Aquestive Therapeutics, Inc. · NASDAQ:AQST
Aquestive Therapeutics — Investor Presentation (November 2024)
57 slides from Aquestive Therapeutics's investor presentation, Nov 6, 2024
Slide 1: Corporate Presentation November 2024
Advancing medicines. Solving problems. Improving lives. September 2024 Advancing medicines. Solving problems. Improving lives. Advancing medicines. Solving problems. Improving lives. 1 Corporate Presentation November 2024
Slide 2: Forward-looking statements
© 2024 Aquestive Therapeutics, Inc. 2 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 Act of 19
Slide 3: Who we are
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 technologies 3 Who we are…
Slide 4: Drug delivery technologies
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, including allergy, topical (dermatological), and more.
Slide 5: Go-to formulation development and manufacturing partner for oral thin film
5 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 1. Libervant approved by U.S. Food and Drug Admin
Slide 6: Diversified pipeline
6 Diversified pipeline 1. Annual revenue includes revenue for patients 12 and up after launch in 2027. 2. Aquestive Therapeutics data on file.
Slide 7: Growth plan
7 Growth plan 1. Assumes satisfaction of all predetermined clinical endpoints and approved by FDA. 2. Estimate is based on an orphan drug market exclusivity block until January of 2027 by an FDA approved nasal spray product. 20261 Launch Anaphylm2024 Launch Libervant (ages 2 to 5) 2028+1 Launch AQST
Slide 8: Our end-to-end capabilities
8 ● 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 f
Slide 9: Financial snapshot
9 Financial snapshot (25.0) (20.0) (15.0) (10.0) (5.0) 0.0 5.0 2019 2020 2021 2022 2023 U SD m ill io ns EBITDA excluding R&D by Year - 5.0 10.0 15.0 20.0 25.0 2019 2020 2021 2022 2023 U SD m ill io ns R&D Investment by Year - 10.0 20.0 30.0 40.0 50.0 60.0 2019 2020 2021 2022 2023 U SD m ill io ns R
Slide 10: Dedicated and experienced leadership team
10 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 Ope
Slide 11: Company key facts
6 of revenue in 2023 $50M+ 150+ employees based in Indiana and New Jersey More than 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 Product la
Slide 12: Anaphylaxis and Unmet Needs
12 Anaphylaxis and Unmet Needs
Slide 13: Anaphylaxis: a potentially fatal allergic reaction
13 Anaphylaxis: a potentially fatal allergic reaction1 1. Turner PJ, et al. World Allergy Org J. 2019;12100066. Poses serious consequences for at-risk patients Often occurs in the community setting Patients at risk for anaphylaxis should have a long-term allergy-management plan Severe systemic hyper
Slide 14: During an allergic reaction, time is the enemy
14 During an allergic reaction, time is the enemy 1. Sampson HA et al. J Allergy Clin Immunol. 2006;117(2):391-397. 2. Lieberman P et al. J Allergy Clin Immunol. 2010;126:477-480. 3. Boyce JA et al; NIAID- Sponsored Expert Panel. J Allergy Clin Immunol. 2010;126(6 suppl):S1-S58. 4. Simons FE. J Alle
Slide 15: What is happening in the allergy rescue space
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 in a carrying case Multiple epinephrine medical devices
Slide 16: Several factors influence epinephrine administration during anaphylaxis
16 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 I
Slide 17: U.S. market has the potential to grow to ~$2B by 2031
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 (EAIs) have increased at a 15% compound annual growth rate (CAGR) from 2021- 2023. 2. https://foodallergy.org/resources/epidemic-infographic. 2
Slide 18: Lead Asset Anaphylm (epinephrine) Sublingual Film
18 Lead Asset AnaphylmTM (epinephrine) Sublingual Film
Slide 19: Anaphylm executive summary
19 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, w
Slide 20: Anaphylm (epinephrine) Sublingual Film
20 + + Easy To Carry Easy To Administer Works Quickly1 First and only non-device based, orally delivered epinephrine product candidate Anaphylm (epinephrine) Sublingual Film 1. Aquestive Therapeutics data on file.
