Insmed, Inc. (NASDAQ:INSM) Q3 2023 Earnings Conference Call October 26, 2023 8:30 AM ET Company Participants Bryan Dunn - IR William Lewis - President, CEO & Chairman Sara Bonstein - CFO Conference Call Participants Andrea Tan - Goldman Sachs Group Jennifer Kim - Cantor Fitzgerald & Co. Vamil Divan - Guggenheim Securities Ritu Baral - TD Cowen Liisa Bayko - Evercore ISI Joseph Schwartz - Leerink Partners Jason Zemansky – Bank of America Merrill Lynch Graig Suvannavejh - Mizuho Securities Leiyang Wang - Barc ...

Drug Development and Approvals - ARIKAYCE is approved in the US, Europe, and Japan for treating MAC lung disease, with accelerated approval received in September 2018[171] - The company plans to propose to the FDA that the Quality of Life – Bronchiectasis (QOL-B) respiratory domain PRO be the primary endpoint for the ENCORE study[174] - Enrollment in the ENCORE study is expected to reach 250 patients by the end of 2023, with topline data anticipated in 2025[174] - The Phase 3 ASPEN trial for brensocatib was completed in Q1 2023, with topline data expected in Q2 2024[174] - ARIKAYCE has received a total of 12 years of exclusivity from the FDA for its indication due to orphan drug and QIDP designations[177] - The company is preparing for the potential launch of brensocatib for bronchiectasis patients, pending approval[174] - The FDA approved a supplemental new drug application for ARIKAYCE, enhancing its label with efficacy data from the Phase 3 CONVERT study[179] - ARIKAYCE received accelerated approval from the FDA in September 2018 for treating refractory MAC lung disease, allowing the company to expand its revenue stream[180] - The ARISE trial demonstrated that 80.6% of patients in the ARIKAYCE arm achieved culture conversion by Month 6, compared to 63.9% in the comparator arm[190] - In the ARISE trial, 43.8% of ARIKAYCE-treated patients showed improvement in QOL-B respiratory scores, compared to 33.3% in the comparator arm[188] - The ASPEN Phase 3 trial for brensocatib has enrolled over 1,700 adult patients across approximately 460 sites in 40 countries, with topline data expected in Q2 2024[198] - Brensocatib received FDA breakthrough therapy designation for treating non-cystic fibrosis bronchiectasis (NCFBE), aimed at reducing exacerbations[200] - The WILLOW study demonstrated a 42% reduction in exacerbation risk for the 10 mg brensocatib group and a 38% reduction for the 25 mg group compared to placebo[203] - The company has commenced post-marketing confirmatory clinical trials for ARIKAYCE, including the ARISE and ENCORE trials, to fulfill FDA requirements for full approval[193] Financial Performance - Product revenues, net, increased by $11.3 million, or 16.7%, to $79.1 million for the three months ended September 30, 2023, driven by growth in ARIKAYCE sales[231] - R&D expenses rose by $9.3 million, or 9.3%, to $109.1 million for the three months ended September 30, 2023, primarily due to increases in compensation and benefit-related expenses[234] - SG&A expenses increased by $15.0 million, or 19.9%, to $90.6 million for the three months ended September 30, 2023, mainly due to higher compensation and professional fees[236] - Interest expense surged by $16.9 million to $20.3 million for the three months ended September 30, 2023, attributed to the Term Loan and Royalty Financing Agreement[240] - Investment income increased significantly to $10.6 million for the three months ended September 30, 2023, compared to $1.8 million in the same period in 2022, due to higher marketable securities and interest rates[239] - Total product revenues, net, for the nine months ended September 30, 2023, reached $221.5 million, a 19.1% increase from $186.1 million in the same period in 2022[242] - Cost of product revenues (excluding amortization of intangible assets) increased by $3.2 million, or 24.0%, to $16.7 million for the three months ended September 30, 2023[233] - R&D expenses increased to $434.0 million during the nine months ended September 30, 2023, from $272.8 million in the same period in 2022, representing a $161.2 million increase or 59.1%[245] - SG&A expenses rose to $255.0 million during the nine months ended September 30, 2023, up from $192.3 million in the same period in 2022, reflecting a $62.7 million increase or 32.6%[247] - Investment income increased to $32.3 million for the nine months ended September 30, 2023, compared to $2.8 million in the same period in 2022, due to an increase in marketable securities balance and interest rates[251] - Interest expense increased to $60.9 million for the nine months ended September 30, 2023, compared to $10.0 million in the same period in 2022, primarily due to the Term Loan and Royalty Financing Agreement[252] Cash Flow and Capital Management - Cash and cash equivalents decreased to $487.1 million as of September 30, 