INSMED Inc

Previous wording

Prior to 2019, we had not generated significant revenue and, through March 31, 2025, we had an accumulated deficit of $4.6 billion. We have financed our operations primarily through the public offerings of our equity securities, debt financings and revenue interest financings. Although it is difficult to predict our future funding requirements, based upon our current operating plan, we anticipate that our cash and cash equivalents and marketable securities as of March 31, 2025 will enable us to fund our operations for at least the next 12 months.

Current wording

Prior to 2019, we had not generated significant revenue and, through June 30, 2025, we had an accumulated deficit of $4.9 billion. We have financed our operations primarily through the public offerings of our equity securities, debt financings and revenue interest financings. Although it is difficult to predict our future funding requirements, based upon our current operating plan, we anticipate that our cash and cash equivalents and marketable securities as of June 30, 2025 will enable us to fund our operations for at least the next 12 months.

Previous wording

•Regulatory submissions for brensocatib in Europe and the UK have been accepted, with submission in Japan planned for 2025. Insmed anticipates commercial launches for each territory in 2026, pending approval.

Current wording

•Regulatory submissions for brensocatib in Europe and the UK have been accepted, with submission in Japan planned for the second half of 2025. Insmed anticipates commercial launches for each territory in 2026, pending approval.

Previous wording

•We completed enrollment in and anticipate reporting topline data from the Phase 2b study of brensocatib in patients with CRSsNP, which we refer to as the BiRCh trial, by the end of 2025.

Current wording

•We completed enrollment in and anticipate topline data from the Phase 2b study of brensocatib in patients with CRSsNP, which we refer to as the BiRCh trial, by the end of 2025.

Previous wording

•We initiated a Phase 2b study of brensocatib in patients with HS, which we refer to as the CEDAR trial, in December 2024.

Current wording

•We initiated a Phase 2b study of brensocatib in patients with HS, which we refer to as the CEDAR trial, in December 2024. We anticipate the interim futility analysis from the first 100 patients to complete Week 16 of the trial in the first quarter of 2026.

Previous wording

•In the fourth quarter of 2024, we received clearance from the FDA for our investigational new drug (IND) application for INS1201, an intrathecally delivered gene therapy for patients with DMD. We anticipate dosing our first patient in the Phase 1 ASCEND trial in the second quarter of 2025.

Current wording

•In the fourth quarter of 2024, we received clearance from the FDA for our investigational new drug (IND) application for INS1201, an intrathecally delivered gene therapy for patients with DMD. We dosed our first patient in the Phase 1 ASCEND trial in July 2025.

Previous wording

We strive to develop and commercialize first- and best-in-class therapies that serve patient communities where the need is greatest. Our first product, ARIKAYCE, is approved in the US as ARIKAYCE® (amikacin liposome inhalation suspension), in Europe as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion and in Japan as ARIKAYCE inhalation 590mg (amikacin sulfate inhalation drug product). We are not aware of any other approved inhaled therapies specifically indicated to treat MAC lung disease in North America, Europe or Japan. We believe that ARIKAYCE has the potential to prove beneficial in other patients with refractory MAC. Our product candidates are brensocatib, our Phase 3 product candidate that we are developing for patients with bronchiectasis and other neutrophil-mediated diseases, TPIP, our Phase 2 product candidate that may offer a differentiated product profile for patients with PH-ILD and PAH, and INS1201, our intrathecally delivered gene therapy product candidate for patients with DMD. We announced positive topline results from our Phase 3 ASPEN trial of brensocatib in May 2024 and the acceptance by the FDA of our NDA, with priority review granted, for brensocatib in patients with bronchiectasis in February 2025, which we anticipate will be followed by filings with the European and Japanese regulatory authorities. We are also advancing our pre-clinical research programs encompassing a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.

