Insmed Incorporated · NASDAQ:INSM
Insmed — Investor Presentation (February 2025)
61 slides from Insmed's investor presentation, Feb 20, 2025
Slide 1: February 2025 Investor Presentation
February 2025 Investor Presentation
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements that involve substantial risks and uncertainties. “Forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a
Slide 3: Additional Disclaimers
Additional Disclaimers With respect to the blended and blinded data observed from the ongoing TPIP study in pulmonary arterial hypertension, the dose titration and efficacy analyses were based on data available as of April 1, 2024, and the safety analyses were based on data available as of January 2
Slide 4: Portfolio of First-in-Class and Potentially Best-in-Class Assets Across Each Stage of Development
Portfolio of First-in-Class and Potentially Best-in-Class* Assets Across Each Stage of Development Pre-clinical Phase 1 Phase 2 Phase 3 Approved ARIKAYCE® Refractory MAC All MAC Lung Disease Brensocatib Bronchiectasis CRSsNP HS TPIP PH-ILD PAH Pre-Clinical Research INS1202: ALS INS1203: Stargardt Di
Slide 5: Total Addressable Patients Has Potential to Grow 80x Through 2030
* If pipeline of assets are approved Bronchiectasis: non-cystic fibrosis bronchiectasis; MAC / MAC LD: Mycobacterium avium complex lung disease; CRSsNP: chronic rhinosinusitis without nasal polyps; PH-ILD: pulmonary hypertension due to interstitial lung disease; PAH: pulmonary arterial hypertension;
Slide 6: Potential Value Creation Will Be Shaped by Two Distinct Drivers
Potential Value Creation Will Be Shaped by Two Distinct Drivers * Pending regulatory approval for bronchiectasis indication; U.S. FDA target action date set for August 12, 2025 ** Guidance growth versus 2024 ARIKAYCE revenue results † Europe/EU5 comprised of France, Germany, Italy, Spain, and the Un
Slide 7: ARIKAYCE Refractory Mycobacterium avium complex lung disease
ARIKAYCE Refractory Mycobacterium avium complex lung disease A rare and chronic disease that can cause irreversible lung damage and is the most common form of NTM respiratory pathogen Mycobacterium avium complex lung disease Image source: Hendrix C, McCrary M, Hou R, Abate G. Diagnosis and Managemen
Slide 8: ARIKAYCE Expected to Deliver Double-Digit Revenue Growth in 2025
ARIKAYCE Expected to Deliver Double-Digit Revenue Growth in 20251 1 Guidance growth vs. 2024 ARIKAYCE revenue results EU5 comprised of France, Germany, Italy, Spain, and the United Kingdom Double-digit growth entering 7th year post launch +19% 2024 vs. 2023 +14% +39% +33% FY 2024 $364M Beat $340-$36
Slide 9: ARIKAYCE has the Potential to be the Best in MAC LD Treatment
ARIKAYCE has the Potential to be the Best in MAC LD Treatment MAC / MAC LD: Mycobacterium avium complex lung disease; TAM: Total Addressable Market †EU5 comprised of France, Germany, Italy, Spain, and the United Kingdom Note: Numbered footnote references related to patient prevalence can be found at
Slide 10: ARIKAYCE Phase 3 Program to Potentially Expand MAC Indication
ARIKAYCE Phase 3 Program to Potentially Expand MAC Indication Trial summary Key Endpoints Screening Double-Blind Registration PMR Trial ARIKAYCE + AZI* + ETH* Placebo + AZI* + ETH* Key Endpoints Month 13 Culture Negativity Endpoint Month 15 R Months 1-6 ARIKAYCE + AZI* + ETH* Off Treatment Months 1-
Slide 11: Brensocatib
