Insmed Incorporated · NASDAQ:INSM
Insmed — Investor Day (March 2015)
97 slides from Insmed's investor day presentation, Mar 24, 2015
Slide 1: Analyst and Investor Day
Analyst and Investor Day March 24, 2015 NASDAQ: INSM Insmed Copyright 2015 © All Rights Reserved.
Slide 2: Agenda
Agenda I. Introduction and Overview – Will Lewis II. NTM Lung Disease & Clinical Trials – Dr. Gina Eagle III. Regulatory – Peggy Berry IV. Research and Development INS1009 – Dr. Walter Perkins and Dr. Gene Sullivan I. Global Commercial Plan & Corporate Development – Wes Kaupinen, Dr. Olaf Bartsch, D
Slide 3: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward-looking statements. Words, and variations of words, such as "intend," "expect," "will," "anticipate," "believe," "continue," "propose" and similar expressions are intended to identify forward-looking statements. Investors are cautioned that su
Slide 4: Insmed Company Vision
Insmed Company Vision We are building a self-sustaining biopharmaceutical company focused on patient needs at the intersection of orphan and pulmonary disease 4
Slide 5: Insmed's Orphan Product Portfolio
Insmed’s Orphan Product Portfolio NTM ARIKAYCE™ liposomal amikacin for inhalation PAH INS1009 inhaled treprostinil-prodrug Additional Programs in Orphan Diseases 5
Slide 6: 2015 – The Year of the Deliverables
2015 – The Year of the Deliverables I. Enroll the “212” NTM Global Phase 3 Trial II. Pursue European Approval of ARIKAYCE – NTM & CF III. Continue Manufacturing Build-out and Redundancy Planning IV. Submit IND for INS1009 and Enroll Phase 1 Trial V. Corporate Development 6
Slide 7: Team Introduction
Team Introduction Introduction Global Commercial Plan & Overview & Corporate Development Will Lewis Wes Kaupinen NTM Disease Dr. Olaf Bartsch & Clinical Trials Dr. Gina Eagle Drayton Wise Regulatory Peggy Berry Tech Ops, Cash R&D, INS1009 & IP and Tax Planning Dr. Walter Perkins Andy Drechsler Dr. G
Slide 8: NTM Lung Disease & Clinical Trials
NTM Lung Disease & Clinical Trials Dr. Gina Eagle 8
Slide 9: ARIKAYCE – NTM and CF Indications
ARIKAYCETM – NTM and CF Indications NTM ARIKAYCE™ liposomal amikacin for inhalation PAH INS1009 inhaled treprostinil-prodrug Additional Programs in Orphan Diseases 9
Slide 10: Nontuberculous Mycobacteria (NTM) Lung Infections
Nontuberculous Mycobacteria (NTM) Lung Infections No Rx Approved Treatment in US and EU Progressive Lung Disease and Increased Mortality in High Risk Patient Groups COPD, bronchiectasis, Common in: asthma and CF Source: Company website; Clarity Pharma Research; American Journal of Respiratory and Cr
Slide 11: Nontuberculous Mycobacteria (NTM) Lung Infections
Nontuberculous Mycobacteria (NTM) Lung Infections Annual Prevalence Diagnosed Treatment Resistant Increase Patients Populations (estimate) US a +13% 55-70K 10-20K EU d +6% 30K 5-10K 20K within EU5 Japan b +11% 30K 5-10K Australia e +9% 5-10K 1-2K Canada c +9% 5-10K 1-2K Source: Company website; Clar
Slide 12: Patient Journey Is Long and Difficult for Most
Patient Journey Is Long and Difficult for Most Only a few are diagnosed correctly from the outset Symptom Presentation Living with NTM Primary Care (NTM Doctor Management) Seek out Physician Flu Bronchitis Treatment initiation COPD Mis-diagnosis Asthma Pulmonary Doctor Treatment initiation Diagnosis
Slide 13: NTM Lung Disease: Risk Factors and Mortality
NTM Lung Disease Risk Factors and Mortality 13
Slide 14: Nontuberculous Mycobacterium (NTM) Overview
