UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of report (Date of earliest event reported):
(Exact name of registrant as specified in its charter)
(State or other jurisdiction | (Commission | (IRS Employer |
of incorporation) | File Number) | Identification No.) |
(Address of principal executive offices) (Zip Code)
(
(Registrant’s telephone number, including area code)
N/A
(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:
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:
| Trading |
| Name of each exchange | |
Title of each class | Symbol(s) | on which registered | ||
Indicate by check mark whether the registrant is an emerging growth company 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 2.02. Results of Operations and Financial Condition.
On January 13, 2025, Harmony Biosciences Holdings, Inc. (the “Company”) issued a press release announcing its preliminary unaudited net product revenue for the fourth quarter and year ended December 31, 2024. In addition, the Company provided guidance related to 2025 net product revenue and provided an update on clinical programs. A copy of this press release is attached as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.
Item 7.01. Regulation FD Disclosure.
The Company will also be presenting at the 43rd Annual JP Morgan Healthcare Conference on January 15, 2025. The presentation is attached as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference.
The information in this report, including Exhibits 99.1 and 99.2, is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that Section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Note Regarding Forward-Looking Statements
Certain statements in this Current Report on Form 8-K constitute “forward-looking statements” within the meaning of the federal securities laws. These statements are based on management’s current opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results. These forward-looking statements are only predictions, not historical fact, and involve certain risks and uncertainties, as well as assumptions. Actual results, levels of activity, performance, achievements and events could differ materially from those stated, anticipated or implied by such forward-looking statements. While the Company believes that its assumptions are reasonable, it is very difficult to predict the impact of known factors, and, of course, it is impossible to anticipate all factors that could affect actual results. There are many risks and uncertainties that could cause actual results to differ materially from the forward-looking statements made herein including the risks discussed under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, which was filed with the Securities and Exchange Commission (“SEC,”) on February 22, 2024, as well as other factors described from time to time in the Company’s filings with the SEC. Such forward-looking statements are made only as of the date of this Current Report on Form 8-K. The Company undertakes no obligation to publicly update or revise any forward-looking statement because of new information, future events or otherwise, except as otherwise required by law. If it does update one or more forward-looking statements, no inference should be made that the Company will make additional updates with respect to those or other forward-looking statements.
Item 9.01(d). Financial Statements and Exhibits.
The following exhibits are furnished as part of this report on Form 8-K:
Exhibit |
| |
No. | Description | |
99.1 | Press release issued by the Company, dated January 13, 2025. | |
99.2 | ||
104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). |
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.
HARMONY BIOSCIENCES HOLDINGS, INC. | ||
Date: January 13, 2025 | By: | /s/ Sandip Kapadia |
Sandip Kapadia | ||
Chief Financial Officer and Chief Administrative Officer | ||
Exhibit 99.1
HARMONY BIOSCIENCES REPORTS STRONG 2024 REVENUES, PROVIDES 2025 REVENUE GUIDANCE AND HIGHLIGHTS KEY PIPELINE CATALYSTS
WAKIX® (pitolisant) Preliminary Net Revenue of ~$201 Million for Fourth Quarter and ~$714 Million for Full Year 2024; Representing Growth of
~23% in Year-Five of Launch
2025 WAKIX Net Revenue Guidance Between $820 - $860 Million; On Track to a Potential $1 Billion+ Opportunity
Value-Creating Catalysts Anticipated Every Quarter in 2025:
Q1 – FDA Decision on File Acceptance of Pitolisant sNDA for Idiopathic Hypersomnia; Potential Approval in 2025
Q2 – Orexin/BP1.15205 Data Presentation at SLEEP 2025 Conference
Q3 – Pivotal Phase 3 Topline Data for ZYN002 in Fragile X Syndrome
Q4 – Initiation of Pitolisant-HD Pivotal Phase 3 Trial in Narcolepsy
Robust, Catalyst-Rich Pipeline Includes Up to Six Phase 3 Clinical Programs by Year End 2025
PLYMOUTH MEETING, Pa., Jan. 13, 2025 /Business Wire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced strong preliminary, unaudited net product revenues for Q4 and full year 2024 of $201 million and $714 million, respectively. Building on this performance, the company is well positioned for a catalyst-rich 2025, with value-creating milestones anticipated every quarter. Harmony will be highlighting its catalysts for 2025 and other key milestones during its presentation at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 11:15 a.m. PT / 2:15 p.m. ET.
