Insmed Provides Business Update at 43rd Annual J.P. Morgan Healthcare Conference -ARIKAYCE (amikacin liposome inhalation suspension) Exceeds the Upper End of Guidance Range for Full-Year 2024 with Unaudited Global Revenu

Insmed Provides Business Update at 43rd Annual J.P. Morgan Healthcare Conference

-ARIKAYCE (amikacin liposome inhalation suspension) Exceeds the Upper End of Guidance Range for Full-Year 2024 with Unaudited Global Revenues of Approximately $363.7 Million-

-2025 Global ARIKAYCE Revenues Expected to be Between $405 Million and $425 Million, Reflecting Continued Double-Digit Growth Compared to 2024-

-NDA for Brensocatib in Bronchiectasis Submitted to FDA in December 2024, Narrowing the Timing for Expected U.S. Launch to the Third Quarter of 2025, Pending Approval Under Priority Review-

-Enrollment for Phase 2 Study of TPIP in Patients with PAH Completed in December 2024; Expected Timing for Topline Data Moved Forward to Mid-2025-

-Phase 3 ENCORE Trial for ARIKAYCE in Patients with Newly Diagnosed or Recurrent MAC Lung Disease Fully Enrolled; Topline Data Anticipated in First Quarter of 2026-

-IND Cleared for Insmed's First Gene Therapy (INS1201) for Patients with DMD; First Patient Dosing Anticipated in the First Half of 2025-

BRIDGEWATER, N.J., January 10, 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 provided an update on the Company's commercial and clinical programs and its outlook for 2025. These updates will be discussed as part of the Company's

presentation at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 13, 2025, at 3:00 p.m. PT (6:00 p.m. ET).

"2024 was an extraordinary year for Insmed, and it is only the beginning of our journey. We believe the upcoming clinical and commercial catalysts have the potential to

redefine Insmed from a company that can serve approximately 30,000 patients today to one able to reach more than 2.5 million patients by the end of the decade," said Will Lewis, Chair and Chief Executive Officer of Insmed. "As we prepare for the

highly anticipated U.S. approval and launch of brensocatib in bronchiectasis in the third quarter of 2025, we continue to advance our additional clinical programs, including TPIP in PH-ILD and PAH, brensocatib in CRSsNP and HS, and our first gene

therapy in DMD. In parallel, we expect to continue to drive double-digit ARIKAYCE growth as we await the readout of ENCORE data, which has the potential to unlock a blockbuster opportunity for the brand."

Preliminary Full-Year 2024 Global Net Product Sales (Unaudited)

Based on preliminary unaudited financial information, the Company expects total global net product sales of ARIKAYCE to be approximately $363.7 million for full-year 2024.

This represents 19% year-over-year growth versus full-year 2023, including growth across each of our regions, as follows:

These preliminary unaudited results are subject to adjustment. Insmed will report its final and complete fourth-quarter and full-year 2024 financial results in late

February 2025. The actual results could be materially different from these preliminary unaudited financial results.

Progress and Anticipated Milestones by Program:

Pre-Clinical Programs

Presentation at the 43rd Annual J.P. Morgan Healthcare Conference

Will Lewis, Chair and Chief Executive Officer of Insmed, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 3:00

p.m. PT (6:00 p.m. ET). A live audio webcast of the presentation will be available on the Investor Relations section of the Company's website at www.insmed.com. A replay

will also be archived for a period of 30 days following the conclusion of the live event.

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

Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis,

CRSsNP, HS, 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

Treprostinil palmitil inhalation powder (TPIP) is a dry powder formulation of treprostinil palmitil, a treprostinil prodrug consisting of treprostinil linked by an ester

bond to a 16-carbon chain. Developed entirely in Insmed's laboratories, TPIP is a potentially highly differentiated prostanoid being evaluated for the treatment of patients with PAH, PH-ILD, and other rare and serious pulmonary disorders. TPIP is

administered in a capsule-based inhalation device. TPIP is an investigational drug product that has not been approved for any indication in any jurisdiction.

IMPORTANT SAFETY INFORMATION AND BOXED WARNING FOR ARIKAYCE IN THE U.S.

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

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

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

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.

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

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.

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 in ammatory conditions, including a therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease. The Company's early-stage 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