BRIDGEWATER, N.J., July 7, 2020 /PRNewswire/ -- Insmed
Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a
mission to transform the lives of patients with serious and rare
diseases, today announced that ARIKAYCE® (amikacin
liposome inhalation suspension) has been included in the new
international treatment guidelines for nontuberculous mycobacterial
(NTM) lung disease, a chronic, debilitating condition that can
cause severe, permanent damage to the lungs. The evidence-based
guidelines, issued by the American Thoracic Society (ATS), European
Respiratory Society (ERS), European Society of Clinical
Microbiology and Infectious Diseases (ESCMID), and Infectious
Diseases Society of America (IDSA), recommend the use of
ARIKAYCE for the treatment of patients with refractory NTM
lung disease caused by Mycobacterium avium complex (MAC) as
part of a combination antibacterial drug regimen for adult patients
with limited or no alternative treatment options.
Specifically, the guidelines strongly recommend the addition of
ARIKAYCE to the standard treatment regimen for patients with MAC
lung disease who have failed to convert to a negative sputum
culture after at least six months of treatment.
The guidelines, published in Clinical Infectious Diseases
and accessible here, are the globally recognized standard for the
prevention, diagnosis, and treatment of NTM lung disease with the
goal of providing the latest evidence-based guidance to improve
patient care and outcomes. This is the first update to the
guidelines in more than a decade, marking a significant milestone
in the management of NTM lung disease.
"The inclusion of ARIKAYCE in the new NTM treatment guidelines
highlights the critical role this innovative therapy can play in
the management of patients with refractory MAC lung disease," said
Martina Flammer, M.D., MBA, Chief
Medical Officer of Insmed. "We appreciate the dedication and hard
work of the medical societies, physicians, and researchers who
developed these guidelines, which will help improve patient care
and outcomes for those battling this difficult-to-treat
disease."
ARIKAYCE is the first and only therapy approved in the United States for the treatment of
refractory NTM lung disease caused by MAC. ARIKAYCE received U.S.
Food and Drug Administration (FDA) accelerated approval based
on results of the Phase 3 CONVERT study, which demonstrated that
once-daily ARIKAYCE, when combined with background regimen therapy,
improved sputum culture conversion rates in patients with
refractory NTM lung disease caused by MAC. In the study, the
addition of ARIKAYCE to background regimen therapy eliminated
evidence of NTM lung disease caused by MAC in sputum by Month 6 in
29% of patients, compared to 9% of patients on background regimen
therapy alone.
About MAC Lung Disease
Mycobacterium avium complex (MAC) lung disease is a
rare and serious disorder that can significantly increase morbidity
and mortality. Patients with MAC lung disease can experience a
range of symptoms that often worsen over time, including chronic
cough, dyspnea, fatigue, fever, weight loss, and chest pain. In
some cases, MAC lung disease can cause severe, even permanent
damage to the lungs, and can be fatal. MAC lung disease is an
emerging public health concern worldwide with significant unmet
need.
About ARIKAYCE® (amikacin liposome inhalation
suspension)
ARIKAYCE is the first and only FDA-approved therapy indicated
for the treatment of Mycobacterium avium complex
(MAC) lung disease as part of a combination antibacterial drug
regimen for adult patients with limited or no alternative treatment
options. Current international treatment guidelines recommend
the use of ARIKAYCE in patients with MAC lung disease who have
failed standard therapy after at least six months of treatment.
ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin,
an established antibiotic that was historically administered
intravenously and associated with severe toxicity to hearing,
balance, and kidney function. Insmed's proprietary PULMOVANCE™
liposomal technology enables the delivery of amikacin directly to
the lungs, where liposomal amikacin is taken up by lung macrophages
where the infection resides. This approach prolongs the release of
amikacin in the lungs while limiting systemic exposure. ARIKAYCE is
administered once daily using the Lamira® Nebulizer
System manufactured by PARI Pharma GmbH (PARI).
About PARI Pharma and the Lamira® Nebulizer
System
ARIKAYCE® (amikacin liposome inhalation
suspension) is delivered by a novel inhalation device, the
Lamira® Nebulizer System, developed by PARI.
Lamira® is a quiet, portable nebulizer that enables
efficient aerosolization of liquid medications, including liposomal
formulations such as ARIKAYCE, via a vibrating, perforated
membrane. Based on PARI's 100-year history working with aerosols,
PARI is dedicated to advancing inhalation therapies by developing
innovative delivery platforms and new pharmaceutical formulations
that work together to improve patient care.
