Genentech, Inc. (NYSE: DNA) and Biogen Idec, Inc. (Nasdaq: BIIB) announced that a Phase II/III study of Rituxan® (rituximab) for primary-progressive multiple sclerosis (PPMS) did not meet its primary endpoint as measured by the time to confirmed disease progression during the 96-week treatment period. Genentech and Biogen Idec will continue to analyze the study results and will submit the data for presentation at an upcoming medical meeting.
"We are disappointed in
the outcome of the primary endpoint, but not surprised given the significant
clinical challenges presented by PPMS," said Hal Barron, M.D., Genentech senior
vice president, development and chief medical officer. "There was some evidence
of biologic activity, and we will continue to review all the data to better
understand the role of B cells in MS."
"While the primary results are not
what we had hoped, we continue to believe in the potential of B cell therapy for
patients living with MS," said Michael Panzara, M.D., MPH, Vice President and
Chief Medical Officer, Neurology Strategic Business Unit, Biogen Idec. "PPMS is
widely considered a difficult form of MS to treat and historically no therapy
has proven efficacy in this disease state."
This Phase II/III randomized, double-blind, placebo-controlled,
multi-center study was designed to evaluate the efficacy, safety and
tolerability of four courses of Rituxan in
patients with PPMS. A total of 439 patients from approximately 60 sites in the
U.S. and Canada were randomized 2:1 to receive either four treatment courses of
Rituxan six months apart or placebo. MRI evaluations were conducted at baseline,
weeks 6, 48, 96 and 122.
Detailed safety data from the study is currently
being evaluated. The incidence of overall adverse events was comparable between
Rituxan and placebo treatment groups. Serious adverse events were 16.4 percent
in the Rituxan arm versus 13.6 percent in the placebo arm, with an incidence of
serious infections of 4.5 percent compared with <1.0 percent respectively.
Infectious events reported in at least 10 percent of patients in either group
included upper respiratory and urinary tract infections. Most infectious events
in the Rituxan arm were reported as mild to moderate in severity, though events
of greater severity were reported more frequently in patients receiving Rituxan.
There were more infusion-related reactions with Rituxan, the majority of which
were mild to moderate in severity. The companies continue to monitor the
long-term safety of Rituxan treatment.
About MS and PPMS
is a chronic autoimmune disease where the body's immune system attacks the
myelin sheath causing inflammation and destruction of this fatty, protective
substance. The myelin sheath surrounds the body's nerve fibers in the central
nervous system, which includes the brain, optic nerves and spinal cord. There
are four generally accepted disease courses of MS with a wide variety of
symptoms and variations of disease progression.
Symptoms of the disease
vary from patient-to-patient. Neurological disability typically accumulates over
time and may include weakness or fatigue; numbness or tingling; blurred vision,
impaired color perception or visual loss; poor coordination of muscle movements;
difficulty with bladder or bowel control; muscle stiffness (spasticity); speech
problems and challenges with cognitive skills.
PPMS affects approximately
10 percent of the MS population and is evident by the slow and continuous
progression of the disease. People with PPMS can experience plateaus in the
progression of their disability, but generally do not experience distinct
periods of relapse or remissions. The progressive nature of PPMS and severe
debilitation associated with the disease can have a devastating impact on a
patient's quality of life and ability to function.
Rituxan, discovered by Biogen Idec, is a therapeutic antibody
that first received Food and Drug Administration (FDA) approval in November 1997
for the treatment of relapsed or refractory, low-grade or follicular,
CD20-positive, B cell non-Hodgkin's lymphoma (NHL). It was also approved in the
European Union under the trade name MabThera® in June 1998. In February 2006,
Rituxan also received FDA approval in combination with methotrexate to reduce
signs and symptoms and, in January 2008, to slow the progression of structural
damage in adult patients with moderately-to-severely active RA who have had an
inadequate response to one or more TNF-antagonist therapies. Rituxan is the
first treatment for RA that selectively targets immune cells known as
CD20-positive B cells. Rituxan does not target the entire immune
CD20 is not found on stem cells, pro-B cells (B cell precursors),
normal plasma cells, or other normal tissues. Rituxan does not target plasma
cells. These cells make antibodies that help fight infections.
does not target stem cells in the bone marrow, and B cells can usually
regenerate and gradually return to normal levels after treatment with Rituxan in
about 12 months for most patients.
In addition, Rituxan received FDA
approval in February 2006 for first-line treatment of previously-untreated
patients with follicular NHL in combination with CVP (cyclophosphamide,
vincristine and prednisolone) chemotherapy and in September 2006, also was
approved for the treatment of non-progressing low-grade, CD20-positive, B cell
NHL as a single agent, in patients with stable disease or who achieve a partial
or complete response following first-line treatment with CVP chemotherapy, and
for previously untreated diffuse large B cell, CD20-positive, NHL in combination
with CHOP or other anthracycline-based chemotherapy regimens.
past ten years, there have been more than 1 million patient exposures to
Important Safety Information
Rituxan has been
associated with fatal infusion reactions, tumor lysis syndrome (TLS), severe
mucocutaneous reactions and progressive multifocal leukoencephalopathy (PML).
Hepatitis B reactivation and cardiac arrhythmias and angina have also been
observed. Patients should be closely observed for signs of infection if biologic
agents and/or disease modifying anti-rheumatic drugs other than methotrexate are
used concomitantly. Common adverse reactions (≥ 5%): hypertension, nausea, upper
respiratory tract infection, arthralgia, pruritus, and pyrexia.
addition to PPMS, for which there is currently no FDA-approved therapy, Rituxan
is also being studied in other autoimmune diseases for significant unmet medical
needs, including systemic lupus erythematosus, lupus nephritis and
antineutrophil cytoplasmic antibodies (ANCA)-associated
Genentech and Biogen Idec co-market Rituxan in the United
States, and Roche markets MabThera in the rest of the world, except Japan, where
Rituxan is co-marketed by Chugai and Zenyaku Kogyo Co. Ltd.
Founded more than 30 years ago, Genentech is a leading
biotechnology company that discovers, develops, manufactures and commercializes
biotherapeutics for significant unmet medical needs. A considerable number of
the currently approved biotechnology products originated from or are based on
Genentech science. Genentech manufactures and commercializes multiple
biotechnology products and licenses several additional products to other
companies. The company has headquarters in South San Francisco, California and
is listed on the New York Stock Exchange under the symbol DNA. For additional
information about the company, please visit http://www.gene.com.
Biogen Idec creates new standards of care in therapeutic areas
with high unmet medical needs. Founded in 1978, Biogen Idec is a global leader
in the discovery, development, manufacturing and commercialization of innovative
therapies. Patients in more than 90 countries benefit from Biogen Idec's
significant products that address diseases such as lymphoma, multiple sclerosis
and rheumatoid arthritis. For product labeling, press releases and additional
information about the company, please visit http://www.biogenidec.com.
release contains a forward-looking statement regarding the potential of B cell
therapy for patients living with MS. Such statement is a prediction and involves
risks and uncertainties such that actual results may differ materially. Actual
results may be affected by a number of factors including, but not limited to,
unexpected safety, efficacy or manufacturing issues, the need for additional
data or clinical studies, FDA actions or delays, failure to obtain or maintain
FDA approval, competition, pricing, reimbursement, the ability to supply
product, product withdrawals and new product approvals and launches, and
intellectual property or contract rights. Please also refer to the risk factors
described in Genentech and Biogen Idec's periodic reports filed with the
Securities and Exchange Commission. Genentech and Biogen Idec disclaim, and do
not undertake, any obligation to update or revise any forward-looking statements
in this press release.
View drug information on Rituxan