Sleep Paralysis

WAKIX® (pitolisant) Net Revenue of $168.4 Million for Fourth Quarter and $582.0 Million for Full Year 2023; Representing Growth of ~31% and ~33%, Respectively U.S. Food and Drug Administration (FDA) Granted Priority Review for WAKIX in Pediatric Narcolepsy; PDUFA Date of June 21, 2024 Meeting with FDA to Discuss Idiopathic Hypersomnia Development Program Scheduled for March 2024 FDA Granted Orphan Drug Designation to Pitolisant for the Treatment of Prader-Willi Syndrome; On Track to Initiate Phase 3 TEMPO Study in First Quarter 2024 2024 Net Product Revenue Projected Between $700 -$720 Million Conference Call and Webcast to be Held Today at 8:30 a.m. ET PLYMOUTH MEETING, Pa., Feb. 22, 2024 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today reported net revenue growth of more than 30% for the fourth quarter and full year ended December 31, 2023. “Harmony delivered another year of outstanding performance in 2023, with continued strong growth for WAKIX, demonstrating its durability going into year five on the market,” stated Jeffrey M. Dayno, M.D., President and Chief Executive Officer of Harmony. “In addition to our strong commercial execution, we advanced all our clinical development programs for pitolisant, moved the Next-Generation formulations of pitolisant into the clinic, expanded our pipeline and diversified our portfolio with the acquisition of Zynerba and the ongoing Phase 3 trial in Fragile X syndrome. Harmony continues to be a growth story and we look forward to continued strong execution in 2024.” Key Business Updates FDA granted priority review of supplemental new drug application (sNDA) for WAKIX in pediatric narcolepsy; PDUFA date of June 21, 2024Meeting with the FDA to discuss Idiopathic Hypersomnia (IH) development program scheduled for March 2024FDA granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS); on track to initiate the Phase 3 TEMPO study in patients with PWS in the first quarter of 2024Reported positive Phase 2 proof-of-concept (POC) data in EDS and fatigue in Myotonic Dystrophy Type 1 (DM1) in the fourth quarter of 2023On track to report pharmacokinetic (PK) data on next-gen pitolisant-based formulations in the first half of 2024Expect to complete patient enrollment in the Phase 3 pivotal RECONNECT trial for Fragile X syndrome (FXS) in the first quarter of 2025 with topline data expected in mid-2025On track to report pre-clinical POC data for HBS-102 in PWS in the first half of 2024Repurchased approximately 3.2 million shares of common stock for $100 million in full-year 2023 and expect to continue the opportunistic repurchase of shares under the remaining share repurchase program authorization of $150 million. Fourth Quarter 2023 Financial Results Net product revenue for the quarter ended December 31, 2023 was $168.4 million, compared to $128.3 million for the same period in 2022. The 31% growth versus the same period in 2022 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 US). The average number of patients on WAKIX increased by approximately 350 sequentially to approximately 6,150 for the quarter ended December 31, 2023. GAAP net income for the quarter ended December 31, 2023, was $26.6 million, or $0.45 per diluted share, compared to GAAP net income of $48.5 million, or $0.79 per diluted share, for the same period in 2022. Non-GAAP adjusted net income was $42.8 million, or $0.73 per diluted share, for the quarter ended December 31, 2023, compared to Non-GAAP adjusted net income of $61.9 million, or $1.01 per diluted share, for the same period in 2022. Reconciliations of applicable GAAP financial measures to Non-GAAP financial measures are included at the end of this press release. Harmony’s operating expenses include the following: Research and Development expenses were $30.3 million in the fourth quarter of 2023, as compared to $10.1 million for the same quarter in 2022, representing a 200% increase. Expenses in the fourth quarter of 2023 included one-time Zynerba transaction related costs of $6.0 million;Sales and Marketing expenses were $26.9 million in the fourth quarter of 2023, as compared to $21.1 million for the same quarter in 2022, representing a 28% increase;General and Administrative expenses were $27.9 million in the fourth quarter of 2023, as compared to $22.6 million for the same quarter in 2022, representing a 23% increase. Expenses in the fourth quarter of 2023 included one-time Zynerba transaction related costs of $3.8 million; andTotal Operating Expenses were $85.1 million in the fourth quarter of 2023, as compared to $53.8 million for the same quarter in 2022, representing a 58% increase. Total Operating Expenses in the fourth quarter of 2023 included one-time Zynerba transaction related costs of $9.8 million. Full Year 2023 Financial Results Net product revenue for the year ended December 31, 2023 was $582.0 million, compared to $437.9 million for 2022. The 33% growth versus the same period in 2022 is primarily attributed to strong commercial sales of WAKIX driven by continued organic demand tapping into a large market opportunity. GAAP net income for the year ended December 31, 2023, was $128.9 million, or $2.13 per diluted share, compared to GAAP net income of $181.5 million, or $2.97 per diluted share, for 2022. The decrease in GAAP net income was primarily driven by the release of the valuation allowance on our deferred tax assets, which resulted in a $74.5 million income tax benefit for the year ended December 31, 2022. Non-GAAP adjusted net income was $188.4 million, or $3.12 per diluted share, for the year ended December 31, 2023, compared to Non-GAAP adjusted net income of $183.5 million, or $3.00 per diluted share, for 2022. Reconciliations of applicable GAAP financial measures to Non-GAAP financial measures are included at the end of this press release. Harmony’s operating expenses include the following: Research and Development expenses were $76.1 million for the year ended December 31, 2023, as compared to $70.9 million for the prior year, representing a 7% increase. Expenses in 2023 included one-time Zynerba transaction related costs of $6.0 million;Sales and Marketing expenses were $97.4 million for the year ended December 31, 2023, as compared to $79.3 million for the prior year, representing a 23% increase;General and Administrative expenses were $95.3 million for the year ended December 31, 2023, as compared to $84.0 million for the prior year, representing a 13% increase. Expenses in 2023 included one-time Zynerba transaction related costs of $3.8 million; andTotal Operating Expenses were $268.8 million for the year ended December 31, 2023, as compared to $234.2 million for the prior year, representing a 15% increase. Total Operating Expenses in 2023 included one-time Zynerba transaction related costs of $9.8 million. As of December 31, 2023, Harmony had cash, cash equivalents and investment securities of $425.6 million, compared to $345.7 million on December 31, 2022. 