“2025 is a pivotal year for Harmony Biosciences, as we focus on unlocking the full potential of our catalyst-rich neuroscience pipeline,” said Jeffrey M. Dayno, M.D.,
| | |
President and Chief Executive Officer of Harmony Biosciences. “Building upon our foundation of strong and continued revenue growth for WAKIX, our multi-franchise pipeline has come into focus with value-creating catalysts expected each and every quarter this year. We are entering 2025 with strong momentum and a clear path toward long-term growth, building a company with the potential to deliver over $3 billion in net revenue going forward."
Fourth Quarter and Full Year 2024 Net Product Revenue for WAKIX (Preliminary and Unaudited)
| ● | Preliminary, unaudited net product revenue for the quarter ended December 31, 2024, was approximately $201 million, compared to $168.4 million for the same period in 2023 |
| ● | Preliminary, unaudited net product revenue for the full year ended December 31, 2024, was approximately $714 million, compared to $582 million for the same period in 2023, representing ~23% growth in its fifth year on the market |
| ● | The average number of patients on WAKIX increased by approximately 300 sequentially to approximately 7,100 for the quarter ended December 31, 2024 |
2025 Net Revenue Guidance
| ● | Net revenue projected between $820 to $860 million |
Key Catalysts for 2025
| ● | Q1 – FDA decision on file acceptance of pitolisant sNDA for Idiopathic Hypersomnia (IH) |
| o | IH sNDA for pitolisant submitted in Q4 2024 |
| o | Potential FDA approval in 2025 |
| ● | Q2 – Preclinical data for BP1.15205 (OX2R agonist) to be presented at SLEEP 2025 |
| o | Data to include preclinical safety and efficacy |
| o | BP1.15205 on track for first-in-human study in Q3 2025 |
| ● | Q3 – Topline data readout for ZYN002 from pivotal Phase 3 trial in Fragile X syndrome (FXS) |
| o | Potential for first and only approved treatment for patients with FXS |
| o | Estimated 80,000 people living with FXS in the US; worldwide rights |
| ● | Q4 – Initiation of Pitolisant-HD Pivotal Phase 3 trial in Narcolepsy |
| o | Designed to address the largest unmet need in patients with narcolepsy by providing greater efficacy for both excessive daytime sleepiness and cataplexy |
| o | Program to include novel endpoint to assess narcolepsy-related fatigue |
| o | Preliminary IP filed out to 2044 to extend the pitolisant franchise |
| | |
Robust, Multi-Franchise Pipeline
| ● | Anticipates up to six phase 3 clinical development programs by year end 2025 |
| o | Initiated pivotal phase 3 study in Lennox-Gastaut syndrome (LGS) for EPX-100 in Q4 2024 |
| ◾ | EPX-100 is the most advanced 5HT2 (serotonin) agonist in clinical development |
| o | Initiation of Pitolisant-GR pivotal bioequivalence study in Q1 2025 with readout anticipated Q3 2025 |
| ● | Pipeline poised to deliver at least one new product or indication approval every year for the next four years (2028) |
| ● | Current pipeline has the potential to generate over $3B in net revenue |
| ● | Additional program updates to be provided during the Management presentation at the upcoming J.P. Morgan Healthcare Conference on January 15, 2025, at 11:15 a.m. PT / 2:15 p.m. ET. Presentation slides are available on the investor page of the Harmony Biosciences website: https://ir.harmonybiosciences.com/ |
The financial information included in this press release is preliminary, unaudited, and subject to change. It does not present all the information necessary for an understanding of the company's financial results for the fourth quarter or full year 2024.
About WAKIX® (pitolisant) Tablets
WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy. It was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018. WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.