2023, from $1,074.0 million as of December 31, 2022, a decrease of $586.9 million[261] - Net cash used in operating activities was $405.4 million for the nine months ended September 30, 2023, compared to $297.3 million for the same period in 2022, primarily for commercial and clinical activities related to ARIKAYCE[262] - The company expects R&D expenses to continue to increase in 2023 relative to 2022, driven by clinical trial activities including the Phase 3 ASPEN trial of brensocatib[246] - The company issued 2,000,000 shares of common stock through its ATM program at a weighted-average public offering price of $19.35 per share, receiving net proceeds of $37.5 million[255] - The company may need to raise additional capital to fund operations and commercialization activities for ARIKAYCE and brensocatib, although it believes it has sufficient funds for at least the next 12 months[260] - Net cash used in investing activities increased to $225.4 million for the nine months ended September 30, 2023, compared to $54.9 million in the same period of 2022[263] - Net cash provided by financing activities decreased to $45.2 million for the nine months ended September 30, 2023, down from $49.9 million in 2022[264] - As of September 30, 2023, the company had $800.0 million of convertible notes outstanding, with interest rates of 1.75% for 2025 Convertible Notes and 0.75% for 2028 Convertible Notes[269] - The company has a $350 million Term Loan accruing interest at SOFR with a floor of 2.5% plus a margin of 7.75% per annum[269] - The Royalty Financing Agreement pays interest at 4% of ARIKAYCE global net sales prior to September 1, 2025, and 4.5% thereafter, along with 0.75% of brensocatib global net sales if approved[269] - As of September 30, 2023, the company's cash and cash equivalents were primarily in cash accounts and money market funds, with marketable securities invested in US treasury notes with an original maturity of greater than 90 days[268] Research and Development Initiatives - The company is advancing its gene therapy program in Duchenne muscular dystrophy (DMD) as part of its early-stage research efforts[173] - The company is actively evaluating in-licensing and acquisition opportunities for a broad range of rare diseases[173] - The company has acquired multiple preclinical stage companies focused on gene therapies for rare genetic disorders, enhancing its early-stage research capabilities[219] - The company expects topline results from the PH-ILD study to be shared in the first half of 2024[218] - There are currently no approved therapies for bronchiectasis in the US, Europe, or Japan, indicating a significant market opportunity[210] Operational Metrics - The discontinuation rate in the ARIKAYCE arm of the ARISE trial was 22.9%, compared to 7.8% in the comparator arm, with no new safety events observed[192] - The average reduction in pulmonary vascular resistance (PVR) in the ongoing PAH study was 21.5%, with 64% of patients experiencing reductions exceeding 65%[216] - TPIP, an investigational inhaled formulation, aims to reduce dosing frequency from four to nine times per day to once daily, potentially improving patient compliance[211] - In the Phase 1 study of TPIP, it was generally well tolerated, with most adverse events being mild and consistent with other inhaled prostanoid therapies[212] - The company plans to seek to increase the maximum tolerated dose of TPIP from 640 µg to up to 1,280 µg for certain PAH patients[216] Financial Condition and Risks - There were no material changes in contractual obligations during the nine months ended September 30, 2023, compared to the previous disclosures[265] - The company does not have any off-balance sheet arrangements that could materially affect its financial condition or results of operations[266] - Fluctuations in foreign currency exchange rates have not materially affected the company's results of operations during the nine months ended September 30, 2023, and 2022[270] - There have been no material changes to the company's critical accounting policies and estimates as disclosed in the previous annual report[267]

Insmed Incorporated (NASDAQ:INSM) Q2 2023 Earnings Conference Call August 3, 2023 8:30 AM ET Company Participants Bryan Dunn - Head, IR William Lewis - Chair & CEO Sara Bonstein - CFO Martina Flammer - Chief Medical Officer Conference Call Participants Judah Frommer - Credit Suisse Vamil Divan - Guggenheim Jennifer Kim - Cantor Fitzgerald Leon Wang - Barclays Jason Zemansky - Bank of America Stephen Willey - Stifel Liisa Bayko - Evercore Andrea Tan - Goldman Sachs Joseph Schwartz - Leerink Partners Operator ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number 000-30739 INSMED INCORPORATED (Exact name of registrant as specified in its charter) Virginia 54-1972729 (State or other jurisdiction ...