Brensocatib Image sources: 1) Fraser CS, José RJ. Insights into Personalised Medicine in Bronchiectasis. Journal of Personalized Medicine. 2023; 13(1):133. https://doi.org/10.3390/jpm13010133. | 2) Rethinkbronchiectasis.com | 3) Yang Y, Zhang N, Crombruggen KV, Lan F, Hu G, Hong S, Bachert C. Differ
Slide 12: U.S. Bronchiectasis Market Primed for a Strong Brensocatib Launch
U.S. Bronchiectasis Market Primed for a Strong Brensocatib Launch* * Pending regulatory approval for bronchiectasis indication ** Since website launch in November 2023 through December 2024 † From February-October 2024; Commercial lives include Veterans Affairs, Tricare, and Dept. of Defense ‡ Highl
Slide 13: Brensocatib in Bronchiectasis Opportunity Could Reach >1M Patients at Launch
Brensocatib in Bronchiectasis Opportunity Could Reach >1M Patients at Launch* * Pending regulatory approval for bronchiectasis indication † Includes misdiagnosed, miscoded, undiagnosedNote: COPD and asthma may be comorbid with bronchiectasis and not all patients with bronchiectasis have comorbid ast
Slide 14: Brensocatib Phase 3 in Bronchiectasis: Trial summary
Brensocatib Phase 3 in Bronchiectasis: Trial summary Off treatment4 weeks Treatment period 52 weeks Baseline day 1 randomization End of trial day 364 End of studyday 392 ± 3 6 weeks Randomization: 1:1:1 for adults 2:2:1 for adolescents Screening Brensocatib 10 mg once daily Brensocatib 25 mg once da
Slide 15: Phase 3 ASPEN Study a Clear Win: Primary Endpoint Achieved Statistical Significance on Both Doses
Phase 3 ASPEN Study a Clear Win: Primary Endpoint Achieved Statistical Significance on Both Doses PE=Pulmonary ExacerbationFEV1=Forced Expiratory Volume Over 1 Second QoL-B=Quality of Life-Bronchiectasis Questionnaire* Statistically significant^ Nominally significant p-value Brensocatib 10 mg compar
Slide 16: Chronic Rhinosinusitis without Nasal Polyps Represents Another Potential Large Patient Opportunity
Chronic Rhinosinusitis without Nasal Polyps Represents Another Potential Large Patient Opportunity Included in Phase 2b BiRCh Trial; >75% Enrolled Annual New Surgical Patients (Incidence)5 0.5M 3.1M 2.6M Steroid Non-responders (Prevalence)5 40K 200K 160K 6M 29M 24M CRSsNP CRSsNP (Prevalence)4 US TAM
Slide 17: Brensocatib Phase 2b in CRSsNP: BiRCh
Brensocatib Phase 2b in CRSsNP: BiRCh Brensocatib 40 mg QD Placebo QD Screening BASELINE EOT (primary analysis) Up to 5 weeks Brensocatib 10 mg QD 4 weeks Treatment period 24 weeks EOS R Off-treatment follow-up N=~270 total Key eligibility criteria: Male or female ≥18 years old and ≤75 years old At
Slide 18: Potential Expansion into Hidradenitis Suppurativa Could Be a New Treatment Option for >600K Patients
Potential Expansion into Hidradenitis Suppurativa Could Be a New Treatment Option for >600K Patients Hurley Stage 2 & 36 Global Diagnosed Prevalence6 30K 275K 300K HS 1.2M – 2.6M US TAM EU5† TAM Japan TAM HS: hidradenitis suppurativa | TAM: Total Addressable Market † EU5 comprised of France, Germany
Slide 19: Brensocatib Phase 2b in HS: CEDAR Trial summary
Primary Endpoint (Week 16) Percent change in total abscess and inflammatory nodule (AN) count Secondary Endpoints (Week 16) Percentage achieving HiSCR50 Percentage achieving HiSCR75 Change from baseline in draining tunnel count Percentage remaining free from HS flare Change from baseline in Internat
Slide 20: TPIP
TPIP Pulmonary Hypertension due to Interstitial Lung Disease A rapidly progressing disease associated with poor survival and decreased quality of life Pulmonary Arterial Hypertension A devastating and debilitating disease that pervades all aspects of a patient’s daily life Image source: Valentini A,
