—Topline Data from the Phase 3 ASPEN Trial of
Brensocatib in Adult Patients with Bronchiectasis Remain on Track
to Read Out in the Latter Part of Second-Quarter 2024—
—Enrollment in the Phase 2 Study of TPIP in
Patients with PH-ILD Completed in November
2023; Topline Data Expected in Second-Quarter 2024 Ahead of
the ASPEN Readout—
—Company Ends 2023 With $780 Million of Cash, Cash Equivalents, and
Marketable Securities, Providing Runway Beyond the Expected
ASPEN Readout—
— ARIKAYCE® (amikacin liposome
inhalation suspension) Total Revenue of $83.7 Million for Fourth-Quarter and $305.2 Million for Full-Year 2023, Reflecting 24%
Annual Growth and Exceeding the Upper End of Full-Year 2023
Guidance Range—
—Company Reiterates Sales Guidance for
2024 Global ARIKAYCE Revenues in the Range of $340 Million to $360
Million, Reflecting Double-Digit Growth Compared to
2023—
BRIDGEWATER, N.J., Feb. 22,
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 reported financial results for the fourth quarter and full
year ended December 31, 2023 and
provided a business update.
"Insmed continued to deliver strong performance in 2023,
demonstrating commercial success evidenced by ARIKAYCE revenues
that exceeded the upper end of our 2023 sales guidance range," said
Will Lewis, Chair and Chief
Executive Officer of Insmed. "The positive ARISE data announced in
September 2023, followed by
encouraging blinded TPIP data shortly thereafter, marked the
beginning of a transformative period of clinical catalysts for the
Company. This series of meaningful data readouts from our mid- to
late-stage pipeline is expected to continue uninterrupted in the
months ahead, with topline results from the PH-ILD and ASPEN trials expected in quick succession in
the second quarter. We believe these near-term data readouts have
the potential to fundamentally change the trajectory for our
company and the patients we serve."
Recent Pillar Highlights
Pillar 1: ARIKAYCE
- ARIKAYCE global revenue grew 24% in 2023 compared to 2022,
reflecting continued strong growth in the U.S., Japan, and Europe.
- Insmed received encouraging written feedback in December 2023 from the U.S. Food and Drug
Administration (FDA) on the patient-reported outcome data produced
in the Phase 3 ARISE study. The Company expects to meet with the
FDA in the coming months to gain additional insights and guidance,
from which it will finalize its statistical plan for the Phase 3
ENCORE study.
- As previously communicated in January
2024, the Company achieved its original target enrollment
goal of 250 patients in the ENCORE trial in patients with newly
diagnosed or recurrent nontuberculous mycobacterial lung infection
caused by Mycobacterium avium complex (MAC) who had not
started antibiotics. Enrollment in the trial remains open.
- The Data Safety Monitoring Committee for the ENCORE study held
its third safety review meeting in November
2023 and recommended that the study continue as
planned.
- Consistent with the Company's expectations, the Pharmaceuticals
and Medical Devices Agency (PMDA) in Japan recently confirmed that it would not
consider a label expansion for ARIKAYCE based on data from the
ARISE study alone due to (i) the lack of Japanese subjects in that
study; (ii) the absence of what PMDA considers a sufficiently long
treatment exposure (12 months); and (iii) the absence of resulting
evidence of culture conversion the PMDA considers durable (15
months). The ongoing ENCORE trial is designed to satisfy the PMDA's
remaining regulatory requirements, including the enrollment of
Japanese patients, sufficient treatment exposure, and an endpoint
for durable culture conversion.
- The Company continues to expect topline data for ENCORE in
2025.
Pillar 2: Brensocatib
- Insmed continues to expect topline data from the Phase 3 ASPEN
study of brensocatib in patients with non-cystic fibrosis
bronchiectasis in the latter part of the second quarter of
2024.
- If ASPEN is successful and
regulatory approval is obtained, the Company anticipates a launch
in bronchiectasis in the U.S. in mid-2025, followed by launches in
Europe and Japan in the first half of 2026. Insmed
continues to advance its launch readiness activities in preparation
for these potential launches.
- The Data Safety Monitoring Committee for the ASPEN study held its fifth and final meeting
in November 2023. No safety signals
were identified, and the Committee recommended that the trial
continue as planned.
