8-K
Harmony Biosciences Holdings, Inc. (HRMY)
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
HARMONY BIOSCIENCES HOLDINGS, INC .
(Exact name of registrant as specified in its charter)
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| Delaware | 001-39450 | 82-2279923 |
| (State or other jurisdiction | (Commission | (IRS Employer |
| of incorporation) | File Number) | Identification No.) |
630 W. Germantown Pike , Suite 215
Plymouth Meeting , PA **** 19462
(Address of principal executive offices) (Zip Code)
( 484 ) 539-9800
(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) |
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| ☐ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
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| ☐ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
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| ☐ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
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Securities registered pursuant to Section 12(b) of the Act:
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| | **** | Trading | **** | Name of each exchange |
| Title of each class | | Symbol(s) | | on which registered |
| Common Stock, $0.00001 par value per share | | HRMY | | The Nasdaq Global Market |
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 February 25, 2025, Harmony Biosciences Holdings, Inc. (the “Company”) issued a press release announcing its financial results for the quarter and year ended December 31, 2024. 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.
On February 25, 2025, the Company posted an investor presentation to its website at ttps://ir.harmonybiosciences.com (the “Investor Presentation”). A copy of the Investor Presentation is attached as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference. The Company expects to use the Investor Presentation, in whole or in part, and possibly with modifications, in connection with presentations to investors, analysts and others.
The information contained in the Investor Presentation is summary information that is intended to be considered in the context of the Company’s Securities and Exchange Commission (“SEC”) filings and other public announcements that the Company may make, by press release or otherwise, from time to time. The Investor Presentation speaks only as of the date of this Current Report on Form 8-K. The Company undertakes no duty or obligation to publicly update or revise the information contained in the Investor Presentation, although it may do so from time to time. Any such updating may be made through the filing of other reports or documents with the SEC, through press releases or through other public disclosure. In addition, the exhibit furnished herewith contains statements intended as “forward-looking statements” that are subject to the cautionary statements about forward-looking statements set forth in such exhibit. By furnishing the information contained in the Investor Presentation, the Company makes no admission as to the materiality of any information in the Investor Presentation that is required to be disclosed solely by reason of Regulation FD.
This Current Report on Form 8-K and its contents (including Exhibits 99.1 and 99.2) are furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section or Sections 11 and 12(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”), nor shall it be deemed incorporated by reference in any filing under the Securities Act or the Exchange Act, regardless of any general incorporation language in such filing, except as shall be 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. Financial Statements and Exhibits.
(d) Exhibits.
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| Exhibit | | |
| No. | | Description |
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| 99.1* | | Press release issued by the Company, dated February 25, 2025. |
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| 99.2* | | Investor Presentation dated February 25, 2025. |
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| 104 | | Cover Page Interactive Data File (embedded within the Inline XBRL document). |
| * | This Exhibit is furnished herewith and will not be deemed “filed” for purposes of Section 18 of the Exchange Act or deemed to be incorporated by reference into any filing under the Exchange Act or the Securities Act except to the extent that Harmony Biosciences Holdings, Inc. specifically incorporates it by reference. |
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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.
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| | HARMONY BIOSCIENCES HOLDINGS, INC. | |
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| Date: February 25, 2025 | By: | /s/ Sandip Kapadia |
| | | Sandip Kapadia |
| | | Chief Financial Officer and Chief Administrative Officer |
Exhibit 99.1
HARMONY BIOSCIENCES REPORTS STRONG 2024 FINANCIAL RESULTS AND REITERATES 2025 NET REVENUE GUIDANCE; HIGHLIGHTS 2025 CATALYSTS IN SLEEP/WAKE AND FRAGILE X SYNDROME DEVELOPMENT PROGRAMS
WAKIX® (pitolisant) Net Revenue of $201.3 Million for Fourth Quarter and $714.7 Million for Full Year 2024; Representing Growth of 23% in Year-Five on the Market
2025 WAKIX Net Revenue Guidance Between $820 - $860 Million; On Track toward a Potential $1 Billion+ Opportunity
Committed to Leadership in Sleep/Wake with Next Generation Pitolisant Formulations and Orexin 2 Receptor Agonist; Phase 3 Registrational Trials with Pitolisant HD in Narcolepsy and IH to Initiate in Q4 2025
Topline Data Readout from Phase 3 Registrational Trial with ZYN002 in Fragile X Syndrome On Track for Q3 2025
Pitolisant Patent Position Strengthened with Favorable Settlement of First Generic Litigation
Conference Call and Webcast to be Held Today at 8:30 a.m. ET
PLYMOUTH MEETING, Pa., February 25, 2025 /Business Wire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced a record $201.3 million in net product revenues for the fourth quarter 2024, and full year 2024 net revenues of $714.7 million, ending the year with $576 million in cash, cash equivalents and investments on the balance sheet. The company has guided 2025 WAKIX® net revenues to $820 - $860 million, targeting a $1B+ opportunity with WAKIX in narcolepsy alone.
