Aquestive Therapeutics, Inc. · NASDAQ:AQST
Aquestive Therapeutics — Investor Presentation (September 2024)
49 slides from Aquestive Therapeutics's investor presentation, Sep 27, 2024
Slide 1: Corporate Presentation September 2024
Advancing medicines. Solving problems. Improving lives. 1 Advancing medicines. Solving problems. Improving lives. Corporate Presentation September 2024
Slide 2: Disclaimer
Disclaimer © 2024 Aquestive Therapeutics, Inc. This presentation and the accompanying oral commentary have been prepared by Aquestive Therapeutics, Inc. (“Aquestive”, the “Company”, “our” or “us”) and contains forward-looking statements within the meaning of the Private Securities Litigation Reform
Slide 3: Who we are
Advancing medicines. Solving problems. Improving lives. Advancing medicines. Solving problems. Improving lives. A publicly traded pharmaceutical company (NASDAQ: AQST) focused on advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologi
Slide 4: Drug delivery technologies
Advancing medicines. Solving problems. Improving lives. 4 Drug delivery technologies AdrenaverseTM Prodrug Platform Adrenaverse platform contains a library of over 20 epinephrine prodrugs that demonstrate control of absorption and conversion rates across a variety of dosage forms and delivery sites,
Slide 5: Go-to formulation and manufacturing partner for oral thin film products
Aquestive is the go-to formulation development and commercial manufacturing partner for oral thin film products worldwide Validation from 5 proprietary and licensed commercial products, supplying over 95% of the world’s prescription oral thin films 5 1. Ondif collaboration with Hypera-Pharma (Brazil
Slide 6: Diversified pipeline
Diversified pipeline 6 1. Annual revenue includes revenue for patients 12 and up after launch in 2027. 2. Aquestive Therapeutics data on file.
Slide 7: Growth plan
Advancing medicines. Solving problems. Improving lives. Growth plan 7 1. Assumes satisfaction of all predetermined clinical endpoints and approved by U.S. Food and Drug Administration (FDA). 2. Estimate is based on an orphan drug market exclusivity block until January of 2027 by an FDA approved nasa
Slide 8: Our end-to-end capabilities
● Formulation & analytical chemistry (CMC) leaders ● Regulatory experts with 6 FDA approvals ● Clinical trial design and execution ● Intellectual property know-how with 150+ patents worldwide Our end-to-end capabilities Development Production Commercialization ● Leading manufacturer of oral thin fil
Slide 9: Financial snapshot
Financial snapshot (25.0) (20.0) (15.0) (10.0) (5.0) 0.0 5.0 2019 2020 2021 2022 2023 U S D m il li o n s EBITDA excluding R&D by Year - 5.0 10.0 15.0 20.0 25.0 2019 2020 2021 2022 2023 U S D m il li o n s R&D Investment by Year - 10.0 20.0 30.0 40.0 50.0 60.0 2019 2020 2021 2022 2023 U S D m il li
Slide 10: Dedicated and experienced leadership team
Dedicated and experienced leadership team Daniel Barber President, CEO & Director Lori J. Braender Chief Legal Officer, Chief Compliance Officer, Corporate Secretary Ernie Toth Chief Financial Officer Peter Boyd SVP, HR & IT Mark Schobel Chief Innovation & Technology Officer Cassie Jung Chief Operat
Slide 11: Company key facts
6 of revenue in 2023 $50M+ 150+ employees based in Indiana and New Jersey More than 2 billion PharmFilm® doses shipped worldwide 19+ years since the company was founded Products are available on 6 continents Over $1.5 billion1 in potential peak annual net sales from pipeline assets (NASDAQ: AQST) 2
Slide 12: Anaphylaxis and Unmet Needs
Anaphylaxis and Unmet Needs 12
Slide 13: Anaphylaxis: a potentially fatal allergic reaction
Advancing medicines. Solving problems. Improving lives. Anaphylaxis: a potentially fatal allergic reaction1 1. Turner PJ, et al. World Allergy Org J. 2019;12100066. Severe systemic hypersensitivity allergic reaction that is rapid in onset and can cause death Poses serious consequences for at-risk pa
Slide 14: Stages of anaphylaxis: early intervention is critical
Stages of anaphylaxis: early intervention is critical 1 1. Dribin et al., J Allergy Clin Immunol, 2021; Xu et al., Allergy Asthma Clin Immunol. 2014. Skin Skin 14 Gastrointestinal Skin Gastrointestinal Respiratory Cardiovascular Skin Gastrointestinal Respiratory Cardiovascular Mild Moderate Severe L
Slide 15: What is happening in the allergy rescue space
Advancing medicines. Solving problems. Improving lives. 15 What is happening in the allergy rescue space • Epinephrine, the only medication proven to stop a life-threatening allergic reaction, is the first-line treatment for anaphylaxis • No oral products are available • By nature, EMDs would be put
