Milestone Pharmaceuticals Inc. · NASDAQ:MIST
Milestone Pharmaceuticals — Investor Presentation (March 2023)
69 slides from Milestone Pharmaceuticals's investor presentation, Mar 31, 2023
Slide 1: Company Overview March 30, 2023
Company Overview March 30, 2023
Slide 2: Forward Looking Statement
Forward Looking Statement 2 Milestone Corporate Overview The Presentation contains forward - looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, as amended. Words such as ‘‘aim,’’ ‘‘anticipate,’’ ‘‘assume,’’ ‘‘believe,’’ ‘‘cont
Slide 3: Recent Progress on PSVT & AFib-RVR Programs for Etripamil
Recent Progress on PSVT & AFib - RVR Programs for Etripamil 3 Milestone Corporate Overview Positive FDA feedback – PSVT NDA filing package proposal considered acceptable On target for NDA filing for PSVT - Q3, 2023 1 2 3 Completed last two Phase 3 PSVT trials – RAPID - Extension & NODE - 303 Initial
Slide 4: PSVT and AFib-RVR Cause Markedly Symptomatic Attacks That Disrupt Patients' Lives
PSVT and AFib - RVR Cause Markedly Symptomatic Attacks That Disrupt Patients’ Lives 4 Milestone Corporate Overview Symptoms include… Many patients feel anxious and powerless • Heart palpitations • Chest pressure or pain • Shortness of breath • Fatigue • Light - headedness • Anxiety Paroxysmal Suprav
Slide 5: Current Treatment of Acute Attacks in the Emergency Department are Burdensome and Costly
Current Treatment of Acute Attacks in the Emergency Department are Burdensome and Costly 5 Milestone Corporate Overview Need for simple, fast - acting treatment, reduce trips to ED and calls to physicians • Time - consuming, disruptive • Often results in a hospital admission • Expensive use of healt
Slide 6: Etripamil Nasal Spray is a Novel L-type Investigational Calcium Channel Blocker
Etripamil Nasal Spray is a Novel L - type Investigational Calcium Channel Blocker Designed to Treat Quickly 6 Milestone Corporate Overview Empowering patients to treat symptomatic attacks Fast onset of action ( T max ≤ 7 min) Patient self - administered Small enough to fit in your pocket
Slide 7: Milestone Pharma - Targeting Vast Unmet Need for Patient Management of Common Heart Conditions
Milestone Pharma - Targeting Vast Unmet Need for Patient Management of Common Heart Conditions PSVT = Paroxysmal Supraventricular Tachycardia; AFib - RVR = Atrial Fibrillation with Rapid Ventricular Rate; 7 Milestone Corporate Overview Targeting Common Arrhythmias Empowering Patients to Treat Themse
Slide 8: Standard of Care is Inadequate
Standard of Care is Inadequate – Most Patients Experience Events Despite Preventative Options 8 Milestone Corporate Overview 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 th
Slide 9: PSVT & AFib-RVR are Sizable and Underserved Markets in the US
PSVT & Atrial Fibrillation with Rapid Ventricular Rate are Sizable and Underserved Markets in the US Source(s): 1. Colilla et al., Am. J. Cardiol . 2013, 112(8), 1142 - 1147; Miyasaka et al., Circulation, 2006, 114, 119 - 125. American Heart Association 2 . HCUP ED & Admissions Data (2016), accessed
Slide 10: Intravenous Calcium Channel Blockers Are Proven Effective for PSVT and AFib-RVR
Intravenous Calcium Channel Blockers (CCB) • Slows conduction signal from the atria to the ventricles over the AV Node • PSVT: breaks the circuit, returning heart to normal rhythm • AFib - RVR: slows heart rate and reduces symptoms while remaining in Atrial Fibrillation Intravenous Calcium Channel B
Slide 11: Etripamil Clinical Pipeline Advancement
