Insmed Incorporated · NASDAQ:INSM
Insmed — Investor Presentation (February 2018)
30 slides from Insmed's investor presentation, Feb 14, 2018
Slide 1: Safe Harbor Statement
Safe Harbor Statement 2 This presentation contains forward looking statements. "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 number of risks and uncertainties. Words herein suc
Slide 2: Our Mission
3 To transform the lives of patients battling serious rare diseases OUR MISSION
Slide 3: Insmed: A Global Biopharmaceutical Company Focused on Rare Disease
Insmed: A Global Biopharmaceutical Company Focused on Rare Disease 4 POSITIVE TOP-LINE PHASE 3 RESULTS FOR ALIS GLOBAL COMMERCIAL OPPORTUNITY ALIS LIFE CYCLE MANAGEMENT GROWING PIPELINE
Slide 4: Treatment of NTM lung disease represents significant unmet need
Treatment of NTM lung disease represents significant unmet need Current antibiotic regimens are inadequate in treating NTM disease, with no known approved therapies in US and EU 5 1 2 3 4 5 Declining lung function Globally prevalent and growing at approximately 8% annually in the US Up to 33% mortal
Slide 5: Estimated 2018 US Market Opportunity in NTM
Estimated 2018 US Market Opportunity in NTM 6 REFRACTORY NTM CAUSED BY MAC 10K - 15K NTM CAUSED BY MAC 60K - 85K DIAGNOSED NTM 75K - 105K
Slide 6: Global Opportunity – Near-Term Focus on US, Japan and EU
Global Opportunity – Near-Term Focus on US, Japan and EU 7 *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 Kingdom 125K – 145K total di
Slide 7: Science of ALIS/Our Solution
Science of ALIS/Our Solution 8 Combination of Free and Liposomal Amikacin Scintigraphy Demonstrates Diffuse Pulmonary Distribution Tested across 5 strains of MAC in vitro ALIS More Effective than Amikacin In Vitro Uptake in the Lung Macrophage Tested Across 5 Strains of NTM
Slide 8: CONVERT study design: ALIS once daily + Guideline Based Therapy
ALIS once daily + Guideline Based Therapy 9 Adult Patients with NTM (MAC) Lung Disease Failing at least 6 months of a multi-drug regimen KEY INCLUSION CRITERIA Guideline Based Therapy R Screening period Month-6 sputum culture PRIMARY ENDPOINT Culture Conversion at 6 months INS-212 non-converters INS
Slide 9: Top-line Data Indicates CONVERT Study Met Primary Endpoint
Top-line Data Indicates CONVERT Study Met Primary Endpoint* Addition of ALIS to GBT** eliminated evidence in sputum of NTM lung disease caused by MAC by Month 6 in 29% of patients, compared to 9% of patients on GBT alone (p <0.0001) 20% absolute difference in treatment groups in favor of ALIS arm 10
Slide 10: Top-line CONVERT Study Results – Secondary Endpoints at Month 6
Top-line CONVERT Study Results – Secondary Endpoints at Month 6 11 Time to Conversion Patients in the GBT-only arm took approximately 30% longer to convert when compared to patients on ALIS plus GBT (p<0.0001) 6-Minute Walk Distance (6MWD) No statistically significant difference between patients in
Slide 11: Top-Line CONVERT Safety Summary
Top-Line CONVERT Safety Summary Adverse events, consistent with those seen with use of inhaled antibiotics, more frequent in ALIS + GBT arm Serious treatment emergent adverse events were similar between treatment arms No distinctions between treatment arms of hearing loss or renal impairment 12 2:1
Slide 12: CONVERT Study Demographics Summary
CONVERT Study Demographics Summary ALIS (n=224) GBT (n=112) Smokers 11.6% (26) 8.9% (10) Guideline Based Therapy Prior to Enrollment On Treatment 89.7% (201) 90.2% (101) Off Treatment for > 3 months 10.3% (23) 9.8% (11) Geography US 41.5% (93) 42.9% (48) Asia 21.4% (48) 17.9% (20) EU + ROW 37.1% (83
Slide 13: Additional interim top-line results from INS-212 and INS-312 announced January 2018
14 Additional interim top-line results from ongoing INS-212 and INS-312 studies announced January 2018
Slide 14: Sputum Culture Conversion in INS-312 Consistent with INS-212
Interim Descriptive Data Show that Sputum Culture Conversion in INS-312 is Consistent with Sputum Culture Conversion in Top-Line Results from INS-212 15 Percentage of Patients with Sputum Culture CONVERSION AS OF DECEMBER 2017* (3 Consecutive Negative Sputum Cultures) 29% of patients initiating ALIS
Slide 15: Continued Treatment with ALIS + GBT Results in More Patients Achieving Culture Conversion
16 29% (65/224) *As of December 2017, 57 of 73 patients in the prior ALIS + GBT Arm in INS-212 were evaluable for Sputum Culture Conversion 12% of prior non-converters from INS-212 ALIS + GBT arm achieved culture conversion by Month 6 in INS-312 Interim Descriptive Data Show Continued Treatment with
