Insmed Incorporated · NASDAQ:INSM
Insmed — Investor Presentation (January 2021)
51 slides from Insmed's investor presentation, Jan 11, 2021
Slide 1: Promising Therapies Inspired by Urgent Unmet Patient Needs
Promising Therapies Inspired by Urgent Unmet Patient Needs
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements that involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a
Slide 3: What a Difference a Year Makes
What a Difference a Year Makes… One Year Ago … One Pillar Today…. Three Pillars Successful U.S. Launch in Refractory MAC Lung Disease ARIKAYCE International expansion: EU Approval & Launch + Japan Filing Strong recommendation for use in international treatment guidelines* Frontline program underway
Slide 4: Our Mission and Vision
Our Mission To transform the lives of patients with serious and rare diseases Our Vision To be a globally-recognized leading biotech company that empowers great people to deliver, with a profound sense of urgency and compassion, life-altering therapies to small patient populations experiencing big h
Slide 5: Ongoing Success of Our Pipeline Coupled with Our Core Franchise Leads to Value Creation
Ongoing Success of Our Pipeline Coupled with Our Core Franchise Leads to Compelling Value Creation Brensocatib Add’l Indications TPIP TPIP World Class Team Research & Development Expertise Brensocatib Bronchiectasis Brensocatib Bronchiectasis Global Commercial Presence Business Development Strength
Slide 6: Three Pillars of Value Creation
Three Pillars of Value Creation PRECLINICALPHASE 1PHASE 2PHASE 3APPROVED ARIKAYCE® (Amikacin Liposome Inhalation Suspension)* Refractory NTM: M. avium complex (MAC) Front Line Label Expansion Brensocatib: DPP1 Inhibitor Bronchiectasis Cystic Fibrosis (IIR) COVID19 Treprostinil Palmitil Inhalation Po
Slide 7: Treprostinil Palmitil Inhalation Powder (TPIP)
Treprostinil Palmitil Inhalation Powder (TPIP) 7
Slide 8: Harnessing the Full Potential of the Prostanoid Pathway in PAH
Harnessing the Full Potential of the Prostanoid Pathway in Pulmonary Arterial Hypertension (PAH) Prostanoids have long been considered the cornerstone therapy for PAH, but clinical use has faced challenges related to rapid metabolism and tolerability issues Treprostinil palmitil has the potential to
Slide 9: TPIP: A Potentially Differentiated Prostanoid
Treprostinil Palmitil Inhalation Powder (TPIP) A Potentially Differentiated Prostanoid TPIP Lung Esterase Convenience Portable, dry powder inhaler QD or BID dosing Potentially prolonged lung residence time, prolonged half life, prolonged duration of pharmacodynamic activity Improved safety/tolerabil
Slide 10: Improved Pharmacokinetics: Prolonged Lung Residence, Lower Cmax
Improved Pharmacokinetics: Prolonged Lung Residence, Lower Cmax, Prolonged Half Life (Rat and Dog) RAT Lung and Plasma Levels DOG Plasma Levels 10000 Lung TRE eq (ng/g) 100 Treprostinil Palmitil 42 μg/kg Treprostinil Palmitil 108 μg/kg Treprostinil 215 μg/kg Treprostinil Palmitil Inhaled TP Inhaled
Slide 11: TPIP at High Dose Shows Superior Effect in Sugen-Hypoxia Rat Model
TPIP at High Dose Shows Superior Effect Overall in the Sugen-Hypoxia Rat Model for PAH Parameter value = (Value – Value of Normal) / (Value of Vehicle – Value of Normal) Amount of blood pumped by the heart per minute Weight ratio Right Ventricle / (Left Ventricle + Septum) % of non-obliterated vesse
Slide 12: Comparison of Prostacyclin Analogs/Agonists
Comparison of Prostacyclin Analogs/Agonists Approved Therapies vs. TPIP (investigational product candidate) Remodulin®Tyvaso®Orenitram®Uptravi®TPIP Route of administrationIV or Subcutaneous Inhaled (nebulized) OralOralInhaled (dry powder) Dosing frequencyContinuous4x per day2x or 3x per day 2x per d
Slide 13: Market Opportunity
Market Opportunity United Therapeutics’ Viewpoint… Building On Their Precedent InnovateGrowExpand Current PAH LandscapeNew PH OpportunitiesOpportunities Beyond PH Prevalence of PH[1] WHO Group 1[2] Prevalence of PH ILD[4] & PH COPD[5] in WHO Group 3[2] Prevalence of Chronic Fibrosing ILD[2] [in U.S.