Current wording

We strive to develop and commercialize first- and best-in-class therapies that serve patient communities where the need is greatest. Our first product, ARIKAYCE, is approved in the US as ARIKAYCE® (amikacin liposome inhalation suspension), in Europe as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion and in Japan as ARIKAYCE inhalation 590mg (amikacin sulfate inhalation drug product). We are not aware of any other approved inhaled therapies specifically indicated to treat MAC lung disease in North America, Europe or Japan. We believe that ARIKAYCE has the potential to prove beneficial in other patients with refractory MAC lung disease. Our product candidates are brensocatib, our Phase 3 product candidate that we are developing for patients with bronchiectasis and other neutrophil-mediated diseases, TPIP, our Phase 2 product candidate that may offer a differentiated product profile for patients with PH-ILD and PAH, and INS1201, our intrathecally delivered gene therapy product candidate for patients with DMD. We announced positive topline results from our Phase 3 ASPEN trial of brensocatib in May 2024 and the acceptance by the FDA of our NDA, with priority review granted, for brensocatib in patients with bronchiectasis in February 2025. Regulatory filings for brensocatib in Europe and the UK have also been accepted and submission in Japan is planned for the second half of 2025. We are also advancing our pre-clinical research programs encompassing a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.

Previous wording

In October 2021, the EMA’s Paediatric Committee approved the brensocatib Pediatric Investigational Plan for the treatment of patients with NCFBE. As a result, the ASPEN trial includes 41 adolescent patients between ages 12 to 17, which will fulfill the pediatric study requirements to support marketing applications in this patient population in the US, Europe and Japan.

Current wording

In October 2021, the EMA’s Paediatric Committee approved the brensocatib Pediatric Investigational Plan for the treatment of patients with NCFBE. As a result, the ASPEN trial included 41 adolescent patients between ages 12 to 17, which will fulfill the pediatric study requirements to support marketing applications in this patient population in the US, Europe and Japan.

Previous wording

We announced topline data for the WILLOW study in February 2020 and full data for the WILLOW study in June 2020. In September 2020, final results from the WILLOW study were published online in the New England Journal of Medicine. The data demonstrate that the WILLOW study met its primary endpoint of time to first pulmonary exacerbation over the 24-week treatment period for both the 10 mg and 25 mg dosage groups of brensocatib compared to placebo (p=0.027, p=0.044, respectively). The risk of exacerbation at any time during the trial was reduced by 42% for the 10 mg group versus placebo (HR 0.58, p=0.029) and by 38% for the 25 mg group versus placebo (HR 0.62, p=0.046). In addition, treatment with brensocatib 10 mg resulted in a significant reduction in the rate of pulmonary exacerbations, a key secondary endpoint, versus placebo. Specifically, patients treated with brensocatib experienced a 36% reduction in the 10 mg arm (p=0.041) and a 25% reduction in the 25 mg arm (p=0.167) versus placebo. Change in concentration of active neutrophil elastase in sputum versus placebo from baseline to the end of the treatment period was also statistically significant (p=0.034 for 10 mg, p=0.021 for 25 mg). Brensocatib was generally well-tolerated in the study. Rates of AEs leading to discontinuation in patients treated with placebo, brensocatib 10 mg, and brensocatib 25 mg were 10.6%, 7.4%, and 6.7%, respectively.

Current wording

We announced topline data for the WILLOW study in February 2020 and full data for the WILLOW study in June 2020. In September 2020, final results from the WILLOW study were published online in the New England Journal of Medicine(NEJM). The data demonstrate that the WILLOW study met its primary endpoint of time to first pulmonary exacerbation over the 24-week treatment period for both the 10 mg and 25 mg dosage groups of brensocatib compared to placebo (p=0.027, p=0.044, respectively). The risk of exacerbation at any time during the trial was reduced by 42% for the 10 mg group versus placebo (HR 0.58, p=0.029) and by 38% for the 25 mg group versus placebo (HR 0.62, p=0.046). In addition, treatment with brensocatib 10 mg resulted in a significant reduction in the rate of pulmonary exacerbations, a key secondary endpoint, versus placebo. Specifically, patients treated with brensocatib experienced a 36% reduction in the 10 mg arm (p=0.041) and a 25% reduction in the 25 mg arm (p=0.167) versus placebo. Change in concentration of active neutrophil elastase in sputum versus placebo from baseline to the end of the treatment period was also statistically significant (p=0.034 for