Slide 21: Most common reasons people don't carry their epinephrine devices
21 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 if there has been no accidental exposure in a long t
Slide 22: Incorporating Anaphylm into patients' daily lifestyle routine
22 Incorporating Anaphylm into patients’ daily lifestyle routine 1. https://www.reviews.org/mobile/cell-phone-addiction; July 2023. 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
23 High epinephrine prescribing physicians have spoken1 1. Aquestive Therapeutics 2024 Survey data on file. 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
24 Patients and caregivers have spoken1 1. Aquestive Therapeutics 2024 Survey data on file.
Slide 25: Intellectual Property
<< Aquestive’ Intellectual Property
Slide 26: Anaphylm patent portfolio
26 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 ►
Slide 27: Anaphylm Clinical Program
27 Anaphylm Clinical Program
Slide 28: Anaphylm program overview
28 Anaphylm program overview Pivotal study meets predetermined endpoints for single dose and repeat dose All planned adult supportive studies are now complete and have met predetermined endpoints Pre-NDA meeting with FDA scheduled in Q4 2024
Slide 29: Anaphylm is fast-acting and well-tolerated
29 Anaphylm is fast-acting and well-tolerated, with a safety profile comparable to standard of care (SOC)1 Consistent time to peak drug concentration (Tmax) of 12-15 minutes Onset of pharmacodynamics (PD) effects within 2-5 minutes Performed consistently in the presence of food (clinically), drink,
Slide 30: Anaphylm Pivotal Study Results
30 Anaphylm Pivotal Study Results
Slide 31: Pivotal study meets primary endpoints of Cmax
31 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, AUC0-45min 520.6 470.2 469.2 308.2 Cmax Bracketing 1. All figures are baseline corrected (removal of b
Slide 32: Primary endpoint of pAUC met
32 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 (minutes) Pa rti al AU C (h r* pg /m l) Anaphy
Slide 33: Anaphylm demonstrated a rapid and robust PK profile
33 Anaphylm demonstrated a rapid and robust PK profile1 Geometric Mean Epinephrine Concentrations by Product After a Single Dose Anaphylm’s epinephrine concentration: • Exceeds Adrenalin manual intramuscular (Manual IM) beginning at 2 minutes • Matches EAIs by 10 minutes • Sustains levels above Manu
Slide 34: Tmax of Anaphylm demonstrates more consistency
34 Time to maximum concentration (Tmax) 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 ● An
Slide 35: Anaphylm demonstrates rapid pharmacodynamic effects
35 Anaphylm demonstrates rapid pharmacodynamic (PD) effects1 Manual IMAnaphylmEpiPenAuvi-Q Median Change in Systolic Blood Pressure Over Time -5 0 5 10 15 20 SB 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: Anaphylm Supportive Studies Results and Clinical Timeline
36 Anaphylm Supportive Studies Results and Clinical Timeline
Slide 37: Temperature/pH study PK results
37 Anaphylm temperature/pH study PK results1 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) 123% 132% Key Takeaways: ● No signifi
Slide 38: Temperature/pH study PD results
38 Anaphylm temperature/pH study PD results1 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 with prior Anaphylm clinical study result
Slide 39: Diversified pipeline
39 Diversified pipeline 1. Annual revenue includes revenue for patients 12 and up after launch in 2027. 2. Aquestive Therapeutics data on file.
