Full Press Release Details
Anaphylaxis and EpinephrineR&D Day March 25, 2021
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forward-looking statements are based on our current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements.
Such risks and uncertainties include, but are not limited to, risks associated with the Company's development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and
plans for AQST-108, AQST-109 and our other drug candidates; risk of delays in FDA approval of our drug candidates, Libervant and AQST-108, AQST-109, and our other drug candidates or failure to receive approval; ability to address the concerns
identified in the FDA's Complete Response Letter dated September 25, 2020 regarding the New Drug Application for Libervant; risk of our ability to demonstrate to the FDA "clinical superiority" within the meaning of the FDA regulations of
Libervant relative to FDA-approved diazepam rectal gel and nasal spray products including by establishing a major contribution to patient care within the meaning of FDA regulations relative to the approved products as well as risks related to
other potential pathways or positions which are or may in the future be advanced to the FDA to overcome the seven year orphan drug exclusivity granted by the FDA for the approved nasal spray product of a competitor in the U.S. and there can be
no assurance that we will be successful; risk that a competitor obtains FDA orphan drug exclusivity for a product with the same active moiety as any of our other drug products for which we are seeking FDA approval and that such earlier approved
competitor orphan drug blocks such other product candidates in the U.S. for seven years for the same indication; risk inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks
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the amounts needed; risk of failure to satisfy all financial and other debt covenants and of any default; our and our competitors' orphan drug approval and resulting drug exclusivity for our products or products of our competitors; short-term
and long-term liquidity and cash requirements, cash funding and cash burn; risk related to government claims against Indivior for which we license, manufacture and sell Suboxone and which accounts for the substantial part of our current
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actions affecting pharmaceutical product pricing, reimbursement or access; claims and risks that may arise regarding the safety or efficacy of the Company's products and product candidates; risk of loss of significant customers; risks related
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business, industry, regulatory and market conditions and other unusual items; and other uncertainties affecting the Company described in the "Risk Factors" section and in other sections included in our Annual Report on Form 10 K, in our
Quarterly Reports on Form 10-Q, and in our Current Reports on Form 8-K filed with the Securities Exchange Commission (SEC). Given those uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as
of the date made. All subsequent forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. The Company assumes no obligation to update
forward-looking statements or outlook or guidance after the date of this press release whether as a result of new information, future events or otherwise, except as may be required by applicable law.PharmFilm and the Aquestive logo are
registered trademarks of Aquestive Therapeutics, Inc. All other registered trademarks referenced herein are the property of their respective owners.This presentation shall not constitute an offer to sell or the solicitation of an offer to buy
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Today's Agenda Topic Presenter(s) Introductions Keith Kendall, CEODan Barber, COO Medical Overview
of Anaphylaxis and Epinephrine David M. Fleischer, MD, FAAAAIProfessor of Pediatrics, Section Head, Allergy and Immunology, Children's Hospital ColoradoUniversity of Colorado Denver School of Medicine Anaphylaxis Market Overview Michael
Arcara, MBA Commercial Lead, Allergy R&D Overview of AQST-108 and AQST-109 Steve Wargacki, PhDVP of R&D, Aquestive Therapeutics Clinical Lessons from Today's Products John Oppenheimer, MD Clinical Professor of Medicine at UMDNJ
Rutgers, Pulmonary and Allergy Associates NJ Clinical Overview of AQST-108 and AQST-109 Mark Lepore, MD Chief Medical Officer, Allergy, Aquestive Therapeutics Q&A Closing Remarks Keith Kendall, CEO
PharmFilm Technology - Where You Need It, When You Need It And Needle Free
David M. Fleischer, MD, FAAAAIProfessor of PediatricsSection Head, Allergy and ImmunologyChildren's
Hospital ColoradoUniversity of Colorado Denver School of Medicine Anaphylaxis and EpinephrineMedical Overview
