Insmed Incorporated · NASDAQ:INSM
Insmed — Investor Day (March 2015)
95 slides from Insmed's investor day presentation, Mar 24, 2015
Slide 1: Analyst and Investor Day March 24, 2015
Insmed Copyright 2015 © All Rights Reserved. Analyst and Investor Day March 24, 2015 NASDAQ: INSM
Slide 2: Agenda
2 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,
Slide 3: Safe Harbor Statement
3 Safe Harbor Statement ARIKAYCE is a trademark of Insmed Incorporated in the United States and various other countries. eFlow® Electronic Nebulizer (eFlow®) is a registered trademark of PARI Pharma GmbH. TOBI (Tobramycin Inhalation Solution) is a registered trademark of Novartis. Cayston (aztreonam
Slide 4: Insmed's Orphan Product Portfolio
5 Insmed’s Orphan Product Portfolio ARIKAYCE™ liposomal amikacin for inhalation INS1009 inhaled treprostinil-prodrug NTM PAH Additional Programs in Orphan Diseases
Slide 5: 2015 – The Year of the Deliverables
6 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
Slide 6: Team Introduction
7 Team Introduction Introduction & OverviewWill Lewis NTM Disease & Clinical TrialsDr. Gina Eagle Regulatory Peggy Berry R&D, INS1009 & IP Dr. Walter Perkins Dr. Gene Sullivan Tech Ops, Cash and Tax Planning Andy Drechsler Global Commercial Plan& Corporate Development Wes Kaupinen Drayton Wise Dr. O
Slide 7: NTM Lung Disease & Clinical Trials
8 NTM Lung Disease & Clinical Trials Dr. Gina Eagle
Slide 8: ARIKAYCE – NTM and CF Indications
9 ARIKAYCE™ liposomal amikacin for inhalation INS1009 inhaled treprostinil-prodrug NTM PAH Additional Programs in Orphan Diseases ARIKAYCETM – NTM and CF Indications
Slide 9: Nontuberculous Mycobacteria (NTM) Lung Infections
10 Nontuberculous Mycobacteria (NTM) Lung Infections Rx No Approved Treatmentin US and EU Progressive Lung Disease and Increased Mortality in High Risk Patient Groups Common in: Source: Company website; Clarity Pharma Research; American Journal of Respiratory and Critical Care Medicine; American Lun
Slide 10: Nontuberculous Mycobacteria (NTM) Lung Infections
11Source: Company website; Clarity Pharma Research; American Journal of Respiratory and Critical Care Medicine; AmericanLung Association; UCSF Medical Center.; Medscape Education; NTM Info and Research; Prevots (2010); (a) Adjemian et al 2012; (b) Yutaki, January 2015 Journal of Infection and chemot
Slide 11: Patient Journey Is Long and Difficult for Most
12 Patient Journey Is Long and Difficult for Most Only a few are diagnosed correctly from the outset Symptom Presentation Seek out Physician Mis-diagnosis Treatment initiation Symptom persistence Physician Referral/Change Diagnosis correction Treatment initiation Living with NTM (NTM Management) Pri
Slide 12: NTM Lung Disease Risk Factors and Mortality
13 NTM Lung Disease Risk Factors and Mortality
Slide 13: Nontuberculous Mycobacterium (NTM) Overview
14 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 disease is possible with many NTM species Nontub
Slide 14: Nontuberculous Mycobacterial Pulmonary Pathogens
15 Nontuberculous Mycobacterial Pulmonary Pathogens } M. Avium Complex (MAC) Common M. avium M. intracellulare M. kansasii M. abscessus M. chelonae Infrequent M. xenopi (zin oh’ pee) M. szulgai (sull’ guy) M. malmoense (mal’ moh en suh) M. fortuitum Rare M. celatum (sell ah’ tum) M. scrofulaceum M.