Etripamil Clinical Pipeline Advancement 11 Milestone Corporate Overview PSVT AFib - RVR Phase I Phase II Phase III NDA Filing / Approval Pharmacology of L - type calcium channel blockers drives broad clinical utility Rapid conversion to sinus rhythm Acute control of rapid ventricular rate
Slide 12: Comprehensive Data Supports FDA New Drug Application for PSVT
Comprehensive Data Supports FDA New Drug Application for Rapid Conversion of PSVT Episodes to Sinus Rhythm in Adults NODE - 1 NODE - 301 NODE - 302 (Ext. of NODE - 301) RAPID NODE - 303 Phase 2 Phase 3 Phase 3 Phase 3 Phase 3 Efficacy (dose finding) Efficacy Safety & Efficacy (Repeat Episodes) Effic
Slide 13: Positive Phase 3 RAPID Trial in Patients with PSVT
Positive Phase 3 RAPID Trial in Patients with PSVT HR = Hazard Ratio; CI = Confidence Interval Source: Milestone Pharmaceuticals Data on File 13 Milestone Corporate Overview Randomized, double - blind, placebo - controlled trial enrolled 706 patients to self - administer Etripamil 70 mg regimen or p
Slide 14: Data Indicates Fast Conversion to Normal Sinus Rhythm
Data Indicates Fast Conversion to Normal Sinus Rhythm (NSR) RAPID Study "+" symbol on graph indicates censoring for signal loss (n=4 over 90 minutes) Source: Milestone Pharmaceuticals Data on File 14 Milestone Corporate Overview 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% 0 5 10 15 20 25 30 35 40 45
Slide 15: Fewer Medical Interventions and Emergency Department Visits
27% 25% 25% 14% 15% 15% 0% 5% 10% 15% 20% 25% 30% 35% 40% NODE-301 Study RAPID Study Pooled Analysis n=49 n=107 n=85 n=99 n=134 n=206 Patients Seeking Medical Intervention P=0.0587 P=0.103 P=0.013 n=13 n=15 n=21 n=15 n=34 n=30 Patients with Emergency Department Visits 25% 21% 22% 13% 14% 14% 0% 5% 1
Slide 16: Etripamil Well-Tolerated with a Favorable Safety Profile
Etripamil Well - Tolerated with a Favorable Safety Profile RAPID Study – Safety Events 16 Milestone Corporate Overview 1 R andomized - period treatment - emergent adverse events, those >5% or those specifically tracked as potentially representing lowered blood pres sure. 2 Safety Population. 3 Six o
Slide 17: Etripamil Has Substantial Potential Value for Stakeholder Groups If Approved
Etripamil Has Substantial Potential Value for Stakeholder Groups If Approved 17 Milestone Corporate Overview Sources: Internal market research, PSVT = Paroxysmal Supraventricular Tachycardia, ED = Emergency Department • Designed for patient self - management • Frees up physician time and office reso
Slide 18: Cardiologists Expect to Prescribe Etripamil to the Majority of Unablated PSVT Patients
Cardiologist Expect to Prescribe Etripamil to the Majority of Unablated PSVT Patients Source: Quantitative market research conducted by Triangle Insights Group (n=250 cardiologists), June - September 2020; Estimated number of unique patients with annual claims for PSVT from Truven MarketScan data, 2
Slide 19: PSVT & AFib-RVR Populations in the US
PSVT & AFib - RVR Populations in the US Source(s): 1. Colilla et al., Am. J. Cardiol . 2013, 112(8), 1142 - 1147; Miyasaka et al., Circulation, 2006, 114, 119 - 125. American Heart Association 2 . HCUP ED & Admissions Data (2016), accessed January 2021. 3. Rehorn et al. Journal of Cardiovascular Ele
Slide 20: Effect of Etripamil on Heart Rate While in SVT (NODE-301)
Effect of Etripamil on Heart Rate (HR) while in SVT (NODE - 301 Study) • Improvement in HR observed within first minute, with maximum difference at 10 minutes • Differences were statistically significant through 40 minutes • Reduction in heart rate in etripamil group sustained for 1 hour • Some pati
