Insmed Reports Fourth-Quarter and Full-Year 2023 Financial Results and Provides Business Update
—Topline Data from the Phase 3 ASPEN Trial of Brensocatib in Adult Patients with Bronchiectasis Remain on Track to
—Enrollment in the Phase 2 Study of TPIP in Patients with PH-ILD Completed in
—Company Ends 2023 With
— ARIKAYCE® (amikacin liposome inhalation suspension) Total Revenue of
—Company Reiterates
Sales Guidance for 2024 Global ARIKAYCE Revenues in the Range of
"
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 , andEurope . -
Insmed received encouraging written feedback inDecember 2023 from theU.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 inNovember 2023 and recommended that the study continue as planned. - Consistent with the Company's expectations, the
Pharmaceuticals and Medical Devices Agency (PMDA) inJapan 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 theU.S. in mid-2025, followed by launches inEurope andJapan 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 inNovember 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:
-
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 theU.S. ,$65.7 million inJapan , and$15.3 million inEurope and rest of world. Full-year 2023 sales demonstrated year-over-year growth of 21% in theU.S. and 16% inJapan , 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 inInsmed'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
(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 (
A replay of the conference call will be accessible approximately 1 hour after its completion through
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Consolidated Statements of Net Loss |
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(in thousands, except per share data) |
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(unaudited) |
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Three Months Ended |
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Twelve Months Ended |
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2023 |
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2022 |
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2023 |
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2022 |
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Product revenues, net |
$ 83,693 |
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$ 59,300 |
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$ 305,208 |
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$ 245,358 |
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Operating expenses: |
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Cost of product revenues (excluding amortization of intangible assets) |
18,443 |
|
13,069 |
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65,573 |
|
55,126 |
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Research and development |
137,029 |
|
124,763 |
|
571,011 |
|
397,518 |
|
Selling, general and administrative |
89,530 |
|
73,479 |
|
344,501 |
|
265,784 |
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Amortization of intangible assets |
1,263 |
|
1,264 |
|
5,052 |
|
5,053 |
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Change in fair value of deferred and contingent consideration liabilities |
15,700 |
|
(1,800) |
|
28,697 |
|
(20,802) |
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Total operating expenses |
261,965 |
|
210,775 |
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1,014,834 |
|
702,679 |
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|
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Operating loss |
(178,272) |
|
(151,475) |
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(709,626) |
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(457,321) |
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Investment income |
9,853 |
|
8,318 |
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42,132 |
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11,081 |
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Interest expense |
(20,784) |
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(16,445) |
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(81,694) |
|
(26,446) |
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Change in fair value of interest rate swap |
1,970 |
|
(1,526) |
|
320 |
|
(1,526) |
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Other income (expense), net |
2,170 |
|
1,130 |
|
1,856 |
|
(5,939) |
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Loss before income taxes |
(185,063) |
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(159,998) |
|
(747,012) |
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(480,151) |
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|
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Provision for income taxes |
998 |
|
125 |
|
2,555 |
|
1,383 |
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Net loss |
$ (186,061) |
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$ (160,123) |
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$ (749,567) |
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$ (481,534) |
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Basic and diluted net loss per share |
$ (1.28) |
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$ (1.21) |
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$ (5.34) |
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$ (3.91) |
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Weighted average basic and diluted common shares outstanding |
144,806 |
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132,694 |
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140,433 |
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123,035 |
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Consolidated Balance Sheets |
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(in thousands, except par value and share data) |
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As of |
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As of |
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Assets |
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Current assets: |
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Cash and cash equivalents |
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$ 482,374 |
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$ 1,074,036 |
Marketable securities |
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298,073 |
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74,244 |
Accounts receivable |
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41,189 |
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29,713 |
Inventory |
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83,248 |
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69,922 |
Prepaid expenses and other current assets |
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24,179 |
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25,468 |
Total current assets |
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929,063 |
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1,273,383 |
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Fixed assets, net |
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65,384 |
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56,491 |
Finance lease right-of-use assets |
|
20,985 |
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23,697 |
Operating lease right-of-use assets |
|
18,017 |
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21,894 |
Intangibles, net |
|
63,704 |
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68,756 |
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|
136,110 |
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136,110 |
Other assets |
|
96,574 |
|
76,104 |
Total assets |
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$ 1,329,837 |
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$ 1,656,435 |
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Liabilities and shareholders' equity |
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Current liabilities: |
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Accounts payable and accrued liabilities |
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$ 214,987 |
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$ 182,117 |
Finance lease liabilities |
|
2,610 |
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1,217 |
Operating lease liabilities |
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8,032 |
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6,909 |
Total current liabilities |
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225,629 |
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190,243 |
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Debt, long-term |
|
1,155,313 |
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1,125,250 |
Royalty financing agreement |
|
155,034 |
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148,015 |
Contingent consideration |
|
84,600 |
|
51,100 |
Finance lease liabilities, long-term |
|
27,026 |
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29,636 |
Operating lease liabilities, long-term |
|
11,013 |
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14,853 |
Other long-term liabilities |
|
3,145 |
|
9,387 |
Total liabilities |
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1,661,760 |
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1,568,484 |
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Shareholders' equity: |
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Common stock, |
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shares, 147,977,960 and 135,653,731 issued and outstanding shares at |
|
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 |
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$ 1,329,837 |
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$ 1,656,435 |
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About ARIKAYCE
ARIKAYCE is approved in the United States as ARIKAYCE® (amikacin liposome inhalation suspension), in
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
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
IMPORTANT SAFETY INFORMATION AND BOXED WARNING FOR ARIKAYCE IN THE
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
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-
Please see Full Prescribing Information .
About
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
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
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
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
Contact:
Investors:
Executive Director, Investor Relations
(646) 812-4030
bryan.dunn@insmed.com
Eleanor Barisser
Associate Director, Investor Relations
(718) 594-5332
eleanor.barisser@insmed.com
Media:
Executive Director, Corporate Communications
(732) 718-3621
amanda.fahey@insmed.com
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