Slides
Page 1
Copyright © 2024 Harmony Biosciences. All rights reserved. Q3 2025 Financial Results & Business Update November 4, 2025
Page 2
Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our full year 2024 net product revenue, expectations for the growth and value of WAKIX, plans to submit an sNDA for pitolisant in idiopathic hypersomnia; our future results of operations and financial position, business strategy, products, prospective products, prod uct approvals, the plans and objectives of management for future operations and future results of anticipated products. These statements are neither p romises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements , including, but not limited to, the following: our commercialization efforts and strategy for WAKIX; the rate and degree of market acceptance and clinical utility of pitolisant in additional indications, if approved, and any other product candidates we may develop or acquire, if approved; our research and development plans, including our plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned cli nical trials; our ability to expand the scope of our license agreements with Bioprojet Société Civile de Recherche (“Bioprojet”); the availability of favorable insu rance coverage and reimbursement for WAKIX; the timing of, and our ability to obtain, regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and additional financing needs; our abilit y to identify, acquire and integrate additional products or product candidates with significant commercial potential that are consistent with our commercia l objectives; our commercialization, marketing and manufacturing capabilities and strategy; significant competition in our industry; our intellect ual property position; loss or retirement of key members of management; failure to successfully execute our growth strategy, including any delays in our planned future growth; our failure to maintain effective internal controls; the impact of government laws and regulations; volatility and fluctuations in the price of our common stock; the significant costs and required management time as a result of operating as a public company; the fact that the pri ce of Harmony's common stock may be volatile and fluctuate substantially; statements related to our intended share repurchases and repurchase timefr ame and the significant costs and required management time as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC") on February 25, 2 025, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward -looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. 2 Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.
Page 3
WAKIX® APPROACHING BLOCKBUSTER STATUS UP TO 5 PHASE 3 REGISTRATIONAL TRIALS BY YEAR-END PROFITABLE, SELF-FUNDING BIOTECH POISED TO ACCELERATE GROWTH STRATEGY UNIQUE COMPANY PROFILE Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.
Page 4
WAKIX®: Q3 2025 Strong Revenue Growth Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.4 29% $186.0 $239.5 Q3 2024 Q3 2025 Year-Over-Year Revenue ($M) DOUBLE-DIGIT REVENUE GROWTH in Year 6 on the Market RECENTLY RAISED 2025 FULL YEAR GUIDANCE TO $845M - $865M WAKIX® Rapidly Approaching Blockbuster StatusKEY TAKEAWAY
Page 5
Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.5 Differentiated Product and Strong Execution Drives Growth Q3 2025 HIGHLIGHTS • ~500 average patient adds represents highest quarterly increase since launch • 8,100 average patients on WAKIX • Highly differentiated product – only non- scheduled treatment option • Growing depth & breadth of prescribers • Strong payer coverage of >80% of lives • Sharpened sales execution, messaging and patient support activities 400 300 300 100 400 300 300 200 350 350 350 150 250 250 300 100 400 500 0 1,000 2,000 3,000 4,000 5,000 6,000 7,000 8,000 9,000 Q2'21 Q3'21 Q4'21 Q1'22 Q2'22 Q3'22 Q4'22 Q1'23 Q2'23 Q3'23 Q4'23 Q1'24 Q2'24 Q3'24 Q4'24 Q1'25 Q2'25 Q3'25 Average # of Patients Net Patient Additions Highest Quarterly Increase in Average Patients Since LaunchKEY TAKEAWAY Average Patients on WAKIX
Page 6
Innovative Late-Stage Pipeline Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.6 PRODUCT / INDICATION DISCOVERY PHASE PRECLINICAL 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) Pitolisant Prader-Willi Syndrome (PWS) Myotonic Dystrophy (DM1) Pitolisant Gastro-Resistant (GR) Pitolisant High-Dose (HD) BP1.15205 (Orexin-2 Receptor Agonist) Sleep/Wake Disorders CBS105^ Treatment-Resistant Narcolepsy HBS-102 PWS Neurobehavioral ZYN002 (Cannabidiol Gel) Fragile X Syndrome (FXS) 22q11.2 Deletion Syndrome (22q) Rare Epilepsy EPX-100 (Clemizole Hydrochloride) Dravet Syndrome (DS) Lennox-Gastaut Syndrome (LGS) EPX-200 (Lorcaserin) Developmental and Epileptic Encephalopathies CBS104^ Refractory Epilepsy 3 CNS FRANCHISES 13 DEVELOPMENT PROGRAMS* 8 ASSETS 5 PHASE 3 PROGRAMS BY YEAR END *Includes additional ongoing clinical and regulatory programs; ^Research collaboration with CiRC Biosciences.
