Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical
company focused on the unmet needs of patients with rare diseases,
today announced that it has submitted its New Drug Application
(NDA) for ALIS (Amikacin Liposome Inhalation Suspension) to the
U.S. Food and Drug Administration (FDA) for adult patients with
Nontuberculous Mycobacterial (NTM) lung disease caused by
Mycobacterium avium complex (MAC).
“We are very excited to announce the completion of this
important milestone which begins the formal process of review by
the FDA of our application for approval of ALIS to treat patients
living with NTM caused by MAC. There are currently no
approved therapies in the U.S. to treat this disease. Our
submission is supported by our pivotal Phase 3 INS-212 study
conducted in subjects with refractory NTM lung disease, the
completed Phase 2 study TR02-112, as well as an expanded safety
database,” remarked Will Lewis, President and Chief Executive
Officer of Insmed. “We look forward to continuing our conversations
with the FDA during this process.”
The Company anticipates receiving a six-month Priority Review
and that the NDA will be reviewed by the Division of Anti-Infective
Products. The FDA will have 60 days to review the submission
of the NDA to determine if it is complete and acceptable for
filing. The FDA has previously designated ALIS as an orphan
drug, a breakthrough therapy and a Qualified Infectious Disease
Product (QIDP) under the Generating Antibiotic Incentives Now
(GAIN) Act.
About NTM Lung Disease
NTM lung disease is a rare and serious disorder associated with
increased rates of morbidity and mortality. There is an increasing
prevalence of lung disease caused by NTM, and we believe it is an
emerging public health concern worldwide. Patients with NTM lung
disease may experience a multitude of symptoms such as fever,
weight loss, cough, lack of appetite, night sweats, blood in the
sputum, and fatigue. Patients with NTM lung disease frequently
require lengthy hospital stays to manage their condition. We
are not aware of any approved inhaled therapies specifically
indicated for refractory NTM lung disease caused by MAC in North
America, Japan or Europe. Current guideline-based approaches
involve use of multi-drug regimens not approved for the treatment
of NTM lung disease, and treatment can be as long as two years or
more.
The prevalence of human disease attributable to NTM has
increased over the past two decades. In a decade long study (1997
to 2007), researchers found that the prevalence of NTM lung disease
in the U.S. was increasing at approximately 8% per year and that
NTM patients on Medicare over the age of 65 were 40% more likely to
die over the period of the study than those who did not have the
disease. In the U.S., we estimate there will be between 75,000 and
105,000 patients with diagnosed NTM lung disease in 2018, of which
we expect 40,000 to 50,000 will be treated for NTM lung disease
caused by MAC. We expect that between 10,000 and 15,000 of
these patients will be refractory to treatment. In Japan, we
estimate there will be between 125,000 and 145,000 patients with
diagnosed NTM lung disease in 2018, with approximately 60,000 to
70,000 of those patients being treated for NTM lung disease caused
by MAC and 15,000 to 18,000 of these treated patients being
refractory to treatment. We also estimate there will be
approximately 14,000 patients with diagnosed NTM lung disease in
the EU5 (comprised of France, Germany, Italy, Spain and the United
Kingdom) in 2018, of which we estimate approximately 4,400 will be
treated for NTM lung disease caused by MAC and approximately 1,400
of these treated patients will be refractory to
treatment.
About ALIS
ALIS is a novel, inhaled, once-daily formulation of amikacin
that is in late-stage clinical development for adult patients with
treatment-refractory NTM lung disease caused by MAC. Amikacin
solution for parenteral administration is an established drug that
has activity against a variety of NTM; however, its use is limited
by the need to administer it intravenously and by toxicity to
hearing, balance, and kidney function associated with this systemic
exposure. Insmed's advanced pulmonary liposome technology uses
charge neutral liposomes to deliver amikacin directly to the lung
where it is taken up by the lung macrophages where the NTM
infection resides. This prolongs the release of amikacin in the
lungs while minimizing systemic exposure thereby offering the
potential for decreased systemic toxicities. ALIS’s ability to
deliver high levels of amikacin directly to the lung distinguishes
it from intravenous amikacin. ALIS is administered once daily using
an optimized, investigational eFlow® Nebulizer System manufactured
by PARI Pharma GmbH (PARI), a portable aerosol delivery
system.