Slide 16: Several factors influence epinephrine administration during anaphylaxis
Several factors influence epinephrine administration during anaphylaxis 1. Nature Reviews Disease Primers on Allergic Rhinitis (2020). 2. Decker et. al. J All Clin Imm (2008). 3. Palmer et. al. All Asthma Proc (2019). 4. Warren et. al. Ann All Asthma Imm (2018). 5. Brooks et. al. Ann All Asthma Imm
Slide 17: U.S. market has the potential to grow to ~$2B by 2031
Advancing medicines. Solving problems. Improving lives. 17 U.S. market has the potential to grow to ~$2B in value by 20311 1. Aquestive Therapeutics data on file, scripts written for epinephrine autoInjectors have increased at a 15% compound annual growth rate (CAGR) from 2021- 2023. 2. https://food
Slide 18: Lead Asset Anaphylm (epinephrine) Sublingual Film
Lead Asset AnaphylmTM (epinephrine) Sublingual Film 18
Slide 19: Anaphylm executive summary
Large Market Opportunity Novel Oral Product Path to Launch • ~$2B anaphylaxis market in value by 2031 with high unmet meet1 • First and only oral epinephrine product candidate in development for anaphylaxis, with patent protection potentially into 2044 • World leader in oral thin film delivery, with
Slide 20: Anaphylm (epinephrine) Sublingual Film
+ + Easy To Carry Easy To Administer Works Quickly1 First and only non-device based, orally delivered epinephrine product candidate 1. Aquestive Therapeutics data on file. 20 Anaphylm (epinephrine) Sublingual Film
Slide 21: Most common reasons people don't carry their epinephrine devices
Advancing medicines. Solving problems. Improving lives. Most common reasons that people don’t carry their epinephrine medical devices (EMDs)1 • Inconvenience • Forgetfulness • Cost • Availability at other places, such as the home, car or school • Expiration of the previous prescription • Complacency
Slide 22: Incorporating Anaphylm into patients' daily lifestyle routine
Incorporating Anaphylm into patients’ daily lifestyle routine 1. https://www.reviews.org/mobile/cell-phone-addiction; July 2023. 22 75% of Americans feel uneasy leaving their phone at home1 Anaphlym, if approved by the FDA, has the potential to be carried on the back of a phone. 71% experience stres
Slide 23: High epinephrine prescribing physicians have spoken
High epinephrine prescribing physicians have spoken1 1. Aquestive Therapeutics 2024 Survey data on file. 23 expressed concern that their at-risk patients don’t consistently have an epinephrine auto injector (EAI) with them when away from home~90% articulated that “A sublingual film is more likely to
Slide 24: Patients and caregivers have spoken
Patients and caregivers have spoken1 1. Aquestive Therapeutics 2024 Survey data on file. 24
Slide 25: Intellectual Property
<% Aquestive’ Intellectual Property
Slide 26: Anaphylm patent portfolio
ANAPHYLM Patent Title Status ENHANCED DELIVERY EPINEPHRINE COMPOSITIONS ► 2 US patents granted ► 2 US applications ► 3 Foreign patents ► 8 Foreign applications ► Priority date: May 5, 2016 ► Possible patent term to 2037 ENHANCED DELIVERY EPINEPHRINE AND PRODRUG COMPOSITIONS ► 2 US applications ► 8 F
Slide 27: Anaphylm Clinical Program
Anaphylm Clinical Program 27
Slide 28: Anaphylm program overview
Anaphylm program overview Pivotal Study meets predetermined endpoints for single dose and repeat dose Temperature/pH Study and Self-Administration Study meet predetermined endpoints; Oral Allergy Syndrome Study underway Continue to anticipate a pre-NDA meeting with FDA prior to filing 28
Slide 29: Anaphylm is fast-acting and well-tolerated
Anaphylm is fast-acting and well-tolerated, with a safety profile comparable to standard of care (SOC)1 1 2 3 Rapid absorption as demonstrated by: Consistent pharmacokinetics (PK) demonstrated across 5 administration procedures: Consistent time to peak drug concentration (Tmax) of 12-15 minutes Onse
Slide 30: Anaphylm Pivotal Study Results
Anaphylm Pivotal Study Results 30
Slide 31: 12mg single dose study meets primary endpoints of Cmax
12mg single dose study meets primary endpoints of Cmax, demonstrating biocomparability to current SOC1 Primary endpoints predefined as Anaphylm values bracketed between injectable products for (1) maximum drug concentration (Cmax) and (2) area under the curve (AUC)0-10min, AUC0-20min, AUC0-30min, AU
Slide 32: Primary endpoint of pAUC met
Primary predetermined endpoint of pAUC, demonstrating biocomparability to SOC¹ to the current SOC is met1Geometric Mean Epinephrine Exposure Levels (pAUC) by Product After a Single Dose 5 10 15 20 40 45 50 55 60 0 50 100 150 200 250 300 25 30 35 Time Post-Dose, min P a rt ia l A U C ( h r* p g /m l)
Slide 33: Anaphylm demonstrated a rapid and robust PK profile