2024 Net Product Revenue Guidance Expect full year 2024 net product revenue of $700 million to $720 million. Conference Call Today at 8:30 a.m. ET We are hosting our fourth quarter and full year 2023 financial results conference call and webcast today 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) 579-2543 (domestic) or +1 (785) 424-1789 (international), and reference passcode HRMYQ423. Non-GAAP Financial MeasuresIn 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) TabletsWAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy and has been commercially available in the U.S. since Q4 2019. 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 UsageWAKIX is indicated for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. Important Safety Information ContraindicationsWAKIX 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 PrecautionsWAKIX 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 (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD). Adverse ReactionsIn the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and 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 infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash. Drug InteractionsConcomitant 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 (see full prescribing information). 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. Therefore, reduced effectiveness of sensitive CYP3A4 substrates may occur when used concomitantly with WAKIX. The effectiveness of hormonal contraceptives may be reduced when used with WAKIX and effectiveness may be reduced for 21 days after discontinuation of therapy. Use in Specific PopulationsWAKIX may reduce the effectiveness of 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. 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 in patients less than 18 years of age. 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 moderate or severe renal impairment. 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 NarcolepsyNarcolepsy 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 HypersomniaIdiopathic 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 Prader-Willi SyndromePWS is an orphan/rare, genetic neurological disorder with many of the symptoms resulting from hypothalamic dysfunction. The hypothalamus is the part of the brain that controls both sleep-wake state stability and signals that mediate the balance between hunger and satiety, resulting in two of the main symptoms in patients with PWS; EDS and hyperphagia (an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety). Other features include low muscle tone, short stature, behavioral problems, and cognitive impairment. Approximately 15,000 to 20,000 people in the U.S. live with PWS, and over half of them experience EDS and the majority of them have behavioral disturbances. About Myotonic Dystrophy Type 1 Myotonic dystrophy Type 1 (DM1) is the most common form of adult-onset muscular dystrophy. It is a genetic disorder inherited in an autosomal-dominant pattern. Latest estimates suggest a prevalence of about one per 2,100 people with the genetic defect for DM1. This equates to about 150,000 people in the U.S. with the genetic defect for DM1. Estimates suggest there are 40,000 people currently diagnosed with DM1 in the U.S., with up to 90% of them reporting EDS and fatigue and over 60% of them experiencing cognitive dysfunction. About Fragile X SyndromeFragile 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 ZYN002ZYN002 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 HBS-102HBS-102, an investigational compound, is a melanin-concentrating hormone (MCH) receptor 1 (MCHR1) antagonist that targets MCH neurons in the brain. It has the potential to be a first-in-class molecule with a novel mechanism of action that could offer a new approach to the treatment of a variety of rare neurological diseases. About Harmony BiosciencesAt Harmony Biosciences, we specialize in developing and delivering treatments for rare neurological diseases that others often overlook. We believe that where empathy and innovation meet, a better life can begin for people living with neurological diseases. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, PA, our team of experts from a wide variety of disciplines and experiences is driven by our shared conviction that innovative science translates into therapeutic possibilities for our patients, who are at the heart of everything we do. For more information, please visit www.harmonybiosciences.com. Forward-Looking StatementsThis 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 product WAKIX and our future capabilities following the acquisition of Zynerba. 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 WAKIX, 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 development activities with Bioprojet, and plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; 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 of our product candidates, including those we are developing with Bioprojet; our failure to achieve the potential benefits of the 2022 LCA with Bioprojet; our ability to recognize the intended benefits of our acquisition of Zynerba Pharmaceuticals; our estimates regarding expenses, future revenue, capital requirements and needs for additional financing; our ability to identify 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 HOLDINGS, INC. AND SUBSIDIARY CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) (In thousands, except share and per share data) Three Months Ended Year Ended December 31, December 31, December 31, December 31, 2023 2022 2023 2022 Net product revenue$168,412 $128,308 $582,022 $437,855 Cost of product sold 43,152 26,885 121,236 83,481 Gross profit 125,260 101,423 460,786 354,374 Operating expenses: Research and development 30,306 10,092 76,063 70,886 Sales and marketing 26,886 21,075 97,404 79,285 General and administrative 27,872 22,643 95,289 84,017 Total operating expenses 85,064 53,810 268,756 234,188 Operating income 40,196 47,613 192,030 120,186 Loss on debt extinguishment — — (9,766) — Other expense (income), net 193 73 159 169 Interest expense (4,796) (5,444) (23,757) (18,795)Interest income 4,096 1,861 14,730 3,126 Income before income taxes 39,689 44,103 173,396 104,686 Income tax benefit (expense) (13,082) 4,406 (44,543) 76,782 Net income$26,607 $48,509 $128,853 $181,468 EARNINGS PER SHARE: Basic$0.46 $0.82 $2.17 $3.07 Diluted$0.45 $0.79 $2.13 $2.97 Weighted average number of shares of common stock - basic 58,320,400 59,478,933 59,469,648 59,173,121 Weighted average number of shares of common stock - diluted 58,853,292 61,620,712 60,372,397 61,097,045 HARMONY BIOSCIENCES HOLDINGS, INC. AND SUBSIDIARYCONSOLIDATED BALANCE SHEETS(In thousands, except share and per share data) December 31, December 31, 2023 2022 ASSETS CURRENT ASSETS: Cash and cash equivalents$311,660 $243,784 Investments, short-term 41,800 79,331 Trade receivables, net 74,140 54,740 Inventory, net 5,363 4,297 Prepaid expenses 12,570 9,347 Other current assets 5,537 8,786 Total current assets 451,070 400,285 NONCURRENT ASSETS: Property and equipment, net 371 573 Restricted cash 270 750 Investments, long-term 72,169 22,568 Intangible assets, net 137,108 160,953 Deferred tax asset 