-- Novartis continues to build on its experience and expertise in MS to
advance care for people with SPMS
EAST HANOVER, NJ, Sept. 17, 2016 /PRNewswire/ - Novartis today announced
positive results of the Phase III EXPAND study showing that oral
once-daily BAF312 (siponimod) significantly reduced the risk of
confirmed disability progression compared with placebo in people with
secondary progressive multiple sclerosis (SPMS). SPMS is a form of MS
characterized by continuous worsening of neurological function over
time, independent of relapses. Topline results of EXPAND were presented
at the 32nd Congress of the European Committee for Treatment and Research in
Multiple Sclerosis (ECTRIMS) in London, UK.
BAF312 is an investigational selective sphingosine-1-phosphate (S1P)
receptor modulator. Initial, first interpretable data from the EXPAND
Treatment with BAF312 reduced the risk of three-month confirmed
disability progression by 21% compared with placebo (p=0.013). The risk
reduction for six-month confirmed disability progression was greater,
further supporting robustness of the data.
A consistent reduction in the risk of three-month confirmed disability
progression across predefined subgroups.
A significant difference in favor of BAF312 compared to placebo over 12
and 24 months in annualized relapse rate, the percent change in brain
volume, and change from baseline in the volume of T2 lesions (brain
lesions identified by a T2-weighted magnetic resonance imaging scan).
The difference in change from baseline in the timed 25-foot walk test
(T25FW) was not significant.
BAF312 was generally safe and well tolerated, with a profile comparable
to other drugs in the same class.
"There are very few available treatment options to delay disease
progression in SPMS, and there is a high unmet need for effective
therapies with an acceptable safety profile for people with the
condition," said Vasant Narasimhan, Global Head of Drug Development and
Chief Medical Officer for Novartis. "Novartis is the global leader in
understanding the role of S1P receptor modulation in the treatment of
MS, and the positive results of the EXPAND study are a continuation of
our ongoing efforts to innovate and meet the needs of patients. These
data are a positive stride forward in an unserved disease area, and we
look forward to evaluating next steps with health authorities."
EXPAND is the largest randomized, controlled study in SPMS to date.
Patients enrolled in EXPAND were representative of a general SPMS
population. They must have been diagnosed with SPMS and also
demonstrated progression of disability in the two years prior to study.
The majority of patients had non-relapsing SPMS. The mean age at study
entry was 48 years, and patients had a median Expanded Disability
Status Scale (EDSS) score of 6.0, which corresponds to the use of a
Novartis will complete full analyses of the EXPAND data and evaluate
next steps in consultation with health authorities. The full study
results, including data from primary and secondary endpoints, will be
submitted for publication.
About the EXPAND Study
The EXPAND study is a randomized, double-blind, placebo-controlled Phase
III study, comparing the efficacy and safety of BAF312 versus placebo
in people with secondary progressive MS (SPMS). It is the largest
randomized, controlled study in SPMS to date, and included 1,651 people
with SPMS from 31 countries. At the time of the study, individuals
enrolled in EXPAND had a mean age of 48 years and had been living with
MS for approximately 17 years. Patients had received a diagnosis of
SPMS, and also demonstrated progression of disability in the two years
prior to study. They also had an Expanded Disability Status Scale
(EDSS) score between 3.0 and 6.5 inclusive, with a median score of 6.0,
which corresponds to the use of a walking aid. Patients were randomized
to receive either 2mg BAF312 or placebo in a 2:1 ratio, respectively.
The primary endpoint of the study was the time to three-month confirmed
disability progression, as measured by the EDSS, versus placebo.
Secondary endpoints included delay in the time to six-month confirmed
disability progression based on EDSS versus placebo, the time to
confirmed worsening of at least 20% from baseline in the timed 25-foot
walk test (T25FW), T2 lesion volume, annualized relapse rate (ARR), and
the safety and tolerability of BAF312 in people with SPMS.
About BAF312 (siponimod)
BAF312 (siponimod) is an investigational selective modulator of specific
types of the sphingosine-1-phosphate (S1P) receptor. The S1P receptor
is commonly found on the surface of specific cells residing in the
central nervous system (CNS), that are responsible for causing CNS
damage that drives loss of function in secondary progressive MS (SPMS).
