Slide 1: Fourth Quarter and Full Year 2022 Financial Results
Fourth Quarter and Full Year 2022 Financial Results February 23, 2023
Slide 2: 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 number of risks and uncert
Slide 3: Upcoming clinical data readouts across four pillars
1H 2023 ASPEN enrollment completion (1Q) Musculoskeletal preclinical data (2Q) CNS preclinical data (2Q) 1st Musculoskeletal IND (2Q) 2H 2023 CRSsNP Phase 2 trial initiation (mid-2023) ARISE topline results (3Q) TPIP Interim dose titration safety & tolerability levels ENCORE enrollment completion (Y
Slide 4: Screening complete in adult patients; future milestones
Screening is complete in adult patients FUTURE MILESTONES Expecting enrollment completion in 1Q23 Toplines Results in 2Q24
Slide 5: Save the Date: Insmed Research Day
SAVE THE DATE Insmed Research Day Date: May 8, 2023 Place: New York City & virtual Time: 8AM ET
Slide 6: Unveiling World-leading Capabilities to Support the Next Chapter of our Growth
Unveiling World-leading Capabilities to Support the Next Chapter of our Growth <20% of overall expenditures in the near term, reflects historical approach Expected to generate at least 6 new INDs and/or Phase 1 studies by year-end 2025 Chemistry, Formulation & Delivery Gene Therapy Deimmunized by De
Slide 7: Abstracts accepted for presentation
All 8 submitted abstracts accepted for presentation, including: ARIKAYCE adverse event mitigation in a real-world setting Brensocatib WILLOW study: analysis by disease severity subgroup May 19 – 24
Slide 8: Brensocatib: Initiate Phase 2 Study in CRS without Nasal Polyps
Brensocatib: Initiate Phase 2 Study in CRS without Nasal Polyps Potential first-in-disease therapy ~400K1,2,3,4 addressable patients worldwide (severe population) ~70M patients in U.S. with CRSsNP1, yet many do not respond to corticosteroids and/or Endoscopic Sinus Surgery (ESS) Initially targeting
Slide 9: ARIKAYCE: ARISE Topline Data
ARIKAYCE: ARISE Topline Data PRO Compare differences in PRO results between the two treatment arms (respiratory and fatigue scores) Goal of ARISE Demonstrate a directional effect on differences in endpoints between treatment groups Culture Conversion Effect on culture conversion Time to culture conv
Slide 10: TPIP: Phase 2 Interim Blinded Dose Titration Data
TPIP: Phase 2 Interim Blinded Dose Titration Data Offers Potential to De-Risk Program Primary Endpoint 10 3:1 active : placebo Safety and tolerability Oxygenation Approximately 32 participants randomized in the study Change from baseline in pulmonary vascular resistance (PVR) at week 16 Primary Endp
Slide 11: ARIKAYCE: ENCORE Enrollment Completion Anticipated Year-End 2023
*Azithromycin (AZI), Ethambutol (ETH) **Patients will be enrolled into separate open label extension study Primary Endpoint Change from Baseline to Month 13 (one month off treatment) in respiratory symptom score Key Secondary Endpoint Proportion of subjects achieving durable culture conversion at Mo
Slide 12: Brensocatib: ASPEN Phase 3 Data Expected
Brensocatib: ASPEN Phase 3 Data Expected Encouraging Blinded Event Rate in Most Recent 3 Months of Study, Consistent with WILLOW ~1M TOTAL DIAGNOSED NON-CF BRONCHIECTASIS PATIENTS AT LAUNCH1 ~450K2,3 U.S. ~150K Japan* ~400K EU5 Market* Up to 6.7M patients misdiagnosed or co-morbid with COPD or asthm
Slide 13: Brensocatib: CF Phase 2 Topline Data – NSP Inhibition
Brensocatib: CF Phase 2 Topline Data Dose & Exposure Dependent Inhibition of Blood NSPs was Observed Cathepsin G Proteinase 3 Neutrophil Elastase 10 mg 25 mg DOSE 40 mg placebo -14.7% 33.2% 54.9% 74.0% 11.3% 69.5% 86.9% 95.6% 2.5% 15.8% 37.2% 55.0% NSP Activity Reduction Relative to Baseline on Day
Slide 14: Brensocatib: CF Phase 2 Topline Data – Well-Tolerated
Brensocatib: CF Phase 2 Topline Data – Well-Toleratedwith No New Safety Signals Detected FINDINGS Thirteen participants in brensocatib arms and two in placebo reported 43 TEAEs One reported SAE of pulmonary exacerbation in brensocatib 40 mg arm (not related). Four TEAEs were related to study drug (3
Slide 15: Global ARIKAYCE Revenues Support Clinical Development Pipeline
Global ARIKAYCE Revenues Support Clinical Development Pipeline *Source: Internal analysis of published NTM epidemiology, primary market research with treating HCPs, and anonymized patient level claims data in US †European 5 comprised of France, Germany, Italy, Spain and the United Kingdom First-in-d
Slide 16: Building Sustainable Growth by 2025
Building Sustainable Growth by 2025 Commercial 1 2 3 4 ARIKAYCE Brensocatib TPIP* Early-Stage Research Refractory MAC lung disease Frontline MAClung disease Next Gen Manufacturing Additional Technologies Phase 3 <20% OF EXPENDITURES Phase 2 Preclinical Bronchiectasis Pulmonary hypertension associate
Slide 17: Reliable Revenue Stream from ARIKAYCE Commercial Franchise
Reliable Revenue Stream from ARIKAYCE Commercial Franchise Cash position $1.15B as of 12/31/22 Supports all inflection points in next 18 months 30% revenue growth in 2022 over 2021 FY22 global revenue – $245.4 million US – $186M JP – $56.5M EU + ROW – $2.9M 2023 Capital allocation >80% of expenditur
Slide 18: Executing Against our Strategic Priorities
Executing Against our Strategic Priorities Leverage our reliable revenue stream while carefully deploying capital Produce topline clinical data readouts near and long term Advance commercial readiness activities to serve significantly more patients with serious and rare diseases Control spending, pr