144,162 85,943 Other noncurrent assets 6,298 2,798 Total noncurrent assets 360,378 273,585 TOTAL ASSETS$811,448 $673,870 LIABILITIES AND STOCKHOLDERS’ EQUITY CURRENT LIABILITIES: Trade payables$17,730 $3,786 Accrued compensation 23,747 11,532 Accrued expenses 99,494 59,942 Current portion of long-term debt 15,000 2,000 Other current liabilities 7,810 1,624 Total current liabilities 163,781 78,884 NONCURRENT LIABILITIES: Long-term debt, net 178,566 189,647 Other noncurrent liabilities 2,109 2,501 Total noncurrent liabilities 180,675 192,148 TOTAL LIABILITIES 344,456 271,032 COMMITMENTS AND CONTINGENCIES (Note 13) STOCKHOLDERS’ EQUITY: Common stock—$0.00001 par value; 500,000,000 shares authorized at December 31, 2023 and December 31, 2022, respectively; - shares 56,769,081 and 59,615,731 issued and outstanding at December 31, 2023 and December 31, 2022, respectively 1 1 Additional paid in capital 610,266 675,118 Accumulated other comprehensive (loss) income 2 (151)Accumulated deficit (143,277) (272,130)TOTAL STOCKHOLDERS’ EQUITY 466,992 402,838 TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY$811,448 $673,870 HARMONY BIOSCIENCES HOLDINGS, INC.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, 2023 2022 2023 2022 GAAP net income$26,607 $48,509 $128,853 $181,468 Non-GAAP Adjustments: Non-cash interest expense (1) 185 422 3,246 1,663 Depreciation 164 107 514 419 Amortization (2) 5,961 5,961 23,845 22,966 Stock-based compensation expense 8,894 7,671 31,205 26,905 Transaction related costs (3) 9,804 - 9,804 - Loss on debt extinguishment - - 9,766 - Licensing fees and milestone payments (4) - - 750 30,000 Valuation allowance release - - - (74,474)Income tax effect related to non-GAAP adjustments (5) (8,789) (731) (19,624) (5,409)Non-GAAP adjusted net income$42,826 $61,939 $188,359 $183,538 GAAP reported net income per diluted share$0.45 $ 0.79 $2.13 $ 2.97 Non-GAAP adjusted net income per diluted share$0.73 $1.01 $3.12 $3.00 Weighted average number of shares of common stock used in non-GAAP diluted per share 58,853,292 61,620,712 60,372,397 61,097,045 (1) Includes amortization of deferred finance charges.(2) Includes amortization of intangible asset related to WAKIX.(3) Includes costs associated with the acquisition of Zynerba in October 2023. There were $2.3M of IPR&D charges and $3.7M of severance recorded in research and development expenses and $3.8M of severance recorded in general and administrative expenses. (4) Includes a $0.8M milestone payment related to HBS-102 preclinical milestone in March 2023 and $30M licensing fee incurred upon closing the 2022 Licensing and Commercialization Agreement with Bioprojet in August 2022. (5) Calculated using the reported effective tax rate for the periods presented less impact of valuation allowance release and discrete items. Harmony Biosciences Investor Contact:Luis Sanay, CFA445-235-8386lsanay@harmonybiosciences.com Harmony Biosciences Media Contact:Cate McCanless202-641-6086cmccanless@harmonybiosciences.com

Arimoclomol NDA resubmission remains on track for end of year filing Proposed acquisition of Acer Therapeutics on track to close Q4 2023 and will diversify Zevra’s revenue with FDA-approved OLPRUVA® for urea cycle disorders (UCDs) Net revenue of $2.9M for Q3 2023 Ended Q3 2023 with $83.4M in cash, cash equivalents, and investments, supporting our forecasted cash runway into 2026 Conference call and live audio webcast scheduled for today, Nov 7, 2023, 8:00 a.m. ET CELEBRATION, Fla., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the quarter ended September 30, 2023. “This is a pivotal time for Zevra as we advance our pipeline programs toward multiple potential value inflection points,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra. “As we look to the end of 2023 and into 2024, we’re focused on three key priorities. First, to close the proposed Acer acquisition and, if consummated, deliver value to patients by commercializing OLPRUVA. Second, to resubmit the arimoclomol NDA. Third, to complete the Phase 2 trial in idiopathic hypersomnia and prepare to advance KP1077 into Phase 3. We are making solid progress towards achieving our mission of building a patient-focused leading rare disease company.” Recent Business and Corporate Highlights: Corporate: Neil F. McFarlane was named President and Chief Executive Officer of Zevra and appointed to the Board of Directors on October 10, 2023. Neil succeeds Christal M.M. Mickle who has been serving as interim CEO and President since June 2023, and who will continue serving in her role as Chief Development Officer. Joseph B. Saluri’s previously announced retirement from the Board of Directors also became effective contemporaneous with Neil’s appointment.Arimoclomol: The new drug application (NDA) for arimoclomol is on track to be resubmitted to the U.S. Food and Drug Administration (FDA) by the end of this year. Arimoclomol is an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC), an ultra-rare, genetic, progressive and fatal neurological disease. If approved, arimoclomol has the potential to be a first-in-class, orally-delivered treatment for NPC. Because a significant amount of new data has been added to the updated NDA, it is expected to be classified as a Class II submission, which will be subject to a six-month review period by the FDA. Arimoclomol is eligible to receive a Priority Review Voucher, if approved.In parallel with the ongoing regulatory work to resubmit the arimoclomol NDA, we are preparing for the potential commercial launch of arimoclomol in the U.S., if approved, with the goal of making this groundbreaking therapy accessible to patients as soon as possible.The Company intends to complete the regulatory pathway for arimoclomol in the U.S., and then continue the evaluation and identification of the optimal regulatory pathway to approval for arimoclomol in the E.U. KP1077: On October 2, 2023, the Company reported interim data from the open-label dose titration phase of its Phase 2 clinical trial evaluating KP1077 in patients with idiopathic hypersomnia (IH). Results demonstrated KP1077 was well-tolerated at all dose levels and both dosing regimens. The interim data related to the secondary and exploratory endpoints showed marked improvements in patient-reported assessments of key IH symptoms from the open-label titration phase, including excessive daytime sleepiness (EDS), sleep inertia, and brain fog.Top-line data from the completed trial are expected in the first half of 2024 after all patients have completed the double-blind withdrawal phase. The combined open-label interim and upcoming topline data are expected to also provide information related to secondary and exploratory endpoints, including excessive daytime sleepiness, sleep inertia, and brain fog. The results from the completed Phase 2 trial will inform the final design of the anticipated Phase 3 trial in IH expected to be initiated by the end of 2024.Phase 1 clinical trial under the narcolepsy IND was completed for KP1077 in healthy volunteers. Data generated from this trial will be analyzed alongside the Phase 2 IH data