“2025 is set up to be a transformational year for Harmony, as our robust, late-stage pipeline comes into focus and we advance our long-term growth strategy,” said Jeffrey M. Dayno, M.D., President and Chief Executive Officer of Harmony Biosciences. “This year will bring major milestones for Harmony, with the topline data readout from our
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Phase 3 registrational trial with ZYN002 in Fragile X syndrome expected in Q3 and the potential, if successful, to bring the first approved treatment to the Fragile X patient community. In Q4, we will be initiating two Phase 3 registrational trials with our next gen Pitolisant HD formulation in both narcolepsy and idiopathic hypersomnia, potentially extending the pitolisant franchise out to the 2040s and further solidifying our leadership in sleep/wake. Taken together, these catalysts underscore the importance of 2025 as a pivotal year in Harmony’s long-term growth strategy. With at least one new product candidate or indication launch each year over the coming years, our pipeline has the potential to deliver over $3 billion in net revenue as the leading CNS company focused on developing and delivering innovative treatments for patients with unmet needs.”
Franchise Highlights
Sleep/Wake Franchise
WAKIX in Narcolepsy
| ● | Net Sales were $201.3M for Q4 2024 and $714.7M for FY 2024 |
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| ● | 2025 Net Revenue projected between $820 to $860 million |
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| ● | Settlement reached in first Abbreviated New Drug Application (ANDA) litigation, giving Novugen Pharma a license to sell its generic product beginning in January 2030 or earlier under certain circumstances |
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| o | This settlement reinforces the strength and durability of Harmony’s intellectual property portfolio and its vigorous defense of its patent estate |
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| ● | On track to obtain pediatric exclusivity which, if granted, would add an additional 6 months of regulatory exclusivity |
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Pitolisant HD (high-dose)
| ● | Higher dose and optimized pharmacokinetic profile designed to address the need for greater efficacy in excessive daytime sleepiness (EDS) and other symptoms in patients with central disorders of hypersomnolence |
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| ● | Phase 3 registrational trial in narcolepsy to include endpoint on narcolepsy-related fatigue in pursuit of differentiated label |
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| o | Trial to initiate in Q4 2025; topline data readout anticipated in 2027 with potential PDUFA date in 2028 |
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| ● | Phase 3 registrational trial in IH to include endpoint on sleep inertia in pursuit of differentiated label |
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| o | Trial to initiate in Q4 2025; topline data readout anticipated in 2027 with potential PDUFA date in 2028 |
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| ● | Provisional IP filed out to 2044 for narcolepsy and IH |
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Pitolisant GR (gastro-resistant)
| ● | Pivotal bioequivalence study on track to initiate Q1 2025 |
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| o | Topline data readout anticipated in Q3 2025 with potential PDUFA in 2026 |
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| ● | Provisional IP filed out to 2044 |
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Orexin-2 receptor agonist (BP1.15205)
| ● | Potential to be best-in-class orexin-2 receptor agonist based on a novel chemical scaffold, preclinical potency, selectivity and safety data, as well as its potential for once-a-day dosing |
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| ● | Preclinical safety and efficacy data to be presented at SLEEP 2025 (June) |
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| ● | IMPD submission on track for mid-2025; first-in-human study expected to initiate 2H 2025 |
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Neurobehavioral Franchise
ZYN002
| ● | Innovative product profile: pharmaceutically manufactured synthetic cannabidiol devoid of THC |
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| ● | The RECONNECT study is designed to confirm the positive findings from the prespecified analysis of the primary outcome in the subgroup of patients with complete methylation from the Phase 2/3 CONNECT study |
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| ● | Potential to be the first and only approved treatment for patients with Fragile X syndrome (FXS) (80,000 patients in the U.S.), and also possess global rights |
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| ● | Topline data readout from Phase 3 registrational trial of ZYN002 in FXS (RECONNECT study) in Q3 2025 with potential PDUFA date in 2026 |
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| ● | Phase 3 registrational trial in 22q11.2 deletion syndrome (22q) to initiate in 2025 |
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Rare Epilepsy Franchise ****
EPX-100 (clemizole hydrochloride}
| ● | Most advanced development program in the 5HT2 (serotonin) agonist class |
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| ● | Topline data readout from Phase 3 registrational trial in Dravet syndrome (ARGUS Study) anticipated in 2026 |
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| ● | Topline data for Phase 3 registrational trial in patients with Lennox-Gastaut syndrome (LIGHTHOUSE study) anticipated in 2026 |
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EPX-200 (lorcaserin hydrochloride)
| ● | Proven mechanism of action in developmental and epileptic encephalopathies (DEEs) confirmed via non-clinical and clinical data |
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| ● | Currently in IND enabling stage |
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Fourth Quarter 2024 Financial Results
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Net product revenues for the quarter ended December 31, 2024, were $201.3 million, compared to $168.4 million for the same period in 2023. The 20% growth versus the same period in 2023 is primarily attributed to strong commercial sales of WAKIX driven by continued organic demand tapping into a large market opportunity (approximately 80,000 patients diagnosed with narcolepsy in the U.S.) and the broad clinical utility of WAKIX across the approximately 9,000 HCPs that we call on (about 5,000 of whom do not participate in an oxybate REMS program). The average number of patients on WAKIX increased by approximately 300 sequentially to approximately 7,100 for the quarter ended December 31, 2024.
GAAP net income for the quarter ended December 31, 2024, was $49.5 million, or $0.85 earnings per diluted share, compared to GAAP net income of $26.6 million, or $0.45 earnings per diluted share, for the same period in 2023. Non-GAAP adjusted net income was $63.0 million, or $1.08 earnings per diluted share, for the quarter ended December 31, 2024, compared to Non-GAAP adjusted net income of $42.8 million, or $0.73 per diluted share, for the same period in 2023.
