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PULMATRIX Corporate Overview February
2018 NASDAQ: PULM Exhibit 99.1
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and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements, including risks that we will not have sufficient working capital, that we will have delays in
obtaining, or we will be unable to obtain, FDA or other regulatory approvals for our products, unable to establish collaborations, or that our products will not be commercially viable, among other risks. A discussion of these and other factors,
including risks and uncertainties with respect to the Company, is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 10-K and its quarterly reports on Form
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Three Study Readouts in 2018-2019 with
Products Exceeding $6B in Estimated Revenue Potential ABPA = Allergic Bronchopulmonary Aspergillosis CF = Cystic Fibrosis LEAD PROGRAM iSPERSE Pulmazole Anti-Fungal PUR1800 novel narrow spectrum kinase inhibitor (NSKI) for AECOPD Acute
exacerbation treatment paradigm, vs prevention, leads to faster and cost efficient development plan First in class (U.S. & E.U.) inhaled non-steroidal anti-inflammatory Demonstrated anti-inflammatory target activity, safety and tolerability in
stable COPD patients 28-day non-clinical safety data 4Q 2018 ~$4.3B U.S. and E.U. estimated revenue opportunity Second PROGRAM iSPERSE PUR1800 Kinase Inhibitor Pulmazole inhaled itraconazole for ABPA Reduced development risk as itraconazole
(Sporanox) was originally approved 25 years ago - 505(b)(2) pathway may allow for accelerated development 1st to market (U.S. & E.U.) inhaled anti-fungal Qualified Infectious Disease Product (QIDP) designation obtained in 2017 for CF and ABPA
Expected Phase 1 data mid-2018 and 28-day biomarker data in ABPA patients mid-2019 ~$2.3B U.S. and E.U. estimated revenue opportunity Accretive Factors Contributing to Probability of Success "De-risked" programs with 505(b)(2) Pulmazole
and precedent PUR1800 data in COPD patients iSPERSE dry-powder delivery technology studied in patients demonstrating safety, tolerability and drug enabling capabilities Underlying iSPERSE intellectual property carries well into
2030's with additional patent protection through specific drug development AECOPD = Acute Exacerbations in Chronic Obstructive Pulmonary Disease LOE = Loss of Exclusivity
Product Indication 2018* 2019*
Milestone Pulmazole Anti-fungal Allergic Bronchopulmonary Aspergillosis (ABPA) in Severe Asthma Phase 1 Mid-2018 Phase 2a Mid-2019 PUR1800 NSKI Acute Exacerbations of COPD (AECOPD) Phase 2a 3Q 2019 PUR5700 NSKI Idiopathic Pulmonary Fibrosis (IPF)
Phase 1 (2019) PUR0200-US LAMA Chronic Obstructive Pulmonary Disease (COPD) Out-Licensed to Vectura for U.S. 09/2017 PUR0200-EU LAMA E.U. Out-Licensing Opportunity Robust Pipeline with Projected Near-Term Value Catalysts NSKI = Narrow Spectrum
Kinase Inhibitor LAMA = Long-Acting Muscarinic Antagonist *Estimated Milestones Pre-Clinical PK Bioequivalence Phase 1 KEY: 2018-2019 Development Focus Phase 2a 28-day NCS Phase 2a Phase 1
iSPERSE Best-in-Class Engineered
Particles Enable Development and Patient Care Advantages PK = Pharmacokinetic iSPERSE enables the sickest patient to get the right dose every time iSPERSE Differentiation iSPERSE Advantages Small, dense, dispersible, respirable
particles Flow rate independent performance (consistent delivery of high drug loads) Scalable platform Broad IP portfolio into 2030s Can be used with most drugs, small molecule or biologic Can be used with virtually any device (i.e., metered-dose,
reservoir, or blister-based inhalers) Requires low inspiratory flow given high particle dispersibility and penetration deep into lung Can deliver large doses into lungs (tens of milligrams) with high lung delivery efficiency Avoids first-pass effect
and systemic side-effects with improved PK profile compared to oral delivery iSPERSE 1 m
Pulmazole - Inhaled Antifungal
Inhaled itraconazole to Treat Allergic Bronchopulmonary Aspergillosis (ABPA) in Asthmatics
3rd Line 2nd Line ABPA-Asthma Overview
Treatment Flow ABPA is a Chronic Disorder, Typically Requiring Both Oral Steroid and Antifungal Treatment Source: Shah. Allergy Asthma Immunol Res. 2016;8(4):282; Agarwal. Clinical & Experimental Allergy. 2013;43:850; Patterson. Proc Am Thorac