to support clinical development of both narcolepsy and IH programs. Acer Therapeutics, Inc. (Acer) Acquisition: The acquisition of Acer, announced by Zevra on August 31, 2023, and subject to customary closing conditions including approval by Acer shareholders, would bring multiple rare disease assets that increase and diversify Zevra’s revenue potential with the addition of OLPRUVA®, which is indicated for the treatment of urea cycle disorders (UCDs), and expand its clinical development portfolio with the addition of EDSIVO™, currently in Phase 3, for vascular Ehlers-Danlos syndrome (vEDS). Acer’s programs strategically complement Zevra’s rare disease portfolio and commercial assets and increase and diversify revenues.OLPRUVA’s commercial operations and capabilities would provide scale and cost synergies to support and accelerate launch and commercialization of arimoclomol, if approved.Acer’s special meeting of shareholders to approve the transaction is scheduled for November 8, 2023. Cash Runway: Balance sheet remains strong with $83.4 million in cash, cash equivalents, and investments as of September 30, 2023, which supports our forecasted operating cash runway into 2026. Cash runway forecast includes the ongoing reimbursements from the French expanded access program for arimoclomol, completion of the arimoclomol NDA resubmission, pull forward of commercial activities to support the launch of arimoclomol, if approved, and completion of the KP1077 development program for IH.Cash runway forecast does not include commercial revenue from arimoclomol following potential FDA approval, or the potential sale of the Priority Review Voucher, which would be received upon approval, or any revenue from sales of OLPRUVA. Overview of Q3 2023 Financial Results: Net revenue for Q3 2023 was $2.9 million compared to prior year Q3 net revenue of $2.9 million. The components of revenue during the current quarter include ongoing royalties from AZSTARYS® and reimbursements from the French early access program for arimoclomol. Research and development (R&D) expenses were $12.3 million for Q3 2023, compared to $5.4 million in Q3 2022. The increase in R&D expenses were primarily driven by the ongoing Phase 2 clinical trial in KP1077, along with the ongoing work to prepare the arimoclomol NDA for resubmission. General and administrative (G&A) expenses were $5.8 million for Q3 2023, compared to $4.0 million in Q3 2022. The period-over-period increase was primarily related to an increase in personnel costs and professional fees associated with our commercial and business development activities. Net loss for Q3 2023 was ($14.0) million, or ($0.40) per basic and diluted share, compared to a net loss of ($6.6) million, or ($0.19) per basic and diluted share for the same period in 2022. As of September 30, 2023, total cash, cash equivalents, and cash investments were $83.4 million, a decrease of ($4.0) million compared to $87.4 million as of June 30, 2023. The decrease was driven, in part, by increased third-party R&D costs related to the KP1077 clinical trial program, the arimoclomol program, and increased G&A expenses during the period. Concomitant with the signing of the merger agreement and announcement of the Acer transaction, Zevra purchased Acer’s outstanding senior secured debt for an amount totaling $28.6 million, and provided a bridge loan to of up to $16.5 million to Acer to provide funding for Acer’s commercial efforts related to OLPRUVA and other corporate purposes. As of September 30, 2023, these assets are included in secured corporate notes in the unaudited condensed consolidated balance sheet and totaled $42.0 million as of September 30, 2023. Upon closing of the Acer transaction, if approved, these amounts are expected to be eliminated as an inter-company transaction. As of September 30, 2023, total shares of common stock outstanding were 36,211,710, and fully diluted common shares outstanding were 51,598,902, which included 4,252,490 shares issuable upon exercise of warrants. Conference Call Information: Zevra will host a conference call and live audio webcast today at 8:00 a.m. ET, to discuss its corporate and financial results for Q3 2023. The audio webcast will be accessible via the Investor Relations section of the Company’s website, http://investors.zevra.com/. An archive of the audio webcast will be available for 90 days beginning at approximately 9:00 a.m. ET, on November 7, 2023. Additionally, interested participants and investors may access the conference call by dialing either: (800) 343-4849 (U.S.)+1 (203) 518-9843 (International)Conference ID: ZVRAQ323 About Urea Cycle Disorders: UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments if ammonia levels are not controlled.i Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels. About OLPRUVA®: ACER-001 (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and has recently been marketed under the brand name, OLPRUVA®. OLPRUVA (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Please see Important Safety Information and full Prescribing Information, including Patient Information. Important Safety Information: Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with OLPRUVA. Tell your doctor about all the medicines you or your child take, especially if you or your child take corticosteroids, valproic acid, haloperidol, and/or probenecid. OLPRUVA can cause serious side effects, including: 1) nervous system problems (neurotoxicity). Symptoms include sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA is right for you. The most common side effects of OLPRUVA include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. About Niemann-Pick Disease Type C (NPC): Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease progression is irreversible and can be fatal within months or take years to be diagnosed and advance in severity. About Arimoclomol: Arimoclomol, Zevra’s orally delivered, first-in-class investigational product candidate for the treatment of NPC, has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, and Orphan Medicinal Product designation for the treatment of NPC by the European Medicines Agency (EMA). The arimoclomol NDA is currently being prepared for resubmission to the FDA. About Idiopathic Hypersomnia (IH): Idiopathic hypersomnia (IH) is a rare sleep disorder characterized by excessive daytime sleepiness. Patients with IH experience daytime lapses into sleep, or an irrepressible need to sleep that persists even with adequate or prolonged nighttime sleep. Additionally, those with IH have extreme difficulty waking, otherwise known as “sleep inertia,” severe “brain fog”, and often fall asleep unintentionally or at inappropriate times. These symptoms of IH often lead to further, even more debilitating problems such as memory lapses, difficulty maintaining focus, and depression. It is estimated, based on claims data, that approximately 37,000 patients in the United States are currently diagnosed with IH treatment, although the total patient population may be much larger due to some patients who have not yet been diagnosed, have been