Slide 21: TPIP has the Potential for Clear Differentiation in PH-ILD and PAH
TPIP has the Potential for Clear Differentiation in PH-ILD and PAH * No head-to-head or convenience studies have been conducted or planned** Safety analysis based on topline safety and tolerability data from the Phase 2 PH-ILD study of TPIP disclosed on May 6, 2024 †Based on most recent publicly ava
Slide 22: PH-ILD and PAH Represent Substantial Commercial Opportunities
PH-ILD and PAH Represent Substantial Commercial Opportunities TPIP: A Potential Best-in-Class* Prostanoid ‡ Wholesale acquisition cost (WAC) as of March 12, 2024 TAM: Total Addressable Market; †EU5 comprised of France, Germany, Italy, Spain and the United Kingdom Note: Numbered footnote references r
Slide 23: TPIP Phase 2 in PH-ILD
TPIP Phase 2 in PH-ILD 4 weeks TPIP 80-640 µg inhalation capsules QD Placebo inhalation capsules QD Screening Treatment period 16 weeks Baseline EOT (primary analysis) EOS 4 weeks Primary Endpoints Secondary Endpoints Follow-up Pharmacokinetics 39 participants randomized in the study 3:1 active : pl
Slide 24: PH-ILD Phase 2 Study Met Primary Objective of Safety and Tolerability
PH-ILD Phase 2 Study Met Primary Objective of Safety and Tolerability, With Lower Rates of Treatment Discontinuation and SAEs vs. Placebo “N” = total participants randomized in specified group | “n” = number of participants within the group1 Based on investigator’s evaluation | 2 All events of cough
Slide 25: PH-ILD Phase 2 Exploratory Efficacy Endpoints Support Advancement into Phase 3
PH-ILD Phase 2 Exploratory Efficacy Endpoints Support Advancement into Phase 3 “N” = total participants randomized in specified group; “n” = number of events, or participants within the group1 Calculation based on Hodges-Lehmann Estimate Location Shift | 2 Calculation based on geometric mean | 3 Cal
Slide 26: TPIP Phase 2b in PAH
TPIP Phase 2b in PAH 4 weeks TPIP 80-640 µg inhalation capsules QD Placebo inhalation capsules QD Screening Treatment period 16 weeks Baseline EOT (primary analysis) EOS 4 weeks Follow-up Approximately 100 participants randomized in the study 2:1 active : placebo Steady-state period 3-week titration
Slide 27: Blended Blinded Phase 2 Data in PAH Indicates Potential of TPIP to Become Best-in-Class Prostanoid
Blended Blinded Phase 2 Data in PAH Indicates Potential of TPIP to Become Best-in-Class* Prostanoid 1 Dose titration analysis based on data available as of April 1, 2024. Dose titration data in PAH reflect first sets of patients in each trial who reached the Week 5 visit, which is the last possible
Slide 28: Gene Therapy & Pre-Clinical Research
Gene Therapy & Pre-Clinical Research Gene Therapy Duchenne Muscular Dystrophy (DMD)1 A rare genetic disorder that causes progressive muscle degeneration, primarily affecting young boys Pre-Clinical Pipeline: INS1202: ALS (Gene Therapy) INS1203: Stargardt Disease (Gene Therapy) Next Gen DPP1 (Multipl
Slide 29: Research Engine Has Produced First Product Candidate
Research Engine Has Produced First Product Candidate Gene Therapy IND for Duchenne Muscular Dystrophy Cleared in December 20241 Our Approach Low Upfront Costs to acquire the technologies Low Ongoing Expense <20% of expenditures Expect ~1-2 IND filings per year Gene Therapy Deimmunized Protein Engine
Slide 30: ASCEND Phase 1 in Duchenne Muscular Dystrophy (DMD)
* 30-day safety observation period between each participant in each cohort IDMC=Independent Data Monitoring Committee Key Trial Information Single dose via intrathecal administration 30-day safety monitoring period between each patient in each cohort Patients must be ambulatory at time of screening
