BRIDGEWATER, N.J., April 8, 2019 /PRNewswire/ -- Insmed Incorporated
(Nasdaq:INSM), a global biopharmaceutical company on a mission to
transform the lives of patients with serious and rare diseases,
today provided an update on the U.S. launch of ARIKAYCE®
(amikacin liposome inhalation suspension), including preliminary
net product sales for the first quarter of 2019. ARIKAYCE was
granted accelerated approval by the U.S. Food and Drug
Administration (FDA) on September 28,
2018, for the treatment of Mycobacterium avium
complex (MAC) lung disease as part of a combination antibacterial
drug regimen for adult patients who have limited or no alternative
treatment options.
"We continue to be very pleased with the U.S. launch progress of
ARIKAYCE and the positive feedback received from the refractory MAC
lung disease community," said Will
Lewis, Chairman and Chief Executive Officer of Insmed. "Six
months into launch, we continue to see steady increases in patients
initiating therapy as well as strong support from payers. We remain
very encouraged by the early trends in our first two quarters of
launch, while carefully monitoring metrics such as patient dropout
rates and duration of therapy. We look forward to providing more
information and additional guidance on the U.S. launch during our
first quarter earnings call in early May."
This update will be discussed as part of the Company's
presentation at the H.C. Wainwright Global Life Sciences Conference
today, Monday, April 8, at
5:50 a.m. ET (10:50 a.m. BST).
Preliminary Q1 2019 Net Product Sales
Based on preliminary financial information, the Company expects
total net product sales of ARIKAYCE to be approximately
$21.9 million for the quarter ended
March 31,
2019, comprising U.S. net product sales
of $21.0 million and ex-U.S. net product sales
of $0.9 million. The ex-U.S. net product sales are comprised
of $0.8 million from the Temporary
Authorization for Use (Autorisation Temporaire d'Utilisation or
ATU) program in France and
$0.1 million from the named patient
program in Germany, both
compassionate use programs.
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 needs. Current guideline-based treatment
involves the use of multi-drug regimens that are not specifically
approved for MAC lung disease. The course of treatment is often two
years or more and is inadequate in treating the disease in many
patients.
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. 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.
IMPORTANT SAFETY INFORMATION
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.
|
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.
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.
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 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 is
ARIKAYCE® (amikacin liposome inhalation
suspension), which is approved in the
United States 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. MAC lung disease is a rare and
often chronic infection that can cause 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 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.
Preliminary Financial Information
The first quarter 2019 net product sales information presented
above is preliminary and subject to revision based on the
completion of the Company's first quarter financial close and Form
10-Q reporting process. During the course of this process, the
Company may identify items that would require adjustments to this
information. These estimates thus constitute forward-looking
statements, and the Company cautions you that they are subject to
risks and uncertainties. Ernst & Young LLP, the Company's
independent registered public accounting firm, has not audited,
reviewed, compiled or performed any procedures with respect to
these preliminary estimates. Accordingly, Ernst & Young LLP
does not express an opinion or any other form of assurance with
respect to these preliminary estimates.
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;
uncertainties in the degree of market acceptance of ARIKAYCE by
physicians, patients, third-party payers 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 successfully complete the confirmatory
post-marketing study required for full approval; 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
payers for ARIKAYCE or acceptable prices for ARIKAYCE; development
of unexpected safety or efficacy concerns related to ARIKAYCE;
inaccuracies in the Company's estimates of the size of the
potential markets for ARIKAYCE, the expected rates of patient
uptake, the duration of expected treatment, or expected patient
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
and the Company's 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 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 regulatory approvals for ARIKAYCE
outside the U.S. or for the Company's product candidates in the
U.S., Europe, Japan or other markets; 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 imposed on
the Company by its material license agreements, 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; limited experience operating internationally;
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 and
uncertainties with respect to the Company's ability to access
future capital; and delays in the execution of plans to build out
and move into the leased space at the Company's new headquarters
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, 2018 and any subsequent Company
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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SOURCE Insmed Incorporated