Company Overview

May 13, 2024

Forward Looking Statement

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,'' ''contemplate,'' ''continue,'' ''could,'' ''design,'' ''due,'' ''estimate,'' ''expect,'' ''goal,'' ''intend,'' ''may,'' ''objective,'' ''plan,'' ''predict,'' ''positioned,'' ''potential,'' ''project,'' ''seek,'' ''should,'' ''target,'' ''will,'' ''would'' (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward- looking statements are based on Milestone's expectations and assumptions as of the date of this Presentation. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this Presentation include statements regarding (i) the design, progress, timing, scope and results of the etripamil clinical trials in PSVT and AFib-RVR, (ii) the potential efficacy, safety and tolerability of etripamil, (iii) the potential of etripamil to deliver a clinically meaningful benefit to patients with PSVT in the home-setting environment and to empower patients to take control of their condition as well as provide value to the healthcare system, (iv) the timing of a potential NDA resubmission, (v) plans relating to commercializing etripamil, if approved, including the geographic areas of focus and sales strategy, (vi) the potential market size and the rate and degree of market acceptance of etripamil and any future product candidates and the implementation of Milestone's business model and strategic plans for its business, etripamil and any future product candidates (vii) Milestone's expected cash runway and (viii) potential royalty payments. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, but are not limited to, the risks inherent in biopharmaceutical product development and clinical trials, including the lengthy and uncertain regulatory approval process, uncertainties as to whether our NDA for etripamil will be approved by the FDA, uncertainties related to the timing of initiation, enrollment, completion and evaluation of clinical trials, including the RAPID and ReVeRA trials, and whether the clinical trials will validate the safety and efficacy of etripamil for PSVT, AFib-RVR, or other indications, among others, as well as risks related to pandemics and public health emergencies, including those related to COVID-19, and risks related the sufficiency of our capital resources and our ability to raise additional capital. These and other risks are set forth in Milestone's filings with the U.S. Securities and Exchange Commission, including in its annual report on Form 10-K for the year ended December 31, 2022, under the caption "Risk Factors", as such discussion may be updated in future filings we make with the SEC including in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, under the caption "Risk Factors". Except as required by law, Milestone assumes no obligation to update any forward- looking statements contained herein to reflect any change in expectations, even as new information becomes available.

This Presentation contains trademarks, trade names and service marks of other companies, which are the property of their respective owners. Certain information contained in this Presentation and statements made orally during this Presentation relate to or is based on studies, publications, surveys and other data obtained from third-party sources and Milestone's own internal estimates and research. While Milestone believes these third-party studies, publications, surveys and other data to be reliable as of the date of the Presentation, it has not independently verified, and makes no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, no independent sources has evaluated the reasonableness or accuracy of Milestone's internal estimates or research and no reliance should be made on any information or statements made in this Presentation relating to or based on such internal estimates and research.

Etripamil is an investigational new drug, which is not approved for commercial distribution in the United States.

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Milestone Pharmaceuticals:

Aspiring to Give Patients Control over Common Heart Conditions

Empowering Patients

Self-Treat Common Arrhythmias

  • CARDAMYSTTM (etripamil) nasal spray: investigational novel calcium channel blocker
  • Fast-acting,well-tolerated, portable, on-demand
  • Shift from Emergency Department to patient self-management

PSVT

Commercial Launch Preparation

  • NDA resubmission - Mar 2024
  • Type A meeting - Feb 2024
  • NDA RTF - Dec 2023
  • Successful Phase 3 - Published in The Lancet
  • Experienced leadership driving commercialization

AFib-RVR

Market Expansion Opportunity

  • End of Phase 2 Meeting expected mid-2024
  • FDA provides Phase 3 study guidance - 1Q 2024
  • Positive Phase 2 Study - ReVeRA - Published in Circulation AE
  • AHA Featured Science Presentation - Nov 2023

PSVT = Paroxysmal Supraventricular Tachycardia; AFib-RVR = Atrial Fibrillation with Rapid Ventricular Rate; NDA = New Drug Application; RTF = Refusal to File; AHA - American Heart Association. Citations: Stambler B et al, The Lancet (2023); Camm AJ et al, Circulation: Arrhythmia & Electrophysiology (2023)

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PSVT and AFib-RVR Cause Markedly Symptomatic Attacks That Disrupt Patients' Lives

Symptoms include…

Heart palpitations

Fatigue

Chest pressure or pain

Light-headedness

Shortness of breath

Anxiety

Many patients feel anxious and powerless

PSVT = Paroxysmal Supraventricular Tachycardia; AFib-RVR = Atrial Fibrillation with Rapid Ventricular Rate