Anaphylm demonstrated a rapid and robust PK profile1 Geometric Mean Epinephrine Concentrations by Product After a Single Dose Anaphylm’s epinephrine concentration: ● Exceeds Adrenalin beginning at 2 minutes ● Matches EAI’s by 10 minutes ● Sustains levels above Adrenalin intramuscular out to 35 minut
Slide 34: Time to maximum concentration more consistent
Time to maximum concentration of Anaphylm demonstrates more consistency1 Time Post-Dosing to Maximum Concentration of Epinephrine ● Tmax is a surrogate for speed of absorption, a critical factor in treating Anaphylaxis ● Tmax consistency is an important measure of clinical performance ● Anaphylm Tma
Slide 35: Anaphylm demonstrates rapid pharmacodynamic effects
Anaphylm demonstrates rapid pharmacodynamic (PD) effects1 Manual IMAnaphylmEpiPenAuvi-Q Median Change in Systolic Blood Pressure Over Time -5 0 5 10 15 20 S B P ( m m H g ) 0 30 60 90 120 150 180 210 240 Time (minutes) Median Change in Diastolic Blood Pressure Over Time 0 30 60 90 120 150 180 210 24
Slide 36: Supportive Studies and Clinical Timeline
Supportive Studies and Clinical Timeline 36
Slide 37: Anaphylm temperature/pH study results
Anaphylm temperature/pH study results1 1. Aquestive Therapeutics data on file. 37 Test Condition Cmax (Test Condition/Room Temperature Water) AUC0-60min (Test Condition/Room Temperature Water) Cold water 106% 98% Hot water 104% 107% Lemon water (target pH: 3) 98% 99% Baking soda water (target pH:8)
Slide 38: Anaphylm temperature/pH study PD results
Anaphylm temperature/pH study PD results1 1. Aquestive Therapeutics data on file. 38 Key Takeaways: ● Topline results demonstrate no statistically significant difference in the maximum increase in systolic blood pressure due to temperature/pH conditions ● PD results for this study are in alignment w
Slide 39: Anaphylm self-administration PK study results
Anaphylm self-administration PK study results1 Key Takeaways: ● Cmax was not statistically different whether Anaphylm was self-administered or administered by an HCP ● Median Tmax was 15 minutes for Anaphylm whether self-administered or administered by an HCP ● Median Tmax for the Adrenalin intramus
Slide 40: Anaphylm self-administration study PD results
Anaphylm self-administration study PD results1 1. Aquestive Therapeutics data on file. 40 Key Takeaways: ● Topline PD results demonstrate no significant difference in the median increase in systolic blood pressure whether Anaphylm is self-administered or HCP-administered ● PD results for this study
Slide 41: Expected clinical and regulatory timeline for Anaphylm
Expected clinical and regulatory timeline for Anaphlym 41 Q2 2024 Q3 2024 Q4 2024 Q1 2025 Temperature/pH Study Completed Oral Allergy Syndrome (OAS) Study Self-Administration Study Completed Pre-NDA Meeting File NDA Pediatric Study
Slide 42: Pipeline Products
<% Aquestive’ Pipeline Products
Slide 43: Expected full launch path for Libervant Buccal Film
Expected full launch path for Libervant (diazepam) Buccal Film Libervant for patients ages six and up • Currently anticipate receiving full FDA approval in January 2027 • Plan to launch for ages six to eleven years, if approved by FDA PDUFA Date • December 23, 2021 Tentative FDA approval received fo
Slide 44: AQST-108 (epinephrine) Topical Gel
AQST-108 (epinephrine) Topical Gel 44 Human Skin Structure • The utility of exogeneous epinephrine for the treatment of medical conditions has been limited due to the molecule’s five- minute half-life as well as poor absorption capabilities¹ • Aquestive’s Adrenaverse technology unlocks the potential
Slide 45: AQST-108 planned Phase 2 Alopecia areata clinical study
45 Phase 2a • N=48, 3 doses and placebo • 12 weeks • Trichoscopy evaluations and labs at baseline, 2, 4, 6, 8 & 12 weeks Objectives of Phase 2a Study Assess the safety and efficacy of AQST-108 in Alopecia Areata patients following 12 weeks of treatment as determined by digital imagery (Canfield) Obj
Slide 46: Planned AQST-108 clinical and regulatory pathway
Planned AQST-108 clinical and regulatory pathway¹ 46 Q4 2024 Q1 2025 Q2 2025 Q3 2025 Open IND Pre-IND Meeting Phase 2A Phase 2B 1. End of phase 2 meeting with the FDA is planned for the fourth quarter of 2025 or the first quarter of 2026. Request Pre-IND Meeting
Slide 47: Financial Guidance
<% Aquestive’ Financial Guidance
Slide 48: 2024 Outlook
2024 expected outlook as of August 6, 2024 Total revenues of approximately $57 to $60 million Non-GAAP adjusted EBITDA loss of approximately $20 to $23 million Cash runway extended into 2026 with completion of $77.5 million underwritten public offering with high quality institutional healthcare inve
Slide 49: Thank You
<% Aquestive’ Thank You
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