Introduction David M. Fleischer, MD, FAAAAIProfessor of PediatricsSection Head, Allergy and
ImmunologyChildren's Hospital ColoradoUniversity of Colorado Denver School of Medicine
Defining Anaphylaxis Simons FE. J Allergy Clin Immunol. 2009;124(4):625-636. Patients
at risk for anaphylaxis should have a long-term allergy-management plan Poses serious consequences for at-risk patients Often occurs in the community setting Anaphylaxis is a severe systemic allergic reaction that is rapid in onset and can
Common Triggers of Anaphylaxis Idiopathic anaphylaxis Exercise Insect venom, medications, and
Common Signs and Symptoms of Anaphylaxis *Potentially life-threatening symptoms. Simons FE. J
Allergy Clin Immunol. 2009;124(4):625-636. Central nervous system10%-15% of episodesUneasiness, throbbing headache, dizziness, confusion, tunnel vision Airway*70% of episodesLarynx: pruritus and tightness in throat; dysphonia and
hoarsenessLung: dyspnea, chest tightness, wheezing/bronchospasm Skin 80%-90% of episodesUrticaria, pruritus, flushingMucosal tissue: pruritus and swelling of lips, tongue, uvula/palate Cardiovascular system*10%-45% of episodesChest pain,
hypotension, tachycardia, weak pulse, dizziness, fainting Gastrointestinal tract30%-45% of episodesNausea, cramping, abdominalpain, vomiting, diarrhea
Patterns of Anaphylaxis UniphasicSigns and symptoms within minutes of exposure to an offending
stimulusBiphasicBiphasic anaphylaxis has an immediate phase with a period of improvement and response to initial therapy, but with recurrent symptoms 2 to 6 hours after the onset of the initial reactionProtractedProtracted anaphylaxis causes
prolonged manifestations (usually respiratory distress or hypotensive shock) for 5 to 32 hours Golden D. Novartis Found Symp. 2004;257:101-110. Anaphylaxissigns and symptoms (minutes to hours) Signs and symptomsoccur 2 to 6 hours
after initial reaction Allergenexposure Likelihood of Occurrence Initial reaction Biphasic reaction 5 m 10 m 15 m 20 m 25 m 30 m 1 h 8 h 72h 2 h 3 h 4 h 5 h 6 h 7 h 9 h 10 h
Epinephrine is first-line treatment for anaphylaxis1Fatality due to anaphylaxis is associated with
delayed epinephrine administration2Failure to administer epinephrine in a timely manner (eg, pre-ED) has been associated with fatalities3Failure to administer epinephrine promptly is the most important factor contributing to death ED,
emergency department.1. Waserman S et al. J Allergy Clin Immunol Pract. 2017;5:1180-1191; 2. Song TT, Lieberman P. Curr Opin Allergy Clin Immunol. 2015;15(4):323-328; 3. Simons FE et al. J Allergy Clin Immunol.
1998;101:33-37. Epinephrine Allergy suspected in mother's death
Use and Carry Practices for Epinephrine Auto-Injectors Correct use of epinephrine auto-injectors (EAIs)
is surprisingly lowData indicate just 16% to 32%1Studies have shown that:Just half of at-risk patients regularly carry an unexpired EAI2Many patients did not have epinephrine available at the time of a reaction3A majority of caregivers did not
give their allergic children epinephrine at the time of their most severe allergic reactions4 1. 1. Bonds RS et al. Misuse of medical devices. Ann Allergy Asthma Immunol. 2015;114(1):74e76.e2; 2. Arkwright PD, Farragher AJ. Pediatr Allergy
Immunol. 2006;17(3):227e229; 3. Polloni L et al. Pediatr Allergy Immunol. 2020;31(4):380e387; 4. Egan M et al. J Allergy Clin Immunol Pract. 2019;7(2):655e658; 5. Warren CM et al. Ann Allergy Asthma Immunol. 2018;121(4):479e489.e2.. Images:
EpiPen Package Insert, Patient Directions for Use Most common reasons for pitfalls in the use of EAIs are:Lack of auto-injector availabilityInadequate education on how to administer the epinephrineConcern for systemic effects, failure to
administer correctly, and accidental administration5
Adverse Events Due to Incorrect Use of EAIs Publications have raised concerns over laceration and
embedded-needle injuries resulting from the use of EAIs1From 1994 to 2007, >15,000 unintentional injections from EAIs were reported to US Poison Control CentersThose unintentionally injected had a median age of 14 years2Even if EAIs are
available, safety concerns and/or fear of needles may prevent them from being administered in a timely manner1 1. Posner L, Camargo CA Jr. Drug Healthc Patient Saf. 2017;9:9-18; 2. Simons FER et al. J Allergy Clin Immunol.
2010;125(2):419-423.e4. 3. Image: Brown J et al. Lacerations and Embedded Needles Caused by EEAI Use in Children. Annals of Emergency Medicine, 2015.
EAI Refill Rates Are Suboptimal Lack of annual EAI refills can leave at-risk patients without immediate
access to epinephrine when they need itIn 1 large HMO over a 5-year period:A total of just 46% of patients refilled their EAI at least once Just 25% refilled multiple timesOnly 11% refilled each year Kaplan. Curr Allergy Asthma Rep.