Reconciliations of applicable GAAP financial measures to Non-GAAP financial measures are included at the end of this press release.
Harmony’s operating expenses included the following:
| ● | Research and Development expenses were $34.7 million in the fourth quarter of 2024, as compared to $30.3 million for the same quarter in 2023, representing a 14% increase; |
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| ● | Sales and Marketing expenses were $27.6 million in the fourth quarter of 2024, as compared to $26.9 million for the same quarter in 2023, representing a 3% increase; |
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| ● | General and Administrative expenses were $28.9 million in the fourth quarter of 2024, as compared to $27.9 million for the same quarter in 2023, representing a 4% increase; and |
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| ● | Total Operating Expenses were $91.1 million in the fourth quarter of 2024, as compared to $85.1 million for the same quarter in 2023, representing a 7% increase. |
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Full Year 2024 Financial Results
Net product revenues for the year ended December 31, 2024, were $714.7 million, compared to $582.0 million for the same period in 2023. The 23% growth versus the same period in 2023 is primarily attributed to strong commercial sales of WAKIX driven by continued organic demand tapping into a large market opportunity (approximately 80,000 patients diagnosed with narcolepsy in the U.S.) and the broad clinical utility of WAKIX across the approximately 9,000 HCPs that we call on (about 5,000 of whom do not participate in an oxybate REMS program).
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GAAP net income for the year ended December 31, 2024, was $145.5 million, or $2.51 earnings per diluted share, compared to GAAP net income of $128.9 million, or $2.13 earnings per diluted share, for the same period in 2023. Non-GAAP adjusted net income was $233.9 million, or $4.04 earnings per diluted share, for the year ended December 31, 2024, compared to Non-GAAP adjusted net income of $188.4 million, or $3.12 per diluted share, for the same period in 2023.
Reconciliations of applicable GAAP financial measures to Non-GAAP financial measures are included at the end of this press release.
Harmony’s operating expenses included the following:
| ● | Research and Development expenses were $145.8 million for the year ended December 31, 2024, as compared to $76.1 million for the prior year, representing an 92% increase, primarily driven by a $25.5 million upfront licensing fee as part of the 2024 Bioprojet Sublicense Agreement for BP1.15205, an orexin-2 receptor agonist, and a $17.1 million IPR&D charge related to the acquisition of Epygenix |
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| ● | Sales and Marketing expenses were $110.9 million for the year ended December 31, 2024, as compared to $97.4 million for the prior year, representing a 14% increase; |
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| ● | General and Administrative expenses were $110.4 million for the year ended December 31, 2024, as compared to $95.3 million for the prior year, representing a 16% increase; and |
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| ● | Total Operating Expenses were $367.1 million for the year ended December 31, 2024, as compared to $268.8 million for the prior year, representing a 37% increase. |
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As of December 31, 2024, Harmony had cash, cash equivalents and investments of $576.1 million, compared to $425.6 million as of December 31, 2023.
2025 Net Product Revenue Guidance
Expect full year 2025 net product revenue of $820 million to $860 million.
Share Repurchase Program
The remaining amount of common stock authorized for repurchases as of December 31, 2024, was $150 million.
Conference Call Today at 8:30 a.m. ET
We are hosting our fourth quarter and full year 2024 financial results conference call and webcast today, beginning at 8:30 a.m. Eastern Time. The live and replay webcast of the call will be available on the investor relations page of our website at https://ir.harmonybiosciences.com/. To participate in the live call by phone, dial 800-
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445-7795 (domestic) or 785-424-1699 (international), and reference passcode HRMYQ424.
Non-GAAP Financial Measures
In addition to our GAAP results, we present certain Non-GAAP metrics including Non-GAAP adjusted net income and Non-GAAP adjusted net income per share, which we believe provides important supplemental information to management and investors regarding our performance. These measurements are not a substitute for GAAP measurements, and the manner in which we calculate Non-GAAP adjusted net income and Non-GAAP adjusted net income per share may not be identical to the manner in which other companies calculate adjusted net income and adjusted net income per share. We use these Non-GAAP measurements as an aid in monitoring our financial performance from quarter-to-quarter and year-to-year and for benchmarking against comparable companies.
Non-GAAP financial measures should not be considered in isolation or as a substitute for comparable GAAP measures; should be read in conjunction with our consolidated financial statements prepared in accordance with GAAP; have no standardized meaning prescribed by GAAP; and are not prepared under any comprehensive set of accounting rules or principles. In addition, from time to time in the future there may be other items that we may exclude for purposes of our Non-GAAP financial measures; and we may in the future cease to exclude items that we have historically excluded for purposes of our Non-GAAP financial measures.
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.
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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.
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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 m^2^.
