Slide 1: Developing Innovative Inhaled Treatments for Serious Lung Infections
Exhibit 99.1 Developing Innovative Inhaled Treatments for Serious Lung Infections June 2012
Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward-looking statements which are made pursuant to provisions of Section 21E of the Securities Exchange Act of 1934. Investors are cautioned that such statements in this presentation, including statements relating to our financial position, results
Slide 3: Insmed: Value Proposition
Insmed: Value Proposition ARIKACE®* is a potentially highly differentiated product opportunity that offers a compelling business model in two orphan diseases w ARIKACE (liposomal amikacin for inhalation), is in Phase 3 (CLEAR-108) for cystic fibrosis (CF) Pseudomonas (Pa) lung infections and Phase 2
Slide 4: Amikacin Summary
ARIKACE: Amikacin Summary Amikacin is an FDA-approved antibiotic with proven efficacy in the treatment of gram-negative infections, including Pseudomonas and NTM w Member of the aminoglycoside class of antibiotics w The value of the IV use has been limited by issues of nephro- toxicity and ototoxici
Slide 5: Proprietary Liposomal Formulation Provides Basis for Differentiation
ARIKACE: Proprietary Liposomal Formulation Provides Basis for Differentiation ARIKACE delivers the potency of Amikacin at the site of the lung infection while providing potential efficacy, safety and convenience benefits Water Core (where Amikacin resides) Lipid Polar Head Groups (at Both Surfaces)
Slide 6: Delivery Using Proprietary eFlow Technology
ARIKACE: Delivery Using Proprietary eFlow® Technology ARIKACE is delivered once daily via the state-of-the-art PARI Optimized, Investigational eFlow Nebulizer System with Advanced Mesh Technology w Fast drug delivery with efficient lung deposition w Small, portable, silent and cordless device weighs
Slide 7: Base Development Plan for 2012
ARIKACE: Base Development Plan for 2012 Insmed is focusing on CLEAR-108 (CF Pa Phase 3 Study) and TARGET-NTM (NTM Phase 2 Study) w ARIKACE vs. Tobi® (inhaled tobramycin solution); N ≈ 300 CLEAR-108 w Builds off of strong Phase 2 efficacy and safety data CF Pseudomonas w Broad population with preferr
Slide 8: ARIKACE—Cystic Fibrosis: Epidemiology and Disease Description
ARIKACE—Cystic Fibrosis Epidemiology and Disease Description Cystic fibrosis is a life-threatening disease with significant unmet needs that is growing in prevalence w Affects about 70,000 children and adults worldwide (30,000 in the U.S.) w Inherited disease that causes thick, sticky mucus to build
Slide 9: Cystic Fibrosis: Need for New Inhaled Antibiotics
ARIKACE: Cystic Fibrosis Need for New Inhaled Antibiotics Current inhaled antibiotics produce modest efficacy in a limited patient population providing an opportunity for ARIKACE to become first-line treatment w Current inhaled antibiotics are not indicated for a significant segment of the CF popula
Slide 10: Cayston vs. Tobi
Cayston® vs. Tobi® CF Phase 3 Trial Results: Pulmonary Function Lung function returned to baseline or lower during each off treatment period and at the end of 24 weeks, both treatment groups showed a decline in lung function from baseline Lung Function Adjusted Mean Relative Change in FEV1 % Predict
Slide 11: Phase 2 Pooled Results (560mg QD): Pulmonary Function
ARIKACE: Cystic Fibrosis Phase 2 Pooled Results (560mg QD): Pulmonary Function ARIKACE demonstrated statistically significant and clinically meaningful improvement in pulmonary function throughout the 28-day treatment period that was sustained through the off-treatment period P = 0.033 P = 0.003 (N)
Slide 12: Open Label Extension (TR02-105): Duration of Response
ARIKACE: Cystic Fibrosis Open Label Extension (TR02-105): Duration of Response An open label extension study demonstrated the sustained efficacy of ARIKACE during and between multiple cycles of therapy Patients Receiving 560 mg ARIKACE Once Daily for 28 Days and Off-Treatment for 56 Days Cycle Cycle
Slide 13: Phase 3 Program Has Been Initiated in Europe and Canada
ARIKACE: Cystic Fibrosis Phase 3 Program Has Been Initiated in Europe and Canada Insmed has reached agreement with EMA and Health Canada on pivotal study requirements for CF patients with Pseudomonas lung infections w CLEAR-108: Phase 3 Primary Efficacy Study (vs. Tobi®, N ≈ 300)* – Primary End-Poin
Slide 14: Non-TB Mycobacteria: Epidemiology and Disease Description
ARIKACE: Non-TB Mycobacteria Epidemiology and Disease Description Total U.S. Patients Diagnosed with NTM Lung Infections1 w Diagnoses of NTM lung infections are growing rapidly 48K - 59K w Current treatment approaches have limited efficacy and are associated with significant toxicity w Mean age of 5
Slide 15: TARGET-NTM Clinical Study Scheduled to be Initiated in Mid-2012
ARIKACE: Non-TB Mycobacteria TARGET-NTM Clinical Study Scheduled to be Initiated in Mid-2012 We believe Insmed is the only company with a clinical program focused on NTM with top-line Phase 2 data projected in 4Q 2013 w Trial Design and Patient Population (N ≈ 100): – Randomized, double-blind, place
Slide 16: Current Overview: Capital Structure and Key Financials
Current Overview: Capital Structure and Key Financials Insmed has a strong cash position Present Capital w 26.2 million fully diluted shares: Structure ü 24.9 million Common Shares (INSM) ü 1.3 million options and restricted stock Pro Forma w Pro forma cash of ~$73 million as of March 31, 2012 Balan