Summary Anaphylaxis is an unpredictable, severe systemic allergic reaction that is rapid in onset and
potentially fatalAt-risk patients should always have immediate access to 2 doses of epinephrineFatality due to anaphylaxis is associated with delayed epinephrine administrationFar too few at-risk patients:Carry their EAI with themRefill their
EAI on an ongoing basisAre confident, timely, and accurate in their use of an EAI during an emergency situationEpinephrine products that are easier for patients to carry and administer would be an important addition to an allergist's treatment
Michael Arcara Anaphylaxis and EpinephrineMarket Overview
Introduction Michael ArcaraCommercial Lead, AllergyMBA, University of Michigan25 years experience in
pharmaceutical commercial developmentEpinephrine-specific experience: Mylan (EpiPen); Valeant (Emerade)
Epidemiology: Life-Threatening Allergic Reaction1,2 Anaphylaxis epidemiology is not well definedStudies
estimate that 1.6% to 5.1% of the US population has actually had an anaphylaxis episodeOne set of data indicates that people dying from food allergy often had previous reactions but these were typically not severe3Per CDC: the prevalence of
food allergies in children increased by 50% between 1997 and 2011 1. Woods et al. J Allergy Clin Immunol. 2014;133:461-467; 2. Food Allergy Research & Education website. https://www.foodallergy.org/resources/facts-and-statistics; 3.
Pumphrey R. Anaphylaxis: can we tell who is at risk of a fatal reaction? Curr Opin Allergy Immunol. 2004.
Overview: Epinephrine Market Prescription market was still growing and peaked just before Teva generic
entered2018-current = significantly diminished HCP/consumer promotionLarge percentage of EAI prescriptions-especially first-time prescriptions-are driven by allergists and pediatriciansTarget HCP universe ~20,000 physiciansCurrent EpiPen
2-Pak WAC: ~$300 Epinephrine: US market 2014 2015 2016 2017 2018 2019 2020 Market prescriptions, millions 3.6 3.8 3.8 3.6 3.1 3.2 3.0 Market $, billions 1.4 1.9 2.4 2.1 1.8 1.9 1.5 Source: Symphony Health PHAST
(Pharmaceutical Audit Suite)
Physician Research: Providers Indicate Preference for Alternative Route of Administration 89% at least
somewhat prefer *Aquestive sponsored online survey; N=500 allergists, pediatricians, and PCPs; fielded 2018. Data on file. To what extent would you prefer an alternative administration route for epinephrine?
How concerned are you with patients and/or their caregivers consistently having their auto-injector(s)
with them when they leave home? Physician Research: Concerns About Patients Having EAI With Them at All Times 88% at least somewhat concerned *Aquestive sponsored online survey; N=500 allergists, pediatricians, and PCPs; fielded 2018. Data
Patient Survey: Unprompted Unmet Need Top 3 Box: 74% Top 4 Box: 88% *Aquestive sponsored online,
5-minute survey; N=75 EAI patients, 75 caregivers; fielded February 2021. Data on file To what extent would you prefer a new way to dose epinephrine?
Interest in the film medicine being available to replace your EAI Patient Survey: First Exposure to
Film Dosing At Least Some Interest: 99% *Aquestive sponsored online, 5-minute survey; N=75 EAI patients, 75 caregivers; fielded February 2021. Data on file
Patient Survey: Impact, Epinephrine On Hand I believe the smaller package would make it more likely for
me to have the film with me when I need it (vs EAI): *Aquestive sponsored online, 5-minute survey; N=75 EAI patients, 75 caregivers; fielded February 2021. Data on file
Patient Survey: Ask Doctor About Film Top 3 Box: 90% Top 4 Box: 96% How likely would you be to ask
your doctor about the film? *Aquestive sponsored online, 5-minute survey; N=75 EAI patients, 75 caregivers; fielded February 2021. Data on file
PharmFilm delivers market share within existing market Aquestive allergy franchise delivers
market expansion Our View of the Epinephrine Commercial Opportunity Features/benefits customers are looking for in a new epinephrine delivery:PortabilityUsabilityNeedle freeTemperature tolerabilityWaterproof/resistant Expected
opportunities to increase the customer base:Previously reluctant (e.g., needle-phobic or size-resistant) patients more readily adopt PharmFilm dosingIncreased carry rates due to size, in turn, increase engagement with the brand and annual
refill ratesGiven current lack of promotion in the market, Aquestive launch plans to re-engage HCPs, patients, and advocacy groups Patient convenience Medical advance + *Target Product Profile
Aquestive PharmFilm - Potential Annual Peak Net Sales* No market expansionPharmFilm share
<30% $500M $250M Modest market expansionPharmFilm share = 30% - 50% Significant market expansionPharmFilm share >50% *Potential sales revenues are based on current sales information from Symphony Health PHAST (Pharmaceutical Audit
Suite) (See Slide 19) assuming peak sales at ~5 years post launch.