Slide 1: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward looking statements. "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 uncertainties. Words herein such
Slide 2: Our Mission: Transform the lives of patients battling serious rare diseases
3 To transform the lives of patients battling serious rare diseases OUR MISSION Top-line data indicates Phase 3 CONVERT trial met primary endpoint of culture conversion Company plans to pursue accelerated approval and request priority review
Slide 3: CONVERT Study Design
ALIS once daily + Guideline Based Therapy 4 Adult Patients with NTM (MAC) Lung Disease Failing at least 6 months of a multi-drug regimen KEY INCLUSION CRITERIA Guideline Based Therapy R Screening period Month-6 sputum culture PRIMARY ENDPOINT Culture Conversion at 6 months INS-212 non-converters INS
Slide 4: Top-line Data Indicates CONVERT Study Met Primary Endpoint
Top-line Data Indicates CONVERT Study Met Primary Endpoint Addition of ALIS to GBT* eliminated evidence in sputum of NTM lung disease caused by MAC by Month 6 in 29% of patients, compared to 9% of patients on GBT alone (p <0.0001) 20% absolute difference in treatment groups in favor of ALIS arm 5 p
Slide 5: Top-line CONVERT Study Results – Secondary Endpoints at Month 6
Top-line CONVERT Study Results – Secondary Endpoints at Month 6 6 Time to Conversion Patients in the GBT-only arm took approximately 30% longer to convert when compared to patients on ALIS plus GBT (p<0.0001) 6-Minute Walk Distance (6MWD) No statistically significant difference between patients in t
Slide 6: CONVERT Safety Summary
CONVERT Safety Summary Adverse events, consistent with those seen with use of inhaled antibiotics, more frequent in ALIS + GBT arm Serious treatment emergent adverse events were similar between treatment arms No distinctions between treatment arms of hearing loss or renal impairment 7 2:1 Randomizat
Slide 7: CONVERT Study Demographics Summary
CONVERT Study Demographics Summary ALIS (n=224) GBT (n=112) Smokers 11.6% (26) 8.9% (10) Guideline Based Therapy Prior to Enrollment On Treatment 89.7% (201) 90.2% (101) Off Treatment for > 3 months 10.3% (23) 9.8% (11) Geography US 41.5% (93) 42.9% (48) Asia 21.4% (48) 17.9% (20) EU + ROW 37.1% (83
Slide 8: Pathway to US Submission
NDA Submission planned based on primary endpoint under Subpart H Pathway to US Submission – Division of Anti-Infective Products 9 Breakthrough Designation Orphan and QIDP Accelerated Approval Full Approval Requirement 12 years of exclusivity (7 orphan and 5 QIDP) and likely priority review Guidance
Slide 9: Insmed: A Global Biopharmaceutical Focused on Rare Disease
Insmed: A Global Biopharmaceutical Focused on Rare Disease 10 POSITIVE TOP-LINE PHASE 3 RESULTS FOR ALIS GLOBAL COMMERCIAL OPPORTUNITY ALIS LIFE CYCLE MANAGEMENT
Slide 10: Insmed: A Global Biopharmaceutical Focused on Rare Disease
Insmed: A Global Biopharmaceutical Focused on Rare Disease ALIS LIFE CYCLE MANAGEMENT GLOBAL POSITIVE TOP-LINE COMMERCIAL PHASE 3 RESULTS OPPORTUNITY FOR ALIS inismed
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