Form 8-K - Current report
February 25 2025 - 4:05PM
Edgar (US Regulatory)
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QC
0001408443
2025-02-25
2025-02-25
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
February 25, 2025
MILESTONE
PHARMACEUTICALS INC.
(Exact name of registrant as specified in its
charter)
Québec |
|
001-38899 |
|
Not
applicable |
(state or other jurisdiction of incorporation) |
|
(Commission File Number) |
|
(I.R.S. Employer Identification No.) |
1111
Dr. Frederik-Philips Boulevard, |
|
|
Suite
420 |
|
|
Montréal,
Québec CA |
|
H4M
2X6 |
(Address of principal executive offices) |
|
(Zip Code) |
Registrant's telephone number, including area
code: (514)
336-0444
(Former
name or former address, if changed since last report.)
Check the appropriate box below if the Form 8-K
filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General
Instruction A.2. below):
¨
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
¨ Soliciting
material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
¨
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
¨
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b)
of the Act:
Title of each class |
|
Trading Symbol(s) |
|
Name of each exchange on which
registered |
Common
Shares |
|
MIST |
|
The
Nasdaq Stock Market LLC |
Indicate by check mark
whether the registrant is an emerging growth company as defined in as defined in Rule 405 of the Securities Act of 1933 (§ 230.405
of this chapter) or Rule 12b–2 of the Securities Exchange Act of 1934 (§ 240.12b–2 of this chapter).
Emerging growth company
¨
If an emerging growth
company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or
revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 7.01. |
Regulation FD Disclosure. |
On February 25, 2025, Milestone Pharmaceuticals
Inc. (“Milestone” or the “Company”) provided a corporate presentation that may be used in connection with presentations
at conferences and investor meetings. The full text of the Company’s corporate presentation is filed as Exhibit 99.1 hereto, and
incorporated herein by reference, and may also be accessed through the “Investors & Media” section of the Company’s
website at www.milestonepharma.com.
The Company intends to
use its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation
FD. Such disclosures will be included on its website in the “Investors & Media” section. Accordingly, investors should
monitor such portions of its website, in addition to following press releases, filings with the U.S. Securities Exchange Commission (the
“SEC”) and public conference calls and webcasts.
The information furnished
under this Item 7.01, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange
Act of 1934, or subject to the liabilities of that section or Sections 11 and 12(a)(2) of the Securities Act of 1933, or the Securities
Act. The information in this Item 7.01, including Exhibit 99.1, shall not be deemed incorporated by reference into any other filing with
the SEC, made by the Company, whether made before or after the date hereof, regardless of any general incorporation language in such filing.
Item 9.01. |
Financial Statements and Exhibits |
(d) Exhibits.
SIGNATURES
Pursuant to the requirements of the Securities
Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
|
MILESTONE PHARMACEUTICALS INC. |
|
|
Date: February 25, 2025 |
By: |
/s/ Amit Hasija |
|
|
Amit Hasija |
|
|
Chief Financial Officer Principal Financial Officer |
Exhibit 99.1
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| Commercial Investor Event:
Preparation for Potential
Launch of CARDAMYST
(etripamil) Nasal Spray
Joe Oliveto, President & CEO
Lorenz Muller, CCO
Investigational Product. Not approved
for commercial use in any jurisdiction. |
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| Disclaimer
This presentation discusses our intended promotional launch strategy for
CARDAMYSTTM (etripamil) nasal spray, which has not yet been approved for
commercial use in any jurisdiction. Our new drug application (“NDA”) remains under
review by the U.S. Food and Drug Administration (“FDA”) with a Prescription Drug
User Fee Act (“PDUFA”) date of March 27, 2025. Any plans described herein remain
subject to FDA approval of the NDA. |
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| Forward Looking Statements
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 3
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 approval by the U.S. Food and Drug Administration of CARDAMYST on the anticipated timeline, or at all; (ii) the design, progress, timing, scope and results of the
etripamil clinical trial in AFib-RVR; (iii) the potential efficacy, safety and tolerability of etripamil for AFib-RVR; (iv) the potential of CARDAMYST to (a) deliver a new PSVT therapeutic option to
market, (b) decrease costs for the healthcare system and reduce emergency department visits and hospital admissions, (c) empower patients to treat symptomatic attacks; (v) plans relating to
