Slide 1: Engineering the Future for Inhaled Therapeutics
March 2016 NASDAQ: PULM Engineering the Future for Inhaled Therapeutics Exhibit 99.1
Slide 2: Disclaimers & Forward Looking Statements
Disclaimers & Forward Looking Statements This presentation contains forward-looking statements. Forward-looking statements may be identified by the use of forward-looking terms such as “anticipates,” “assumes,” “believes,” “can,” “could,” “estimates,” “expects,” “forecasts,” “guides,” “intends,” “is
Slide 3: Pulmatrix: Unlocking full potential of inhaled drugs
Enhanced lung delivery via iSPERSE engineered particle platform High percent of drugs to the lung Reduced systemic toxicities Pulmatrix: Unlocking full potential of inhaled drugs $33 BB + Market by 20191 Select Products: Address millions of patients and broad range of diseases Pulmonary Disease Mark
Slide 4: iSPERSE Technology Platform: Engineered for Exceptional Performance
iSPERSE Platform: Small, dense, dispersible, and respirable salt-containing particles Variegated formulation concepts to achieve product advantages (particle size, formulation, etc.) Clinically validated advantages over conventional technologies Broad novel IP portfolio into 2030s Particle Engineeri
Slide 5: Pulmatrix/Capsugel Alliance
Pulmatrix/Capsugel Alliance Capsugel Commercial Capability: Wide ranging expertise in dosage forms, including inhalation GMP through commercial scale spray-drying Internal R&D expertise in formulation science Provides Pulmatrix a CMO partner for all proprietary and out-licensed projects Pulmatrix iS
Slide 6: iSPERSE Technology Platform: Enabling High Value Product Opportunities
iSPERSE Technology Platform: Enabling High Value Product Opportunities iSPERSE Pipeline: Initial focus on 505(b)2 products Proprietary focus on rare/orphan pulmonary disease Partner strategy facilitates platform advancement in the context of valuable programs NCEs for Respiratory Disease Branded Gen
Slide 7: Pulmatrix Pipeline: iSPERSE Pulmonary Therapeutics
Pulmatrix Pipeline: iSPERSE Pulmonary Therapeutics Product Class Indication Formulation/Feasibility Non-Clinical Phase 1 Phase 2 Addressable Market PUR0200 LAMA COPD $7.9 BB by 20191 PUR1900 Anti-fungal Cystic Fibrosis Enter clinic 2016 20k patients Immuno- compromised 200k patients Allergic/invasiv
Slide 8: Pulmonary Fungal Infections in CF: High Incidence and Impact
Source: Chotirmall. Int J Biochem Cell Biol. 2014;1(52):161; Moss. Semin Respir Crit Care Med. 2015;36(2):207; Physician Interviews; ClearView Analysis. CF: Cystic Fibrosis. Pulmonary Fungal Infections in CF: High Incidence and Impact A combination of the host’s underlying immune state as well as pa
Slide 9: Current CF Anti-fungal Therapy Limited by PK and Side Effects
Current CF Anti-fungal Therapy Limited by PK and Side Effects Poor oral bioavailability and lung penetration in CF patients Subtherapeutic doses compromise efficacy and lead to recurrence Extensive drug-drug interactions Substrate and inhibitor of CYP3A4 Ivacaftor (Kalydeco) Lumacaftor (Orkambi) 21
Slide 10: PUR1900: Inhaled Itraconazole for CF
Relieves high treatment burden of oral antifungal therapy and improve outcomes Inhalation delivery provides improved safety and (likely superior) efficacy at a much lower total dose of drug Targeted delivery achieves high therapeutic dose directly to the site of infection avoiding GI/systemic exposu
Slide 11: PUR1900's Superior Market Profile
PUR1900’s Superior Market Profile PUR1900 Product Comparisons Oral Anti-Fungals 10-20mg inhaled Total Dose Delivered 100-250mg oral QD Dosage Frequency BID 10-100X higher lung levels with sustained (QD) profile Target Organ PK Profile (Lung) 50% of patients receive sub therapeutic lung levels >>100X
Slide 12: PUR1900 Reverses the Profile of Oral Dosing
PUR1900 Reverses the Profile of Oral Dosing PUR1900 achieves high lung concentrations and low systemic exposure Higher lung concentration achieved than oral dosing Lower systemic exposure achieved than oral dosing
Slide 13: PUR1900 Development Status
PUR1900 Development Status PUR1900 Phase 1 formulation selected; manufacturing work on-going at Bend / Capsugel PUR1900 drug product targets same aerodynamic properties and lung deposition as fungal spores Non-clinical 7-day multi-dose PK study complete 28-day GLP toxicity studies in life completed
Slide 14: PUR1900 Phase 1 Clinical Plan
PUR1900 Phase 1 Clinical Plan Phase 1/1b study to evaluate single ascending doses of PUR1900 in HNV and CF patients Establish safety, tolerability and PK Timing: FPI target Q3 2016 HNV data expected EOY 2016 CF patient dosing completion target Q1 2017 Cohort 1 N=6 10 mg Cohort 2 N=6 20 mg Cohort 3 N
Slide 15: PUR1900 Product Profile Feedback
PUR1900 Product Profile Feedback Source: Physician Interviews; ClearView Analysis. DPI: Dry Powder Inhaler; SOC: Standard of Care; QOL: Quality of Life; CFQ-R: CF Respiratory Questionnaire. Profile Element Safety and Tolerability Dosing and ROA “If anything, I would anticipate that itraconazole deli
Slide 16: PUR1900 Product Profile Feedback
