8-K
INSMED Inc (INSM)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of
The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): May 8, 2025
INSMED INCORPORATED
(Exact name of registrant as specified in its charter)
| Virginia | 000-30739 | 54-1972729 |
|---|---|---|
| (State or other jurisdiction of incorporation) | (Commission File Number) | (IRS Employer Identification No.) |
| 700 US Highway 202/206<br><br> <br>Bridgewater, New Jersey | 08807<br><br> <br>(Zip Code) | |
| --- | --- | |
| (Address of principal executive offices) |
Registrant’s telephone number, including area code: (908) 977-9900
Not Applicable
(Former name or former address, if changed since last report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
| ☐ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
|---|---|
| ☐ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| --- | --- |
| ☐ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| --- | --- |
| ☐ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
| --- | --- |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which<br><br> <br>registered |
|---|---|---|
| Common Stock, par value $0.01 per share | INSM | Nasdaq Global Select Market |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR 230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR 240.12b-2).
Emerging growth company☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
ITEM 2.02 – Results of Operations and Financial Condition.
On May 8, 2025, Insmed Incorporated (the “Company”) issued a press release regarding its financial results for the three months ended March 31, 2025. A copy of this press release is furnished herewith as Exhibit 99.1 pursuant to this Item 2.02 and is incorporated herein by reference. The slide presentation to be used during the conference call referenced in the press release is furnished herewith as Exhibit 99.2.
The information contained herein, including Exhibits 99.1 and 99.2 attached hereto, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as shall be expressly set forth by specific reference in such a filing.
ITEM 9.01 – Financial Statements and Exhibits.
(d) Exhibits
| Exhibit<br><br> <br>No. | Description |
|---|---|
| 99.1 | Press release issued by Insmed Incorporated on May 8, 2025. |
| 99.2 | Insmed Incorporated May 8, 2025 Presentation. |
| 104 | Cover Page Interactive Date File (embedded within the Inline XBRL document). |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Dated: May 8, 2025 | INSMED INCORPORATED | |
|---|---|---|
| By: | /s/ Michael A. Smith | |
| Name: | Michael A. Smith | |
| Title: | Chief Legal Officer and Corporate Secretary |
Exhibit 99.1
Insmed Reports First-Quarter 2025 Financial Results and Provides Business Update
—ARIKAYCE^®^ (amikacin liposome inhalation suspension) Total Revenue of $92.8 Million for the First Quarter of 2025, Reflecting 23% Growth Over the First Quarter of 2024—
—NDA for Brensocatib in Patients with Bronchiectasis Remains on Track, with a PDUFA Target Action Date of August 12, 2025—
—Phase 2b Study of TPIP in Patients with PAH Completed; Topline Data Expected in June 2025—
—Enrollment Completed for Phase 2b BiRCh Study of Brensocatib in Patients with CRSsNP; Topline Data Anticipated By the End of 2025—
—MAA Filings for Brensocatib in Patients with Bronchiectasis Accepted by EMA and MHRA—
—Company Reiterates 2025 Global ARIKAYCE Revenue Guidance Range of $405 Million to $425 Million, Reflecting Double-Digit Growth Compared to 2024—
BRIDGEWATER, N.J., May 08, 2025 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today reported financial results for the first quarter ended March 31, 2025 and provided a business update.
“In the first quarter of 2025, Insmed demonstrated executional excellence across our commercial and clinical programs, while simultaneously preparing for critical near-term milestones, including our Phase 2 data readout of TPIP in PAH, and our anticipated FDA approval of brensocatib for bronchiectasis,” said Will Lewis, Chair and Chief Executive Officer of Insmed. “Against the backdrop of an evolving regulatory environment, brensocatib’s FDA review process remains on track, and we are relentlessly focused on preparing for a frictionless launch for the patients counting on us to succeed. This momentum is anticipated to continue as we expect to unveil data from our Phase 2 BiRCh study of brensocatib in chronic rhinosinusitis without nasal polyps by year-end 2025 and our Phase 3 ENCORE study of ARIKAYCE in all MAC lung disease patients in the first half of 2026. As I reflect on these upcoming catalysts, I couldn’t be prouder of the Insmed team working tirelessly to achieve our ambitious vision.”
