Aquestive Therapeutics, Inc. · NASDAQ:AQST
Aquestive Therapeutics — Investor Presentation (August 2023)
61 slides from Aquestive Therapeutics's investor presentation, Aug 11, 2023
Slide 1: Corporate Presentation August 2023
Advancing medicines. Solving problems. Improving lives. Advancing medicines. Solving problems. Improving lives. Corporate Presentation August 2023 1
Slide 2: Disclaimer
Advancing medicines. Solving problems. Improving lives. Disclaimer This presentation and the accompanying oral commentary has been prepared by Aquestive Therapeutics, Inc. (the “Company”, “our” or “us”) and contains forward-looking statements within the meaning of the Private Securities Litigation R
Slide 3: I. Corporate Overview
Advancing medicines. Solving problems. Improving lives. I. Corporate Overview 3
Slide 4: Our Quest
Advancing medicines. Solving problems. Improving lives. Our Quest ●Advancing medicines, solving therapeutic problems, and improving lives ●Our pipeline of product candidates aims to overcome barriers that patients face with existing treatment options and provide new paradigms for treating critical a
Slide 5: Aquestive Is a Growth Story With Multiple Assets
Advancing medicines. Solving problems. Improving lives. Aquestive Is a Growth Story With Multiple Assets 5 Revenue-Generating Base of Existing Collaborations • 5 FDA-approved products • 10+ years of product sales on 6 continents • Multiple product launches since 2022 • 150+ patents worldwide Potenti
Slide 6: We Have a Strong Vision for Building the Company
Advancing medicines. Solving problems. Improving lives. We Have a Strong Vision for Building the Company In the next five years, we aim to: • Grow the existing and ex-U.S. collaboration revenue • Secure FDA approval for Anaphylm™ in the U.S. • Launch Libervant™ in the U.S. in 2027 • Utilize our epin
Slide 7: Where You Need It, When You Need It: PharmFilm
Advancing medicines. Solving problems. Improving lives. Where You Need It, When You Need It™ 7 Our Core Technology is Branded as PharmFilm®
Slide 8: Our Future Technology Is Already In-house
Advancing medicines. Solving problems. Improving lives. 8 And Our Future Technology Is Already In-house AQST-108 Anaphylm (AQST-109)
Slide 9: Product Portfolio – Significant Licensing Opportunities in 2023
Advancing medicines. Solving problems. Improving lives. Product Portfolio – Significant Licensing Opportunities in 2023 AQST-108 formulation (epinephrine) (Alternative Indications) Proprietary growth drivers Preclinical Clinical Filed Marketed 9 Hypera – Brazil Haisco - China Libervant™ (diazepam) B
Slide 10: Potential for Two Transformative Launches
Advancing medicines. Solving problems. Improving lives. Potential for Two Transformative Launches 10 Anaphylm™ (epinephrine) Sublingual Film • Potential indication of treatment of severe allergic reactions, including anaphylaxis • Anticipate submitting New Drug Application (NDA) by the end of 2024 •
Slide 11: Strong Leadership Team
Advancing medicines. Solving problems. Improving lives. Strong Leadership Team Daniel Barber President, CEO and Director Lori J. Braender SVP, General Counsel Ken Marshall Chief Commercial Officer Mark Schobel Chief Innovation & Technology Officer Ernie Toth Chief Financial Officer 11 Cassie Jung SV
Slide 12: Executed on Key Deliverables in the Last Months
Advancing medicines. Solving problems. Improving lives. Executed on Key Deliverables in the Last 12 Months Since management change in May 2022, the team has: • Raised $47M in non-dilutive financing • Signed 3 new licensing agreements on 3 continents • Supported two new product launches of licensees
Slide 13: We Are Now Focused on the Next Chapter
Advancing medicines. Solving problems. Improving lives. We Are Now Focused on the Next Chapter Over the near term, the Company aims to: • Submit pivotal protocol for Anaphylm to the FDA for review • Continue to strengthen the balance sheet • Refinance the existing debt (anticipate standard 5 yr, 3 y
Slide 14: Potential Near-term Milestones Targeted
Advancing medicines. Solving problems. Improving lives. 14 Potential Near-term Milestones Targeted - Complete debt refinancing - Start Anaphylm pivotal study - License Libervant in China H1 2024 - Receive Anaphylm topline pivotal data - AQST-108 first-in-human data - License ex-U.S. rights to Anaphy
Slide 15: II. Anaphylm (epinephrine) Sublingual Film
Advancing medicines. Solving problems. Improving lives. II. Anaphylm™ (epinephrine) Sublingual Film 15
Slide 16: Anaphylaxis Market Overview
Advancing medicines. Solving problems. Improving lives. Anaphylaxis Market Overview 16
Slide 17: Anaphylaxis: A Serious Systemic Hypersensitivity Reaction
Advancing medicines. Solving problems. Improving lives. Anaphylaxis: A Serious Systemic Hypersensitivity Reaction That is Usually Rapid in Onset And May Be Fatal 17 1. Turner PJ, et al. World Allergy Org J. 2019;12100066. 2. FARE, 2022; https://www.foodallergy.org/resources/facts-and-statistics. 3.