Previous wording

We announced positive topline results from the ASPEN trial in May 2024. Results from the ASPEN trial were published in the New England Journal of Medicine in April 2025. The primary efficacy analysis included data from 1,680 adult patients and 41 adolescent patients. Brensocatib was well-tolerated in the study. In addition, the study met its primary endpoint, with both dosage strengths of brensocatib demonstrating statistically significant reductions in the annualized rate of adjudicated PEs versus placebo. The study also met several of its prespecified secondary endpoints with statistical significance. In February 2025, the FDA accepted our NDA, with priority review granted, for brensocatib in patients with bronchiectasis. The FDA indicated that it does not plan to hold an advisory committee meeting to discuss the NDA.

Current wording

We announced positive topline results from the ASPEN trial in May 2024. Results from the ASPEN trial were published in the NEJM in April 2025. The primary efficacy analysis included data from 1,680 adult patients and 41 adolescent patients. Brensocatib was well-tolerated in the study. In addition, the study met its primary endpoint, with both dosage strengths of brensocatib demonstrating statistically significant reductions in the annualized rate of adjudicated PEs versus placebo. The study also met several of its prespecified secondary endpoints with statistical significance. In February 2025, the FDA accepted our NDA, with priority review granted, for brensocatib in patients with bronchiectasis. The FDA indicated that it does not plan to hold an advisory committee meeting to discuss the NDA.

Previous wording

We are conducting further studies to explore the potential of brensocatib in additional neutrophil-mediated diseases, including CRSsNP and HS. CRSsNP currently has one approved pharmacological therapy (corticosteriod nasal spray); however, many patients do not respond to corticosteroids or endoscopic sinus surgery. The Phase 2b BiRCh trial of brensocatib in patients with CRSsNP is underway, with enrollment of 288 patients completed. We anticipate reporting topline data from the BiRCh trial by the end of 2025. In the fourth quarter of 2024, we initiated a Phase 2b study of brensocatib in patients with HS.

Current wording

We are conducting further studies to explore the potential of brensocatib in additional neutrophil-mediated diseases, including CRSsNP and HS. CRSsNP currently has one approved pharmacological therapy (corticosteriod nasal spray); however, many patients do not respond to corticosteroids or endoscopic sinus surgery. The Phase 2b BiRCh trial of brensocatib in patients with CRSsNP is underway, with enrollment of 288 patients completed. We anticipate topline data from the BiRCh trial by the end of 2025. In the fourth quarter of 2024, we initiated a Phase 2b study of brensocatib in patients with HS. We anticipate the interim futility analysis from the first 100 patients to complete Week 16 of the trial in the first quarter of 2026.

Previous wording

In the fourth quarter of 2024, we received clearance from the FDA for our IND application for INS1201, a microdystrophin adeno-associated virus gene replacement therapy for patients with DMD. Administered intrathecally, this approach has the potential to target both skeletal and cardiac muscles at lower doses than intravenous DMD gene therapies. We anticipate dosing our first patient in the Phase 1 ASCEND trial in the second quarter of 2025.

Current wording

In the fourth quarter of 2024, we received clearance from the FDA for our IND application for INS1201, a microdystrophin adeno-associated virus gene replacement therapy for patients with DMD. Administered intrathecally, this approach has the potential to target both skeletal and cardiac muscles at lower doses than intravenous DMD gene therapies. We dosed our first patient in the Phase 1 ASCEND trial in July 2025.

Previous wording

•Product revenues, net, increased $17.3 million, or 22.9%, as compared to the same period in the prior year as a result of the growth in ARIKAYCE sales;

Current wording

•Product revenues, net, increased $17.1 million, or 18.9%, as compared to the same period in the prior year as a result of the growth in ARIKAYCE sales;

Previous wording

•Cost of product revenues (excluding amortization of intangible assets) increased $3.8 million, or 21.9%, as compared to the same period in the prior year as a result of the growth in ARIKAYCE sales discussed above;

Current wording

•Cost of product revenues (excluding amortization of intangible assets) increased $7.1 million, or 33.9%, as compared to the same period in the prior year primarily as a result of the growth in ARIKAYCE sales discussed above;