Soc. 2010;7:237; Tracy. J. Fungi. 2016;2:17; Physician Interviews; ClearView Analysis. IgE = Immunoglobulin E Antibodies 1st Line Follow-up: Chest Radiograph, Spirometry, Total Serum IgE Glucocorticoids (4 Months) Acute ABPA Patient (Stage I)
Remission (Stage II) Response Exacerbation (Stage III) Steroids (4 Months) & antifungal (6 Months) Long Term Therapy: Azoles, Glucocorticoids, Omalizumab, Methylprednisolone Pulses, Nebulized Amphotericin Treatment-Dependent (Stage IV) ABPA in
asthmatics is caused by hypersensitivity to Aspergillus fumigatus Persistence of A. fumigatus in the airways leads to local inflammation, mucus production and airway hyper-reactivity Untreated ABPA may result in pulmonary fibrosis, respiratory
failure and potentially death Antifungals are mostly reserved for Stage III and IV patients but also used in Stage I in ~30% of patients Itraconazole (Sporanox) is the most commonly used antifungal
Reducing Fungal Burden Plays an
Important Role in ABPA Treatment Pulmazole expected to reduce fungal burden and attenuate inflammatory response DC = dendritic cell; AM = alveolar macrophage Moss (2014) Eur Resp J 43:1487-1500 Antifungals Steroids Omalizumab Oral antifungals,
including itraconazole, are limited by poor pharmacokinetics and safety concerns associated with high plasma concentrations ABPA diagnosis: High IgE (>1000IU/mL) Skin prick positivity to Aspergillus Eosinophilia (>500 cells L) Elevated
IgE/IgG Antibodies to Aspergillus Radiographic pulmonary opacities consistent with ABPA Growing fungus in the airway activates a Th2 lymphocyte response Resulting eosinophilic inflammation and IgE production drive airway obstruction and clinical
symptoms Steroids are immunosuppressive and may exacerbate fungal growth or antigen production Antifungals inhibit fungal growth, improve fungal clearance and reduce antigen production Act to reduce inflammation, improve clinical outcomes and may be
Literature Indicates Antifungals
Improve ABPA Outcomes Literature and Physicians Both Confirm the Importance of Antifungals in ABPA Treatment Physicians View Antifungals as Clinically Effective* Experts interviewed indicated antifungal agents reduce exacerbations and chronic
steroid use Physicians attribute results to reduction in ABPA patients' fungal burden Physicians interviewed currently use antifungals in ~50% of patients Itraconazole is currently used in majority of patients receiving antifungal therapy
Antifungal agents have been shown to reduce exacerbations, improve lung function and reduce steroid use in ABPA Physicians consider antifungals as add-ons to steroids, although use is limited by a lack of robust data and known safety risks 1999,
Salez et al. Itraconazole improved pulmonary function, and decreased total IgE, exacerbations, and steroid use in a cohort of 14 patients 2000, Stevens et al. Itraconazole improved pulmonary function, and decreased steroid use and total IgE in a
randomized double-blind trial with 55 patients 2003, Wark et al. Itraconazole decreased total IgE and the exacerbation frequency in a randomized, double-blind trial with 29 patients 2012 Chishimba et al. A review indicated that vori./posaconazole
improved QOL, and reduced steroid use and exacerbations in 20 patients 2016, Ram et al. Nebulized amphotericin B reduced exacerbations compared to nebulized budesonide in a study in 21 patients Source: Chishimba. Journal of Asthma. 2012; 49(4):423;
Stevens. NEJM.2000;342(11):757; Wark. J Allergy Clin Immuno. 2003;111; 952; Salez.Chest. 1999; 116(6):1665; Ram. Journal of Asthma. 2016:517; Physician Interviews; ClearView Analysis. QOL = Quality of Life *Based on certain physicians interviewed by
Pulmazole Targets ABPA
Patients' Unmet Need for an Efficacious and Well Tolerated Antifungal Meaningful ABPA Disease Burden ~1.5% of adult asthma patients (~300K in U.S.) suffer 1-3 exacerbations annually Untreated ABPA may result in pulmonary fibrosis, respiratory
failure and potentially death Severe asthma patients annually average 5 hospitalization days at ~$15K Approximately 45% of patients experience recurrent relapses or steroid dependence when treated with steroids alone Pulmazole Anticipated Benefits
