Milestone Pharmaceuticals Inc. · NASDAQ:MIST

Milestone Pharmaceuticals — American Heart Association Scientific Sessions 2022 (AHA22)

18 slides from Milestone Pharmaceuticals's conference presentation, Nov 7, 2022

  1. Slide 1: Self-administered Etripamil for Termination of Spontaneous PSVT: Primary Analysis from the RAPID Study

    #AHA22 Self-administered Etripamil for Termination of Spontaneous Paroxysmal Supraventricular Tachycardia: Primary Analysis from the RAPID Study Bruce S. Stambler1, A John Camm2, Marco Alings3, Paul Dorian4, Hein Heidbuchel5, Jaco Houtgraaf 6, Jose L. Merino7, Blandine Mondésert8, Philip Sager9, Atu

  2. Slide 2: Disclosures

    Disclosures James E. Ip, MD: • Received compensation as study investigator and steering committee member for Milestone Pharmaceuticals. • Received honoraria/speaking/consulting fee for Abbott Medical, Boston Scientific, and Medtronic Inc • Membership on advisory committee and/or steering committee f

  3. Slide 3: Etripamil: Potential New Treatment for PSVT

    Etripamil: Potential New Treatment for PSVT • Novel, investigational, L-type calcium channel blocker • Formulated for intranasal spray with: • Rapid onset of action (Tmax ≤ 7 min) • Short-lasting: inactivated by blood esterases • Developed to satisfy unmet need for self-administered therapy that is

  4. Slide 4: Phase 3 Clinical Study Design

    RAPID Phase 3 Clinical Study Design Objective: Evaluate the efficacy and safety of etripamil nasal spray in patients experiencing a PSVT episode in an at-home setting Screening visit Outcome measures Key inclusion criteria Test dose visit At-Home Treated Independent Adjudication, PSVT Event  Patien

  5. Slide 5: RAPID Study Patient Populations

    RAPID Study Patient Populations Overall safety population1 N = 706 Test dose1 N = 706 Blinded Adjudication 14 not randomized, (9 test dose failures, 5 of Safety Population Other) No ECG Randomized2 N = 692 (9%) 102 discontinued by time of RAPID data cut.3 Non-PSVT 335 no treated PSVT (19%) episode a

  6. Slide 6: Demographics & Baseline Characteristics (Safety Population)

    Demographics & Baseline Characteristics (Safety Population) Placebo (N=120) Etripamil (N=135) Overall (N=255) Age, years Mean (SD) 56.2 (12.0) 52.4 (14.0) 54.2 (13.2) Median (range) 58.0 (21, 78) 52.0 (19, 82) 55.0 (19, 82) Sex, female, n (%) 88 (73.3) 93 (68.9) 181 (71.0) Race, n (%) American India

  7. Slide 7: Primary Endpoint: Conversion of Adjudicated PSVT to NSR at 30 Minutes

    Primary Endpoint: Conversion of Adjudicated PSVT to NSR at 30 min At 30 min., HR = 2.62 (95% CI, 1.659-4.147); p<0.001 100% Kaplan Meier Probability of Conversion, % 80% 64.3% 60% 40% 31.2% 20% Placebo Etripamil 0% 0 5 10 15 20 25 30 Time Since Study Drug Administration (minutes) Placebo 85 78 67 64

  8. Slide 8: 300-Minute Endpoint: Conversion of Adjudicated PSVT to NSR

    300-Minute Endpoint: Conversion of Adjudicated PSVT to NSR At 300 min.: HR = 1.70 (95% CI, 1.213-2.383); p<0.001 100% 82.7% Cumulative Conversion Rate (%) 80% 73.5% 72.0% 60% 54.5% 40% 20% Placebo Etripamil 0% 0 30 60 90 120 150 180 210 240 270 300 Time Since Study Drug Administration (minutes) Plac

  9. Slide 9: Median Time to Conversion of PSVT to NSR Reduced in Etripamil Arm

    Median Time to Conversion of PSVT to NSR, Reduced in Etripamil Arm Compared to Placebo 100% 90% Kaplan Meier Probability of Conversion, (%) 80% 70% Etripamil Placebo 60% 17.2 min 53.5 min 50% 40% 30% 20% 10% 0% 0 5 10 15 20 25 30 35 40 45 50 55 60 65 70 75 80 85 90 Number of Patients at Risk Time Si