Insmed Incorporated (NASDAQ:INSM) Q1 2023 Results Conference Call May 4, 2023 8:30 AM ET Company Participants Bryan Dunn - Head of Investor Relations Will Lewis - Chair and Chief Executive Officer Sara Bonstein - Chief Financial Officer Martina Flammer - Chief Medical Officer Conference Call Participants Nick Lenard - JPMorgan Andrea Tan - Goldman Sachs Judah Frommer - Credit Suisse Jennifer Kim - Cantor Fitzgerald Leon Wang - Barclays Stephen Willey - Stifel Jason Zemansky - Bank of America Liisa Bayko - ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number 000-30739 INSMED INCORPORATED (Exact name of registrant as specified in its charter) Virginia 54-1972729 (State or other jurisdictio ...

Financial Data and Key Metrics Changes - Total net revenue for ARIKAYCE was $245.4 million for the full year 2022, reflecting a 30% growth over 2021 [26] - The company ended 2022 with $1.15 billion in cash and cash equivalents and marketable securities, positioning itself for a re-inflection point over the next 18 months [46][72] - Research and development expenses for the full year 2022 were $397.5 million, while SG&A expenses were $265.8 million [52] Business Line Data and Key Metrics Changes - ARIKAYCE revenue in the US was $186 million, $56.5 million in Japan, and $2.9 million in Europe and the rest of the world for 2022 [26] - The company anticipates global revenues for ARIKAYCE to be between $285 million and $300 million for the full year 2023 [51] - The gross to net in the US was approximately 13%, consistent with prior years [71] Market Data and Key Metrics Changes - The company expects a one-time price cut in Japan in the high single to mid-teen range, consistent with prior product launches [49] - Europe remains a small percentage of global revenues, with a one-time charge of $7.5 million recorded in Q4 2022 due to a lower-than-expected reimbursement price in France [70] Company Strategy and Development Direction - The company aims to evolve from addressing patient populations of tens of thousands to over a million, with each program representing first-in-class or best-in-class treatment [29] - The strategic approach involves using cash flow from advanced programs to fund medium to long-term development work, aiming to become a self-sustaining biotechnology company [7] - The company plans to initiate a Phase 2 trial of Brensocatib in chronic rhinosinusitis without nasal polyps in mid-2023 [59] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement for the future, anticipating the impact of multiple investments made over the last several years to begin materializing in 2023 [22] - The company is optimistic about the potential for growth in the US market, citing positive trends in key metrics such as enrollment forms and new patient starts [131] - Management noted that the fundamentals in Japan are strong, with expectations for growth in the second half of the year as COVID-related pressures ease [69][108] Other Important Information - The company plans to present at the American Thoracic Society International Conference, with eight abstracts accepted for presentation [37] - The ASPEN study is more than 50% larger than previous Phase 3 programs in this indication, with all adult screenings completed [129] Q&A Session Summary Question: How should we think about the magnitude of growth for ARIKAYCE in 2023? - Management acknowledged that Q4 was slightly lower than expected due to inventory shifts but remains positive about growth in the US as the market normalizes post-COVID [55] Question: What are the dynamics shaping expectations in key geographies? - Management indicated that Japan remains crucial, with strong demand expected to grow as lockdowns ease, while Europe continues to face pricing challenges [69][100] Question: How confident is the company in the PRO's ability to detect clinically meaningful changes? - Management expressed high confidence in the PRO based on extensive experience treating patients and the data collected [105][161]

February 23, 2023 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 timings discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors ...

