Company Overview
June 6, 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 CARDAMYST for PSVT and etripamil for AFib-RVR, (iii) the potential of CARDAMYST to deliver a new PSVT therapeutic option to market, (iv) plans relating to commercializing CARDAMYST, if approved, including the geographic areas of focus and sales strategy, (v) the potential market size and the rate and degree of market acceptance of CARDAMYST (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 (vi) Milestone's expected cash runway and (vii) 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 filing for CARDAMYST 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 CARDAMYST for PSVT, etripamil for 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, 2023, under the caption "Risk Factors", as such discussion may be updated in future filings we make with the SEC. 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.
CARDAMYSTTM (etripamil) nasal spray is an investigational new drug, which is not approved for commercial distribution in the United States.
Milestone Corporate Overview | 2 |
Milestone Pharmaceuticals:
Aspiring to Give Patients Control over Common Heart Conditions
Empowering Patients | PSVT | AFib-RVR | |||||
• | Self-Treat Common Arrhythmias | • | Commercial Launch Preparation | • | Market Expansion Opportunity | ||
CARDAMYSTTM (etripamil) | NDA accepted May 2024, | End of Phase 2 Meeting | |||||
nasal spray: investigational | PDUFA action date Mar 2025 | expected mid-2024 | |||||
novel calcium channel | • | Successful Phase 3 -Published | • | FDA Phase 3 study | |||
blocker | |||||||
in The Lancet | guidance - 1Q 2024 | ||||||
• | Fast-acting,well-tolerated, | • | Experienced leadership | • | Positive Phase 2 ReVeRA | ||
portable, on-demand | |||||||
driving commercialization | Study - AHA Presentation | ||||||
• | Shift from Emergency | and published in Circulation | |||||
Department to patient | AE - Nov 2023 | ||||||
self-management | |||||||
PSVT = Paroxysmal Supraventricular Tachycardia; AFib-RVR = Atrial Fibrillation with Rapid Ventricular Rate; NDA = New Drug Application; AHA - American Heart Association. Citations: Stambler B et al, The Lancet (2023); Camm AJ et al, Circulation: Arrhythmia & Electrophysiology (2023)
Milestone Corporate Overview | 3 |
Atrial Arrhythmias with a Common Patient Burden & Cardiac Target
BURDEN: Patients with PSVT & AFib-RVR report
marked, often-disabling symptoms
PSVT | AFib-RVR |
(AVNRT and AVRT) | (a subset of AFib) |
Regular rapid heart rate | Irregular rapid heart rate |
Commonly 150 - 250 bpm | Commonly 100 - 175 bpm |
Episode frequency and duration is highly variable; occurring without warning
Common | Heart palpitations | Chest pressure or pain |
Symptoms | Shortness of breath | Fatigue |
Include | Light-headedness | Anxiety / Loss-of-control |
PSVT = Paroxysmal Supraventricular Tachycardia; AFib-RVR = Atrial Fibrillation with Rapid Ventricular Rate Sources: adapted from https://en.ecgpedia.org/index.php?title=Supraventricular_Rhythms, accessed 2/2021
TARGET: PSVT & AFib-RVR
Rely on AV Nodal Properties
Atrial Fibrillation
(multiple atrial
wavelets)
SA Node
AV Node
AV Nodal | AV Reentrant |
Reentrant Tachycardia | Tachycardia |
(AVNRT) | (AVRT) |
Milestone Corporate Overview | 4 |
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
Milestone Corporate Overview | 5 |
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.
Milestone Corporate Overview | 6 |
CARDAMYST 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
Milestone Corporate Overview | 7 |
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
Milestone Corporate Overview | 8 |
Core PSVT Market is Addressable Now*, with Large Potential for Expansion
Initial Target | Market |
Future Target | Market |
*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.
Milestone Corporate Overview | 9 |
CARDAMYST 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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Milestone Pharmaceuticals Inc. published this content on 06 June 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 06 June 2024 11:17:01 UTC.