Slide 21: ReVeRA Phase II Proof of Concept Trial in AFib-RVR
ReVeRA - Phase II Proof of Concept Trial of Etripamil in AFib - RVR in the Emergency Department Setting 21 Milestone Corporate Overview Patient presents to ED with episode of AFib - RVR Dosing & Assessment Efficacy Analysis 1. Baseline ECG for ≥ 10 min 2. Administer double blind study drug 70 mg etr
Slide 22: Potential Effect of Etripamil on Heart Rate in AFib-RVR
Potential Effect of Etripamil on Heart Rate on Patients in AFib - RVR 22 Milestone Corporate Overview PSVT Patients HR Reduction NODE - 301 Afib - RVR In PSVT Program AFib - RVR in REVERA (interim) Public Release 2023 Medical Conference Q2’ 2023 Utilizing Proof of Concept Data to Design Pivotal AFib
Slide 23: Peak US Market Opportunity for Etripamil in PSVT and AFib-RVR
Peak US Market Opportunity for Etripamil in PSVT and AFib - RVR Sources: Internal estimates based on market research, Milestone Pharmaceuticals Inc. 23 Milestone Corporate Overview ~2.6 million patients US Prevalence (2030) ~40 - 60% ~1.0 - 1.6 million patients ~500k - 800k patients 4 - 6/year Addre
Slide 24: Strategic Financing with RTW Extends Runway to Mid-2025
Strategic Financing with RTW Extends Runway to Mid - 2025 Helps fund launch and commercialization of Etripamil in PSVT 24 Milestone Corporate Overview 1 VWAP – Volume Weighted Average Price as of 3/27/23 2 Rate can increase by 2.5% if certain annual net sales thresholds are not met 3 Annual net prod
Slide 25: Finances as of December 31, 2022
Finances – as of December 31, 2022 Cash and short - term investments of $64.6M at year - end (proforma for convertible notes 114.6M (1) ) • March 2023, $125.0M strategic financing strengthens cash position (1) Cash balance plus proceeds from March 2023 financing (1) funds operations to mid - 2025 (1
Slide 26: Milestone Pharma - Targeting Vast Unmet Need for Patient Management of Common Heart Conditions
Milestone Pharma - Targeting Vast Unmet Need for Patient Management of Common Heart Conditions PSVT = Paroxysmal Supraventricular Tachycardia; AFib - RVR = Atrial Fibrillation with Rapid Ventricular Rate; 26 Milestone Corporate Overview Targeting Common Arrhythmias Empowering Patients to Treat Thems
Slide 27: Appendix
Milestone Corporate Overview 27 Appendix
Slide 28: Core PSVT Market Addressable Now, if approved, with Potential for Expansion
Core PSVT Market Addressable Now, if approved, with Potential for Expansion Source: 1) assumes annual incidence rate for PSVT of ~300k from longitudinal claims analysis and the average time to diagnosi s ( currently 2 - 3 years) can be reduced to <6 months 2) Calculated as the difference between PSV
Slide 29: Management of Patients with PSVT and Call Point Targeting
Management of Patients with PSVT and Call Point Targeting • Targeted sales force to reach majority of available opportunity • Significant overlap with most common CV portfolio call points Source: Internal market research 29 Clinical Cardiologists Primary Care Physicians Electro - physiologists % of
Slide 30: Published Disease Data Likely Under-Reports Burden of PSVT
Published Disease Data Likely Under - Reports Burden of PSVT Strengths • Provides important demographic and clinical characteristic data on patients with PSVT • Positive Predictive Values from PREEMPT useful • Less than 40% of incident cases in MESA would have been detected by PSVT ICD - 9 Code 427.
