Insmed Incorporated · NASDAQ:INSM
Insmed — IPO Roadshow (September 2012)
32 slides from Insmed's IPO roadshow presentation, Sep 28, 2012
Slide 1: Developing Innovative Inhaled Treatments for Serious Lung Infections
Free Writing Prospectus Registration Statement No. 333-182124 Developing Innovative Inhaled Treatments for Serious Lung Infections August 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 ARIKACE (liposomal amikacin for inhalation), is in Phase 3 (CLEAR-108) for cystic fibrosis (CF) Pseudomonas (Pa) lung infections and Phase 2 Attractive (TARGE
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 Aminoglycoside antibiotic Value of the IV use has been limited by nephro-toxicity and ototoxicity ARIKACE (liposomal amikacin for inhalati
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 lunginfection; engineered specifically for improved PK-PD* profile in the lung providing for potential enhanced efficacy, safety and convenience bene
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 Fast drug delivery with efficient lung deposition Small, portable, silent and cordlessdevice weighs les
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) ARIKACEvs. Tobi ® (inhaled tobramycin solution); N 300 CLEAR-108 Builds off of strong Phase 2 efficacy and safety data CF Pseudomonas Broad population with preferred trial design Study
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 Pseudomonas Lung Infections Increase with Patient Age Affects about 70,000 children 80.0 and adults worldwide (30,000 in U.S. and Europe, each) 70.0 60.0 Inherited
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 Current inhaled antibiotics are not indicated for a significant segment of the CF populati
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 Changein FEV1 % Predicte
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 18% % Change in FEV 1 (
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 20 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 ® CLEAR-108: Phase 3 Primary Efficacy Study (vs. Tobi ,N 300)* Primary End-Point: Re
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 NTMare intracellular organisms that invade and multiply chiefly within macrophages in t
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”* 2 U.S. Patients Diagnosed with NTM Lung Infections in 20111 Diagnosisgrowing at~ 8%annually 60,000 MAC a
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 NTM lung infections are difficult to treat since NTM are taken up and multiply ins
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 Trial Design and Patient Population (N 100): – Randomized, double-blind, placebo controlled Phase 2 study in pat
Slide 18: Current Overview: Capital Structure and Key Financials
Current Overview: Capital Structure and Key Financials Insmed has a strong cash position Present Capital 26.5 million fully diluted shares: Structure 24.9 million Common Shares (INSM) 1.6 million options, restricted stock units, and warrants Cash of ~$75 million as of June 30, 2012 consisting of cas
Slide 19: Appendix
Appendix Addressing the Potential for Cross-Resistance of ARIKACE 18
Slide 20: Summary: Addressing Potential for Cross-Resistance in ARIKACE
Summary: Addressing Potential for Cross-Resistance in ARIKACE While resistance to TOBI (tobramycin) has been documented, we believe there is no cross-resistance in ARIKACE (amikacin) for the following reasons. Well-characterized clinical isolates of Pseudomonas aeruginosa (Pa) from Dr. Burns’ collec