- The Company is currently enrolling patients in the Phase
2b BiRCh trial of brensocatib in
patients with chronic rhinosinusitis without nasal polyps
(CRSsNP).
- The Company expects to initiate a Phase 2 study of brensocatib
in patients with hidradenitis suppurativa (HS) in the second half
of 2024, pending positive results from the ASPEN study.
Pillar 3: TPIP
- In October 2023, Insmed announced
encouraging blended and blinded data from two ongoing Phase 2
studies of treprostinil palmitil inhalation powder (TPIP) in
pulmonary hypertension associated with interstitial lung disease
(PH-ILD) and pulmonary arterial hypertension (PAH).
- The Company completed enrollment of 39 patients in the Phase 2
safety study in PH-ILD in November
2023, exceeding its initial enrollment target of 32
patients. Topline data from the study are anticipated in advance of
Phase 3 ASPEN data in the second quarter of 2024.
- Enrollment remains ongoing in the Phase 2 study in PAH. Insmed
anticipates sharing updated blinded data from approximately 40
patients in the PAH study at the same time topline results
from the PH-ILD study become available in the second quarter of
2024. Topline results from the Phase 2 PAH study continue to be
expected in 2025.
- Insmed has submitted a protocol amendment to the FDA and other
regulatory authorities for the open-label extension of the Phase 2
PAH study. This amendment, if approved, would allow investigators
to continue to increase the dose of TPIP up to a maximum of 1,280
micrograms once daily.
Pillar 4: Early-Stage Research
- Insmed's early-stage research efforts include more than 30
identified pre-clinical programs in development, all of which have
the potential to become first-in-class or best-in-class
therapies.
- The Company continues to anticipate the totality of its
early-stage research programs will comprise less than 20% of
overall annual spend.
Fourth-Quarter and Full-Year 2023 Financial Results
- Total revenue for the fourth quarter ended December 31, 2023, was $83.7 million, reflecting 41% growth compared to
total revenue of $59.3 million for
the fourth quarter of 2022. Total revenue for the full-year 2023
was $305.2 million, compared to total
revenue of $245.4 million for the
full-year 2022, reflecting 24% year-over-year growth.
- Total revenue for the full-year 2023 was comprised of ARIKAYCE
net sales of $224.2 million in the
U.S., $65.7 million in Japan, and $15.3
million in Europe and rest
of world. Full-year 2023 sales demonstrated year-over-year growth
of 21% in the U.S. and 16% in Japan, reflecting continued strong growth
trends for ARIKAYCE in these regions.
- Cost of product revenues (excluding amortization of
intangibles) was $18.4 million for
the fourth quarter of 2023, compared to $13.1 million for the fourth quarter of 2022,
primarily reflecting the increase in sales volumes. For the
full-year 2023, cost of product revenues (excluding amortization of
intangibles) was $65.6 million
compared to $55.1 million for the
full-year 2022.
- Research and development (R&D) expenses were $137.0 million for the fourth quarter of 2023,
compared to $124.8 million for the
fourth quarter of 2022. For the full-year 2023, R&D expenses
were $571.0 million compared to
$397.5 million in 2022, reflecting
one-time, non-cash asset acquisition costs and the continued
investment in Insmed's early and mid- to late-stage pipeline
programs, including increases in headcount to support those
existing and acquired programs.
- Selling, general and administrative (SG&A) expenses for the
fourth quarter of 2023 were $89.5
million, compared to $73.5
million for the fourth quarter of 2022. For the full-year
2023, SG&A expenses were $344.5
million, compared to $265.8
million for the full-year 2022. The year-over-year increase
in SG&A expenses resulted primarily from commercial readiness
activities for brensocatib and an increase in headcount.
- For the fourth-quarter 2023, Insmed reported a net loss of
$186.1 million, or $1.28 per share, compared to a net loss of
$160.1 million, or $1.21 per share, for the fourth-quarter 2022. For
the full-year 2023, Insmed reported a net loss of $749.6 million, or $5.34 per share, compared to a net loss of
$481.5 million, or $3.91 per share, for the full-year 2022.
Balance Sheet, Financial Guidance, and Planned
Investments
- As of December 31, 2023, Insmed
had cash, cash equivalents, and marketable securities totaling
$780.4 million.
- Insmed is reiterating its sales guidance for full-year 2024
global ARIKAYCE revenues in the range of $340 million to $360
million, representing 15% year-over-year growth at the
midpoint compared to 2023.