Improved efficacy with consistent, and potentially greater, drug levels in the lung Improved safety & tolerability from lower systemic exposure via inhalation Reduced steroid burden by reduction in fungal growth and antigen production Source:
Shah. AAIR. 2016;8(4):282; Wark. JACI. 2003;111; 952; Salez. Chest. 1999;116(6):1665; GINA 2015; Patterson. Arch Intern Med. 1986: 146:916-8; Physician Interviews; ClearView Analysis. QOL = Quality of Life. MOA = Mechanism of Action UNMET NEED
Reduce exacerbations & prevent development of progressive lung injury Minimize toxicity associated with systemic absorption by delivering drug locally Optimize efficacy by delivering higher dose of drug directly to lung Reduce steroid use &
minimize steroid toxicity
Lower Plasma Concentration of
Pulmazole vs. Oral Sporanox 5X Greater Lung Concentration of Pulmazole vs. Oral Sporanox Pulmazole: Anticipated Efficacy & Safety Benefits vs Oral Itraconazole (Sporanox) In preclinical dog studies, Pulmazole reverses the systemic to lung
exposure ratio observed with oral Sporanox Data indicates potential for efficacy and tolerability advantages with Pulmazole compared to the preferred ABPA-Asthma oral antifungal treatment (Sporanox) Pulmazole 1.25 mg/kg Sporanox 5 mg/kg NOTE: Dosing
reflects estimated Pulmazole lung dose vs. Sporanox oral dose Pulmazole 1.25 mg/kg Sporanox 5 mg/kg
Survival Pulmazole Compared to Oral
Sporanox and Placebo Pulmazole: Inhaled Itraconazole Improved Survival in an Immunosuppressed Guinea Pig Model In a lethal model with aspergillus infected immunosuppressed guinea pigs, Pulmazole achieved ~90% survival vs 30% with oral Sporanox at 14
days Data re-asserts potential efficacy advantages for Pulmazole compared to the preferred ABPA-Asthma oral antifungal treatment (Sporanox) Pulmazole 1 mg/kg Sporanox 10 mg/kg NOTE: Dosing reflects estimated Pulmazole lung dose vs. Sporanox oral
Part 1: Single Ascending Dose
Healthy volunteers (n=6/cohort) Part 2: Multiple Ascending Dose Healthy volunteers (n=6/cohort) Part 3: Single Dose Crossover Asthmatics (n=16) Ph1/1b Safety-Tolerability Data plus PK Data Will Highlight PK Advantages Over Oral Sporanox Pulmazole
(25 mg) Pulmazole (10 mg) Pulmazole (5 mg) Pulmazole (20 mg) Pulmazole (10 mg) Phase 1: Safety, Tolerability and PK Study in Healthy Volunteers and Asthmatics 2018 2019 1Q 2Q 3Q 4Q 1Q 2Q 3Q Phase 1 Data Phase 1 Start Study Design & Protocol
Synopsis Part 1: SAD 6 patients X 3 cohorts (optional 4th cohort up to 35 mg) Doses 5, 10, 25 mg Safety and tolerability endpoints including PFTs Part 2: MAD 6 patients X 2 cohorts (optional 3rd cohort up to 35 mg) 14 days @ 10mg or 20 mg QD Safety
and tolerability endpoints including PFTs Part 3: Single Dose Crossover PK 16 mild-to-moderate asthmatics single dose crossover Oral Sporanox (200 mg) or inhaled Pulmazole (20mg) NOTE: Pulmazole doses > 5mg are expected to provide equivalent lung
exposure to 200 mg oral Sporanox Lung PK assessed by induced sputum Plasma PK 14-day washout between crossover doses Pulmazole (20 mg) Sporanox (200 mg; oral) Sporanox (200 mg; oral) Pulmazole (20 mg)
Ph2a 28-day Safety-Tolerability
Study Also Evaluating Pulmonary Function and Biomarkers of Interest Randomized, double-blind, placebo controlled study (1:1 randomization; each cohort n = 16) Exploratory endpoints: Pulmonary Function (FEV1) Biomarker IgE plasma concentrations
Sputum and plasma eosinophils IgE and IgG specific to A. fumigatus antigens in plasma Aspergillus burden in sputum Phase 2a: 28-day Safety, Tolerability, Pulmonary Function and Biomarker Study in Asthmatic ABPA Patients Pulmazole (10 mg) Pulmazole
(20 mg) Placebo ABPA Patients Safety Tolerability Pulm. Function Biomarkers 2018 2019 1Q 2Q 3Q 4Q 1Q 2Q 3Q Phase 2a Start Phase 2a Data Dose to be confirmed upon completion of phase 1/1b Study Design & Protocol Synopsis Patient Profile
Moderate-to-severe asthmatics with confirmed and stable ABPA, meeting diagnostic criteria for ABPA as per Modified ISHAM working group 2013 criteria for diagnosis of ABPA M/F, Ages 18-65 Double blind, PBO-controlled safety and tolerability study 1:1
randomization; each cohort n = 16 Sample size/arm consistent with Wark study. 28 days @ PBO QD 28 days @ 5 mg Pulmazole QD 28 days @ 10 mg Pulmazole QD 28 days @ 20 mg Pulmazole QD NOTE: Pulmazole doses > 5mg are expected to provide equivalent
