Aquestive Therapeutics, Inc. · NASDAQ:AQST

Aquestive Therapeutics — Corporate Update (March 2024)

35 slides from Aquestive Therapeutics's corporate update, Mar 15, 2024

  1. Slide 1: Pivotal Study and FDA Meeting Supplemental Slides

    Advancing medicines. Solving problems. Improving lives. Advancing medicines. Solving problems. Improving lives. Pivotal Study and FDA Meeting Supplemental Slides March 2024 1

  2. Slide 2: Disclaimer

    Advancing medicines. Solving problems. Improving lives. Disclaimer © 2024 Property of 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 stat

  3. Slide 3: Anaphylm Pivotal Study Results

    Advancing medicines. Solving problems. Improving lives. Anaphylm Pivotal Study Results 3

  4. Slide 4: Pivotal study meets all primary and secondary endpoints

    Phase 3 pivotal study meets all primary and secondary endpoints 4 Advancing medicines. Solving problems. Improving lives. Pivotal Study meets endpoints for single dose and repeat dose  Single dose: maximum concentration (Cmax) and partial area under the curve (AUC) measures met as predefined  Repe

  5. Slide 5: Anaphylm pivotal study design

    AnaphylmTM pivotal study design Healthy volunteers (n=36) Anaphylm Adrenalin (Manual IM) EpiPen® Anaphylm Adrenalin (Manual IM) EpiPen ® 15 min Dose #1 Repeat Dose Dose #2 Single Dose Healthy volunteers (n=64) Anaphylm Adrenalin (Manual IM) Auvi-Q® EpiPen 5 Advancing medicines. Solving problems. Imp

  6. Slide 6: Key endpoints

    Key endpoints Pharmacokinetic (PK) Pharmacodynamic (PD) • Maximum plasma concentration (Cmax) • Time to maximum plasma concentration (Tmax) • Partial area under the curve at 10, 20, 30, and 45 minutes • Subjects reaching 100pg/mL over time • Change in systolic blood pressure (SBP) • Change in diasto

  7. Slide 7: Anaphylm Topline Pivotal Study Results: Single Dose (n=64)

    Advancing medicines. Solving problems. Improving lives. Anaphylm Topline Pivotal Study Results: Single Dose (n=64) 7

  8. Slide 8: Anaphylm 12mg single dose study meets primary endpoints

    8 Anaphylm 12mg single dose study meets primary endpoints AUC0-10min AUC0-20min AUC0-30min AUC0-45min Adrenalin 3.3 11.3 29.7 74.6 Anaphylm 17.7 71.5 104.8 138.6 EpiPen 23.4 62.0 109.7 179.8 Auvi-Q 21.8 61.5 112.4 200.3 Amnt (pg/mL) Auvi-Q 520.6 Anaphylm 470.2 EpiPen 469.2 Adrenalin 308.2 Cmax Brack

  9. Slide 9: Anaphylm is biocomparable to injectors

    Anaphylm is biocomparable to injectors Geometric Mean Epinephrine Exposure Levels (pAUC) by Product After a Single Dose 9 Advancing medicines. Solving problems. Improving lives.  Anaphylm partial AUC values are comparable to autoinjectors for 30 minutes post dosing and remain bracketed past 60 minu

  10. Slide 10: Anaphylm continues to show rapid uptake during the first minutes after dosing

    Anaphylm continues to show rapid uptake during the first 10 minutes after dosing CONFIDENTIAL10 Advancing medicines. Solving problems. Improving lives. Geometric Mean Epinephrine Timepoint Concentrations by Product After a Single Dose  Rapid uptake follows film dissolution and absorption profile

  11. Slide 11: Anaphylm remains above 100 pg/mL after dosing

    Anaphylm remains above 100 pg/mL at 60 minutes after dosing  Anaphylm achieves rapid PK within first 10 minutes similar to autoinjectors  Anaphylm exceeds Adrenalin beginning at 2 minutes  PK is sustained greater than Adrenalin out to 35 minutes  PK is sustained greater than 100 pg/mL for durati