Nontuberculous Mycobacterium (NTM) Overview ● To date >150 identified NTM species ● MAC (Mycobacterium avium Complex) is the most common ● Ubiquitous organisms: found in soil and water ● Animal to human and human to human transmission not widely documented ● Asymptomatic infections and symptomatic d
Slide 15: Nontuberculous Mycobacterial Pulmonary Pathogens
Nontuberculous Mycobacterial Pulmonary Pathogens Infrequent Common } M. xenopi (zin oh’ pee) M. avium M. Avium Complex M. szulgai (sull’ guy) M. intracellulare (MAC) M. malmoense M. kansasii (mal’ moh en suh) M. abscessus M. fortuitum M. chelonae Rare M. celatum (sell ah’ tum) M. scrofulaceum M. sim
Slide 16: Risk Factors in MAC Lung Disease
Risk Factors in MAC Lung Disease 5-year-all-cause overall mortality (Ito et al *): 25.6% untreated chronic MAC patients: 33.3% treated MAC patients: 22.2% p = 0.30 Adjusting for clinical, microbiological and radiological confounders, independent factors for 5-year mortality were a high Charlson
Slide 17: Risk Factors in MAC Lung Disease
Risk Factors in MAC Lung Disease Literature on Risk Factors in MAC Advanced Stage Cavitary Lung Low BMI Macrolide- Lung Disease at Diagnosis Resistance at Diagnosis Ito et al INT J TUBERC LUNG DIS 16(3):408–414 Masao Okumura et al, Inter Med 47: 1465-1472, 2008 DOI: 10.2169/internalmedicine.47
Slide 18: High Risk Factor: Cavitary Lung
High Risk Factor: Cavitary Lung 18
Slide 19: NTM Lung Disease: Advantages of LAI
NTM Lung Disease Advantages of LAI 19
Slide 20: Liposomal Amikacin for Inhalation (LAI)
Liposomal Amikacin for Inhalation (LAI) ● Amikacin is an aminoglycoside antibiotic with in vitro evidence of activity against NTM ● Novel liposomal formulation for inhalation via nebuliser ● High lung Cmax, AUC, and t½ improved AUC:MIC ratio ● Liposomes have a neutral charge and can be taken up by l
Slide 21: NTM Lung Disease: Recap of Study
NTM Lung Disease Recap of Study 112 21
Slide 22: Study 112: Study Design
Study 112: Study Design Randomized 90 Subjects (1:1): Stratified: CF vs non-CF MAC vs M. abscessus 12 Weeks Once-Daily Dosing 12 Weeks Once-Daily Dosing, Background therapyd Open-Label + LAI* once daily 4 Weeks by eFlow® Screening perioda,b,c 28-Day follow-up Day –42 to Day –2 R Day 1 LAI once daily
Slide 23: Study 112: Patient Profiles
Study 112 : Patient Profiles • Length of Time on NTM Treatment Regimen Prior to Baseline >24 Months >12 - 24 Months 6 - 12 Months 0 5 10 15 20 25 30 35 40 Patients (n) • Lung Lesion Type on HRCTscan at Baseline Lesion Type N (%) Cavitary 6 (6.70%) Nodular and Cavitary 63 (70.0%) Nodular 21 (23.3%) 2
Slide 24: Study 112: Recap of Main Results
Study 112: Recap of Main Results ● Reduction in mycobacterium 25% Percent of patients 25.0% burden (numerically superior, (11/44) whose sputum culture tested negative not statistically significant) 20% for NTM at Day 84 (mITT population.) ● Achievement of a negative 15% sputum culture in 11 out of 4
Slide 25: Study 112: Patients with at least 1 negative sputum culture for NTM
Study 112: Patients with at least 1 negative sputum culture for NTM Length of NTM Treatment Prior to Baseline 28 Day Follow- Early Term Arm CF Status NTM Oragnism (Months) Baseline Day 28 Day 56 Day 84 Day 112 Day 140 Day 168 Up Reason >24 >24 >24 >24 Broth Broth >24 >12 - 24 Early Term Resp distres
Slide 26: Study 112: Patients with Sputum Culture Conversion