commercializing CARDAMYST, if approved, including timing, the geographic areas of focus and sales strategy; (vi) the potential market size and the rate and degree of market acceptance of
CARDAMYST (etripamil) and any future product candidates; (vii) the projected use of CARDAMYST in the future; (viii) anticipated commercial and Medicare coverage of CARDAMYST; (ix) the
implementation of Milestone’s business model and strategic plans for its business, etripamil and any future product candidates; (x) Milestone’s expected cash runway; and (xi) 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; whether our future interactions with the FDA will have satisfactory outcomes; whether and when, if at all, our NDA for etripamil will be
approved by the FDA; uncertainties related to the timing of initiation, enrollment, completion, evaluation and results of our clinical trials; risks and uncertainty related to the complexity inherent
in cleaning, verifying and analyzing trial data; and whether the clinical trials will validate the safety and efficacy of etripamil for PSVT or other indications, among others, general economic,
political, and market conditions, including deteriorating market conditions due to investor concerns regarding inflation, international tariffs; Russian hostilities in Ukraine and ongoing disputes in
Israel and Gaza and overall fluctuations in the financial markets in the United States and abroad; risks related to pandemics and public health emergencies; and risks related the sufficiency of
Milestone's capital resources and its ability to raise additional capital in the current economic climate. These and other risks are set forth in Milestone's filings with the U.S. Securities and
Exchange Commission (“SEC”), 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. |
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| Agenda
4
Introduction and Overview
The Problem
Our Innovation
The Opportunity
Commercial Strategy and Execution
02
03
04
05
01
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 |
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| Our Mission
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 5
We are a patient-centric
biopharmaceutical company intending
to bring etripamil nasal spray to
patients living with paroxysmal
supraventricular tachycardia (PSVT)
and atrial fibrillation with rapid rate
(AFib-RVR) |
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| 2025 – A Potentially Transformative Year for Milestone
*Feb 2025 – US Patent Office issued notice of allowance for patent covering repeat dose regimen
Key: PDUFA = Prescription Drug User Fee Act /
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 6
• Potential approval of CARDAMYST, PDUFA March 27, 2025
• Promotional launch of CARDAMYST, expected mid-2025
• $75M payment expected upon approval from royalty financing
• Expected to fund operations into mid-2026, combined with current cash
• Commercial launch of CARDAMYST is first priority
• Patent estate potentially extended to 2042*
• Planned Phase 3 study start for etripamil in second indication
• AFib-RVR represents larger potential target population than PSVT
PSVT
AFib-RVR
Finances & Other |
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| Our Vision for Commercial Success of CARDAMYST
Key: PSVT = Paroxysmal Supraventricular Tachycardia
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 7
Patients
Empowerment
Prescribers
Provides a solution
Payers
Efficiency
GOAL
Easy to use Easy to prescribe Limit need to manage
Plan to Leverage Experience in PSVT to Launch Confidently |
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| Agenda
8
Introduction and Overview
The Problem
Our Innovation
The Opportunity
Commercial Strategy and Execution
02
03
04
05
01
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 |
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| Agenda
9
Introduction and Overview
The Problem
Our Innovation
The Opportunity
Commercial Strategy and Execution
02
03
04
05
01
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 |
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| PSVT Puts a Significant Burden on Patients
Paroxysmal Supraventricular Tachycardia is a common
symptomatic arrhythmia (abnormal heart rhythm) characterized
by attacks of very rapid heart rate
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 10
Disease Characteristics
• Chronic condition with symptomatic attacks
• Incidence and duration unpredictable
• Often anxiety-provoking
• 50% of patients are under 65
• 65% of patients are women
• Mostly managed by cardiologists
Patient impact
• Discomfort during attacks, symptoms
• Anxiety between attacks, unpredictability
• Per year >650,000 claims seeking treatment
― ~100,000 ablations
― >140,000 ED visits
― >40,000 hospitalizations
Key: ED = Emergency Department
