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PULMATRIX Corporate Overview August
2018 NASDAQ: PULM Exhibit 99.1
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FDA, or any other regulatory entity, to market our therapeutic candidates in the United States or in any other jurisdictions. Our therapeutic candidates, including Pulmazole, PUR1800, and PUR0200 are classified by the FDA as investigational drugs
and are limited by Federal (or United States) law to investigational use only that require additional studies to make definitive conclusions and claims about such candidates' safety or efficacy.
Pulmatrix: Building Value by Meeting
Important Unmet Medical Needs in ABPA and AECOPD ABPA = Allergic Bronchopulmonary Aspergillosis; CF = Cystic Fibrosis; AECOPD = Acute Exacerbations in Chronic Obstructive Pulmonary Disease LEAD PROGRAM: iSPERSE Pulmazole Anti-Fungal Pulmazole
is inhaled Itraconazole for allergic bronchopulmonary aspergillosis (ABPA) - seeking to be the first to market inhaled anti-fungal in US and EU Reduced development risk as oral itraconazole (Sporanox) has been on the market for over 25 years
Itraconazole use in ABPA, though effective, has limited utility given poor bioavailability and toxicity concerns Phase 1/1b study results demonstrated Pulmazole safety/tolerability and ability to achieve significantly higher lung exposure and lower
plasma exposure than oral Sporanox, supporting the belief that Pulmazole could significantly improve upon known efficacy and safety profile of Sporanox Qualified Infectious Disease Product (QIDP) designation obtained in 2017 for ABPA and CF Second
PROGRAM: iSPERSE PUR1800 Kinase Inhibitor PUR1800 is a novel narrow spectrum kinase inhibitor (NSKI) for COPD first in class (US/EU) inhaled non-steroidal anti-inflammatory to treat acute exacerbations in COPD (AECOPD) NSKI in PUR1800 demonstrated
anti-inflammatory activity, safety and tolerability in stable COPD patients Underlying iSPERSE Platform Technology Supports Future Value Growth iSPERSE dry powder technology significant patient experience and product opportunities iSPERSE
intellectual property carries well into 2030's on platform and specific products
iSPERSE Best-in-Class Engineered
Particles Enable Development and Patient Care Advantages Small, dense, dispersible, respirable particles Highly efficient inhaled delivery Flow rate independent performance (consistent delivery of high drug loads) Scalable platform Broad IP
portfolio into 2030s Can be used with a broad range of drugs, small molecule to biologic Can be used with any device (i.e., metered-dose, reservoir, or blister-based inhalers) Requires low inspiratory flow for penetration deep into lung based on
high dispersibility 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
iSPERSE Differentiation Potential iSPERSE Advantages iSPERSE Enables Sick Patients to Get More Effective Doses
Robust Pipeline with Projected
Significant Value Catalysts* LAMA = Long-Acting Muscarinic Antagonist; * Estimated Milestones Product Pipeline Indication 2H 2018 2019 1H 2020 Milestone Pulmazole Anti-fungal Allergic Bronchopulmonary Aspergillosis (ABPA) in Patients with Asthma
Phase 2 4Q 2019 PUR1800 NSKI Acute Exacerbations of COPD (AECOPD) Phase 2a 2Q 2020 PUR5700 NSKI Idiopathic Pulmonary Fibrosis (IPF) Pre-clinical Potential Future Revenue Opportunities PUR0200-US LAMA Chronic Obstructive Pulmonary Disease (COPD)
Out-Licensed to Vectura for U.S. 09/2017 Phase 2 Phase 2a Phase 1 28-Day Tox Pre-Clinical
Pulmazole - Inhaled Antifungal
Inhaled Itraconazole to Treat Allergic Bronchopulmonary Aspergillosis (ABPA) in Asthmatics "I am very encouraged by the phase 1 results demonstrating that it is both feasible to administer itraconazole by inhalation and further that high