Study 112: Patients with Sputum Culture Conversion* Length of NTM Treatment Prior to Baseline 28 Day Follow- Early Term Arm CF Status NTM Oragnism (Months) Baseline Day 28 Day 56 Day 84 Day 112 Day 140 Day 168 Up Reason >24 >24 >24 >24 Broth Broth >24 >12 - 24 LAI Non-CF MAC >12 - 24 >12 - 24 >12 -
Slide 27: Study 112: Six Minute Walk Test
Study 112: 6 Minute Walk Test Change From Baseline in 6MWT Distance Missing Values Excluded (mITT Population) 40 30 20 Least Squares Mean (SE) 10 0 Arikayce -10 P = .009 Placebo -20 -30 -40 -50 -60 0 28 56 84 112 Study Day 27
Slide 28: Study 112: Safety Recap
Study 112: Safety Recap Treatment-Emergent Adverse Events with >10% occurrence Through End of Open-label Phase (Safety Population) Infective exacerbation of bronchiectasis Dysphonia Cough Oropharyngeal pain Haemoptysis Nausea Fatigue Pyrexia LAI (n=44) Wheezing Placebo (n=45) Chest discomfort 0% 10%
Slide 29: NTM Lung Disease: Insmed Study
Insmed NTM Lung Disease Study 212
Slide 30: Study 212-Global Sites
Study 212-Global Sites North America Europe Europe Asia-Pac ● United States (34) ● UK (15) ● Poland (4) ● Australia (17) ● Canada (5) ● Germany (13) ● Austria (2) ● Japan (8) ● Italy (12) ● Netherlands (1) ● New Zealand (3) ● Spain (7) ● France (6) Goal is to have 80+ sites active in the 1H 2015 30
Slide 31: Study 212: Study Design Overview
Study 212: Study Design Overview Key Facts: INS-212 Trial • Randomized ~300 patients (2:1) • Non-CF MAC Only • Stratifications: (1) Smokers vs. non-smokers; (2) Background Therapy last 6 months Converters Continue Treatment for 12 Months Post Conversion 8 Months Daily Dosing ARIKAYCE once daily 3 Mo
Slide 32: Study 212: Sputum Sampling
Study 212: Sputum Sampling Primary Endpoint: ● 3 sputum samples will be collected for each monthly visit (Baseline to Month 6) ● Patients will interrupt LAI administration on day -2 until they visit the clinic No LAI No LAI No LAI Re-Start 24 Hours 48 Hours 72 Hours LAI Dosing Last LAI Sputum #1 Spu
Slide 33: Central Micro Labs Globally
Central Micro Labs Globally Region Central Micro Lab Microbiology Director USA/Canada University of Texas at Tyler Health Richard J. Wallace Jr. M.D. Northeast (USA) Europe Radboud University Medical Center Jakko van Ingen, MD, PhD (Netherlands) Australia/Asia Queensland Mycobacterium Dr. Chris Coul
Slide 34: Methodology Across Global Central Micro Labs
Methodology Across Global Central Micro Labs Methodology/Supplies Tyler Radbound QMRL North America Europe Australia/Asia Control strain ATC700898 ATC700898 ATCC700898 Liquid medium VersaTrek BD MGIT BDMGIT Solid medium Agar 7H11 Agar 7H11 Agar 7H11 Mac Growth (ID) Gen-Probe Gen-Probe Hain CM Line P
Slide 35: Study 212 Sample Size Calculation (Primary Endpoint)
Study 212 Sample Size Calculation ● Primary Endpoint Proportion of subjects achieving culture conversion by Month 6 ● Assumptions Month 6 culture conversion rate: 20% for LAI+MDR; 5% for MDR Chi-square test at a 2-sided significance level of 0.05 A 2:1 randomization ratio to either LAI+MDR or MDR At
Slide 36: Study 212 Sample Size Calculation (Secondary Endpoint)
Study 212 Sample Size Calculation ● Secondary Endpoint Change from baseline to Month 6 in 6MWT distance ● Assumptions A common standard deviation (SD) of 100 A between-treatment difference of 50 meters in mean change two group t-test at a 2-sided significance level of 0.05 A 2:1 randomization ratio
Slide 37: Study 212 Statistical Methods
Study 212 Statistical Methods ● Stratification Randomization stratified by: Smoking Status (current smoker or not) and Prior Multi- drug Regimen (on treatment or off treatment for at least 3 months) ● Primary Analysis Population Modified Intent-to-treat population: all randomized subjects who receiv