Recent Progress and Anticipated Milestones by Program:
ARIKAYCE
| • | ARIKAYCE global revenue grew 23% in the first quarter of 2025 compared to the first quarter of 2024, reflecting double-digit year-over-year growth in the U.S., Japan, and Europe. |
|---|---|
| • | The Company anticipates the topline readout of the Phase 3 ENCORE trial in the first half of 2026 in patients with newly diagnosed or recurrent Mycobacterium avium complex (MAC) lung disease who have not started antibiotics, which will include the change from baseline in respiratory symptom score at Month 13 and the percentage of patients achieving<br> durable culture conversion at Month 15. |
| --- | --- |
| • | The Company plans to submit a supplementary new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) for ARIKAYCE in all patients with MAC lung disease in the U.S. in the second<br> half of 2026. |
| --- | --- |
Brensocatib
| • | In February 2025, the FDA accepted the Company’s New Drug Application (NDA) for brensocatib for patients with bronchiectasis, granting the application Priority Review designation with a Prescription<br> Drug User Fee Act (PDUFA) target action date of August 12, 2025. If approved, Insmed expects to immediately launch brensocatib in the U.S. |
|---|---|
| • | Regulatory submissions for brensocatib in the EU and UK have been accepted, with submission in Japan planned for 2025. Insmed anticipates commercial launches for each territory in 2026, pending<br> approval. |
| --- | --- |
| • | Insmed completed enrollment in the Phase 2b BiRCh study of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) with 288 patients. The Company remains on track to report<br> topline results by the end of 2025. |
| --- | --- |
| • | The Company continues to enroll patients in the Phase 2b CEDAR study of brensocatib in patients with hidradenitis suppurativa (HS). Based on current enrollment rates, the Company anticipates the<br> interim futility analysis from the first 100 patients to complete Week 16 of the trial in the first half of 2026. |
| --- | --- |
TPIP
| • | Insmed completed the Phase 2b study of treprostinil palmitil inhalation powder (TPIP) in pulmonary arterial hypertension (PAH), with topline data anticipated in June of 2025. |
|---|---|
| • | The Company plans to initiate a Phase 3 study of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) in the second half of 2025. |
| --- | --- |
Gene Therapy
| • | Insmed initiated the Phase 1 ASCEND clinical study of INS1201, an intrathecally-delivered gene therapy for patients with Duchenne muscular dystrophy (DMD), in the first quarter of 2025, and expects<br> to dose the first patient in the second quarter of 2025. |
|---|---|
| • | The Company’s next two gene therapy candidates, which target amyotrophic lateral sclerosis (ALS) and Stargardt disease, are currently advancing toward the clinic. |
| --- | --- |
Pre-Clinical Programs
| • | Insmed’s 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 for the indications<br> being pursued. |
|---|---|
| • | The Company anticipates submitting an average of one to two INDs per year from its pre-clinical research programs. |
| --- | --- |
| • | Insmed continues to anticipate that the totality of its pre-clinical research programs will comprise less than 20% of overall expenditures. |
| --- | --- |
Corporate Updates
| • | In April 2025, data from the Phase 3 ASPEN study of brensocatib in patients with bronchiectasis were published in the New England<br> Journal of Medicine. |
|---|---|
| • | In April 2025, the Company issued a notice of redemption for all $569.5 million aggregate principal amount of its remaining outstanding 0.75% Convertible Senior Notes Due 2028, with a redemption date<br> of June 6, 2025. |
| --- | --- |
| • | Insmed plans to present eleven abstracts from across its respiratory portfolio (ARIKAYCE, brensocatib, and TPIP) at the American Thoracic Society (ATS) 2025 International Conference, taking place May<br> 18-21, 2025. |
| --- | --- |
| • | Insmed intends to expand its U.S. manufacturing footprint and has a project underway to establish a secondary source of brensocatib manufacturing in the U.S. |
| --- | --- |