misdiagnosed, or are not currently seeking treatment. About Narcolepsy: Narcolepsy is a chronic debilitating central disorder of hypersomnolence. The primary symptom of narcolepsy is excessive daytime sleepiness characterized by daily episodes of an irrepressible need to sleep or daytime lapses into sleep. Patients with narcolepsy have an abnormal rapid eye movement (REM) sleep phase which can cause disrupted nighttime sleep, sleep paralysis and sleep-related hallucinations during sleep-wake transitions. Narcolepsy has severe personal, social, and economic consequences. Patients with narcolepsy experience substantial impairment of their mental and physical wellbeing, and depression and anxiety are common. Cognitive dysfunctions such as difficulty to focus and memory lapses (also referred to as ‘brain fog’) are frequently reported. The many symptoms experienced by patients with narcolepsy result in a high disease burden and poor quality of life. Narcolepsy is categorized in two types: narcolepsy type 1 (NT1) and type 2 (NT2). NT1 is considered a distinct disease entity characterized in part by loss of hypocretin neurons and symptoms of cataplexy (sudden, brief attacks of muscle weakness sometimes resulting in the body to fall uncontrollably, often triggered by strong emotions). When narcolepsy presents without cataplexy and with normal hypocretin-1 concentrations in the cerebrospinal fluid (CSF), it is categorized as NT2 (Hypocretin-1 is also known as orexin-A, a neuropeptide involved in regulating sleep-wake cycles). The combined worldwide prevalence of both types of narcolepsy has been estimated to be 25-50 per 100,000 people. Epidemiological studies using well-defined criteria for assessing the prevalence of narcolepsy (both NT1 and NT2) estimate incidence rates ranging from 31 to 79 per 100,000 people, corresponding to approximately 100,000 to 260,000 total patients in the United States. About SDX and KP1077: Serdexmethylphenidate (SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and the sole active pharmaceutical ingredient (API) in KP1077, Zevra’s lead clinical candidate being developed as a treatment for idiopathic hypersomnia (IH) and narcolepsy. Zevra is currently enrolling for a multicenter, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal Phase 2 clinical trial to evaluate safety and efficacy of KP1077 as a treatment for IH. For more information regarding the Phase 2 trial, visit www.clinicaltrials.gov. KP1077 has been granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of IH, and the U.S. Drug Enforcement Agency (DEA) has classified SDX, the sole API in KP1077, as a Schedule IV controlled substance based on evidence suggesting SDX has a lower potential for abuse when compared to d-MPH, a Schedule II controlled substance. About Zevra Therapeutics: Zevra Therapeutics is a rare disease company melding science, data, and patient need to create transformational therapies for diseases with limited or no treatment options. With unique, data-driven clinical, regulatory, and commercialization strategies, the Company is overcoming complex drug development challenges to bring much-needed therapies to patients. With both regulatory and clinical-stage product candidates, the Company is building its commercial capability to make new therapies available to the rare disease community. Expanded access programs are made available by Zevra Therapeutics and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy as published on its website at zevra.com. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion. Caution Concerning Forward-Looking Statements: This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, and which can be identified by the use of words such as "may," "will," "expect," "project," "estimate," "anticipate," "plan," "believe," "potential," "should," "continue," "could," "intend," "target," "predict," or the negative versions of those words or other comparable words or expressions, although not all forward-looking statements contain these identifying words or expressions. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include without limitation statements regarding the potential sale of the Priority Review Voucher, the promise and potential impact of our preclinical or clinical trial data, including without limitation the initiation, timing and results of any clinical trials or readouts, the content, information used for, timing or results of any IND applications and NDA submissions or resubmissions for arimoclomol, KP1077, or any other product candidates for any specific disease indication or at any dosage, the potential achievement of commercial sales or revenue milestones for AZSTARYS and the timing thereof, the potential launch or commercialization of any of product candidates or products, including in the event of the consummation of the proposed merger transaction, or the timing thereof, the sufficiency of our cash, cash equivalents and investments to fund our operating activities for any specific period of time, statements regarding the proposed merger transaction, its timing and its consummation, the anticipated financial performance of Zevra and Acer related thereto, including the anticipated closing of, benefits of, accounting impact of, and synergies related to the proposed merger transaction, potential strategic implications as a result of the proposed merger transaction, our plans to build out commercial teams for products or product candidates, our commercial infrastructure investments and the impact of the proposed transactions on them, and our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, 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. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2022, as updated in Zevra’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, and Zevra’s other filings with the Securities and Exchange Commission (the SEC). While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Additional Information about the Proposed Merger between Acer and Zevra, the Special Meeting and Where to Find It In connection with the proposed merger, Zevra has filed a registration statement on Form S-4 with the SEC, including a proxy statement / prospectus. The registration statement was declared effective on October 10, 2023. Additionally, Acer’s proxy statement was filed on October 10, 2023. On October 30, 2023, Acer filed a supplement to the proxy statement to make certain supplemental disclosures. Acer shareholders are urged to read these materials because they contain important information about Acer, Zevra and the proposed merger. The proxy statement / prospectus and other relevant materials, including any proxy supplements, and any other documents filed by Zevra and/or Acer with the SEC, may be obtained free of charge at the SEC website at www.sec.gov. In addition, Acer shareholders will be able to attend the Acer special meeting via the Internet at https://www.cstproxy.com/acertx/sm2023 and view the Acer 2023 Special Meeting Proxy Statement and the Zevra Therapeutics, Inc. Forms 10-K, 10-Qs and 8-Ks. Acer shareholders are urged to read