Slide 31: Our Culture is Our Greatest Strength
Our Culture is Our Greatest Strength In a recent survey* >90% of Employees who responded said they felt: * The 2024 annual Insmed Pulse Survey included 89.4% participation across the organization Proud to work at Insmed Inspired by what we do Confident in Insmed’s future Driven to do their best work
Slide 32: Appendix
Appendix
Slide 33: Strong, Long-Dated Patent Exclusivity
Strong, Long-Dated Patent Exclusivity a Based on U.S. Patent Application No.18/024,040 and ex-U.S. counterpart applications issuing as patents. Additionally, given the complexity of drug product (liposomal inhalation with specific nebulizer), required bioequivalence testing could be difficult and le
Slide 34: Manufacturing
Manufacturing Brensocatib ARIKAYCE TPIP API Drug Product Spain Canada API Drug Product Taiwan US API Drug Product Italy UK To come online Canada
Slide 35: ARISE: QoL-B Respiratory Score Changes Show Clear Separation
ARISE: QoL-B Respiratory Score Changes* Show Clear Separation When PGI-S Category Improved vs. No Change or Decline QoL-B: Quality of Life-Bronchiectasis Respiratory Domain questionnaire | PGI-S: Patient Global Impression of Severity | MAC: Mycobacterium avium complex * Includes patient data from bo
Slide 36: ARISE: Comparison Between Treatment Arms Shows Clear Favorable Trend for ARIKAYCE Arm
QoL-B Respiratory Domain ALIS+AZI+ETH N=48 ELC+AZI+ETH N=51 Difference # of Participants Evaluated for Change from Baseline to Month 7 43 48 # of Missing Change from Baseline to Month 7 Value 5 3 Change in PRO Score from Baseline to Month 7 (LS-Mean) 12.24 7.76 4.48 (95% Confidence Interval) (7.96,
Slide 37: ARISE: QoL-B Respiratory Score Changes from Baseline to Month 7
ARISE: QoL-B Respiratory Score Changes from Baseline to Month 7 Show Clear Separation for ARIKAYCE Group CDF: cumulative distribution function | ALIS: amikacin liposome inhalation suspension | AZI: azithromycin; ELC: empty liposome control | ETH: ethambutol | QoL-B: Quality of Life-Bronchiectasis Re
Slide 38: ARISE: Significantly Higher, More Persistent Culture Conversion in ARIKAYCE Arm
ARISE: Significantly* Higher, More Persistent Culture Conversion in ARIKAYCE Arm ALIS: amikacin liposome inhalation suspension | AZI: azithromycin | ELC: empty liposome control | ETH: ethambutol * “Nominally statistically significant” because no hierarchical testing or adjustment for multiplicity wa
Slide 39: ARISE: Patients in ARIKAYCE Arm Converted Faster
ARISE: For Those Who Achieved Culture Conversion, Patients in ARIKAYCE Arm Converted Faster ALIS: amikacin liposome inhalation suspension | AZI: azithromycin | ELC: empty liposome control | ETH: ethambutol ~74% of converters on ARIKAYCE achieved clinical conversion at the first possible time point (
Slide 40: ARISE: Correlation Between Culture Conversion and QoL-B Respiratory Score Changes
ALIS+AZI+ETH (N=48) Culture Converted by Month 6 Not Culture Converted by Month 6 Difference Change in QoL-B Resp. Score (Baseline to Month 7) +15.74 +3.53 +12.21 (95% Confidence Interval) (+11.45, +20.03) (-5.34, +12.41) (+2.33, +22.08) p-value 0.0167 Culture Converted by Month 7 Not Culture Conver
Slide 41: ARISE: No New or Unexpected Safety Signals
ALIS+AZI+ETH N=48 ELC+AZI+ETH N=51 Total N=99 Study completion (%) 44 (91.7) 48 (94.1) 92 (92.9) Treatment completion with all 3 drugs (%) 35 (72.9) 47 (92.2) 82 (82.8) Early ALIS/ELC discontinuation (%) 11 (22.9) 4 (7.8) 15 (15.2) Any TEAE (%) 44 (91.7) 41 (80.4) 85 (85.9) Dysphonia (%) 20 (41.7) 2