Slide 38: Study 212 Statistical Methods (Secondary Endpoint)
Study 212 Statistical Methods ● Hypothesis – Secondary Endpoint H0: No between-treatment difference in mean change from baseline to Month 6 in 6MWT distance H1: There is a between-treatment difference in mean change ● Analysis of Secondary Endpoint Mixed-effects Model for Repeated Measures (MMRM) ov
Slide 39: Cystic Fibrosis Program
Cystic Fibrosis Program TR02-110 Update 39
Slide 40: Cystic Fibrosis Program Update
Cystic Fibrosis Program Update LAI for the Management of Pa in Patients with Cystic Fibrosis TR02-110 Mean Relative Change from Baseline in FEV1 (L) Missing Values Excluded mITT Population (Interim Data Cut - 149 Complete Day 337 [1 Year]) 25 20 15 Relative Mean Change from Baseline (L) 10 5 0 -5 -1
Slide 41: Reasons for Patient Discontinuation in TR02-110
Cystic Fibrosis Program Update Reasons for Patient Discontinuation in TR02-110 Prior LAI arm in TR02-108 Prior TOBI arm in TR02-108 18 16 16 15 14 12 Number of Patients 12 10 10 8 8 6 4 3 2 1 1 1 0 0 0 0 Death Adverse Event Withdrawl of Lost to Follow-Up Permature Other Concent Termination of Study
Slide 42: Regulatory
Regulatory Peggy Berry 42
Slide 43: Regulatory Overview
Regulatory Overview ARIKAYCE ● European update ● US update ● Rest of World activities INS1009 ● US update ● Rest of World activities 43
Slide 44: EMA Regulatory Timeline
EMA Regulatory Timeline Pre-submission Assessment of new marketing authorisation application Decision-making phase Clock Clock Stop Stop Day 1 Day 120 Day 121 Day 180 Day 181 Day 210 Day 277: (Feb 2015) List of List of Opinion EC Decision Questions Outstanding issues or opinion European Marketing Ap
Slide 45: ARIKAYCE – US Update
ARIKAYCE – US Update Reached agreement on protocol INS-212 Anticipate beginning rolling NDA submission in mid-2016 with final at end of 2016 for possible SubPart H approval ● Permits an approval based upon culture conversation & 6-minute walk test (6MWT) distance at the 6 month timepoint ● Requires
Slide 46: SubPart H Approval
SubPart H Approval ● If agreed requirements are met and the data on the final endpoints are achieved, the FDA could issue a full approval of the drug with updated labeling that incorporates information from the study results ● If agreed requirements are not met or if the clinical data do not succeed
Slide 47: ARIKAYCE – Rest of World Update
ARIKAYCE – Rest of World Update Australia ● Orphan drug designation achieved in February 2015 for CF and refractory NTM Canada ● Held pre-submission meetings with regulatory agencies Japan ● Reached agreement on clinical study requirements for phase 3 study and PK 47
Slide 48: INS1009 – US & RoW update
INS1009 – US & RoW update ● Reached general agreement on development program ● Agreed that 505(b)(2) is possible – pending review of the data confirming that INS1009 is a prodrug of treprostinil (FDA data review to occur 2Q 2015) ● Anticipated Benefits of 505(b)(2) ● No carcinogenicity study ● Only
Slide 49: Research and Development INS1009
Research and Development INS1009 Dr. Walter Perkins Dr. Gene Sullivan 49
Slide 50: Pulmonary Arterial Hypertension (PAH): Our Next Target Indication
Pulmonary Arterial Hypertension (PAH): Our Next Target Indication NTM ARIKAYCE™ liposomal amikacin for inhalation PAH INS1009 inhaled treprostinil-prodrug Additional Programs in Orphan Diseases 50