Citations: Internal market research and longitudinal analysis of Truven/Marketscan and Medicare claims data; IQVIA Pharmetrics Plus 2019 Commercial claims for patients <65yo and Medicare
LDS 5% for patients >65yo (ICD: I47.1); Healthcare Utilization Project (HCUP) 2019, https://hcup-us.ahrq.gov/databases.jsp; accessed 11/2024; https://youtu.be/oqwNSC-Jkaw
Donnette from Huntsville AL |
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| Majority of Patients with PSVT have Anxiety and Reduced QoL
Both During and Between Episodes
Key: ED = Emergency Department. Citations: ADD REFERENCES
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 11
>80%
of all patients with PSVT
report some level of
anxiety, with the majority
having moderate or
Social and Lifestyle Limitations severe symptoms
Physical Symptoms and Emotional Distress
Fear of Recurrence and Generalized Anxiety
Concerns Health and Long-Term Outlook
Impact on Daily Functioning and Productivity
Key: QoL = Quality of Life
Citations: Internal Market Research |
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| Many Current Treatments in the Market are Inconvenient,
Intrusive, and/or Ineffective
Key: BB = Beta Blocker; CCB = Calcium Channel Blocker
Citations: 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
patients with supraventricular tachycardia: executive summary. Circulation. 2016;133:e471–e505
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 12
“Pill in Pocket” = off label oral CCB or BB,
or Vagal maneuver
• poor efficacy
• “do no harm”
Acute
ED, Urgent Care Visit or Hospitalization
• IV adenosine, IV CCB or DC cardioversion
• time consuming
• costly
• stressful
Most Patients Experience Attacks Despite Preventive Options
Oral BBs and CCBs
• reduce episodes
• only somewhat effective
• require daily administration
• unwanted side effects
Chronic / Preventive
Catheter ablation
• often curative
• risks associated with invasive procedure
• only elected by ~15% patients (mostly young) |
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| Underappreciated Journey of a Patient with PSVT:
Drawn-out Diagnoses & Difficult, Limited Treatment Options
Key: Rx = prescriptiont
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 13
Diagnosis
Off-label
Chronic Oral Rx
Off-label
“Pill in Pocket” Rx
No Treatment
Catheter Ablation
Attack of PSVT
Emergency
Department “Tough it out”
Very frequent
and/or severe
Not infrequent and
moderate/severe Infrequent or
mild |
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| PSVT Drives Significant Costs for the Healthcare System
Citations: 1. Milestone Pharmaceuticals. Data on file. 2. Chew DS, et al. Am Heart J. 2021;233:132-140. 3. Murman DH, et al. Acad Emerg Med. 2007;14(6):578-581. 4. Sacks NC et al. Am J Cardiol.
2020 Jan 15;125(2):215-221. 5. Jiang S et al. J Manag Care Spec Pharm. 2022 Nov;28(11):1321-1330. 6. Deshmukh A et al. Heart Rhythm O2. 2022 7. Data on file (HCUP NIS 2019 and CMS
hospitalization payments, 2021
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 14
>$5B Total annual US spending for patients with PSVT1
>140K Emergency department visits per year due to PSVT1
patients who present to ED
are hospitalized3
Costs post-diagnosis are primarily driven by2:
Inpatient services Diagnostic testing Ablation procedures
~25% $2.5k $15k
average cost of
ED visit4,5
average cost of
hospitalization4.6,7 |
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| Agenda
15
Introduction and Overview
The Problem
Our Innovation
The Opportunity
Commercial Strategy and Execution
02
03
04
05
01
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 |
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| CARDAMYST is a Novel Investigational CCB Designed to Treat
Episodes of PSVT Safely and Quickly
NCE
Short-acting CCB
MOA very well understood and trusted
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 16
Fast onset of action, well tolerated
Convenient patient self-administered nasal spray
Portable, on demand
Key: NCE = New Chemical Entity; CCB = Calcium Channel Blocker; MOA = Mechanism of Action
Empowering patients to treat symptomatic attacks |
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| CARDAMYST Would Put Patients in Control of Their PSVT
Sources: Internal market research,
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 17
Efficacy
Fast onset delivers quick relief from symptoms of PSVT
Convenience
On-demand use reduces reliance on healthcare system
Safety
Safety and tolerability supports confident use at home
Cost
Cost effective compared with an ED visit/hospitalization
Quiet Mind
Reduce worry between episodes
with versatile on-demand treatment
Active Living
Allow a return to normal rhythm
Patient Empowerment
Empower in disease management
& avoid unneeded healthcare visits
Rapid & Reliable Relief
Provide meaningful relief from
episode symptoms and duration
Investigational Product. Not
approved for commercial
use in any jurisdiction |
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| Fast Relief to More Patients
