Insmed (INSM) investor relations material

Recent achievements and pipeline progress

• Successful ASPEN trial readout and FDA approval of brensocatib for bronchiectasis, with early launch showing strong initial revenue and broad prescribing patterns.

• TPIP data validated its potential in multiple indications, with phase 3 trials launching in PAH, PH-ILD, IPF, and PPF; regulatory feedback allows for single phase 3 studies in some indications.

• ARIKAYCE, already approved in several regions, is positioned for significant market expansion pending ENCORE trial results, potentially increasing the addressable population from 30,000 to 250,000.

• Brensocatib is being studied for additional diseases, including CRS and HS, with key data readouts expected by early next year; CRS could represent a larger market than bronchiectasis.

• Next-generation DPP-1 inhibitors and gene therapy programs for DMD, ALS, and Stargardt are advancing, with several entering the clinic next year.

Commercial strategy and market outlook

• Focus on multi-franchise commercial execution, leveraging existing salesforce for new launches and maintaining list price parity across the U.S., Europe, and Japan.

• Early Brinsupri launch generated $28 million in partial quarter revenue, with market access and payer decisions expected to shape uptake over the next two quarters.

• Expansion into Europe and Japan is supported by established infrastructure, with pricing strategies adapting to local market dynamics and MFN pressures.

• COPD and asthma comorbidities could significantly expand the bronchiectasis market, with millions of potential patients beyond the initial 250,000 target group.

• Revenue from Brinsupri and ARIKAYCE is expected to drive the company toward cash flow positivity, supporting ongoing R&D and business development.

R&D prioritization and future opportunities

• Nearly 30 preclinical programs span gene therapy, deimmunized proteins, and synthetic rescue technologies, with AI-driven approaches in development.

• Selective business development will focus on novel mechanisms of action with high return potential and low upfront costs.

• CRS without nasal polyps represents a major unmet need, with over 30 million U.S. patients; positive trial results could unlock broader applications for DPP-1 inhibitors.

• Overlap between bronchiectasis, CRS, and NTM populations may facilitate cross-indication expansion and integrated treatment strategies.

• TPIP's once-daily, dry powder formulation and strong efficacy data position it as a best-in-class candidate for multiple pulmonary indications.