Indications and Usage
WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of excessive daytime sleepiness (EDS) in pediatric patients 6 years of age and older with narcolepsy.
Important Safety Information
| | |
Contraindications
WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.
Warnings and Precautions
WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.
The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is contraindicated in patients with severe hepatic impairment and not recommended in patients with end-stage renal disease (ESRD).
Adverse Reactions
In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and at least twice placebo) for WAKIX were insomnia (6%), nausea (6%), and anxiety (5%). Other adverse reactions that occurred at ≥2% and more frequently than in patients treated with placebo included headache, upper respiratory tract infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash.
In the placebo-controlled phase of the clinical trial conducted in pediatric patients 6 years and older with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and greater than placebo) for WAKIX were headache (19%) and insomnia (7%). The overall adverse reaction profile of WAKIX in the pediatric clinical trial was similar to that seen in the adult clinical trial program.
Drug Interactions
Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half.
Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required.
H1 receptor antagonists that cross the blood-brain barrier may reduce the effectiveness of WAKIX. Patients should avoid centrally acting H1 receptor antagonists.
| | |
WAKIX is a borderline/weak inducer of CYP3A4. WAKIX may reduce the effectiveness of sensitive CYP3A4 substrates, including hormonal contraceptives. Patients using hormonal contraception should be advised to use an alternative non-hormonal contraceptive method during treatment with WAKIX and for at least 21 days after discontinuing treatment.
Use in Specific Populations
There is a pregnancy exposure registry that monitors pregnancy outcomes in women who are exposed to WAKIX during pregnancy. Patients should be encouraged to enroll in the WAKIX pregnancy registry if they become pregnant. To enroll or obtain information from the registry, patients can call 1-800-833-7460.
The safety and effectiveness of WAKIX have not been established for treatment of excessive daytime sleepiness in pediatric patients less than 6 years of age with narcolepsy.
The safety and effectiveness of WAKIX have not been established for treatment of cataplexy in pediatric patients with narcolepsy.
WAKIX is extensively metabolized by the liver. WAKIX is contraindicated in patients with severe hepatic impairment. Dosage adjustment is required in patients with moderate hepatic impairment.
WAKIX is not recommended in patients with end-stage renal disease. Dosage adjustment of WAKIX is recommended in patients with eGFR <60 mL/minute/1.73 m2.
Dosage reduction is recommended in patients known to be poor CYP2D6 metabolizers; these patients have higher concentrations of WAKIX than normal CYP2D6 metabolizers.
Please see the Full Prescribing Information for WAKIX for more information.
To report suspected adverse reactions, contact Harmony Biosciences at 1-800-833-7460 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
About Narcolepsy
Narcolepsy is a rare, chronic, debilitating neurological disease of sleep-wake state instability that impacts approximately 170,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy – its two cardinal symptoms – along with other manifestations of REM sleep dysregulation (hallucinations and sleep paralysis), which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin/orexin, a neuropeptide in the brain that supports sleep-wake state stability. This disease affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed.
| | |
About Idiopathic Hypersomnia
Idiopathic Hypersomnia (IH) is a rare and chronic neurological disease that is characterized by excessive daytime sleepiness (EDS) despite sufficient or even long sleep time. EDS in IH cannot be alleviated by naps, longer sleep or more efficient sleep. People living with IH experience significant EDS along with the symptoms of sleep inertia (prolonged difficulty waking up from sleep) and 'brain fog' (impaired cognition, attention, and alertness). The cause of IH is unknown, but it is likely due to alterations in areas of the brain that stabilize states of sleep and wakefulness. IH is one of the central disorders of hypersomnolence and, like narcolepsy, is a debilitating sleep disorder that can result in significant disruption in daily functioning.
