MONMOUTH JUNCTION, N.J.,
May 30, 2012 /PRNewswire/
-- Insmed Incorporated (Nasdaq CM: INSM), a biopharmaceutical
company, today provided a corporate update, including the Company's
current strategic development priorities for ARIKACE® (liposomal
amikacin for inhalation).
"Insmed has determined that its current resources will be
applied towards the CLEAR-108 phase 3 European and Canadian
registrational study of ARIKACE in patients with Cystic Fibrosis
(CF)-related Pseudomonas aeruginosa lung infections, the
TARGET-NTM phase 2 trial of ARIKACE in patients with
non-tuberculous mycobacteria (NTM) lung disease and the 9-month dog
toxicity study," said Timothy
Whitten, President and CEO of Insmed.
"We believe this ARIKACE development program prioritizes the
most compelling near-term opportunities for Insmed and its
shareholders," continued Mr. Whitten. "The CLEAR-108 trial
provides Insmed with the earliest opportunity to garner critical
phase 3 ARIKACE data in a broad cystic fibrosis patient population
while generating important information on the efficacy and safety
of ARIKACE as compared to the current standard of care. In
NTM, ARIKACE has the opportunity to become a part of standard
therapy for a rapidly growing chronic disease that has a high unmet
medical need with limited treatment options. We project our
current cash position to be sufficient to generate top-line data
from the three prioritized ARIKACE studies."
"Insmed believes that ARIKACE has the potential to address
important unmet medical needs in CF and NTM, two key orphan
indications with combined global market opportunity of up to
$1 billion," concluded Mr.
Whitten.
Cystic Fibrosis
CLinical Evaluation of ARIKACE
(CLEAR-108) Phase 3 CF Study
- Randomized, phase 3 trial comparing ARIKACE 560 mg, delivered
once daily via an optimized, investigational eFlow® Nebulizer
System (PARI Pharma GmbH), to twice daily TOBI®(1) 300 mg
(inhaled tobramycin solution), a commercially available inhaled
antibiotic delivered via the PARI LC® Plus nebulizer
- Trial is underway in Europe
and Canada and will include
approximately 300 patients
- Primary endpoint is change in pulmonary function (FEV-1)
measured after three 28 day on-treatment and three 28 day
off-treatment cycles (approximately six months)
- Key secondary endpoint is time to pulmonary exacerbation
- Top-line data expected in mid-2013
The study design has been agreed upon by Insmed and the European
Medicines Agency. The study's Principal Investigator is
Diana Bilton, M.D., Director of
Adult CF Centre at the Royal Brompton Hospital in London, England. Eligible patients will
have the option to participate in a longer-term open-label
multi-cycle safety study, called CLEAR-110.
CF Market
CF is a rare, life-threatening genetic disease affecting
approximately 70,000 children and adults worldwide. Today,
the median predicted age of survival for a person with CF is
approximately 38 years, but the median age of death remains in the
mid-20's. More than half of all CF patients have acquired
Pseudomonas lung infections by age 18 and receive extensive
and often chronic antibiotic treatments. Antibiotics
delivered via inhalation have become part of standard treatment for
CF patients with Pseudomonas lung infections. However,
due to the thick sticky mucous these patients produce in their
lungs, CF patients seldom clear the Pseudomonas, and they
become chronically infected. This results in a continuous
decline in lung function, despite all currently available
antibiotic treatments.
Non-TB Mycobacteria
Treatment with ARIKACE to Realize
Greater Efficacy Trial (TARGET-NTM) Phase 2
Clinical Trial
- Randomized, placebo-controlled study of ARIKACE in
approximately 100 adult patients that will include two of the most
common species of NTM that cause lung disease - Mycobacterium
avium complex (MAC) and Mycobacterium abscessus
- NTM culture positive patients will continue with their
antibiotic regimen, and receive additionally either ARIKACE 560 mg,
delivered once daily via an optimized, investigational eFlow
Nebulizer System, or placebo, once daily
- Primary efficacy endpoint will be change in mycobacterial
density from baseline to the end of 84 days of treatment, which is
the end of the randomized portion of the trial
- At the conclusion of the randomized portion of the study,
eligible patients may receive ARIKACE 560 mg once daily for an
additional 84 days in an open-label design. Open-label means
patients will know they are receiving ARIKACE.
