BRIDGEWATER, N.J., June 8, 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 U.S. Food and Drug
Administration (FDA) has granted breakthrough therapy designation
for brensocatib (formerly known as INS1007) for the treatment of
adult patients with non-cystic fibrosis bronchiectasis (NCFBE) for
reducing exacerbations. 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.
The FDA's breakthrough therapy designation is designed to
expedite the development and review of therapies that are
intended to treat a serious or life-threatening disease and for
which preliminary clinical evidence indicates that the drug may
demonstrate substantial improvement over available therapy. The
benefits of breakthrough therapy designation include more frequent
communication and meetings with FDA, eligibility for rolling and
priority review, intensive guidance on an efficient drug
development program, and organizational commitment from the FDA
involving senior managers.
"We are very pleased that the FDA has granted breakthrough
therapy designation to brensocatib for treating patients with
NCFBE, recognizing the strength of data from our Phase 2 WILLOW
study and the potential for brensocatib to offer a novel,
first-in-class treatment approach to bronchiectasis," said
Martina Flammer, M.D., MBA, Chief
Medical Officer of Insmed. "There are currently no approved
therapies specifically targeting this severe and chronic pulmonary
disease in the United States,
Europe, or Japan. We look forward to continuing to work
with the FDA as we advance the development of brensocatib to
address this urgent medical need."
The breakthrough therapy designation for brensocatib is based on
positive results from the global randomized, double-blind,
placebo-controlled Phase 2 WILLOW study of brensocatib in adults
with NCFBE. As previously announced, full results from this study
will be presented during a virtual clinical trials session hosted
by the American Thoracic Society (ATS) on Wednesday, June 24, 2020. Insmed will hold a
conference call following the oral session during which the lead
study investigator will further discuss the WILLOW data and Insmed
management will provide a business assessment of brensocatib.
Insmed expects to initiate a Phase 3 program for brensocatib in
bronchiectasis in the second half of 2020.
Post-ATS Conference Call Details
Insmed will host a conference call on Wednesday, June 24, 2020 at 4:00 pm ET. Shareholders and other interested
parties may participate in the conference call by dialing (888)
317-6003 (domestic) or (412) 317-6061 (international) and
referencing conference ID number 9628054. The call will also be
webcast live on the company's website at www.insmed.com.
A replay of the conference call will be accessible approximately
one hour after its completion through July 8, 2020, by
dialing (877) 344-7529 (domestic) or (412) 317-0088 (international)
and referencing replay access code 10144366. A webcast of
the call will also be archived for 90 days under the Investor
Relations section of the Company's website
at www.insmed.com.
About WILLOW
WILLOW was a randomized, double-blind, placebo-controlled,
parallel-group, multi-center, multi-national, Phase 2 study to
assess the efficacy, safety and tolerability, and pharmacokinetics
of brensocatib administered once daily for 24 weeks in patients
with non-cystic fibrosis bronchiectasis (NCFBE). WILLOW was
conducted at 116 sites and enrolled 256 adult patients diagnosed
with NCFBE who had at least two documented pulmonary exacerbations
in the 12 months prior to screening. Patients were randomized 1:1:1
to receive either 10 mg or 25 mg of brensocatib or matching
placebo. The primary efficacy endpoint was the time to first
pulmonary exacerbation over the 24-week treatment period in the
brensocatib arms compared to the placebo arm.
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 Non-Cystic Fibrosis Bronchiectasis
Non-cystic fibrosis bronchiectasis (NCFBE) 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. NCFBE affects approximately 340,000 to
520,000 patients in the U.S. Today, there are no approved therapies
specifically targeting NCFBE in the U.S., Europe,
or Japan for the treatment of patients with NCFBE.
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 treprostinil
palmitil, 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 the full data set from the WILLOW study,
our six-month Phase 2 trial of brensocatib in patients with
NCFBE or data generated in further
clinical trials of brensocatib will not be consistent with the
top-line results of the study; 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; 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, our Quarterly Report on Form 10-Q for
the quarter ended March 31,
2020 and any subsequent Company filings with the 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