- CVT-301 Phase 3 SPAN-PD study met
primary endpoint of improvement in UPDRS III, in data presented at
2017 International Congress of Parkinson’s Disease and Movement
Disorders (MDS)
- Multiple secondary endpoints were
supportive of primary endpoint result
- Acorda plans to file New Drug
Application (NDA) in U.S. by end of Q2 2017
- Company to host investor webcast to
review data from CVT-301 clinical program on Monday, June 5 at 4:30
pm Eastern / 1:30 pm Pacific
Acorda Therapeutics, Inc. (Nasdaq:ACOR) presented data from its
Phase 3 SPAN-PD clinical trial of CVT-301 (levodopa inhalation
powder) that showed a statistically significant, clinically
meaningful improvement in motor function, as measured by the
Unified Parkinson’s Disease Rating Scale – Part III (UPDRS III) in
people with Parkinson’s experiencing OFF periods. Multiple
secondary endpoints, including achievement of an ON state with
maintenance through 60 minutes and Patient Global Impression of
Change (PGIC), were supportive of the primary endpoint result.
These findings are being presented at the International Congress of
Parkinson’s Disease and Movement Disorders (MDS), being held in
Vancouver, British Columbia from June 4-8, 2017.
Acorda is developing CVT-301 as a treatment for symptoms of OFF
periods in people with Parkinson’s taking a carbidopa / levodopa
regimen. OFF periods refer to the re-emergence of Parkinson’s
symptoms.
Key Efficacy and Safety Findings: Phase 3 SPAN-PD
Study
The poster “Inhaled levodopa (CVT-301, 84-mg dose) significantly
improves motor function during OFF periods in Parkinson’s disease
subjects: A Phase 3 Study (SPAN-PD)” (Poster #LBA34) highlighted
findings from a 12-week, placebo-controlled trial that enrolled 339
participants.
The study met its primary endpoint, with CVT-301 84 mg showing
statistically significant improvement in motor function compared to
placebo as measured by mean change in the UPDRS III at 30-minutes
post-dose at Week 12 (-9.83 vs. -5.91; p = 0.009).
Secondary efficacy analyses were performed using a pre-specified
hierarchy. The order of hierarchy was set based on probability of
success, guided by the Phase 2b results. The primary endpoint
(CVT-301 84 mg vs. placebo) was tested first for statistical
significance. Upon achieving significance, the secondary endpoints
were tested for CVT-301 84 mg vs. placebo followed by CVT-301 60 mg
in the hierarchical order, as long as each preceding endpoint
reached a significance level of P < 0.05. The hierarchical
sequence did not reach statistical significance at Step 3.
Unadjusted (nominal) p-values are presented below for all key
secondary endpoints.
Endpoint Hierarchy Order
∆ P-Value 84 mg
vs. placebo
Change in UPDRS III at 30 min (primary)
1 -3.92
0.009** % OFF to ON and remaining ON at 60 min
2 21.60 0.003**
Change in UPDRS III at 20 min 3
-2.55 0.062 % subjects improved on PGIC
4 25.00
<0.001* Change in UPDRS III at 10 min
5 -2.26 0.046*
Change in PD diary OFF time 6
0.01 0.975 60 mg vs. placebo
Change in UPDRS III at 30 min 7
-3.07 0.039* % OFF to ON
and remaining ON at 60 min 8
19.50 0.006* Change in UPDRS III at 20
min 9 -1.98
0.147 % subjects improved on PGIC 10
15.20 0.026* Change in
UPDRS III at 10 min 11
-0.97 0.387 Change in PD diary OFF time
12 -0.10
0.722
** Statistically significant on a nominal
and adjusted basis
* Statistically significant on a nominal
basis only
The most commonly reported adverse events in the CVT-301 84 mg
group compared to the placebo group were: cough (14.9% vs. 1.8%,
reported mostly once/subject), upper respiratory tract infection
(6.1% vs. 2.7%), nausea (5.3% vs. 2.7%), sputum discoloration (5.3%
vs. 0%) and dyskinesia (3.5% vs. 0.0%). When cough was reported, it
was typically characterized as mild. Two of 114 participants
receiving CVT-301 84 mg discontinued the study due to cough.
Key Safety and Exploratory Efficacy Findings: Long-Term
Safety Study
In addition to the data presented at the MDS congress, Acorda
announced interim data from an ongoing long-term safety study
(CVT-301-005). This is a 52-week open-label study comparing
treatment with CVT-301 84 mg with an observational control group.
Participants were randomized in a 2:1 ratio into either the CVT-301
84 mg arm (n=271) or the observational control arm (n=127) of the
study.
The primary objective of this study is to assess pulmonary
function. Measures include Forced Expiratory Volume in 1 second
(FEV1) and diffusing capacity of the lung for carbon monoxide
(DLco).
There were no statistical differences in the mean changes in
FEV1 and DLco from baseline to Week 52 between the CVT-301 84
mg and observational control groups.