Page 7
Extending Our Leadership Position • Pitolisant HD: IND submission completed; to initiate Phase 3 registrational trials in narcolepsy and IH Q4 2025; utility patents filed with potential protection until 2044 • Pitolisant GR: Pivotal BE study topline data readout Q4 2025; Dosing optimization study completed with positive topline data; utility patents filed with potential protection until 2044 • BP1.15205: potential best-in-class orexin-2 agonist; FIH study to commence Q4 2025, clinical data anticipated 2026 Ongoing Review of RECONNECT Phase 3 Data • The ZYN002 phase 3 RECONNECT study in Fragile X syndrome did not meet the primary endpoint of improvement in social avoidance primarily due to a higher-than-expected placebo response rate; a review of the full data set is ongoing • The ZYN002 development program in 22q11.2 deletion syndrome (22q) has been paused pending the full review of the RECONNECT data One of the Most Advanced 5-HT2 Development Program • EPX-100: Phase 3 registrational trials ongoing in Dravet syndrome (ARGUS study) & Lennox-Gastaut syndrome (LIGHTHOUSE study) o Presenting efficacy, safety and tolerability data from the ARGUS open label extension study at the American Epilepsy Society Meeting in December o Topline data anticipated in 2026 from the ARGUS and the LIGHTHOUSE studies Advancement of late-stage, catalyst-rich pipeline SLEEP/WAKE NEURO BEHAVIORAL EPILEPSY Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.
Page 8
Pitolisant HD: Phase 3 Registrational Trials in Narcolepsy & IH – Q4 2025 Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.8 Q4 2025 Initiation of Phase 3 Registrational Trials of Pitolisant HD • Narcolepsy • IH ENHANCED FORMULATION WITH OPTIMIZED PK PROFILE AND HIGHER DOSE Designed to address the need for greater efficacy in excessive daytime sleepiness (EDS) in patients with central disorders of hypersomnolence PROGRAMS TO PURSUE A DIFFERENTIATED LABEL Fatigue in narcolepsy; sleep inertia in IH IND SUBMITTED; NARCOLEPSY AND IH PHASE 3 REGISTRATIONAL TRIALS TO BE INITIATED Q4 2025 Topline data readouts anticipated 2027; PDUFA dates targeted for 2028 UTILITY PATENTS FILED TO EXTEND PITOLISANT FRANCHISE INTO 2040s
Page 9
Pitolisant GR: Fast To Market Strategy Designed to Demonstrate Bioequivalence to WAKIX® Formulation 9 Q1 2027 Target PDUFA Date PIVOTAL BIOEQUIVALENCE STUDY COMPLETED Topline Data in Q4 2025 DOSING OPTIMIZATION STUDY COMPLETED 100% of the patients (46/46) able to initiate pitolisant GR at the therapeutic dose,17.8mg, without titration; No safety or tolerability issues reported NDA SUBMISSION EARLY 2026 TARGET PDUFA Q1 2027 Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.
Page 10
ZYN002: Pharmaceutically Manufactured Synthetic Cannabinoid Gel in FXS Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.10 ZYN002: INNOVATIVE PRODUCT PROFILE 100% synthetic, pharmaceutically manufactured cannabidiol (CBD), devoid of THC, in a patent-protected, permeation-enhanced gel formulation RECENTLY COMPLETED PHASE 3 RECONNECT STUDY RECONNECT phase 3 study in FXS did not meet the primary endpoint of improvement in social avoidance primarily due to a higher-than-expected placebo response rate; a review of the full data set is ongoing • The ZYN002 development program in 22q11.2 deletion syndrome (22q) has been paused pending the full review of the RECONNECT data MARKET OPPORTUNITY ~80,000 patients in the US with FXS; worldwide rights VERY HIGH UNMET NEED No approved products for FXS Data Review ongoing: RECONNECT Phase 3 Trial
Page 11
EPX-100: One of Most Advanced 5-HT2 (Serotonin) Agonist Programs in DEEs Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.11 2026 Anticipate Topline Data from Ongoing Global Phase 3 Trials • DS • LGS ESTABLISHED 5-HT2 (SEROTONIN) AGONIST MECHANISM OF ACTION MoA validated via the zebrafish model PHASE 3 STUDIES IN DS AND LGS Recruitment ongoing for Phase 3 registrational trials in patients with Dravet syndrome (ARGUS study) and Lennox-Gastaut syndrome (LIGHTHOUSE study) • Anticipate topline data in 2026 SAFETY: POTENTIAL TO OFFER A VERY UNIQUE RISK/BENEFIT PROPOSITION No additional laboratory or special safety monitoring BID DOSING REGIMEN Convenient for patients and caregivers
