CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme,
a Sanofi company (EURONEXT: SAN and NYSE: SNY) announced today
that key data from the TOWER trial were presented at the 28th
Congress of the European Committee for Treatment and Research in
Multiple Sclerosis (ECTRIMS). In the study, once-daily, oral AUBAGIO®
14 mg significantly reduced the annualized relapse rate and slowed
progression of disability in patients with relapsing forms of multiple
sclerosis (MS) compared to placebo. In addition, the proportion of
patients treated with AUBAGIO who were relapse-free was significantly
higher compared to placebo.
“AUBAGIO is the first and only oral MS therapy to significantly slow
the progression of disability in two Phase III trials”
TOWER (Teriflunomide Oral
in people With relapsing multiplE
scleRosis) is a randomized, double-blind
Phase III trial that enrolled 1,169 patients with relapsing MS across 26
countries and compared 7 mg or 14 mg once-daily, oral AUBAGIO against
placebo. The company announced positive top-line results in June. In
September, the FDA approved AUBAGIO as a once-daily oral treatment for
patients with relapsing forms of MS. Marketing applications for AUBAGIO
are currently under review by the EMA and other regulatory authorities.
“Slowing the progression of disability is a major goal in treating MS
and remains a significant unmet need for many patients,” said Ludwig
Kappos, M.D., Chair of Neurology, University Hospital Basel,
Switzerland, who presented the key TOWER results. “The TOWER study
results are consistent with the Phase lll TEMSO data, both in terms of
the effect on progression of disability and the manageable safety
profile of AUBAGIO.”
TOWER data presented for the first time today for the 14 mg dose include:
A 36.3 percent reduction in annualized relapse rate (ARR=0.319), the
primary endpoint of the trial, as compared to placebo (ARR=0.501)
(p=0.0001); Fifty-two percent of patients treated with this dose were
also relapse-free, meaning they did not experience any relapses during
the study, compared to 38 percent with placebo (37 percent risk
A 31.5 percent reduction in the risk of 12-week sustained accumulation
of disability, the main secondary endpoint, as measured by the
Expanded Disability Status Scale (EDSS), compared to placebo
In addition, a 22.3 percent reduction in annualized relapse rate
(ARR=0.389) was observed in patients treated with AUBAGIO 7 mg compared
to placebo (p=0.189); Further, 55 percent of patients treated with 7 mg
AUBAGIO were relapse-free, as compared with 38 percent on placebo
(p=0.0016). There was no statistically significant difference observed
between AUBAGIO 7 mg and placebo for the risk of 12-week sustained
accumulation of disability.
“AUBAGIO is the first and only oral MS therapy to significantly slow
the progression of disability in two Phase III trials,” said David
Meeker, M.D., President and CEO, Genzyme. “The convenience of a once
daily oral therapy offers a meaningful alternative for patients wanting
to avoid the burden of regular injections.”
Patients who completed the trial were followed for a period between 48
and 173 weeks. The average duration of AUBAGIO exposure in TOWER was 18
Adverse events observed in the trial were consistent with those seen in
previous studies of AUBAGIO in MS. The proportion of patients with
treatment-emergent adverse events was similar across all treatment arms.
The most common adverse events reported more frequently in the AUBAGIO
arms were headache, ALT (Alanine aminotransferase) elevations, hair
thinning, diarrhea, nausea and neutropenia. As previously reported,
there was one death from a respiratory infection in the placebo arm and
three deaths in the teriflunomide arms from a motor vehicle accident,
suicide and sepsis.
The FDA has approved AUBAGIO as a once-daily, oral immunomodulator
indicated for patients with relapsing forms of multiple sclerosis (MS).
The ongoing AUBAGIO clinical development program, involving more than
5,000 patients in 36 countries, is among the largest of any MS therapy.
Some patients in extension trials have been treated up to 10 years.
AUBAGIO is an immunomodulator with anti-inflammatory properties.
Although the exact mechanism of action for AUBAGIO is not fully
understood, it may involve a reduction in the number of activated
lymphocytes in the central nervous system (CNS).
The U.S. AUBAGIO label includes a boxed warning citing the risk of
hepatotoxicity and, teratogenicity (based on animal data). In MS
clinical studies included in the U.S. label, the incidence of serious
adverse events were similar among AUBAGIO and placebo-treated patients.
The most common adverse events associated with AUBAGIO in MS patients
included increased ALT levels, alopecia, diarrhea, influenza, nausea and
The labeling for AUBAGIO was also informed by the estimated 2.1 million
years of patient exposure globally since the launch of leflunomide,
which is indicated in the U.S. for the treatment of rheumatoid
arthritis. AUBAGIO is the principal active metabolite of leflunomide.
Severe liver injury including fatal liver failure has been reported in
patients treated with leflunomide.
About the TOWER Trial
TOWER is a Phase III, multi-center double-blind parallel-group
placebo-controlled study of the efficacy and safety of AUBAGIO in
patients with relapsing MS followed by an open-label extension period.
The TOWER study included patients ages 18 to 55. The primary endpoint
was the annualized relapse rate, defined as the number of confirmed
relapses per patient-year. The key secondary endpoint was time to
disability progression confirmed for a minimum of 12-weeks. Safety
variables were defined as adverse events reported by the patients or
noted by the investigator during the study period.
About Genzyme, a Sanofi Company
Genzyme has pioneered the development and delivery of transformative
therapies for patients affected by rare and debilitating diseases for
over 30 years. We accomplish our goals through world-class research and
with the compassion and commitment of our employees. With a focus on
rare diseases and multiple sclerosis, we are dedicated to making a
positive impact on the lives of the patients and families we serve. That
goal guides and inspires us every day. Genzyme’s portfolio of
transformative therapies, which are marketed in countries around the
world, represents groundbreaking and life-saving advances in medicine.
As a Sanofi company, Genzyme benefits from the reach and resources of
one of the world’s largest pharmaceutical companies, with a shared
commitment to improving the lives of patients. Learn more at www.genzyme.com.
Sanofi, a global and diversified healthcare leader, discovers, develops
and distributes therapeutic solutions focused on patients’ needs. Sanofi
has core strengths in the field of healthcare with seven growth
platforms: diabetes solutions, human vaccines, innovative drugs,
consumer healthcare, emerging markets, animal health and the new
Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York
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