First-Quarter 2025 Financial Results
| • | The following table summarizes first-quarter 2025 and 2024 revenues and revenue growth for ARIKAYCE across all commercial regions: | ||||||
|---|---|---|---|---|---|---|---|
| Three Months Ended<br><br> <br>March 31, | |||||||
| --- | --- | --- | --- | --- | --- | --- | --- |
| (in millions) | 2025 | 2024 | Growth | ||||
| U.S. | $ | 64.3 | $ | 56.3 | 14.1 | % | |
| Japan | 22.1 | 14.9 | 48.3 | % | |||
| Europe & Rest of World | 6.5 | 4.3 | 51.8 | % | |||
| Total Revenues | $ | 92.8 | $ | 75.5 | 22.9 | % | |
| • | Cost of product revenues (excluding amortization of intangibles) was $21.3 million for the first quarter of 2025, compared to $17.5 million for the first quarter of 2024. The increase in cost of<br> product revenues primarily reflects growth in ARIKAYCE sales. | ||||||
| --- | --- | ||||||
| • | Research and development (R&D) expenses were $152.6 million for the first quarter of 2025, compared to $121.1 million for the first quarter of 2024. The increase in R&D expenses was primarily<br> related to increases in compensation and benefit-related expenses and stock-based compensation costs, due to an increase in headcount, as well as an increase in manufacturing expenses. | ||||||
| --- | --- | ||||||
| • | Selling, general and administrative (SG&A) expenses for the first quarter of 2025 were $147.5 million, compared to $93.1 million for the first quarter of 2024. The increase in SG&A expenses<br> was primarily related to increases in compensation and benefit-related expenses, as well as stock-based compensation costs, predominantly due to an increase in headcount in preparation for the anticipated launch of brensocatib in the U.S.,<br> pending regulatory approval. | ||||||
| --- | --- | ||||||
| • | For the first quarter of 2025, Insmed reported a net loss of $256.6 million, or $1.42 per share, compared to a net loss of $157.1 million, or $1.06 per share, for the first quarter of 2024. | ||||||
| --- | --- |
Balance Sheet, Financial Guidance, and Planned Investments
| • | As of March 31, 2025, Insmed had cash, cash equivalents, and marketable securities totaling approximately $1.2 billion. |
|---|---|
| • | Insmed continues to anticipate full-year 2025 global ARIKAYCE revenues in the range of $405 million to $425 million, representing between 11% and 17% year-over-year growth compared to 2024. |
| --- | --- |
| • | The Company plans to continue to invest in the following key activities in 2025: |
| --- | --- |
| (i) | commercialization and expansion of ARIKAYCE globally; |
| --- | --- |
| (ii) | commercial launch of brensocatib in the U.S., if approved, with advancement of regulatory submissions and interactions in the EU, UK, and Japan; |
| --- | --- |
| (iii) | advancement of clinical trial programs for brensocatib, including the ongoing Phase 2b BiRCh study in patients with CRSsNP and the Phase 2b CEDAR study in patients with HS; |
| --- | --- |
| (iv) | advancement of the Phase 3 ENCORE study for ARIKAYCE, which is intended to satisfy the post-marketing requirement for full approval of its current indication and potentially support label expansion<br> to include all patients with a MAC lung disease; |
| --- | --- |
| (v) | advancement of the clinical development programs for TPIP, including the Phase 2b study in patients with PAH and the initiation of a Phase 3 study in patients with PH-ILD; |
| --- | --- |
| (vi) | advancement of the Phase 1 ASCEND study for INS1201 in DMD; and |
| --- | --- |
| (vii) | continued development of its pre-clinical research programs. |
| --- | --- |
Conference Call
Insmed will host a conference call beginning today, May 8, 2025, at 8:00 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 May 15, 2025, 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 March 31, | ||||||
|---|---|---|---|---|---|---|
| 2025 | 2024 | |||||
| Product revenues, net | $ | 92,823 | $ | 75,500 | ||
| Operating expenses: | ||||||
| Cost of product revenues (excluding amortization of intangible assets) | 21,278 | 17,457 | ||||
| Research and development | 152,577 | 121,083 | ||||
| Selling, general and administrative | 147,545 | 93,102 | ||||