About CONVERT (INS-212)
CONVERT was a randomized, open-label, global Phase 3 study
designed to confirm the sputum culture conversion results seen in
Insmed's Phase 2 clinical study of ARIKAYCE in patients with
refractory NTM lung disease caused by MAC. CONVERT was conducted in
18 countries at more than 125 sites. The primary efficacy endpoint
was the proportion of patients who achieved sputum culture
conversion at Month 6 in the ARIKAYCE plus GBT arm compared to the
GBT-only arm. Patients who achieved sputum culture conversion by
Month 6 continued in the CONVERT study for an additional 12 months
of treatment following the first monthly negative sputum culture.
The CONVERT study also evaluated the proportion of patients who
maintained sputum culture conversion 3 months off all MAC
treatment.
IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.
WARNING: RISK OF
INCREASED RESPIRATORY ADVERSE REACTIONS
ARIKAYCE has been
associated with an increased risk of respiratory adverse reactions,
including hypersensitivity pneumonitis, hemoptysis, bronchospasm,
and exacerbation of underlying pulmonary disease that have led to
hospitalizations in some cases.
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Hypersensitivity Pneumonitis has been reported with the
use of ARIKAYCE in the clinical trials. Hypersensitivity
pneumonitis (reported as allergic alveolitis, pneumonitis,
interstitial lung disease, allergic reaction to ARIKAYCE) was
reported at a higher frequency in patients treated with ARIKAYCE
plus background regimen (3.1%) compared to patients treated with a
background regimen alone (0%). Most patients with hypersensitivity
pneumonitis discontinued treatment with ARIKAYCE and received
treatment with corticosteroids. If hypersensitivity pneumonitis
occurs, discontinue ARIKAYCE and manage patients as medically
appropriate.
Hemoptysis has been reported with the use of ARIKAYCE in
the clinical trials. Hemoptysis was reported at a higher frequency
in patients treated with ARIKAYCE plus background regimen (17.9%)
compared to patients treated with a background regimen alone
(12.5%). If hemoptysis occurs, manage patients as medically
appropriate.
Bronchospasm has been reported with the use of
ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma,
bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea
exertional, prolonged expiration, throat tightness, wheezing) was
reported at a higher frequency in patients treated with ARIKAYCE
plus background regimen (28.7%) compared to patients treated
with a background regimen alone (10.7%). If bronchospasm occurs
during the use of ARIKAYCE, treat patients as medically
appropriate.
Exacerbations of underlying pulmonary disease has
been reported with the use of ARIKAYCE in the clinical trials.
Exacerbations of underlying pulmonary disease (reported as chronic
obstructive pulmonary disease (COPD), infective exacerbation of
COPD, infective exacerbation of bronchiectasis) have been reported
at a higher frequency in patients treated with ARIKAYCE plus
background regimen (14.8%) compared to patients treated with
background regimen alone (9.8%). If exacerbations of
underlying pulmonary disease occur during the use of ARIKAYCE,
treat patients as medically appropriate.
Anaphylaxis and Hypersensitivity Reactions: Serious
and potentially life-threatening hypersensitivity reactions,
including anaphylaxis, have been reported in patients taking
ARIKAYCE. Signs and symptoms include acute onset of skin and
mucosal tissue hypersensitivity reactions (hives, itching,
flushing, swollen lips/tongue/uvula), respiratory difficulty
(shortness of breath, wheezing, stridor, cough), gastrointestinal
symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and
cardiovascular signs and symptoms of anaphylaxis (tachycardia, low
blood pressure, syncope, incontinence, dizziness). Before therapy
with ARIKAYCE is instituted, evaluate for previous hypersensitivity
reactions to aminoglycosides. If anaphylaxis or a hypersensitivity
reaction occurs, discontinue ARIKAYCE and institute appropriate
supportive measures.
Ototoxicity has been reported with the use of ARIKAYCE in
the clinical trials. Ototoxicity (including deafness, dizziness,
presyncope, tinnitus, and vertigo) were reported with a higher
frequency in patients treated with ARIKAYCE plus background regimen
(17%) compared to patients treated with background
regimen alone (9.8%). This was primarily driven by tinnitus
(7.6% in ARIKAYCE plus background regimen vs 0.9% in the background
regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background
regimen vs 2.7% in the background regimen alone arm). Closely
monitor patients with known or suspected auditory or vestibular
dysfunction during treatment with ARIKAYCE. If ototoxicity occurs,
manage patients as medically appropriate, including potentially
discontinuing ARIKAYCE.