The most common adverse events that were reported in any study
arm at >5% were:
Adverse Event
Observational Control
CVT-301 84 mg
n (%)
(n=127)
(n=271)
Cough 1 (0.8%) 35 (12.9%)
Nasopharyngitis 6 (4.7%)
17 (6.3%) Dyskinesia 4 (3.1%)
15 (5.5%) Fall 3 (2.4%)
14 (5.2%) Bone Fracture (various types)
3 (2.4%) 14 (5.2%)
All reported fractures were judged by the investigators to be
unlikely related or not related to study drug. Most reports of
cough were mild (91%), none was severe. Three of 271 participants
(1.1%) receiving CVT-301 discontinued the study due to cough.
Participants reporting serious adverse events (SAEs) were as
follows: 13 (10.2%) in the observational control arm and 40 (14.8%)
in the CVT-301 84 mg arm. Urinary tract infection occurred in four
participants (1.5%) receiving CVT-301 84 mg. No other SAEs in the
CVT-301 treatment group were reported at greater than 1%. There was
one death in the study, a drowning in the CVT-301 84 mg group,
judged by the investigator to be not related to study drug.
The study also included several uncontrolled, exploratory
efficacy measurements that were only conducted in the CVT-301 arm
of the study. Exploratory endpoints are hypothesis-generating.
Findings included:
- At Week 52, the least-square mean of
UPDRS III change from pre-dose at 30 minutes post-dose was -15.13
(n=130).
- 85% of participants (n=129) maintained
an ON state 60 minutes post-dose at Week 52.
- 73% of participants (n=165) reported
improvement as measured by PGIC at Week 52.
- The least-square mean reduction in
daily OFF time among participants who completed the Week 52 visit
(n=108) was 1.15 hours.
- Over the course of the study, the
average daily usage of CVT-301 was 2.3 doses per day.
Acorda plans to file an NDA for CVT-301 with the U.S. Food and
Drug Administration (FDA) by the end of the second quarter of 2017,
and a Marketing Authorization Application (MAA) with the European
Medicines Agency (EMA) by the end of the year.
Investor Webcast Information
The Company will host a webcast for investors to provide an
overview of the CVT-301 clinical program on June 5, 2017 at 4:30 pm
Eastern / 1:30 pm Pacific. This will include data being presented
at the MDS Congress, as well as additional interim findings from a
long-term safety study.
The webcast will include presentations by Matthew Stern, M.D.,
University of Pennsylvania, Peter LeWitt, M.D., Wayne State
University School of Medicine, and Donald Grosset, M.D., Institute
of Neurological Sciences, Queen Elizabeth University Hospital
(Glasgow).
The webcast will be available on the Investor Events page
of www.acorda.com and will be archived in the same location
for replay. To participate via conference call, please dial
800-806-5484 (U.S.) or 416-340-2217 (international) and reference
the access code 8170198#. An audio replay will be available until
June 12, 2017 at 1:00 pm Eastern at 800-408-3053 (U.S.) or
905-694-9451 (international) using access code 5949682#.
About Parkinson’s disease and OFF Periods
Approximately one million people in the U.S. and 1.2 million
Europeans are diagnosed with Parkinson’s disease (PD); OFF periods
are experienced by approximately 350,000 in the U.S. and 420,000
in Europe.
Parkinson’s is a progressive neurodegenerative disorder
resulting from the gradual loss of certain neurons responsible for
producing dopamine. It causes a range of symptoms including
impaired movement, muscle stiffness and tremors. As PD progresses,
people with Parkinson’s experience OFF periods, which are
characterized by the re-emergence of PD symptoms. This re-emergence
can occur even when an individual’s treatment regimen has been
optimized.
OFF periods can be very disruptive to the lives of people with
Parkinson’s, their families and caregivers. OFF periods can
increase in frequency and severity during the course of the
disease.
About CVT-301 (levodopa inhalation powder) and
ARCUS®
CVT-301 is a self-administered, inhaled levodopa (L-dopa)
therapy in development for the treatment of symptoms of OFF periods
in people with Parkinson’s disease taking a carbidopa / levodopa
regimen.
CVT-301 utilizes Acorda’s investigational ARCUS® platform
for inhaled therapeutics. CVT-301 was designed to deliver a precise
dose of a dry powder formulation of L-dopa to the lung. Oral
medication can be associated with variable onset of action, as the
medicine is absorbed through the gastrointestinal (digestive) tract
before reaching the brain. Inhaled treatments enter the body
through the lungs and reach the brain, bypassing the digestive
system.
About Acorda Therapeutics
Founded in 1995, Acorda Therapeutics is a
biopharmaceutical 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 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 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., which
will likely be materially adversely affected by the recently
announced court decision in our litigation against filers of
Abbreviated New Drug Applications (each, an “ANDA”) to market
generic versions of 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 INBRIJA (CVT-301, levodopa inhalation powder),
or any other acquired or in-licensed programs; we may not be able
to complete development of, obtain regulatory approval for, or
successfully market INBRIJA, 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
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 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.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170605006069/en/
Acorda Therapeutics, Inc.Felicia Vonella,
914-326-5146fvonella@acorda.com
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