Milestone Pharmaceuticals Inc. · NASDAQ:MIST
Milestone Pharmaceuticals — Corporate Update (March 2020)
16 slides from Milestone Pharmaceuticals's corporate update, Mar 23, 2020
Slide 1: NODE-301 Topline Data Conference Call March 23, 2020
NODE - 301 Topline Data Conference Call March 23, 2020
Slide 2: Disclaimers
Disclaimers This Presentation contains forward - looking statements . Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify thes
Slide 3: Call Participants
Call Participants Prepared Remarks • Joseph Oliveto, Chief Executive Officer Additional Q&A Participants • Amit Hasija, Chief Financial Officer • Lorenz Muller, Chief Commercial Officer • Jeff Nelson, Chief Operating Officer • Francis Plat, MD, Chief Medical Officer 3
Slide 4: Opportunity to Shift the Standard of Care out of the Acute-Care Setting
Opportunity to Shift the Standard of Care out of the Acute - Care Setting for ~2 Million PSVT Patients Sources: Internal estimates based on market research and longitudinal analysis of Truven/ Marketscan and Medicare claims data; Page RL et al, 2015 ACC/AHA/HRS guideline for the management of adult
Slide 5: Overview top-line
Overview NODE - 301 top - line: • Missed its primary endpoint over 5 hours • Showed clinically meaningful efficacy during the first 45 minutes consistent with the known pharmacology of etripamil • Human factors had little impact on study execution or results • Demonstrated a positive safety profile
Slide 6: Pivotal Phase Study Design
Pivotal Phase 3 Study Design PSVT = Paroxysmal Supraventricular Tachycardia; SR = Sinus Rhythm; Etr = etripamil; Pbo = placebo 6 Documented diagnosis of PSVT History of longer episodes A djudicated PSVT events Efficacy Dataset (N=156, 79%) Etr =107, Pbo =49) Objective: Superiority of etripamil over
Slide 7: Primary Endpoint – Time to Conversion Analysis
NODE - 301 Primary Endpoint – Time to Conversion Analysis 7 Pbo 49 32 18 12 5 1 1 0 Etripamil 107 47 36 31 28 22 15 13 11 9 3 NODE - 301 study missed its primary endpoint over 5 hours, but showed early efficacy Number of subjects remaining to resolve their event 0% 10% 20% 30% 40% 50% 60% 70% 80% 90
Slide 8: NODE-Pharmacological Study Results
NODE - 102 Pharmacological Study Results 8 Effective pharmacologic activity of etripamil occurs between 5 and 45 minutes Placebo Etripamil 70mg -10 -5 0 5 10 15 20 0 10 20 30 40 50 PR Interval Prolongation Change from Baseline (%, with 95% CI) Time (min) 10% PR Prolongation Threshold
Slide 9: Efficacy – Time to Conversion over 45 Minutes
NODE - 301 Efficacy – Time to Conversion over 45 Minutes 9 0% 10% 20% 30% 40% 50% 60% 70% 0 5 10 15 20 25 30 35 40 45 % Converted to SR Time to Conversion (minutes) Placebo Etripamil 70mg HR (95% CI) 1.668 ( 1.026 , 2.712) p=0.02
Slide 10: Key Secondary Endpoints
43.27 57.42 0 10 20 30 40 50 60 70 80 90 100 Score on TSQM - 9 Questionnaire TSQM - 9 Global Satisfaction With 95% CI bars Placebo (N=49) Etripamil (N=104) P=0.0069 P=0.0015 NODE - 301 Key Secondary Endpoints 10 Key secondary endpoints from NODE - 301 support benefit of etripamil to patients and pay
Slide 11: Safety Analysis: Randomized Treatment Emergent Adverse Events (RTEAE)
Randomized Treatment Emergent Adverse Events (RTEAE) Etripamil N=138 Placebo N=60 Subjects with any RTEAE 53 (38.4) 12 (20.0) Maximum severity of RTEAE Mild 45 (32.6) 10 (16.7) Moderate 8 (5.8) 3 (3.3) Severe 0 (0.0) 0 (0.0) Subjects with any Serious Adverse Event (SAE) 0 (0.0) 1 (1.7) Subjects with
Slide 12: Randomized Treatment Emergent Adverse Events
Randomized Treatment Emergent Adverse Events Etripamil (N=138) Placebo (N=60) Nasal discomfort 27 (19.6) 4 (6.7) Nasal congestion 11 (8.0) 2 (3.3) Epistaxis 9 (6.5) 0 (0.0) Rhinorrhea 8 (5.8) 1 (1.7) Throat irritation 7 (5.1) 1 (1.7) Headache 4 (2.9) 0 (0.0) Sneezing 3 (2.2) 0 (0.0) Atrioventricular
Slide 13: Etripamil PSVT Phase Development Plan
Etripamil PSVT Phase 3 Development Plan 2018 Efficacy & Safety Efficacy & Safety Safety NODE - 301 NODE - 301B NODE - 302 Open - Label Extension Safety NODE - 303 Open - Label Safety 2019 2020 2021 Data Primary Utility PSVT = Paroxysmal Supraventricular Tachycardia 13
Slide 14: Overview top-line
Overview NODE - 301 top - line: • Missed its primary endpoint over 5 hours • Showed clinically meaningful efficacy during the first 45 minutes consistent with the known pharmacology of etripamil • Human factors had little impact on study execution or results • Demonstrated a positive safety profile
Slide 15: Thank You
Thank You 15
Slide 16: Kaplan-Meier Plot of Conversion up to Hour 5
HR (95% CI) 1.086 ( 0.726 , 1.623) p<0.1212 peto - peto Kaplan - Meier Plot of Conversion up to Hour 5 Efficacy Population 16
Milestone Pharmaceuticals — Corporate Update (March 2020) on 6ix