the proxy statement / prospectus, including any supplements thereto, and the other relevant materials before making any voting or investment decision with respect to the proposed merger. No Offer or Solicitation This communication is for informational purposes only and not intended to and does not constitute an offer to subscribe for, buy or sell, the solicitation of an offer to subscribe for, buy or sell or an invitation to subscribe for, buy or sell any securities or the solicitation of any vote or approval in any jurisdiction pursuant to or in connection with the proposed transaction or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. No offer of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended, and otherwise in accordance with applicable law. Participants in the Solicitation Zevra, Acer and their respective directors and executive officers may be considered participants in the solicitation of proxies in connection with the proposed transaction. Information about the directors and executive officers of Acer is set forth in its Annual Report on Form 10-K for the year ended December 31, 2022, which was filed with the SEC on March 27, 2023, and its proxy statement for its 2023 annual meeting of shareholders, which was filed with the SEC on April 14, 2023. Information about the directors and executive officers of Zevra is set forth in its Annual Report on Form 10-K for the year ended December 31, 2022, which was filed with the SEC on March 7, 2023, and its proxy statement for its 2023 annual meeting of stockholders, which was filed with the SEC on March 15, 2023, the definitive proxy statement filed by Daniel J. Mangless, together with the other participants named therein, which was filed with the SEC on March 17, 2023, and Zevra’s Current Reports on Form 8-K, filed with the SEC on March 30, 2023, May 8, 2023, May 15, 2023, and August 7, 2023. Other information regarding the participants in the proxy solicitations and a description of their direct and indirect interests, by security holdings or otherwise, are contained in the proxy statement/prospectus and other relevant materials filed with the SEC. Contacts: Nichol Ochsner +1 (732) 754-2545 nochsner@zevra.com Jennifer Arcure+1 (917) 603-0681 Jennifer.arcure@evokegroup.com ZEVRA THERAPEUTICS, INC.UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share amounts) Three months endedSeptember 30, Six months ended September 30, 2023 2022 2023 2022 Revenue, net $2,895 $2,874 $14,244 $8,139 Operating expenses: Cost of revenue 144 141 946 200 Research and development 12,297 5,385 28,574 13,262 Selling, general and administrative 5,818 3,974 19,657 10,266 Acquired in-process research and development — — — 17,663 Total operating expenses 18,259 9,500 49,177 41,391 Loss from operations (15,364) (6,626) (34,933) (33,252)Other (expense) income: Interest expense (366) (124) (745) (165)Fair value adjustment related to derivative and warrant liability — 22 — 295 Fair value adjustment related to investments 124 (139) 451 (634)Interest and other income, net 1,738 218 4,331 482 Total other income (expense): 1,496 (23 4,037 (22)Loss before income taxes (13,868) (6,649) (30,896) (33,274)Income tax benefit (177) 33 — 752 Net loss $(14,045) $(6,616) $(30,896) $(32,522) Basic and diluted net loss per share of common stock: Net loss $(0.40) $(0.19) $(0.90) $(0.94)Weighted average number of shares of common stock outstanding: Basic and diluted 34,724,614 34,494,702 34,364,075 34,482,791 ZEVRA THERAPEUTICS, INC.UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS(in thousands, except share and par value amounts) September 30, December 31, 2023 2022Assets Current assets: Cash and cash equivalents $43,269 $65,466 Securities at fair value 39,672 16,900 Secured corporate notes 41,999 — Short-term investments - other 485 481 Accounts and other receivables 9,927 8,299 Prepaid expenses and other current assets 1,661 1,877 Total current assets: 137,013 93,023 Inventories 481 671 Property and equipment, net 642 794 Operating lease right-of-use assets 698 988 Long-term investments - other — 20,000 Other long-term assets 148 53 Total assets: $139,982 $115,529 Liabilities and stockholders' equity Current liabilities: Accounts payable and accrued expenses $13,080 $6,169 Current portion of operating lease liabilities 433 480 Current portion of discount and rebate liabilities 7,890 4,655 Other current liabilities 311 422 Total current liabilities: 21,714 11,726 Line of credit payable 38,801 12,800 Secured promissory note 5,073 — Operating lease liabilities, less current portion 517 843 Discount and rebate liabilities, less current portion 4,987 4,327 Other long-term liabilities 420 26 Total liabilities: 71,512 29,722 Commitments and contingencies Stockholders’ equity: Preferred stock: Undesignated preferred stock, $0.0001 par value, 10,000,000 shares authorized, no shares issued or outstanding as of September 30, 2023, or December 31, 2022 — — Common stock, $0.0001 par value, 250,000,000 shares authorized, 37,787,402 shares issued and 36,211,710 shares outstanding as of June 30, 2023; 35,450,257 shares issued and 34,540,304 shares outstanding as of December 31, 2022 3 3 Additional paid-in capital 418,138 401,799 Treasury stock, at cost (10,983) (7,536)Accumulated deficit (339,468) (308,572)Accumulated other comprehensive (loss) income (220) 113 Total stockholders' equity: 67,470 85,807 Total liabilities and stockholders' equity: $138,982 $115,529 i Ah Mew N, et al. Urea cycle disorders overview [updated June 22, 2017]. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews® [Internet]. University of Washington; 1993-2022. Accessed March 20, 2022.

Continued Strong Growth with WAKIX® (pitolisant) Net Revenue of $160.3 Million for Third Quarter 2023; Increased ~37% Year-over-Year Average Number of Patients on WAKIX Increased by ~350 Sequentially to ~5,800 for Third Quarter 2023 Remain Committed and Continue to Pursue Idiopathic Hypersomnia Indication; Next Step to Meet with FDA Informed by Review of Full Data Set Expanded and Diversified Pipeline with Acquisition of Zynerba Pharmaceuticals; Zygel™ in Pivotal Phase 3 Trial for Fragile X syndrome Repurchased ~1.4 Million Shares of Common Stock for $50 Million in Third Quarter 2023; Board of Directors Authorized New $200 Million Share Repurchase Program Conference Call and Webcast to be Held Today at 8:30 a.m. ET PLYMOUTH MEETING, Pa., Oct. 31, 2023 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. (“Harmony” or the “Company”) (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today reported financial results and business updates for the quarter ended September 30, 2023. “We continue to demonstrate very strong growth in our commercial business, with WAKIX in narcolepsy delivering the strongest revenue quarter in Harmony's history,” stated Jeffrey M. Dayno, M.D., President and Chief Executive Officer of Harmony. “In addition, we advanced our pitolisant pipeline programs, and expanded our pipeline with the addition of Zygel through the closing of the Zynerba acquisition.” “Given our continued confidence in the underlying