lung exposure to 200 mg oral Sporanox Primary safety and tolerability endpoints Exploratory Endpoint Evaluation Note FEV1 data following 16 weeks in Wark: increase of 7.9% with itraconazole and a decrease of 1.9% with placebo (P = 0.5 -
perhaps because of small sample size)) Note, biomarker endpoints descriptive, but based on the Wark paper, we should expect to see reductions in serum IgE, sputum eosinophils and IgG antibodies specific to A fumigatus (25-40% reduction depending on
marker) as early as 4 weeks. Pulmazole (5 mg)
Pulmazole - Commercial
1st Line Antifungal Usage More than
Doubles Pulmazole: Efficacy and Safety Advantages Drive Prescriber Preference The Pulmazole Advantage Pulmazole Converts Majority of Oral Azole ABPA Market Patients Receiving Pulmazole Total Antifungal Therapy Pivotal Trial Design Oral Prednisone +
Placebo vs. Oral Prednisone + Pulmazole 4-month treatment following patients for 8 additional months Target Product Profile Primary Endpoint: 50% improvement in exacerbation number or rate Secondary Endpoints: FEV1 Unmet Need in ABPA-Asthma Frequent
exacerbations due to poor disease management Dependence on oral steroids as primary treatment option Safety / tolerability issues with both oral steroids and oral antifungals Pulmazole Value Drivers Inhaled delivery minimizes systemic exposure to
improve side-effect profile iSPERSE lung delivery enables high drug concentrations to lung with potential efficacy benefit itraconazole MOA and effectiveness in ABPA-Asthma is established Source: Physician Interviews; Payer Interviews; ClearView
Pulmazole: Payers Anticipate
Biologic Pricing with Minimal Access Restrictions Source: Chishimba. Journal of Asthma. 2012;49(4):423; Stevens. NEJM. 2000;342(11):757; Payer Interviews; ClearView Analysis. Red Book; Payer Interviews; Source: Payer Interviews; ClearView Analysis.
PA = Prior Authorization. Annual Gross Cost (U.S.) $100 K $10 K $30 K $70 K $50 K Anticipated Optimized Pricing Range Payer Tactics Overview Access Liberal Restricted Case-by-case Evaluation Restrict Beyond Trial Pop. Proof of Efficacy for Renewal
PA (e.g., Restrict to Trial Pop.) $50 K+ Access Liberal Restricted Case-by-case Evaluation Restrict Beyond Trial Pop. Proof of Efficacy for Renewal PA (e.g., Restrict to Trial Pop.) $30 - 50 K Access Liberal Restricted $5 - 30 K
Case-by-case Evaluation Restrict Beyond Trial Pop. Proof of Efficacy for Renewal PA (e.g., Restrict to Trial Pop.) Xolair is indicated for severe asthma poorly controlled by ICS with a reactivity to aeroallergen Annual Price: ~$40K Xolair Nucala is
indicated for patients with severe asthma and an eosinophilic phenotype Annual Price: ~$35K Nucala Cinqair is indicated for patients with severe asthma and an eosinophilic phenotype Annual Price: ~$25K Cinqair Pulmazole Pricing Potential Asthma
Biologic Price Analogues
Pulmazole: ABPA-Asthma Peak Revenue
Forecast at Loss of Exclusivity in 2033 Source: Physician Interviews; Payer Interviews; ClearView Analysis. * Also includes discount for patient compliance, patient persistence, and gross-to-net adjustment. U.S. Other Indications U.S. ABPA Asthma
E.U. ABPA Asthma US Patients at Peak Use (2033) 260 - 315 K ABPA patients 170 - 200 K diagnosed and treated 120 - 140 K inhaled antifungal eligible Pulmazole Penetration (2033) 80 - 95 K treated with Pulmazole (~67% of
eligible patients) Pricing Potential & Market Access (2033) 2033 Pricing: ~$55 K per year ($40 K at launch) 80% market access Peak US Net Sales* $1.3 Billion Pulmazole U.S. 2033 Forecast & Assumptions Pulmazole U.S. & E.U. 2033
Pulmazole Profile Meets Need for a
More Effective / Well Tolerated Antifungal and Drives Value Source: Physician Interviews; Payer Interviews; ClearView Analysis Pulmazole Clinicians told Pulmatrix they need safe and effective treatments that reduce the burden of exacerbations and
steroid-use High Unmet Need Prescribers indicate Pulmazole would penetrate 67% of addressable ABPA-Asthma market Physician Demand Clinical trial landscape survey identified limited competition (Pulmocide) for Pulmazole in ABPA-Asthma Limited
Competition Significant opportunity to expand Pulmazole to other indications beyond ABPA-Asthma Meaningful Upside Value Payers indicated favorable market access with minimal use restrictions at respiratory biologic pricing Potential for Favorable
PUR1800 - Narrow Spectrum