Previous wording

•SG&A expenses increased$54.4 million, or 58.5%, as compared to the same period in the prior year primarily as a result of increases in compensation and benefit-related expensesand stock-based compensation costs;

Current wording

•SG&A expenses increased $48.2 million, or 45.2%, as compared to the same period in the prior year primarily as a result of increases in compensation and benefit-related expensesand stock-based compensation costs;

Previous wording

•Investment income increased $5.1 million, or 58.3%, as compared to the same period in the prior year primarily as a result of the increase in our average cash and cash equivalents and marketable securities balances; and

Current wording

•Investment income increased $2.9 million, or 28.6%, as compared to the same period in the prior year primarily as a result of the increase in our average cash and cash equivalents and marketable securities balances; and

Previous wording

Product revenues, net, for the three months ended March 31, 2025 were $92.8 million as compared to $75.5 million forthe same period in 2024, an increase of $17.3 million, or 22.9%. This increase was a result of the growth in sales of ARIKAYCE in the US, Japan, and Europe and the rest of the world.

Current wording

Product revenues, net, for the three months ended June 30, 2025 were $107.4 million as compared to $90.3 million forthe same period in 2024, an increase of $17.1 million, or 18.9%. This increase was a result of the growth in sales of ARIKAYCE in the US, Japan, and Europe and rest of world.

Previous wording

Cost of product revenues (excluding amortization of intangible assets) were $21.3 million for the three months ended March 31, 2025 as compared to $17.5 million forthe same period in 2024, an increase of $3.8 million, or 21.9%. This increase was primarily attributable to the increase in total product revenues discussed above.

Current wording

Cost of product revenues (excluding amortization of intangible assets) were $28.1 million for the three months ended June 30, 2025 as compared to $21.0 million forthe same period in 2024, an increase of $7.1 million, or 33.9%. This increase was primarily attributable to the increase in total product revenues discussed above.

Previous wording

R&D expenses were $152.6 million for the three months ended March 31, 2025 as compared to $121.1 million for the same period in 2024, an increase of$31.5 million, or 26.0%. This increase was primarily due to a $19.1 million increasein compensation and benefit-related expenses and stock-based compensation costs due to an increase in headcount and a $7.7 million increase in manufacturing expenses.

Current wording

R&D expenses were $177.2 million for the three months ended June 30, 2025 as compared to $146.7 million for the same period in 2024, an increase of$30.4 million, or 20.7%. This increase was primarily due to a $25.2 million increase in manufacturing expenses and a $17.2 million increasein compensation and benefit-related expenses and stock-based compensation costs due to an increase in headcount.

Previous wording

SG&A expenses were $147.5 million for the three months ended March 31, 2025 as compared to $93.1 million for the same period in 2024, an increase of $54.4 million, or 58.5%. This increase was primarily due to a $33.0 million increase in compensation and benefit-related expenses and stock-based compensation costs due to an increase in headcount and a$15.9 million increase in professional fees and other external expenses, both driven by commercial readiness activities for brensocatib. We expect SG&A expenses to increase in 2025 relative to 2024 due, in part, to commercial readiness activities, and commercial activities for brensocatib, if approved.

Current wording

SG&A expenses were $154.8 million for the three months ended June 30, 2025 as compared to $106.6 million for the same period in 2024, an increase of $48.2 million, or 45.2%. This increase was primarily due to a $31.3 million increase in compensation and benefit-related expenses and stock-based compensation costs due to an increase in headcount and an$11.8 million increase in professional fees and other external expenses, both driven by commercial readiness activities for brensocatib. We expect SG&A expenses to increase in 2025 relative to 2024 due, in part, to commercial readiness activities, and commercial activities for brensocatib, if approved.

Previous wording

The change in fair value of deferred and contingent consideration for the three months ended March 31, 2025 was $18.3 million and was primarily due to the increase in our share price. The change is related to the fair value of the potential future consideration to be paid to former equityholders of the businesses we acquired.