- Insmed continues to anticipate that over 80% of total annual
expenditures will be on its mid- to late-stage and commercial
programs (ARIKAYCE, brensocatib, and TPIP), and that less than 20%
of overall spend will be on its early-stage research programs,
reflecting the Company's historical approach to spending.
- The Company plans to continue to invest in the following key
activities in 2024:
(i) commercialization and expansion of ARIKAYCE
globally;
(ii) advancement of brensocatib, including the
Phase 3 ASPEN study in patients with bronchiectasis, and commercial
launch readiness activities, the ongoing Phase 2 trial in patients
with CRSsNP, and the Phase 2 program in HS to be initiated in the
second half of the year if the ASPEN result is positive;
(iii) advancement of the clinical trial program
for ARIKAYCE, which is intended to satisfy the post-marketing
requirement for full approval of its current indication and
potentially support label expansion to include all patients with a
MAC lung infection;
(iv) advancement of its Phase 2 clinical
development programs for TPIP; and
(v) development of its early-stage research
programs.
Conference Call
Insmed will host a conference call beginning today
at 8:30 AM Eastern Time. Shareholders and other interested
parties may participate in the conference call by dialing (888)
210-2654 (U.S. and international) and referencing access code
7862189. 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
1 hour after its completion through March
23, 2024, by dialing (800) 770-2030 (U.S. and international)
and referencing access code 7862189. 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.
INSMED INCORPORATED
|
|
Consolidated Statements of Net
Loss
|
|
(in thousands, except per share
data)
|
|
(unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
December 31,
|
|
Twelve Months Ended
December 31,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
|
|
|
|
|
|
Product revenues,
net
|
$ 83,693
|
|
$ 59,300
|
|
$
305,208
|
|
$
245,358
|
|
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Cost of product
revenues (excluding amortization of intangible assets)
|
18,443
|
|
13,069
|
|
65,573
|
|
55,126
|
|
Research and
development
|
137,029
|
|
124,763
|
|
571,011
|
|
397,518
|
|
Selling, general and
administrative
|
89,530
|
|
73,479
|
|
344,501
|
|
265,784
|
|
Amortization of
intangible assets
|
1,263
|
|
1,264
|
|
5,052
|
|
5,053
|
|
Change in fair value
of deferred and contingent consideration liabilities
|
15,700
|
|
(1,800)
|
|
28,697
|
|
(20,802)
|
|
Total operating
expenses
|
261,965
|
|
210,775
|
|
1,014,834
|
|
702,679
|
|
|
|
|
|
|
|
|
|
|
Operating
loss
|
(178,272)
|
|
(151,475)
|
|
(709,626)
|
|
(457,321)
|
|
|
|
|
|
|
|
|
|
|
Investment
income
|
9,853
|
|
8,318
|
|
42,132
|
|
11,081
|
|
Interest
expense
|
(20,784)
|
|
(16,445)
|
|
(81,694)
|
|
(26,446)
|
|
Change in fair value of
interest rate swap
|
1,970
|
|
(1,526)
|
|
320
|
|
(1,526)
|
|
Other income (expense),
net
|
2,170
|
|
1,130
|
|
1,856
|
|
(5,939)
|
|
Loss before income
taxes
|
(185,063)
|
|
(159,998)
|
|
(747,012)
|
|
(480,151)
|
|
|
|
|
|
|
|
|
|
|
Provision for income
taxes
|
998
|
|
125
|
|
2,555
|
|
1,383
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
$
(186,061)
|
|
$
(160,123)
|
|
$
(749,567)
|
|
$
(481,534)
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
$
(1.28)
|
|
$
(1.21)
|
|
$
(5.34)
|
|
$
(3.91)
|
|
|
|
|
|
|
|
|
|
|
Weighted average basic
and diluted common shares outstanding
|
144,806
|
|
132,694
|
|
140,433
|
|
123,035
|
|
|
|
|
|
|
|
|
|
|
INSMED INCORPORATED
|
Consolidated Balance Sheets
|
(in thousands, except par value and share
data)
|
|
|
|
|
