BRIDGEWATER, N.J., Sept. 30, 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 progress across its portfolio of
therapies and pipeline candidates, which will be discussed in
further detail today at the Company's virtual Research &
Development (R&D) Day. The event will feature presentations
from the Insmed management team and Ronald
J. Oudiz, M.D., FACP, FACC, FCCP, Professor of Medicine at
the David Geffen School of Medicine at the University of California Los Angeles (UCLA), the Interim Chief, Division of Cardiology,
and a Director at the Liu Center for Pulmonary Hypertension at the
Lundquist Institute for Biomedical Innovation at Harbor-UCLA
Medical Center, and the lead investigator in the Company's planned
Phase 2a trial for treprostinil palmitil inhalation powder
(TPIP).
"Our success in building a commercial company with a global
footprint and strong pipeline sets the stage for Insmed's next
chapter of growth," said Will Lewis,
Chair and Chief Executive Officer of Insmed. "With ARIKAYCE, our
global expansion efforts and upcoming study in front-line NTM
patients has the potential to establish this first-in-disease
program as the standard of care in NTM lung disease worldwide. We
believe brensocatib, our DPP1 inhibitor, offers an entirely new
approach to address a wide array of neutrophil-mediated diseases.
We plan to build upon our early leadership position in this space
with our anticipated near-term registrational study in NCFBE,
followed by a new development program in cystic fibrosis and plans
to explore other opportunities across the neutrophil-mediated
disease landscape. We are also pleased to announce the initiation
of a Phase 1 trial of TPIP, which represents an exciting
opportunity to more fully harness the potential of the prostanoid
pathway for the treatment of pulmonary arterial hypertension and
may offer a disease-modifying effect. We are well capitalized to
execute against our strategic objectives during this next phase of
growth for Insmed."
Select R&D Day Highlights
Initiated Phase 1 Trial for Treprostinil Palmitil Inhalation
Powder
The Company announced today that it has initiated dosing of the
first subjects in the Phase 1 healthy volunteer trial of TPIP in
the United States. The objective
of this first-in-human single ascending dose and multiple ascending
dose study is to assess the pharmacokinetics and tolerability
profile of TPIP. Top-line data are expected in the first quarter of
2021. The Company also announced plans to initiate a Phase 2a study
in patients with pulmonary arterial hypertension (PAH) in early
2021.
Today, Dr. Oudiz will provide a review of the PAH treatment
landscape and the remaining unmet medical need. Additionally, the
Company will provide an update on TPIP and review key highlights of
preclinical data supporting the product candidate's potentially
differentiated product profile that could prove to be an
advantageous addition to the therapeutic arsenal for the treatment
of patients with PAH.
Treprostinil palmitil is a prodrug consisting of treprostinil
linked by an ester bond to a 16-carbon chain. The prodrug becomes
active when esterases cleave off the 16-carbon chain, resulting in
the active molecule treprostinil. TPIP is formulated as a dry
powder for administration in a capsule-based inhalation device.
Building a Leading Neutrophil-Mediated Portfolio with
Brensocatib
Insmed will outline its plans to leverage the potential of
brensocatib, its small molecule, oral, reversible inhibitor of
dipeptidyl peptidase I (DPP1), in an array of neutrophil-mediated
diseases, beginning with NCFBE. The Company will discuss the study
design for its registrational Phase 3 trial of brensocatib for the
treatment of NCFBE. The trial, known as ASPEN, was developed with input from both the
U.S. Food and Drug Administration (FDA) and the European Medicines
Agency (EMA). Insmed remains on track to launch the ASPEN trial in the fourth quarter of 2020.
The Company also announced plans to advance a clinical
development program for brensocatib in cystic fibrosis (CF) and to
discuss this program with health authorities in the first half of
2021.
Global Expansion for ARIKAYCE® (Amikacin Liposome
Inhalation Suspension)
ARIKAYCE is the first and only FDA-approved therapy for the
treatment of Mycobacterium avium complex (MAC)
lung disease as part of a combination antibacterial drug regimen
for adult patients with limited or no alternative treatment
options. Today the Company will provide an update on its
global expansion efforts in Europe
and Japan. Insmed recently
received a positive opinion from the Committee for Medicinal
Products for Human Use of the EMA for the treatment of
nontuberculous mycobacterial (NTM) lung infections caused by MAC in
adults with limited treatment options who do not have CF. The
Company anticipates receiving Marketing Authorization in the
European Union by the end of October, with a planned first European
launch by end of 2020. In addition, Insmed filed a Japanese New
Drug Application (JNDA) with the Japanese Ministry of Health,
Labour and Welfare in March of this year. Following an anticipated
12-month review, if ARIKAYCE is approved, the Company would
anticipate launching ARIKAYCE in Japan in the middle of 2021.
Additionally, the Company will provide details around its
planned registration program to pursue regulatory approval of
ARIKAYCE as a front-line therapy designed to establish a new
standard of care for patients suffering from NTM lung disease. The
clinical study design includes two separate but inter-related
clinical trials to be conducted in parallel. ENCORE is the pivotal
study intended to fulfill the post-marketing requirement to allow
full approval of ARIKAYCE in the United
States. The ARISE trial is an interventional study designed
to validate the patient-reported outcome (PRO) tool that will be
used to measure efficacy in ENCORE. The Company plans to initiate
both trials globally in the fourth quarter of 2020.
Financial Update
As of June 30, 2020, Insmed had
cash and cash equivalents of $641.9M.
As of today, the Company expects its cash runway to extend over
three years based on currently planned development programs.
Conference Call
The R&D Day is scheduled to take place today from
10:00 a.m. to 12:30 p.m. ET in a
virtual format. The call and accompanying slides will be webcast
live on the Company's website at
https://investor.insmed.com/events. To listen to the conference
call live, please dial (888) 317-6003 (domestic) or (412) 317-6061
(international) and reference conference ID number 0587949.
A replay of the conference call will be accessible approximately
on hour after its completion through October
14, 2020 by dialing (877) 344-7529 (domestic) or (412)
317-0088 (international) and referencing replay access code
10147009. A webcast of the call will also be archived for 90 days
under the Investor Relations section of the Company's website at
https://investor.insmed.com/events.
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 inhalation powder, a treprostinil prodrug
formulated as a dry powder for inhalation, which 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: 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 to successfully
commercialize or maintain U.S. 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; 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 is 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 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 its
inability to enroll or retain sufficient patients to conduct and
complete the trials or generate data necessary for regulatory
approval; 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 FDA-approved
third-party manufacturing facility and unexpected expenses
associated with those plan.
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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SOURCE Insmed Incorporated