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
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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
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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
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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 25, 2025, and our other filings with the SEC could cause actual results to differ materially from those indicated by
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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 HOLDINGS, INC. AND SUBSIDIARY CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)
(In thousands, except share and per share data)
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|---|---|---|---|---|---|---|---|---|---|---|---|---|
| | | Three Months Ended | | Year Ended | ||||||||
| | | December 31, | | December 31, | | December 31, | | December 31, | ||||
| | | 2024 | | 2023 | | 2024 | | 2023 | ||||
| Net product revenue | | $ | 201,267 | | $ | 168,412 | | $ | 714,734 | | $ | 582,022 |
| Cost of product sold | | 54,409 | | 43,152 | | 156,815 | | 121,236 | ||||
| Gross profit | | 146,858 | | 125,260 | | 557,919 | | 460,786 | ||||
| Operating expenses: | | | | | ||||||||
| Research and development | | 34,666 | | 30,306 | | 145,825 | | 76,063 | ||||
| Sales and marketing | | 27,600 | | 26,886 | | 110,916 | | 97,404 | ||||
| General and administrative | | 28,865 | | 27,872 | | 110,352 | | 95,289 | ||||
| Total operating expenses | | 91,131 | | 85,064 | | 367,093 | | 268,756 | ||||
| Operating income | | 55,727 | | 40,196 | | 190,826 | | 192,030 | ||||
| Loss on debt extinguishment | | — | | — | | — | | (9,766) | ||||
| Other expense (income), net | | 160 | | 193 | | (68) | | 159 | ||||
| Interest expense | | | (4,209) | | | (4,796) | | | (17,496) | | | (23,757) |
| Interest income | | 4,477 | | 4,096 | | 18,542 | | 14,730 | ||||
| Income before income taxes | | 56,155 | | 39,689 | | 191,804 | | 173,396 | ||||
| Income tax benefit (expense) | | (6,680) | | (13,082) | | (46,311) | | (44,543) | ||||
| Net income | | $ | 49,475 | | $ | 26,607 | | $ | 145,493 | | $ | 128,853 |
| EARNINGS PER SHARE: | | | | | ||||||||
| Basic | | $ | 0.87 | | $ | 0.46 | | $ | 2.56 | | $ | 2.17 |
| Diluted | | $ | 0.85 | | $ | 0.45 | | $ | 2.51 | | $ | 2.13 |
| Weighted average number of shares of common stock - basic | | 57,097,092 | | 58,320,400 | | 56,885,455 | | 59,469,648 | ||||
| Weighted average number of shares of common stock - diluted | | 58,218,052 | | 58,853,292 | | 57,869,915 | | 60,372,397 |
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HARMONY BIOSCIENCES HOLDINGS, INC. AND SUBSIDIARIES
CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share data)
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| | **** | December 31, | **** | December 31, | ||
| | 2024 | 2023 | ||||
| ASSETS | | | ||||
| CURRENT ASSETS: | | | ||||
| Cash and cash equivalents | | $ | 453,001 | | $ | 311,660 |
| Investments, short-term | | | 14,185 | | | 41,800 |
| Trade receivables, net | | 83,033 | | 74,140 | ||
| Inventory, net | | 7,198 | | 5,363 | ||
| Prepaid expenses | | 13,714 | | 12,570 | ||
| Other current assets | | 8,121 | | 5,537 | ||
| Total current assets | | 579,252 | | 451,070 | ||
| NONCURRENT ASSETS: | | | ||||
| Property and equipment, net | | 1,257 | | 371 | ||
| Restricted cash | | 270 | | 270 | ||
| Investments, long-term | | | 108,874 | | | 72,169 |
| Intangible assets, net | | 113,263 | | 137,108 | ||
| Deferred tax asset | | | 190,398 | | | 144,162 |
| Other noncurrent assets | | 5,886 | | 6,298 | ||
| Total noncurrent assets | | 419,948 | | 360,378 | ||
| TOTAL ASSETS | | $ | 999,200 | | $ | 811,448 |
| LIABILITIES AND STOCKHOLDERS’ EQUITY | | | ||||
| CURRENT LIABILITIES: | | | ||||
| Trade payables | | $ | 13,744 | | $ | 17,730 |
| Accrued compensation | | 18,776 | | 23,747 | ||
| Accrued expenses | | 120,640 | | 99,494 | ||
| Current portion of long-term debt | | | 16,250 | | | 15,000 |
| Other current liabilities | | 5,672 | | 7,810 | ||
| Total current liabilities | | 175,082 | | 163,781 | ||
| NONCURRENT LIABILITIES: | | | ||||
| Long-term debt, net | | 163,016 | | 178,566 | ||
| Other noncurrent liabilities | | 1,947 | | 2,109 | ||
| Total noncurrent liabilities | | 164,963 | | 180,675 | ||
| TOTAL LIABILITIES | | 340,045 | | 344,456 | ||
| COMMITMENTS AND CONTINGENCIES (Note 13) | | | ||||
| STOCKHOLDERS’ EQUITY: | | | ||||
| Common stock—$0.00001 par value; 500,000,000 shares authorized at December 31, 2024 and December 31, 2023, respectively; 57,144,887 and 56,769,081 shares issued and outstanding at December 31, 2024 and December 31, 2023, respectively | | 1 | | 1 | ||
| Additional paid in capital | | 656,872 | | 610,266 | ||