Slide 18: Treatment of Anaphylaxis – Epinephrine
Advancing medicines. Solving problems. Improving lives. 18 Treatment of Anaphylaxis – Epinephrine Epinephrine is the first line of treatment for anaphylaxis • Epinephrine is the only medication proven to stop a life-threatening allergic reaction Epinephrine dosage (current medication delivery system
Slide 19: Epinephrine Market On Track for up to 5M TRx in 2023
Advancing medicines. Solving problems. Improving lives. 19 Epinephrine Market On Track for up to 5M TRx in 2023 1. Symphony Health Data April 2023. The 2022 Epinephrine market surpassed 4 million TRx and has rebounded to historical highs following a downturn due to generics and the Covid-19 pandemic
Slide 20: Generic Market With High Levels of Dissatisfaction and Unmet Need
Advancing medicines. Solving problems. Improving lives. 20 Generic Market With High Levels of Dissatisfaction and Unmet Need 1. KOL feedback; Aquestive Market Research. 2. Fromer L. The American Journal of Medicine (2016);129, 1244-1250. 3. Warren et al. Ann Allergy Asthma Immunol (2018). 4. Brooks
Slide 21: Recent FDA Public Document Highlighted the Barriers to Epinephrine Use
Advancing medicines. Solving problems. Improving lives. 21 Recent FDA Public Document Highlighted the Barriers to Epinephrine Use 1 1. https://www.fda.gov/media/168054/download/Slide 14.
Slide 22: Anaphylm (epinephrine) Sublingual Film
Advancing medicines. Solving problems. Improving lives. CONFIDENTIAL22 Anaphylm™ (epinephrine) Sublingual Film First and only non-device based, orally delivered epinephrine product candidate Fast absorption into the bloodstream Non-device administration Portability + +
Slide 23: PharmFilm Platform Projecting Robust Stability
Advancing medicines. Solving problems. Improving lives. 23 PharmFilm® Platform Projecting Robust Stability Chemical Stability • > 2 years room temperature • > 6 months accelerated conditions • Successful Scale Up Executed Environmental Stability • Light resistant • Water resistant • Withstands extre
Slide 24: Patents/Patent Applications Extending into 2042
Advancing medicines. Solving problems. Improving lives. 24 Patents/Patent Applications Extending into 2042 Title Patent Status ENHANCED DELIVERY EPINEPHRINE COMPOSITIONS • Granted U.S. Patent 11,191,737 (5/4/2037) • 8 Foreign applications • Priority date: May 5, 2016 • Possible patent term to 2037 E
Slide 25: Competitive Product Summary
Advancing medicines. Solving problems. Improving lives. 25 Competitive Product Summary ORAL AUTO INJECTOR INTRA NASAL Company Brand Anaphylm EpiPen/Generic Adrenaclick® Auvi-Q® Symjepi® neffy® Utuly™ N/A Administration Sublingual Auto-Injector Auto-Injector Auto- Injector Syringe Device Nasal Spray
Slide 26: Anaphylm: Product Development
Advancing medicines. Solving problems. Improving lives. Anaphylm: Product Development 26
Slide 27: Scientific Advisory Board
Advancing medicines. Solving problems. Improving lives. 27 Scientific Advisory Board David Bernstein, MD University of Cincinnati Carlos Camargo, MD Harvard Medical School David M. Fleischer, MD Children’s Hospital Colorado David Golden, MD Sinai Hospital, Baltimore Matthew Greenhawt, MD Children’s
Slide 28: Anaphylm Clinical Trials to Date
Advancing medicines. Solving problems. Improving lives. 28 Anaphylm Clinical Trials to Date Study Description Study Status N 210010 First-in-Human (FIH), Single Ascending Dose (SAD) study to evaluate safety and tolerability, as well as pharmacokinetic (PK) performance and pharmacodynamic (PD) effect