Slide 51: Pulmonary Arterial Hypertension (PAH) Orphan Disease
Pulmonary Arterial Hypertension (PAH) Orphan Disease Progressive Disease Leading to Heart Failure ● No curative treatments ● 15% one-year mortality ● ≈100,000* patients afflicted globally ● ≈25,000 treated patients in the U.S.1,2,3 ● Treatment is progressive combo therapy, including: ● Calcium chann
Slide 52: Current Marketed Prostanoids for Pulmonary Arterial Hypertension
Current Marketed Prostanoids for Pulmonary Arterial Hypertension Drug Dosing Pros Cons Intravenous Intravenous – central line infection Flolan, Veletri Continuous via continuous Requires infusion pump (epoprostinil) drug level infusion Swings in peak/trough plasma levels Ventavis Inhaled with
Slide 53: INS1009: Treprostinil-Prodrug Formulated in a Nanoparticle
INS1009: Treprostinil-Prodrug Formulated in a Nanoparticle 24 hour Coverage via Convenient Once-Daily Dosing without Large Peak/Trough Swings in Plasma Levels C16TR Hexadecyl-treprostinil (C16TR) Prodrug Nanoparticles in aqueous suspension Administered by state-of-the-art nebulizer Pat
Slide 54: INS1009 Provides Sustained Release of Treprostinil
INS1009 Provides Sustained Release of Treprostinil 10.00 INS1009 Treprostinil Plasma Level (ng/mL) Sustained Release Cmax reduced significantly 1.00 INS1009 0.10 Therapeutic threshold* Treprostinil 0.01 0 1 2 3 4 5 6 7 Time (hr) A single 6 µg/kg dose administered by inhalation to ventilated rats
Slide 55: INS1009 Extends Vasodilation Period Significantly
INS1009 Extends Vasodilation Period Significantly Rat Acute Hypoxia Model of PAH mPAP (as % initial hypoxic value) 110.0 Buffer Control 100.0 90.0 Treprostinil 80.0 70.0 INS1009 60.0 50.0 N’s= 8-10 40.0 -5 0 0.5 1 1.5 2 2.5 3 5 10 20 30 40 50 60 70 80 90 100 110 120 130 140 150 160 170 180 Time (min
Slide 56: INS1009 Provides Sustained Release of Treprostinil over Hours
INS1009 Provides Sustained Release of Treprostinil over 24 Hours Rats dosed by Nose-Only Inhalation 10 Dose of 6 µg/kg Treprostinil Plasma Level (ng/mL) 1 Stays above threshold 0.1 of 0.1ng/mL for full vasodilatory effect* 0.01 0 5 10 15 20 25 Time (h) With nose-only inhalation plasma levels are hig
Slide 57: Inhaled INS1009 Provides Sustained Release of Treprostinil in Dogs
Inhaled INS1009 Provides Sustained Release of Treprostinil in Dogs Lung Dose in Ventilated Dogs (Single Inhalation) 10 Tre 5 µg/kg Tre 16 µg/kg in Plasma INS1009 7 µg/kg Level INS1009 22 µg/kg INS1009 46 µg/kg Plasma 1 INS1009 95 µg/kg (ng/mL) (ng/mL) Compound Treprostinil 0.1 Active 0.01 0 6 12 18
Slide 58: INS1009 Better Tolerated in Dogs than Inhaled Treprostinil
INS1009 Better Tolerated in Dogs than Inhaled Treprostinil Compound Lung Dose (µg/kg) Observed Side Effects Treprostinil 5 0 of 3 dogs 16 4 of 5 dogs INS1009 7 0 of 3 dogs 22 0 of 5 dogs 46 0 of 3 dogs 95 1 of 3 dogs Observed side effects after drug delivery: Treprostinil -- cough, rapid shallow b
Slide 59: INS1009 for PAH: Path to Market
INS1009 for PAH: Path to Market Pursuing 505(b)(2) FDA NDA Approval ● Can be faster to market* ● Lower risk by leveraging prior drug approval ● Lower costs due to fewer studies FDA IND ● IND: expect to file in 2H 2015 INS1009 Clinical (graphical representation) ● Phase 1 Study: Expect to Start by Ye
Slide 60: INS1009 for PAH: Clinical Plans through Phase 2
INS1009 for PAH: Clinical Plans through Phase 2 Phase 1 A single ascending dose (SAD) study in healthy volunteers for tolerability and pharmacokinetics Phase 2 A single 12-week dose exploration study in PAH patients 60