  10. Slide 10: Subgroup Analyses: Responders At 30 Minutes

    Subgroup Analyses: Responders At 30 Minutes Responder = conversion of PSVT to normal sinus rhythm. Age groups are not inclusive of the lower bound: Q2 45-54, includes patients >45 years old; Q3 54-62, includes patients >54 years old. PSVT = paroxysmal supraventricular tachycardia; AVNRT = atrioventr

  11. Slide 11: Secondary Endpoints: Medical Interventions, Emergency Department Utilization

    Secondary Endpoints: Medical Interventions, Emergency Department Utilization Patients Seeking Medical Intervention Patients with Emergency Department Visits 40% Placebo Etripamil 40% Placebo Etripamil P=0.0587 P=0.103 P=0.013 P=0.076 P=0.209 P=0.035 35% 35% 30% 30% 27% 25% 25% 25% 25% 25% 22% 21% 20

  12. Slide 12: Safety – Direct ECG CMS Reading

    RAPID Safety – Direct ECG CMS Reading1 Placebo Etripamil Randomized Dose2 Randomized Dose2 N=116 N=128 Non-sustained ventricular tachycardia 19 (16.4) 18 (14.1) Sustained ventricular tachycardia (≥ 30 seconds) 1 (0.9)3 0 PSVT Recurrence 5 (4.3) 4 (3.1) Atrial Fibrillation ≥ 30 seconds 4 (3.5) 1 (0.8

  13. Slide 13: Safety Analysis

    RAPID Safety Analysis Placebo Etripamil Randomized Dose1 Randomized Dose1 N=120 N=135 Subjects with any randomized-period TEAE, n (%) 20 (16.7) 68 (50.4) Maximum severity, and n (%) of subjects with any randomized-period TEAE Mild 15 (75.0%) 46 (67.6%) Moderate 4 (20.0%) 21 (30.9%) Severe 1 (5.0%) 1

  14. Slide 14: Safety – Adverse Events

    RAPID Safety – Adverse Events Placebo Etripamil Randomized Dose2 Randomized Dose2 Subjects with Randomized-period TEAE, Incidence >5%, n (%) (n=120) (n=135) Nasal discomfort 6 (5.0) 31 (23.0) Nasal congestion 1 (0.8) 17 (12.6) Rhinorrhea 3 (2.5) 12 (8.9) Epistaxis 2 (1.7) 8 (5.9)3 Placebo Etripamil

  15. Slide 15: RAPID Study: Summary and Conclusions

    RAPID Study: Summary and Conclusions • RAPID study achieved primary efficacy endpoint of terminating PSVT with self- administered etripamil, using symptom-based optional repeat dosing (HR 2.62, p < 0.001)  Conversion of PSVT to sinus rhythm: 64.3% at 30 minutes, 73.5% at 60 minutes • Median Time to

  16. Slide 16: Acknowledgements

    Acknowledgements  Study Participants  Data Safety Monitoring Committee  Participating study sites / Principal Daniel Beyerbach, MD (Chair and physician) Investigators / Study Coordinators Sean Pokorney, MD (Interim Physician) across 9 countries Benjamin Steinberg, MD (Physician)  Adjudication Co

  17. Slide 17: Trial Sites and Investigators

    RAPID Trial Sites and Investigators UNITED STATES Glenn Meininger, Baltimore, MD Jacqueline Joza, Montreal, PQ Andrea Hornyik, Encs Pawel Derejko, Warszawa Saleem Akbar, Allen, TX Jared Morton, Idaho Falls, ID Yaariv Khaykin, Newmarket, ON Ferenc Lakatos, Bekescsaba Jacek Gniot, Pulawy Jeffrey Ander

  18. Slide 18: Thank You!

    #AHA22 Self-administered Etripamil for Termination of Spontaneous Paroxysmal Supraventricular Tachycardia: Primary Analysis from the RAPID Study THANK YOU!

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