BRIDGEWATER, N.J., Feb. 19, 2021 /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 topline results from the Phase 1 study of
treprostinil palmitil inhalation powder (TPIP) in healthy
volunteers. Data from the study demonstrated that TPIP was
generally safe and well tolerated, with a pharmacokinetic profile
that supports once-daily dosing. A conference call will be held
today, February 19, 2021, at
8:30 am ET, with Insmed management to
further discuss these results and provide an update on the planned
development pathway for TPIP.
"We are very pleased to share these encouraging Phase 1 results,
which we believe validate several critical aspects of the TPIP
profile and continue to build on the momentum of our earlier
preclinical work," said Martina
Flammer, M.D., MBA, Chief Medical Officer of Insmed.
"Importantly, these findings support the continued development of
TPIP with once-daily dosing in a clinical trial program for
patients with pulmonary arterial hypertension (PAH). This is a
serious, progressive, and rare disease in which the current
standard of care is limited by tolerability issues and a cumbersome
dosing regimen."
The Phase 1 study of 42 healthy volunteers was designed to
assess the safety, tolerability, and pharmacokinetics of TPIP in
the setting of single-dose and multiple-dose administration. The
highest dose tested as a single dose was 675 µg, and the highest
dose tested in repeated dosing was 225 µg.
The study demonstrated that TPIP was generally safe and well
tolerated. The most common adverse events (AEs) across all
cohorts in the study were cough, dizziness, headache, and nausea.
Most AEs were mild in severity and consistent in nature with those
typically seen with other inhaled prostanoid therapies. There were
few moderate AEs and no severe or serious AEs. Subjects in the
multiple dose panel that incorporated an up-titration approach
beginning at 112.5 µg once-daily and progressing to 225 µg
once-daily reported fewer AEs compared to the panel dosed with 225
µg once-daily from the first dose.
Overall pharmacokinetic results demonstrated that treprostinil
exposure (AUC and Cmax) was dose-proportional, with low
to moderate inter-subject variability. Treprostinil was detected in
the plasma at 24 hours at all doses and throughout the 48-hour
sampling period for the two highest doses. Compared with currently
available inhaled treprostinil therapy, TPIP showed substantially
lower Cmax and longer half-life.
Insmed plans to present full data from this study at an upcoming
medical meeting.
"The positive results from this Phase 1 study provide clear
support for advancing TPIP to the next stage of clinical
development in PAH as well as exploring its potential in other
serious pulmonary disorders," said Will
Lewis, Chair and CEO of Insmed. "These findings represent a
significant step toward unlocking the full potential of prostanoid
therapy. With continued development of TPIP, we look forward to
evaluating whether this novel treatment candidate may offer the
potential for improved tolerability, dosing convenience, and
efficacy for patients with PAH."
Insmed plans to advance the development of TPIP with two studies
in patients with PAH. The first is an open-label,
proof-of-mechanism study to understand the impact of TPIP on
pulmonary vascular resistance (PVR) over a 24-hour period. The
Company anticipates sharing topline data from this study in the
second half of 2021. The second will aim to investigate the effect
of TPIP on PVR and 6-minute walk distance over a 16-week treatment
period using an up-titration, once-daily dosing schedule. The
Company plans to initiate this trial in the fourth quarter of
2021.
Beyond PAH, Insmed continues to explore potential development
pathways for TPIP in patients with pulmonary hypertension
associated with interstitial lung disease (PH-ILD) and idiopathic
pulmonary fibrosis (IPF), and plans to initiate a study in patients
with PH-ILD using an up-titration, once-daily dosing schedule.
About the TPIP Phase 1 Study
The Phase 1 study was intended to assess the safety,
tolerability, and pharmacokinetics of TPIP in healthy volunteer
subjects in the setting of single-dose and seven-day, multiple-dose
administration. Serial cohorts of subjects were enrolled. In the
first panel, subjects were randomized to receive single doses of
112.5 µg, 225 µg, or 450 µg of TPIP. In the next panel, subjects
were randomized to receive single doses of 675 µg of TPIP or
placebo.
In the next panel, which was the first multiple-dose panel,
subjects were randomized to receive 225 µg of TPIP once-daily for
seven days, or matching placebo. The final cohort incorporated a
placebo-controlled, up-titration approach in which subjects began
at 112.5 µg once-daily for four days, then advanced to 225 µg
once-daily for three days.
Conference Call
Insmed will host a conference call today beginning at
8:30 a.m. ET. Shareholders and other
interested parties may participate in the conference call by
dialing (833) 340-0284 (domestic) or (236) 712-2425 (international)
and referencing conference ID number 1963113. 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
two hours after its completion through March
21, 2021 by dialing (800) 585-8367 (domestic) or (416)
621-4642 (international) and referencing conference ID number
1963113. 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 TPIP
Treprostinil palmitil inhalation powder (TPIP) is a dry powder
formulation of treprostinil palmitil, a treprostinil prodrug
consisting of treprostinil linked by an ester bond to a 16-carbon
chain. Developed entirely in Insmed's laboratories, TPIP is a
potentially highly differentiated prostanoid being evaluated for
the treatment of patients with PAH and other rare and serious
pulmonary disorders. TPIP is administered in a capsule-based
inhalation device. TPIP 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 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 (EU),
including the United Kingdom as a
result of the United Kingdom's
exit from the EU, or for the Company's product candidates in the
U.S., Europe, Japan or other markets; failure to
successfully commercialize ARIKAYCE, the Company's only approved
product, in the U.S. or the EU (amikacin liposome inhalation
suspension and Liposomal 590 mg Nebuliser Dispersion,
respectively), or to maintain U.S. or EU approval for ARIKAYCE;
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 to be effective or safe for patients in
ongoing and future clinical studies, including the ASPEN study; the risk that TPIP does not prove
to be effective or safe for patients in ongoing and future clinical
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 U.S. Food and Drug Administration,
including the risk that the Company will not timely and
successfully 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 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 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 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 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 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, 2019, the Company's
Quarterly Reports on Form 10-Q for the quarters ended March 31, 2020, June 30,
2020 and September 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:
Eleanor Barisser
Associate Director, Investor Relations
Insmed
eleanor.barisser@insmed.com
Media:
Mandy Fahey
Senior Director, Corporate Communications
Insmed
(732) 487-7468
amanda.fahey@insmed.com
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SOURCE Insmed Incorporated