Slide 14: Next Steps for TPIP Development
Next Steps for TPIP Development Phase 1 Complete SAD/MAD Study Objective Establish PK and tolerability profile Top line results expected in Q1 2021 Expect to Initiate Phase 2a in Early 2021 Exploratory, single dose study Objective Demonstrate proof of mechanism, understand PK/PD relationship, confir
Slide 15: Brensocatib
Brensocatib 15
Slide 16: Pathogenesis of Bronchiectasis: A Vicious Cycle
Pathogenesis of Bronchiectasis: A Vicious Cycle INFLAMMATION Persistent inflammatory response, causing lung damage BACTERIAL COLONIZATION Increased susceptibility to chronic bacterial infection and colonization AIRWAY DESTRUCTION An environmental insult or disease process leads to bronchial wall des
Slide 17: Brensocatib Inhibits Activation of NSPs in Neutrophil-mediated Diseases
By Inhibiting the Activation of NSPs, Brensocatib May Offer Applicability in a Range of Neutrophil-mediated Diseases DPP1 is an enzyme that catalyzes the activation of NSPs in neutrophils when they are formed in the bone marrow Neutrophils are the most common type of white blood cell and play an ess
Slide 18: WILLOW Study Success Recognized Worldwide
WILLOW Study Success Recognized Worldwide Both 10 mg & 25 mg group met primary endpoint with statistical significance Exacerbation-related hospitalization rates was higher & duration of hospitalization was longer in the placebo arm Risk of exacerbation over six months reduced by 42% for 10 mg group,
Slide 19: WILLOW vs. ASPEN Trial Details
Strong Phase 2 Data Lead to Confidence in Phase 3 WILLOW vs. ASPEN Trial Details WILLOWASPEN Sample Size256 Pts (~85 pts/arm) 1,620 Pts (540 pts/arm) Powering Assumptions80% for a 40% reduction90% for a 30% reduction Robust Definition of Pulmonary ExacerbationConsistentConsistent Duration of Therapy
Slide 20: ASPEN Phase 3 Study Design
ASPEN Phase 3 Study Design Treatment period Off treatment Up to 6 weeks 52 weeks 4 weeks Brensocatib 25 mg QD N=540 40 Countries 1:1:1 Screening Brensocatib 10 mg QD N=540 ~480 sites Stratified by: Geographic region Pseudomonas aeruginosa on sputum culture Number of prior PEs (2, or ≥3) in previous
Slide 21: The Unmet Need in Bronchiectasis
The Unmet Need in Bronchiectasis No Available Treatment Options for 1.7M – 6M Diagnosed Bronchiectasis Patients Total Diagnosed BE PatientsAdditional Market Opportunity ~340K – ~520K * U.S. Between 650K – 9M COPD patients in the US (4%1-2 ~350K – ~500K ** EU5† COPD 54% ) may also have bronchiectasis
Slide 22: Building a Portfolio of Treatments for Neutrophil-mediated Diseases
Strategically Building a Portfolio of Treatments for Neutrophil-mediated Diseases Bronchiectasis Program Serious Unmet Need Conditions Cystic Fibrosis Granulomatosis with Polyangiitis Apha-1 Antitrypsin Deficiency Larger Markets Inflammatory Bowel Disease* Rheumatoid Arthritis* Lupus Nephritis* COPD
Slide 23: ARIKAYCE
ARIKAYCE 23 Not for promotional use
Slide 24: First-in-Disease Therapy for Refractory MAC Lung Disease
First-in-Disease Therapy for Refractory MAC Lung Disease Gaining Global Momentum MAC lung disease is a rare, progressive, and chronic condition that can cause severe, permanent damage to the lungs ARIKAYCE Novel, inhaled, once-daily formulation of liposomal amikacin taken with a nebulizer Symptoms o
Slide 25: Building on the Success of the Launch with Recent Advancements
Building on the Success of the Launch with Recent Advancements International guideline inclusion with a strong recommendation for use (1) U.S. sNDA approval adding Culture Conversion Data Beyond 12 Months to Label Approved in Europe in late 2020 Planned expansion into Japan by mid-2021 European Laun
Slide 26: Clear Vision for Label Expansion & Global Market
Clear Vision for Label Expansion & Global Market U.S. 95-115K Diagnosed NTM Patients (2019E)* 12K-17K refractory MAC patients (2019E)* EU† Market 14K Diagnosed NTM Patients (2018E)* 1,400 refractory MAC patients (2018E)* Japan 125-145K Diagnosed NTM Patients (2018E)* 15K-18K refractory MAC patients