Slide 29: Agency Interactions on Anaphylm Program to Date
Advancing medicines. Solving problems. Improving lives. 29 Agency Interactions on Anaphylm Program to Date Interaction Key Takeaways Pre-IND Meeting (December 1, 2021) • 505(b)(2) NDA regulatory approval pathway acceptable (no efficacy trials required) • Bracket PK to 0.3mg IM and safety to 0.5mg IM
Slide 30: EPIPHAST I Part 3: Favorable Pharmacokinetic (PK) Per Initial Data
Advancing medicines. Solving problems. Improving lives. 30 EPIPHAST I Part 3: Favorable Pharmacokinetic (PK) Per Initial Data ● Median time to peak concentration (Tmax) of 12 minutes at target 4 minute hold time*, compared to 50 minutes for 0.3mg Intramuscular Injection (IM) ● Partial area under the
Slide 31: EPIPHAST I Part 3: Rapid Absorption With Comparable PK After Peanut Butter
Advancing medicines. Solving problems. Improving lives. 31 EPIPHAST I Part 3: Rapid Absorption With Comparable PK After Consuming Peanut Butter From Part 3 of EPIPHAST Trial ● Study results for the sublingual administration of Anaphylm sublingual film after consuming a peanut butter sandwich demonst
Slide 32: EPIPHAST II: Topline Results
Advancing medicines. Solving problems. Improving lives. 32 Treatment B as el in e A dj us te d E pi C m ax 15 (p g/ m L) 0 250 500 750 1000 1250 1500 1750 2000 IM 0.3 mg single (R1) EpiPen 0.3 mg single (R3) AQST-109 12 mg single (T1) EPIPHAST II: Topline Results Geometric means presented for Cmax.
Slide 33: EPIPHAST II: Time to Maximum Concentration (Tmax)
Advancing medicines. Solving problems. Improving lives. 33 Baseline Adjusted Epinephrine Tmax EPIPHAST II Treatment Ba se lin e Ad ju st ed E pi T m ax (m in ut es ) 0 15 30 45 60 75 90 IM 0.3 mg single (R1) EpiPen 0.3 mg single (R3) AQST-109 12 mg single (T1) EPIPHAST II: Time to Maximum Concentrat
Slide 34: AQ109106: Similar Exposure to Auto-injectors During First 10 Minutes
Advancing medicines. Solving problems. Improving lives. 34 AQ109106: Anaphylm had Similar Exposure to Auto-injectors During the First 10 Minutes Following Dosing Parameter 0.3mg Manual (N=27) Auvi-Q (N=29) Anaphylm (n=12) EpiPen (generic) (N=29) EpiPen (N=27) AUC0-10min (hr*pg/mL) 5.3 26.7 28.3 37.7
Slide 35: Repeat Dose – Minutes
Advancing medicines. Solving problems. Improving lives. 35 Repeat Dose – 25 Minutes Description 0.3mg Manual Injection Repeat Dose (10 min) Anaphylm Repeat Dose (25 min) # Subjects 23 27 Cmax (pg/mL) 755 882 AUC0-t (hr*pg/mL) 1300 776 AUC0-45 (hr*pg/mL) 181 207 Tmax (minutes) 50 33 Tmax Range (minut
Slide 36: Study AQ109103 Design and Objective
Advancing medicines. Solving problems. Improving lives. Study AQ109103 Design and Objective DESIGN • Single-center, 3-way crossover • N = 24 healthy volunteers • Treatments o T1 = Anaphylm 12mg “revised administration language” o T2 = Anaphylm 14mg “revised administration language” o T3 = Anaphylm 1
Slide 37: Study Bridges to Study 106C with Concordant Outcomes
Advancing medicines. Solving problems. Improving lives. Study 103 Bridges to Study 106C with Concordant Outcomes Using Same Administration Conditions 37 Comparison of 106C to 103 under the same administration conditions: data were within the expected variability Parameter Anaphylm 12mg Manual hold,
Slide 38: pAUC Comparison of Anaphylm 12mg and 14mg to Auto-Injectors
Advancing medicines. Solving problems. Improving lives. 38 0 50 100 150 200 250 300 350 AUC0-5min AUC0-10min AUC0-20min AUC0-30min AUC0-40min AUC0-45min AUC0-60min Ep in ep hr in e Ex po su re L ev el s (h r* pg /m L) Time Post Administration (minutes) pAUC Comparison of Anaphylm 12mg and 14mg to Co