levels of the drug may be achieved within the airways. I believe that Pulmazole has the potential to significantly improve upon both the efficacy and safety profile of oral Sporanox, and I look forward to working with the other members of the
advisory board to assist Pulmatrix in advancing the development of Pulmazole to treat patients with fungal asthma, focusing initially on ABPA." - David Denning, MD Professor of Infectious Diseases in Global Health and Director of the
National Aspergillosis Centre, Manchester, UK
PULM: Investment Opportunity $25M Raise
Funds Pulmatrix Through Pulmazole Ph2 Data Expected in 4Q 2019 Clinical Stage Antifungal Pulmazole is an inhaled reformulation of itraconazole in Ph2 for airway fungal infections Ph1/1b study demonstrated safety/tolerability in healthy normal
volunteers dosed up to 14 days and asthmatics following single dose Ph1/1b results support belief that Pulmazole could significantly improve upon known efficacy and safety profile of Sporanox Attractive Market Opportunity Up to $1.5B U.S. in
Asthma-ABPA and in other Aspergillus pulmonary disease (ex. Cystic Fibrosis - ABPA)* Following 505(b)(2) pathway for registration as Sporanox has been on market for 25+ years Additional 5 years exclusivity with QIDP status Clinical Data &
Upcoming Milestones Phase 2 28-day proof-of-mechanism in ABPA patients scheduled to begin 4Q 2019 (FPI 4Q 2018) Phase 2 proof-of-concept efficacy/safety trial in ABPA patients scheduled to begin 2Q 2020 Superior Technology and Team iSPERSE
best-in-class particle engineering technology for lung delivery Clinical stage COPD assets (PUR0200 and PUR1800) with substantial follow on development opportunities Experienced management team and board with success in commercializing respiratory
products ABPA = Allergic Bronchopulmonary Aspergillosis; COPD = Chronic Obstructive Pulmonary Disease; FPI = First Patient In * Peak revenues expected at loss of market exclusivity, ~11 years post launch Pulmazole
Pulmazole Has Potential to Transform
the Standard of Care for ABPA Source: Denning DW, Pleuvry A, Cole DC. Global burden of allergic bronchopulmonary aspergillosis with asthma and its complication chronic pulmonary aspergillosis in adults. Med Mycol. 2013;51(4):361-370 Ref:
Clearview Analysis.; IDSA = Infectious Diseases Society of America; SAD/MAD = Single Ascending Dose / Multiple Ascending Dose; POM = Proof of Mechanism; POC = Proof of Concept Significant Product Opportunity Estimated 300K US and 5M WW ABPA patients
No approved antifungal therapy available Limited/no novel competition anticipated for ABPA market Improving Upon Standard of Care Sporanox (oral itraconazole) use is limited due to poor bioavailability, safety/tolerability issues and drug-drug
concerns associated with high oral plasma exposure Target Product Profile (TPP) Pulmazole is anticipated to provide higher lung exposure and lower plasma exposure than Sporanox, potentially improving both efficacy and safety/tolerability Ph1/1b
Clinical Study Results Pulmazole was safe and well tolerated in SAD/MAD study in healthy normal volunteers and SD administration to asthmatics Pulmazole demonstrated significantly higher lung exposure than Sporanox in asthmatics, despite inhaling
1/10 of the dose administered orally Pulmazole plasma exposure in asthmatics and healthy normal volunteers was significantly lower than that of Sporanox Pulmazole Clinical Value Drivers Investment required to deliver Phase 2 28-day POM data expected
in 4Q 2019 Potential registration Phase 2b/3 POC trial start date 2Q 2020
ABPA Represents a Large Addressable
Antifungal Patient Population in Aspergillus Related Diseases 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;
Source: Denning DW, Pleuvry A, Cole DC. Med Mycol. 2013;51(4):361-370; Physician Interviews; ClearView Analysis.; IgE = Immunoglobulin E Antibodies; IgG = Immunoglobulin G Antibodies Persistence of A. fumigatus in the airways leads to local