Slide 27: ARIKAYCE Front Line Program Ongoing Trial Summary
ARIKAYCE Front Line Program Ongoing Trial Summary ARISE Primary Objective Demonstrate reliability, validity and responsiveness of the PRO/symptom scores Screening Adults with new ARISE Psychometric ARIKAYCE + AZI* + ETH* RMonths 1-6 Off Treatment** Secondary Objective Demonstrate effect of ARIKAYCE
Slide 28: Financial Strength to Achieve Our Objectives
Financial Strength to Achieve Our Objectives $588.8M as of 9/30/20 102M* as of 9/30/20 Cash and Cash Equivalents Common Shares Outstanding *Excludes stock options, unvested RSUs and shares underlying outstanding convertible notes 28
Slide 29: Building on Our Global Momentum to Create Shareholder Value
Building on Our Global Momentum to Create Shareholder Value 2020 2019 ARIKAYCE 2021 (anticipated) ARIKAYCE Japan approval / 2018 ARIKAYCE Top 10 Launch Brensocatib Completed enrollment in WILLOW EU launch Brensocatib NEJM publication of WILLOW data TPIP Positive preclinical data & initiated clinical
Slide 30: Anticipated Near-Term Events Support All Three Pillars
Anticipated Near-Term Events Support All Three Pillars of Value Creation Strategy ARIKAYCE: Commercial Expansion And Label Extension EU Approval And Launch Frontline Program Initiation (ARISE / ENCORE) Japan Approval Japan Launch Brensocatib: Unlocking Options For Neutrophil-mediated Diseases ASPEN
Slide 31: Investor Relations
We are committed to bringing forth technologies and medicines in therapeutic areas with the greatest potential to make a difference in patients’ lives. Investor Relations investor.relations@insmed.com 646-351-0954 Powered by Purpose 3131
Slide 32: Appendix
Appendix 32
Slide 33: Robust Development Cycle Capabilities with Clear Commercialization Path
Robust Development Cycle Capabilities with Clear Commercialization Path Pipeline Highlight: Populations with Urgent Unmet Patient Needs Drug Discovery Research ARIKAYCE® Treprostinil Palmitil Inhalation Powder (TPIP) Clinical Development Management of the Regulatory Pathway Global Commercialization
Slide 34: Insmed Differentiated
Insmed Differentiated With ARIKAYCE, Brensocatib and TPIP, we have developed three value drivers, plus Robust Pipeline and Development Capabilities: Clinical Development Drug Discovery Research Business Development Management of the Regulatory Pathway Global Commercialization Strong expertise and po
Slide 35: Protected with Significant, Global IP
Protected with Significant, Global IP Brensocatib US, Europe, Japan, China In-licensed compound patent exclusivity until 2035 TPIP Non-CF Bronchiectasis Treatment Methods patent application filed, projected to expire in 2037 Oral formulation and ANCA vasculitis patent applications filed, projected t
Slide 36: Manufacturing
Manufacturing Brensocatib ARIKAYCE TPIP API API API Spain 300kg capacity Canada 200L capacity Taiwan 50g capacity Drug Product Drug Product Drug Product Canada 2mm capacity UK 450L capacity US 10,000 capsules capacity 36
Slide 37: WILLOW Study Data Demonstrate Potential for First-in-Class Bronchiectasis Treatment
W I L L O W S T U D Y• Data Demonstrate Potential for First-in-Class Bronchiectasis Treatment Randomized, double-blind, Phase 2 study; tested 10 mg, 25 mg and placebo CT scan-confirmed NCFBE; ≥2 pulmonary exacerbations in past 12 months Risk of exacerbation over six months reduced by 42% for 10 mg g
Slide 38: WILLOW Study Summary
Willow Study Summary Study Schema NCFBE confirmed by CT scan With documented history of ≥2 pulmonary exacerbations in prior 12 months Efficacy Top-Line Data: WILLOW Study Achieves Primary Endpoint Risk of having an exacerbation over the course of six months reduced by up to 40% with Brensocatib Rand
Slide 39: Brensocatib Phase 2 WILLOW Study Design
Brensocatib Phase 2 WILLOW Study Design Treatment period Off treatment Primary objective Time to first exacerbation over 24 weeks in patients with non-cystic fibrosis bronchiectasis (NCFBE) 6 weeks 24 weeks 4 weeks Screening Stratified by Pseudomonas 1:1:1 Brensocatib 25 mg QD Brensocatib 10 mg QD P