Slide 39: Key PK Parameters Compare Favorably to Existing Treatments
Advancing medicines. Solving problems. Improving lives. 39 Key PK Parameters Compare Favorably to Existing Treatments Bars above show highest and lowest 75% quartile ranges of approved products. 1. Cross-study comparison of AQ109102 and AQ109103. Anaphylm 12mg and 14mg provide a consistently fast Tm
Slide 40: 12mg and 14mg Anaphylm Resulted in Favorable Pharmacodynamic Effects
Advancing medicines. Solving problems. Improving lives. 40 Both 12mg and 14mg Anaphylm Resulted in Clinically Favorable Pharmacodynamic (PD) Effects Anaphylm demonstrates a rapid increase in systolic blood pressure (SBP), pulse and diastolic blood pressure (DBP) within 2 minutes. Minimal impact to P
Slide 41: Anaphylm Safety and Tolerability
Advancing medicines. Solving problems. Improving lives. 41 Anaphylm Safety and Tolerability • In the clinical program to date, treatment emergent adverse events (TEAEs) were assessed by both incidence and severity. o The vast majority of reported TEAEs were mild or moderate in severity. o The majori
Slide 42: Summary and Next Steps
Advancing medicines. Solving problems. Improving lives. 42 Summary and Next Steps ● AQ109102 compared Anaphylm to multiple epinephrine auto-injectors • Confirmation of target range between existing reference listed drug (RLD) epinephrine injections ● AQ109106 focused on administration instructions •
Slide 43: Regulatory Path Potentially De-risked by Nasal Spray FDA Advisory Committee
Advancing medicines. Solving problems. Improving lives. 43 Regulatory Path Potentially De-risked by Recent Epinephrine Nasal Spray FDA Advisory Committee Meeting VOTING RESULTS 16:6 VOTING RESULTS 17:5 VOTE: Do the PK/PD results support a favorable benefit-risk assessment for ARS-1 in adults for the
Slide 44: Anaphylm 2023-2024 Critical Path
Advancing medicines. Solving problems. Improving lives. 44 Anaphylm 2023-2024 Critical Path 2023 2024 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Jan Feb Mar Apr May Jun Jul Aug Sept Oct Nov Dec Jan Feb Mar Apr May Jun Jul Aug Sept Oct Nov Dec Pilot Studies Pivotal Study Pediatric Study Pre-NDA Meeting NDA Submission
Slide 45: III. Libervant (diazepam) Buccal Film
Advancing medicines. Solving problems. Improving lives. III. Libervant™ (diazepam) Buccal Film 45
Slide 46: The Unmet Need in Refractory Seizures
Advancing medicines. Solving problems. Improving lives. 46 1. Laxer, Ketal, The consequences of Refractory Epilepsy and its treatment; Epilepsy & Behavior, Vol 37, Aug 2014, Pgs 59 –70; https://doi.org/10.1016/j.yebeh.2014.05.031, 2. Triangle Insights Group (2017). Synthesis of Epilepsy (ARS) Primar
Slide 47: Current Treatments are Either Rectal or Intra Nasal Options
Advancing medicines. Solving problems. Improving lives. 47 Current Treatments are Either Rectal or Intra Nasal Options
Slide 48: Seizure Rescue Market
Advancing medicines. Solving problems. Improving lives. 48 Seizure Rescue Market The seizure rescue market continues to grow with new products being promoted. Based upon publicly available data, Valtoco® has flat to growing market share in all age groups in which it competes. 1. Symphony Health Data
Slide 49: Seizure Rescue Market by Age
Advancing medicines. Solving problems. Improving lives. 49 Seizure Rescue Market by Age 1. Symphony Health Data April 2023. 1
Slide 50: Seizure Rescue Market Size By Age 12+ Years
Advancing medicines. Solving problems. Improving lives. 50 Seizure Rescue Market Size By Age 12+ Years The 18+ Age group has experienced rapid market size growth with the introduction of multiple nasal products. 1. Symphony Health Data April 2023. 1
