Aquestive Therapeutics, Inc. · NASDAQ:AQST
Aquestive Therapeutics — Investor Presentation (June 2023)
65 slides from Aquestive Therapeutics's investor presentation, Jun 29, 2023
Slide 1: Corporate Presentation June 2023
Advancing medicines. Solving problems. Improving lives. Corporate Presentation June 2023 1
Slide 2: Disclaimer
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 Reform Act of 1995. Words such as “believe,” “anticipate,
Slide 3: Corporate Overview
<< Aquestive’ Il. Corporate Overview Advancing medicines. Solving problems. Improving lives.
Slide 4: Our Quest
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 and complex conditions 4
Slide 5: Aquestive Is a Growth Story With Multiple Assets
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 Potential for 2 Commercialization Events Prior to 2027 Lead pipeline pr
Slide 6: We Have a Strong Vision for Building the Company
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 epinephrine prodrug platform for future product launches after Anaphyl
Slide 7: Where You Need It, When You Need It
Where You Need It, When You Need It™ 7 Our Core Technology is Branded as PharmFilm®
Slide 8: And Our Future Technology Is Already In-house
8 And Our Future Technology Is Already In-house AQST-108 Anaphylm (AQST-109)
Slide 9: Product Portfolio – Significant Licensing Opportunities
Product Portfolio – Significant Licensing Opportunities in 2023 AQST-108 formulation (epinephrine) (Alternative Indications) Indivior license Zambon EU license / Mitsubishi Tanabe US license Sunovion license Hypera Brazil license Proprietary growth drivers Preclinical Clinical Filed Marketed 9 Hyper
Slide 10: Potential for Two Transformative Launches
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 Estimated Total Addressable Market of ~ $1B 1. Estimated tota
Slide 11: Strong Leadership Team
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 SVP, Operations Steve Wargacki SVP, R&D Gary Slatko Chief
Slide 12: Executed on Key Deliverables in the Last Months
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 Received FDA tentative approval for Libervant Successfully clo
Slide 13: We Are Now Focused on the Next Chapter
We Are Now Focused on the Next Chapter Over the next 90 days, the Company aims to: Continue to strengthen the balance sheet Refinance the existing debt (anticipate standard 5 yr, 3 yr i/o deal) Out-license Libervant in China Submit pivotal protocol for Anaphylm to the FDA for review 13
Slide 14: Potential Near-term Milestones Targeted
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 Anaphylm H2 2023 Anaphylm pre-NDA meeting Anaphylm NDA submission Ex-U.S.
Slide 15: Anaphylm (epinephrine) Sublingual Film
Anaphylm™ (epinephrine) Sublingual Film 15
Slide 16: Anaphylaxis Market Overview
Anaphylaxis Market Overview 16
Slide 17: Anaphylaxis: A Serious Systemic Hypersensitivity Reaction
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. Fromer L. The American Journal of Medicine (2016);129, 1
Slide 18: Treatment of Anaphylaxis – Epinephrine
Advancing medicines. Solving problems. Improving lives. 18 Treatment of Anaphylaxis – Epinephrine 0.10 mg (for children 16.5 to 33 pounds) — AUVI-Q® brand only 0.15 mg (for children under 66 pounds) 0.3 mg (for children and adults over 66 pounds) 1 Epinephrine is the first line of treatment for anap
Slide 19: Epinephrine Market
Epinephrine Market 4M TRx in 2022 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. TRx counts have exceeded prior year for 9 consecutive months. 1
Slide 20: Generic Market With High Levels of Dissatisfaction and Unmet Need
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 et al. Ann Allergy Asthma Immunol (2017). 5. Asthma and All
Slide 21: Recent FDA Public Document Highlighted Barriers to Epinephrine Use
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
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
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 extreme cold conditions High temperature excursions while maintaining shelf-
