--Advisory committee supports surrogate endpoint
of sputum culture conversion--
Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical
company focused on the unmet needs of patients with rare diseases,
today announced that the U.S. Food and Drug Administration’s (FDA)
Antimicrobial Drugs Advisory Committee voted 12 to 2 in favor of
the safety and effectiveness of ALIS (amikacin liposome inhalation
suspension) for adults with nontuberculous mycobacterial (NTM) lung
disease caused by Mycobacterium avium complex (MAC) who have
limited or no treatment options. The committee also voted in favor
of the surrogate endpoint of sputum culture conversion used in the
Phase 3 CONVERT study being reasonably likely to predict clinical
benefit. If approved, ALIS will be the first and only therapy in
the U.S. specifically indicated for the treatment of patients with
NTM lung disease caused by MAC.
“We are very pleased by the outcome of today’s advisory
committee meeting, which recognized the role ALIS may be able to
play in addressing the significant unmet medical need among
patients suffering from NTM lung disease caused by MAC, a chronic,
debilitating and potentially fatal infection,” said Will Lewis,
President and Chief Executive Officer of Insmed.
The advisory committee’s recommendation was based on briefing
materials developed from Insmed’s new drug application (NDA) for
ALIS, which was submitted under accelerated approval provisions and
includes data from the Phase 3 CONVERT study. The study met its
primary endpoint of culture conversion by Month 6 with statistical
significance for once-daily ALIS when added to guideline-based
therapy (GBT) compared with GBT alone in patients with refractory
NTM lung disease due to MAC.
“The patients included in our Phase 3 clinical trial represent
the most difficult-to-treat segment of the NTM lung disease
population, having already failed treatment with current
guideline-based therapy. The committee’s favorable recommendation
brings us one step closer to providing the first and only
FDA-approved treatment for these patients. We look forward to
working closely with the FDA as it completes its review of our
application,” said Paul Streck, M.D., Chief Medical Officer of
Insmed.
In a separate vote, the committee voted against the safety and
effectiveness of ALIS in the broadest population of adult patients
with NTM lung disease caused by MAC.
The FDA is not bound by the committee’s recommendation but takes
its input into consideration when evaluating whether to approve
Insmed’s NDA for ALIS, which is currently under Priority Review by
the FDA with an action date of September 28, 2018 under
the Prescription Drug User Fee Act (PDUFA). The FDA has
previously designated ALIS 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 Insmed believes 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. Insmed is
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., Insmed estimates there will be between 75,000
and 105,000 patients with diagnosed NTM lung disease in 2018, of
which the Company expects 40,000 to 50,000 will be treated for NTM
lung disease caused by MAC. Insmed expects that between 10,000 and
15,000 of these patients will be refractory to treatment. In Japan,
Insmed estimates 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. Insmed also estimates 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 the Company estimates 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 and under regulatory
review by the FDA for adult patients with 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. 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 achieved culture conversion at Month 6
in the ALIS plus GBT arm compared to the GBT-only arm. Patients who
achieved culture conversion by Month 6 are continuing in the
CONVERT study for an additional 12 months of treatment following
the first monthly negative sputum culture. Patients who did not
culture convert may have been eligible to enroll 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, non-converting 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 plus
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 and under regulatory review by the FDA for adult
patients with 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 remainder of the data from the treatment
and off-treatment phases of INS-212 will not be consistent with the
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
INS-312 study); risks that subsequent data from the INS-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, extensions of action dates under PDUFA or complexity in
the review process for combination products; imposition of
significant post-approval regulatory requirements on our product
candidates or 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; 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 become a party; 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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