Insmed Announces Promising Results From IPLEX Phase II Myotonic Muscular Dystrophy Clinical Study

    RICHMOND, VA., May 3 /CNW/ - Insmed Inc. (Nasdaq:INSM) today announced
positive results from a Phase II investigator-sponsored study of the company's
drug, IPLEX(TM), 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(TM)

    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.

For further information: Investor Relations International Haris Tajyar,
818-382-9702 htajyar@irintl.com