Slide 21: Aztreonam for Inhalation Solution vs. Tobramycin Inhalation Solution
Aztreonam for Inhalation Solution (AZLI!) vs. Tobramycin Inhalation Solution (TIS): Gg A 6-month Comparative Trial in Cystic Fibrosis (CF) Patients with Pseudomonas aeruginosa (PA) @ GILEAD (CM Cermann,’ B 7c SR Boas,* MB: S Lewis," ABM: ys and T OSs Lekeaiae betes ‘Cotaoe of Mesicing, Nounton, TUBA
Slide 22: Percent Change in FEV1—ITT
Percent Change in FEV1—ITT Arikace 560 p=0.016 p=0.005 p=0.04 Arikace 280 p=0.07 Placebo Visit Day Arikace 560 * 15.4% (16.5) 18.4% (21.3) 13.2% (15.3) 13.2% (16.2) 11.5% (16.4) 13.2% (24.3) Arikace 280 * 10.9% (10.6) 9.4% (12.6) 9.6% (12.5) 10.1% (12.8) 1.7% (9.0) 2.0% (8.6) Placebo * 0.6% (11.7) -
Slide 23: Change in FEV1 (% predicted)—ITT
Change in FEV1 (% predicted)—ITT Arikace 560 P=0.009 P=0.019 P=0.021 Arikace 280 P=0.124 Placebo Visit Day Arikace 560 * 12.9% (17.2) 15.8% (22.5) 10.5% (15.6) 11.0% (16.4) 8.6% (17.7) 13.8% (26.2) Arikace 280 * 10.8% (10.8) 9.2% (13.1) 9.4% (12.9) 9.6% (13.7) 1.6% (9.6) 1.8% (8.8) Placebo * -0.9% (
Slide 24: ARIKACE—TR02-05 PFT: Prior Use of Inhalation Antibiotic
ARIKACE—TR02-05 PFT: Prior Use of Inhalation Antibiotic Relative Change FEV 1 (ml) Arikace ™ Placebo (N=8) (N=4) Day 28 10 % -5 % Day 56 5% -1 % 23
Slide 25: Tobramycin FEV1 (L) Absolute
Tobramycin FEV1 (L) Absolute 24
Slide 26: ARIKACE—TR02-05 By Prior Tobramycin Use
ARIKACE—TR02-05 By Prior Tobramycin Use * AbsoluteChangefrom Baseline- FEV1 (L) Cohort I –280 mg Patients With Prior Patients Without Prior Tobramycin Use Tobramycin Use Arikace ™ N=5 Placebo N=3 Arikace ™ N=16 Placebo N=8 Day 28 0.326 (0.290) 5 -0.083 (0.123) 3 0.126 (0.203) 16 -0.016 (0.144) 8 Day
Slide 27: ARIKACE—TR02-05 By Prior Tobramycin Use
ARIKACE—TR02-05 By Prior Tobramycin Use 280mg Cohort 280mg Cohort Patients with Tobramycin Patients without Tobramycin Arikace Arikace Placebo Placebo Visit Day Visit Day Arikace * 326 (290) 152 (186) Arikace * 126 (203) 1 (161) Placebo * -83 (123) -40 (284) Placebo * -16 (144) -120 (168) * Mean (SD
Slide 28: Tobramycin FEV1 (L) Relative
Tobramycin FEV1 (L) Relative 27
Slide 29: ARIKACE—TR02-05 By Prior Tobramycin Use
ARIKACE—TR02-05 By Prior Tobramycin Use * Relative Changefrom Baseline- FEV1 (L) Cohort I –280 mg Patients With Prior Patients Without Prior Tobramycin Use Tobramycin Use Arikace ™ N=5 Placebo N=3 Arikace ™ N=16 Placebo N=8 Day 28 0.136 (0.088) 5 -0.052 (0.075) 3 0.091 (0.138) 16 -0.002 (0.067) 8 Da
Slide 30: ARIKACE—TR02-05 By Prior Tobramycin Use
ARIKACE—TR02-05 By Prior Tobramycin Use 280mg Cohort 280mg Cohort Patients with Tobramycin Patients without Tobramycin Arikace Arikace Placebo Placebo Visit Day Visit Day Arikace * 13.6% (8.8) 5.1% (9.3) Arikace * 9.1% (13.8) 0.9% (8.4) Placebo * -5.2% (7.5) -1.0% (14.8) Placebo * -0.2% (6.7) -5.3%
Slide 31: Arikace Efficacy in Patients with Prior Tobramycin Use: TR02-106
Arikace™- Efficacy in Patients with Prior Tobramycin Use: TR02-106 Change in FEV1(ml) Change in Log10 CFU Subjects with 5-6 Cycles of TOBI in Subjects with 5-6 Cycles of TOBI in prior 12 months prior 12 months 3 250 200 2 150 Placebo 100 1 50 Arikace 560 0 0 -50 -100 -1 -150 -200 Placebo -2 Arikace
Slide 32: Insmed has filed a registration statement with the SEC
Insmed has filed a registration statement (including a prospectus) with the Securities and Exchange Commission (the “SEC”) for the offering to which this communication relates. Before you invest, you should read the prospectus in that registration statement and other documents Insmed has filed with