PUR1900 Product Profile Feedback Physicians highlighted that ~85% of treated AB patients would likely be prescribed an inhaled antifungal, largely due to current dissatisfaction with available agents Source: Physician Interviews; ClearView Analysis. ABPA: Allergic Bronchopulmonary Aspergillosis. AB:
Slide 17: PUR1900: U.S. Payer Reaction
PUR1900: U.S. Payer Reaction Payers expressed enthusiasm for PUR1900, highlighting that an inhaled antifungal addresses a key unmet medical need in cystic fibrosis. Source: Payer Interviews; ClearView Analysis. ROA: Route of Administration; CF: Cystic Fibrosis. “I like the product. Seems like a TOBI
Slide 18: PUR1900 for CF: Significant Commercial Opportunity
PUR1900 for CF: Significant Commercial Opportunity First product designed for topical delivery to the lung of an infected CF patient Anticipate a much better superior safety, tolerability, and efficacy profile to current standard of care Worldwide sales potential estimated at >$250MM annual peak sal
Slide 19: PUR1900 Value Proposition outside CF
PUR1900 Value Proposition outside CF Source: Physician Interviews; ClerView Analysis. SAFS: Severe Asthma with Fungal Sensitization. Potential PUR1900 LCM Opportunities Potential LCM Opportunities for PUR1900 SAFS Fungal Pneumonia Other Organ Transplant* Asthma ABPA Lung Transplant Immuno- comprimis
Slide 20: iSPERSE Technology Platform: Enabling High Value Product Opportunities
iSPERSE Technology Platform: Enabling High Value Product Opportunities iSPERSE Technology Platform: Small, dense, dispersible, and respirable salt-containing particles Enabling efficient lung delivery of high drug loads Flexible formulation concepts enable small molecules, anti-infectives and biolog
Slide 21: PUR0200: Bronchodilator for COPD
PUR0200: Bronchodilator for COPD Low Mid High Drug Product Profile Branded generic iSPERSE version of a marketed $5B WW once daily LAMA bronchodilator Uses commercially available capsule-based passive DPI device Commercial scale capsule based device Clinical PD Data in COPD Patients Black circles sh
Slide 22: PUR0200 PK Data from COPD Patients: Cmax/AUC
PUR0200 PK Data from COPD Patients: Cmax/AUC Bioequivalence Plasma levels increase with dose and with similar kinetics At lowest iSPERSE dose, matched Cmax with lower AUC Matching Cmax at 1/6 reference dose Triple exposure at ½ reference dose
Slide 23: PUR0200 Clinical Design To Define Bioequivalent Formulation Targets
PUR0200 Clinical Design To Define Bioequivalent Formulation Targets Single dose PK study in HNV Primary outcomes: plasma PK Seven period cross-over design 42 subjects 5 PUR0200 formulations 2 periods of reference administration Formulation parameters designed to encompass reference product Clinicall
Slide 24: PUR0200 Fast Follower: Significant Near-Term Market Potential
PUR0200 Fast Follower: Significant Near-Term Market Potential Drug Class Muscarinic antagonist (bronchodilator) Dosing profile Once daily, inhaled Regulatory Path EU PK Bioequivalence Development Status Clinical trial Q1 2016 Addressable Market Size (2019)* 22MM in the EU Data from current PK Bioequ
Slide 25: Pulmatrix: Building Future Value
NCEs for Respiratory Disease Profile: Novel compounds to address high unmet need in COPD, CF and IPF iSPERSE enables: High drug loading and targeted lung delivery to maximize efficacy Evaluating multiple existing, development stage pharma compounds Pulmatrix: Building Future Value Reformulation for
Slide 26: iSPERSE for IPF
iSPERSE for IPF Need for improvements on approved drugs Nintedanib and Pirfenidone Despite efficacy, significant unmet need remains GI side effects and oral bioavailability limit tolerability, use and efficacy Critical need for inhaled options to overcome side effects and enable drugs against new ta
Slide 27: Pulmatrix Team: A History of Biotech Success
Pulmatrix Team: A History of Biotech Success Terry McGuire BOD Investor (AIR, Cubist, Ironwood) Robert Clarke, PhD CEO (12+ years) (AIR, Alkermes) Steve Gillis, PhD BOD Investor (Immunex, Bluebird) Kurt Graves BOD Independent (Vertex) Michael Higgins BOD-Audit Chair (Genzyme) Scott Rocklage, PhD BOD
Slide 28: Share Statistics
Share Statistics Trading Symbol PULM Total Shares Outstanding 14.5 million Financials Cash on Hand At Dec. 31, 2015 $18.9 million Runway based on anticipated expenses mid 2017 Cap Structure Insider Ownership 55% Key Financial Data
Slide 29: Anticipated Near Term Milestones
Anticipated Near Term Milestones PUR0200 Jan 2016 Initiated pilot BE PK study in EU Q2 2016 Clinical data from pilot BE PK study Q4 2016 Initiate development towards pivotal BE study with partner PUR1900 Jan 2016 Initiated GLP non-clinical safety program supporting Phase 1 Q2 2016 Completion of GLP
Slide 30: Pulmatrix Investment Opportunity
Pulmatrix Investment Opportunity High Value Pipeline PUR1900: Inhaled anti-fungal for Cystic Fibrosis PUR0200: Clinical-stage COPD candidate (R&D collaboration with Mylan) Opportunities for pipeline expansion Proprietary iSPERSE Technology Engineered particles enable highly efficient delivery of dru
Slide 31: Engineering the Future for Inhaled Therapeutics
March 2016 NASDAQ: PULM Engineering the Future for Inhaled Therapeutics