Slide 31: Estimating Prevalence, Incidence, and Annually Treated Patients Using Claims Data
Estimating Prevalence, Incidence, and Annually Treated Patients Using Longitudinal Claims Data Source: Data on file from IBM Marketscan ® Commercial Research Database (<65y) and the Medicare Limited Dataset (≥65y), with demographic, enrollment and claims data f or commercially insured (Truven) and M
Slide 32: Current US PSVT Market
• Prevalence ~2M diagnosed PSVT patients • ~300K newly diagnosed per year • ~600K patients treated per year • >150K ED/hospital visits per year • ~80K ablations per year No Rx ~25% Chronic Therapy ~65% Ablation ~10% Current US PSVT Market Source: Sacks, N.C. et al; Prevalence of Paroxysmal Supravent
Slide 33: New Data Enhances Understanding of Burden of PSVT
New Data Enhances Understanding of Burden of PSVT Analysis of Prospective Patient Reported Outcomes Longitudinal Data • Phase 1: Baseline Survey (medical and SVT episode history) • Phase 2: Longitudinal Weekly Surveys (episode survey if experienced an episode, QoL survey if not) 5,277 surveys comple
Slide 34: Peak US Market Opportunity for Etripamil in PSVT
Peak US Market Opportunity for Etripamil in PSVT Patients with PSVT *Patient stated severity of SVT episode (mild, moderate, or severe) Sources: Internal estimates based on market and outcomes research, Milestone Pharmaceuticals. 1. Rehorn et al. Journal of Cardiovascular Electrophysiology. 2021 Aug
Slide 35: PRO Analyses Provide A Clearer Picture of Burden of PSVT
PRO Analyses Provide A Clearer Picture of Burden of PSVT than Market Research Alone Sources: Internal estimates based on market and outcomes research, Milestone Pharmaceuticals. 1. PSVT patient market researc h c onducted by Triangle Insights Group, 2018 (n =250). 2. PSVT patient market research con
Slide 36: Phase 3 PSVT Program - Other than RAPID
Phase 3 PSVT Program – Other than RAPID • NODE - 301 • NODE - 302
Slide 37: NODE-301 Study Design
NODE - 301 Study Design 37 Milestone Corporate Overview Test Dose Randomization Event Primary Efficacy Analysis 70 mg dose of etripamil Administer in office while in sinus rhythm for safety evaluation (N=431) Randomized 2:1 70 mg etripamil : placebo (N=419) 1. Patient recognizes symptoms 2. Applies
Slide 38: NODE-301 Primary Endpoint Over 5 Hours
Primary Endpoint - over 5 hours Hazard Ratio = 1.09; p<0.12 NODE - 301 Kaplan - Meier Plot of Conversion to Sinus Rhythm 38 Milestone Corporate Overview Post hoc analysis: over 30 minutes Etripamil 54% vs. Placebo 35% HR = 1.87; p<0.02 Primary analysis: over 5 hours HR = 1.086; p<0.12 Source: Data o
Slide 39: NODE-301 Conversion up to 5 Hours with Medical Intervention as Treatment Failures
NODE - 301 Conversion up to Hour 5 with Medical Intervention Patients Analyzed as Treatment Failures at 5 hours (post - hoc) 39 Milestone Corporate Overview Etripamil 70mg Placebo Subjects who convert following medical assistance are censored at 5 hours. Subjects who present missing data from time t
Slide 40: NODE-301 Efficacy - Time to Conversion over 45 Minutes
NODE - 301 Efficacy – Time to Conversion over 45 Minutes 40 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 32% 14% 54% 35% Milestone Corporate Overview New Primary Endpoint Second Dose Option for RAPID HR 1.87, p=0.02
Slide 41: NODE-301 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 41 Key secondary endpoints from NODE - 301 support benefit of etripamil to patients and pay
Slide 42: NODE-301 Safety Analysis
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) Most Common Adverse Events (>5%) Nasal discomfort 27 (19.6) 4 (6.7)
Slide 43: NODE-301 Safety Analysis
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 44: NODE-301 Safety Analysis
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 45: NODE-302 Study Design: Single-arm, Open-label Extension Study
NODE - 302 Study Design: Single - arm, Open - label Extension Study From NODE - 301 Study Procedures 1. Patient perceived PSVT episode 2. Patient applied CMS 3. Patient performed trained VM 4. If episode persisted, patient self - administered etripamil 70 mg intranasally 5. CMS ECG monitoring contin
