BRIDGEWATER, N.J., Dec. 2, 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 the first patient has been dosed in the Phase
3 ASPEN study of brensocatib in patients with bronchiectasis.
Brensocatib is a novel, first-in-class, oral, reversible inhibitor
of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the
treatment of bronchiectasis and other inflammatory diseases.
"There is an urgent need for an approved therapy for the
treatment of bronchiectasis that has the potential to reduce
exacerbations—the major driver of morbidity and mortality in these
patients," said Martina Flammer,
M.D., MBA, Chief Medical Officer of Insmed. "Building on the
strength of data from our Phase 2 WILLOW study, which showed that
treatment with brensocatib both prolonged the time to first
exacerbation and reduced the risk of exacerbation over the 24-week
treatment period, we are very pleased to initiate the
registrational ASPEN trial. We
believe this is a significant step toward enabling regulatory
submissions and potentially bringing brensocatib to patients in
need."
ASPEN is a global, randomized,
double-blind, placebo-controlled Phase 3 study to assess the
efficacy, safety, and tolerability of brensocatib in patients with
bronchiectasis. Patients with bronchiectasis due to cystic fibrosis
may not be enrolled in the study. Patients will be randomized to
receive brensocatib 10 mg, brensocatib 25 mg, or placebo once daily
for 52 weeks. The primary endpoint is the rate of pulmonary
exacerbations over the 52-week treatment period. Secondary
endpoints include time to first pulmonary exacerbation, percentage
of subjects who remain pulmonary exacerbation-free, change from
baseline in post-bronchodilator FEV1, rate of severe pulmonary
exacerbations, change from baseline in the Bronchiectasis (QOL-B)
Respiratory Symptoms Domain Score, and incidence and severity of
treatment-emergent adverse events.
The study is expected to enroll approximately 1,620 patients
(540 in each arm) at approximately 480 sites in 40 countries. More
information on this study is available at clinicaltrials.gov
(NCT04594369).
Brensocatib has received Breakthrough Therapy Designation from
the U.S. Food and Drug Administration as well as Priority Medicines
(PRIME) designation from the European Medicines agency for patients
with bronchiectasis.
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.
About Bronchiectasis
Bronchiectasis is a severe, chronic pulmonary disorder in which
the bronchi become permanently dilated due to a cycle of infection,
inflammation, and lung tissue damage. The condition is marked by
frequent pulmonary exacerbations requiring antibiotic therapy
and/or hospitalizations. Symptoms include chronic cough, excessive
sputum production, shortness of breath, and repeated respiratory
infections, which can worsen the underlying condition.
Bronchiectasis affects approximately 340,000 to 520,000 patients in
the U.S. Today, there are no approved therapies specifically
targeting bronchiectasis in the U.S., Europe,
or Japan.
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 and the European Union 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
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 obtain, or delays in obtaining, regulatory
approvals for ARIKAYCE outside the U.S. or European Union or for
the Company's product candidates in the
U.S., Europe, Japan or other markets, including the
United Kingdom as a result of the
United Kingdom's exit from the
European Union; failure to successfully commercialize ARIKAYCE, the
Company's only approved product, in the
United States or European Union (amikacin liposome
inhalation suspension and Liposomal 590 mg Nebuliser Dispersion,
respectively), or to maintain U.S. or EU approval for ARIKAYCE;
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 the Company's business, employees, including key
personnel, patients, partners and suppliers; the risk that
brensocatib does not prove to be effective or safe for patients in
ongoing and future clinical studies, including the ASPEN and STOP-COVID19 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 FDA, including the risk that the Company will not
timely and successfully 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 Pharma
GmbH (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 or its
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; failure to successfully
conduct future clinical trials for ARIKAYCE, brensocatib,
treprostinil palmitil inhalation powder (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; 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 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, 2019, the Company's Quarterly Reports on
Form 10-Q for the quarters ended March 31, 2020, June 30, 2020 and September 30, 2020, 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:
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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SOURCE Insmed Incorporated