About ZYN002
ZYN002 is the first-and-only pharmaceutically manufactured synthetic cannabidiol devoid of THC and formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. The product is manufactured through a synthetic process in a cGMP facility and is not extracted from the cannabis plant. ZYN002 does not contain THC, the compound that causes the euphoric effect of cannabis, and has the potential to be a nonscheduled product if approved. Cannabidiol, the active ingredient in ZYN002, has been granted orphan drug designation by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of FXS and for the treatment of 22q. Additionally, ZYN002 has received FDA Fast Track designation for the treatment of behavioral symptoms in patients with FXS.
About Fragile X Syndrome
Fragile X syndrome (FXS) is a rare genetic disorder that is the leading known cause of both inherited intellectual disability and autism spectrum disorder. The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability. While the exact prevalence is unknown, upwards of 80,000 patients in the U.S. and 121,000 patients in the European Union and the UK are believed to have FXS, based on FXS prevalence estimates of approximately 1 in 4,000 to 7,000 in males and approximately 1 in 8,000 to 11,000 in females. There is a significant unmet medical need in patients living with FXS as there are currently no FDA-approved treatments for this disorder.
FXS is caused by a mutation in FMR1, a gene which modulates a number of systems, including the endocannabinoid system, and most critically, codes for a protein called FMRP. The FMR1 mutation manifests as multiple repeats of a DNA segment, known as the CGG triplet repeat, resulting in deficiency or lack of FMRP. FMRP helps regulate the production of other proteins and plays a role in the development of synapses, which are critical for relaying nerve impulses, and in regulating synaptic plasticity. In people with full mutation of the FMR1 gene, the CGG segment is repeated more than 200 times, and in most cases causes the gene to not function. Methylation of the FMR1 gene also plays
| | |
a role in determining functionality of the gene. In approximately 60% of patients with FXS, who have complete methylation of the FMR1 gene, no FMRP is produced, resulting in dysregulation of the systems modulated by FMRP.
About Clemizole Hydrochloride (EPX-100)
EPX-100, clemizole hydrochloride, is under development for the treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). EPX-100 acts by targeting central 5-hydroxytryptamine receptors to modulate serotonin signaling. The drug candidate is administered orally twice a day in a liquid formulation and has been developed based on a proprietary phenotype-based zebrafish drug screening platform. DS is caused by a loss of function mutation in the SCN1A gene, and scn1 mutant zebrafish replicate the genetic etiology and phenotype observed in the majority of DS patients. The scn1Lab mutant zebrafish model that expresses voltage gated sodium channels has been used for high-throughput screening of compounds that modulate Nav1.1 in the central nervous system.
About Dravet Syndrome
Dravet syndrome (DS) is a severe and progressive epileptic encephalopathy that begins in infancy and causes significant impact on patient functioning. DS begins in the first year of life and is characterized by high seizure frequency and severity, intellectual disability, and a risk of sudden unexpected death in epilepsy. Approximately 85% of Dravet syndrome cases are caused by de novo loss-of-function (LOF) mutations in a voltage-gated sodium channel gene, SCN1A1. DS has an estimated incidence rate of 1:15,700.
About Lennox-Gastaut Syndrome
Lennox-Gastaut syndrome (LGS) is a rare and drug-resistant epileptic encephalopathy characterized by onset in children between 3-5 years of age. The underlying cause of LGS is unknown and can be related to a wide range of factors including genetic differences and structural differences in the brain. As a result, patients experience multiple seizure types, including atonic seizures, and developmental, cognitive, and behavioral issues. LGS affects approximately 48,000 patients in the U.S.
About Harmony Biosciences
Harmony Biosciences is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Driven by novel science, visionary thinking, and a commitment to those who feel overlooked, Harmony Biosciences is nurturing a future full of therapeutic possibilities that may enable patients with rare neurological diseases to truly thrive. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, Pa., we believe that when empathy and innovation meet, a better future can begin; a vision evident in the therapeutic innovations we advance, the culture we cultivate, and the community programs we foster. For more information, please visit www.harmonybiosciences.com.