Slide 46: Conversion of Adjudicated PSVT to Sinus Rhythm - NODE-302 and NODE-301
Conversion of Adjudicated PSVT to Sinus Rhythm at 30 and 60 Minutes – NODE - 302 and NODE - 301 Source: Ip, JE et al; Etripamil Nasal Spray Is Effective and Safe for Conversion of Repeated Spontaneous Episodes of Paroxysm al Supraventricular Tachycardia During Long - term Follow - up: Results From t
Slide 47: NODE-302: Number of Positively Adjudicated PSVT Episodes Per Patient
NODE - 302: Number of Positively Adjudicated PSVT Episodes Per Patient Treated With Etripamil Source: Ip, JE et al; Etripamil Nasal Spray Is Effective and Safe for Conversion of Repeated Spontaneous Episodes of Paroxysm al Supraventricular Tachycardia During Long - term Follow - up: Results From the
Slide 48: NODE-302: Conversion of Adjudicated PSVT - 1st Episode
NODE - 302: Conversion of Adjudicated PSVT to Sinus Rhythm – 1st Episode Source: Ip, JE et al; Etripamil Nasal Spray Is Effective and Safe for Conversion of Repeated Spontaneous Episodes of Paroxysm al Supraventricular Tachycardia During Long - term Follow - up: Results From the NODE - 302 Study; He
Slide 49: NODE-302: Conversion of Adjudicated PSVT - 2nd Episode
NODE - 302: Conversion of Adjudicated PSVT to Sinus Rhythm – 2nd Episode Source: Ip, JE et al; Etripamil Nasal Spray Is Effective and Safe for Conversion of Repeated Spontaneous Episodes of Paroxysm al Supraventricular Tachycardia During Long - term Follow - up: Results From the NODE - 302 Study; He
Slide 50: NODE-302: Consistency of Conversion between 1st and 2nd Episodes
NODE - 302 Consistency of Conversion at 30 Minutes between the 1 st and 2nd Adjudicated PSVT Episodes • 75% of patients (30/40) had a consistent response between the 1st and 2nd episode (Chi - square=8.09; P =0.0045) • 21/26 patients (81%) who converted on their 1st episode also successfully convert
Slide 51: NODE-302: Additional Medical Intervention
NODE - 302: Additional Medical Intervention • 13% of patients and 8.5% of positively adjudicated PSVT episodes required additional medical intervention • Additional medical interventions included: ― Intravenous adenosine (n=12) ― Physician - initiated vagal maneuver (n=2) ― Orally self - administere
Slide 52: NODE-302: Most Frequent Etripamil-related TEAEs
NODE - 302: Most Frequent Etripamil - related TEAEs • Majority of TEAEs were nasal/local, mild, and brief • No reported cases of syncope or symptoms of hypotension • No episodes of AV block or pauses after PSVT conversion with etripamil Source: Ip, JE et al; Etripamil Nasal Spray Is Effective and Sa
Slide 53: Comparison of Etripamil-Related AEs with Repeat Dosing
Comparison of Etripamil - Related AEs with Repeat Dosing – NODE - 301 and NODE - 302 Source: Ip, JE et al; Etripamil Nasal Spray Is Effective and Safe for Conversion of Repeated Spontaneous Episodes of Paroxysm al Supraventricular Tachycardia During Long - term Follow - up: Results From the NODE - 3
Slide 54: Phase 1 and Phase 2 Clinical Studies
Phase 2 and Phase 1 Clinical Studies
Slide 55: Etripamil Nasal Spray: A Novel CCB Designed to be Fast, Portable, and Patient-Empowering
Etripamil Nasal Spray: A Novel CCB Designed to be Fast, Portable, and Patient - Empowering 55 Milestone Corporate Overview PK Plots of Intranasally Administered Etripamil (single and repeat dosing) • Developed to rapidly terminate episodes of PSVT • Designed for patient self - administration where
Slide 56: Etripamil Phase 1 Pharmacology PR Prolongation Used to Select Doses
Etripamil Phase 1 Pharmacology PR Prolongation Used to Select Doses for Phase 2 Error bars indicate standard error of the mean 56 20 40 60 80 100 120 140 placebo 8 4 6 2 10 12 14 16 18 20 0 Target PR threshold PR Interval Prolongation (%) Etripamil Dose (mg) Milestone Corporate Overview
Slide 57: Etripamil Nasal Spray Pharmacological Results (NODE-102)