Slide 24: Patents/Patent Applications Extending into 2042
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 ENHANCED DELIVERY EPINEPHRINE AND PRODRUG COMPOSITIONS 2 U.S. applic
Slide 25: Competitive Product Summary
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 Nasal Spray Nasal Spray Dosing (Adult/Jr) TBD 0.3 / 0.15 mg
Slide 26: Anaphylm: Product Development
Anaphylm:Product Development 26
Slide 27: Scientific Advisory Board
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 Hospital Colorado Ruchi Gupta, MD, MPH Northwestern Jay Lie
Slide 28: Anaphylm Clinical Trials to Date
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, of DESF (Anaphylm) Complete 44 EPIPHAST Part 1 Evaluate m
Slide 29: Agency Interactions on Anaphylm Program to Date
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 Evaluate potential for extrinsic factors to impact DESF (ANAPH
Slide 30: Anaphylm Timeline To Date
Anaphylm Timeline To Date 2020 2020 2021 2023 2022 Q4 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Oct Nov Dec Jan Feb Mar Apr May Jun Jul Aug Sept Oct Nov Dec Jan Feb Mar Apr May Jun Jul Aug Sept Oct Nov Dec EpiPhast II First In Human (FIH) Transfer, Scale Up and Registration Mfg. Formulation EpiPhast EOP2 Meeting 1
Slide 31: EPIPHAST I Part 3: Favorable Pharmacokinetic Per Initial Data
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 curve (AUC) within clinically relevant periods of 10, 20 & 30
Slide 32: EPIPHAST I Part 3: Comparable PK After Consuming Peanut Butter
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 demonstrate consistent performance Consistent Tmax of 12 minutes Com
Slide 33: EPIPHAST II: Topline Results
EPIPHAST II: Topline Results Geometric means presented for Cmax. Anaphylm Cmax values within the timeframe critical to abate the cascade of anaphylaxis is comparable to and well bracketed by the 0.3mg IM and the EpiPen® 90th percentile 10th percentile
Slide 34: EPIPHAST II: Time to Maximum Concentration (Tmax)
EPIPHAST II: Time to Maximum Concentration (Tmax) Anaphylm showed a shorter median Tmax than both 0.3mg IM and the 0.3mg EpiPen Range of Tmax values across the study is consistent with EpiPen Both EpiPen and Anaphylm provide faster Tmax values than the 0.3mg IM Fastest Median Tmax
Slide 35: Similar Exposure to Auto-injectors During First 10 Minutes
Anaphylm: 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 43.7 1. Cross-study comparison from AQ109102 and AQ109106. Comparison o
Slide 36: Anaphylm Data Brackets Existing Products
Anaphylm Data Brackets Existing Products to 45 Minutes FDA recommended bracketing between the exposures produced by auto-injectors and manual injection across a range of relevant time points characterized as pAUC. Manual Injection Level and Below Auto-injector Levels Anaphylm Critical early phase si
Slide 37: Key PK Parameters Compare Favorably to Existing Treatments
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 AQ109106. Anaphylm 12mg provides a consistently fast Tmax with median and mean Cmax levels bracketed by the current FDA ap
Slide 38: Clinically Favorable PD from Anaphylm
Clinically Favorable PD from Anaphylm Anaphylm demonstrates a rapid increase in systolic blood pressure (SBP), pulse and diastolic blood pressure (DBP) within 2 minutes. Injected epinephrine produces moderate increases in SBP and pulse with no measurable effect on DBP. Mean Baseline Adjusted Changes
Slide 39: Repeat Dose
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 (minutes) 30 - 70 10 - 70 Geometric Means presented for Cmax, AUC
Slide 40: Summary and Next Steps
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 Confirmation of Anaphylm Cmax comparability Confirmation that Anap
Slide 41: Regulatory Path Potentially De-risked by Nasal Spray Advisory Committee
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 emergency treatment of allergic reactions (Type 1) and ana
Slide 42: Anaphylm 2023-2024 Critical Path
Anaphylm 2023-2024 Critical Path 2023 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 43: Libervant (diazepam) Buccal Film