Nephrotoxicity was observed during the clinical
trials of ARIKAYCE in patients with MAC lung disease but not at a
higher frequency than background regimen alone. Nephrotoxicity has
been associated with the aminoglycosides. Close monitoring of
patients with known or suspected renal dysfunction may be needed
when prescribing ARIKAYCE.
Neuromuscular Blockade: Patients with neuromuscular
disorders were not enrolled in ARIKAYCE clinical trials. Patients
with known or suspected neuromuscular disorders, such as myasthenia
gravis, should be closely monitored since aminoglycosides may
aggravate muscle weakness by blocking the release of acetylcholine
at neuromuscular junctions.
Embryo-Fetal Toxicity: Aminoglycosides can cause
fetal harm when administered to a pregnant woman. Aminoglycosides,
including ARIKAYCE, may be associated with total, irreversible,
bilateral congenital deafness in pediatric patients exposed in
utero. Patients who use ARIKAYCE during pregnancy, or become
pregnant while taking ARIKAYCE should be apprised of the potential
hazard to the fetus.
Contraindications: ARIKAYCE is contraindicated in
patients with known hypersensitivity to any aminoglycoside.
Most Common Adverse Reactions: The most common adverse
reactions in Trial 1 at an incidence ≥5% for patients using
ARIKAYCE plus background regimen compared to patients treated with
background regimen alone were dysphonia (47% vs 1%), cough (39% vs
17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%),
ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%),
musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs
10%), exacerbation of underlying pulmonary disease (15% vs 10%),
diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%),
headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash
(6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs
1%), and chest discomfort (5% vs 3%).
Drug Interactions: Avoid concomitant use of ARIKAYCE
with medications associated with neurotoxicity, nephrotoxicity, and
ototoxicity. Some diuretics can enhance aminoglycoside toxicity by
altering aminoglycoside concentrations in serum and tissue. Avoid
concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea,
or intravenous mannitol.
Overdosage: Adverse reactions specifically associated
with overdose of ARIKAYCE have not been identified. Acute
toxicity should be treated with immediate withdrawal of ARIKAYCE,
and baseline tests of renal function should be undertaken.
Hemodialysis may be helpful in removing amikacin from the body. In
all cases of suspected overdosage, physicians should contact the
Regional Poison Control Center for information about effective
treatment.
U.S. INDICATION
LIMITED POPULATION: ARIKAYCE® is
indicated in adults, who have limited or no alternative treatment
options, for the treatment of Mycobacterium
avium complex (MAC) lung disease as part of a combination
antibacterial drug regimen in patients who do not achieve negative
sputum cultures after a minimum of 6 consecutive months of a
multidrug background regimen therapy. As only limited clinical
safety and effectiveness data for ARIKAYCE are currently available,
reserve ARIKAYCE for use in adults who have limited or no
alternative treatment options. This drug is indicated for
use in a limited and specific population of patients.
This indication is approved under accelerated approval based
on achieving sputum culture conversion (defined as 3 consecutive
negative monthly sputum cultures) by Month 6. Clinical benefit has
not yet been established. Continued approval for this indication
may be contingent upon verification and description of clinical
benefit in confirmatory trials.
Limitation of Use: ARIKAYCE has only been studied
in patients with refractory MAC lung disease defined as patients
who did not achieve negative sputum cultures after a minimum of 6
consecutive months of a multidrug background regimen therapy. The
use of ARIKAYCE is not recommended for patients with non-refractory
MAC lung disease.
Patients are encouraged to report negative side effects of
prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or call 1–800–FDA–1088. You
can also call the Company at 1-844-4-INSMED.
Please see Full Prescribing
Information.
