Insmed Announces Promising Results From IPLEX Phase II Myotonic Muscular Dystrophy Clinical Study
03 Mai 2007 - 3:15PM
Business Wire
Insmed Inc. (Nasdaq:INSM) today announced positive results from a
Phase II investigator-sponsored study of the company�s drug,
IPLEXTM, in patients with myotonic muscular dystrophy (DM1).
Preliminary results of the clinical study, being conducted at the
University of Rochester School of Medicine and Dentistry, showed
that six months of treatment with doses of IPLEX up to 1 mg/kg/day
in six patients met the primary study endpoints of being safe and
well tolerated. In addition, IPLEX treatment was associated with
improvements in muscle mass, cholesterol and triglycerides. During
the six months of treatment, 5 out of 6 patients experienced an
improvement in lean muscle mass. Patients also reported improvement
in gastrointestinal function, endurance and cognitive function
during treatment with IPLEX. �We are very encouraged by the results
from this preliminary study of IPLEX in myotonic dystrophy. The
results not only indicate IPLEX was safe, well tolerated and had a
positive effect on muscle and lipid metabolism, they also suggest
IPLEX may have a positive effect on aspects of the disease that
affect patients� daily living,� said Dr. Geoffrey Allan, president
and CEO of Insmed. Study Description The primary objectives of this
ongoing open-label, Phase II dose escalation study are to examine
the safety and tolerability of once-daily, subcutaneous injection
of IPLEX in up to 15 patients with DM1 and to identify the maximum
tolerated dose.�The next cohort of patients will be treated for six
months with a dose of IPLEX which will be titrated up to 2
mg/kg/day. The study is funded by the National Institutes of Health
and the Muscular Dystrophy Association, with supply of IPLEX drug
provided by Insmed. A Phase II, placebo-controlled study to further
investigate the safety and efficacy of IPLEX in a larger number of
patients with DM1 is being designed based on the preliminary
results of this study. About Myotonic Muscular Dystrophy Myotonic
muscular dystrophy, also known as myotonic dystrophy, dystrophia
myotonica or Steinert's disease, is the most common type of adult
muscular dystrophy, affecting 1 in 8000 individuals (approximately
40,000 people in the United States). Myotonic dystrophy patients
develop progressive muscle wasting and weakness in the hands,
forearms, legs, neck and face, as well as cataracts and cardiac
arrhythmias, and eventually can become totally disabled, dying
usually from respiratory or cardiac failure. At present, there is
no treatment to reverse most of these symptoms. For more
information about myotonic muscular dystrophy, please visit
www.mda.org. About IPLEX� IPLEX was approved in the United States
in December 2005 for the treatment of children with growth failure
due to severe primary IGF-I deficiency (Primary IGFD). IPLEX
(rhIGF-I/rhIGFBP-3), a complex of recombinant human insulin-like
growth factor-I (rhIGF-I) and its predominant binding protein
IGFBP-3 (rhIGFBP-3). The drug is also being investigated for
various other indications with unmet medical needs, including
HIV-associated adipose redistribution syndrome, retinopathy of
prematurity and amyotrophic lateral sclerosis (otherwise known as
ALS or Lou Gehrig�s disease). About Insmed Insmed is a
biopharmaceutical company focused on the development and
commercialization of drugs for the treatment of metabolic diseases
with unmet medical needs. For more information, please visit
www.insmed.com. About the University of Rochester School of
Medicine and Dentistry The University of Rochester School of
Medicine and Dentistry is a leading medical university that
consistently defines health care worldwide by conducting advanced
biomedical research and providing leading life science education
and complex patient care. It has been designated by the National
Institutes of Health (NIH) as one of several "centers of
excellence" for muscular dystrophy research and is receiving up to
$1 million per year for five years in federal NIH funding and up to
$500,000 per year for three years from MDA, for a total of up to
$6.5 million, to identify potential muscular dystrophy therapies.
For more information, please visit www.urmc.rochester.edu. About
The Muscular Dystrophy Association The Muscular Dystrophy
Association (MDA) is a voluntary health agency -- a dedicated
partnership between scientists and concerned citizens aimed at
conquering neuromuscular diseases that affect more than a million
Americans. MDA combats neuromuscular diseases through programs of
worldwide research, comprehensive medical and community services,
and far-reaching professional and public health education. MDA is
the world's largest non-governmental sponsor of research seeking
the causes of and effective treatments for neuromuscular diseases,
sponsoring some 400 research projects annually. For more
information, please visit www.mda.org. Forward Looking Statements
Statements included within this press release, which are not
historical in nature, may constitute forward-looking statements for
purposes of the safe harbor provided by the Private Securities
Litigation Reform Act of 1995. Forward-looking statements in this
press release include, but are not limited to, statements regarding
planned clinical study design, our regulatory and business
strategies, plans and objectives of management and growth
opportunities for existing or proposed products. Such
forward-looking statements are subject to numerous risks and
uncertainties, including risks that product candidates may fail in
the clinic or may not be successfully marketed or manufactured, the
company may lack financial resources to complete development of
product candidates, the FDA may interpret the results of our
studies differently than we have, competing products may be more
successful, demand for new pharmaceutical products may decrease,
the biopharmaceutical industry may experience negative market
trends and other risks detailed from time to time in the company's
filings with the Securities and Exchange Commission. As a result of
these and other risks and uncertainties, actual results may differ
materially from those described in this press release. For further
information with respect to factors that could cause actual results
to differ from expectations, reference is made to reports filed by
the Company with the Securities and Exchange Commission under the
Securities Exchange Act of 1934, as amended. The forward-looking
statements made in this release are made only as of the date hereof
and Insmed disclaims any intention or responsibility for updating
predictions or financial guidance contained in this release.
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