Current wording

The change in fair value of deferred and contingent consideration liabilities for the three months ended June 30, 2025 was $59.0 million and was primarily due to the increase in our share price. The change is related to the fair value of the potential future consideration to be paid to former equityholders of the businesses we acquired.

Previous wording

Interest expense for the three months ended March 31, 2025 was $21.6 million as compared to $21.0 million for the same period in 2024, an increase of $0.5 million, or 2.5%. This increase was primarily due to interest related to the Swap Contract in 2024. See Note 10 - Debtand Note 11 - Royalty Financing Agreement in this Quarterly Report on Form 10-Q for further details.

Current wording

Interest expense for the three months ended June 30, 2025 was $21.2 million as compared to $21.3 million for the same period in 2024. See Note 10 - Debtand Note 11 - Royalty Financing Agreement in this Quarterly Report on Form 10-Q for further details.

Previous wording

•R&D expenses increased$31.5 million, or 26.0%, as compared to the same period in the prior year primarily as a result ofincreases in compensation and benefit-related expensesand stock-based compensation costs;

Current wording

•R&D expenses increased $61.9 million, or 23.1%, as compared to the same period in the prior year primarily as a result ofincreases in compensation and benefit-related expensesand stock-based compensation costs;

Previous wording

•Interest expense increased $0.5 million, or 2.5%, as compared to the same period in the prior year primarily as a result of the interest related to the Swap Contract in 2024.

Current wording

•Interest expense increased $0.5 million, or 1.2%, as compared to the same period in the prior year primarily as a result of the interest income related to the Swap Contract in 2024.

Previous wording

In May 2024, we completed an underwritten offering of 14,514,562 shares of our common stock at a public offering price of $51.50 per share. 1,893,203 of the shares of common stock were issued pursuant to the exercise in full of the underwriters' option to purchase additional shares. Our net proceeds from the sale of the shares, after deducting underwriting discounts and estimated offering expenses of $34.3 million, were $713.2 million.

Current wording

In May 2024, we completed an underwritten offering of 14,514,562 shares of our common stock at a public offering price of $51.50 per share. 1,893,203 of the shares of common stock were issued pursuant to the exercise in full of the underwriters' option to purchase additional shares. Our net proceeds from the sale of the shares, after deducting underwriting discounts and offering expenses of $34.3 million, were $713.2 million.

Previous wording

We may need to raise additional capital to fund our operations, the continued commercialization of ARIKAYCE, launch readiness activities for the potential launch of brensocatib for the treatment of patients with bronchiectasis, if approved, clinical trials for brensocatib, TPIP, and our future product candidates, and to develop, acquire, in-license or co-promote other products or product candidates, including those that address orphan or rare diseases. While we believe we currently have sufficient funds to meet our financial needs for at least the next 12 months, we may opportunistically raise additional capital and may do so through equity or debt financing(s), strategic transactions or otherwise. Our cash requirements for the next 12 months will be impacted by a number of factors, the most significant of which we expect to be expenses related to our commercialization efforts for ARIKAYCE and if approved, brensocatib, development costs for our clinical-stage assets, and, to a lesser extent, our pre-clinical research programs.

Current wording

We may need to raise additional capital to fund our operations, the continued commercialization of ARIKAYCE, launch readiness activities for the potential launch of brensocatib for the treatment of patients with bronchiectasis, if approved, clinical trials for brensocatib, TPIP, INS1201, and our future product candidates, and to develop, acquire, in-license or co-promote other products or product candidates, including those that address orphan or rare diseases. While we believe we currently have sufficient funds to meet our financial needs for at least the next 12 months, we may opportunistically raise additional capital and may do so through equity or debt financing(s), strategic transactions or otherwise. Our cash requirements for the next 12 months will be impacted by a number of factors, the most significant of which we expect to be expenses related to our commercialization efforts for ARIKAYCE and if approved, brensocatib, development costs for our clinical-stage assets, and, to a lesser extent, our pre-clinical research programs.

