Insmed Provides Interim Update from Two-Year, Open-Label Extension Study of ARIKACE to Treat Pseudomonas aeruginosa in Cystic

  Insmed Provides Interim Update from Two-Year, Open-Label Extension Study of
  ARIKACE to Treat Pseudomonas aeruginosa in Cystic Fibrosis Patients

 Initial 12-Month Data Demonstrate Long-Term Safety and Durability of Effect

Business Wire

MONMOUTH JUNCTION, N.J. -- February 19, 2014

Insmed Incorporated (Nasdaq GS:INSM), a biopharmaceutical company focused on
developing an inhaled anti-infective to treat patients battling serious lung
diseases in orphan indications that are often life-threatening, provides an
interim update from the Company’s CLEAR-110 study, an ongoing, two-year,
open-label extension study of once-daily ARIKACE^®, or liposomal amikacin for
inhalation, to treat Pseudomonas aeruginosa (Pa) in cystic fibrosis (CF)
patients. These data are from 98 patients who have completed the first 12
months (six cycles) of the CLEAR-110 extension study. The data were collected
as part of a scheduled data safety monitoring board (DSMB) review of the
CLEAR-110 extension study. The data showed that ARIKACE was well tolerated,
and there was a sustained improvement from baseline level in Forced Expiratory
Volume in One Second (FEV[1]). These patients also experienced a sustained
reduction in density of Pa sputum while on treatment.

The Two-Year Open-label Extension Study

Eligible patients from the open-label, multicenter, randomized Phase 3
CLEAR-108 trial (as described below) were given the option to participate in a
two-year, open-label, multi-cycle extension study designed to evaluate the
long-term safety and tolerability of ARIKACE in CF patients. 206 patients
enrolled in this study, representing 77% of the patients that completed the
randomized portion of the Phase 3 trial, and received at least one dose of
study drug. The data presented in the attached charts and discussed below
includes 98 patients who had completed one year in the CLEAR-110 study by
December 31, 2013, the time of data cut-off for DSMB review.

These data demonstrate that patients receiving ARIKACE for six cycles (12
months) in the extension study showed mean increase in relative change in
FEV[1] which is sustained during both on-treatment and off-treatment months.
Overall, ARIKACE was well-tolerated during the six cycles administered over
this 12-month period, with adverse events being consistent with those expected
in a population of CF patients receiving inhaled medicines.

"As we announced on July 1, 2013, the Phase 3 study of ARIKACE achieved its
primary endpoint and demonstrated that ARIKACE administered once a day is
non-inferior to the standard of care which is administered twice a day. This
longer-term study demonstrates our continued commitment to the CF patients and
to gathering longer term data to clarify the safety and efficacy profile of
our drug candidate. The data we are reporting today are consistent with the
findings from our earlier longer term study of ARIKACE and we believe further
strengthen the clinical data packages we plan to submit to European and
Canadian regulatory authorities later this year," said Renu Gupta, MD, FAAP,
Executive Vice President Development and Chief Medical Officer of Insmed.

“We are especially pleased that these data demonstrated that patients taking
ARIKACE actually saw their FEV[1] levels remain above baseline on average. In
addition we continue to see reduction of bacterial density and consistency in
our overall safety profile. Further, we believe that once-daily administration
of ARIKACE will support patient convenience and compliance,” said Will Lewis,
President and Chief Executive Officer of Insmed. “With the results achieved in
our Phase 3 trial, along with these positive interim data, we continue our
preparation for regulatory filings with the European Medicines Agency and
Health Canada, which remain on target for mid-year.”

“I would like to recognize Dr. Gupta and our clinical team for designing and
conducting such an important trial. This is the first trial to investigate the
safety and efficacy of inhaled antibiotics in CF patients for a period of up
to two years and it speaks to Insmed’s ongoing commitment to understanding the
safe and effective use of this drug in this target population,” added Mr.

Recap of ARIKACE Data from Phase 3 Clinical Trial to Treat Pa in CF Patients

The Phase 3 trial was designed to assess the comparative safety and efficacy
of ARIKACE and TOBI in CF patients with Pa. A total of 302 adult and pediatric
CF patients with chronic Pa were enrolled at 75 sites in Europe and Canada,
and randomized to receive 28 days of ARIKACE treatment delivered once-daily
via an investigational eFlow^® Nebulizer System, orTOBI delivered twice-daily
via the PARI LC Plus^® Nebulizer System over a 24-week treatment period.

The primary endpoint was relative change in FEV[1] measured after three
treatment cycles, with each cycle consisting of 28 days “on” treatment and 28
days “off” treatment. Secondary endpoints measured were relative changes in
FEV[1] at other time points, time to and number of pulmonary exacerbations,
time to antibiotic rescue treatment, change in density of Pa in sputum,
respiratory hospitalizations and changes in Patient Reported Outcomes
assessing Quality of Life.

The Phase 3 clinical trial of ARIKACE achieved its primary endpoint of
non-inferiority to TOBI for relative change in FEV[1] from baseline to end of
study. Overall, the secondary endpoints showed comparability of once-daily
ARIKACE compared with twice-daily TOBI consistent with the primary endpoint of
the study. The overall safety profile of ARIKACE was comparable to TOBI, with
adverse events consistent with those seen in similar studies and expected in a
population of CF patients receiving inhaled antibiotics. There was no
difference between arms in the reporting of serious adverse events and there
were no unexpected adverse events.

