—New Graph Depicting 52-Week FEV1 Data
Illustrate Significantly Less Decline in Lung Function for
Brensocatib 25 mg Versus Placebo—
—Additional Exploratory Endpoints to be
Presented, Including FVC and Patient-Reported BEST Score—
BRIDGEWATER, N.J., July 3, 2024
/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
additional positive results from the ASPEN study, a global, randomized,
double-blind, placebo-controlled Phase 3 study to assess the
efficacy, safety, and tolerability of brensocatib in patients with
non-cystic fibrosis bronchiectasis, will be presented tomorrow,
July 4, 2024, at the 7th
World Bronchiectasis Conference (WBC) in Dundee, Scotland. Slides from this
presentation can be found here.
As previously announced, the ASPEN study met its primary endpoint, with
both dosage strengths of brensocatib achieving statistical and
clinical significance for the reduction in the annualized rate of
pulmonary exacerbations (PEs) versus placebo over the 52-week
treatment period. The annualized rate of exacerbations was 1.015
for the brensocatib 10 mg group, 1.036 for the brensocatib 25 mg
group, and 1.286 for placebo, representing a 21.1% risk reduction
from placebo for the brensocatib 10 mg group (p=0.0019) and a 19.4%
risk reduction for the 25 mg group (p=0.0046). Both dosage
strengths of brensocatib also met several secondary endpoints,
including significantly prolonging the time to first exacerbation
and significantly increasing the odds of remaining
exacerbation-free over the treatment period.
"The ASPEN findings are
critically important given that there is no approved treatment for
bronchiectasis and there remains an urgent need for a therapy that
can both reduce pulmonary exacerbations and lessen the burden of
this disease. The data announced today further underscore the
positive impact brensocatib may have on patients if approved," said
lead study investigator James
Chalmers, MBChB, Ph.D., Professor and Consultant Respiratory
Physician at the School of Medicine, University of
Dundee, UK. "Bronchiectasis is a
progressive disease that causes patients to lose lung function over
time. Therefore, I am particularly encouraged by the data which
showed that the 25 mg dose of brensocatib may slow the rate of
decline of FEV1 and FVC, which represent clinically meaningful
parameters of lung function that physicians consider important
outcome measures."
The study assessed change in lung function, as measured by
change from baseline in post-bronchodilator forced expiratory
volume over one second (FEV1) at Week 52, a key secondary endpoint.
Patients treated with brensocatib 25 mg demonstrated significantly
less FEV1 decline, preserving more lung function as compared to
placebo (LS mean change of 38 mL, p=0.0054). Patients in the
placebo arm lost on average 62 mL of FEV1 in one year. In addition,
new data will be presented at WBC measuring the change from
baseline in post-bronchodilator forced vital capacity (FVC) at Week
52, another measure of lung function and an exploratory endpoint in
the study. Patients treated with brensocatib 25 mg showed nominally
significantly less decline in FVC compared to placebo (LS mean
change of 75 mL, p<0.0001).
"We are incredibly excited to share additional results from the
ASPEN study with the
bronchiectasis community at the World Bronchiectasis Conference,
further building on the positive topline results we previously
shared," said Martina Flammer, M.D.,
MBA, Chief Medical Officer of Insmed. "Importantly, the additional
data to be presented include exploratory endpoints that further
support our belief that brensocatib may have a transformational
impact on the management of bronchiectasis. The efficacy
demonstrated in ASPEN, combined
with a favorable safety profile that was comparable to placebo,
underscore the potential for brensocatib to be used as a chronic
treatment for patients with bronchiectasis, pending approval."
Patients in both dosage groups of brensocatib experienced
numerical improvements in change from baseline in the Quality of
Life-Bronchiectasis (QOL-B) Respiratory Symptom Domain Score, with
the brensocatib 25 mg dose group demonstrating a nominally
significant improvement of 3.8 points versus placebo (p=0.0004).
Improvements in patient reported QOL-B Respiratory Symptom Domain
Score were seen as early as 4 weeks in both brensocatib arms. New
data will also be presented at WBC on the change in average daily
bronchiectasis exacerbation and symptom tool (BEST) score, an
exploratory endpoint, which is a novel symptom diary for
bronchiectasis symptom burden and detection of exacerbations.
Patients treated with brensocatib 25 mg showed a nominally
significant 1-point decrease in BEST score compared to placebo.