Etripamil Nasal Spray Pharmacological Results (NODE - 102) 57 Milestone Corporate Overview Anticipated therapeutic effect within 45 minutes; peak within 10 minutes -10% 0% 10% 20% 0 20 40 60 80 100 120 140 160 0 10 20 30 40 50 60 70 80 90 100 PR Interval – Mean change from baseline (%) Mean Etripami
Slide 58: PK of Etripamil Repeat Administration
PK of Etripamil 30 mg Repeat Administration at T=10 min (Study MSP - 2017 - 1096) 58 0 10 20 30 40 50 60 70 80 90 100 0 10 20 30 40 50 60 Plasma Concentration (ng/ml) Time (minutes) 30mg 30mg Milestone Corporate Overview Repeat administration increases both Cmax and AUC N=7, Error bars are standard
Slide 59: Phase 2a/b Study Design (NODE-1)
Placebo n=20 Objectives: Demonstrate superiority of etripamil over placebo in terminating SVT and dose - ranging trend analysis Etripamil 140mg n=21 15 min Etripamil 105mg n=20 0 min - 5 min Phase 2a/b Study Design (NODE - 1) EP = electrophysiology. SVT = supraventricular tachycardia, PSVT = paroxys
Slide 60: Phase 2 Primary Endpoint
Phase 2 Primary Endpoint Source: Stambler , B.S. et al.; Etripamil Nasal Spray for Rapid Conversion of Supraventricular Tachycardia to Sinus Rhythm; J Am Coll Cardiol . 2018;72(5):489 – 97 60 Etripamil three highest doses demonstrated 75 - 95% conversion rates, statistically significant compared to
Slide 61: Phase 2 Time to Conversion
Phase 2 Time to Conversion Source: Stambler , B.S. et al.; Etripamil Nasal Spray for Rapid Conversion of Supraventricular Tachycardia to Sinus Rhythm; J Am Coll Cardiol . 2018;72(5):489 – 97 61 Milestone Corporate Overview 70mg etripamil dose showed rapid time to conversion (median < 3 min) Kaplan M
Slide 62: Phase 2 Mean Systolic Blood Pressure Effects
Phase 2 Mean Systolic Blood Pressure Effects Source: Stambler , B.S. et al.; Etripamil Nasal Spray for Rapid Conversion of Supraventricular Tachycardia to Sinus Rhythm; J Am Coll Cardiol . 2018;72(5):489 – 97 62 Milestone Corporate Overview 1 2 1 Baseline is defined as the average of the 20 - min an
Slide 63: RAPID Detailed Slides
Milestone RAPID Detailed Slides
Slide 64: Phase 3 RAPID Clinical Study Design & Enrollment
▪ ECG of blinded event is adjudicated for being true PSVT (efficacy population) & PSVT termination time ▪ Primary Endpoint = PSVT conversion to NSR, Kaplan Meier analysis over 30 min ▪ Power : 90%, alpha < 0.05; delta = 19% in conversion rates between arms ▪ Study concluded when target met of 180 bl
Slide 65: RAPID Baseline Patient Demographics
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 Indian or Alaska native 0 1 (0.7) 1 (0.4) Asian 4 (3.3) 2 (1.5) 6
Slide 66: RAPID Efficacy
0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% 0 5 10 15 20 25 30 35 40 45 50 55 60 65 70 75 80 85 90 Kaplan Meier Probability of Conversion, (%) Time Since Study Drug Administration (minutes) RAPID Efficacy "+" symbol on graph indicates censoring for signal loss (n=4 over 90 minutes) Source: Milestone
Slide 67: RAPID Safety Analysis
RAPID Safety Analysis 1 Safety Population. 2 Three events were: Frequent PVCs and couplets after PSVT termination; non - sustained VT after PSVT termination; allergic reactio n, treated with oral Benadryl. TEAE timing – up to 24 hours following drug administration. TEAE = treatment - emergent advers
Slide 68: RAPID Safety - Adverse Events
RAPID Safety – Adverse Events Source: Milestone Pharmaceuticals Data on File0 69 Milestone Corporate Overview 1 Adverse events specifically acquired as adverse events of interest, as potentially representing lowered blood pressure. 2 Safety Population. 3 Six of 8 rated as mild, 2 of 8 rated as moder
Slide 69: Conversion Rates from PSVT to Normal Rhythm (all studies)
Conversion Rates from PSVT to Normal Rhythm (all studies) Source: Milestone Pharmaceuticals Data on File 70 Milestone Corporate Overview R&D Phase, Study Etripamil, 70 mg (% Conversion) Placebo (% Conversion) Hazard Ratio p value Statistical Analysis Notes Phase 3 RAPID 64 31 2.62 <0.001 Kaplan Meie
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