inflammation, mucus production, reduction in lung function and worsening of asthma symptoms. Untreated ABPA may result in pulmonary fibrosis, respiratory failure and potentially death. Healthy Lung Diseased Lung ABPA is a Debilitating Disease ABPA
Pathophysiology ABPA in asthmatics is caused by hypersensitivity to Aspergillus species lung infection ~300K adult asthma ABPA patients in U.S. and ~5M worldwide Diagnostic criteria include: High IgE (>1000 IU/mL) Skin prick positivity to
Aspergillus Eosinophilia (>500 cells/ L) Elevated IgE/IgG Antibodies to Aspergillus Radiographic pulmonary opacities consistent with ABPA
ABPA Progression and Treatment
Options ABPA is Treated by Allergists and Pulmonologists Greenberger P., B.R., Demain J, et. al., Allergic Bronchopulmonary Aspergillosis. J Allergy Clin Immunol Pract., 2015. 2(6): p. 703-708.; Physician Research: Clearview Analysis ABPA-Asthma
Treatment Goals Treatment is Limited Primarily to Steroids and Antifungal Therapy Limited Treatment Options Impede Ability to Achieve Treatment Goals Symptom control of asthma or cystic fibrosis Prevent or treat pulmonary exacerbations Reduce or
remit pulmonary inflammation Mitigate progression to end-stage fibrotic or cavitary disease Oral steroid therapy is the standard of care for treating ABPA In patients with insufficient treatment response, antifungal therapy is added to steroid
therapy for ~4 months Antifungals are sometimes used 1st line in conjunction with steroids ~50% of ABPA patients have inadequate response to oral steroids alone ~20% of ABPA patients become steroid dependent Antifungal agents are believed to reduce
fungal burden (antigen induces inflammatory response) Antifungal treatment improves clinical outcomes and can enable a reduction in steroid burden While the majority of antifungal use in ABPA is itraconazole, overall antifungal use is limited by
safety/tolerability concerns
Literature Establishes Strong
Clinical Precedent for Itraconazole Use in ABPA Source: Denning. Chest.1991; 100:813; 116(6):1665; Stevens. NEJM.2000;342(11):757; Wark. J Allergy Clin Immuno. 2003;111; 952; Agarwal Chest 2018; doi: 10.1016/j.chest.2018.01.005; Ram. Journal of
Asthma. 2016:517; Physician Interviews; ClearView Analysis.; FEV1 = Forced Expiratory Volume in 1 Second Pulmazole is Anticipated to Improve Upon Sporanox Outcomes (FEV1, Exacerbation Rate, Asthma Control and Steroid Burden) Demonstrated in Clinical
Literature Literature Indicates Antifungals Improve ABPA Outcomes 1991, Denning et al. Itraconazole improved pulmonary function, decreased corticosteroid use and total IgE in 6 patients with ABPA (3 cystic fibrosis and 3 asthma) 2018, Agarwal et al.
In acute stage treatment naive ABPA patients, monotherapy itraconazole is effective in considerable number with less side-effects compared to prednisolone 2016, Ram et al. Nebulized amphotericin B reduced exacerbations compared to nebulized
budesonide in a study in 21 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
Randomized Placebo Controlled
Studies Support Efficacy of Oral Sporanox in Asthma-ABPA NEJM 2000; 342:756-62.; J Allergy Clin Immunol 2003; 111: 952-57; BID = 2x/Day; FVC = Forced Vital Capacity; PEF = Peak Expiratory Flow; PBO = Placebo Stevens & Wark Studies - Key
Takeaways Both clinical trials support inclusion of Sporanox into current ABPA treatment guidelines Key biomarkers of ABPA activity can be reduced as early as 4 weeks of treatment Both studies show improvements in FEV1 at 16 weeks, a potentially
approvable endpoint Both studies are the foundation for Pulmazole Phase 2 POM and Phase 2b/3 POC development plan 2003, Wark et al. Journal of Allergy & Clinical Immunology 2000, Stevens et al. New England Journal of Medicine Design: Part 1: 200