Slide 15: Risk Factors in MAC Lung Disease
16 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 16: Risk Factors in MAC Lung Disease Literature
17 Risk Factors in MAC Lung Disease Literature on Risk Factors in MAC Lung Disease Advanced Stage at Diagnosis Cavitary Lung Low BMI Macrolide- 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 17: High Risk Factor: Cavitary Lung
18 High Risk Factor: Cavitary Lung
Slide 18: NTM Lung Disease Advantages of LAI
19 NTM Lung Disease Advantages of LAI
Slide 19: Liposomal Amikacin for Inhalation (LAI)
20 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 lu
Slide 20: NTM Lung Disease Recap of Study
21 NTM Lung Disease Recap of Study 112
Slide 21: Study 112: Study Design
22 Study 112: Study Design Randomized 90 Subjects (1:1): Stratified: CF vs non-CF MAC vs M. abscessus Primary Endpoint: Efficacy • Change on Semi-Quantitative Scale for mycobacterial culture at Day 84 Screening perioda,b,cDay –42to Day –2 12 Weeks Once-Daily Dosing Background therapyd+ LAI* once dai
Slide 22: Study 112: Patient Profiles
23 Study 112 : Patient Profiles 0 5 10 15 20 25 30 35 40 6 - 12 Months >12 - 24 Months >24 Months Patients (n) •Length of Time on NTM Treatment Regimen Prior to Baseline Lesion Type N(%) Cavitary 6 (6.70%) Nodularand Cavitary 63 (70.0%) Nodular 21 (23.3%) • Lung Lesion Type on HRCTscan at Baseline
Slide 23: Study 112: Recap of Main Results
24 Study 112: Recap of Main Results . Reduction in mycobacterium burden (numerically superior, not statistically significant) .Achievement of a negative sputum culture in 11 out of 44 patients at Day 84 (statistically significant) .Safety: more frequent respiratory events; consistent similar inhaled
Slide 24: Study 112: Patients with negative sputum culture for NTM
25 Study 112: Patients with at least 1 negative sputum culture for NTM Treatment Arm CF Status NTM Oragnism Length of NTM Prior to Baseline (Months) Baseline Day 28 Day 56 Day 84 Day 112 Day 140 Day 16828 24>24>24>24 Broth Broth >24 >12 - 24 Early Term Resp 12 - 24>12 - 24>12 - 24>12 - 24>12 - 24 Br
Slide 25: Study 112: Patients with Sputum Culture Conversion
26 Study 112: Patients with Sputum Culture Conversion* Treatment Arm CF Status NTM Oragnism Length of NTM Prior to Baseline (Months) Baseline Day 28 Day 56 Day 84 Day 112 Day 140 Day 16828 24>24>24>24 Broth Broth>24>12 - 24>12 - 24>12 - 24>12 - 24 Broth Broth Broth 6 - 126 - 12 Broth 6 - 12 Broth>24
Slide 26: Study 112: Six Minute Walk Test
27 Study 112: 6 Minute Walk Test -60 -50 -40 -30 -20 -10 0 10 20 30 40 0 28 56 84 112 Least Squares Mean (SE) Study Day Change From Baseline in 6MWT Distance Missing Values Excluded (mITT Population) Arikayce Placebo P= .009
Slide 27: Study 112: Safety Recap
28 Study 112: Safety Recap 0% 10% 20% 30% 40% 50% Chest discomfort Wheezing Pyrexia Fatigue Nausea Haemoptysis Oropharyngeal pain Cough Dysphonia Infective exacerbation of bronchiectasis LAI (n=44) Placebo (n=45) LAI, liposomal amikacin for inhalation. Treatment-Emergent Adverse Events with >10% occ
Slide 28: NTM Lung Disease Study 212
Insmed NTM Lung Disease Study 212
Slide 29: Study 212 - Global Sites
30 Study 212-Global Sites North America . United States (34) . Canada (5) Europe . UK (15) . Germany (13) . Italy (12) . Spain (7) . France (6) Europe . Poland (4) . Austria (2) . Netherlands (1) Asia-Pac . Australia (17) . Japan (8) . New Zealand (3) Goal is to have 80+ sites active in the 1H 2015
Slide 30: Study 212: Study Design Overview
31 Study 212: Study Design Overview Primary Endpoint at 6 Months for Accelerated Approval . Percentage of patients that achieve culture conversion . Secondary: Six-minute walk test Screening Period 8 Months Daily Dosing ARIKAYCE once daily + Background Therapy Background Therapy Converters Continue
Slide 31: Study 212: Sputum Sampling
32 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 . Sputum processing will be . Performed per standard lab procedures . Globally aligned
Slide 32: Central Micro Labs Globally
33 Central Micro Labs Globally Region Central Micro Lab Microbiology Director USA/Canada University of Texas at Tyler Health Northeast (USA) Richard J. Wallace Jr.M.D. Europe Radboud University Medical Center (Netherlands) Jakko van Ingen, MD, PhD Australia/Asia Queensland Mycobacterium Reference La
Slide 33: Methodology Across Global Central Micro Labs