| Amortization of intangible assets | 1,263 | 1,263 | ||||
| Change in fair value of deferred and contingent consideration liabilities | 18,300 | (11,900 | ) | |||
| Total operating expenses | 340,963 | 221,005 | ||||
| Operating loss | (248,140 | ) | (145,505 | ) | ||
| Investment income | 13,906 | 8,783 | ||||
| Interest expense | (21,569 | ) | (21,042 | ) | ||
| Change in fair value of interest rate swap | - | 2,362 | ||||
| Other income (expense), net | 132 | (1,100 | ) | |||
| Loss before income taxes | (255,671 | ) | (156,502 | ) | ||
| Provision for income taxes | 912 | 589 | ||||
| Net loss | $ | (256,583 | ) | $ | (157,091 | ) |
| Basic and diluted net loss per share | $ | (1.42 | ) | $ | (1.06 | ) |
| Weighted average basic and diluted common shares outstanding | 180860 | 148,456 |
INSMED INCORPORATED
Consolidated Balance Sheets
(in thousands, except par value and share data)
| As of | |||||
|---|---|---|---|---|---|
| December 31, 2024 | |||||
| Assets | |||||
| Current assets: | |||||
| Cash and cash equivalents | 403,247 | $ | 555,030 | ||
| Marketable securities | 796,204 | 878,796 | |||
| Accounts receivable | 47,746 | 52,012 | |||
| Inventory | 100,713 | 98,578 | |||
| Prepaid expenses and other current assets | 54,782 | 37,245 | |||
| Total current assets | 1,402,692 | 1,621,661 | |||
| Fixed assets, net | 88,358 | 80,052 | |||
| Finance lease right-of-use assets | 17,595 | 18,273 | |||
| Operating lease right-of-use assets | 10,832 | 17,257 | |||
| Intangibles, net | 57,389 | 58,652 | |||
| Goodwill | 136,110 | 136,110 | |||
| Other assets | 89,759 | 93,226 | |||
| Total assets | 1,802,735 | $ | 2,025,231 | ||
| Liabilities and shareholders' equity | |||||
| Current liabilities: | |||||
| Accounts payable and accrued liabilities | 232,674 | $ | 285,209 | ||
| Finance lease liabilities | 3,054 | 2,961 | |||
| Operating lease liabilities | 3,505 | 9,358 | |||
| Total current liabilities | 239,233 | 297,528 | |||
| Debt, long-term | 1,105,068 | 1,103,382 | |||
| Royalty financing agreement | 162,508 | 161,067 | |||
| Contingent consideration | 159,900 | 144,200 | |||
| Finance lease liabilities, long-term | 23,266 | 24,064 | |||
| Operating lease liabilities, long-term | 8,480 | 9,112 | |||
| Other long-term liabilities | 5,121 | 499 | |||
| Total liabilities | 1,703,576 | 1,739,852 | |||
| Shareholders' equity: | |||||
| Common stock, 0.01 par value; 500,000,000 authorized shares, 181,900,074 and 179,382,635 issued and outstanding<br> shares at March 31, 2025 and December 31, 2024, respectively | 1,819 | 1,794 | |||
| Additional paid-in capital | 4,714,742 | 4,645,791 | |||
| Accumulated deficit | (4,616,500 | ) | (4,359,917 | ) | |
| Accumulated other comprehensive loss | (902 | ) | (2,289 | ) | |
| Total shareholders' equity | 99,159 | 285,379 | |||
| Total liabilities and shareholders' equity | 1,802,735 | $ | 2,025,231 |
All values are in US Dollars.
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, chronic rhinosinusitis without nasal polyps, hidradenitis suppurativa, 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 pulmonary arterial hypertension (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.
About INS1201
INS1201 is an investigational micro-dystrophin adeno-associated virus gene replacement therapy that Insmed is developing as a potential treatment for patients with Duchenne muscular dystrophy. Administered intrathecally, this approach has the potential to target both skeletal and cardiac muscles at lower doses. INS1201 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<br><br> <br><br><br> <br>ARIKAYCE has been associated with an increased risk of respiratory adverse<br> 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 people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases. The Company is advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery focused on serving patient communities where the need is greatest. Insmed's most advanced programs are in pulmonary and inflammatory conditions, including a therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease. The Company's early-stage programs encompass a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.