| | |
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, product approvals, the plans and objectives of management for future operations and future results of anticipated products. These statements are neither promises 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 clinical trials; our ability to expand the scope of our license agreements with Bioprojet Société Civile de Recherche (“Bioprojet”); the availability of favorable insurance 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 ability to identify, acquire and integrate additional products or product candidates with significant commercial potential that are consistent with our commercial objectives; our commercialization, marketing and manufacturing capabilities and strategy; significant competition in our industry; our intellectual 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 price of Harmony's common stock may be volatile and fluctuate substantially; statements related to our intended share repurchases and repurchase timeframe 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 22, 2024, 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.
| | |
Harmony Biosciences Investor Contact:
Brennan Doyle
484-539-9700
bdoyle@harmonybiosciences.com
Harmony Biosciences Media Contact:
Cate McCanless
202-641-6086
cmccanless@harmonybiosciences.com
| | |
| 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 |
| 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 Biosciences Holdings, Inc.’s (the “Company”) future financial position, preliminary financial results, business strategy and plans and objectives of management for future operations, should be considered forward-looking statements. Forward-looking statements use words like “believes,” “plans,” “expects,” “intends,” “will,” “would,” “anticipates,” “estimates,” “may,” “could,” “might,” “continue,” “potential,” and similar words or expressions in discussions of the Company’s future operations, financial performance or the Company’s strategies, but the absence of these words does not mean that a statement is not forward-looking. These statements are based on current expectations or objectives that are inherently uncertain. These forward-looking statements involve significant risks and uncertainties that could cause the actual results to differ materially from the expressed or implied forwarding-looking statements, including, but not limited to the risk factors discussed under the caption “Risk Factors” in the Company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (the “SEC”) on February 22, 2024 and its other filings with the SEC. While the Company may elect to update such forward-looking statements at some point in the future, it disclaims any obligation to do so, even if subsequent events cause its views to change. This presentation includes information related to market opportunity as well as cost and other estimates obtained from internal analyses and external sources. The internal analyses are based upon management’s understanding of market and industry conditions and have not been verified by independent sources. Similarly, the externally sourced information has been obtained from sources the Company believes to be reliable, but the accuracy and completeness of such information cannot be assured. Neither the Company, nor any of its respective officers, directors, managers, employees, agents, or representatives, (i) make any representations or warranties, express or implied, with respect to any of the information contained herein, including the accuracy or completeness of this presentation or any other written or oral information made available to any interested party or its advisor (and any liability therefore is expressly disclaimed), (ii) have any liability from the use of the information, including with respect to any forward-looking statements, or (iii) undertake to update any of the information contained herein or provide additional information as a result of new information or future events or developments. This presentation concerns product candidates that are or have been under clinical investigation and which have not yet been approved for marketing by the U.S. Food and Drug Administration, European Medicines Agency or other foreign regulatory authorities. These product candidates are currently limited by U.S. Federal law to investigational use, and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated. Preliminary Financial Results The preliminary financial results of the Company for the fiscal year ended December 31, 2024 included in this presentation are estimates and represent