scale used to assess walking capacity.
The study found that 23 of 121 (19.0%) participants receiving 10 mg of
dalfampridine BID and 17 of 121 (14.0%) participants receiving 7.5 mg of
dalfampridine BID showed at least a 20% improvement on the 2MinWT,
compared to 17 of 126 (13.5%) participants receiving placebo.
In this study, dalfampridine was well tolerated in the post-stroke
population. The safety profile overall was similar to that observed in
multiple sclerosis clinical trials and post-marketing surveillance. The
most common adverse events (= 5%) reported in this study were: falls (10
mg: 10.7%, 7.5 mg: 9.5%, placebo: 5.6%), urinary tract infections (10
mg: 9.0%, 7.5 mg: 6.3%, placebo: 2.4%), dizziness (10 mg: 3.3%, 7.5 mg:
7.9%, placebo: 2.4%) and fatigue (10mg: 2.5%, 7.5 mg: 3.2%, placebo:
6.3%). There were no seizures reported in the dalfampridine 10 mg group.
There were 2 seizures reported in the 7.5 mg group and 3 reported in the
Additional data from the MILESTONE study will be presented at a future
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,
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.
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 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 acquired with 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
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 press release 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 press release.