mg Sporanox BID vs PBO for 16 weeks (n=55) Part 2: open label, all patients received 200 mg Sporanox QD (n=50) Results: 46% response rate in Sporanox group vs 19% response rate in placebo group (p=0.04) Greater improvements in lung function (FEV1,
FVC, PEF) noted in Sporanox treatment group versus placebo Design: 200 mg oral Sporanox bid vs PBO for 16 weeks (n=29) Results: Sputum eosinophils (35% decrease/week first 4 weeks) and serum IgE (6% decrease/week) significantly reduced Clinically
significant improvement in FEV1 following 16 weeks of treatment (9.8% treatment difference between groups (p=0.5, likely due to small sample size) Fewer exacerbations requiring oral corticosteroids in those treated with Sporanox (p=0.03)
Pulmazole Potentially Addresses the
Significant Limitations of Oral Antifungals in ABPA Denning. Clinical and Experimental Dermatology. 2001;26:648; Source: Wang. AMAC. 2010;54:2409. Denning. Clinical Infectious Diseases. 2002;34:563; Lestner. Clinical Infectious Diseases 2009;
49:928-30; Waljee. BMJ. 2017;357:1415; Greenberger. Allergy Asthma Proc. 2007;28(4):489. Physician Interviews; ClearView Analysis, October 2017 Gastro Intestinal Intolerance Hepatic Abnormality Cutaneous Reactions Variable and Poor PK Drug to
Drug Interactions Fatigue Antifungals reduce both exacerbations and chronic steroid use Sporanox has been on the market for 25+ years and is the preferred antifungal Oral antifungal dose dependent side-effects can be significant restricting
physician use and variability in lung concentration limit the ability to maximize therapeutic effect Physicians desire additional antifungal treatment options Inhalation of itraconazole directly to the airways should allow for higher exposure where
Aspergillus is present Pulmazole profile would lead to significant treatment adoption Visual Changes Antifungal Limitations Physician Feedback Supports Pulmazole as a Preferred Treatment
Pulmazole Has Up to $1.5B Peak
Revenue Potential in the U.S. Source: Physician Interviews; Payer Interviews; ClearView Analysis. *Also includes discount for patient compliance, patient persistence, and gross-to-net adjustment; ** Estimate based on ClearView Analysis, which
included qualitative physician surveys and interviews; *** Estimate based on ClearView Analysis, which took into account uninsured patients, patients who are unwilling to pay, and projected access restrictions placed by payers; * Peak revenues
expected at loss of market exclusivity, ~11 years post launch ~$1.3B ~$200M Payers and Physicians Interviewed See Logic in Pulmazole Value Proposition Pulmazole U.S. Peak Revenue Forecast* Payers suggested ~70% market access*** Payers indicated
~$40K/year asthma biologic price with restrictions to specialty use and label Physicians indicated 67% addressable market penetration with Pulmazole** 1st Line Antifungal Usage May More than Double U.S. Other Indications U.S. ABPA Asthma Net Revenue
% of Antifungal Use 1st Line with Steroids
Phase 1/1b Data Highlights Potential
of Pulmazole to Improve Upon Clinical Utility of Oral Sporanox Pulmazole (20 mg) Sporanox (200 mg; oral) Sporanox (200 mg; oral) Pulmazole (20 mg) Parts 1 and 2 in HNV Demonstrated safety and tolerability of Pulmazole administered up to
14 days Following inhalation of Pulmazole, total systemic exposure over 24 hours is ~100-400 fold lower than that expected with Sporanox Part 3 SD in Stable Asthmatics Demonstrated safety and tolerability of Pulmazole administered as a single dose
in asthmatics ~50 fold higher lung exposure following inhalation of Pulmazole compared to Sporanox despite inhaling only 1/10 the dose ~85 fold lower plasma exposure than oral Sporanox Pulmazole (25 mg) Pulmazole (10 mg) Pulmazole (5 mg) Pulmazole
(35 mg) Pulmazole (20 mg) Pulmazole (10 mg) Pulmazole (35 mg) Ph1/1b Study SUCCESSFULLY Met All Endpoints Part 1: Single Ascending Dose Healthy Normal Volunteers (n=6/cohort): Optional 4th cohort of 35 mg Part 2: Multiple Ascending Dose Healthy