Harmony Biosciences Holdings, Inc. · NASDAQ:HRMY
Harmony Biosciences — 43rd Annual JP Morgan Healthcare Conference (January 2025)
25 slides from Harmony Biosciences's conference presentation, Jan 13, 2025
Slide 1: 43rd Annual JP Morgan Healthcare Conference
Copyright © 2025 Harmony Biosciences. All rights reserved. 4 3 r d Annual JP Morgan Healthcare Conference JEFFREY M. DAYNO, MD January 15, 2025 | San Francisco
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in these materials or elsewhere, including statements regarding Harmony Bios
Slide 3: Innovative, Patient-Focused, Profitable Biotech, Catalyst-Rich Pipeline
INNOVATIVE PATIENT-FOCUSED PROFITABLE BIOTECH CATALYST-RICH PIPELINE
Slide 4: Key Catalysts
4 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference 4 Key Catalysts One every Q in 2025 $1B+ Potential opportunity for WAKIX in narcolepsy $3B+ Establishing leadership position in CNS 6 Phase 3 Development programs by year end 2025 MULTIPLE MILESTONES IN 2025 DURABLE LONG-TERM
Slide 5: Each Franchise With Potential Peak Sales of $1-2B
SLEEP/WAKE NEURO BEHAVIORAL EPILEPSY EACH FRANCHISE WITH POTENTIAL Peak Sales of $1-2B 5 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference
Slide 6: Innovative Late-Stage Pipeline
Innovative Late-Stage Pipeline 6 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference PRODUCT / INDICATION PRE-IND PHASE 1 PHASE 2 PHASE 3 REGULATORY FILING MARKETED PRODUCT Sleep/Wake WAKIX® EDS in Narcolepsy (Adults) Cataplexy in Narcolepsy (Adults) EDS in Narcolepsy (Pediatric
Slide 7: WAKIX Is One of the Most Successful Orphan/Rare Launches
WAKIX® Is One of the Most Successful Orphan/Rare Launches With Demonstrated Durable Revenue Generation 7 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference WAKIX Net Revenue Growth 2020–2024 $160 $305 $438 $582 $714 $0 $200 $400 $600 $800 2020 2021 2022 2023 2024 Net Revenue ($
Slide 8: 2025 Net Revenue Guidance
2025 Net Revenue Guidance 8 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference Chris Living with Narcolepsy KEY TAKEAWAY $820M-$860M NET REVENUE GUIDANCE WAKIX represents a potential $1B+ opportunity in narcolepsy alone
Slide 9: 2025 Anticipated Catalysts
2025 Anticipated Catalysts 9 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference 1Q25 Pitolisant IH sNDA FDA decision on file acceptance (potential approval in 2025) 2Q25 BP1.15205 (OX2R agonist) • Preclinical data presentation at SLEEP 2025 • IMPD submission (IND submission 2H)
Slide 10: Q1 Catalyst: Pitolisant for Idiopathic Hypersomnia
TOTALITY OF DATA FROM THE PHASE 3 INTUNE STUDY Open-label, randomized withdrawal and long-term extension data REAL WORLD AND CLINICAL EXPERIENCE DATA Data from compassionate use program and top KOL clinical experience FAVORABLE BENEFIT / RISK PROFILE Unmet medical need/unique safety profile VOICE OF
Slide 11: Strong and Durable Improvement in EDS in Patients With IH
0 2 4 6 8 10 12 14 16 18 Strong and Durable Improvement in EDS in Patients With IH (as Measured by ESS) 11 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference The mean ESS Score stayed within the normal level of wakefulness throughout the long-term extension period KEY TAKEAWAY
Slide 12: Percentage of Pitolisant Patients within Normal Level of Wakefulness
0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% Percentage of Pitolisant Patients within the Normal Level of Wakefulness 12 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference Pitolisant demonstrates high patient response rates in maintaining normal level of wakefulness observed thr
Slide 13: Q2 Catalyst: Potential Best-in-Class Orexin Receptor Agonist
Q2 Catalyst: Potential Best-in-Class Orexin 2 Receptor (OX2R) Agonist 13 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference TEIJIN - COLLABORATION WITH PROF. YANAGISAWA Tokyo-based Pharma; innovator of TPM-1116 (now BP1.15205) Discovered orexin receptors and implications on sle
Slide 14: BP1.15205: Most Potent OX2R Agonist
