BRIDGEWATER, N.J., April 23, 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 it will provide funding and clinical
drug supply for the STOP-COVID19 (Superiority Trial
of Protease inhibition in COVID-19) trial, an
investigator-initiated study of brensocatib (formerly known as
INS1007) in up to 300 hospitalized patients with COVID-19 sponsored
by the University of Dundee. The study,
which has been prioritized and designated an Urgent Public Health
trial by the UK's National Institute for Health Research, is
expected to begin enrollment in May
2020.
Brensocatib is a novel oral, reversible inhibitor of dipeptidyl
peptidase 1 (DPP1) currently being developed by Insmed for the
treatment of bronchiectasis and other inflammatory diseases. DPP1
is an enzyme that catalyzes the activation of neutrophil serine
proteases (NSPs) 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. By inhibiting the activation of NSPs, brensocatib may
offer applicability in a range of neutrophil-mediated diseases.
Neutrophil influx into the lungs is a defining characteristic of
acute respiratory distress syndrome (ARDS), a severe outcome of
COVID-19 that is associated with high mortality. Reduction of
neutrophil proteases may reduce the progression of lung injury and
the need for ventilation in these patients.
"The global COVID-19 pandemic has generated an extraordinary
response from the biopharmaceutical industry to bring to bear all
potential means of fighting this disease and preventing its most
severe outcomes, including the need for ventilation and ICU stays,"
said Martina Flammer, M.D., Chief
Medical Officer of Insmed. "At the start of the outbreak, Insmed
began pursuing an in vivo mouse model to better understand
the potential of brensocatib in preventing ARDS. As we rapidly
advance this early-stage research simultaneously, we are very
pleased to support Professor James
Chalmers and the University of
Dundee in leading a controlled clinical trial that will help
us evaluate the potential impact of brensocatib on hospitalized
patients suffering from severe COVID-19."
The STOP-COVID19 trial is a prospective, randomized,
double-blind, placebo-controlled trial of brensocatib in patients
with severe COVID-19. The multicenter study is expected to enroll
up to 300 patients at 10 sites in the UK who present to the
hospital with confirmed COVID-19 and are at risk of needing
increased levels of supplemental oxygen and/or ventilation.
Patients will be randomized 1:1 to receive either brensocatib 25 mg
once daily or matching placebo on top of standard of care. The
primary endpoint is clinical improvement on a seven-point ordinal
scale as defined by the World Health Organization. Patients will be
treated for 28 days, with a sample-size reassessment performed once
100 patients have been enrolled and treated.
"The medical community has never faced a more urgent need for
treatment than the unprecedented situation we face today with
COVID-19," said lead study investigator James Chalmers, MBChB, Ph.D., Professor and
Consultant Respiratory Physician at the School of
Medicine, University of Dundee,
UK. "The mechanism of action of brensocatib that we observed
in a Phase 2 study in patients with non-cystic fibrosis
bronchiectasis provides a strong rationale for evaluating this
novel treatment candidate in other neutrophil-driven inflammatory
conditions. It is my hope that this novel approach will have
applicability in patients at risk of ARDS—a devastating outcome of
COVID-19 for which there are currently no approved therapies."
In February 2020, Insmed reported
positive top-line results from the global randomized, double-blind
placebo-controlled Phase 2 WILLOW study evaluating the efficacy,
safety, and pharmacokinetics of brensocatib in adults with
non-cystic fibrosis bronchiectasis. In this study of more than 250
patients, brensocatib was generally well-tolerated. The study met
both its primary and key secondary endpoint.
Insmed will continue to develop brensocatib in patients with
bronchiectasis and expects to begin enrollment in a Phase 3
program in the second half of 2020 following anticipated
discussions later this year with health authorities on the design
of the program.
About Brensocatib (Formerly INS1007)
Brensocatib is a small molecule, oral, reversible inhibitor of
dipeptidyl peptidase I (DPP1) being developed by Insmed for the
treatment of patients with bronchiectasis. 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 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 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.
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: 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; the risk that the full data set from the WILLOW
study, our six-month Phase 2 trial of brensocatib in patients with
NCBFE or data generated in further clinical trials of brensocatib
will not be consistent with the top-line results of the study;
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 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 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:
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