Slide 40: Self-administration PK study results
40 Anaphylm self-administration PK study results1 Key Takeaways: ● Cmax was not statistically different whether Anaphylm was self-administered or administered by a healthcare provider (HCP) ● Median Tmax was 15 minutes for Anaphylm whether self-administered or administered by an HCP ● Median Tmax fo
Slide 41: Self-administration study PD results
41 Anaphylm self-administration study PD results1 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 are in alignment with prior study resul
Slide 42: Oral allergen challenge study (OASIS) design
42 Oral allergen challenge study (OASIS) induced subject reactions Screening Clinician tracks patient symptoms until resolution First subject visit Second subject visit Dosing 1. Subjects received either single dose or repeat dose of Anaphylm 2. Clinician tracks symptoms from time of dosing until re
Slide 43: OASIS study: rapid symptom resolution after Anaphylm
43 OASIS study - complete symptom resolution occurs rapidly after Anaphylm administration¹ 0% 20% 40% 60% 80% 100% 0 10 20 30 40 50 60 Po pu la tio n w ith a ll sy m pt om s re so lv ed Time (minutes) Screening Single Dose Repeat Dose Time of initial Anaphylm dosing Time from allergen exposure to co
Slide 44: OASIS study: symptom relief correlates to PK levels
44 OASIS study - symptom relief correlates to Anaphylm PK levels¹,² 1. Aquestive Therapeutics data on file. 2. Data represent per protocol patient population. Time comparison of geometric mean baseline corrected epinephrine concentration and symptom resolution following allergen exposure and single
Slide 45: OASIS study: symptom relief with repeat dosing
45 OASIS study - symptom relief was also observed with repeat dosing of Anaphylm¹,² 1. Aquestive Therapeutics data on file. 2. Data represent per protocol patient population. Time comparison of geometric mean baseline corrected epinephrine concentration and symptom resolution following allergen expo
Slide 46: OASIS study: PK profile with and without allergen exposure
46 OASIS study - Anaphylm PK profile remains consistent with and without allergen exposure¹,² 1. Aquestive Therapeutics data on file. 2. Data represent per protocol patient population. Geometric mean baseline-adjusted epinephrine concentration over time in OAS subjects after single dose administrati
Slide 47: OASIS study: single dose meets primary endpoints
47 OASIS study - Anaphylm single dose meets predetermined primary endpoints¹,² Administration AUC0- 10min AUC0- 20min AUC0- 30min AUC0- 45min Manual IM (n=24) 6.0 18.9 39.0 76.0 Anaphylm with allergen (n=23) 14.4 63.2 97.0 132.1 Anaphylm without allergen (n=15) 11.0 50.3 82.6 124.1 Administration Cm
Slide 48: OASIS study: repeat dose meets primary endpoints
48 OASIS study - Anaphylm repeat dose meets predetermined primary endpoints¹,² Administration AUC0- 10min AUC0- 20min AUC0- 30min AUC0- 45min Manual IM (n=22) 5.1 15.5 39.2 99.4 Anaphylm with allergen (n=23) 10.1 62.6 216.8 360.5 Anaphylm without allergen (n=13) 9.2 35.0 106.5 180.4 Administration C
Slide 49: Expected clinical timeline for Anaphylm
49 Q2 2024 Q3 2024 Q4 2024 Q1 2025 Temperature/pH Study Completed Self-Administration Study Completed Pre-NDA Meeting File NDA Pediatric Study Expected clinical timeline for Anaphlym • OASIS study: Assessing PK profile for Anaphylm in the presence of oral physiologic change in subjects with oral all
Slide 50: Pipeline Products
<< Aquestive’ Pipeline Products
Slide 51: Expected full launch path for Libervant
51 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 submit NDA and launch for ages 6 to 11, if approved by FDAPDUFA Date • December 23, 2021 Tentative FDA approval recei
Slide 52: AQST-108 (epinephrine) Topical Gel
52 AQST-108 (epinephrine) Topical Gel 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 53: AQST-108 planned Phase 2a study for alopecia areata
53 AQST-108 planned Phase 2a clinical study for alopecia areata¹ 1. Plan on commencing study after alignment with the FDA. Phase 2a Study Design • 24-48 subjects, 4 doses • 12 – 24 weeks² • Change from baseline ≥10% in Severity of Alopecia Tool (SALT) score at Week 12 • Trichoscopy evaluations and l
Slide 54: Planned AQST-108 clinical and regulatory timeline
54 Planned AQST-108 clinical and regulatory approval timeline¹ 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.
Slide 55: Financial Guidance
<< Aquestive’ Financial Guidance
Slide 56: 2024 Outlook
56 2024 expected outlook as of November 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 in Q1 2024 with high quality institutional
Slide 57: Thank You
<< Aquestive’ Thank You
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