Slide 42: ASPEN: Annualized Rate of Adjudicated Pulmonary Exacerbations Over 52 Weeks
ASPEN: Annualized Rate of Adjudicated Pulmonary Exacerbations Over 52 Weeks a Exacerbations were adjudicated events in the ITT analysis set analyzed using a negative binomial model b P value is statistically significant when adjusted for multiplicity control c See Appendix for subgroup analysis | IT
Slide 43: ASPEN: Change From Baseline in Post-Bronchodilator FEV1 at Week 52
ASPEN: Change From Baseline in Post-Bronchodilator FEV1 at Week 52 a % Predicted FEV1 analyzed using a linear repeated measures model in the ITT analysis set. b P value is statistically significant when adjusted for multiplicity controlc See Appendix for subgroup analysis BD=bronchodilator | FEV1=fo
Slide 44: ASPEN: Change From Baseline in QoL-B Respiratory Symptom Domain Score
ASPEN: Change From Baseline in QoL-B Respiratory Symptom Domain Score at Week 52 a Adult patients only. b P value vs placebo calculated using a linear repeated measures model in the ITT analysis set. c Nominally significant P value.LS=least squares | QoL-B RSS=Quality of Life-Bronchiectasis Question
Slide 45: ASPEN: TEAEs and Treatment Discontinuations Comparable in Brensocatib and Placebo Arms
ASPEN: TEAEs and Treatment Discontinuations Were Comparable in Brensocatib and Placebo Arms TEAE=treatment-emergent adverse event * As reported by study investigators Brensocatib 10 mgn=582 Brensocatib 25 mgn=574 Placebon=563 Any TEAE, n (%) 452 (77.7) 440 (76.7) 448 (79.6) Severe TEAE, n (%) 74 (12
Slide 46: ASPEN: Change From Baseline in Post-Bronchodilator FVC at Week 52
ASPEN: Change From Baseline in Post-Bronchodilator FVC at Week 52 Brensocatib aFVC analyzed using a linear repeated measures model in the ITT analysis set. bNominally significant P value.BD=bronchodilator; FVC=forced vital capacity; ITT=intention-to-treat; LS=least squares. Exploratory endpoint Bren
Slide 47: ASPEN: Change From Baseline in Average Daily BEST Score
ASPEN: Change From Baseline in Average Daily BEST Score Brensocatib a Average daily change in the BEST score analyzed with an analysis of covariance model in adult patients in the ITT analysis set. | b Nominally significant P value.BEST, Bronchiectasis Exacerbation and Symptoms Tool. Exploratory end
Slide 48: ASPEN: Exacerbation Rate Subgroup Analysis (1 of 3)
Subgroup Brensocatib 10 mg (N=583) n (%) Brensocatib 25 mg (N=575) n (%) Placebo (N=563) n (%) Brensocatib 10 mg Rate ratio (95% CI) Brensocatib 10 mg Rate ratio (95% CI) Brensocatib 25 mg Rate ratio (95% CI) Brensocatib 25 mg Rate ratio (95% CI) Overall 583 (100.0) 575 (100.0) 563 (100.0) 0.79 (0.6
Slide 49: ASPEN: Exacerbation Rate Subgroup Analysis (2 of 3)
Subgroup Brensocatib 10 mg (N=583) n (%) Brensocatib 25 mg (N=575) n (%) Placebo (N=563) n (%) Brensocatib 10 mg Rate ratio (95% CI) Brensocatib 10 mg Rate ratio (95% CI) Brensocatib 25 mg Rate ratio (95% CI) Brensocatib 25 mg Rate ratio (95% CI) Chronic use of antibiotics Yes 146 (25.0) 154 (26.8)
Slide 50: ASPEN: Exacerbation Rate Subgroup Analysis (3 of 3)
COPD=chronic obstructive pulmonary disease; PE=pulmonary exacerbationbIncludes the United States and Canada. cIncludes Austria, Belgium, Bulgaria, Denmark, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Latvia, Netherlands, Poland, Portugal, Serbia, Slovakia, Spain, Turkey, and United Kin
Slide 51: ASPEN: FEV1 Subgroup Analysis (1 of 3)