About CONVERT (INS-212) and INS-312
CONVERT is a randomized, open-label, global Phase 3 trial
designed to confirm the culture conversion results seen in Insmed’s
Phase 2 clinical trial of ALIS in patients with refractory NTM lung
disease caused by MAC. CONVERT is being conducted in 18 countries
at more than 125 sites. The primary efficacy endpoint is the
proportion of patients who achieve sputum culture conversion
(defined as 3 consecutive negative monthly sputum cultures) by
Month 6 in the ALIS plus GBT arm compared to the GBT-only arm.
Patients who achieve culture conversion by Month 6 will continue in
the CONVERT study for an additional 12 months of treatment
following the first monthly negative sputum culture. Patients who
do not culture convert have the option of enrolling in our INS-312
study. INS-312 is a single-arm, open-label extension study for
patients who completed six months of treatment in the INS-212
study, but did not demonstrate culture conversion by Month 6. Under
the study protocol, patients in the ALIS plus GBT arm of the
INS-212 study will receive an additional 12 months of ALIS plus
GBT. Patients who crossed over from the GBT-only arm of the INS-212
study will receive 12 months of treatment of ALIS + GBT.
About Insmed
Insmed Incorporated is a global biopharmaceutical Company
focused on the unmet needs of patients with rare diseases. The
Company’s lead product candidate is ALIS, which is in late-state
development for adult patients with treatment refractory NTM lung
disease caused by MAC, which is a rare and often chronic infection
that is capable of causing irreversible lung damage and can be
fatal. Insmed's earlier-stage clinical pipeline includes
INS1007, a novel oral reversible inhibitor of dipeptidyl peptidase
1 with therapeutic potential in non-cystic fibrosis bronchiectasis
and other inflammatory diseases, and INS1009, an inhaled
nanoparticle formulation of a treprostinil prodrug that may offer a
differentiated product profile for rare pulmonary disorders,
including pulmonary arterial hypertension. For more
information, visit www.insmed.com.
Forward-looking Statements
This press release contains forward-looking statements that
involve substantial risks and uncertainties. "Forward-looking
statements," as that term is defined in the Private Securities
Litigation Reform Act of 1995, are statements that are not
historical facts and involve a number of risks and uncertainties.
Words herein such as "may," "will," "should," "could," "would,"
"expects," "plans," "anticipates," "believes," "estimates,"
"projects," "predicts," "intends," "potential," "continues," and
similar expressions (as well as other words or expressions
referencing future events, conditions or circumstances) may
identify forward-looking statements.