Page 12
Catalyst-Rich Pipeline Driving Value Beyond 2025 Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved..12 Pipeline Poised to Deliver Multiple New Product or Indication Launches Over the Next Several Years KEY TAKEAWAY Q4 2025 Pitolisant HD Initiation of Phase 3 registrational trials in narcolepsy and IH Pitolisant GR Pivotal BE topline data 2026 Pitolisant PWS Phase 3 TLD EPX-100 DS/LGS Phase 3 topline data (TLD) OX2R Phase 1 clinical PK data 2027 – 2028 Pitolisant GR PDUFA (2027) Pitolisant-HD Phase 3 TLD in narcolepsy and IH (2027) Pitolisant-HD PDUFA narcolepsy and IH (2028) EPX-100 DS/LGS PDUFA Pitolisant PWS PDUFA
Page 13
Financial Highlights Q3 2025 13 1. Non-GAAP Adjusted Net Income= GAAP Net Income excluding non-cash interest expense, depreciation, amortization, stock-based compensation, and tax effect of these items. Three Months Ended September 30, 2025 (In millions, USD) $186.0 $239.5 Q3 2024 Q3 2025 Net Product Revenue Non-GAAP Adjusted Net Income1 Cash, Cash Equivalents & Investments 11% $57.3 $63.5 Q3 2024 Q3 2025 29% $504.7 $576.1 $610.2 $672.3 $778.4 Q3 2024 Q4 2024 Q1 2025 Q2 2025 Q3 2025 +$106 UNIQUE COMPANY PROFILE 29% REVENUE GROWTH Year 6 on the market 2025 FULL YEAR REVENUE GUIDANCE $845M-$865M STRONG PROFITABILITY 4+ Years SIGNIFICANT CASH GENERATION $778M+ Cash, Cash Equivalents and Investments Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.
Page 14
Financial Summary Q3 2025 Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.14 Totals may not foot due to rounding Three Months Ended September 30, % Change Nine Months Ended September 30, % Change2025 2024 2025 2024 Net Product Revenue $239.5 $186.0 29% $624.7 $513.5 22% Cost of Product Sold 59.7 42.8 39% 129.8 102.4 27% TotalOperating Expenses $114.3 $81.6 40% $325.0 $276.0 18% R&D Expense 55.0 25.4 117% 139.7 111.2 26% S&M Expense 29.5 27.6 7% 90.3 83.3 8% G&A Expense 29.8 28.6 4% 95.0 81.5 17% Net Income $50.9 $46.1 10% $136.2 $96.0 42% Cash, cash equivalents & investments $778.4 $504.7 54% (In millions, USD)
Page 15
GAAP vs NON-GAAP Reconciliation Q3 2025 Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.15 (In millions, USD) Totals may not foot due to rounding Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 GAAP net income1 $50.9 $46.1 $136.2 $96.0 Non-cash interest expense2 0.2 0.2 0.5 0.5 Depreciation 0.0 0.1 0.0 0.3 Amortization3 6.0 6.0 17.9 17.9 Stock-based compensation expense 10.8 11.4 34.7 32.8 Income tax effect related to Non-GAAP adjustments4 (4.3) (6.4) (11.6) (15.0) Non-GAAP adjusted net income1 $63.5 $57.3 $177.7 $132.5 GAAP net income per diluted share $0.87 $0.79 $2.32 $1.66 Non-GAAP adjusted net income per diluted share $1.08 $0.99 $3.02 $2.29 Weighted average number of shares of common stock used in non-GAAP diluted per share 58.7 58.1 58.7 57.8 (1) Includes a $15.0 million IPR&D charge related to a clinical milestone achieved for ZYN002 during the three and nine months ended September 30, 2025. Includes a $15.0 million IPR&D charge related to an upfront fee incurred upon closing the CiRC research collaboration agreement for the nine months ended September 30, 2025. Includes a $1.0 million IPR&D charge related to a preclinical milestone achieved for HBS-102 during the three and nine months ended September 30, 2024. Includes a $25.5 million charge related to an upfront license fee incurred upon closing the 2024 Bioprojet Sublicense Agreement and a $17.1 million IPR&D charge related to the acquisition of Epygenix for the nine months ended September 30, 2024. (2) Includes amortization of deferred finance charges. (3) Includes amortization of intangible asset related to WAKIX. (4) Calculated using the reported effective tax rate for the periods presented less impact of discrete items.
Page 16
Harmony Biosciences | Q3 2025 Financial Results & Business Update | All rights reserved.16 Commitment to patients Addressing unmet medical needs Delivering meaningful treatment options Helping patients thrive DELIVER ON PROMISE TO PATIENTS Executional excellence Innovative, catalyst-rich pipeline Profitable, self-funding biotech Meaningful investment opportunity DELIVER STONG VALUE TO SHAREHOLDERS