Product Approvals and Development - ARIKAYCE received accelerated approval in the US in September 2018 for the treatment of refractory MAC lung disease, with subsequent approvals in Europe (October 2020) and Japan (March 2021) for similar indications [19][24][32]. - The company anticipates sharing topline efficacy and safety data from the ARISE trial in Q3 2023 and completing enrollment in the ENCORE trial by the end of 2023 [20][30]. - Brensocatib is in Phase 3 development for bronchiectasis and CF, with plans to advance CRSsNP into Phase 2 development in mid-2023 [20][21]. - The FDA granted ARIKAYCE a total of 12 years of exclusivity due to its orphan drug and Qualified Infectious Disease Product (QIDP) designations [25]. - The CONVERT study showed that 63.1% of patients on ARIKAYCE plus guideline-based therapy maintained durable culture conversion for three months off therapy compared to 0% on guideline-based therapy alone (p<0.0002) [34]. - The ARISE trial completed enrollment in Q4 2022, with a blinded treatment discontinuation rate of 15% reported in January 2023, and topline efficacy and safety data expected in Q3 2023 [37]. - The ENCORE trial is currently enrolling approximately 250 patients, with completion of enrollment anticipated by the end of 2023 [38]. - The estimated number of patients diagnosed with NTM lung disease in the US is between 95,000 and 115,000, with 12,000 to 17,000 patients refractory to treatment [42]. - The potential addressable market for bronchiectasis is estimated at 450,000 patients in the US, 400,000 in the European 5, and 150,000 in Japan [57]. - Brensocatib demonstrated a 42% reduction in the risk of exacerbation for the 10 mg group and a 38% reduction for the 25 mg group compared to placebo in the WILLOW study [51]. - The FDA granted breakthrough therapy designation for brensocatib for treating adult patients with non-cystic fibrosis bronchiectasis, expediting its development [47]. - The ASPEN trial for brensocatib is expected to enroll approximately 1,620 patients across 480 sites in 40 countries, with topline data anticipated in Q2 2024 [45]. Research and Development Initiatives - The company is actively evaluating in-licensing and acquisition opportunities for a broad range of rare diseases to complement its internal R&D efforts [22]. - The company plans to share interim data from the PH-ILD study in the second half of 2023 and topline results in the first half of 2024 [25]. - The company plans to explore brensocatib's potential in additional neutrophil-mediated diseases, including CRSsNP, with Phase 2 development anticipated in mid-2023 [55]. - Two ongoing Phase 2 studies for TPIP are assessing safety in PH-ILD and efficacy in PAH, with topline results from the PH-ILD study expected in the first half of 2024 [61][62]. - The company acquired a proprietary protein deimmunization platform and two preclinical stage companies in 2021 to enhance its gene therapy capabilities [63]. - The first IND filing is anticipated in the first half of 2023, with preclinical data expected in musculoskeletal and CNS indications [65]. Commercialization and Market Strategy - The company is advancing commercial readiness activities in 2023 in preparation for the potential launch of brensocatib [21]. - The company is working to ensure an uninterrupted supply of ARIKAYCE in Germany while negotiating pricing agreements with local health authorities [31]. - The company plans to rely on third-party manufacturers for the commercial supply of product candidates, with an estimated investment of approximately $99 million to increase ARIKAYCE production capacity [68]. - The company has invested significant resources in the commercialization of ARIKAYCE, which may exceed those required for more established technologies [217]. - The company may seek partnerships for marketing and distribution of ARIKAYCE, which could limit control over commercialization efforts [215]. Financial and Market Position - The company has a history of operating losses and expects to incur losses for the foreseeable future, impacting its financial condition [213]. - The company may need to raise additional funds to continue operations, facing uncertainties regarding capital access [213]. - The market price of the company's stock has been highly volatile, which could lead to shareholder litigation [213]. Regulatory Environment and Exclusivity - The FDA has 60 days to determine if an NDA or BLA is accepted for filing, with a goal to complete 90% of standard applications within 10 months [118]. - The FDA may require substantial post-approval testing, known as Phase 4 studies, to gather additional information on the drug's effects and side effects [121]. - The FDA's accelerated approval program allows for drugs to be approved based on surrogate endpoints, potentially reducing time to market [122]. - Fast track designation can expedite development and review for drugs intended to treat serious conditions with unmet medical needs [126]. - Breakthrough therapy designation provides intensive guidance on drug development and expedited review, with a response required from the FDA within 60 days [128]. - Drugs designated as Qualified Infectious Disease Products (QIDPs) may receive priority review and fast track