- Patient enrollment currently expected to begin in mid-2012,
with top-line results projected in the fourth quarter of 2013
The clinical trial design has been agreed upon by Insmed and the
U.S. Food and Drug Administration, and several sites have recently
begun screening patients. The Principal Investigator of the
study is Kenneth N. Olivier, M.D.,
M.P.H., staff pulmonologist in the Laboratory of Clinical
Infectious Diseases at the National Institute of Allergy and
Infectious Diseases, part of the National Institutes of
Health.
NTM Market
According to a recent 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. More than half of these patients were
treated with antibiotics for NTM.
MAC and Mycobacterium abscessus account for the vast
majority of NTM lung disease, with combined prevalence rates
reported from 75 percent to over 85 percent in the U.S.
U.S. CF Status
Insmed will defer plans to initiate a phase 3 study of ARIKACE
in the U.S. for CF patients until the Company reviews top-line
results from CLEAR-108, as the data generated from CLEAR-108 could
provide additional clarity on the scope, design and conduct of a
U.S. CF phase 3 clinical trial. In addition, the deferral of
a U.S. CF phase 3 clinical trial allows Insmed to focus its current
capital resources on the prioritized ARIKACE clinical studies,
CLEAR-108 (together with the follow-on CLEAR-110) and TARGET-NTM,
as well as the dog toxicity study, which recently started and is
expected to generate top-line results in the second quarter of
2013.
About Insmed
Insmed Incorporated is a biopharmaceutical company focused on
the development of innovative inhaled pharmaceuticals for the
site-specific treatment of serious lung diseases. Insmed's
primary focus is on the development of inhaled antibiotic therapy
delivered via proprietary advanced liposomal pulmonary technology
in areas of high unmet need. For more information, please
visit http://www.insmed.com.
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 to 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 it 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. Online at
www.paripharma.com.
Forward-Looking Statements
This release contains forward-looking statements which are made
pursuant to provisions of Section 21E of the Securities Exchange
Act of 1934. Investors are cautioned that such statements in this
release, including statements relating to our financial position,
results of operations, the status and the results of pre-clinical
studies and clinical trials and pre-clinical and clinical data
described herein, the timing of and costs associated with
pre-clinical studies and clinical trials, the development of our
products, our estimates of the size of the potential markets for
our product candidates, and the business strategies, plans and
objectives of management, constitute forward-looking statements
which involve risks and uncertainties that could cause actual
results to differ materially from those anticipated by the
forward-looking statements. Our results may be affected by
such factors as the receipt and timing of FDA and other regulatory
reviews and approvals, if at all, competitive developments
affecting our product development, delays in product development or
clinical trials, and patent disputes involving currently developing
products. The risks and uncertainties include, without
limitation, we may experience unexpected regulatory actions, delays
or requests, our future clinical trials may not be successful, we
may be unsuccessful in developing our product candidates or
receiving necessary regulatory approvals, we may experience delays
in our product development or clinical trials, our product
candidates may not prove to be commercially successful, our
expenses may be higher than anticipated and other risks and
challenges detailed in our filings with the U.S. Securities and
Exchange Commission, including our Annual Report on Form 10-K for
the year ended December 31, 2011 and
our Quarterly Report on Form 10-Q for the quarter ended
March 31, 2012. Investors are
cautioned not to place undue reliance on any forward-looking
statements which speak only as of the date of this release.
We undertake no obligation to publicly release the results of
any revisions to these forward-looking statements that may be made
to reflect events or circumstances that occur after the date of
this release or to reflect the occurrence of unanticipated
events.
(1) TOBI® is a Registered Trademark of Novartis Pharmaceuticals
Corporation
Investor Relations Contact:
Brian Ritchie – FTI Consulting
212-850-5683
brian.ritchie@fticonsulting.com
Media Contact:
Irma Gomez-Dib – FTI Consulting
212-850-5761
irma.gomez-dib@fticonsulting.com
SOURCE Insmed Incorporated