BRIDGEWATER, N.J., Nov. 13, 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 European Medicines Agency (EMA) has
granted Priority Medicines (PRIME) designation to brensocatib for
the treatment of non-cystic fibrosis bronchiectasis (NCFBE).
Brensocatib is a novel, first-in-class, oral, reversible inhibitor
of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the
treatment of NCFBE and other inflammatory diseases.
The EMA's PRIME designation is designed to enhance support for
the development of medicines that target unmet medical needs. To be
eligible, treatment candidates must demonstrate the potential to
offer a major therapeutic advantage over existing treatments, or
benefit patients without treatment options, based on early clinical
data. The benefits of PRIME designation include early and enhanced
interaction with the EMA to optimize development plans and the
potential for accelerated assessment. More information on PRIME
designation can be found on the EMA website at ema.europe.eu.
"We are very pleased that the EMA has recognized the potential
for brensocatib to offer an entirely new treatment approach for
NCFBE, a severe and chronic disease with significant unmet needs,"
said Martina Flammer, M.D., MBA,
Chief Medical Officer of Insmed. "We are building on the strength
of the Phase 2 WILLOW study with the initiation of a pivotal Phase
3 program for brensocatib that we hope will bring forth this
urgently needed solution."
The PRIME designation is based on positive results from the
global, randomized, double-blind, placebo-controlled Phase 2 WILLOW
study of brensocatib in adults with NCFBE. Insmed plans to initiate
the registrational Phase 3 ASPEN trial of brensocatib in patients
with NCFBE by the end of 2020. More information on this study is
available at clinicaltrials.gov (NCT04594369).
In addition to PRIME designation, brensocatib has received
Breakthrough Therapy Designation from the U.S. Food and Drug
Administration.
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
Brensocatib is a small molecule, oral, reversible inhibitor of
dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the
treatment of patients with non-cystic fibrosis bronchiectasis
(NCFBE) 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 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 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; failure to
successfully commercialize or maintain U.S. or EU approval for
ARIKAYCE, the Company's only approved product; 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
effective or safe for patients in 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 complete the confirmatory
post-marketing study 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, 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 our 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 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 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 and
June 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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