| Accumulated other comprehensive (loss) income | | | 66 | | | 2 |
| Retained earnings (accumulated deficit) | | 2,216 | | (143,277) | ||
| TOTAL STOCKHOLDERS’ EQUITY | | 659,155 | | 466,992 | ||
| TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY | | $ | 999,200 | | $ | 811,448 |
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HARMONY BIOSCIENCES HOLDINGS, INC. AND SUBSIDIARIES
RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL RESULTS
(In thousands except share and per share data)
| | | Three Months Ended | | Year Ended | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| | | December 31, | | December 31, | | December 31, | | December 31, | ||||
| | | 2024 | | 2023 | | 2024 | | 2023 | ||||
| GAAP net income | | $ | 49,475 | | $ | 26,607 | | $ | 145,493 | | $ | 128,853 |
| Non-GAAP Adjustments: | | | | | ||||||||
| Non-cash interest expense (1) | | 169 | | 185 | | 700 | | 3,246 | ||||
| Depreciation | | 6 | | 164 | | 267 | | 514 | ||||
| Amortization (2) | | 5,962 | | 5,961 | | 23,845 | | 23,845 | ||||
| Stock-based compensation expense | | 9,856 | | 8,894 | | 42,701 | | 31,205 | ||||
| Licensing fee and milestone payments (3) | | | - | | | - | | | 26,500 | | | 750 |
| Loss on debt extinguishment (4) | | | - | | | - | | | - | | | 9,766 |
| Transaction related costs (5) | | | - | | | 9,804 | | | 17,095 | | | 9,804 |
| Income tax effect related to non-GAAP adjustments (6) | | (2,442) | | (8,789) | | (22,657) | | (19,624) | ||||
| Non-GAAP adjusted net income | | $ | 63,026 | | $ | 42,826 | | $ | 233,944 | | $ | 188,359 |
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| GAAP reported net income per diluted share | | $ | 0.85 | | $ | 0.45 | | $ | 2.51 | | $ | 2.13 |
| Non-GAAP adjusted net income per diluted share | | $ | 1.08 | | $ | 0.73 | | $ | 4.04 | | $ | 3.12 |
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| Weighted average number of shares of common stock used in non-GAAP diluted per share | | 58,218,052 | | 58,853,292 | | 57,869,915 | | 60,372,397 |
(1) Includes amortization of deferred finance charges.
(2) Includes amortization of intangible asset related to WAKIX.
(3) Amount represents upfront licensing fee incurred upon closing the 2024 Bioprojet Sublicense Agreement, milestone payment related to HBS-102 in September 2024 and milestone payment related to HBS-102 in March 2023.
(4) Includes loss on extinguishment of the Blackstone Credit Agreement.
(5) Includes IPR&D charge related to the acquisitions of Epygenix in April 2024 and Zynerba in October 2023.
(6) Calculated using the reported effective tax rate for the periods presented less impact of discrete items.
Harmony Biosciences Investor Contact:
Brennan Doyle
484-539-9700
bdoyle@harmonybiosciences.com
Harmony Biosciences Media Contact:
Cate McCanless
202-641-6086
cmccanless@harmonybiosciences.com
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Exhibit 99.2
| Copyright © 2025 Harmony Biosciences. All rights reserved.<br>Q4 & FY 2024<br>Financial Results<br>&<br>Business Update<br>February 25, 2025 |
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| Forward-Looking Statements<br>This presentation includes forward‐looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of<br>historical facts contained in these materials or elsewhere, including statements regarding Harmony Biosciences Holdings, Inc.’s (the “Company”) future financial position,<br>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<br>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<br>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<br>statements involve significant risks and uncertainties that could cause the actual results to differ materially from the expressed or implied forwarding-looking statements,<br>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<br>Exchange Commission (the “SEC”) on February 25, 2025 and its other filings with the SEC. While the Company may elect to update such forward-looking statements at some<br>point in the future, it disclaims any obligation to do so, even if subsequent events cause its views to change.<br>This presentation includes information related to market opportunity as well as cost and other estimates obtained from internal analyses and external sources. The internal<br>analyses are based upon management’s understanding of market and industry conditions and have not been verified by independent sources. Similarly, the externally<br>sourced information has been obtained from sources the Company believes to be reliable, but the accuracy and completeness of such information cannot be assured.<br>Neither the Company, nor any of its respective officers, directors, managers, employees, agents, or representatives, (i) make any representations or warranties, express or<br>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<br>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<br>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<br>future events or developments.<br>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<br>Drug Administration, European Medicines Agency or other foreign regulatory authorities. These product candidates are currently limited by U.S. Federal law to investigational<br>use, and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated.<br>2 |