strength of the business and our conviction in the growth potential for the company, we are announcing a new share repurchase program of $200 million.” Third Quarter 2023 Financial Results Net product revenues for the quarter ended September 30, 2023 were $160.3 million, compared to $117.2 million for the same period in 2022. The 37% growth versus the same period in 2022 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 United States). The average number of patients on WAKIX increased by approximately 350 sequentially to approximately 5,800 for the quarter ended September 30, 2023. GAAP net income for the quarter ended September 30, 2023 was $38.5 million, or $0.63 earnings per diluted share, compared to GAAP net income of $87.9 million, or $1.44 earnings per diluted share, for the same period in 2022. The decrease in GAAP net income was primarily driven by the release of the valuation allowance on our deferred tax assets, which resulted in a $74.5 million income tax benefit for the quarter ended September 30, 2022, partially offset by a $30.0 million initial licensing fee as part of the 2022 Licensing and Commercialization Agreement with Bioprojet (the “2022 LCA”). For the quarter ended September 30, 2023, we also incurred a $9.8 million loss on debt extinguishment. Non-GAAP adjusted net income was $58.8 million, or $0.97 earnings per diluted share, for the quarter ended September 30, 2023, compared to Non-GAAP adjusted net income of $58.1 million, or $0.95 per diluted share, for the same period in 2022. Reconciliations of applicable GAAP financial measures to Non-GAAP financial measures are included at the end of this press release. Harmony’s operating expenses include the following: Research and Development expenses were $17.5 million in the third quarter of 2023, as compared to $40.5 million for the same quarter in 2022, representing a 57% decrease, driven by a $30.0 million licensing fee as part of the 2022 LCA incurred during the third quarter of 2022;Sales and Marketing expenses were $23.4 million in the third quarter of 2023, as compared to $20.5 million for the same quarter in 2022, representing a 14% increase;General and Administrative expenses were $22.5 million in the third quarter of 2023, as compared to $21.3 million for the same quarter in 2022, representing a 6% increase; andTotal Operating Expenses were $63.5 million in the third quarter of 2023, as compared to $82.3 million for the same quarter in 2022, representing a 23% decrease. As of September 30, 2023, Harmony had cash, cash equivalents and investment securities of $438.4 million, compared to $345.7 million as of December 31, 2022. Company Updates Reported topline results from the Phase 3 INTUNE study in adult patients with idiopathic hypersomnia (IH). While the primary endpoint did not reach statistical significance during the randomized withdrawal phase, a robust clinical effect was demonstrated in the open label phase of the study. Based on the totality of the data, the company remains committed and continues to pursue an indication for pitolisant in IH. Next step is to meet with the FDA informed by the review of the full data set. Received FDA alignment on the protocol for the Phase 3 TEMPO study in patients with Prader-Willi syndrome (PWS) which will satisfy the requirements for both the registrational trial and now pediatric exclusivity as well. We expect to initiate the study in the first quarter of 2024. On track for topline data from the Myotonic Dystrophy (DM1) Phase 2 proof-of-concept signal detection trial in the fourth quarter of 2023. On track to submit a supplemental new drug application (sNDA) for a pediatric narcolepsy indication to the FDA in the fourth quarter of 2023.Advancing new pitolisant based formulations into the clinic in the fourth quarter of 2023. Anticipate data in the first half of 2024.Expanded and diversified our pipeline with the acquisition of Zynerba Pharmaceuticals. Zygel is in a Pivotal Phase 3 trial for Fragile X syndrome (FXS).During the third quarter of 2023, the company repurchased 1,439,792 shares of common stock at an aggregate cost of $50 million, as part of the prior $125 million share repurchase program, which we closed.Our Board of Directors authorized a new $200 million share repurchase program. Conference Call Today at 8:30 a.m. ET We are hosting our third quarter 2023 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) 245-3047 (domestic) or +1 (203) 518-9765 (international), and reference passcode HRMYQ323. Non-GAAP Financial MeasuresIn 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) TabletsWAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy and has been commercially available in the U.S. since Q4 2019. 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 UsageWAKIX is indicated for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. Important Safety Information ContraindicationsWAKIX 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 PrecautionsWAKIX 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 (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD). Adverse ReactionsIn 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. Drug InteractionsConcomitant 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 (see full prescribing information). 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. Therefore, reduced effectiveness of sensitive CYP3A4 substrates may occur when used concomitantly with WAKIX. The effectiveness of hormonal contraceptives may be reduced when used with WAKIX and effectiveness may be reduced for 21 days after discontinuation of therapy. Use in Specific PopulationsWAKIX may reduce the effectiveness of 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. 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 in patients less than 18 years of age. 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 moderate or severe renal impairment. 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 NarcolepsyNarcolepsy is a rare, chronic, debilitating neurological disease of sleep-wake state instability that impacts approximately 165,000 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 HypersomniaIdiopathic 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 Prader-Willi SyndromePWS is an orphan/rare, genetic neurological disorder with many of the symptoms resulting from hypothalamic dysfunction. The hypothalamus is the part of the brain that controls both sleep-wake state stability and signals that mediate the balance between hunger and satiety, resulting in two of the main symptoms in patients with PWS; EDS and hyperphagia (an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety). Other features include low muscle tone, short stature, behavioral problems, and cognitive impairment. Approximately 15,000 to 20,000 people in the U.S. live with PWS, and over half of them experience EDS and the majority of them have behavioral disturbances. About Zygel™ Zygel is the first and only pharmaceutically manufactured, synthetic cannabidiol, non-euphoric cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. Zygel is manufactured through a synthetic process in a cGMP facility and is not extracted from the cannabis plant. Therefore, it is devoid of THC, which is what causes the euphoric effect of cannabis, and has the potential to be a nonscheduled product if approved. Cannabidiol, the active ingredient in Zygel, has been granted orphan drug designation by the FDA and the EMA for the treatment of FXS and for the treatment of 22q11.2 deletion syndrome (22q). Additionally, Zygel has received FDA Fast Track designation for the treatment of behavioral symptoms in patients with FXS. About Fragile X SyndromeFragile X syndrome (FXS) is a rare genetic disorder that is the leading known cause of both inherited intellectual disability and autism spectrum disorder, affecting 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females. 