Jazz JZP441 Alkermes6 2680 Takeda4 TAK-994 Takeda5 TAK-925 Takeda4 TAK-861 Eisai3 E2086 Centessa2 ORX750 HRMY/BP1 BP1.15205 Select DMPK parameters 0.015 nM 0.11 nM 2.3 nM 2.5 nM 5.5 nM 19 nM Not reported Not reported Potency (hOX2R, EC50) > 600x 9800x > 2000x 3000x > 5000x Not reported Not reported
Slide 15: Orexin-2 Receptor Agonist: Distinct Chemical Structure and Properties
Orexin-2 Receptor Agonist: Distinct Chemical Structure with Unique Properties and Potential Clinical Benefits 15 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference Potential best-in-class OX2R agonist with possibility for broad clinical utility KEY TAKEAWAY Combination of high
Slide 16: Q3 Catalyst: ZYN002 for Fragile X Syndrome
Q3 Catalyst: ZYN002 for the Treatment of Fragile X Syndrome 16 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference Topline Data Readout from Pivotal Phase 3 RECONNECT Trial ZYN002: INNOVATIVE PRODUCT PROFILE Purely synthetic cannabidiol (CBD); devoid of THC LEAD PROGRAM IN FRAGI
Slide 17: Phase 3 CONNECT Trial: Completely Methylated Subgroup
Phase 2 CONNECT Trial1: Completely Methylated Subgroup 17 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference Key secondary outcomes SOCIAL INTERACTION 63% vs 37% (p=0.005*) IRRITABLE/DISRUPTIVE BEHAVIORS 54% vs 33% (p=0.027*) SOCIAL AVOIDANCE (≥ 3 POINTS) 56% vs 37% (p=0.030*)
Slide 18: Open-Label Extension Sustained Improvement in Complete Methylation Patients
Open-Label Extension Patients on ZYN002 Sustained Improvement in Patients With Complete Methylation of FMR1a 18 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference Change in ABC-CFXS Social Avoidance CONNECT-FX Double-Blindb RECONNECT Population Continued improvement through 4 m
Slide 19: Pivotal Phase 3 RECONNECT Trial Design
Pivotal Phase 3 RECONNECT Trial: Design Optimized from Phase 2 CONNECT-FX Trial 19 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference Successful completion of Phase 3 pivotal trial expected to satisfy requirements for an NDA submission in the U.S. and a marketing authorization
Slide 20: Q4 Catalyst: Pitolisant-HD in Narcolepsy
Q4 Catalyst: Pitolisant-HD in Narcolepsy 20 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference Initiation of pivotal Phase 3 trial of Pitolisant-HD in narcolepsy DESIGNED TO ADDRESS THE LARGEST UNMET MEDICAL NEED IN NARCOLEPSY • Providing greater efficacy for both excessive day
Slide 21: Pitolisant Franchise Poised to Drive Durable Growth to Mid-2040s
Pitolisant Franchise Poised to Drive Durable Patient and Revenue Growth to the Mid-2040s • Two meaningfully differentiated product profiles building off WAKIX with PDUFAs prior to LOE • Provisional patents filed out to 2044 to extend durable patient and net revenue growth • Pursuing other indication
Slide 22: EPX-100: Most Advanced 5HT2 Agonist Development Program
EPX-100: Most Advanced and Differentiated 5HT2 (serotonin) Agonist Development Program 22 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference EPX-100 https://pubchem.ncbi.nlm.nih.gov/compound/Clemizole PROVEN MoA Established serotonergic mechanism of action – confirmed via highl
Slide 23: Catalyst-Rich Pipeline Driving Value Beyond 2025
Catalyst-Rich Pipeline Driving Value Beyond 2025 23 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference 2026 • ZYN002 FXS PDUFA • Pitolisant-GR PDUFA • EPX100 DS/LGS Phase 3 topline data (TLD) • Pitolisant PWS Phase 3 TLD • OX2R SAD/MAD PK readouts 2027 – 2028 • Pitolisant-HD Ph
Slide 24: Key Catalysts
24 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference 4 Key Catalysts One every Q in 2025 $1B+ Potential opportunity for WAKIX in narcolepsy $3B+ Establishing leadership position in CNS 6 Phase 3 Development programs by year end 2025 MULTIPLE MILESTONES IN 2025 DURABLE LONG-TER
Slide 25: company/harmonybiosciences/ @harmonybio harmony_biosciences www.harmonybiosciences.com
company/harmonybiosciences/ @harmonybio harmony_biosciences www.harmonybiosciences.com
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