Slide 40: WILLOW: Hospitalization Rate due to Exacerbations
WILLOW: Hospitalization Rate due to Exacerbations 10% 8% Patients (%) 4% 2% 8.0% Exacerbation-Related Hospitalization Rate a 6.1% 4.6% Days of Hospitalization Due to Exacerbation 12 10.1 10 8 7.0 Days (N) 6 4 2 Exacerbation-related hospitalization rates was higher & duration of hospitalization was l
Slide 41: Rate of Exacerbations by Patient Subgroups
25 mg brensocatib vs. placebo Rate of Exacerbations by Patient Subgroups 10 mg brensocatib vs. placebo Chalmers et al, ERS International Congress 2020, 7-9 September. RCT4135 BSI, bronchiectasis severity index; FEV1, forced expiratory volume in 1 second aBulgaria/Poland 41
Slide 42: Dose Response Effect Across Sputum Neutrophil Serine Proteases
Dose Response Effect Seen Consistently Across Sputum Neutrophil Serine Proteases (NSPs) Mean (SE) Change from Baseline to Week 24 (log10 [mg/mL]) Neutrophil Elastase Proteinase 3Cathepsin G Chalmers et al, ERS International Congress 2020, 7-9 September. RCT413542
Slide 43: WILLOW Safety Data
WILLOW Safety Data no. (%)Placebo (n=85) Brensocatib 10 mg (n=81) Brensocatib 25 mg (n=89) TEAE resulting in study discontinuation3 (3.5)3 (3.7)4 (5.5) TEAE resulting in treatment discontinuation9 (10.6)6 (7.4)6 (6.7) Serious TEAE19 (22.4)11 13.6)10 (11.2) Serious TEAEs in ≥ 3% of patients in any gr
Slide 44: ASPEN Study: Phase 3 Pivotal Program Ongoing
ASPEN Study: Phase 3 Pivotal Program Ongoing Single Trial Designed to Support Global Filing Breakthrough Therapy Designation & PRIME designation granted for brensocatib in non-cystic fibrosis bronchiectasis (NCFBE) Robust efficacy endpoints, supported by WILLOW results Integrates feedback from Healt
Slide 45: ASPEN Timeline Considerations
ASPEN Timeline Considerations 40 Countries ~480 sites 6 Fast Track Countries Targeting WILLOW study sites 45
Slide 46: Well Positioned to Support a Successful Global Launch in Bronchiectasis
Well Positioned to Support a Successful Global Launch in Bronchiectasis Uniquely positioned to commercialize brensocatib given the synergies that exist between bronchiectasis and NTM lung disease Proven US launch capabilities with ARIKAYCE Geographic and target HCP overlap with current US Sales stru
Slide 47: ARIKAYCE Treats Refractory MAC Lung Disease
ARIKAYCE Treats Refractory MAC Lung Disease Potential Progression Toward Use as Front-line and/or Maintenance Therapy 12-17 K refractory US: 100K patients Approved Treatment in MAC Lung Disease First and only therapy approved in the United States as part of a combination antibacterial drug regimen f
Slide 48: European Reimbursement and Launch Timelines
Germany Ireland Anticipated timelines shown Insmed has been accepted to participate in an accelerated reimbursement process being piloted by the Dutch Health Care Institute (ZIN). While ARIKAYCE is currently well advanced in the accelerated review process, there is no guarantee ARIKAYCE will ultimat
Slide 49: On Track for Anticipated Approval, Mid-Year 2021 Japan Launch
On Track for Anticipated Approval, Mid-Year 2021 Japan Launch 2020 March+12 Months~ 3 monthsMid-Year 2021 ARIKAYCE JNDA Filing JDNA ApprovalPrice NegotiationLaunch 2020 JuneJNDA Approval~ 3 monthsMid-Year 2021 Lamira Nebulizer Notification Form submitted; considered approved Request for health insur
Slide 50: Pulmonary Hypertension: Diagnostic Classification
Pulmonary 3. 1. Hypoxemia Hypertension: Diagnostic classification Idiopathic PAH (IPAH) Heritable BMPR2 ALK1, ENG, SMAD9, CAV1, KCNK3 1.2.3. Unknown Drug-and toxin-induced Associated with Connective tissue diseases HIV infection Portal hypertension Congenital heart diseases Schistosomiasis 1’ Pulmon
Slide 51: Treprostinil Palmitil Inhalation Solution Phase 1 Study Plasma PK
Treprostinil Palmitil Inhalation Solution Phase 1 Study Plasma PK in Healthy Volunteers Plasma Treprostinil Concentration (ng/mL) TPIS 85 Substantially lower Cmax than Tyvaso® Persistent plasma levels of drug beyond 12 hours consistent with sustained release Treprostinil Plasma PK Summary Cohort 1 (