--Real-World Evidence Evaluating Reduction in
Hospitalizations Following Initiation of use of
ARIKAYCE® (amikacin liposome inhalation suspension) to
be Presented in Poster Discussion Session--
--Analysis of Phase 2 WILLOW Study Evaluating the
Benefit-Risk Profile of Brensocatib in Adult Patients with
Non-Cystic Fibrosis Bronchiectasis to be Presented in
Mini-Symposium--
BRIDGEWATER, N.J., March 28,
2022 /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 seven posters across three of its
pillars—ARIKAYCE, brensocatib, and treprostinil palmitil inhalation
powder (TPIP)—will be presented at the American Thoracic Society
(ATS) 2022 International Conference, taking place May 13-18, 2022, in San
Francisco.
"ATS provides us with the opportunity to present the first
real-world data on adding ARIKAYCE to the standard treatment
regimen for patients living with refractory MAC lung disease," said
Martina Flammer, M.D., M.B.A, Chief
Medical Officer of Insmed. "This year's conference will also
highlight Insmed's growing pipeline of potential therapies for
individuals living with serious and rare diseases. The wide range
of data presentations reflects our steadfast commitment to
scientific advancement that has the potential to make a significant
impact on patients' lives."
Presentations include:
- Thematic poster session, Sunday, May
15, 11:15 AM-1:15 PM PT:
A1945/P250 – Treprostinil Exerts Anti-Fibrotic Effects via the
Prostanoid Receptor Subtype EP2 in Human Lung Fibroblast
- Thematic poster session, Sunday, May 15, 11:15 AM-1:15 PM PT: A1471/P670 – Pulmonary
Exacerbations (PEx) and Hospitalizations in Commercially Insured
Patients with Non-Cystic Fibrosis Bronchiectasis (NCFBE) Over 1-
and 2-Year Follow-Up Periods
- Poster discussion session, Tuesday, May
17, 9:30-11:00 AM PT:
A3866/302 – Reduction in Hospitalizations Following Initiation of
Amikacin Liposome Inhalation Suspension: A Retrospective Cohort
Study of Patients in Real-world Settings
- Poster discussion session, Tuesday, May
17, 9:30-11:00 AM PT:
A3879/315 – The Hospitalization Burden Among Treatment-refractory
Nontuberculous Mycobacterial Lung Disease Patients in Japan
- Thematic poster session, Tuesday, May
17, 11:15 AM-1:15 PM PT:
A4613/P1002 –Administration of Treprostinil to the Basolateral
Surface but Not the Apical Surface of Human Bronchial Air-Liquid
Interface Epithelial Cells Induces Release of Prostaglandin E2
- Thematic poster session, Tuesday, May
17, 11:15 AM-1:15 PM PT:
A4612/P1001 – Binding Affinity of Treprostinil to Rat Recombinant
Prostanoid Receptors IP and EP2
- Mini symposium, Tuesday, May 17,
2:55-3:05 PM PT – A4778
– Brensocatib for the Treatment of Non-Cystic Fibrosis
Bronchiectasis (NCFBE): Number Needed to Treat (NNT) and Number
Needed to Harm (NNH)
About ARIKAYCE®
ARIKAYCE is approved in the United
States as ARIKAYCE® (amikacin liposome inhalation
suspension), in Europe as
ARIKAYCE® Liposomal 590 mg Nebuliser Dispersion, and in
Japan as ARIKAYCE®
inhalation 590 mg (amikacin sulfate inhalation drug product).
Current international treatment guidelines recommend the use of
ARIKAYCE for appropriate patients. 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, 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 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 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 to improve patient care.
About Brensocatib
Brensocatib is a small molecule, oral, reversible inhibitor of
dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the
treatment of patients with bronchiectasis and other
neutrophil-mediated diseases. DPP1 is an enzyme responsible for
activating neutrophil serine proteases (NSPs), such as neutrophil
elastase, in neutrophils when they are formed in the bone marrow.
Neutrophils are the most common type of white blood cell and play
an essential role in pathogen destruction and inflammatory
mediation. In chronic inflammatory lung diseases, neutrophils
accumulate in the airways and result in excessive active NSPs that
cause lung destruction and inflammation. Brensocatib may decrease
the damaging effects of inflammatory diseases such as
bronchiectasis by inhibiting DPP1 and its activation of NSPs.
Brensocatib is an investigational drug product that has not been
approved for any indication in any jurisdiction.