the most current information available to the Company’s management, as financial closing procedures for the fourth quarter and fiscal year ended December 31, 2024 are not yet complete. The Company expects that its actual results to be reported in its Annual Report on Form 10-K for the year ended December 31, 2024 will not differ materially from the preliminary results, however, these results are subject to change following the completion of year-end accounting procedures and adjustments, including the execution of the Company’s internal control over financial reporting, the completion of the preparation and audit of the Company’s financial statements and the subsequent occurrence or identification of events prior to the formal issuance of the audited financial statements for fiscal year 2024. 2 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference |
| INNOVATIVE PATIENT-FOCUSED PROFITABLE BIOTECH CATALYST-RICH PIPELINE |
| 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 VALUE CREATION |
| 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 |
| 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) Pitolisant Idiopathic Hypersomnia (IH) Prader-Willi Syndrome (PWS) Myotonic Dystrophy (DM1) Pitolisant Gastro-Resistant (GR) Pitolisant High-Dose (HD) BP1.15205 (Orexin-2 Receptor Agonist) Sleep/Wake Disorders HBS-102 PWS Neurobehavioral ZYN-002 (Cannabidiol Gel) Fragile X Syndrome (FXS) 22q11.2 Deletion Syndrome (22q) Rare Epilepsy EPX-100 (Clemizole Hydrochloride) Dravet Syndrome (DS) Lenox-Gastaut Syndrome (LGS) EPX-200 (Lorcaserin) Developmental and Epileptic Encephalopathies (DEE) 3 CNS FRANCHISES 13 DEVELOPMENT 8 PROGRAMS ASSETS 6PHASE 3 PROGRAMS BY YEAR END |
| 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 $800 2020 2021 2022 2023 2024 Net Revenue ($M) 1 KEY TAKEAWAY 1. Net Revenue for fiscal year 2024 is preliminary, unaudited and subject to change 2. https://narcolepsynetwork.org/accessed Feb 2024 80,000 90,000 > 50% of Patients Undiagnosed ~170,000 Diagnosed Narcolepsy Patients Undiagnosed Narcolepsy Population People Living With Narcolepsy in the U.S² Sustained, durable revenue for WAKIX over 5 years on the market |
| 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 |
| 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) 3Q25 ZYN002 FXS Phase 3 topline data readout Pitolisant-GR Pivotal BE study readout 4Q25 Pitolisant-HD initiation of pivotal Phase 3 trial in Narcolepsy Late-stage pipeline driving a catalyst-rich 2025 KEY TAKEAWAY |
| 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 THE PATIENT REPORT Output from Externally-Led Patient-focused Drug Development meeting with active FDA participation ESTABLISHED SAFETY Non-scheduled and simple dosing regimen Q1 Catalyst: Pitolisant for Idiopathic Hypersomnia (IH) 10 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference FDA decision on IH sNDA file acceptance (potential approval in 2025) |
| 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 NORMAL LEVEL OF WAKEFULNESS Start of Study End of 8 Weeks Pitolisant RWD Placebo Mean ESS Score DAY 22 MONTH 7 MONTH 13 Open Label Extension (52 weeks) Randomized Withdrawal Phase (4 Weeks) Open Label Phase (8 Weeks) 9.4 POINT IMPROVEMENT SUSTAINED RESPONSE WITHIN NORMAL RANGE UP TO 13 MONTHS ITT population: Data on file |
| 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 through 13 months of treatment KEY TAKEAWAY Start of Study End of 8 Weeks Pitolisant RWD Placebo DAY 22 MONTH 7 MONTH 13 Open Label Extension (52 weeks) Randomized Withdrawal Phase (4 Weeks) Open Label Phase (8 Weeks) 78.42% 73.9% 70.3% 75.6% % of Patients on Pitolisant with ESS < 10 ITT population: Data on file NEW DATA |
| 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 sleep/wake UNIQUE STRUCTURE/CHEMICAL SCAFFOLD Differentiated from other known OX2R agonist chemical structures CLINICAL POTENTIAL • Potency and selectivity • Potent on-target effects • Potentially better AE profile • Once-daily dosing PRECLINICAL DATA PRESENTATION AT SLEEP IN JUNE Preclinical data presentation at SLEEP 2025 meeting in June Preclinical Data Presentation at SLEEP 2025 OREXIN CLASS Next wave of Sleep/Wake therapeutic innovation |
| 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 Not reported Selectivity for hOX2R vs hOX1R Not reported Once a day dosing Twice a day dosing IV dosing Twice a day dosing Not reported Once a day dosing Potential for once-daily oral dosing Dosing regimen BP1.15205: Most potent OX2R Agonist (In Vitro Pharmacology Data) 14 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference The most potent orexin-2 receptor agonist (based on publicly available data) KEY TAKEAWAY 1.Bioprojet/Harmony data on file; 2. Lack et al., World Sleep 2023, abstract; 3. Hatanaka et al., ACNP 2022, poster; 4. Kimura et al., World Sleep 2023, abstract; 5. Yukitake et al., Pharmacol Biochem Behav. 2019, publication; 6. Clinicaltrials.gov. |
