Insmed Incorporated · NASDAQ:INSM
Insmed — Investor Presentation (January 2020)
24 slides from Insmed's investor presentation, Jan 13, 2020
Slide 1: Investor Presentation January 2020
Investor Presentation January 2020 Not for promotional use
Slide 2: Forward-looking statements
This presentation contains forward - looking statements that involve substantial risks and uncertainties. "Forward - looking stateme nts," 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 u
Slide 3: ARIKAYCE Indication and Use
• LIMITED POPULATION: ARIKAYCE is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum
Slide 4: Our Mission
To transform the lives of patients with serious and rare diseases Our Mission Not for promotional use 4
Slide 5: 2020 Strategic Priorities
5 2020 Strategic Priorities Continue to drive strong commercial performance of ARIKAYCE in U.S. Initiate ARIKAYCE confirmatory clinical study in front - line setting of patients with MAC lung disease and a life - cycle management study in patients with NTM caused by M. Abscessus Accelerate global ex
Slide 6: About ARIKAYCE
About ARIKAYCE 6 Not for promotional use
Slide 7: What is MAC Lung Disease?
What is MAC Lung Disease? 7 Mycobacterium avium complex (MAC) lung disease is a rare, progressive, and chronic condition that can cause severe, permanent damage to the lungs. The disease is caused by bacteria in the environment and is more likely to affect those with a history of lung conditions, li
Slide 8: ARIKAYCE Developed to Address Significant Unmet Need in MAC Lung Disease
8 ARIKAYCE Developed to Address Significant Unmet Need in MAC Lung Disease Pulmovance™ liposomal technology delivers drug directly to site of infection; prolongs release of amikacin in the lungs while limiting systemic exposure Not for promotional use An inhaled, innovative, once - daily formulation
Slide 9: Commercial Approach: Near-Term Focus on U.S. Launch; Expansion to EU and Japan
Commercial Approach: Near - Term Focus on U.S. Launch; Expansion to EU and Japan 9 *Source: Internal analysis of published NTM epidemiology, primary market research with treating HCPs, and anonymized patient level claims data in US † EU5 comprised of France, Germany, Italy, Spain and the United King
Slide 10: U.S. Launch Update as of 9/30/19
U.S. Launch Update as of 9/30/19 10 Strong Initial Uptake • $38.9M 3Q19 total net product sales » $37.8M in U.S. net sales » $1.1M in ex - U.S. net sales from French ATU program and German named patient program Engaged HCP and Patient Community • Positive physician feedback • Over 1,600 unique presc
Slide 11: Total Revenue by Quarter
$9.8 $21.9 $30.0 $38.9 4Q18 1Q19 2Q19 3Q19 Total Revenue (in millions) Quarter Total Revenue by Quarter 11 Revenue Ramp since ARIKAYCE Launch Performance Catalysts: • Direct to Patient Campaign • Direct to Physician Campaign • Potential Treatment Guidelines Update • Deployment of Patient Access Liai
Slide 12: Estimated 2019 U.S. Market Opportunity in NTM Refractory MAC
12 Estimated 2019 U.S. Market Opportunity in NTM Refractory MAC 12K - 17K NTM Caused By MAC* 80K - 90K Diagnosed NTM* 98K - 113K Launch Population Life - Cycle Management and Label Expansion Potential Potential progression toward use as front - line or maintenance therapy Life Cycle Management* Laun
Slide 13: Proposed Registration Study for Frontline Indication
13 Screen Failure but pass required subset of screen tests Proposed Registration Study for Frontline Indication Not for promotional use Screening Blinded Registration Study ARIKAYCE + AZI* + ETH* Placebo + AZI + ETH Key Endpoints Month 13 Culture Negativity Endpoints Month 15 R Months 1 - 12 E ARIKA
Slide 14: Established Supply Chain
• Redundant drug supply chain • Commercial - scale manufacturing capacity on - line and expansion under way 14 Established Supply Chain *Patheon long term project initiated 4Q 2017 but not yet completed * Not for promotional use 50L 200L 450L
Slide 15: ARIKAYCE: Multiple Layers of Market Exclusivity
15 ARIKAYCE: Multiple Layers of Market Exclusivity 2033 2027 2030 2028 2029 2026 2031 2032 2034 2035 U.S. patent portfolio Regulatory exclusivity periods (U.S./EU) Japanese patent portfolio* • Coverage into 2035 • U.S.: 12 - year exclusivity (orphan and QIDP designations) • EU: 10 - year exclusivity
Slide 16: Growing Pipeline
Growing Pipeline 16 PRECLINICAL PHASE 1 PHASE 2 PHASE 3 APPROVED ARIKAYCE ® ( Amikacin Liposome Inhalation Suspension) Refractory NTM: M. avium complex (MAC) ARIKAYCE ® (Amikacin Liposome Inhalation Suspension) Lifecycle Management Front line, maintenance, other non - MAC NTM species, e.g., M. Absce
Slide 17: About INS1007 for Non-Cystic Fibrosis Bronchiectasis (NCFBE)
About INS1007 for Non - Cystic Fibrosis Bronchiectasis (NCFBE) 17 Not for promotional use
Slide 18: Model for Bronchiectasis Pathogenesis
Model for Bronchiectasis Pathogenesis 18 An environmental insult or disease process leads to bronchial wall destruction and dilation Structural damage impairs protective mucociliary clearance Increased susceptibility to chronic bacterial infection and colonization AIRWAY DESTRUCTION ABNORMAL MUCOCIL
Slide 19: INS1007 for Non-Cystic Fibrosis Bronchiectasis (NCFBE)
INS1007 for Non - Cystic Fibrosis Bronchiectasis (NCFBE) 19 Novel Mechanism Severe, Chronic Pulmonary Disorder Status • Oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) • DPP1 catalyzes activation of neutrophil serine proteases (NSPs) • NSPs are key agents of neutrophil - mediated inflamm
Slide 20: INS1007 is a Potent DPP1 Inhibitor that Prevents NSP Activation
20 INS1007 is a Potent DPP1 Inhibitor that Prevents NSP Activation During Neutrophil Maturation Neutrophil serine proteases (NSPs) are activated by dipeptidyl peptidase 1 (DPP1) during neutrophil maturation in bone marrow INS1007 inhibits DPP1, preventing activation of NSPs; neutrophils mature and a
Slide 21: INS1007 in NCFBE: The Willow Study
INS1007 in NCFBE: The Willow Study 21 NCFBE confirmed by CT scan With documented history of ≥2 pulmonary exacerbations in prior 12 months INS1007 10 mg once daily Placebo Randomized 1:1:1 Double Blind 256 Patients Primary Efficacy: Time to first pulmonary exacerbation Secondary Efficacy: • Rate of p
Slide 22: Framing Expectations for INS1007 Willow Study
≥20% difference in rate of pulmonary exacerbations vs. placebo would justify proceeding to Phase 3 Framing Expectations for INS1007 Willow Study 22 Exacerbation rate represents key endpoint in Phase 2 study Insmed is seeking to avoid repeating the history of Phase 3 failures in bronchiectasis • FDA
Slide 23: Financial Highlights
23 Financial Highlights Financial Guidance • Full - year 2019 revenues for ARIKAYCE in the range of $133 million to $138 million • Second half 2019 adjusted operating expenses in the range of $140 million to $155 million* As of September 30, 2019 • Cash and cash equivalents $535.6 million • Common s
Slide 24: Thank You
Thank You Not for promotional use 24