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Sunday, September 18, 2016
Novartis BAF312 reduced the risk of disability progression in pivotal phase III study in secondary progressive MS patients
·EXPAND study data presented at ECTRIMS
2016 show that treatment with BAF312 (siponimod) reduced the risk of
three-month confirmed disability progression by 21% vs. placeboin patients with secondary
progressive multiple sclerosis (SPMS)
·SPMS is a progressive and highly
disabling form of MS and there are no approved treatments currently available
in the US
·Novartis continues to build on its
experience and expertise in MS to advance care for people with SPMS
Hanover, N. J., September 17, 2016– 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.
is an investigational selective sphingosine-1-phosphate (S1P) receptor
modulator. Initial, first interpretable data from the EXPAND study show:
·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.
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 walking aid.
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
the EXPAND Study
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.
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
(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).
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 SPMS.
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 studies.
In the US, the Sandoz Division of Novartis markets
Glatopa® (glatiramer acetate injection) 20mg/mL, the first generic
version of Teva's Copaxone®* 20mg.
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.
Novartis Pharmaceuticals Corporation offers a broad range
of medicines for cancer, cardiovascular disease, endocrine disease,
inflammatory disease, infectious disease, neurological disease, organ
transplantation, psychiatric disease, respiratory disease and skin
in East Hanover, NJ Novartis Pharmaceuticals Corporation is an affiliate of
Novartis AG, which provides innovative
healthcare solutions that address the evolving needs of patients and societies.
Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to
best meet these needs: innovative medicines, eye care and cost-saving generic
pharmaceuticals. Novartis is the only global company with leading positions in
these areas. In 2015, the Group achieved net sales of USD 49.4 billion, while
R&D throughout the Group amounted to approximately USD 8.9 billion (USD 8.7
billion excluding impairment and amortization charges). Novartis Group
companies employ approximately 118,000 full-time-equivalent associates. Novartis
products are available in more than 180 countries around the world. For more
information, please visit http://www.novartis.com.