Poster Presentation at the American Thoracic
Society Annual Meeting 2014Reports Twenty-One Culture Negative
Patients at Day 168 of Study
Insmed Incorporated (Nasdaq:INSM) today announced additional
results from the Company’s phase 2 clinical trial of ARIKAYCETM, or
liposomal amikacin for inhalation, for the treatment of patients
with treatment resistant nontuberculous mycobacterial (NTM) lung
infections. The results are highlighted in a poster entitled, “A
Randomized, Double-Blind, Placebo-Controlled Study of Liposomal
Amikacin for Inhalation (ARIKAYCE) in Patients with Recalcitrant
Nontuberculous Mycobacterial Lung Disease,” that will be presented
today at the American Thoracic Society’s Annual Meeting 2014 in San
Diego, California by Kenneth N. Olivier, M.D., M.Ph., National
Institute of Allergy and Infectious Diseases and a co-Principal
Investigator of the study. The poster is available on the Company’s
website at www.insmed.com.
At the conclusion of the 84-day double blind phase of the trial,
78 of the 80 patients agreed to receive once-daily ARIKAYCE plus
standard of care treatment for an additional 84 days. Data from 68
of these patients who completed the visits during the additional
open label phase were available for inclusion in the poster. These
results collected from the open label phase show that 21 of these
patients were culture negative for NTM at Day 168. This data
reflects 10 patients who were culture negative at Day 84 as well as
5 additional patients from the ARIKAYCE arm and 6 additional
patients who were on placebo, switching to ARIKAYCE during the
open-label phase. The number of patients with negative cultures
increased from the double blind phase of the trial in which 11 out
of 44 patients treated with ARIKAYCE (added to standard of care
treatment) demonstrated negative cultures by day 84 of the study as
compared to 3 out of 45 patients treated with standard of care plus
placebo.
“We are encouraged by these additional and durable culture
conversions which we believe is the ultimate goal in the treatment
of NTM lung infections,” said Will Lewis, President and Chief
Executive Officer of Insmed. “The patients screened for admission
to this trial are recalcitrant to treatment. While the entry
criteria for this trial required a minimum of 6 months on standard
of care therapy, over 75% of patients entering this trial were
treated with standard of care therapy for more than a year, yet
remained culture positive. In addition, a majority of these
patients suffer from at least one additional pulmonary
co-morbidity, such as bronchiectasis or cystic fibrosis, making the
hurdle quite high for showing any improvement and making these
results that much more encouraging for patients suffering from this
disease. We now look forward to the regulatory discussions in the
United States and Europe that will guide our path forward.”
In the next several months, the Company plans to incorporate the
trial results into discussions with the regulatory agencies in the
United States and Europe to determine next steps for ARIKAYCE in
the treatment of NTM lung infections. Based upon the culture
conversion results, in April 2014 the Company applied for
Breakthrough Therapy Designation for ARIKAYCE in the United States.
ARIKAYCE has already received Orphan Drug, Qualified Infectious
Disease Product (QIDP) and Fast Track designations from the U.S.
Food and Drug Administration (FDA) for the treatment of NTM lung
infections and recently received Orphan Drug Designation from the
European Medicines Agency (EMA).
Clinical Trial Design and Key Endpoints
The randomized, double-blind, placebo-controlled phase 2
clinical trial compared ARIKAYCE (590 mg delivered once daily for
84 days), added to standard of care treatment, versus standard of
care treatment plus placebo, in 89 adult patients with treatment
resistant NTM lung disease at 19 sites in North America.
Eligibility for the study required patients to have been on the
American Thoracic Society/Infectious Diseases Society of America
(ATS/IDSA) guidelines-based therapy for at least six months prior
to screening with persistently positive mycobacterial cultures.
Following the randomized portion of the study, all eligible
patients had the option to receive ARIKAYCE once daily for an
additional 84 days in an open-label design.
The primary efficacy endpoint of the study was a
semi-quantitative measurement of the change in mycobacterial
density on a seven-point scale from baseline (Day 1) to the end of
the randomized portion of the trial (Day 84). ARIKAYCE did not meet
the pre-specified level for statistical significance although there
was a positive trend (p=0.148) in favor of ARIKAYCE. The key
secondary endpoint of culture conversion reached statistical
significance in favor of ARIKAYCE.
Safety
Patients receiving ARIKAYCE experienced adverse events
consistent with those seen in similar patient populations receiving
inhaled antibiotics. Overall, mild to moderate upper respiratory
irritation was more common in the ARIKAYCE arm compared to the
placebo arm and there was no difference in severe serious adverse
reactions or hemoptysis between the two arms. There was one
Suspected Unexpected Serious Adverse Reaction (SUSAR) observed in
the double-blind phase and one SUSAR observed in the open-label
phase. Instances of hearing loss or tinnitus, a side effect more
commonly associated with intravenous dosing of amikacin, were
evenly balanced between the ARIKAYCE and placebo arms.
About Nontuberculous Mycobacteria (NTM)
Nontuberculous mycobacteria (NTM) are organisms found in the
soil and water that can cause serious lung disease in susceptible
individuals, for which there are currently limited effective
treatments and no approved therapies. The prevalence of NTM disease
is reported to be increasing, and according to reports from the
American Thoracic Society is believed to be greater than that of
tuberculosis in the U.S. According to the National Center for
Biotechnology Information, epidemiological studies show that
presence of NTM infection is increasing in developing countries,
perhaps because of the implementation of tap water. Women with
characteristic phenotype are believed to be at higher risk of
acquiring NTM infection along with patients with defects on cystic
fibrosis transmembrane conductance regulators.
NTM lung disease is often a chronic condition that can lead to
progressive inflammation and lung damage, and is characterized by
bronchiectasis and cavitary disease. NTM infections often require
lengthy hospital stays for medical management. Treatment usually
involves multi-drug regimens that can be poorly tolerated and have
limited effectiveness, especially in patients with severe disease
or in those who have failed prior treatment attempts. According to
a company-sponsored patient chart study conducted by Clarity Pharma
Research, approximately 50,000 patients suffering from NTM lung
disease visited physician offices in the U.S. during 2011.
About ARIKAYCE™
ARIKAYCE 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. ARIKAYCE 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.
This is the first controlled clinical trial of an antibiotic in
patients suffering from NTM lung infections. There are no drugs
approved by the FDA for the treatment of this chronic, debilitating
disease.
About eFlow® Technology and PARI Pharma
ARIKAYCE is delivered by an investigational eFlow® Nebulizer
System developed by PARI Pharma and optimized specifically for
ARIKAYCE. 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.
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
ARIKAYCE, or liposomal amikacin for inhalation, for at least two
identified orphan patient populations: patients with nontuberculous
mycobacteria (NTM) lung infections and cystic fibrosis (CF)
patients with Pseudomonas aeruginosa lung infections. For more
information, please visit http://www.insmed.com.
Forward-looking Statements
This release contains forward-looking statements. 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
Company’s prior disclosures, 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, the ability to obtain Breakthrough
Therapy Designation for ARIKAYCE in the U.S., the inability to make
product candidates commercially successful, changes in anticipated
expenses, changes in the Company’s financing requirements or
ability to raise additional capital, and other risks and challenges
detailed in the Company’s filings with the U.S. Securities and
Exchange Commission, including, without limitation, its Annual
Report on Form 10-K for the year ended December 31, 2013
and its subsequent quarterly reports on Form 10-Q. 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.
LHAAnne Marie Fields, 212-838-3777Senior Vice
Presidentafields@lhai.comorBruce Voss,
310-691-7100Managing Directorbvoss@lhai.com
Insmed (NASDAQ:INSM)
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