|
|
|
As of
|
|
As of
|
|
|
December 31, 2023
|
|
December 31, 2022
|
|
|
|
|
|
Assets
|
|
|
|
|
Current
assets:
|
|
|
|
|
Cash and cash
equivalents
|
|
$
482,374
|
|
$
1,074,036
|
Marketable
securities
|
|
298,073
|
|
74,244
|
Accounts
receivable
|
|
41,189
|
|
29,713
|
Inventory
|
|
83,248
|
|
69,922
|
Prepaid expenses and
other current assets
|
|
24,179
|
|
25,468
|
Total current
assets
|
|
929,063
|
|
1,273,383
|
|
|
|
|
|
Fixed assets,
net
|
|
65,384
|
|
56,491
|
Finance lease
right-of-use assets
|
|
20,985
|
|
23,697
|
Operating lease
right-of-use assets
|
|
18,017
|
|
21,894
|
Intangibles,
net
|
|
63,704
|
|
68,756
|
Goodwill
|
|
136,110
|
|
136,110
|
Other assets
|
|
96,574
|
|
76,104
|
Total
assets
|
|
$
1,329,837
|
|
$
1,656,435
|
|
|
|
|
|
Liabilities and shareholders'
equity
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
Accounts payable and
accrued liabilities
|
|
$
214,987
|
|
$
182,117
|
Finance lease
liabilities
|
|
2,610
|
|
1,217
|
Operating lease
liabilities
|
|
8,032
|
|
6,909
|
Total current
liabilities
|
|
225,629
|
|
190,243
|
|
|
|
|
|
Debt,
long-term
|
|
1,155,313
|
|
1,125,250
|
Royalty financing
agreement
|
|
155,034
|
|
148,015
|
Contingent
consideration
|
|
84,600
|
|
51,100
|
Finance lease
liabilities, long-term
|
|
27,026
|
|
29,636
|
Operating lease
liabilities, long-term
|
|
11,013
|
|
14,853
|
Other long-term
liabilities
|
|
3,145
|
|
9,387
|
Total
liabilities
|
|
1,661,760
|
|
1,568,484
|
|
|
|
|
|
Shareholders'
equity:
|
|
|
|
|
Common stock, $0.01
par value; 500,000,000 authorized
|
|
|
|
|
shares, 147,977,960
and 135,653,731 issued and outstanding shares at
December 31, 2023 and December 31, 2022, respectively
|
|
1,480
|
|
1,357
|
Additional paid-in
capital
|
|
3,113,487
|
|
2,782,416
|
Accumulated
deficit
|
|
(3,446,145)
|
|
(2,696,578)
|
Accumulated other
comprehensive (loss) income
|
|
(745)
|
|
756
|
Total shareholders'
(deficit) equity
|
|
(331,923)
|
|
87,951
|
Total liabilities and
shareholders' equity
|
|
$
1,329,837
|
|
$
1,656,435
|
|
|
|
|
|
About ARIKAYCE
ARIKAYCE is approved in the United States as
ARIKAYCE® (amikacin liposome inhalation
suspension), in Europe as
ARIKAYCE® Liposomal 590 mg Nebuliser Dispersion,
and in Japan as
ARIKAYCE® inhalation 590 mg (amikacin sulfate inhalation
drug product). Current international treatment guidelines
recommend the use of ARIKAYCE for appropriate patients. ARIKAYCE is
a novel, inhaled, once-daily formulation of amikacin, an
established antibiotic that was historically administered
intravenously and associated with severe toxicity to hearing,
balance, and kidney function. Insmed's proprietary
PULMOVANCE® liposomal technology enables the delivery of
amikacin directly to the lungs, where liposomal amikacin is taken
up by lung macrophages where the infection resides, while limiting
systemic exposure. ARIKAYCE is administered once daily using the
Lamira® Nebulizer System manufactured by PARI
Pharma GmbH (PARI).
About PARI Pharma and the Lamira® Nebulizer
System
ARIKAYCE is delivered by a novel inhalation device, the
Lamira® Nebulizer System, developed by PARI.
Lamira® is a quiet, portable nebulizer that enables
efficient aerosolization of ARIKAYCE via a vibrating, perforated
membrane. Based on PARI's 100-year history working with aerosols,
PARI is dedicated to advancing inhalation therapies by developing
innovative delivery platforms to improve patient care.
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 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, PH-ILD, 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.
IMPORTANT SAFETY INFORMATION AND BOXED WARNING
FOR ARIKAYCE IN THE U.S.