Slide 61: Insmed's Orphan Product Portfolio
Insmed’s Orphan Product Portfolio NTM ARIKAYCE™ liposomal amikacin for inhalation PAH INS1009 inhaled treprostinil-prodrug Additional Programs in Orphan Diseases 61
Slide 62: Global Commercial Plan & Corporate Development
Global Commercial Plan & Corporate Development Wes Kaupinen Drayton Wise Dr. Olaf Bartsch 62
Slide 63: ARIKAYCE: Near-term Commercial Launches
ARIKAYCE: Near-term Commercial Launches Estimated Diagnosed Treatment Resistant Approval Patients Populations (estimate) Timing 2016 EU 30K 5-10K LAUNCH ALONE; EXPERIENCED EU LEADERSHIP IN PLACE 2017 55-70K 10-20K US LAUNCH ALONE; BUILDING AN EXPERIENCED U.S. TEAM 2018 Japan 30K 5-10K PARTNERSHIP DI
Slide 64: Europe
Europe Dr. Olaf Bartsch 64
Slide 65: Targeted EU Build-out to Pursue NTM and CF Indications
Targeted EU Build-out to Pursue NTM and CF Indications Medical Science Liaison Focus Phase I Initial Hires In Place ● Germany ● France Phase II ● UK ● Italy ● Sweden ● Netherlands 65
Slide 66: Diagnosed NTM Prevalence in EU 5
Diagnosed NTM Prevalence in EU 5 ● Includes both newly diagnosed patients and those with active ongoing disease diagnosed in a UK (4,103) previous year ● Treatment of NTM often extends Germany (5,373) well into the second year France (3,857) ● Of the ~30k diagnosed NTM Italy (3,714) patients Spain (
Slide 67: The Unmet Need - NTM
The Unmet Need - NTM NTM infections mostly occur in patient with underlying pulmonary diseases like COPD, bronchiectasis and asthma and CF. ● No on-label treatment available in Europe ● Cocktail of 3-4 antibiotics used off-label with significant side effects ● KOL quote in Europe: “Make sure you get
Slide 68: CF Patients with Pseudomonas Lung Infections
CF Patients with Pseudomonas Lung Infections 85% 2 Adults CF patients 80% with chronic Pseudomonas Increase in aeruginosa infection1 resistance to TOBI® Fatal genetic disease EU CF PATIENTS Life expectancy of 38-40 years 35K 1% to 3% 3 Annual decline in lung function 1 Adapted from Cystic Fibrosis F
Slide 69: The Need – CF / Pseudomonas aeruginosa
The Need – CF / Pseudomonas aeruginosa ● Several inhaled antibiotics Product Treatment Status already available with ArikayceTM Inhaled, liposomal formulation of amikacin Phase 3 trial completed in Europe and Canada acceptable treatment results TOBI® Inhaled tobramycin (Novartis) Marketed Combined t
Slide 70: The Addressable Market
The Addressable Market CF /Pseudomonas: slow market penetration foreseen ● 80% of the adult CF patients have chronic Pseudomonas infections ● The current gold standard is Tobramycin ● ARIKAYCE has some advantages in QoL (once daily) and is just another option for HCP´s All NTM: Major unmet need ● 33
Slide 71: Key Considerations for Insmed Europe
Key Considerations for Insmed Europe ● Launch strategy taking into account market sizes & market access possibilities, i.e. start in the 2 major countries (Germany & France), explore the UK and Italy and be opportunistic elsewhere (e.g. NL, Sweden) ● European launch provides learning opportunity for
Slide 72: EU Launch Preparation
EU Launch Preparation ● Focused build-out of presence in 2015 / 2016 ● Direct country organizations (Germany & France) ● 4 markets under evaluation for direct approach (UK, Italy, Sweden, NL) ● All other EU markets initially mapped through 3rd party consultants ● Supply & distribution through 3rd pa
Slide 73: Example NTM Lung Disease in Germany