Slide 16: Durability of Culture Conversion Higher in ALIS + GBT Arm
Interim Descriptive Data Show that in INS-212, Durability of Culture Conversion is Substantially Higher in ALIS + GBT Arm (61% vs. 0% as of December 2017)* 17 Percentage of Converters from each arm who remain culture negative three months off treatment ALIS + GBT shows a more durable culture convers
Slide 17: INS-212 and INS-312 Safety Summary
INS-212 and INS-312 Safety Summary No Change in Overall Safety Assessment for ALIS Based on Interim Data INS-212 Adverse events, consistent with those seen with use of inhaled antibiotics, more frequent in ALIS + GBT arm Serious treatment emergent adverse events were similar between treatment arms N
Slide 18: NDA Submission planned under Subpart H Pathway to US Submission
NDA Submission planned based on primary endpoint under Subpart H Pathway to US Submission – Division of Anti-Infective Products 19 Breakthrough Designation Orphan and QIDP Accelerated Approval Full Approval Requirement 12 years of exclusivity (7 orphan and 5 QIDP) and anticipates priority review Gui
Slide 19: Therapeutic Specialists Expected to Cover the US Market
72 Therapeutic Specialists Expected to Cover the US Market Based on Concentration of NTM Treatment Among Providers 20 Source: Symphony Health, PatientSourceTM November 2013 – October 2016 ~5K physicians manage 70% of diagnosed NTM patients 8 in 10 diagnosed NTM patients managed by Pulmonologist or I
Slide 20: Actively Educating the Community About NTM
Actively Educating the Community About NTM 21 “1,000 Words about NTM” “Over & Over” Healthcare Professional Disease Awareness Campaign NTMFacts.com Patient Disease Awareness Campaign AboutNTM.com
Slide 21: Estimated Average First-Year Insurance Spending in NTMLD
NTMLD COPD IPF PAH Lung Cancer $48,169 $22,901 $44,313 $51,753 $69,005 *First year insurance spending was summarized from a national managed care claims database following the first diagnosis of each disease between 2007 and 2016 and standardized using RBRVS (Resource Based Relative Value Scale) by
Slide 22: Estimated Annual Cost of Leading Respiratory Orphan Drugs
¹ Based on Wholesale Acquisition Cost (WAC). Source: Redbook (7/7/17) ² IPF and PAH patients frequently require use of multiple agents making annual costs significantly higher ³ Cystic Fibrosis agents’ WAC represents 6 months of therapy due to every other month dosing Cystic Fibrosis ³ 30,000 patien
Slide 23: ALIS Value Proposition – Treatment Reduces Cost
ALIS Value Proposition – Treatment Reduces Cost Pulmonary Nontuberculous Mycobacterial Infections: Healthcare Resource Utilization and Costs in Medicare Patients at a US Health Plan. Abraham et al. Methods: Medical and pharmacy claims between January 1, 2007, and May 31, 2014, were used to identify
Slide 24: Established Supply Chain
Established Supply Chain Redundant drug supply chain Commercial-scale manufacturing capacity on-line 25 50L 200L Patheon* (part of Thermo Fisher Scientific) 450L Patheon long term project initiated 4Q 2017
Slide 25: ALIS: Multiple Layers of Market Exclusivity
Potential to extend exclusivity to May 2033 with system claims Japanese patent portfolio 2033 2027 2030 2028 2029 2026 ALIS: Multiple Layers of Market Exclusivity 26 US patent portfolio Coverage into Jan. 2034 Potential 6-month pediatric extension US: 12-year exclusivity (orphan and QIDP designation
Slide 26: Growing Pipeline
PRECLINICAL PHASE 1 PHASE 2 PHASE 3 ARIKAYCE®: Amikacin Liposome Inhalation Suspension (ALIS) Refractory NTM: M. avium complex (MAC) ARIKAYCE®: Amikacin Liposome Inhalation Suspension (ALIS) Front line, maintenance, other non MAC NTM species, e.g. M Abcessus INS1007: DPP1 Inhibitor Non-CF bronchiect
Slide 27: 2018 Financial Guidance – 1st Half
2018 Financial Guidance – 1st Half 28 ALIS Commercial Activities: Hire and deploy: Therapeutic Specialists, Patient Services and Key Account Directors Continue to invest in physician and patient disease awareness campaigns Advance Japan/global expansion ALIS Manufacturing Investments: Continue build
Slide 28: Insmed: A Global Biopharmaceutical Company Focused on Rare Disease
Insmed: A Global Biopharmaceutical Company Focused on Rare Disease 29 POSITIVE TOP-LINE PHASE 3 RESULTS FOR ALIS GLOBAL COMMERCIAL OPPORTUNITY ALIS LIFE CYCLE MANAGEMENT GROWING PIPELINE
Slide 29: Insmed: A Global Biopharmaceutical Company Focused on Rare Disease
Insmed: A Global Biopharmaceutical Company Focused on Rare Disease GROWING PIPELINE ALIS LIFE CYCLE GLOBAL MANAGEMENT POSITIVE TOP- COMMERCIAL LINE PHASE 3 OPPORTUNITY RESULTS FOR ALIS
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