34 Methodology Across Global Central Micro Labs Methodology/Supplies TylerNorth America Radbound Europe QMRL 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
Slide 34: Study 212 Sample Size Calculation – Primary Endpoint
35 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
Slide 35: Study 212 Sample Size Calculation – Secondary Endpoint
36 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 rat
Slide 36: Study 212 Statistical Methods
37 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 37: Study 212 Statistical Methods – Secondary Endpoint
38 Study 212 Statistical Methods .Hypothesis –Secondary Endpoint H0: No between-treatment difference in mean change from baseline to Month 6 in 6MWT distanceH1: There is a between-treatment difference in mean change .Analysis of Secondary Endpoint Mixed-effects Model for Repeated Measures (MMRM) ove
Slide 38: Cystic Fibrosis Program TR02-110 Update
39 Cystic Fibrosis Program TR02-110 Update
Slide 39: Cystic Fibrosis Program Update
40 Cystic Fibrosis Program Update LAI for the Management of Pain Patients with Cystic Fibrosis Anecdotal Trial Experience •8 patients have requested compassionate use of LAI to follow TR02-110 . Very positive feedback from PIs in general regarding slowing of disease progression (even reversal in som
Slide 40: Reasons for Patient Discontinuation in TR02-110
41 Cystic Fibrosis Program Update Reasons for Patient Discontinuation in TR02-110 0 2 4 6 8 10 12 14 16 18 Death Adverse Event Withdrawl of Concent Lost to Follow-Up PermatureTermination of Study Other Prior LAI arm in TR02-108 Prior TOBI arm in TR02-108 Reason for Early Discontinuation Number of Pa
Slide 41: Regulatory Peggy Berry
42 Regulatory Peggy Berry peggy-berry-headshot.jpg
Slide 42: Regulatory Overview
43 Regulatory Overview ARIKAYCE .European update .US update .Rest of World activities INS1009 .US update .Rest of World activities
Slide 43: EMA Regulatory Timeline
44 EMA Regulatory Timeline Clock Stop Clock Stop Pre-submission Assessment of new marketing authorisation application Decision-making phase Day 1 (Feb 2015) Day 120 List of Questions Day 121 Day 180 List of Outstanding issues or opinion Day 181 Day 210 Opinion Day 277: EC Decision European Marketing
Slide 44: ARIKAYCE – US Update
45 D:\INSMED\2012\IR Deck\Artwork\New Logo\Logo Master v1a Small .pngARIKAYCE – 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-m
Slide 45: SubPart H Approval
46 SubPart H Approval .If agreed requirements are metand 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 46: ARIKAYCE – Rest of World Update
47 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
Slide 47: INS1009 – US & RoW update
48 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 one
Slide 48: Research and Development INS1009
49 Research and Development INS1009 Dr. Walter Perkins Dr. Gene Sullivan
Slide 49: Pulmonary Arterial Hypertension (PAH): Our Next Target Indication
50 Pulmonary Arterial Hypertension (PAH): Our Next Target Indication ARIKAYCE™ liposomal amikacin for inhalation INS1009 inhaled treprostinil-prodrug NTM PAH Additional Programs in Orphan Diseases
Slide 50: Pulmonary Arterial Hypertension (PAH)
51 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 channel
Slide 51: Current Marketed Prostanoids for PAH
52 Current Marketed Prostanoids for Pulmonary Arterial Hypertension Drug Dosing Pros Cons Flolan, Veletri (epoprostinil) Intravenous via continuous infusion Continuous drug level .Intravenous – central line infection .Requires infusion pump Ventavis (iloprost) Inhaled with 6-9x daily dosing Less inv
Slide 52: INS1009: Treprostinil-Prodrug Formulated in a Nanoparticle
53 INS1009: Treprostinil-Prodrug Formulated in a Nanoparticle ~100 nm in diameter C16TR .Hexadecyl-treprostinil (C16TR) Prodrug .Nanoparticles in aqueous suspension .Administered by state-of-the-art nebulizer .Patent applications filed 24 hour Coverage via Convenient Once-Daily Dosing without Large
Slide 53: INS1009 Provides Sustained Release of Treprostinil
54 A single 6 µg/kg dose administered by inhalation to ventilated rats *Estimation based on correlation of treprostinil plasma levels with changes in mean pulmonary arterial pressure (mPAP) from multiple experiments in rats From ERS 2014 Poster 2367. 0.01 0.10 1.00 10.00 0 1 2 3 4 5 6 7 Treprostinil
Slide 54: INS1009 Extends Vasodilation Period Significantly