About Insmed
Insmed Incorporated is a global biopharmaceutical company on a
mission to transform the lives of patients with serious and rare
diseases. Insmed's first commercial product, ARIKAYCE®
(amikacin liposome inhalation suspension), is the first and only
therapy approved in the United States for the treatment
of refractory Mycobacterium avium complex (MAC)
lung disease as part of a combination antibacterial drug regimen
for adult patients with limited or no alternative treatment
options. MAC lung disease is a chronic, debilitating condition that
can cause severe and permanent lung damage. Insmed's earlier-stage
clinical pipeline includes brensocatib, a novel oral reversible
inhibitor of dipeptidyl peptidase 1 with therapeutic potential in
non-cystic fibrosis bronchiectasis and other inflammatory diseases,
and treprostinil palmitil, an inhaled 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: failure to successfully commercialize or maintain U.S.
approval for ARIKAYCE, the Company's only approved product; the
risk that brensocatib does not prove effective or safe for patients
in the STOP-COVID19 study; business or economic disruptions due to
catastrophes or other events, including natural disasters or public
health crises; impact of the novel coronavirus (COVID-19) pandemic
and efforts to reduce its spread on our business, employees,
including key personnel, patients, partners and suppliers;
uncertainties in the degree of market acceptance of ARIKAYCE by
physicians, patients, third-party payors and others in the
healthcare community; the Company's inability to obtain full
approval of ARIKAYCE from the FDA, including the risk that the
Company will not timely and successfully complete the study to
validate a PRO tool and complete the confirmatory post-marketing
study required for full approval of ARIKAYCE; inability of the
Company, PARI or the Company's other third party manufacturers to
comply with regulatory requirements related to ARIKAYCE or the
Lamira® Nebulizer System; the Company's inability to
obtain adequate reimbursement from government or third-party payors
for ARIKAYCE or acceptable prices for ARIKAYCE; development of
unexpected safety or efficacy concerns related to ARIKAYCE or
brensocatib; inaccuracies in the Company's estimates of the size of
the potential markets for ARIKAYCE or brensocatib or in data the
Company has used to identify physicians; expected rates of patient
uptake, duration of expected treatment, or expected patient
adherence or discontinuation rates; the Company's inability to
create an effective direct sales and marketing infrastructure or to
partner with third parties that offer such an infrastructure for
distribution of ARIKAYCE; failure to obtain regulatory approval to
expand ARIKAYCE's indication to a broader patient population;
failure to successfully conduct future clinical trials for
ARIKAYCE, brensocatib and the Company's other product candidates,
including due to the Company's limited experience in conducting
preclinical development activities and clinical trials necessary
for regulatory approval and the Company's inability to enroll or
retain sufficient patients to conduct and complete the trials or
generate data necessary for regulatory approval; risks that the
Company's clinical studies will be delayed or that serious side
effects will be identified during drug development; failure to
obtain, or delays in obtaining, regulatory approvals for ARIKAYCE
outside the U.S. or for the Company's product candidates in the
U.S., Europe, Japan or other markets, including the
United Kingdom as a result of its
recent exit from the European Union; failure of third parties on
which the Company is dependent to manufacture sufficient quantities
of ARIKAYCE or the Company's product candidates for commercial or
clinical needs, to conduct the Company's clinical trials, or to
comply with laws and regulations that impact the Company's business
or agreements with the Company; the Company's inability to attract
and retain key personnel or to effectively manage the Company's
growth; the Company's inability to adapt to its highly competitive
and changing environment; the Company's inability to adequately
protect its intellectual property rights or prevent disclosure of
its trade secrets and other proprietary information and costs
associated with litigation or other proceedings related to such
matters; restrictions or other obligations imposed on the Company
by its agreements related to ARIKAYCE or the Company's product
candidates, including its license agreements with PARI and
AstraZeneca AB, and failure of the Company to comply with its
obligations under such agreements; the cost and potential
reputational damage resulting from litigation to which the Company
is or may become a party, including product liability claims; the
Company's limited experience operating internationally; changes in
laws and regulations applicable to the Company's business,
including any pricing reform, and failure to comply with such laws
and regulations; inability to repay the Company's existing
indebtedness and uncertainties with respect to the Company's
ability to access future capital; and delays in the execution of
plans to build out an additional FDA-approved third-party
manufacturing facility and unexpected expenses associated with
those plans.
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, 2019, our Quarterly
Report on Form 10-Q for the quarter ended March 31, 2020 and any subsequent Company filings
with the SEC.
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 SEC, 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:
Investors:
Argot Partners
Laura Perry or Heather Savelle
(212) 600-1902
Insmed@argotpartners.com
Media:
Mandy Fahey
Senior Director, Corporate Communications
Insmed
(732) 718-3621
amanda.fahey@insmed.com
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