Slide 1: Developing Innovative Inhaled Treatments for Serious Lung Infections
Exhibit 99.1 Developing Innovative Inhaled Treatments for Serious Lung Infections July 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, project
Slide 3: Insmed: Value Proposition
Insmed: Value Proposition ARIKACE®* is a highly differentiated product that offers a compelling business opportunity 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 (TARGET- Attracti
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 ARIKACE (liposomal amikacin for inhalation) delivers high, sustained levels of drug to the lung while reducing systemic exposure to well b
Slide 5: Proprietary Liposomal Formulation Provides Basis for Important Potential Benefits
ARIKACE: Proprietary Liposomal Formulation Provides Basis _ for Important Potential Benefits ARIKACE delivers the potency of Amikacin at the site of the lung infection; engineered specifically for improved PK-PD* profile in the lung providing for potential enhanced efficacy, safety and convenience b
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: Development Plan
ARIKACE: Development Plan 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 preferred trial desig
Slide 8: Arikace—Cystic Fibrosis
Arikace—Cystic Fibrosis Epidemiology and Disease Description Cystic fibrosis is a life-threatening disease with significant unmet needs that is growing in prevalence Pseudomonas Lung Infections Increase with Patient Age w Affects about 70,000 children and adults worldwide (30,000 in U.S. and Europe,
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): Durability of Response
ARIKACE: Cystic Fibrosis Open Label Extension (TR02-105): Durability 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 Cyc
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: Disease Description and High Unmet Need
ARIKACE: Non-TB Mycobacteria Disease Description and High Unmet Need Non-TB mycobacteria (NTM) are intracellular pathogens that can cause severe, chronic pulmonary disease with limited effective treatment options w NTM are intracellular organisms that invade and multiply chiefly within macrophages i
Slide 15: Non-TB Mycobacteria: Market Opportunity
ARIKACE: Non-TB Mycobacteria Market Opportunity “The prevalence of this debilitating chronic disease continues to grow, and the current NTM treatment paradigm lacks acceptable treatment options”* w Diagnosis growing at~ 8% annually2 U.S. Patients Diagnosed with NTM Lung Infections in 20111 w MAC and
Slide 16: Non-TB Mycobacteria: Rationale for ARIKACE
ARIKACE: Non-TB Mycobacteria Rationale for ARIKACE ARIKACE opportunity: achieve superior efficacy in NTM treatment by better penetrating lung macrophages where NTM bacteria reside while limiting systemic drug exposure w NTM lung infections are difficult to treat since NTM are taken up and multiply i
Slide 17: TARGET-NTM Clinical Study Initiated in Mid-2012
ARIKACE: Non-TB Mycobacteria TARGET-NTM Clinical Study Initiated in Mid-2012 Insmed appears to be the only company with an NTM clinical program; top -line Phase 2 data projected in 4Q 2013 w Trial Design and Patient Population (N ≈ 100): – Randomized, double-blind, placebo controlled Phase 2 study i
Slide 18: Current Overview: Capital Structure and Key Financials
Current Overview: Capital Structure and Key Financials Insmed has a strong cash position Present Capital w 26.5 million fully diluted shares: Structure ü 24.9 million Common Shares (INSM) ü 1.6 million options and restricted stock units w Cash of ~$73 million as of March 31, 2012 consisting of cash,