About Cystic Fibrosis

CF is an inherited chronic disease that affects roughly 70,000 children and
adults worldwide, including 30,000 children and adults in the US (Cystic
Fibrosis Foundation Patient Registry, 2011) and 35,000 patients in Europe
(Hoiby, BMC Medicine, 2011, 9:32). In CF patients, a defective gene and its
protein product cause the body to produce unusually thick, sticky mucus that
clogs the lungs and leads to life-threatening lung infections and obstructs
the pancreas and stops natural enzymes from helping the body break down and
absorb food. There is no cure for CF.


ARIKACE  is a form of the antibiotic amikacin, which is enclosed in
nanocapsules of lipid called liposomes. This advanced pulmonary liposome
technology prolongs the release of amikacin in the lungs while minimizing
systemic exposure. The treatment uses biocompatible lipids endogenous to the
lung that are formulated into small (0.3 micron), charge-neutral liposomes.
ARIKACE  is administered once-daily using an optimized, investigational eFlow®
Nebulizer System manufactured by PARI Pharma GmbH, a novel, highly efficient
and portable aerosol delivery system.

ARIKACE  has been granted orphan drug designation in the U.S. by the FDA for
the treatment of Pseudomonas infections in patients with CF and for the
treatment of NTM lung infections. ARIKACE has also received orphan drug
designation in Europe by the EMA for the treatment of Pseudomonas infections
in patients with CF.

About eFlow® Technology and PARI Pharma

ARIKACE is delivered by an investigational eFlow® Nebulizer System developed
by PARI Pharma and optimized specifically for ARIKACE. The optimized device
uses eFlow Technology to enable highly efficient aerosolization of medication
including liposomal formulations via a vibrating, perforated membrane that
includes thousands of laser-drilled holes. Compared with other nebulization
technologies, eFlow Technology produces aerosols with a very high density of
active drug, a precisely defined droplet size and a high proportion of
respirable droplets delivered in the shortest possible period of time. eFlow
Technology is not an ultrasonic nebulizer technology and is not a general
purpose electronic aerosol generator nebulizer technology. Combined with its
quiet mode of operation, small size, light weight and battery use, eFlow
Technology reduces the burden of taking daily, inhaled treatments. PARI Pharma
focuses on the development of aerosol delivery devices and inhalation drug
development to advance aerosol therapies where drug and device can be
optimized together. For more information, please visit

* TOBI^® is a Registered Trademark of Novartis Pharmaceuticals Corporation.

About Insmed

Insmed Incorporated is a biopharmaceutical company dedicated to improving the
lives of patients battling serious lung diseases. Insmed is focused on the
development and commercialization of ARIKACE^®, or liposomal amikacin for
inhalation, for at least two identified orphan patient populations: patients
with non-tuberculous mycobacteria (NTM) lung infections and cystic fibrosis
(CF) patients with Pseudomonas aeruginosa lung infections. For more
information, please visit

Forward-Looking Statements

This release contains forward-looking statements that are made pursuant to
provisions of Section 21E of the Securities Exchange Act of 1934. Words, and
variations of words, such as "intend," "expect," "will," "anticipate,"
"believe," "continue," "propose" and similar expressions are intended to
identify forward-looking statements. Investors are cautioned that such
statements in this release, including statements relating to the status,
results and timing of clinical trials and clinical data, the anticipated
benefits of Insmed's products, the anticipated timing of regulatory
submissions, and the ability to obtain required regulatory approvals, bring
products to market and successfully commercialize products constitute
forward-looking statements that involve risks and uncertainties that could
cause actual results to differ materially from those in the forward-looking
statements. Such risks and uncertainties include, without limitation, failure
or delay of European, Canadian, U.S. Food and Drug Administration and other
regulatory reviews and approvals, competitive developments affecting the
Company’s product candidates, delays in product development or clinical trials
or other studies, patent disputes and other intellectual property developments
relating to the Company’s product candidates, unexpected regulatory actions,
delays or requests, the failure of clinical trials or other studies or results
of clinical trials or other studies that do not meet expectations, the fact
that subsequent analyses of clinical trial or study data may lead to different
(including less favorable) interpretations of trial or study results or may
identify important implications of a trial or study that are not reflected in
the statements contained in this press release, and the fact that trial or
study results or subsequent analyses may be subject to differing
interpretations by regulatory agencies, the inability to successfully develop
the Company’s product candidates or receive necessary regulatory approvals,
inability to make product candidates commercially successful, changes in
anticipated expenses, changes in the Company’s financing requirements or
ability raise additional capital, and other risks and challenges detailed in
the Company’s filings with the U.S. Securities and Exchange Commission,
including its Annual Report on Form 10-K for the year ended December 31, 2012.
Investors are cautioned not to place undue reliance on any forward-looking
statements that speak only as of the date of this news release. The Company
undertakes no obligation to update these forward-looking statements to reflect
events or circumstances or changes in its expectations.

Photos/Multimedia Gallery Available:



Anne Marie Fields, 212-838-3777
Senior Vice President
Bruce Voss, 310-691-7100
Managing Director
Press spacebar to pause and continue. Press esc to stop.