Insmed Incorporated · NASDAQ:INSM
Insmed — Investor Presentation (February 2020)
33 slides from Insmed's investor presentation, Feb 25, 2020
Slide 1: Investor Presentation February 2020
Investor Presentation February 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: Insmed 2020: Supporting the Next Stage of Growth
5 Insmed 2020: Supporting the Next Stage of Growth ARIKAYCE Successful commercial launch in the U.S. INS1007 Late - stage program with novel mechanism of action and broad potential Potential $1B market opportunities Strong patent protection Commitment to Patients Focus on unmet needs and delivering
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. Commercial Update as of 12/31/19
U.S. Commercial Update as of 12/31/19 10 Strong 2019 Sales • $136.5 million for full - year 2019 • $45.7 million in 4Q 2019 Engaged HCP and Patient Community • Positive physician feedback • Over 1,900 unique prescribers since launch Supportive Payor Landscape • Continued positive reimbursement trend
Slide 11: ARIKAYCE Total Revenue by Quarter
$21.9 $30.0 $38.9 $45.7 1Q19 2Q19 3Q19 4Q19 Total Revenue (in millions) Quarter Total Revenue by Quarter 11 ARIKAYCE Revenues Expected Drivers for 2020 Performance • Peer - reviewed publication of potential solutions to manage most common adverse events • Potential update to NTM treatment guidelines
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* 95K - 115K 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* • Patent exclusivity to 2035 • U.S.: 12 - year exclusivity (orphan and QIDP designations) • EU: 10 - year exc
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: Oral, Reversible Inhibitor of Dipeptidyl Peptidase 1 (DPP1)
INS1007: Oral, Reversible Inhibitor of Dipeptidyl Peptidase 1 (DPP1) 19 Novel Mechanism Bronchiectasis: A Severe, Chronic Pulmonary Disorder • DPP1 catalyzes activation of neutrophil serine proteases (NSPs) in neutrophils • NSPs are key agents of neutrophil - mediated inflammation, tissue damage, an
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: Top-Line Data: WILLOW Study Achieves Primary Endpoint
Top - Line Data: WILLOW Study Achieves Primary Endpoint 22 ^ Two sided p - value Time to First Exacerbation vs. Placebo INS1007 10 mg INS1007 25 mg p - value ^ 0.027 0.044 Hazard Ratio p - value ^ 0.58 0.029 0.62 0.046 Risk of having an exacerbation over the course of six months reduced by up to 40%
Slide 23: Top-Line Data: WILLOW Study Achieves Key Secondary Endpoint
Frequency of Pulmonary Exacerbations INS1007 10 mg (N=82) INS1007 25 mg (N=87) Reduction vs. Placebo 36% 25% p - value 0.041 0.167 23 Top - Line Data: WILLOW Study Achieves Key Secondary Endpoint FDA Guidance: Frequency of pulmonary exacerbation should be the primary endpoint in Phase 3
Slide 24: WILLOW Safety Data
WILLOW Safety Data • INS1007 was generally well - tolerated in the WILLOW study • Most common adverse events (AEs) in patients treated with INS1007 were cough, headache, sputum increase, dyspnea, fatigue, and upper respiratory tract infection 24 Placebo INS1007 10 mg INS1007 25 mg Rates of adverse e
Slide 25: Financial Highlights
25 Financial Highlights Full Year 2020 Financial Guidance • Full year 2020 revenues for ARIKAYCE expected to be $180M - $220M • Full year adjusted operating expenses* expected to be $340M - $360M As of December 31, 2019 • Cash and cash equivalents $487.4 million • Common shares outstanding** 89.7 mi
Slide 26: Appendix
Appendix 26
Slide 27: WILLOW: Baseline Characteristics in ITT Population
WILLOW: Baseline Characteristics in ITT Population Placebo (n=87) INS1007 10 mg (n=82) INS1007 25 mg (n=87) Sex, Female (%) 55 (63.2) 57 (69.5) 62 (71.3) Age ( yrs ), mean ± SD 64.0 (11.86) 64.6 (12.42) 63.7 (12.67) Age ≥65 years Age ≥75 years 54 (62.1) 14 (16.1) 48 (58.5) 20 (24.4) 48 (55.2) 14 (16
Slide 28: WILLOW: Hazard Ratio Pulmonary Exacerbation by Subgroup
28 WILLOW: Hazard Ratio Pulmonary Exacerbation by Subgroup
Slide 29: WILLOW: Frequency of Pulmonary Exacerbations by Subgroup
29 WILLOW: Frequency of Pulmonary Exacerbations by Subgroup
Slide 30: INS1009: Dry Powder Inhaled Prostanoid for Pulmonary Arterial Hypertension (PAH)
• Dry powder, inhaled treprostinil prodrug formulation • Designed to provide sustained release of treprostinil to the lung over a prolonged period. • Potential benefits of INS1009: • Potential to be disease - modifying, with impact on vascular remodeling • Superior tolerability observed in animal mo
Slide 31: RV94: For the Treatment of Gram-Positive Infections
RV94: For the Treatment of Gram - Positive Infections 31 • Semisynthetic lipoglycopeptide designed to be used as a once - daily inhaled therapy for CF patients with pulmonary MRSA infection New Chemical Entity • Up to 60x more potent than vancomycin in pulmonary MRSA infections in animal models • 30
Slide 32: Opportunity for Synergistic Portfolio in Additional Lung Disease Indications
Opportunity for Synergistic Portfolio in Additional Lung Disease Indications 32 NTM Cystic Fibrosis ARIKAYCE INS1007 Bronchiectasis RV94 INS1007
Slide 33: Thank You
Thank You Not for promotional use 33