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| Innovation driving growth of the portfolio<br>Extending Our Leadership<br>Position<br>• Initiating Phase 3 registrational trials of<br>Pitolisant HD in narcolepsy & IH Q4 ‘25<br>• Initiating pivotal BE study of Pitolisant<br>GR Q1 ‘25; topline data readout Q3 ‘25<br>• Provisional IP for Pitolisant HD & GR<br>out to 2044 to extend franchise<br>• Potential best-in-class orexin-2 agonist<br>(BP1.15205) data at SLEEP 2025<br>Next Major Clinical Catalyst<br>• On track for topline data readout<br>of Phase 3 registrational trial of<br>ZYN002 in Fragile X syndrome in<br>Q3 2025<br>• Plan to initiate Phase 3<br>registrational trial in 22q deletion<br>syndrome in Q4 2025<br>Most Advanced 5-HT2<br>Development Program<br>• EPX-100: validated MOA<br>• Ongoing Phase 3 registrational trial<br>in Dravet syndrome; topline data in<br>2026<br>• Phase 3 registrational trial in<br>Lennox-Gastaut syndrome initiated<br>Q4 2024<br>SLEEP/WAKE<br>NEURO<br>BEHAVIORAL EPILEPSY<br>Harmony Biosciences data on file. |
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| $168.4<br>$201.3<br>HIGHLIGHTS<br> Q4 ‘24 represented the highest quarter of Net<br>Revenue in our history, $201.3M<br> FY ‘24 Net Revenue of $714.7M<br> Durable double-digit sales growth continued<br>through year five on the market<br> $576M of Cash and cash equivalents on the<br>Balance Sheet<br>2025 Full Year Guidance of $820-$860M<br>Q4 ‘23 Q4 ‘24<br>Q4 ‘24 WAKIX Net Revenue ($M)<br>WAKIX® Net Revenue Performance<br>4<br>20%<br>Confident in WAKIX being a potential $1B+ opportunity in narcolepsy alone<br>$582.0<br>$714.7<br>Confident in WAKIX being a potential $1B+ opportunity in narcolepsy alone<br>FY ‘23 FY ‘24<br>23%<br>FY ‘24 WAKIX Net Revenue ($M) |
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| +300<br>+400<br>+300<br>+300<br>+100<br>+400<br>+300<br>+300<br>+200<br>+350<br>+350<br>+350<br>+150<br>+250<br>+250<br>+300<br>0<br>1000<br>2000<br>3000<br>4000<br>5000<br>6000<br>7000<br>8000<br>Q4<br>20<br>Q1<br>21<br>Q2<br>21<br>Q3<br>21<br>Q4<br>21<br>Q1<br>22<br>Q2<br>22<br>Q3<br>22<br>Q4<br>22<br>Q1<br>23<br>Q2<br>23<br>Q3<br>23<br>Q4<br>23<br>Q1<br>24<br>Q2<br>24<br>Q3<br>24<br>Q4<br>24<br>Average # of Patients Net Patient Additions Column1<br>Meaningfully Differentiated Product Profile Key Driver in Strong<br>Durable Growth in Patients on WAKIX®<br>5<br>Q4 24 Highlights<br>More unique prescribers of WAKIX® than<br>sodium oxybate<br>Strong market access coverage (>80%) –<br>even with the availability of generic and new<br>1 oxybate options<br>~Average # of Patients on WAKIX<br>~Approximate Patient Counts<br>~7,100<br>1. Net Patient Additions based on previously disclosed quarterly average number of patients on WAKIX<br>~7,100 Average<br>number of<br>patients on<br>WAKIX and<br>growing<br>On our way<br>to a $1B+<br>opportunity<br>in adult<br>narcolepsy |
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| Pitolisant Franchise: Patient-Centric Drug Development<br>Building Our Leadership Position in Sleep/Wake<br>6<br>Non-scheduled<br>Gastro-resistant<br>coating<br>EDS and<br>Cataplexy<br>No titration<br>Fatigue indication<br>Higher dose,<br>enhanced efficacy<br>Non-scheduled<br>EDS and<br>Cataplexy<br>Non-scheduled<br>EDS and<br>Cataplexy<br>Gastro-resistant<br>coating<br>No titration<br>Well tolerated;<br>safety profile<br>Well tolerated;<br>safety profile<br>Well tolerated;<br>safety profile<br>WAKIX®* Pitolisant GR Pitolisant HD<br>Products require<br>titration<br>NARCOLEPSY UNMET NEEDS<br>FDA-approved treatments<br>are scheduled (CII – CIV)<br>Only 1 FDA-approved treatment<br>indicated for EDS and cataplexy<br>1. McCullough et al. Novel treatment options in narcolepsy, Chicago Rush Memorial Center - SLEEP 2019 Abstract; 2. Droogleever et al. (2012). Severe fatigue in narcolepsy with cataplexy. Sleep, 21(2), 163-169; 3. Barateau et al., Dauvilliers, 2019; 4. Wang et al., 2023;<br>4. Zhan et al., 2023; 5. Postmarketing study; 6. Versta Research, Know Narcolepsy Survey (“Know Narcolepsy”), October 2018; * WAKIX attributes based on FDA-approved adult narcolepsy product labelling.<br>Residual symptoms¹<br>Report fatigue²<br>Report GI<br>disturbances³,<br>⁴<br>Cite nausea as<br>a side effect⁵<br>Cite frustration with<br>side effects⁶<br>Don’t achieve<br>clinical benefit<br>60%<br>75%<br>100%<br>88%<br>56%<br>100%<br>1<br>33%<br>1 in 5 |