Example NTM lung Disease in Germany Est. Number of NTM Country Lung Disease Mild Moderate Severe DX Patterns in DE are similar to other EU or Region Patients markets… France 3,857 945 2,175 737 ● Roughly 20% deemed “severe” Germany 5,373 1,171 3,116 1,086 ● DE system of referrals drives initial DX a
Slide 74: Example Germany
Example Germany Launch possible shortly after approval Pre launch strategy focused on: ● Medical education ● Disease Awareness (NTM) ● Definition of the unmet need ● (NTM & CF) ● KOL Management ● Advisory boards ● PR ● Market Access preparation ● G-BA consultation process ● AMNOG Dossier development
Slide 75: German Disease Awareness Campaign NTM Lung Disease
German Disease Awareness Campaign NTM Lung Disease ● German Pulmonologist Introductory Mailing ● DA website www.ntmfakten.de ● German Advisory boards ● German Ad Campaign ● Medical Education Campaign 75
Slide 76: NTM Lung Disease Awareness Launch
NTM Lung Disease Awareness Launch Disease Awareness Launch Phase 1: Germany Disease Awareness Doc Check Email Microsite Launch (Third party vendor) Newsletter (Third party vendor) Banner Ads* Banner Ads Digital February March April Print Insmed Introduction Letter Mailer DGP Booth NTMinfo Literature
Slide 77: US Commercial Planning
US Commercial Planning Drayton Wise 77
Slide 78: Key Elements of the U.S. ARIKAYCE Launch Planning
Key Elements of the U.S. ARIKAYCE Launch Planning Our Approach 1. Targeted Disease State Awareness, including our key learnings from EU experience 2. SALES + MEDICAL + MARKETING field-oriented and working as one 3. Augmenting therapy with services that maximize patient experience and convenience 4.
Slide 79: Hallmark of a Successful Orphan Disease Approach
Hallmark of a Successful Orphan Disease Approach: Begins Early with Highly Targeted Disease State Awareness Campaign 79
Slide 80: U.S. ARIKAYCE Launch Planning: Targeted Sales and Medical Presence
U.S. ARIKAYCE Launch Planning: Targeted Sales and Medical Presence Focused on Pulmonologists Field-based pulmonary 30 sales specialists Pulmonologists represent 3,000 over 80% of NTM patients Field Medical Science MSLs Liaisons To Convey NTM Medical Story Patient Maximizing convenience: Centricity ●
Slide 81: ARIKAYCE: Pricing and Reimbursement in NTM
ARIKAYCE: Pricing and Reimbursement in NTM Building the Value for Money Story US burden of illness study with major US payor confirms NTM is a costly disease: ● Monthly medical + pharmacy expenditures ≈ $5,000 less per month for those diagnosed and treated with existing therapies vs. those diagnosed
Slide 82: Corporate Development
Corporate Development Wes Kaupinen 82
Slide 83: Corporate Development Focus: Build on our Commitment to NTM Patients
Corporate Development Focus: Build on our Commitment to NTM Patients 2015 Corporate Goal: Acquire at least one external asset #1: Product/Product Candidate Serves an Orphan / Pulmonary Orphan Patient Population Key Corporate Development Criteria: #2: Therapeutic Indication is “NTM-Like” 83
Slide 84: Differential Criteria Based on Development Stage
Differential Criteria Based on Development Stage Commercial Development World-Class Patient Minimal Upfront Payment: Management in Rare Disease(s): ● Consideration structured to reward ● Enables leverage of successful success orphan commercial infrastructure Asset Currently “Under the Affordably
Slide 85: Broad Spectrum of Pulmonary Orphan Diseases
