—Topline Data Remains on Track to be Reported
in Q2 of 2024—
—More than 1,700 Adult Patients Enrolled in
Global, Registrational Study of First-in-Class Treatment
Candidate—
BRIDGEWATER, N.J., March 31,
2023 /PRNewswire/ -- Insmed Incorporated
(Nasdaq:INSM), a global biopharmaceutical company on a mission to
transform the lives of patients with serious and rare diseases,
announced that adult patient enrollment is complete as of today in
its pivotal ASPEN study of
brensocatib.
ASPEN is a global, randomized,
double-blind, placebo-controlled Phase 3 study to assess the
efficacy, safety, and tolerability of brensocatib in patients ages
12 to 85 years with non-cystic fibrosis bronchiectasis. More than
1,700 adult patients have been enrolled and randomized (1:1:1) to
receive brensocatib 10 mg, brensocatib 25 mg, or placebo once daily
for 52 weeks, followed by 4 weeks off treatment. The primary
endpoint is the rate of pulmonary exacerbations over the 52-week
treatment period. Insmed anticipates reporting topline data from
adults in the ASPEN trial in the
second quarter of 2024.
"We are thrilled to have completed adult patient enrollment on
time in the ASPEN study, a
critical milestone in our efforts to develop a potentially
life-transforming therapy for people living with bronchiectasis,"
said Martina Flammer, M.D., MBA,
Chief Medical Officer of Insmed. "ASPEN is the largest single Phase 3 study ever
conducted in bronchiectasis, a serious pulmonary disease that today
has no approved treatments. We believe the pace of enrollment and
enthusiasm of the treating community reflect the significant need
for a therapy specifically developed for this disease. We are
grateful to the patients, their families, and the clinical trial
investigators for their support and commitment to advancing this
important work."
The ASPEN study is being
conducted at more than 460 sites in nearly 40 countries. The
enrollment of adolescent patients ages 12 through <18 is ongoing
and does not have an impact on planned submission timelines with
regulatory authorities. More information on the ASPEN 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. Insmed is also advancing the development of
brensocatib in additional neutrophil-driven inflammatory diseases
with significant health burdens and unmet treatment needs.
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 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, 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 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 timings 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® (amikacin liposome inhalation
suspension) outside the U.S., Europe or Japan, or for the Company's product candidates
in the U.S., Europe, Japan or other markets,
including separate regulatory approval for the
Lamira® Nebulizer System 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 or TPIP does not prove to
be effective or safe for patients in ongoing and future clinical
studies, including, for brensocatib, the ASPEN study; 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 patient reported outcome 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,
brensocatib, TPIP or the Company's other 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 risks and uncertainties associated with,
and the perceived benefits of, the Company's secured senior loan
with certain funds managed by Pharmakon Advisors, LP and the
Company's royalty financing with OrbiMed Royalty & Credit
Opportunities IV, LP, including our ability to maintain compliance
with the covenants in the agreements for the senior secured loan
and royalty financing and the perceived impact of the restrictions
on the Company's operations under these agreements; 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; 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; deterioration in general economic
conditions in the U.S., Europe,
Japan and globally, including the
effect of prolonged periods of inflation, affecting the Company,
its suppliers, third-party service providers and potential
partners; 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 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, 2022 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:
Bryan Dunn
Executive Director, Investor Relations
Insmed
(646) 812-4030
bryan.dunn@insmed.com
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
View original content to download
multimedia:https://www.prnewswire.com/news-releases/insmed-completes-enrollment-of-adult-patients-in-phase-3-aspen-study-of-brensocatib-in-bronchiectasis-301786434.html
SOURCE Insmed Incorporated