Headquartered in Bridgewater, New Jersey, Insmed has offices and research locations throughout the United States, Europe, and Japan. Insmed is proud to be recognized as one of the best employers in the biopharmaceutical industry, including spending four consecutive years as the No. 1 Science Top Employer. 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 continue to successfully commercialize ARIKAYCE, our 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; our inability to obtain full approval of ARIKAYCE from the FDA, including the risk that we will not successfully or in a timely manner complete the confirmatory post-marketing clinical trial required for full approval of ARIKAYCE, or our failure to obtain regulatory approval to expand ARIKAYCE's indication to a broader patient population; failure to obtain, or delays in obtaining, regulatory approvals for brensocatib, TPIP or our other product candidates in the U.S., Europe or Japan or for ARIKAYCE outside the US, Europe or Japan, including separate regulatory approval for Lamira^®^ in each market and for each usage; failure to successfully commercialize brensocatib, TPIP or our other product candidates, if approved by applicable regulatory authorities, or to maintain applicable regulatory approvals for brensocatib, TPIP or our other product candidates, if approved; uncertainties or changes in the degree of market acceptance of ARIKAYCE or, if approved, brensocatib, TPIP or our other product candidates by physicians, patients, third-party payors and others in the healthcare community; our inability to obtain and maintain adequate reimbursement from government or third-party payors for ARIKAYCE or, if approved, brensocatib, TPIP or our other product candidates, or acceptable prices for ARIKAYCE or, if approved, brensocatib, TPIP or our other product candidates; inaccuracies in our estimates of the size of the potential markets for ARIKAYCE, brensocatib, TPIP or our other product candidates or in data we have used to identify physicians, expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; failure of third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE, brensocatib, or TPIP 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; the risks and uncertainties associated with, and the perceived benefits of, our secured senior loan with certain funds managed by Pharmakon Advisors LP and our royalty financing with OrbiMed Royalty & Credit Opportunities IV, LP, including our ability to maintain compliance with the covenants in the agreements for the senior secured loan and royalty financing and the impact of the restrictions on our operations under these agreements; our 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 our product candidates that are approved in the future; failure to successfully conduct future clinical trials for ARIKAYCE, brensocatib, TPIP or our other product candidates and our 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; development of unexpected safety or efficacy concerns related to ARIKAYCE, brensocatib, TPIP or our other product candidates; risks that our clinical studies will be delayed, that serious side effects will be identified during drug development, or that any protocol amendments submitted will be rejected; failure to successfully predict the time and cost of development, regulatory approval and commercialization for novel gene therapy products; the risk that interim, topline or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available or may be interpreted differently if additional data are disclosed, or that blinded data will not be predictive of unblinded data; risk that our competitors may obtain orphan drug exclusivity for a product that is essentially the same as a product we are developing for a particular indication; our inability to attract and retain key personnel or to effectively manage our growth; our inability to successfully integrate our recent acquisitions and appropriately manage the amount of management's time and attention devoted to integration activities; risks that our acquired technologies, products and product candidates will not be commercially successful; inability to adapt to our highly competitive and changing environment; inability to access, upgrade or expand our technology systems or difficulties in updating our existing technology or developing or implementing new technology; risk that we are unable to maintain our significant customers; risk that government healthcare reform materially increases our costs and damages our financial condition; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; risk that our current and potential future use of AI 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 us, our suppliers, third-party service providers and potential partners; the risk that we could become involved in costly intellectual property disputes, be unable to adequately protect our intellectual property rights or prevent disclosure of our trade secrets and other proprietary information, and incur costs associated with litigation or other proceedings related to such matters; restrictions or other obligations imposed on us by agreements related to ARIKAYCE, brensocatib or our other product candidates, including our license agreements with PARI and AstraZeneca AB, and failure to comply with our obligations under such agreements; the cost and potential reputational damage resulting from litigation to which we are or may become a party, including product liability claims; risk that our operations are subject to a material disruption in the event of a cybersecurity attack or issue; our limited experience operating internationally; changes in laws and regulations applicable to our business, including any pricing reform and laws that impact our ability to utilize certain third parties in the research, development or manufacture of our product candidates, and failure to comply with such laws and regulations; our history of operating losses, and the possibility that we never achieve or maintain profitability; goodwill impairment charges affecting our results of operations and financial condition; inability to repay our existing indebtedness and uncertainties with respect to our 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, 2024 and any subsequent Company filings with the Securities and Exchange Commission (SEC).
The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.