Efficacy Data from RAPID
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 18
Key: CI = confidence interval; HR = hazard ratio; SR = sinus rhythm
1. Stambler BS, et al. Lancet. 2023;402(10396):118-128.
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
Time since study drug administration (minutes)
Placebo
31%
Etripamil
64%
Placebo 85 78 67 64 60 58 56 53 49 45 44 40 37 37 36 36 35 32
Etripamil 99 79 67 55 45 40 35 33 31 29 28 27 26 25 23 22 20 19
35
19
Number of subjects at risk
61%
81%
Conversion of PSVT to SR at 30 min: HR = 2.62 (95% CI: 1.659, 4.147); P<0.001
55%
73%
More Converted
>2x patients converted to SR
within 30 minutes of taking
CARDAMYST vs doing nothing
Kaplan-Meier probability of conversion, (%)
Faster Conversion
>3x faster conversion of PSVT to SR
with CARDAMYST vs doing nothing
Fewer ED Visits
~40% reduction in ED use for
CARDAMYST vs doing nothing |
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| Well-Tolerated with a Favorable Safety Profile
Safety Data from RAPID
1. Randomized-period treatment-emergent adverse events, those ≥5% or those specifically tracked as potentially representing lowered blood pressure. 2. Safety Population. 3. Six of eight rated
as mild, two of eight rated as moderate, zero needing intervention.
Key: AV = Atrioventricular
Citations: American Heart Association Scientific Sessions, Late-Breaking Clinical Trial Presentation, November 2022; and The Lancet (2023).
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 19
Subject-reported AEs from the RAPID Study,1 n (%)
Placebo2
n=120
Etripamil2
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
Adverse Events were predominantly mediated by the route of administration
No syncope, pre-syncope, or 2nd/3rd degree AV block |
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| Our Vision for Commercial Success of CARDAMYST
Key: HCP = Healthcare Provider
Citations: Internal market research
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 20
Patients
Empowerment
Fast, reliable self-administration
Less disruption, reliance on ED
Less fear over when the next
event will occur
Prescribers
Provides a solution
Trusted CCB mechanism
Evidence-based new tool
Frees up HCP time and office
resources
Novel and cost-effective
treatment
Potential to reduce ED visits/
hospital admissions
Payers
Efficiency
Goal
Easy to use Easy to prescribe Limit need to manage |
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| Payers Recognize the Value of CARDAMYST to Fill an Unmet
Need for Cardiology HCPs
Citations: Internal Market Research
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 21
Given rational pricing,
not expected to be
“budget-impacting”
enough to warrant
active management
Minimal market
competition less
likely to drive large
rebate pressure
See potential for efficacy
endpoints to translate
into reductions in
healthcare resource
utilization
Unmet Need
Payers report they will
defer to cardiologists
to interpret clinical
unmet need |
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| Agenda
22
Introduction and Overview
The Problem
Our Innovation
The Opportunity
Commercial Strategy and Execution
02
03
04
05
01
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 |
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| 2M+ Patients Cost the Healthcare System at Least $5 Billion Annually
Key: PSVT = Paroxysmal Supraventricular Tachycardia
Citations: 1. Rehorn M, et al. J Cardiovasc Electrophysiol. 2021 Aug;32(8):2199-2206. 2. IQVIA Pharmetrics Plus 2019 Commercial claims for patients <65yo and Medicare LDS 5% for patients >65yo
(ICD: I47.1) 3. Healthcare Utilization Project (HCUP) 2019, https://hcup-us.ahrq.gov/databases.jsp; accessed 11/2024; range reflects ED visits/IP admissions with PSVT as primary diagnosis code to
ED visits/IP admissions with PSVT in any diagnostic position.
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 23
140,000 – 525,000 Emergency
Department Visits per Year3
~100,000 Ablations
Performed per Year2
650,000 to 1M Patients
Treated per Year2
40,000 – 120,000 In-patient
Admissions per Year3
~2M+
Patients
Diagnosed with
PSVT1 |
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| 3-5 times per year
Most Patients with a Diagnosis of PSVT Over Time Could Use
CARDAMYST Multiple Times per Year
Citations:. 1. Stambler BS, et al. Lancet 2023 Jul 8;402(10396):118-128.; Stambler BS. et al. CIRCEP. 2022 Dec;15(12):e010915. 2. Ip JE et al. JACC. 2024 May 21;83(20):2032-2034.; Ip JE et al. JAHA.
2023 Oct 3;12(19):e028227. 3. PSVT patient longitudinal study (n=247 US & UK), conducted by Blueprint Research Group, 2019-2020. 4. Quantitative market research (n=250 clinical cardiologists)
conducted by Triangle Insights Group, 2020-2021.