  12. Slide 12: Tmax is a surrogate for speed of absorption

     Tmax is a surrogate for speed of absorption, a critical factor in treating anaphylaxis  Tmax consistency is an important measure of clinical performance  Anaphylm Tmax interquartile range (5 min) is significantly more consistent than EpiPen, Auvi Q, and Adrenalin  Anaphylm median Tmax of 12 min

  13. Slide 13: Anaphylm reaches 100 pg/mL threshold as rapidly as EpiPen

    Anaphylm reaches 100 pg/mL threshold as rapidly as EpiPen and faster than Adrenalin 13 Advancing medicines. Solving problems. Improving lives. Common FDA-cited key threshold level is an important target to achieve hemodynamic stability and curb the progression of anaphylaxis. Proportion of Subjects

  14. Slide 14: Anaphylm overlaps with comparators at later timepoints (PK Sustainability)

    Anaphylm overlaps with comparators at later timepoints (PK Sustainability) Timepoint: 30 minutes Timepoint: 60 minutes Timepoint: 90 minutes  Anaphylm remains above the 100 pg/mL threshold well past the critical time for treatment  Overlapping exposures even after 30 minutes with comparators reinf

  15. Slide 15: Pharmacodynamics consistent with previous results

    15 Pharmacodynamics consistent with previous results Median Change in Systolic Blood Pressure over Time AQ109301 Part B Manual IM EpiPen Auvi-Q Anaphylm Median Change in Diastolic Blood Pressure over Time AQ109301 Part B Manual IM EpiPen Median Change in Pulse Rate over Time AQ109301 Part B Manual I

  16. Slide 16: Tolerability summary

    Advancing medicines. Solving problems. Improving lives. 16 Tolerability summary  All TEAE categorized as mild (Grade 1).  No SAEs; No severe AEs  TEAEs were transient and resolved without major intervention  Primary cardiovascular TEAE associated with mild palpitations  No intervention required

  17. Slide 17: Anaphylm TopLine Pivotal Study Results: Repeat Dose (n=36)

    Advancing medicines. Solving problems. Improving lives. Anaphylm TopLine Pivotal Study Results: Repeat Dose (n=36) 11 17 7 7

  18. Slide 18: Anaphylm 12mg repeat dose study meets primary endpoints

    Anaphylm 12mg repeat dose study meets primary endpoints 18 Advancing medicines. Solving problems. Improving lives. AUC0-10min AUC0-20min AUC0-30min Adrenalin 4.1 13.8 39.0 Anaphylm 20.3 120.8 378.5 EpiPen 21.0 69.8 172.3 45 minutes 60 minutes Cmax CV% Anaphylm 380 275 2028 91% EpiPen 497 347 899 47%

  19. Slide 19: Subjects rapidly exceed 100 pg/mL threshold after second dose

    100% of subjects were > 100 pg/mL within 5 minutes of receiving a second dose of Anaphylm compared to 30 minutes for EpiPen. Overall study data demonstrates repeat dosing is well-tolerated for all evaluated subjects. Error bars: + SE Repeat dose study indicates subjects at risk for needing a second

  20. Slide 20: Anaphylm median Tmax after second dosing demonstrates rapid absorption

     Anaphylm median Tmax of 10 minutes after second dosing demonstrates rapid absorption, a critical factor for initial treatment failures  Anaphylm Tmax interquartile range (5 min) is significantly more consistent than EpiPen and Adrenalin  Anaphylm median Tmax is the same as EpiPen and is signific

  21. Slide 21: Proportion of Subjects Achieving 100 pg/mL in Repeat Dose Pivotal Study

    Time (minutes) Number and Proportion of Subjects >100 pg/mL Adrenalin (N=36) EpiPen (N=35) Anaphylm (N=36) 5 13 ( 36%) 25 ( 74%) 15 ( 42%) 10 15 ( 42%) 30 ( 86%) 30 ( 83%) 15 17 ( 47%) 31 ( 89%) 34 ( 94%) 20 27 ( 75%) 34 ( 97%) 36 (100%) 30 33 ( 92%) 34 ( 97%) 36 (100%) 45 34 ( 94%) 35 (100%) 36 (10