Contact:
Investors:
Bryan Dunn
Vice President, Investor Relations
(646) 812-4030
investor.relations@insmed.com
Media:
Claire Mulhearn
Vice President, Corporate Communications
(862) 842-6819
media@insmed.com
Exhibit 99.2
May 8, 2025 First-Quarter 2025 Earnings Presentation
Forward Looking Statements This presentation 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 presentation 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 continue to successfully commercialize ARIKAYCE, our 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 US, European or Japanese approval for ARIKAYCE; our inability to obtain full approval of ARIKAYCE from the FDA, including the risk that we will not successfully or in a timely manner complete the confirmatory post-marketing clinical trial required for full approval of ARIKAYCE, or our failure to obtain regulatory approval to expand ARIKAYCE’s indication to a broader patient population; failure to obtain, or delays in obtaining, regulatory approvals for brensocatib, TPIP or our other product candidates in the US, Europe or Japan or for ARIKAYCE outside the US, Europe or Japan, including separate regulatory approval for Lamira® in each market and for each usage; failure to successfully commercialize brensocatib, TPIP or our other product candidates, if approved by applicable regulatory authorities, or to maintain applicable regulatory approvals for brensocatib, TPIP or our other product candidates, if approved; uncertainties or changes in the degree of market acceptance of ARIKAYCE or, if approved, brensocatib, TPIP or our other product candidates by physicians, patients, third-party payors and others in the healthcare community; our inability to obtain and maintain adequate reimbursement from government or third-party payors for ARIKAYCE or, if approved, brensocatib, TPIP or our other product candidates, or acceptable prices for ARIKAYCE or, if approved, brensocatib, TPIP or our other product candidates; inaccuracies in our estimates of the size of the potential markets for ARIKAYCE, brensocatib, TPIP or our other product candidates or in data we have used to identify physicians, expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; failure of third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE, brensocatib, or TPIP 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; the risks and uncertainties associated with, and the perceived benefits of, our senior secured loan with certain funds managed by Pharmakon Advisors LP and our royalty financing with OrbiMed Royalty & Credit Opportunities IV, LP, including our ability to maintain compliance with the covenants in the agreements for the senior secured loan and royalty financing and the impact of the restrictions on our operations under these agreements; our 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 our product candidates that are approved in the future; failure to successfully conduct future clinical trials for ARIKAYCE, brensocatib, TPIP or our other product candidates and our 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; development of unexpected safety or efficacy concerns related to ARIKAYCE, brensocatib, TPIP or our other product candidates; risks that our clinical studies will be delayed, that serious side effects will be identified during drug development, or that any protocol amendments submitted will be rejected; failure to successfully predict the time and cost of development, regulatory approval and commercialization for novel gene therapy products; the risk that interim, topline or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available or may be interpreted differently if additional data are disclosed, or that blinded data will not be predictive of unblinded data; risk that our competitors may obtain orphan drug exclusivity for a product that is essentially the same as a product we are developing for a particular indication; our inability to attract and retain key personnel or to effectively manage our growth; our inability to successfully integrate our recent acquisitions and appropriately manage the amount of management’s time and attention devoted to integration activities; risks that our acquired technologies, products and product candidates will not be commercially successful; inability to adapt to our highly competitive and changing environment; inability to access, upgrade or expand our technology systems or difficulties in updating our existing technology or developing or implementing new technology; risk that we are unable to maintain our significant customers; risk that government healthcare reform materially increases our costs and damages our financial condition; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; 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 US, Europe, Japan and globally, including the effect of prolonged periods of inflation, affecting us, our suppliers, third-party service providers and potential partners; the risk that we could become involved in costly intellectual property disputes, be unable to adequately protect our intellectual property rights or prevent disclosure of our trade secrets and other proprietary information, and incur costs associated with litigation or other proceedings related to such matters; restrictions or other obligations imposed on us by agreements related to ARIKAYCE, brensocatib or our other product candidates, including our license agreements with PARI and AstraZeneca AB , and failure to comply with our obligations under such agreements; the cost and potential reputational damage resulting from litigation to which we are or may become a party, including product liability claims; risk that our operations are subject to a material disruption in the event of a cybersecurity attack or issue; our limited experience operating internationally; changes in laws and regulations applicable to our business, including any pricing reform and laws that impact our ability to utilize certain third parties in the research, development or manufacture of our product candidates, and failure to comply with such laws and regulations; our history of operating losses, and the possibility that we never achieve or maintain profitability; goodwill impairment charges affecting our results of operations and financial condition; inability to repay our existing indebtedness and uncertainties with respect to our 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, 2024 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 presentation. 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. Additional Disclaimers: Please be aware that brensocatib and TPIP are investigational products that have not been approved for sale or found safe or effective by the FDA or any regulatory authority. In addition, ARIKAYCE has not been approved for the treatment of all patients with MAC lung disease. This presentation is not promotion or advertisement of ARIKAYCE, brensocatib, or TPIP. Insmed and ARIKAYCE are registered trademarks of Insmed Incorporated. All other trademarks are property of their respective owner(s).