| 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 potency and excellent selectivity translates to potentially low dose(s) for efficacy which provides dosing flexibility across all central disorders of hypersomnolence Selectivity Excellent selectivity in the context of potency that is several orders of magnitude higher compared to other OX2R agonists Over 140-fold selectivity over OX1R based on highest anticipated human dose from pre-clinical efficacy data Over 1000-fold selectivity against 150 other targets of interest Highest potency compared to other OX2R agonists to-date Excellent Potency and |
| 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 FRAGILE X SYNDROME (FXS) Additional opportunity in related disorder, 22q deletion syndrome (22q) MARKET OPPORTUNITY ~80,000 patients in the US with FXS; similar for 22q, Worldwide rights VERY HIGH UNMET NEED No approved products for FXS or 22q Would be first approved treatment for patients with FXS |
| 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*) Clinically meaningful improvement in patients with complete methylation KEY TAKEAWAY 40% MEDIAN IMPROVEMENT in socially avoidant behaviors (p=0.027*) 1. Berry-Kravis et al 2022 * nominal statistical significance |
| 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 months used to support increasing treatment period in RECONNECT to 4 months -6 -5 -4 -3 -2 -1 0 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 Change from baseline c Placebo (n) ZYN002 (n) 48 47 47 45 49 46 45 43 43 44 36 40 31 33 31 33 10 14 15 19 Month ZYN002 Placebo Switch from Placebo to ZYN002 a. Patients matching primary efficacy population in RECONNECT. b. ZYN2-CL-016 (CONNECT-FX). c. Least square mean ± SE; reduction equals improvement. |
| 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 application in the EU. Primary endpoint: Patients with complete methylation Extended treatment period to 16 weeks Increased dose for individuals >50 kg More patient and family friendly Learnings from Connect-FX trial informed RECONNECT study design KEY TAKEAWAY |
| 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 daytime sleepiness and cataplexy PRELIMINARY IP FILED TO EXTEND PITOLISANT FRANCHISE INTO 2040’S PROGRAM TO INCLUDE NOVEL ENDPOINT TO ASSESS NARCOLEPSY-RELATED FATIGUE |
| 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 indications (IH, DM1) to drive incremental patient, net revenue growth 21 January 15, 2025 Harmony Biosciences | J.P. Morgan Healthcare Conference *With pediatric exclusivity; on-track to meet the requirements for pediatric exclusivity WAKIX ® (LOE Q3 2030*) $1B+ foundation in narcolepsy alone PITOLISANT-GR (PDUFA DATE 2026) Expands pitolisant patient base $300–$500M Opportunity PITOLISANT-HD (PDUFA DATE 2028) Further expands pitolisant patient base and extends pitolisant franchise $1B+ opportunity in narcolepsy alone 2019 2026 2028 2030 2040 • Extension of Pitolisant franchise beyond 2030 with enhanced product profiles • Poised to deliver durable patient growth and significant revenue to the mid 2040s KEY TAKEAWAY |
| 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 highly predictive zebra-fish model PROVEN SAFETY • Clemizole was marketed for ~ 20 yrs with no significant safety and/or tolerability signals from post marketing exposure • Emerging safety and tolerability profile from the Phase 3 study in DS is promising; no need for special laboratory or cardiac monitoring required DOSING REGIMEN BID dosing; very important clinical consideration for patients with DEEs ADVANCED CLINICAL DEVELOPMENT • Actively enrolling patients in US and EU in a phase 3 registrational study in Dravet syndrome; Topline data in 2026 • Initiated Phase 3 registrational study in LGS in Q4 2024; Topline Data in 2026 |
| 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 Phase 3 TLD in narcolepsy (2027) • Pitolisant-HD PDUFA for narcolepsy (2028) • ZYN002 22q deletion syndrome – Phase 3 TLD • EPX100 DS/LGS PDUFA • Pitolisant PWS PDUFA Pipeline poised to deliver one or more new product or indication launches each year over the next 4 years KEY TAKEAWAY |
| 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-TERM VALUE CREATION |
| company/harmonybiosciences/ @harmonybio harmony_biosciences www.harmonybiosciences.com |