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| Pitolisant Franchise Poised to Drive Durable Patient and Revenue<br>Growth to the Mid-2040s<br>• Two meaningfully differentiated product profiles building off WAKIX with PDUFAs prior to LOE<br>• Provisional patents filed out to 2044 to extend durable patient and net revenue growth<br>• Pursuing other indications (IH, DM1) to drive incremental patient, net revenue growth<br>*On track toward pediatric exclusivity – WAKIX LOE would be Q3 2030<br>WAKIX® (LOE Q1 2030*)<br>$1B+ foundation in narcolepsy alone<br>PITOLISANT GR (TARGET PDUFA DATE 2026)<br>Expands pitolisant patient base<br>Potential $300–$500M Opportunity<br>PITOLISANT HD (TARGET PDUFA DATE 2028)<br>Grows pitolisant patient base and extends pitolisant franchise<br>Potential $1B+ opportunity in narcolepsy alone<br>2019 2026 2028 2030 2040<br>• Pitolisant franchise strengthens leadership position in sleep/wake<br>• Poised to deliver durable patient growth and significant revenue to the mid 2040s<br>7 |
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| Innovative Late-Stage Pipeline<br>8<br>PRODUCT / INDICATION PRE-IND PHASE 1 PHASE 2 PHASE 3 REGULATORY FILING MARKETED PRODUCT<br>Sleep/Wake<br>WAKIX®<br>EDS in Narcolepsy (Adults)<br>Cataplexy in Narcolepsy (Adults)<br>EDS in Narcolepsy (Pediatric)<br>Pitolisant<br>Prader-Willi Syndrome (PWS)<br>Myotonic Dystrophy (DM1)<br>Pitolisant Gastro-Resistant (GR)<br>Pitolisant High-Dose (HD)<br>BP1.15205 (Orexin-2 Receptor Agonist)<br>Sleep/Wake Disorders<br>HBS-102<br>PWS<br>Neurobehavioral<br>ZYN-002 (Cannabidiol Gel)<br>Fragile X Syndrome (FXS)<br>22q11.2 Deletion Syndrome (22q)<br>Rare Epilepsy<br>EPX-100 (Clemizole Hydrochloride)<br>Dravet Syndrome (DS)<br>Lenox-Gastaut Syndrome (LGS)<br>EPX-200 (Lorcaserin)<br>Developmental and Epileptic Encephalopathies (DEE)<br>3 CNS<br>FRANCHISES 13 DEVELOPMENT 8 PROGRAMS* ASSETS 6PHASE 3 PROGRAMS<br>BY YEAR END<br>*includes additional ongoing clinical and regulatory programs |
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| 2025 Anticipated Catalysts<br>9<br>2Q25<br>BP1.15205 (OX2R<br>agonist)<br>• Preclinical data<br>presentation at<br>SLEEP 2025<br>• IMPD submission<br>(IND submission 2H)<br>3Q25<br>ZYN002<br>FXS Phase 3<br>Registrational Trial<br>topline data readout<br>Pitolisant GR Pivotal<br>BE study readout<br>4Q25<br>Pitolisant HD<br>Initiation of Phase 3<br>registrational trials in<br>narcolepsy and idiopathic<br>hypersomnia (IH)<br>Late-stage pipeline driving a catalyst-rich 2025 KEY<br>TAKEAWAY |
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| BP1.15205: Potential Best-in-Class Orexin 2 Receptor (OX2R) Agonist<br>10<br>TEIJIN<br>Tokyo-based Pharma; innovator of TPM-1116 (now BP1.15205)<br>UNIQUE STRUCTURE/CHEMICAL SCAFFOLD<br>Differentiated from other known OX2R agonist chemical structures<br>CLINICAL POTENTIAL<br>• Potency and selectivity<br>• Potent on-target effects<br>• Potentially better AE profile<br>• Once-daily dosing<br>PRECLINICAL DATA PRESENTATION AT SLEEP IN JUNE<br>Preclinical safety and efficacy data presentation at SLEEP 2025 meeting in<br>June<br>Preclinical Safety<br>and Efficacy Data<br>Presentation at<br>SLEEP 2025<br>OREXIN CLASS<br>Next wave of Sleep/Wake therapeutic innovation |
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| ZYN002: Potential for First Approved Treatment of Fragile X Syndrome<br>11<br>Topline Data<br>Readout from Phase<br>3 Registrational<br>Trial, RECONNECT<br>Study: 3Q 2025<br>ZYN002: INNOVATIVE PRODUCT PROFILE<br>Purely synthetic cannabidiol (CBD); devoid of THC; Permeation enhanced gel<br>LEAD PROGRAM IN FRAGILE X SYNDROME (FXS)<br>Additional opportunity in related disorder, 22q deletion syndrome (22q)<br>MARKET OPPORTUNITY<br>~80,000 patients in the US with FXS; similar for 22q; Worldwide rights<br>VERY HIGH UNMET NEED<br>No approved products for FXS or 22q<br>Would be first approved treatment for patients with FXS |
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| Pitolisant HD:<br>Phase 3 Registrational Trials in Narcolepsy and IH in Q4 2025<br>12<br>Initiation of Phase 3<br>Registrational Trials<br>of Pitolisant HD in<br>Narcolepsy and IH<br>OPTIMIZED PK PROFILE AND HIGHER DOSE<br>• Designed to address the need for greater efficacy in EDS and other<br>symptoms in patients with central disorders of hypersomnolence<br>PROVISIONAL IP FILED TO EXTEND PITOLISANT<br>FRANCHISE INTO 2040’S<br>PROGRAMS TO PURSUE A DIFFERENTIATED LABEL<br> Fatigue in narcolepsy; sleep inertia in idiopathic hypersomnia<br>NARCOLEPSY AND IH PHASE 3 REGISTRATIONAL TRIALS<br>TO BE INITIATED Q4 2025<br>Topline data readout anticipated 2027; PDUFA targeted for 2028 |