  22. Slide 22: Pharmacodynamics remain consistent with past performance

    Pharmacodynamics remain consistent with past performance 22 Advancing medicines. Solving problems. Improving lives. Median Change in Systolic Blood Pressure over Time Median Change in Diastolic Blood Pressure over Time Median Change in Pulse over Time Maximum Systolic Blood Pressure Maximum Diastoli

  23. Slide 23: Tolerability summary

    Tolerability summary 23  Most TEAE categorized as mild (Grade 1)  No SAEs; No severe AEs; No severe cardiac events observed  TEAEs were transient and resolved without major intervention  Primary cardiovascular TEAE associated with mild palpitations  No intervention required  No AEs of AV block

  24. Slide 24: Program Timeline

    Advancing medicines. Solving problems. Improving lives. 24 Program Timeline

  25. Slide 25: Anaphylm clinical trials to date

    Advancing medicines. Solving problems. Improving lives. 25 Anaphylm clinical trials to date Study Description Study Status N Data Publicly Disclose d 210010 First-in-Human (FIH), Single Ascending Dose (SAD) study to evaluate safety and tolerability, as well as pharmacokinetic (PK) performance and ph

  26. Slide 26: Agency interactions on Anaphylm program to date

    Agency interactions on Anaphylm program to date 26 Advancing medicines. Solving problems. Improving lives. 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

  27. Slide 27: Projected clinical timeline

    Projected clinical timeline Q1 2024 Q2 2024 Q3 2024 Q4 2024 Temperature / pH study Self administration study Allergen challenge study Pediatric study File NDAPre-NDA Mtg 27 Advancing medicines. Solving problems. Improving lives. Pivotal Study Completed FDA Type C Mtg Completed

  28. Slide 28: Plan for allergen-induced study

    Plan for allergen-induced study Utilize known OAS population to conduct allergen-challenge study  Step 1: introduce allergen to patient  Step 2: monitor for symptoms (minimum 15 minutes)  Step 3: dose Anaphylm  Step 4: conduct blood draws to assess Anaphylm pharmacokinetics Note: a 2nd non-chall

  29. Slide 29: FDA Type C Meeting

    Advancing medicines. Solving problems. Improving lives. FDA Type C Meeting 29

  30. Slide 30: Type C meeting designed to address open items from End-of-Phase 2 meeting

    The Type C meeting was designed to address the open items from our End-of-Phase 2 meeting held in November 2022 30 Advancing medicines. Solving problems. Improving lives. Aquestive press release from December 22, 2022: “The FDA also provided comments on potential issues the Company will need to addr

  31. Slide 31: (1) The impact of any product hold time

    (1) the impact of any product hold time 31 Advancing medicines. Solving problems. Improving lives. Our position We removed hold time from our dosing administration thereby alleviating the issue. Our interpretation of FDA meeting minutes We have adequately addressed their concerns. Additional informa

  32. Slide 32: (2) The potential for emesis (vomiting)

    (2) the potential for emesis (vomiting) 32 Advancing medicines. Solving problems. Improving lives. Our position We will characterize events of emesis for consolidated analysis in the NDA. Our interpretation of FDA meeting minutes Recommended additional information (such as narratives for patients wh

  33. Slide 33: (3) Potential for mouth conditions (such as angioedema)

    (3) potential for mouth conditions (such as angioedema) 33 Advancing medicines. Solving problems. Improving lives. Our position We proposed a histamine study in healthy volunteers followed by the administration of Anaphylm. Our interpretation of FDA meeting minutes The FDA did not agree with this mo

  34. Slide 34: Completeness of clinical development program

    Completeness of clinical development program 34 Advancing medicines. Solving problems. Improving lives. In addition to addressing the FDA comments from our EOP2 meeting, we requested the FDA provide feedback on the completeness of our overall program. Our position Completion of the planned clinical

  35. Slide 35: Thank You

    Advancing medicines. Solving problems. Improving lives. Thank You 35

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