BRIDGEWATER, N.J., Jan. 4, 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 unaudited net product sales for the fourth
quarter of 2018, and shared the Company's strategic priorities for
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.
These updates will be discussed as part of the Company's
presentation at the 37th Annual J.P. Morgan Healthcare
Conference in San Francisco on Wednesday, January 9, at 4:00 p.m. PT (7:00 p.m.
ET).
"We are very pleased with the U.S. launch progress of ARIKAYCE
and the strides we have made in a short period of time to reach
patients with refractory MAC lung disease," said Will Lewis, President, Chief Executive Officer,
and Chairman of the Board of Insmed. "Importantly, we've seen
steady increases in patients initiating therapy during the first
three months of launch and have been pleased with the rates of
reimbursement to date from payers. The strong prescription uptake
we've seen thus far is complemented by positive anecdotal feedback
from treating physicians. As we begin the new year, we are excited
to continue this momentum and will provide a further update on the
U.S. launch during our fourth quarter earnings call. We also look
forward to pursuing a number of corporate and clinical milestones
throughout 2019 that we believe will deliver value both to
shareholders and to the rare disease community."
Preliminary Q4 2018 Net Product Sales (Unaudited) and
Additional Launch Metrics
Based on preliminary unaudited financial information, the
Company expects total net product sales of ARIKAYCE to be
approximately $9.8 million for the
quarter ended December 31,
2018, comprising U.S. net sales of $9.2
million and ex-U.S. net sales of $0.6 million. The
ex-U.S. net sales reflect utilization from the Temporary
Authorisation for Use (ATU) program in France. As of
December 31, 2018, more than 500
patients in the U.S. have initiated treatment with ARIKAYCE and
approximately 600 physicians in the U.S have written at least one
prescription for the therapy. The Company commenced
commercialization efforts promptly following FDA approval.
Insmed expects to release final results for the fourth quarter
and audited results for the full year of 2018 on Friday, February 22, 2019.
Strategic Priorities
Insmed has established the following strategic priorities for
2019:
- Continue our efforts to ensure a successful U.S. launch of
ARIKAYCE.
- Complete the design and protocol of the confirmatory clinical
study during the first half of 2019 required for the full approval
of ARIKAYCE by the FDA in the U.S in a front-line setting of
patients with MAC lung disease.
- Accelerate our global expansion to support potential regulatory
filings for ARIKAYCE in Europe in
mid-2019 and in Japan in the first
half of 2020.
- Advance our pipeline, which is intended to bring additional
therapies to market for patients with serious and rare diseases,
including completing enrollment in the WILLOW study, our six-month
Phase 2 trial of INS1007 in patients with non-cystic fibrosis
bronchiectasis, in mid-2019.
Presentation at 37th Annual J.P. Morgan Healthcare
Conference
Will Lewis, President, Chief Executive Officer, and
Chairman of the Board of Insmed, will present at the 37th annual
J.P. Morgan Healthcare Conference on Wednesday, January 9, 2019, at 4:00 p.m. PT (7:00 p.m.
ET). A live audio webcast of the presentation will be
available on the Investor Relations section of the Company's
website at www.insmed.com. A replay will also be archived for 90
days on the Investor Relations section of the site.
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 it 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 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.
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.
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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.
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.
Preliminary Financial Information
The fourth quarter 2018 net product sales information and
additional launch metrics presented above are preliminary and
subject to revision. The preliminary net product sales information
may be revised based on the completion of the Company's fourth
quarter financial close and reporting process and the year-end
audit to be performed by Ernst & Young LLP, the Company's
independent registered public accounting firm. 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 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 US
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 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 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 US or for the Company's product candidates in the US,
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; and inability to
repay the Company's existing indebtedness and uncertainties with
respect to the Company's ability to access future
capital.
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 Quarterly Report on Form 10-Q for the quarter ended
September 30, 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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