WARNING: RISK OF
INCREASED RESPIRATORY ADVERSE REACTIONS
|
ARIKAYCE has been
associated with an increased risk of respiratory adverse reactions,
including hypersensitivity pneumonitis, hemoptysis, bronchospasm,
and exacerbation of underlying pulmonary disease that have led to
hospitalizations in some cases.
|
Hypersensitivity Pneumonitis has been reported with the
use of ARIKAYCE in the clinical trials. Hypersensitivity
pneumonitis (reported as allergic alveolitis, pneumonitis,
interstitial lung disease, allergic reaction to ARIKAYCE) was
reported at a higher frequency in patients treated with ARIKAYCE
plus background regimen (3.1%) compared to patients treated with a
background regimen alone (0%). Most patients with hypersensitivity
pneumonitis discontinued treatment with ARIKAYCE and received
treatment with corticosteroids. If hypersensitivity pneumonitis
occurs, discontinue ARIKAYCE and manage patients as medically
appropriate.
Hemoptysis has been reported with the use of ARIKAYCE in
the clinical trials. Hemoptysis was reported at a higher frequency
in patients treated with ARIKAYCE plus background regimen (17.9%)
compared to patients treated with a background regimen alone
(12.5%). If hemoptysis occurs, manage patients as medically
appropriate.
Bronchospasm has been reported with the use of
ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma,
bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea
exertional, prolonged expiration, throat tightness, wheezing) was
reported at a higher frequency in patients treated with ARIKAYCE
plus background regimen (28.7%) compared to patients treated
with a background regimen alone (10.7%). If bronchospasm occurs
during the use of ARIKAYCE, treat patients as medically
appropriate.
Exacerbations of underlying pulmonary disease has been
reported with the use of ARIKAYCE in the clinical trials.
Exacerbations of underlying pulmonary disease (reported as chronic
obstructive pulmonary disease (COPD), infective exacerbation of
COPD, infective exacerbation of bronchiectasis) have been reported
at a higher frequency in patients treated with ARIKAYCE plus
background regimen (14.8%) compared to patients treated with
background regimen alone (9.8%). If exacerbations of
underlying pulmonary disease occur during the use of ARIKAYCE,
treat patients as medically appropriate.
Anaphylaxis and Hypersensitivity Reactions: Serious and
potentially life-threatening hypersensitivity reactions, including
anaphylaxis, have been reported in patients taking ARIKAYCE. Signs
and symptoms include acute onset of skin and mucosal tissue
hypersensitivity reactions (hives, itching, flushing, swollen
lips/tongue/uvula), respiratory difficulty (shortness of breath,
wheezing, stridor, cough), gastrointestinal symptoms (nausea,
vomiting, diarrhea, crampy abdominal pain), and cardiovascular
signs and symptoms of anaphylaxis (tachycardia, low blood pressure,
syncope, incontinence, dizziness). Before therapy with ARIKAYCE is
instituted, evaluate for previous hypersensitivity reactions to
aminoglycosides. If anaphylaxis or a hypersensitivity reaction
occurs, discontinue ARIKAYCE and institute appropriate supportive
measures.
Ototoxicity has been reported with the use of ARIKAYCE in
the clinical trials. Ototoxicity (including deafness, dizziness,
presyncope, tinnitus, and vertigo) were reported with a higher
frequency in patients treated with ARIKAYCE plus background regimen
(17%) compared to patients treated with background
regimen alone (9.8%). This was primarily driven by tinnitus
(7.6% in ARIKAYCE plus background regimen vs 0.9% in the background
regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background
regimen vs 2.7% in the background regimen alone arm). Closely
monitor patients with known or suspected auditory or vestibular
dysfunction during treatment with ARIKAYCE. If ototoxicity occurs,
manage patients as medically appropriate, including potentially
discontinuing ARIKAYCE.
Nephrotoxicity was observed during the clinical
trials of ARIKAYCE in patients with MAC lung disease but not at a
higher frequency than background regimen alone. Nephrotoxicity has
been associated with the aminoglycosides. Close monitoring of
patients with known or suspected renal dysfunction may be needed
when prescribing ARIKAYCE.
Neuromuscular Blockade: Patients with neuromuscular
disorders were not enrolled in ARIKAYCE clinical trials. Patients
with known or suspected neuromuscular disorders, such as myasthenia
gravis, should be closely monitored since aminoglycosides may
aggravate muscle weakness by blocking the release of acetylcholine
at neuromuscular junctions.