Brensocatib was well-tolerated in the study and demonstrated a
favorable safety profile. Treatment-emergent adverse events (TEAEs)
occurring in at least 5.0% of patients treated with either dose of
brensocatib and more frequently than in placebo were COVID-19
(15.8%, 20.9%, 15.8%), nasopharyngitis (7.7%, 6.3%, 7.6%), cough
(7.0%, 6.1%, 6.4%), and headache (6.7%, 8.5%, and 6.9%) for
brensocatib 10 mg, brensocatib 25 mg, and placebo,
respectively.
Insmed plans to file a New Drug Application with the U.S. Food
and Drug Administration for brensocatib in patients with
bronchiectasis in the fourth quarter of 2024. Pending regulatory
approvals, Insmed anticipates a U.S. launch for brensocatib in
mid-2025 followed by launches in Europe and Japan in the first half of 2026. If approved,
brensocatib would be the first approved treatment for patients with
bronchiectasis as well as the first approved dipeptidyl peptidase 1
(DPP1) inhibitor—a new mechanism of action with the potential to
address a range of neutrophil-mediated diseases.
About ASPEN
As part of the ASPEN study's
conduct, more than 460 trial sites were engaged in nearly 40
countries. After excluding sites that did not enroll any patients
and all sites in Ukraine, the
total number of active sites in ASPEN was 391 sites in 35 countries. Adult
patients (ages 18 to 85 years) were randomized 1:1:1 and adolescent
patients (ages 12 to <18 years) were randomized 2:2:1 for
treatment with brensocatib 10 mg, brensocatib 25 mg, or placebo
once daily for 52 weeks, followed by 4 weeks off treatment. The
primary efficacy analysis included data from 1,680 adult patients
and 41 adolescent patients.
About Bronchiectasis
Bronchiectasis is a serious, chronic lung disease 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 500,000 patients in the U.S.,
600,000 patients in Europe, and
150,000 patients in Japan, and
there are currently no approved therapies specifically targeting
bronchiectasis in these regions.
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, CRSsNP, 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 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 progressing a robust pipeline of investigational
therapies targeting areas of serious unmet need, including
neutrophil-mediated inflammatory diseases and rare pulmonary
disorders. Insmed is also advancing an early-stage research engine
encompassing a wide range of technologies and modalities, including
artificial intelligence-driven protein engineering, gene therapy,
and protein manufacturing. Insmed is headquartered
in Bridgewater, New Jersey, with additional offices and
research locations throughout the United States, Europe,
and Japan. Visit www.insmed.com to learn more.
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: the risk that the full data set from the ASPEN study or data generated in further
clinical trials of brensocatib will not be consistent with the
topline results of the ASPEN study
or any additional results of the ASPEN study; failure to obtain, or delays in
obtaining, regulatory approvals for brensocatib in the U.S.,
Europe or Japan; failure to successfully commercialize
brensocatib, if approved by applicable regulatory authorities, in
the U.S., Europe or Japan, or to maintain U.S., European or
Japanese approval for brensocatib once approved; uncertainties in
the degree of market acceptance of brensocatib by physicians,
patients, third-party payors and others in the healthcare
community; inaccuracies in the Company's estimates of the size of
the potential markets for 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; inability of the Company, Esteve
Pharmaceuticals, S.A., Thermo Fisher Scientific, Inc. or the
Company's other third-party manufacturers to comply with regulatory
requirements related to brensocatib; the Company's inability to
obtain adequate reimbursement from government or third-party payors
for brensocatib or acceptable prices for brensocatib; development
of unexpected safety or efficacy concerns related to brensocatib;
failure to obtain regulatory approval for potential future
brensocatib indications; restrictions or other obligations imposed
on us by agreements related to brensocatib, including our license
agreement with AstraZeneca AB, and failure to comply with our
obligations under such agreements; failure to successfully conduct
future clinical trials for brensocatib, including due to the
Company's 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 brensocatib 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 strength and
enforceability of the Company's intellectual property rights or the
rights of third parties; the cost and potential reputational damage
resulting from litigation to which the Company may become a party,
including product liability claims; changes in laws and regulations
applicable to the Company's business and failure to comply with
such laws and regulations; business or economic disruptions due to
catastrophes or other events, including natural disasters or public
health crises; and inability to repay the Company's existing
indebtedness and uncertainties with respect to the Company's need
and ability to access future capital.
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, 2023 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/additional-positive-data-from-pivotal-aspen-study-of-brensocatib-in-patients-with-bronchiectasis-to-be-presented-at-the-7th-world-bronchiectasis-conference-302188927.html
SOURCE Insmed Incorporated