Insmed Reports Fourth-Quarter and Full-Year 2024 Financial Results and Provides Business Update

—ARIKAYCE® (amikacin liposome inhalation suspension) Total Revenue of $104.4 Million for Fourth-Quarter and $363.7 Million for Full-Year 2024, Reflecting 19% Annual Growth and Exceeding the Upper End of Full-Year 2024 Guidance Range— —Company Reiterates 2025 Global ARIKAYCE Revenue Guidance of $405 Million to $425 Million, Reflecting Double-Digit Growth Compared to 2024— —NDA for Brensocatib in Patients with Bronchiectasis Accepted by FDA and Granted Priority Review with a PDUFA Target Action Date of August 12, 2025— —Phase 2b Study of TPIP in Patients with PAH, Phase 2b BiRCh Study of Brensocatib in Patients with CRSsNP, and Phase 3 ENCORE Study of ARIKAYCE Remain on Track for Topline Data in Mid-2025, End of 2025, and First Quarter of 2026, Respectively— —Company Ends 2024 with Approximately $1.4 Billion of Cash, Cash Equivalents, and Marketable Securities— , /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 fourth quarter and full year ended December 31, 2024 and provided a business update. "As we report on the most important year in Insmed's history, we feel we are in a rare position in our industry, with extraordinary opportunities ahead. Our success across all areas of the business in 2024—most notably the delivery of positive data from the landmark Phase 3 ASPEN study of brensocatib in bronchiectasis—has positioned us to potentially reach many more patients suffering from serious diseases, and resulted in significant value creation," said Will Lewis, Chair and Chief Executive Officer of Insmed. "In 2025, we look forward to delivering the highly anticipated U.S. launch of brensocatib, pending FDA approval, and we are delighted to be one step closer to achieving that goal with the FDA's acceptance of our NDA with Priority Review. Simultaneously, we expect to continue to advance our robust mid- to late-stage pipeline while generating double-digit worldwide revenue growth for ARIKAYCE. We believe that Insmed's strong balance sheet and unique company culture will enable us to deliver on behalf of patients in need." Recent Progress and Anticipated Milestones by Program: ARIKAYCE ARIKAYCE global revenue grew 19% in 2024 compared to 2023, reflecting continued strong growth in the U.S., Japan, and Europe. In the fourth quarter of 2024, the Company completed enrollment in the Phase 3 ENCORE study for patients with newly diagnosed or recurrent Mycobacterium avium complex (MAC) lung disease who had not started antibiotics. Total enrollment in the study was 425 patients. The Company continues to anticipate a topline readout for ENCORE in the first quarter of 2026, with the submission of a supplementary new drug application (sNDA) to the FDA for ARIKAYCE in all patients with MAC lung disease in the U.S. expected later in 2026. Brensocatib In February 2025, the FDA accepted the Company's New Drug Application (NDA) for brensocatib for patients with bronchiectasis and granted the application Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 12, 2025. Insmed continues to expect brensocatib will launch in the U.S. in the third quarter, if approved. Regulatory submissions for brensocatib in the EU, UK, and Japan are planned for 2025, with commercial launches anticipated in 2026, pending approval in each territory. The Phase 2b BiRCh study of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) remains on track to report topline results by the end of 2025. The Company has initiated dosing of patients in the Phase 2b CEDAR study of brensocatib in patients with hidradenitis suppurativa (HS). TPIP Insmed presented additional lung imaging data from the Phase 2a study of treprostinil palmitil inhalation powder (TPIP) in pulmonary hypertension associated with interstitial lung disease (PH-ILD) at the Pulmonary Vascular Research Institute's 2025 Annual World Congress on February 1, 2025. Treatment with TPIP resulted in a significant increase in the fraction of blood volume in small arteries and a directional improvement in small-to-large artery volume ratio versus placebo, suggesting small vessel vasodilation and improved pulmonary arteriole recruitment. In addition, a numerical decrease in high-attenuation abnormality score was observed in patients treated with TPIP. The Company plans to initiate a Phase 3 study of TPIP in patients with PH-ILD in the second half of 2025. Enrollment in the Phase 2b study of TPIP in pulmonary arterial hypertension (PAH) completed in December 2024, with 102 patients randomized. Insmed continues to anticipate topline data from the study in the middle of 2025, ahead of the anticipated U.S. launch of brensocatib. Gene Therapy The Company received clearance from the FDA for its investigational new drug (IND) application for INS1201, an intrathecally-delivered gene therapy for patients with Duchenne muscular dystrophy (DMD), in December 2024. Insmed plans to initiate a clinical study of INS1201 in the first half 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 being pursued. The Company anticipates submitting an average of one to two INDs per year from its pre-clinical research program. Insmed continues to anticipate the totality of its pre-clinical research programs will comprise less than 20% of overall expenditure. Fourth-Quarter and Full-Year 2024 Financial Results The following table summarizes fourth-quarter and full-year 2024 and 2023 revenues and revenue growth for ARIKAYCE across all commercial regions: Three Months EndedDecember 31, Twelve Months EndedDecember 31, (in millions) 2024 2023 Growth 2024 2023 Growth U.S. $67.8 $58.3 16.4 % $254.8 $224.2 13.7 % Japan 30.7 21.0 46.6 % 87.7 65.7 33.4 % Europe & Rest of World 5.9 4.5 32.5 % 21.2 15.3 38.8 % Total Revenues $104.4 $83.7 24.8 % $363.7 $305.2 19.2 % Cost of product revenues (excluding amortization of intangibles) was $26.2 million for the fourth quarter of 2024, compared to $18.4 million for the fourth quarter of 2023. For the full-year 2024, cost of product revenues (excluding amortization of intangibles) was $85.7 million compared to $65.6 million for the full-year 2023. The increase in cost of product revenues in the fourth-quarter and full-year primarily reflects growth in ARIKAYCE sales. Research and development (R&D) expenses were $179.7 million for the fourth quarter of 2024, compared to $137.0 million for the fourth quarter of 2023. For the full-year 2024, R&D expenses were $598.4 million compared to $571.0 million for the full-year 2023. The increase in R&D expenses for the fourth-quarter and full-year was primarily related to increases in compensation and benefit-related expenses, as well as stock-based compensation, due to an increase in headcount. Selling, general and administrative (SG&A) expenses for the fourth quarter of 2024 were $142.5 million, compared to $89.5 million for the fourth quarter of 2023. For the full-year 2024, SG&A expenses were $461.1 million, compared to $344.5 million for the full-year 2023. The increase in SG&A expenses for the fourth-quarter and full-year was primarily related to increases in compensation and benefit-related expenses, as well as stock-based compensation, due to an increase in headcount in preparation for the anticipated launch of brensocatib in the U.S., pending regulatory approval. For the fourth-quarter 2024, Insmed reported a net loss of $235.5 million, or $1.32 per share, compared to a net loss of $186.1 million, or $1.28 per share, for the fourth-quarter 2023. For the full-year 2024, Insmed reported a net loss of $913.8 million, or $5.57 per share, compared to a net loss of $749.6 million, or $5.34 per share, for the full-year 2023. Balance Sheet, Financial Guidance, and Planned Investments As of December 31, 2024, Insmed had cash, cash equivalents, and marketable securities totaling approximately $1.4 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 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 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;(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 clinical trial program for INS1201 in DMD; and(vii) continued development of its pre-clinical research programs. Conference Call Insmed will host a conference call beginning today, February 20, 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 February 27, 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 EndedDecember 31, Twelve Months EndedDecember 31, 2024 2023 2024 2023 Product revenues, net $ 104,442 $ 83,693 $ 363,707 $ 305,208 Operating expenses: Cost of product revenues (excluding amortization of intangible assets) 26,151 18,443 85,742 65,573 Research and development 179,727 137,029 598,367 571,011 Selling, general and administrative 142,515 89,530 461,116 344,501 Amortization of intangible assets 1,263 1,263 5,052 5,052 Change in fair value of deferred and contingent consideration liabilities (14,800) 15,700 91,682 28,697 Total operating expenses 334,856 261,965 1,241,959 1,014,834 Operating loss (230,414) (178,272) (878,252) (709,626) Investment income 17,257 9,853 53,307 42,132 Interest expense (21,550) (20,784) (84,913) (81,694) Change in fair value of interest rate swap 870 1,970 (236) 320 Other (expense) income, net (445) 2,170 29 1,856 Loss before income taxes (234,282) (185,063) (910,065) (747,012) Provision for income taxes 1,266 998 3,707 2,555 Net loss $ (235,548) $ (186,061) $ (913,772) $ (749,567) Basic and diluted net loss per share $ (1.32) $ (1.28) $ (5.57) $ (5.34) Weighted average basic and diluted common shares outstanding 179,021 144,806 164,043 140,433 INSMED INCORPORATED Consolidated Balance Sheets (in thousands, except par value and share data) As of As of December 31, 2024 December 31, 2023 Assets Current assets: Cash and cash equivalents $ 555,030 $ 482,374 Marketable securities 878,796 298,073 Accounts receivable 52,012 41,189 Inventory 98,578 83,248 Prepaid expenses and other current assets 37,245 24,179 Total current assets 1,621,661 929,063 Fixed assets, net 80,052 65,384 Finance lease right-of-use assets 18,273 20,985 Operating lease right-of-use assets 17,257 18,017 Intangibles, net 58,652 63,704 Goodwill 136,110 136,110 Other assets 93,226 96,574 Total assets $ 2,025,231 $ 1,329,837 Liabilities and shareholders' equity Current liabilities: Accounts payable and accrued liabilities $ 285,209 $ 214,987 Finance lease liabilities 2,961 2,610 Operating lease liabilities 9,358 8,032 Total current liabilities 297,528 225,629 Debt, long-term 1,103,382 1,155,313 Royalty financing agreement 161,067 155,034 Contingent consideration 144,200 84,600 Finance lease liabilities, long-term 24,064 27,026 Operating lease liabilities, long-term 9,112 11,013 Other long-term liabilities 499 3,145 Total liabilities 1,739,852 1,661,760 Shareholders' equity: Common stock, $0.01 par value; 500,000,000 authorized shares, 179,382,635 and 147,977,960 issued and outstanding shares at December 31, 2024 and December 31, 2023, respectively 1,794 1,480 Additional paid-in capital 4,645,791 3,113,487 Accumulated deficit (4,359,917) (3,446,145) Accumulated other comprehensive loss (2,289) (745) Total shareholders' equity (deficit) 285,379 (331,923) Total liabilities and shareholders' equity (deficit) $ 2,025,231 $ 1,329,837 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 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 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 1800FDA1088. 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 pre-clinical research 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 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 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 DunnVice President, Investor Relations(646) 812-4030[email protected] Michael V. Morabito, Ph.D.Director, Investor Relations(917) 936-8430[email protected] Gianna De PalmaManager, Investor Relations(973) 886-2236[email protected] Media: Mandy FaheyVice President, Corporate Communications(732) 718-3621[email protected] SOURCE Insmed Incorporated WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In

Insmed Reports Fourth-Quarter and Full-Year 2024 Financial Results and Provides Business Update

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