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 24
Source Reported Frequency of
use of CARDAMYST/yr
Randomized Clinical Studies1 3-9
Open Label Studies2 2-4
Observational Study3 4-6
Market Research4 3-4
Projected use of CARDAMYST:
~1 per year
2025
~1-2 per year
2026
~3-5 per year
2027+ |
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| Patients with PSVT are Primarily Treated by Cardiologists
in the Outpatient Setting
Citations: Internal Market Research
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 25
Clinical Cardiologists Primary Care
Physicians
Electrophysiologists
10%
30% 60% |
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| Versatility of CARDAMYST Leads to Multiple Use Cases
Citations: Internal Market Research
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 26
Diagnosis
Off-label
Chronic Oral Rx
Off-label
“Pill in Pocket” Rx
No Treatment
Catheter Ablation
← Add
← Switch
← Add
← Switch
← Add
← Bridge
← Alternative
CARDAMYST
Attack of PSVT
Emergency
Department “Tough it out”
Very frequent
and/or severe
Not infrequent and
moderate/severe Infrequent or
mild |
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| Cardiologists Expect to Prescribe CARDAMYST to the
Majority of Unablated Patients with PSVT
Citation: 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, 2008-2016 analyzed by Precision Xtract, 2019
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 27
Cardiologists’ Stated Adoption
of CARDAMYST per Segment
Current
Management of PSVT
Pill-in-Pocket 64%
Chronic Meds Alone 43%
No Current Treatment 53%
Impact of Expected Cardiologist
Adoption of CARDAMYST
Pill-in-Pocket 34%
Chronic Medications
Alone 37%
No
Treatment
17%
Ablation
12%
CARDAMYST
46%
12%
21%
8%
12% |
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| Electrophysiologists Have an Important Role to Play
with CARDAMYST
Key: KOL = Key Opinion Leader; P&T = Pharmacy & Therapeutics; PSVT = Paroxysmal Supraventricular Tachycardia; AFib-RVR = Atrial Fibrillation with Rapid Ventricular Rate; EPs = Electrophysiologists
Citations: Internal Market Research
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 28
• KOLs in arrhythmias respected for their input on P&T and guidelines
• Perform ~100,000 cardiac ablations annually in the U.S.
• Treat ~10% of patients with PSVT
Bridge to ablation
between consultation
and procedure (33%)
Alternative to ablation
for patients hesitant
about procedure (24%)
EPs report willingness to use CARDAMYST in majority of cases1 |
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| Accessible and Compelling Commercial Opportunity
for CARDAMYST
Key: MOA = Mechanism of Action; HRU = Healthcare Resource Utilization
Citations: Internal Market Research and Longitudinal Analysis of Truven/Marketscan and Medicare claims data
No anticipated
branded competition
Low barrier
to prescribing
Opportunity for early
demand generation
and quality coverage
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 29
~650k patients treated
annually
~50% commercially-insured patients
Cost offsets driven by
lower HRU
Familiar and trusted
MOA
Evidence-based
100% share of voice
Lower rebate
pressure |
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| Agenda
30
Introduction and Overview
The Problem
Our Innovation
The Opportunity
Commercial Strategy and Execution
02
03
04
05
01
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 |
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| Fully Integrated and Experienced Launch Leadership Team
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 31
Lorenz Muller
Chief Commercial Officer
Jeff Moore
VP of Sales
Anita Holtz MSN, CRNP, CMD
VP Head of Medical Affairs
Roshan Girglani
VP of Marketing
Joseph Oliveto
President and CEO
John Jackimiec
VP of Market Access
David Bharucha, MD PhD
Chief Medical Officer |
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| Go-to-Market Model for CARDAMYST
Balancing Need to Generate Demand with Managing Commercial Burn
Key: HCPs = Healthcare Providers
Citations: Adopted from Sandberg Orne, L. et al; “Empowering the Next-Generation Launch Model”; Trinity White Paper, June 2022. Data on File (Symphony Open Claims,
Veeva Patient Compass, 2021-2024).
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 32 Resourcing and Infrastructure Build
L-1+ Years Pre-Approval Launch Launch +3 Years
Phase 1 Prioritizing
Awareness and Access
Launch +2 Years
TARGETS
Planned Phased Commercial Launch Plan
KOL Engagement
Payer Profiling
Launch +1 Year
Phase 2 Fit-for-Purpose
Field Deployment
10-12k HCPs
Commercial
Access
High-value
Prescribers
Phase 3 Expand as You Go
17-22k HCPs 25-30k HCPs
Expand Sales
Force
Medicare
Access
IDNs |
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| Plans to Deliver Broad Quality Payer Coverage
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 33
Citations: Internal market research.