Libervant™ (diazepam) Buccal Film 43
Slide 44: The Unmet Need in Refractory Seizures
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) Primary Research. Internal Aquestive report: unpublished, 3. Epileps
Slide 45: Current Treatments are Either Rectal or Intra Nasal Options
Current Treatments are Either Rectal or Intra Nasal Options
Slide 46: Seizure Rescue Market
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 April 2023. 1
Slide 47: Seizure Rescue Market by Age
Seizure Rescue Market by Age 1. Symphony Health Data April 2023. 1
Slide 48: Seizure Rescue Market Size By Age 12+ Years
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 49: Seizure Rescue Market Size By Age 2-11 Years
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 50: Strong Patient Preference – What Patients Want
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 take the medicine as quickly as I possibly can when I need
Slide 51: Strong Patient Preference – Willingness to Request
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 prefer my doctor prescribe me: 6% 7% 16% 21% 50% 71% Likel
Slide 52: Libervant Buccal Film Path to Launch
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. 1. Estimate is based on an orphan drug market exclusivity block until January of 2027
Slide 53: Existing Collaborations
IV. Existing Collaborations 53
Slide 54: Services and Capabilities
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 Systematic approach utilized to characterize complex formulati
Slide 55: Product Licenses Across the Globe
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 56: Existing Product Portfolio Has Generated Over $500M In Revenue
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 Haisco (China) Zambon (EU) TBD (China) Clobazam FDA Approval
Slide 57: Global Diversification of Suboxone
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 Denmark, Norway Sweden Finland Switzerland UK Israel Australa
Slide 58: The Suboxone U.S. Market Has Been Stable for Several Years
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 experienced price stability for several years. 1. Elsevier Gold Standard P
Slide 59: Appendix
59 Appendix << Aquestive’ Advancing medicines. Solving problems. Improving lives.
Slide 60: Partial AUC0-10min by Study
Partial AUC0-10min by Study Geometric Means presented 60
Slide 61: Recent Single Administration Studies of AQST-109 12mg
Recent Single Administration Studies of AQST-109 12mg Study # # Subjects Swallow Hold Time Dosing Instructions pAUC0-10min Endpoint > Manual IM pAUC0-20min Endpoint > Manual IM pAUC0-30min Endpoint > Manual IM pAUC0-45min Endpoint > Manual IM GM Cmax > Manual IM AQST109102 29 None Anaphylm was appli
Slide 62: Pharmacokinetic Summary Results: Impact of Food Residue
Pharmacokinetic Summary Results: Impact of Food Residue (Reference Slide #32) Geometric Means presented for Cmax and AUC, Median presented for Tmax Study Results AQST-109 12mg 4-minute hold time (N=22 doses) AQST-109 12mg PB sandwich (N=20 doses) Epinephrine IM injection 0.3mg (Part 2) (N=48 doses)
Slide 63: EPIPHAST II: Comparative Changes in Systolic Blood Pressure and Cardiovascular AEs
EPIPHAST II: Comparative Changes in Systolic Blood Pressure and Cardiovascular Adverse Event Profiles EpiPen (N=22) Manual Injection (N=23) Anaphylm (N=23) SBP>30mm Hg 9% 0% 13% SBP>40mm Hg 0% 0% 9% EpiPen (N=22) Manual Injection (N=23) Anaphylm (N=23) Cardiac Disorders 7 (31.8) 1 (4.3) 2 (8.7) Palp
Slide 64: Comparative Changes in Systolic Blood Pressure and Cardiovascular AEs
Study 102 & 106: Comparative Changes in Systolic Blood Pressure and Cardiovascular Adverse Event Profiles EpiPen (102, N=27) EpiPen Generic (102, N=29) Auvi-Q (102, N=29) Manual Injection (102, N=27) Anaphylm (106, N=12) SBP>30mm Hg 7% 7% 7% 7% 42% SBP>40mm Hg 4% 3% 0% 4% 8% EpiPen (102, N=27) EpiPe
Slide 65: Thank You
1 Thank You << Aquestive’ Advancing medicines. Solving problems. Improving lives.
Aquestive Therapeutics — Investor Presentation (June 2023) on 6ix