Opening Remarks Brensocatib Updates TPIP Updates ARIKAYCE® Updates Financial Results 5-6 8-9 11 13 16-20 Slides Will Lewis Chair & CEO Sara Bonstein Chief Financial Officer Agenda Speakers
Opening Remarks Will Lewis | Chair & CEO
First-Quarter 2025 Highlights * Pending regulatory approval for bronchiectasis indication | FDA: Food and Drug Administration | PDUFA: Prescription Drug User Fee Act Mid- to late-stage programs refers to all clinical programs in Phase 2 and Phase 3 Double-digit ARIKAYCE growth All mid- to late-stage clinical programs on or ahead of schedule FDA review of brensocatib undisrupted PDUFA target action date of Aug. 12th for potential U.S. approval of brensocatib*
Bronchiectasis: Ph3 ASPEN ✔ CRSsNP: Ph2 BiRCh HS: Ph2 CEDAR 1 2 3 PH-ILD: Phase 2 ✔ PAH: Phase 2 Refractory MAC: Ph3 CONVERT ✔ All MAC LD: Ph3 ARISE ✔ All MAC LD: Ph3 ENCORE Brensocatib ARIKAYCE® TPIP Three Mid- to Late-Stage Programs Bronchiectasis: non-cystic fibrosis bronchiectasis; MAC / MAC LD: Mycobacterium avium complex lung disease; CRSsNP: chronic rhinosinusitis without nasal polyps; TPIP: Treprostinil Palmitil Inhalation Powder; PH-ILD: pulmonary hypertension due to interstitial lung disease; PAH: pulmonary arterial hypertension; HS: hidradenitis suppurativa | Mid- to late-stage programs refers to all clinical programs in Phase 2 and Phase 3 | Ph: Phase | ✔ trial successfully readout Achieved Clinical Success In At Least One Indication
Brensocatib Updates Will Lewis | Chair & CEO
Brensocatib FDA Review Remains On Track CRSsNP Phase 2b BiRCh enrolled 288 patients Topline readout expected by YE:25 Bronchiectasis Phase 3 ASPEN results published in NEJM Focused on a frictionless launch Regulatory submissions accepted in Europe & UK NEJM: New England Journal of Medicine; April 23, 2025 |
Bronchiectasis: non-cystic fibrosis bronchiectasis | FDA: Food and Drug Administration | CRSsNP: chronic rhinosinusitis without nasal polyps | HS: hidradenitis suppurativa | YE: year-end | 1H: first-half HS Phase 2 CEDAR trial enrollment on
track Interim futility analysis anticipated in 1H:26 Bronchiectasis Patients Are Motivated to Act1 ~1 Million Unique visits to U.S. disease state website\* ~53K Patients‡ who have engaged in a high-value action\* Visit
SpeakUpInBronchiectasis.com1 1 Patient website metrics dashboard \* Since website launch in November 2023 through April 2025 ‡ Cumulative visitors that have self-reported as patients that have taken a high-value action like downloading
support tools and registering for Customer Relationship Management \(CRM\)
Next Gen DPP1 Leading the Way in Neutrophil-Mediated Disease Management DPP1: dipeptidyl peptidase 1 enzyme that plays a crucial role in the activation of neutrophil serine proteases (NSPs). | COPD: Chronic obstructive pulmonary disease Next-Generation DPP1s COPD Other Indications Rheumatoid Arthritis Anticipate first indication to enter clinic as early as 2026
TPIP Updates TPIP: Treprostinil Palmitil Inhalation Powder Will Lewis | Chair & CEO
Phase 2b PAH Trial Topline Readout Expected in June 2025 PAH Key Trial Details PVR measured at trough1 Max tolerated dose of 640 μg ~95% of completed patients enrolled in OLE permitting dose up to 1,280 μg PH-ILD Expect to initiate Phase 3 trial in 2H:25 TPIP: Treprostinil Palmitil Inhalation Powder | PAH: pulmonary arterial hypertension | PVR: pulmonary vascular resistance | OLE: open-label extension | 6MWD: six-minute walk distance | “Best-in-class" indicates a profile that could be considered more attractive than other treatment options for a particular disease. Head-to-head clinical trials are not anticipated. Placebo-Adjusted Reduction from Baseline PVR2 ≥20% a “clear win”* ~25% “best-in-class” Hope to see 15-20m directional benefit on 6MWD favoring treatment** 1 Implies measurement approximately 24 hours after prior dose administration 2 Phase 2b TPIP for PAH trial primary endpoint * A “clear win” refers to an outcome that could be considered clinically meaningful and competitive with results shown for other currently available prostanoid therapies ** Phase 2b TPIP for PAH trial secondary endpoint: change from baseline 6MWD vs. placebo; not powered to show definitive effect
ARIKAYCE® Updates Will Lewis | Chair & CEO