About TPIP
Treprostinil palmitil inhalation powder (TPIP) is a dry powder
formulation of treprostinil palmitil, a treprostinil prodrug
consisting of treprostinil linked by an ester bond to a 16-carbon
chain. Developed entirely in Insmed's laboratories, TPIP is a
potentially highly differentiated prostanoid being evaluated for
the treatment of patients with PAH, PH-ILD, and other rare and
serious pulmonary disorders. TPIP is administered in a
capsule-based inhalation device. TPIP is an investigational drug
product that has not been approved for any indication in any
jurisdiction.
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.
|
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 is a first-in-disease
therapy approved in the United States, Europe,
and Japan to treat a chronic, debilitating lung disease.
The Company is also progressing a robust pipeline of
investigational therapies targeting areas of serious unmet need,
including neutrophil-mediated inflammatory diseases and rare
pulmonary disorders. Insmed is headquartered in Bridgewater,
New Jersey, with a growing
footprint across Europe and in Japan. For more
information, visit https://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 obtain, or delays in obtaining, regulatory
approvals for ARIKAYCE outside the
U.S., Europe or Japan, or for the Company's product
candidates in the U.S., Europe, Japan or other
markets, including regulatory approval for the Lamira®
Nebulizer System and the drug delivery device for TPIP in each
market and for each usage; failure to successfully commercialize
ARIKAYCE, the Company's only approved product, in the
U.S., Europe or Japan (amikacin liposome
inhalation suspension, Liposomal 590 mg Nebuliser Dispersion, and
amikacin sulfate inhalation drug product, respectively), or to
maintain U.S., European or Japanese approval for ARIKAYCE; business
or economic disruptions due to catastrophes or other events,
including natural disasters or public health crises; impact of the
COVID-19 pandemic and efforts to reduce its spread on the Company's
business, employees, including key personnel, patients, partners
and suppliers; risk that brensocatib does not prove effective or
safe for patients in ongoing and future clinical studies, including
the ASPEN study; risk that TPIP does not prove to be
effective or safe for patients in ongoing and future clinical
studies; 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 U.S. Food and Drug Administration,
including the risk that the Company will not successfully or in a
timely manner complete the study to validate a PRO tool and the
confirmatory post-marketing clinical trial 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 the Company's product
candidates; inaccuracies in the Company's estimates of the size of
the potential markets for ARIKAYCE, brensocatib, TPIP or the
Company's other product candidates 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 or any of the Company's product candidates
that are approved in the future; failure to obtain regulatory
approval to expand ARIKAYCE's indication to a broader patient
population; risk that the Company's competitors may obtain orphan
drug exclusivity for a product that is essentially the same as a
product the Company is developing for a particular indication;
failure to successfully predict the time and cost of development,
regulatory approval and commercialization for novel gene therapy
products; failure to successfully conduct future clinical trials
for ARIKAYCE, brensocatib, TPIP and the Company's other product
candidates due to the Company's limited experience in conducting
preclinical development activities and clinical trials necessary
for regulatory approval and its potential inability to enroll or
retain sufficient patients to conduct and complete the trials or
generate data necessary for regulatory approval, among other
things; risks that the Company's clinical studies will be delayed
or that serious side effects will be identified during drug
development; 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 the
Company's agreements or laws and regulations that impact the
Company's business or agreements with the Company; failure to
comply with the Company's obligations in the Company's third party
agreements; the Company's inability to attract and retain key
personnel or to effectively manage the Company's growth; the
Company's inability to successfully integrate its recent
acquisitions and appropriately manage the amount of management's
time and attention devoted to integration activities; risks that
the Company's acquired technologies, products and product
candidates are not commercially successful; the Company's inability
to adapt to its highly competitive and changing environment; risk
that the Company is unable to maintain its significant customers;
risk that government healthcare reform materially increases the
Company's costs and damages its financial condition; 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 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; risk that the Company's operations are subject to a
material disruption in the event of a cybersecurity attack or
issue; business disruptions or expenses related to the upgrade to
the Company's enterprise resource planning (ERP) system; 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; the Company's history of operating losses, and the
possibility that the Company may never achieve or maintain
profitability; goodwill impairment charges affecting the Company's
results of operations and financial condition; 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 third-party
manufacturing facility approved by the appropriate regulatory
authorities 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, 2021 and any subsequent Company filings
with the Securities and Exchange Commission (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:
Eleanor Barisser
Associate Director, Investor Relations
Insmed
(718) 594-5332
eleanor.barisser@insmed.com
Media:
Mandy Fahey
Executive Director, Corporate Communications
Insmed
(732) 718-3621
amanda.fahey@insmed.com
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