Slide 51: Seizure Rescue Market Size By Age 2-11 Years
Advancing medicines. Solving problems. Improving lives. 51 Seizure Rescue Market Size By Age 2-11 Years The 2-5 year-old age group has not experienced growth while the 6-11 year-old age group has experienced modest growth with one nasal option. 1. Symphony Health Data April 2023. 1
Slide 52: Strong Patient Preference – What Patients Want
Advancing medicines. Solving problems. Improving lives. 52 Strong Patient Preference – What Patients Want % Indicating 1: Not at all Important 2 3: Somewhat Important 4 5: Highly Important Top 2 Box Ability to have the repetitive seizure medicine with me at all times 3% 7% 20% 26% 45% 71% Ability to
Slide 53: Strong Patient Preference – Willingness to Request
Advancing medicines. Solving problems. Improving lives. 53 Strong Patient Preference – Willingness to Request % Choosing Strongly Prefer Nasal Prefer Nasal No Preference Prefer Film Strongly Prefer Film Film Preference If both medicines worked just as well at stopping my repetitive seizures, I would
Slide 54: Libervant Buccal Film Path to Launch
Advancing medicines. Solving problems. Improving lives. 54 Libervant™ (diazepam) Buccal Film Path to Launch Libervant LAUNCH • Expected January 2027 PDUFA Date • December 23, 2021 Tentative Approval Received • August 30, 2022 • Continue to pursue early U.S. market access with FDA. • Submitted NDA fo
Slide 55: IV. Existing Collaborations
Advancing medicines. Solving problems. Improving lives. IV. Existing Collaborations 55
Slide 56: Services and Capabilities
Advancing medicines. Solving problems. Improving lives. Services and Capabilities Formulation Development ● Systematic approach applied to address permeation barriers ● Robust formulation design capabilities utilize quality- by- design principles to control risk and optimize performance Analytical ●
Slide 57: Product Licenses Across the Globe
Advancing medicines. Solving problems. Improving lives. 57 Product Licenses Across the Globe Indivior Assertio Hypera Haisco Pharmanovia Zambon Mitsubishi Tanabe Pharma America We currently have eight active worldwide licensing and manufacturing contracts; five more than just two years ago.
Slide 58: Existing Product Portfolio Has Generated Over $500M In Revenue
Advancing medicines. Solving problems. Improving lives. 58 Existing Product Portfolio Has Generated Over $500M In Revenue Exservan (2021 Launch) Emylif (2023 Launch) Pharmanovia (ex-US, China) TBD(US) Expected 2024 Launch Expected 2027 Launch1 Riluzole FDA Approval 2019 Diazepam FDA Approval 2022 Ha
Slide 59: Global Diversification of Suboxone
Advancing medicines. Solving problems. Improving lives. 59 Global Diversification of Suboxone 1. Aquestive Therapeutics data on file. 2. Data from Indivior Jeffries Healthcare Conference Presentation June 7, 2023. Suboxone ROW Market2 N. America Canada Europe & Middle East EU France Italy Germany De
Slide 60: The Suboxone U.S. Market Has Been Stable for Several Years
Advancing medicines. Solving problems. Improving lives. 60 The Suboxone U.S. Market Has Been Stable for Several Years ● Suboxone U.S. market TRx is growing despite lack of promotion and alternative product forms. ● Suboxone U.S. market share is on consistent trajectory. ● Suboxone U.S. has experienc
Slide 61: Thank You
Advancing medicines. Solving problems. Improving lives. Thank You 61
Aquestive Therapeutics — Investor Presentation (August 2023) on 6ix