Patients
Accessible and
Affordable
Coverage with Low
Need to Control
Payers
Not Onerous to
Prescribe
Healthcare
Providers
Available via local pharmacy
Reasonable out of pocket cost
Limit need for paperwork
or authorizations
Net pricing below specialty tier
Protection from overuse
Commercial expected before
Medicare |
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| Strong Value Proposition for CARDAMYST for Payers Can Lead to
Broad Coverage without Onerous Restrictions for HCPs or Patients
Key: U/M = Utilization Management; PA = Prior Authorization; I/E = Inclusion/ Exclusion; IDNs = Integrated Delivery Networks
Citations: CRA Pricing Research 6/2022
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 34
Commercial Coverage
Goal: Nominal access rebates with patient copay
support provided to reduce out-of-pocket cost
burden
Medicare Coverage
Goal: Rebate to specialty Tier threshold to avoid
excessive patient out of pocket
• PA to label
• Reasonable quantity
limits
• Specialty prescribing
Utilization Management Priorities for Coverage
Commercial
2025 2026 2027
Medicare
Gov’t Other
IDNs |
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| Key Elements of Launch Timeline for CARDAMYST
Potential FDA approval expected March 27
Medical Information Center live
Patient support services live
Product available for order by pharmacies
Market Access reimbursement support live
Field Medical engaging with KOLs
Sales reps generating demand mid-2025
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 35
Key anticipated
milestones for launch
of CARDAMYST
Key: FDA = Food and Drug Administration; KOLs = Key Opinion Leaders |
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| Priorities for Launching CARDAMYST
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 36
Awareness and
Demand Generation
Sales Leadership
Sales Force Territories
Sales Rep Hiring following potential approval
Promotional Launch mid-2025
Access and HCP
Experience
Distribution Contracts
Account Managers Profiling Payers
Fit-for-Purpose Reimbursement Support Programs
Patient Experience and
Affordability
Patient Support Services
Enable Contracting for Target Coverage
Copay Mitigation (Commercial) |
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| Factors Supporting Planned Launch Year Uptake of CARDAMYST
Citations: Internal Market Research
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 37
• Initial sales force covers 50% of potential
• Patients seeking treatment weekly
• HCP familiarity and evidence-based approach
• Accessibility through retail distribution
• Fit-for-purpose HCP and patient support programs
• No anticipated branded competition
• Healthcare cost offsets
Scripts
Written
Scripts
Filled |
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| Key Brand Performance Indicators for Planned Launch Year
• New to Brand Prescriptions (NBRx)
• Prescription Growth (week over week)
*Acceptable coverage includes, for example, absence of step edits and reasonable prior authorizations / quantity limits
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 38
• % Target Lives Covered (emphasis on commercial)
• % of Targets with “Acceptable” Coverage*
Drive HCP Awareness
& Adoption
Establish & Maintain
Broad, High-Quality
Access & Affordability
Strategic Objective: |
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| Anticipated Commercial Strategy
Demonstrate Demand and Expand
Key: NDA = New Drug Application; RWE = Real World Evidence
FOUNDATION
• NDA submission & review
• Pubs & scientific presentations
• KOL engagement
• RWE generation and payer engagement
• Commercial leadership in place
POTENTIAL LAUNCH
• FDA approval
• Retail distribution
• Staged field force deployment
• Patient support services
• Focus on commercial coverage
EXPAND PROMOTION
• Expand field force
• Initiate patient activation
• Engage peer-to-peer
promotion
• Expand access to government
payers and IDNs
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 39
2024
2025
2026 |
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| Key Takeaways for Investment Thesis
• Milestone offers near-term commercial and mid-term clinical development catalysts
to investors
• PSVT is a symptomatic and unpredictable condition without good patient-driven
treatment options
• PSVT affects 2M+ Americans and costs the healthcare system over $5B per year
• CARDAMYST, if approved, will be the first and only FDA-approved on-demand
therapy for patients with PSVT
• Milestone is working to implement a cost-efficient launch strategy designed to:
― drive near-term demand;
― have a reasonable time to breakeven; and
― offer substantial opportunities for growth and expansion
Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 40 |
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| Commercial Investor Event: Preparation for Potential Launch of CARDAMYST │ February 25, 2025 41
Guy Rousseau, PhD
SVP Regulatory Affairs &
Quality Management
Jeff Nelson
Chief Operating Officer
Amit Hasija
Chief Financial Officer,
EVP Corporate Development
Q&A
Lorenz Muller
Chief Commercial Officer
Joseph Oliveto
President and CEO
David Bharucha, MD PhD
Chief Medical Officer |
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| Thank you |
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