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 (FEV1). 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 FEV1 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 FEV1 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. Lewis.
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, or TOBI delivered twice-daily via the PARI LC Plus®
Nebulizer System over a 24-week treatment period.
The primary endpoint was relative change in FEV1 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 FEV1 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 FEV1 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.
About ARIKACE®
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 www.paripharma.com.
* 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 www.insmed.com.
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.
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