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. There are approximately 80,000 people in the U.S. and approximately 121,000 people in the European Union and UK living with FXS. 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 Harmony BiosciencesAt Harmony Biosciences, we specialize in developing and delivering treatments for rare neurological diseases that others often overlook. We believe that where empathy and innovation meet, a better life can begin for people living with neurological diseases. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, PA, our team of experts from a wide variety of disciplines and experiences is driven by our shared conviction that innovative science translates into therapeutic possibilities for our patients, who are at the heart of everything we do. For more information, please visit www.harmonybiosciences.com. Forward-Looking StatementsThis 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 product WAKIX and our future capabilities following the acquisition of Zynerba. 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 WAKIX, 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 development activities with Bioprojet, and plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; 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 of our product candidates, including those we are developing with Bioprojet; our failure to achieve the potential benefits of the 2022 LCA with Bioprojet; our ability to recognize the intended benefits of our acquisition of Zynerba Pharmaceuticals; our estimates regarding expenses, future revenue, capital requirements and needs for additional financing; our ability to identify 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 21, 2023, 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 HOLDINGS, INC. AND SUBSIDIARY CONSOLIDATEDSTATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (In thousands, except share and per share data) Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022Net product revenues $ 160,268 $ 117,206 $ 413,610 $ 309,547Cost of product sold 32,296 22,959 78,084 56,596Gross profit 127,972 94,247 335,526 252,951Operating expenses: Research and development 17,499 40,548 45,757 60,794Sales and marketing 23,418 20,467 70,518 58,210General and administrative 22,546 21,331 67,417 61,374Total operating expenses 63,463 82,346 183,692 180,378Operating income 64,509 11,901 151,834 72,573Loss on debt extinguishment (9,766) — (9,766) —Other (expense) income, net (5) 56 (34) 96Interest expense, net (2,906) (3,990) (8,327) (12,086)Income before income taxes 51,832 7,967 133,707 60,583Income tax (expense) benefit (13,371) 79,976 (31,461) 72,376Net income $ 38,461 $ 87,943 $ 102,246 $ 132,959Unrealized income (loss) on investments 6 (149) (365) (178)Comprehensive income $ 38,467 $ 87,794 $ 101,881 $ 132,781 EARNINGS PER SHARE: Basic $ 0.64 $ 1.48 $ 1.71 $ 2.25Diluted $ 0.63 $ 1.44 $ 1.68 $ 2.18Weighted average number of shares of common stock - basic 59,863,102 59,234,720 59,856,941 59,070,063Weighted average number of shares of common stock - diluted 60,681,676 61,207,625 60,892,992 60,921,482 HARMONY BIOSCIENCES HOLDINGS, INC.CONSOLIDATED BALANCE SHEETS(In thousands except share and per share data) September 30, December 31, 2023 2022 ASSETS CURRENT ASSETS: Cash and cash equivalents $324,603 $243,784 Investments, short-term 46,071 79,331 Trade receivables, net 67,264 54,740 Inventory, net 5,087 4,297 Prepaid expenses 14,269 9,347 Other current assets 5,704 8,786 Total current assets 462,998 400,285 NONCURRENT ASSETS: Property and equipment, net 428 573 Restricted cash 250 750 Investments, long-term 67,700 22,568 Intangible assets, net 143,069 160,953 Deferred tax asset 100,485 85,943 Other noncurrent assets 2,836 2,798 Total noncurrent assets 314,768 273,585 TOTAL ASSETS $777,766 $673,870 LIABILITIES AND STOCKHOLDERS’ EQUITY CURRENT LIABILITIES: Trade payables $6,539 $3,786 Accrued compensation 10,322 11,532 Accrued expenses 72,761 59,942 Current portion of long-term debt 15,000 2,000 Other current liabilities 7,786 1,624 Total current liabilities 112,408 78,884 NONCURRENT LIABILITIES: Long-term debt, net 182,131 189,647 Other noncurrent liabilities 1,895 2,501 Total noncurrent liabilities 184,026 192,148 TOTAL LIABILITIES 296,434 271,032 COMMITMENTS AND CONTINGENCIES (Note 12) STOCKHOLDERS’ EQUITY: Common stock—$0.00001 par value; 500,000,000 shares authorized at September 30, 2023 and December 31, 2022, respectively; 58,571,944 shares and 59,615,731 issued and outstanding at September 30, 2023 and December 31, 2022, respectively 1 1 Additional paid in capital 651,731 675,118 Accumulated other comprehensive (loss) income (516) (151)Accumulated deficit (169,884) (272,130)TOTAL STOCKHOLDERS’ EQUITY 481,332 402,838 TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY $777,766 $673,870 HARMONY BIOSCIENCES HOLDINGS, INC.RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL RESULTS(In thousands except share and per share data) Three Months Ended Nine Months Ended September 30, September 30, September 30, September 30, 2023 2022 2023 2022 GAAP net income $38,461 $87,943 $102,246 $132,959 Non-GAAP Adjustments: Non-cash interest expense (1) 2,221 418 3,061 1,241 Depreciation 144 101 350 312 Amortization (2) 5,962 5,962 17,884 17,005 Stock-based compensation expense 7,957 6,967 22,311 19,234 Licensing fee (3) - 30,000 - 30,000 Loss on debt extinguishment 9,766 - 9,766 - Valuation allowance release - (74,474) - (74,474)Income tax effect related to non-GAAP adjustments (4) (5,723) 1,175 (10,835) (2,341)Non-GAAP adjusted net income $58,788 $58,092 $144,783 $123,936 GAAP reported net income per diluted share $0.63 $1.44 $1.68 $2.18 Non-GAAP adjusted net income per diluted share $0.97 $0.95 $2.38 $2.03 Weighted average number of shares of common stock used in non-GAAP diluted per share 60,681,676 61,207,625 60,892,992 60,921,482 (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 2022 Licensing and Commercialization Agreement with Bioprojet.(4) Calculated using the reported effective tax rate for the periods presented less impact of discrete items Harmony Biosciences Investor Contact:Luis Sanay, CFA445-235-8386lsanay@harmonybiosciences.com Harmony Biosciences Media Contact:Cate McCanless202-641-6086cmccanless@harmonybiosciences.com