The forward-looking statements in this press release are based
upon the Company’s current expectations and beliefs, and involve
known and unknown risks, uncertainties and other factors, which may
cause the Company’s actual results, performance and achievements
and the timing of certain events to differ materially from the
results, performance, achievements or timing discussed, projected,
anticipated or indicated in any forward-looking statements. Such
risks, uncertainties and other factors include, among others, the
following: risks that the full six-month data from the INS-212
study or subsequent data from the remainder of the study’s
treatment and off-treatment phases will not be consistent with the
top-line six-month results of the study; uncertainties in the
research and development of the Company’s existing product
candidates, including due to delays in data readouts, such as the
full data from the INS-212 study, patient enrollment and retention
or failure of the Company’s preclinical studies or clinical trials
to satisfy pre-established endpoints, including secondary endpoints
in the INS-212 study and endpoints in the INS-212 extension study
(the 312 study); risks that subsequent data from the 312 study will
not be consistent with the interim results; failure to obtain, or
delays in obtaining, regulatory approval from the U.S. Food and
Drug Administration, Japan’s Ministry of Health, Labour and
Welfare, Japan’s Pharmaceuticals and Medical Devices Agency, the
European Medicines Agency, and other regulatory authorities for the
Company’s product candidates or their delivery devices, such as the
eFlow Nebulizer System, including due to insufficient clinical
data, selection of endpoints that are not satisfactory to
regulators, complexity in the review process for combination
products or inadequate or delayed data from a human factors study
required for U.S. regulatory approval; failure to maintain
regulatory approval for the Company’s product candidates, if
received, due to a failure to satisfy post-approval regulatory
requirements, such as the submission of sufficient data from
confirmatory clinical studies; safety and efficacy concerns related
to the Company’s product candidates; lack of experience in
conducting and managing preclinical development activities and
clinical trials necessary for regulatory approval, including the
regulatory filing and review process; failure to comply with
extensive post-approval regulatory requirements or imposition of
significant post-approval restrictions on the Company’s product
candidates by regulators; uncertainties in the rate and degree of
market acceptance of product candidates, if approved; inability to
create an effective direct sales and marketing infrastructure or to
partner with third parties that offer such an infrastructure for
distribution of the Company’s product candidates, if approved;
inaccuracies in the Company’s estimates of the size of the
potential markets for the Company’s product candidates or
limitations by regulators on the proposed treatment population for
the Company’s product candidates; failure of third parties on which
the Company is dependent to conduct the Company’s clinical trials,
to manufacture sufficient quantities of the Company’s product
candidates for clinical or commercial needs, including the
Company’s raw materials suppliers, or to comply with the Company’s
agreements or laws and regulations that impact the Company’s
business; inaccurate estimates regarding the Company’s future
capital requirements, including those necessary to fund the
Company’s ongoing clinical development, regulatory and
commercialization efforts as well as milestone payments or
royalties owed to third parties; failure to develop, or to license
for development, additional product candidates, including a failure
to attract experienced third-party collaborators; uncertainties in
the timing, scope and rate of reimbursement for the Company’s
product candidates; changes in laws and regulations applicable to
the Company’s business and failure to comply with such laws and
regulations; inability to repay the Company’s existing indebtedness
or to obtain additional capital when needed on desirable terms or
at all; failure to obtain, protect and enforce the Company’s
patents and other intellectual property and costs associated with
litigation or other proceedings related to such matters;
restrictions imposed on the Company by license agreements that are
critical for the Company’s product development, including the
Company’s license agreements with PARI Pharma GmbH and AstraZeneca
AB, and failure to comply with the Company’s obligations under such
agreements; competitive developments affecting the Company’s
product candidates and potential exclusivity related thereto; the
cost and potential reputational damage resulting from litigation to
which the Company is or may be a party, including, without
limitation, the class action lawsuit against the Company that
recently was dismissed without prejudice; loss of key personnel;
and lack of experience operating internationally.
The Company may not actually achieve the results, plans,
intentions or expectations indicated by the Company’s
forward-looking statements because, by their nature,
forward-looking statements involve risks and uncertainties because
they relate to events and depend on circumstances that may or may
not occur in the future. For additional information about the risks
and uncertainties that may affect the Company’s business, please
see the factors discussed in Item 1A, "Risk Factors," in the
Company’s Annual Report on Form 10-K for the year ended December
31, 2017 and any subsequent filings with the Securities and
Exchange Commission.
The Company cautions readers not to place undue reliance on any
such forward-looking statements, which speak only as of the date of
this press release. The Company disclaims any obligation, except as
specifically required by law and the rules of the Securities and
Exchange Commission, to publicly update or revise any such
statements to reflect any change in expectations or in events,
conditions or circumstances on which any such statements may be
based, or that may affect the likelihood that actual results will
differ from those set forth in the forward-looking statements.
Contact:
Blaine Davis Insmed Incorporated (908) 947-2841
blaine.davis@insmed.com
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