Embryo-Fetal Toxicity: Aminoglycosides can cause
fetal harm when administered to a pregnant woman. Aminoglycosides,
including ARIKAYCE, may be associated with total, irreversible,
bilateral congenital deafness in pediatric patients exposed in
utero. Patients who use ARIKAYCE during pregnancy, or become
pregnant while taking ARIKAYCE should be apprised of the potential
hazard to the fetus.
Contraindications: ARIKAYCE is contraindicated in
patients with known hypersensitivity to any aminoglycoside.
Most Common Adverse Reactions: The most common adverse
reactions in Trial 1 at an incidence ≥5% for patients using
ARIKAYCE plus background regimen compared to patients treated with
background regimen alone were dysphonia (47% vs 1%), cough (39% vs
17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%),
ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%),
musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs
10%), exacerbation of underlying pulmonary disease (15% vs 10%),
diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%),
headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash
(6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs
1%), and chest discomfort (5% vs 3%).
Drug Interactions: Avoid concomitant use of ARIKAYCE
with medications associated with neurotoxicity, nephrotoxicity, and
ototoxicity. Some diuretics can enhance aminoglycoside toxicity by
altering aminoglycoside concentrations in serum and tissue. Avoid
concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea,
or intravenous mannitol.
Overdosage: Adverse reactions specifically associated
with overdose of ARIKAYCE have not been identified. Acute
toxicity should be treated with immediate withdrawal of ARIKAYCE,
and baseline tests of renal function should be undertaken.
Hemodialysis may be helpful in removing amikacin from the body. In
all cases of suspected overdosage, physicians should contact the
Regional Poison Control Center for information about effective
treatment.
U.S. INDICATION
LIMITED POPULATION: ARIKAYCE® is
indicated in adults, who have limited or no alternative treatment
options, for the treatment of Mycobacterium avium complex
(MAC) lung disease as part of a combination antibacterial drug
regimen in patients who do not achieve negative sputum cultures
after a minimum of 6 consecutive months of a multidrug background
regimen therapy. As only limited clinical safety and effectiveness
data for ARIKAYCE are currently available, reserve ARIKAYCE for use
in adults who have limited or no alternative treatment
options. This drug is indicated for use in a limited and
specific population of patients.
This indication is approved under accelerated approval based
on achieving sputum culture conversion (defined as 3 consecutive
negative monthly sputum cultures) by Month 6. Clinical benefit has
not yet been established. Continued approval for this indication
may be contingent upon verification and description of clinical
benefit in confirmatory trials.
Limitation of Use: ARIKAYCE has only been studied
in patients with refractory MAC lung disease defined as patients
who did not achieve negative sputum cultures after a minimum of 6
consecutive months of a multidrug background regimen therapy. The
use of ARIKAYCE is not recommended for patients with non-refractory
MAC lung disease.
Patients are encouraged to report negative side effects of
prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088. You
can also call the Company at 1-844-4-INSMED.
Please see Full Prescribing
Information.
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: failure to obtain, or delays in obtaining, regulatory
approvals for ARIKAYCE 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 continue 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; 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 or changes 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 successfully or in a timely manner
validate a PRO tool and complete 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 the Company's 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 or maintain 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 of our
product candidates or to permit the use of ARIKAYCE in the broader
population of patients with MAC lung disease, among other
things; risks that the Company's clinical studies will be
delayed, that serious side effects will be identified during drug
development, or that any protocol amendments submitted will be
rejected; risks that topline, interim or partial data sets are not
representative of a complete or larger data set and are subject to
change as more patient data becomes available or that blinded data
will not be predictive of unblinded data; 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; the
Company's inability to access, upgrade or expand its technology
systems or difficulties in updating our existing technology or
developing or implementing new technology; 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; risk that our current
and potential future use of artificial intelligence and machine
learning may not be successful; 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; 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, 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.
With respect to the blended and blinded data observed from the
ongoing TPIP studies noted above, the dose titration, efficacy, and
safety analyses were based on data available as of August 28, September
12, and October 23, 2023,
respectively. These findings may not be representative of results
after the studies are completed and all data is collected and
analyzed. As a result, later interim data readouts and final data
from these studies may be materially different than the
observations described above, including with respect to efficacy,
safety and tolerability of TPIP.
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
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SOURCE Insmed Incorporated