designation, with ARIKAYCE being a notable example [129]. - Non-patent exclusivity for new chemical entities (NCEs) lasts for five years, preventing the FDA from accepting abbreviated NDAs during this period [134]. - A three-year non-patent exclusivity is granted for drugs with new clinical investigations essential for approval, covering only the new conditions of use [135]. - QIDP designation can extend non-patent exclusivities by five years, potentially allowing for a combined 12 years of exclusivity for drugs like ARIKAYCE [137]. - Reference product exclusivity prevents the FDA from accepting a BLA for a proposed biosimilar product for 4 years and from approving it for 12 years after the reference product's first licensure [138]. Workforce and Corporate Governance - As of December 31, 2022, the company had a total of 736 full-time employees, with 579 in the US, 85 in Europe, and 72 in Japan [194]. - The company anticipates increasing its headcount in 2023 [194]. - Women represent 38% of the executive team, 28% of the leadership team, 33% of the board of directors, and 51% of the overall workforce [199]. - The company has a compensation program designed to attract and retain talent, with total compensation generally positioned within a competitive range of the peer market median [196]. - The company is committed to equitable pay and conducts annual internal equity reviews to ensure a fair compensation system [199]. - The company has a cross-functional group focused on Environmental, Social, and Governance (ESG) considerations and strategy [200]. - The company supports several green measures and community service programs to improve sustainability efforts [201]. - The company has implemented flexible work arrangements for employees to manage business and personal responsibilities during COVID-19 [202]. - The company is dedicated to promoting patient advocacy and safety as part of its corporate governance [201]. - The company aims to ensure diverse succession plans in its workforce and board of directors [199]. Competitive Landscape - The company faces competition from various sectors, including established pharmaceutical and biotechnology firms, which may have more advanced products [97]. - There are currently no approved inhaled therapies for refractory NTM lung infections in North America, Europe, or Japan, positioning ARIKAYCE uniquely in the market [99]. - The company expects competition in gene therapy and protein engineering to intensify, impacting its market position if approved products are developed by competitors [100]. Intellectual Property and Licensing - The company holds over 450 patents related to its products, including 12 issued US patents for ARIKAYCE, with coverage expected through May 15, 2035 [70][75]. - The company has licensed multiple patents related to brensocatib, with the earliest expiring in March 2035 [79]. - TPIP is protected by several US patents expiring in October 2034, covering its use in treating pulmonary hypertension [80]. - The company has filed applications to register trademarks for INSMED and ARIKAYCE, with some registrations already granted [83]. - The licensing agreement with PARI includes rights to use the optimized Lamira Nebulizer System for ARIKAYCE, which is approved in the US, EU, and Japan [85]. - Milestone payments to PARI totaled €3.0 million upon FDA and EMA approvals of ARIKAYCE, with ongoing royalty payments in the mid-single digits on global net sales [87]. - The agreement with Resilience mandates a minimum payment of $6 million annually for ARIKAYCE production, with an initial term of five years starting in October 2018 [91]. - The investment to increase long-term production capacity with Patheon is estimated at approximately $99 million, with agreements remaining in effect for successive renewal terms [92]. - Cystic Fibrosis Foundation Therapeutics, Inc. funding agreements resulted in milestone payments owed totaling $13.4 million through 2025, with $4.9 million paid as of December 31, 2022 [93]. - The AZ License Agreement with AstraZeneca includes an upfront payment of $30 million and potential milestone payments up to $72.5 million for clinical development [96]. Regulatory Designations and Orphan Drug Status - ARIKAYCE has received orphan drug designation for treating NTM infections and bronchiectasis, qualifying the company for various development incentives [102]. - The European Commission granted orphan drug designation for ARIKAYCE for the treatment of NTM lung disease, promoting its development for conditions affecting fewer than 5 in 10,000 people in the EU [105]. - Orphan exclusivity lasts for 10 years, extendable by 2 years if a pediatric investigation plan is implemented, but can be reduced to 6 years if the product is sufficiently profitable [106]. - Orphan drug designation provides opportunities for fee reductions and exemptions for small and medium enterprises, as well as potential national benefits from EU member states [107]. - In Japan, ARIKAYCE did not qualify for orphan drug designation due to the estimated number of NTM patients exceeding 50,000 [110].

placebo -14.7% 95.6% 2.5% patients with insmed 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 timings discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, un ...