Subgroup Brensocatib 10 mg (N=583) n (%) Brensocatib 25 mg (N=575) n (%) Placebo (N=563) n (%) Brensocatib 10 mg LS mean difference (95% CI) Brensocatib 10 mgLS mean difference (95% CI) Brensocatib 25 mg LS mean difference (95% CI) Brensocatib 25 mg LS mean difference (95% CI) Overall 583 (100.0) 57
Slide 52: ASPEN: FEV1 Subgroup Analysis (2 of 3)
Subgroup Brensocatib 10 mg (N=583) n (%) Brensocatib 25 mg (N=575) n (%) Placebo (N=563) n (%) Brensocatib 10 mg LS mean difference (95% CI) Brensocatib 10 mgLS mean difference (95% CI) Brensocatib 25 mg LS mean difference (95% CI) Brensocatib 25 mg LS mean difference (95% CI) Chronic use of antibio
Slide 53: ASPEN: FEV1 Subgroup Analysis (3 of 3)
COPD=chronic obstructive pulmonary disease; FEV1=forced expiratory volume in 1 second; LS=least squares; PE=pulmonary exacerbationbIncludes the United States and Canada. cIncludes Austria, Belgium, Bulgaria, Denmark, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Latvia, Netherlands, Pola
Slide 54: TPIP Phase 1 Study: Key Takeaways
TPIP showed substantially lower Cmaxand longer half-life Supports the potential for improved tolerability, efficacy and convenience Safety profile was generally well tolerated, AEs were mild and consistent with inhaled prostanoid Tolerability was improved with an up-titration approach Findings sugge
Slide 55: TPIP: Change From Baseline in Small and Large Blood Vessels
TPIP TPIP: Change From Baseline in Small and Large Blood Vessels aEffect size was calculated as a percentage of the treatment effect divided by the placebo arm baseline mean. SD=standard deviation; TPIP=treprostinil palmitil inhalation powder; BV5A=volume of pulmonary arteries smaller than 5 mm2 in
Slide 56: TPIP: Change From Baseline in High Attenuation Abnormality Score
TPIP TPIP: Change From Baseline in High Attenuation Abnormality Scorea bThe HAA score is expressed as a percentage of total lung volume. cEffect size was calculated as a percentage of the treatment effect divided by the placebo arm baseline mean. HAA=high-attenuation abnormality; SD=standard deviati
Slide 57: TPIP: FRI in Representative Patients Treated with TPIP vs. Placebo
Color indicates vessel size. Small vessels with cross-sectional area (CSA) <5 mm2 are colored in red (●), vessels with CSA 5-10 mm2 – in yellow (●), and CSA >10 mm2 – in blue (●). Parameters indicated as BV5 and BV10 represent the total volume of vessels with CSA <5 mm2 and >10 mm2, respectively. No
Slide 58: Epidemiological Footnotes (1 of 3)
Epidemiological Footnotes (1 of 3) 1. Internal analysis of published NTM epidemiology, including internal market research and US patient level claims data analysis: Jennifer Adjemian, Kenneth N Olivier, Amy E Seitz, Steven M Holland, D Rebecca Prevots: Prevalence of nontuberculous mycobacterial lung
Slide 59: Epidemiological Footnotes (2 of 3)
Epidemiological Footnotes (2 of 3) 4. Internal analysis and estimations based on published epidemiology studies: National Health Interview Survey (NHIS) Data (2021) Alshabanat A, Zafari Z, Albanyan O, Dairi M, FitzGerald JM (2015) Asthma and COPD Overlap Syndrome (ACOS): A Systematic Review and Meta
Slide 60: Epidemiological Footnotes (3 of 3)
Epidemiological Footnotes (3 of 3) 7. Internal assessment of published epidemiology and US patient level claims data analysis, including: Kirson, N. Y., Birnbaum, H. G., Ivanova, J. I., Waldman, T., Joish, V., & Williamson, T. (2011). Prevalence of pulmonary arterial hypertension and chronic thrombo
Slide 61: Count us in.