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| EPX-100: Most Advanced and Differentiated 5HT2 (serotonin)<br>Agonist Development Program<br>13<br>EPX-100<br>PROVEN MoA<br>Established serotonergic mechanism of action – confirmed via highly predictive<br>zebra-fish model<br>PROVEN SAFETY<br>• Clemizole was marketed for ~ 20 yrs with no significant safety and/or<br>tolerability signals from post marketing exposure<br>• Emerging safety and tolerability profile from the Phase 3 study in DS is<br>promising; no need for special laboratory or cardiac monitoring required<br>DOSING REGIMEN<br>BID dosing; very important clinical consideration for patients with DEEs<br>ADVANCED CLINICAL DEVELOPMENT<br>• Actively enrolling patients in US and EU in a Phase 3 registrational trial in<br>Dravet syndrome; Topline data in 2026<br>• Initiated Phase 3 registrational study in LGS in Q4 2024; topline Data in 2026 |
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| Financial Highlights<br>Net Product<br>Revenue<br>Non-GAAP<br>Adjusted<br>Net Income(1)<br>Cash, Cash<br>Equivalents<br>& Investments<br>$168.4<br>$201.3<br>Q4 2023 Q4 2024<br>$42.8 $63.0<br>$582.0<br>$714.7<br>2023 YTD 2024 YTD<br>$188.4 $233.9<br>2023 YTD 2024 YTD<br>$425.6 $453.6 $504.7<br>$576.1<br>Dec 31 '24<br>(1) Non-GAAP Adjusted Net Income= GAAP Net Income excluding non-cash interest expense, depreciation, amortization, stock-based compensation, other non-operating items and tax effect of these items<br>20% 23%<br>24%<br>(In millions, USD) Three Months Ended<br> December 30, 2024<br>Q4 2023 Q4 2024<br>47%<br>Twelve Months Ended<br> December 30, 2024<br>14<br>14<br>$434.1<br>Dec 31 ‘23 Mar 31 ‘24 Jun 30 ‘24 Sep 30 ‘24<br>+$71 |
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| Financial Summary<br>NM denotes not meaningful % change<br>(1) Includes upfront licensing fee of $25.5M related to the 2024 Bioprojet Sublicense Agreement and IPR&D charge of $17.1M related to the acquisition of Epygenix for the twelve months ended December 31, 2024<br>(In millions, USD)<br>Totals may not foot due to rounding<br>Three Months Ended<br>December 31, % Change<br>Twelve Months Ended<br>December 31, % Change<br>2024 2023 2024 2023<br>Net Product Revenue $201.3 $168.4 20% $714.7 $582.0 23%<br>Cost of Product Sold 54.4 43.2 26% 156.8 121.2 29%<br>Total Operating Expenses $91.1 $85.1 7% $367.1 $268.8 37%<br>R&D Expense (1) 34.7 30.3 14% 145.8 76.1 92%<br>S&M Expense 27.6 26.9 3% 110.9 97.4 14%<br>G&AExpense 28.9 27.9 4% 110.4 95.3 16%<br>Net Income $49.5 $26.6 86% $145.5 $128.9 13%<br>Cash, cash equivalents &<br>investments<br>$576.1<br>15 |
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| GAAP vs NON-GAAP Reconciliation<br>(In millions, USD)<br>Totals may not foot due to rounding<br>Three Months Ended<br>December 31,<br>Twelve Months Ended<br>December 31,<br>2024 2023 2024 2023<br>GAAP net income $49.5 $26.6 $145.5 $128.9<br>Non-cash interest expense(1) 0.2 0.2 0.7 3.2<br>Depreciation 0.0 0.2 0.3 0.5<br>Amortization(2) 6.0 6.0 23.8 23.8<br>Stock-based compensation expense 9.9 8.9 42.7 31.2<br>Licensing fee and milestone payments(3) - - 26.5 0.8<br>Loss on debt extinguishment(6) - - - 9.8<br>Transaction related costs(4) - 9.8 17.1 9.8<br>Income tax effect related to Non-GAAP adjustments(5) (2.4) (8.8) (22.7) (19.6)<br>Non-GAAP adjusted net income $63.0 $42.8 $233.9 $188.4<br>GAAP net income per diluted share $0.85 $0.45 $2.51 $2.13<br>Non-GAAP adjusted net income per diluted share $1.08 $0.73 $4.04 $3.12<br>Weighted average number of shares of common stock used in non-GAAP<br>diluted per share<br>58,218,052 58,853,292 57,869,915 60,372,397<br>(1) Includes amortization of deferred finance charges.<br>(2) Includes amortization of intangible asset related to WAKIX.<br>(3) Amount represents upfront licensing fee incurred upon closing the 2024 Bioprojet Sublicense Agreement and milestones related to HBS102 in September 2024 and March 2023.<br>(4) Includes IPR&D charge related to the acquisition of Epygenix.<br>(5) Calculated using the reported effective tax rate for the periods presented less impact of discrete items.<br>(6) Includes loss on extinguishment of the Blackstone Credit Agreement.<br>16 |
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| 17<br>Commitment to patients<br>Addressing unmet medical needs<br>Delivering meaningful treatment options<br>Helping patients thrive<br>DELIVER ON<br>PROMISE<br>TO PATIENTS<br>Innovative<br>Catalyst-rich pipeline<br>Profitable biotech company<br>Meaningful investment opportunity<br>DELIVER<br>STONG VALUE<br>TO SHAREHOLDERS |
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| company/harmonybiosciences/ @harmonybio harmony_biosciences<br>www.harmonybiosciences.com |
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