55 INS1009 Extends Vasodilation Period Significantly Rat Acute Hypoxia Model of PAH 40.0 50.0 60.0 70.0 80.0 90.0 100.0 110.0 -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) mPAP (as % initial hypoxic value) Buffer Control Treprostinil INS1009 Mean
Slide 55: INS1009 Sustained Release over 4 Hours
56 INS1009 Provides Sustained Release of Treprostinil over 24 Hours 0.01 0.1 1 10 0 5 10 15 20 25 Time (h) Stays above threshold of 0.1ng/mL for full vasodilatory effect* Dose of 6 µg/kg Treprostinil Plasma Level (ng/mL) With nose-only inhalation plasma levels are higher than those in ventilated rat
Slide 56: Inhaled INS1009 Provides Sustained Release in Dogs
57 Tre 5 µg/kg Tre 16 µg/kg INS1009 7 µg/kg INS1009 22 µg/kg INS1009 46 µg/kg INS1009 95 µg/kg 0 6 12 18 24 30 36 42 48 0.01 0.1 1 10 Active Lung Dose in Ventilated Dogs (Single Inhalation) Treprostinil Plasma Level (ng/mL) Inhaled INS1009 Provides Sustained Release of Treprostinil in Dogs
Slide 57: INS1009 Better Tolerated in Dogs than Inhaled Treprostinil
58 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 shallo
Slide 58: INS1009 for PAH: Path to Market
59 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 Clinical . Phase 1 Study: Expect to Start by Year-end 2015 . Phase 2 Study: Exp
Slide 59: INS1009 for PAH: Clinical Plans through Phase 2
60 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 INS1009 for PAH: Clinical Plans through Phase 2
Slide 60: Insmed's Orphan Product Portfolio
61 Insmed’s Orphan Product Portfolio ARIKAYCE™ liposomal amikacin for inhalation INS1009 inhaled treprostinil-prodrug NTM PAH Additional Programs in Orphan Diseases
Slide 61: Global Commercial Plan & Corporate Development
62 Global Commercial Plan & Corporate Development Wes Kaupinen Drayton Wise Dr. Olaf Bartsch Drayton
Slide 62: ARIKAYCE: Near-term Commercial Launches
63 ARIKAYCE: Near-term Commercial Launches 2016 2017 2018 30K Diagnosed Patients Treatment Resistant Populations (estimate) 30K 5-10K 5-10K 55-70K 10-20K Estimated Approval Timing PARTNERSHIP DISCUSSIONS UNDERWAY LAUNCH ALONE;EXPERIENCED EU LEADERSHIP IN PLACE LAUNCH ALONE; BUILDING AN EXPERIENCED U
Slide 63: Europe Dr. Olaf Bartsch
Insmed Europe Dr. Olaf Bartsch
Slide 64: Targeted EU Build-out to Pursue NTM and CF Indications
65 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
Slide 65: Diagnosed NTM Prevalence in EU
66 Diagnosed NTM Prevalence in EU 5 . Includes both newly diagnosed patients and those with active ongoing disease diagnosed in a previous year . Treatment of NTM often extends well into the second year . Of the ~30k diagnosed NTM patients . ~20k are in EU5 . Germany has the largest population with
Slide 66: The Unmet Need - NTM
67 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 67: EU CF Patients
68 EU CF PATIENTS 35K Fatal genetic disease Life expectancyof 38-40 years CF Patients with Pseudomonas Lung Infections Adults CF patients with chronic Pseudomonas aeruginosa infection1 80% Increase inresistance to TOBI® 1Adapted from Cystic Fibrosis Foundation, Patient Registry Annual Data Reports,
Slide 68: The Need – CF / Pseudomonas aeruginosa
69 The Need – CF / Pseudomonas aeruginosa .Several inhaled antibiotics already available with acceptable treatment results .Market very competitive with downward pressure on prices .ARIKAYCE adds another treatment option for those patients intolerant or resistant to existing therapies Product Treatm
Slide 69: The Addressable Market
70 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 70: Key Considerations for Insmed Europe
71 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 fo
Slide 71: EU Launch Preparation
72 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 part
Slide 72: Example NTM Lung Disease in Germany
73 Example NTM lung Disease in Germany DX Patterns in DE are similar to other EU markets .Roughly 20% deemed “severe” .DE system of referrals drives initial DX at Internal or General Medicine (38%) followed by Pulmonologist (34%) while TX Patterns in DE are not. .Majority of patients seen in academi
Slide 73: Example Germany Launch
74 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 74: German Disease Awareness Campaign
75 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
Slide 75: NTM Lung Disease Awareness Launch
76 NTM Lung Disease Awareness Launch February March April Digital Print Disease Awareness Microsite Launch Insmed Introduction Letter Mailer Newsletter (Third party vendor) Banner Ads Doc Check Email (Third party vendor) Web Key Banner Ads* DGP Booth NTMinfo Literature Web Key Journal Ad Mailing of