There is a Broad Spectrum of Pulmonary Orphan Diseases; Many of These Patient Populations are Severely Underserved Auto-Immune Diseases Genetic Surfactant Disorders Lung-Limited idiopathic disorders Trafficking and Lysosomal Storage Disorders Anti-basement membrane syndrome ABCA3 Idiopathic eosinoph
Slide 86: Insmed Vision: Leading Sustainable Pulmonary Orphan Company
Insmed Vision: Leading Sustainable Pulmonary Orphan Company Today NTM CF PAH 86
Slide 87: Insmed Vision: Leading Sustainable Pulmonary Orphan Company
Insmed Vision: Leading Sustainable Pulmonary Orphan Company Today Future Vision AATD NCFB NTM SARC LAM NTM CF PAH CF PAP PAH 87
Slide 88: Tech Ops, Cash and Tax Planning
Tech Ops, Cash and Tax Planning Andy Drechsler 88
Slide 89: Manufacturing and Supply Chain
Manufacturing and Supply Chain Producing Product at Scale for Clinical and Commercial Supply ● Multi-sourced supply chain ● Global distribution ● Building out commercial scale manufacturing capabilities 89
Slide 90: Manufacturing and Global Supply Chain – ARIKAYCE
Manufacturing and Global Supply Chain – ARIKAYCE Formulation and Filling of ARIKAYCE ● Current: 50kg Althea (US) ● Future: 50kg Althea (US) & 200kg Therapure (CAN) Testing ● Covance (UK) Labelling and Packaging ● For Clinical ● Xerimis (US) and Biotec (UK) ● For Commercial ● Almac (US) and Almac (UK
Slide 91: Manufacturing and Global Supply Chain – ARIKAYCE
Manufacturing and Global Supply Chain – ARIKAYCE Goal: Dual sources throughout the supply chain #1 - Amikacin (Italy) Almac UK #2 - Amikacin Device Patients (Germany) (China) Almac US #1 - Cholesterol (France) ARIKAYCE Manufacture (US & Canada) #2 - Cholesterol Xerimis US (India) Clinical Sites Biot
Slide 92: Manufacturing and Global Supply Chain – ARIKAYCE
Manufacturing and Global Supply Chain – ARIKAYCE Althea: Therapure: ● Current Clinical and Commercial ● Future Commercial production site launch site - On-line 2H 2015 ● 50kg scale ~ 5,000 vials of ● 200kg scale ~ 20,000 vials of ARIKAYCE per batch ARIKAYCE per batch 92
Slide 93: Manufacturing and Supply Chain – INS1009
Manufacturing and Supply Chain – INS1009 Formulation and Filling ● Toxicology studies ● Axcellerate (NJ) – 1 liter batch size ● Phase 1 Clinical studies ● AMRI (MA) – 1 liter batch size Testing ● PPD (WI) Labelling and Packaging ● For Clinical ● Xerimis (PA) Axcellerate April May June July Aug Sep O
Slide 94: Cash Needs from Operations
Cash Needs from Operations ● Cash Balance at 12/31/14 = $159.2M Cash Burn from Operations ● Timing/pace of 2015 spending ($ in millions) dependent on: 58 60 56 48 50 46 ● 212 trial enrollment 40 40 35 ● Manufacturing scale up 30 ● Manufacturing pace/schedule 20 10 ● European infrastructure build 0 1
Slide 95: Tax Planning
Tax Planning ● Commenced long term tax planning efforts in 2014 ● Anticipate that Ireland will be our IP and tax hub for EU ● New entities: Ireland, Germany, France & Netherlands ● Long term plans for manufacturing and distribution ● Current: Althea (San Diego, California, US) ● 2015: Addition of Th
Slide 96: Key Takeaways
Key Takeaways Mission ● Building a patient-focused and sustainable biopharmaceutical company focused on orphan, pulmonary indications ARIKAYCE ● Significant global NTM market opportunity ● Phase 3 global study in NTM underway ● MAA submitted/validated in EU for NTM and CF INS1009 ● IND to be filed 2
Slide 97: Thank You
Building a Patient-Focused and Sustainable Biopharmaceutical Company Focused on Orphan, Pulmonary Indications Thank You www.insmed.com Insmed Copyright 2015 © All Rights Reserved.