Phase 3 ENCORE Trial Progressing On Schedule ENCORE Primary Endpoint United States: PRO (Month 13) Japan: Durable culture conversion (Month 15) Expect topline readout in 1H:26 after Month 15 results are available Worldwide Sales* +22.9% Refractory MAC LD All MAC LD * unaudited MAC LD: Mycobacterium avium complex lung disease | PRO: patient reported outcomes | 1H: first-half
Let’s Recap Each clinical program showing meaningful progress Regulatory filings and launch preparations for brensocatib advancing on or ahead of schedule Strong commercial execution keeps us on track to achieve 2025 ARIKAYCE guidance Committed to delivering on behalf of patients and stakeholders
Financial Results Sara Bonstein | Chief Financial Officer
Strong Fundamentals Minimize Tariff Exposure * Does not include announced tariffs that have explicitly carved out pharmaceutical products | IP: intellectual property Based on the tariffs currently in place, anticipate single-digit million $ impact annually* U.S. IP is domiciled in the U.S.; expected to limit tariff exposure to actual cost base of the product Intellectual Property Intend to expand U.S. manufacturing footprint; project underway to establish brensocatib secondary source manufacturing in the U.S. Manufacturing
ARIKAYCE Growing Double-Digits in 7th Year Post Launch 6th consecutive quarter of double-digit growth in each region Strength ex-U.S. driven by increased new patient starts Reiterating 2025 ARIKAYCE guidance of $405 to $425M* United States $64.3M, +14.1% GTN: ~20%2 Japan $22.1M, +48.3% Europe & Rest of World $6.5M, +51.8% Worldwide $92.8M +22.9% Three Months Ended March 31, 20251 * Does not include revenues associated with the anticipated launch of brensocatib, pending regulatory approval | GTN: gross-to-net 1 unaudited | 2 Full-year 2025 U.S. gross-to-net guidance for ARIKAYCE expected to be in the range of high-teens to low-twenties percent
Well-Capitalized As We Approach Upcoming Catalysts Sequential increase in burn driven by annual employee incentive compensation1 Following brensocatib launch*, expect sales growth to accelerate faster than spend The strength of our business affords us many options for accessing capital * Pending regulatory approval for bronchiectasis indication 1 Annual employee incentive compensation payout which occurs in the first-quarter of each year † Unaudited cash, cash equivalents, and marketable securities position as of March 31, 2025 ~$1.2B March 31, 2025† In Cash, Cash Equivalents, and Marketable Securities
Called Remaining Convertible Debt* Expect conversion to lower ongoing interest expense and reduce outstanding debt Remaining Principal1,2 Underlying Shares3 ~$570M ~17.8M (if all notes are converted) * CUSIP 457669AB5; 0.75% convertible senior notes set to mature in June 2028 1 Insmed issued a redemption notice on April 24, 2025, as permitted under Section 11.03 of the indenture governing the notes. Redemption date is June 6, 2025. 2 Original principal amount of approximately $575M excludes the approximately $6M of previously redeemed notes as of April 24, 2025 3 Approximately $570M principal divided by approximately $31.96 per share conversion price equals approximately 17.8M shares
Investing to Advance Our Commercial & Clinical Programs Three Months Ended* March 31, 2025 March 31, 2024 Product Revenues $92.8 $75.5 Cost of Product Revenues** As a % of Revenues (21.3) 22.9% (17.5) 23.1% R&D (152.6) (121.1) SG&A (147.5) (93.1) Other† (19.6) 10.6 Total Operating Expenses $(341.0) $(221.0) Operating Loss $(248.1) $(145.5) Cost of product revenues as a % of revenues was in-line with historical R&D and SG&A increased y/y reflecting investment in pipeline & launch readiness Sequential step-down in R&D spend1 * unaudited ** Excluding amortization of intangible assets R&D: research and development | SG&A: selling, general, and administrative expenses | y/y: year-over-year † Includes amortization of intangible assets and change in fair value of deferred and contingent consideration liabilities 1 Compared to prior quarter R&D expense of $179.7M in the three months ended December 31, 2024 (in $ millions, except for percentages)
Closing Remarks In a unique position of strength financially & operationally Brensocatib launch* expected to accelerate already strong revenue growth Strong cash position to support upcoming commercial and clinical catalysts * Pending regulatory approval for bronchiectasis indication | Bronchiectasis: non-cystic fibrosis bronchiectasis
Q&A Session Will Lewis Chair & CEO Sara Bonstein Chief Financial Officer Martina Flammer Chief Medical Officer