In-vitro studies show that BAF312 enters the brain and by binding to
these specific receptors, may prevent the activation of these harmful
cells, helping to reduce loss of physical and cognitive function
associated with SPMS.
About Multiple Sclerosis
Multiple sclerosis (MS) is a chronic disorder of the central nervous
system (CNS) that disrupts the normal functioning of the brain, optic
nerves and spinal cord through inflammation and tissue loss. The
evolution of MS results in an increasing loss of both physical (e.g.,
walking) and cognitive (e.g., memory) function. There are three types
of MS: relapsing-remitting MS (RRMS), secondary progressive MS (SPMS)
and primary progressive MS (PPMS).
SPMS is characterized by gradual worsening of neurological function over
time. This leads to a progressive accumulation of disability,
independent of relapses, which can severely affect patients' abilities
to carry out everyday activities. It usually follows an initial phase
of RRMS, which accounts for approximately 85% of all MS diagnoses; a
quarter of people with RRMS will eventually go on to develop SPMS
within 10 years of their initial RRMS diagnosis, rising to more than
three-quarters after 30 years. There remains a high unmet need for
effective and safe treatments to help delay disability progression in
MS affects around 400,000 people in the US.
About Novartis in Multiple Sclerosis
The Novartis multiple sclerosis (MS) portfolio includes Gilenya
(fingolimod, an S1P modulator), which is indicated for relapsing forms
of MS and is also in development for pediatric MS. Extavia® (interferon beta-1b for subcutaneous injection) is approved in the US
for the treatment of relapsing forms of MS. In Europe, Extavia is
approved to treat people with relapsing-remitting MS, secondary
progressive MS (SPMS) with active disease and people who have had a
single clinical event suggestive of MS.
In addition to BAF312 (siponimod) in development in SPMS,
investigational compounds include ofatumumab (OMB157), a fully human
monoclonal antibody in development for relapsing MS. Ofatumumab targets
CD20, and is currently being investigated in two Phase III pivotal
In the US, the Sandoz Division of Novartis markets Glatopa® (glatiramer acetate injection) 20mg/mL, the first generic version of
Teva's Copaxone®* 20mg.
The foregoing release contains forward-looking statements that can be
identified by words such as "continues," "investigational,"
"continuation," "ongoing efforts," "stride forward," "look forward,"
"next steps," "will," "in development," "being investigated," or
similar terms, or by express or implied discussions regarding potential
marketing approvals for BAF312 and OMB157, potential new indications or
labeling for Gilenya or Extavia, or regarding potential future revenues
from BAF312, Gilenya, Extavia, OMB157 and Glatopa. You should not place
undue reliance on these statements. Such forward-looking statements are
based on the current beliefs and expectations of management regarding
future events, and are subject to significant known and unknown risks
and uncertainties. Should one or more of these risks or uncertainties
materialize, or should underlying assumptions prove incorrect, actual
results may vary materially from those set forth in the forward-looking
statements. There can be no guarantee that BAF312 or OMB157 will be
approved for sale in any market, or at any particular time. Neither can
there be any guarantee that Gilenya or Extavia will be submitted or
approved for any additional indications or labeling in any market, or
at any particular time. Nor can there be any guarantee that any of
BAF312, Gilenya, Extavia, OMB157 or Glatopa will be commercially
successful in the future. In particular, management's expectations
regarding such products and investigational compounds could be affected
by, among other things, the uncertainties inherent in research and
development, including unexpected clinical trial results and additional
analysis of existing clinical data; unexpected regulatory actions or
delays or government regulation generally; the company's ability to
obtain or maintain proprietary intellectual property protection;
general economic and industry conditions; global trends toward health
care cost containment, including ongoing pricing pressures; unexpected
safety, quality or manufacturing issues, and other risks and factors
referred to in Novartis AG's current Form 20-F on file with the US
Securities and Exchange Commission. Novartis is providing the
information in this press release as of this date and does not
undertake any obligation to update any forward-looking statements
contained in this press release as a result of new information, future
events or otherwise.
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*Copaxone® is a registered trademark of Teva Pharmaceutical Industries Ltd.