Previous wording

As of March 31, 2025, we had cash and cash equivalents of $403.2 million, as compared to $555.0 million as of December 31, 2024. In addition, as of March 31, 2025, we had marketable securities of$796.2 million, as compared to$878.8 million as of December 31, 2024. Thedecrease of $151.8 million in cash and cash equivalents anddecrease of $82.6 million in marketable securitieswas primarily due to our cash used in operating activities. Our working capital was $1.2 billion as of March 31, 2025, as compared with $1.3 billion as of December 31, 2024.

Current wording

As of June 30, 2025, we had cash and cash equivalents of $1,284.3 million, as compared to $555.0 million as of December 31, 2024. This increase of $729.3 million in cash and cash equivalents was primarily due to the June 2025 underwritten offering of common stock, which provided us with $823.6 million in net proceeds, partially offset by our cash used in operating activities. In addition, as of June 30, 2025, we had marketable securities of$572.4 million, as compared to$878.8 million as of December 31, 2024. This decrease of $306.4 million in marketable securitieswas primarily due to our cash used in operating activities. Our working capital was $1.8 billion as of June 30, 2025, as compared with $1.3 billion as of December 31, 2024.

Previous wording

Net cash used in operating activities was $262.1 million and $184.0 million for the three months ended March 31, 2025 and 2024, respectively. The net cash used in operating activities during the three months ended March 31, 2025 and 2024 was primarily for the commercial, clinical, and manufacturing activities related to ARIKAYCE, commercial readiness activities for brensocatib, as well as other SG&A expenses and clinical trial expenses related to brensocatib and TPIP. The increase in cash used in operating activities for the three months ended March 31, 2025 as compared to the same period in 2024 was primarily due to the increase in net loss, excluding the adjustments to reconcile net loss to net cash used in operating activities.

Current wording

Net cash used in operating activities was $467.7 million and $307.0 million for the six months ended June 30, 2025 and 2024, respectively. The net cash used in operating activities during the six months ended June 30, 2025 and 2024 was primarily for the commercial, clinical, and manufacturing activities related to ARIKAYCE, commercial readiness activities for brensocatib, as well as other SG&A expenses and clinical trial expenses related to brensocatib and TPIP. The increase in cash used in operating activities for the six months ended June 30, 2025 as compared to the same period in 2024 was primarily due to the increase in net loss, excluding the adjustments to reconcile net loss to net cash used in operating activities.

Previous wording

Net cash provided by investing activities was $80.4 million and $295.3 million for the three months ended March 31, 2025 and 2024, respectively. During the three months ended March 31, 2025, net cash provided by investing activities consisted primarily of maturities of marketable securities, mostly offset by purchases of marketable securities. Net cash provided by investing activities during thethree months ended March 31, 2024, primarily consisted of maturities of marketable securities.

Current wording

Net cash provided by investing activities was $308.2 million and $288.5 million for the six months ended June 30, 2025 and 2024, respectively. During the six months ended June 30, 2025, net cash provided by investing activities consisted primarily of maturities of marketable securities, partially offset by purchases of marketable securities. During the six months ended June 30, 2024, net cash provided by investing activities primarily consisted of maturities of marketable securities.

Previous wording

Net cash provided by financing activities was $29.0 million and $3.0 million for the three months ended March 31, 2025 and 2024, respectively. The increase in cash provided by financing activities for thethree months ended March 31, 2025 as compared to the same period in 2024 isdue to the proceeds from stock options and our ESPP.

Current wording

Net cash provided by financing activities was $886.8 million and $784.5 million for the six months ended June 30, 2025 and 2024, respectively. During thesix months ended June 30, 2025 and 2024, net cash provided by financing activities consisted primarily of proceeds from the issuance of common stock in our underwritten public equity offerings, and proceeds from the exercise of stock options and ESPP.

Previous wording

There were no material changes outside of the ordinary course of business in our contractual obligations during the three months ended March 31, 2025 from those disclosed in Item 7, “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Liquidity and Capital Resources—Contractual Obligations” in our Annual Report on Form 10-K for the year ended December 31, 2024.

Current wording

There were no material changes outside of the ordinary course of business in our contractual obligations during the six months ended June 30, 2025 from those disclosed in Item 7, “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Liquidity and Capital Resources—Contractual Obligations” in our Annual Report on Form 10-K for the year ended December 31, 2024.