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Current Treatment of Acute Attacks in the Emergency

Department are Burdensome and Costly

For many patients, physicians and payers:

  • Time-consuming,disruptive
  • Often results in a hospital admission
  • Expensive use of healthcare system resources

Need for simple, fast-acting treatment, reduce trips to ED and calls to physicians

ED = Emergency Department

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Etripamil Nasal Spray is a Novel L-type Investigational Calcium Channel Blocker Designed to Treat Episodes Quickly

Fast onset of action

Patient

Small enough to

(Tmax ≤ 7 min)

self-administered

fit in your pocket

Empowering patients to treat symptomatic attacks

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PSVT and AFib-RVR Populations in the US

PSVT

Atrial Fibrillation

Total Patients (2030)

2.6 Million3

10 Million1

Discharged ED Visits

145 Thousand

785 Thousand

& Hospital Admissions (2016)2

Target Addressable Market (2030)

1.0-1.6 Million5

~3-4 Million4

Patient Population

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 Electrophysiology. 2021 Aug; 32(8): 2199-2206. doi: 10.1111/jce.15109. Epub 2021 Jun 14. 2018 prevalence of 2M anticipated to grow at a CAGR of ~2% 4. Quantitative Survey conducted by Triangle Insights, May 2021, N=250 Clinical Cardiologists, Interventional Cardiologists, and Electrophysiologists. 5. Estimate of TAM (~40%- 60% of prevalence) based on internal PSVT patient market research (BluePrint Research Group, n=247) and longitudinal analysis of claims data.

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Current US PSVT Market

Total annual US healthcare expenditures of ~$3B

  • Prevalence ~2M patients diagnosed with PSVT
  • ~650K patients treated per year
    • ~300K newly diagnosed per year
    • >150K ED/hospital visits per year
    • ~80K ablations per year

Current Management

Ablations

12%

Pill-in-

No Treatment Pocket 34%

17%

Chronic Medications

Alone 37%

PSVT = Paroxysmal Supraventricular Tachycardia; ED = Emergency Department

Source: (1) Sacks, N.C. et al; Prevalence of Paroxysmal Supraventricular Tachycardia (PSVT) in the US in Patients Under 65 Years of Age; Abstract and Oral Presentation at the International Academy of Cardiology Annual Scientific Sessions 2018, 23rd World Congress on Heart Disease; Precision Xtract, Boston, MA, USA; and data-on-file from IBM Marketscan® Commercial Research Database (<65y) and the Medicare Limited Dataset (≥65y), with demographic, enrollment and claims data for commercially insured (Truven) and Medicare covered patients using PSVT code 427.0 or I47.1 for up to a 9-year interval between 2008 and 2016 inclusive; (2) Quantitative market research conducted by Triangle Insights Group (n=250 cardiologists), June-September 2020

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Core PSVT Market is Addressable Now*, with Large Potential for Expansion

Market

Initial Target

Market

Future Target

*if approved

~650k/year3

~1.4 Million2

~600-900k1

Diagnosed, Annually Treated Patients

Diagnosed, Not Annually Treated Patients

Undiagnosed Patients

2.1

Million

Sources: 1) assumes annual incidence rate for PSVT of ~300k from longitudinal claims analysis and the average time to diagnosis (currently 2-3 years) can be reduced to <6 months 2) Calculated as the difference between PSVT prevalence of 2.1M and annual treatment rate of ~650k from Truven MarketScan data, 2008-2016 analyzed by Precision Xtract, 2019 3) Estimated number of unique patients with annual claims for PSVT from Truven MarketScan data, 2008-2016 analyzed by Precision Xtract, 2019.

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Etripamil Has Substantial Potential Value for Stakeholder Groups If Approved

Patients - Empowerment

Physicians -

Payers - More Efficient

Dependable Tool

Use of Resources

Fast, reliable self-administration

Designed for patient self-

Novel and cost-effective

Less disruption, reliance on the

management

treatment

Emergency Department

Frees up physician time

Reduction in ED/hospital

Less fear over when the next

and office resources

admissions

event will occur

Trusted CCB mechanism

Sources: Internal market research, PSVT = Paroxysmal Supraventricular Tachycardia, CCB = Calcium Channel Blocker, ED = Emergency Department

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Disclaimer

Milestone Pharmaceuticals Inc. published this content on 13 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 22 May 2024 21:44:09 UTC.