Slide 76: Key Elements of the U.S. ARIKAYCE Launch Planning
78 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
Slide 77: Hallmark of a Successful Orphan Disease Approach
79 Hallmark of a Successful Orphan Disease Approach: Begins Early with Highly Targeted Disease State Awareness Campaign
Slide 78: U.S. Launch Planning: Targeted Sales and Medical Presence
80 U.S. ARIKAYCE Launch Planning: Targeted Sales and Medical Presence Focused on Pulmonologists Field-based pulmonary sales specialists Pulmonologists represent over 80% of NTM patients Field Medical Science Liaisons To Convey NTM Medical Story Maximizing convenience: .1. Patient services hub .2. Sp
Slide 79: ARIKAYCE: Pricing and Reimbursement in NTM
81 ARIKAYCE: Pricing and Reimbursement in NTMBuilding 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 diagnos
Slide 80: Corporate Development Wes Kaupinen
82 Corporate Development Wes Kaupinen
Slide 81: Corporate Development Focus
83 Corporate Development Focus: Build on our Commitment to NTM Patients 2015 Corporate Goal: Acquire at least one external asset Key Corporate Development Criteria: #1: Product/Product Candidate Serves an Orphan / Pulmonary Orphan Patient Population #2: Therapeutic Indication is “NTM-Like”
Slide 82: Differential Criteria Based on Development Stage
84 Differential Criteria Based on Development Stage Commercial Development . World-Class Patient Management in Rare Disease(s): .Enables leverage of successful orphan commercial infrastructure . Affordably Priced: .Naturally limits pursuit of some therapeutic areas . Minimal Upfront Payment: .Consid
Slide 83: Broad Spectrum of Pulmonary Orphan Diseases
85 There is a Broad Spectrum of Pulmonary Orphan Diseases; Many of These Patient Populations are Severely Underserved Auto-Immune Diseases Anti-basement membrane syndrome Pulmonary alveolar proteinosis Lung-Limited idiopathicdisorders Idiopathiceosinophilic pneumonias Tracheobronchopathiaosteochondr
Slide 84: Insmed Vision: Leading Sustainable Pulmonary Orphan Company
86 Insmed Vision: Leading Sustainable Pulmonary Orphan Company CF Today NTM PAH
Slide 85: Insmed Vision: Future Vision
87 Insmed Vision: Leading Sustainable Pulmonary Orphan Company CF Today Future Vision NTM PAH CF NTM PAH AATD LAM SARC NCFB PAP
Slide 86: Tech Ops, Cash and Tax Planning
88 Tech Ops, Cash and Tax Planning Andy Drechsler p
Slide 87: Manufacturing and Supply Chain
89 Manufacturing and Supply ChainProducing Product at Scale for Clinical and Commercial Supply .Multi-sourced supply chain .Building out commercial scale manufacturing capabilities .Global distribution
Slide 88: Manufacturing and Global Supply Chain – ARIKAYCE
90 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) Dev
Slide 89: Manufacturing and Global Supply Chain – ARIKAYCE
91 Manufacturing and Global Supply Chain – ARIKAYCE #1 - Cholesterol (France) #1 - DPPC (Germany) Device (Germany) Testing Sites (UK) Almac US Xerimis US Clinical Sites ARIKAYCE Manufacture (US & Canada) #2 - Cholesterol (India) #2 - Amikacin (China) #1 - Amikacin (Italy) #2 - DPPC (Japan) Biotec UK
Slide 90: Manufacturing and Global Supply Chain – ARIKAYCE
92 Manufacturing and Global Supply Chain –ARIKAYCE Althea: .Current Clinical and Commercial launch site .50kg scale ~ 5,000 vials of ARIKAYCE per batch Therapure: .Future Commercial production site - On-line 2H 2015 .200kg scale ~ 20,000 vials of ARIKAYCE per batch
Slide 91: Manufacturing and Supply Chain – INS1009
93 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) Z:\Presentations\Nanoparticle Cartoons\Manufa
Slide 92: Cash Needs from Operations
94 Cash Needs from Operations .Cash Balance at 12/31/14 = $159.2M .Timing/pace of 2015 spending dependent on: .212 trial enrollment .Manufacturing scale up .Manufacturing pace/schedule .European infrastructure build .Debt $25M: interest only, due 1/1/16 .Potentially able to modify 35 40 46 48 56 58
Slide 93: Tax Planning
95 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 Thera
Slide 94: Key Takeaways
96 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 2H
Slide 95: Thank You
97 Insmed Copyright 2015 © All Rights Reserved. Thank You Building aPatient-Focused and Sustainable Biopharmaceutical Company Focused on Orphan, Pulmonary Indications www.insmed.com