Initial 12-Month Data Demonstrate Long-Term
Safety and Durability of Effect
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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LHAAnne Marie Fields, 212-838-3777Senior Vice
Presidentafields@lhai.comorBruce Voss,
310-691-7100Managing Directorbvoss@lhai.com
Insmed (NASDAQ:INSM)
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