Parasomnias

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

New paper in The Lancet Neurology proposes the first iteration of a research framework for defining and staging Parkinson's disease (PD) based on biology, rather than clinical symptoms The framework defines PD based on the neuronal alpha-synuclein pathology that is a hallmark for the disease, building on the 2023 discovery of a biomarker that detects alpha-synuclein in living people Based on this new definition, the paper's authors propose a staging system — the neuronal alpha-synuclein disease integrated staging system (NSD-ISS) — that promises to be a research accelerator by improving the development of new drugs across the disease continuum, enabling clinical trials for disease prevention and clarifying regulatory review NEW YORK, Jan. 23, 2024 /PRNewswire/ -- Today, an international working group has proposed a new research framework — the neuronal alpha-synuclein disease integrated staging system (NSD-ISS) —that, for the first time, stages Parkinson's disease (PD) and defines it based on the disease's underlying biology. The paper, published in The Lancet Neurology, describes the framework, its applications, gaps in knowledge and future plans. The NSD-ISS framework is considered a milestone for the field, and a critical starting point — as more data is developed, its concepts and definitions are expected to evolve. The foundation of NSD-ISS defines Parkinson's by the presence of its hallmark pathology, neuronal alpha-synuclein (asyn), that can now be detected by reliable biomarkers, often before symptoms appear. By applying an objective approach to detecting PD and standardizing its tracking on a biological continuum, NSD-ISS represents a major leap into the new and emerging biological era of Parkinson's science. The new research framework stands to accelerate and increase the success of scientific discovery and therapeutic development at all stages of Parkinson's. And ultimately, a treatment that targets the biology of the disease — rather than just its symptoms — is the way to reach a cure. (A similar biological framework in Alzheimer's disease resulted in successful trials and new drug approvals, with the first drugs to slow Alzheimer's-related cognitive decline appearing in 2022 and 2023.) This landmark step forward was made possible by the breakthrough discovery of a test — validated in April 2023 by The Michael J. Fox Foundation's Parkinson's Progression Markers Initiative — capable of detecting the earliest believed pathology of PD even prior to the onset of any visible symptoms. The test, known as the alpha-synuclein seed amplification assay (αSyn-SAA), detects dysfunctional alpha-synuclein protein, one of the two biological hallmarks of PD alongside dopaminergic transport dysfunction, which can be visualized later in the disease process in the brain using DaTScan. Together, these two hallmarks serve as the basis for the NSD-ISS staging system. Key details include: The NSD-ISS integrates two core biomarkers: aggregated asyn (as currently detected by αSyn-SAA) and dopamine dysfunction (as currently measured by DaTScan dopamine transporter imaging in the brain). Stages one and two are based on these objective biomarkers, and stages three to six require these biomarkers and progressive motor and other nonmotor symptoms, such as REM sleep-behavior disorder, tremor, severe loss of smell, cognitive impairments and gait and balance problems. While disease occurs on a continuum and progression varies from person to person, defining discrete biological stages is critical to provide an objective framework for therapeutic development. Researchers are still investigating and refining the timeline for biological disease progression. "This new research framework promises to transform clinical trial design as we know it," said Diane Stephenson, PhD, executive director, Critical Path for Parkinson's Consortium, Critical Path Institute, and a coauthor on The Lancet Neurology paper. "This is how the field will meaningfully and tangibly achieve smarter and faster drug trials and treatments that can slow or halt disease progression, and perhaps one day, prevent the disease process from occurring altogether." Because the framework encompasses clinical syndromes for which dysfunctional asyn is the defining biological hallmark, it applies to — and stands to improve treatment for — both PD and dementia with Lewy Bodies (DLB). The NSD-ISS unifies PD and DLB along the same continuum of progression based on a standardized biological diagnosis. Framework Developed through Open Multistakeholder Collaboration There is widespread recognition that to achieve a paradigm shift in how PD is defined and staged, multiple diverse stakeholders need to come together and engage in open and transparent dialogue. As part of the effort, the framework's international working group hosted a multistakeholder discussion to collect input on the concepts for NSD-ISS from people and families living with PD, neuroscience and clinical leaders, industry experts, federal research funders (i.e., the National Institutes of Health), disease-focused nonprofit organizations and regulatory authorities (i.e., the Food and Drug Administration). A draft manuscript also was posted online for public comment. The process — which engaged more than 550 individuals and organizations from six continents — resulted in a framework informed by collective community input. "Inviting scientific discourse from a multitude of perspectives is a key principle and a shared value of every organization and individual in the working group effort," said Sohini Chowdhury, The Michael J. Fox Foundation's chief program officer and a coauthor on the paper. "This new research framework is the product of a diverse community of contributors, whose inputs have helped to align and harmonize this first meaningful step toward a shared tool for researchers and the biopharma industry." NSD-ISS Will Become a Research Accelerator for the Field The first iteration of the NSD-ISS framework is expected to play a transformative role in guiding development of therapies that affect disease progression by: Stimulating new approaches to develop and test therapies targeting relevant biology across the disease continuum, which could lead to precision treatments for people at every stage of disease. Enabling clinical trials even prior to symptom onset though biomarker-based disease detection. These trials could ultimately lead to therapies that prevent the onset of clinical symptoms. Providing academic researchers, industry investigators and regulators a consistent and uniform definition for each disease stage so that molecular investigations and clinical trial outcomes can be objectively assessed, standardized and compared efficiently. "Our shared hope is that this new framework will foster innovation in clinical development, making trials more efficient and streamlining regulatory review. In short, the NSD-ISS is a research accelerator. And it is expected to evolve with accumulating scientific knowledge," said Tanya Simuni, MD, lead author on the paper, professor of neurology and director of the Parkinson's Disease Movement Disorders Center at Northwestern University. "The success that the Alzheimer's field has had with its biological framework provides the inspiration and motivation to achieve similar accelerated timelines in Parkinson's. Ten years from now, we hope we will look back and say this framework was the key that finally opened the door to next-generation treatments in Parkinson's." As Simuni and her coauthors note in The Lancet Neurology paper, the framework offers an actionable proposal to researchers and industry creating a new path for efficient clinical trials, but at present, it is not intended for use in routine clinical care. The paper includes acknowledgement that data supporting concepts of the NSD-ISS framework have emerged largely from the Foundation's landmark PPMI study. Major funding for PPMI comes from Aligning Science Across Parkinson's (ASAP) (), a coordinated research initiative focused on accelerating the pace of discovery and informing the path to a cure for PD. ASAP support is enabling a seismic expansion of PPMI to increase recruitment efforts and remote testing for those at-risk for PD as well as expanding assay development efforts to enable breakthroughs such as the alpha-synuclein seed amplification assay. PPMI is additionally funded by a consortium of more than 40 biotech and pharmaceutical firms providing financial and in-kind support, and by tens of thousands of individual donors to The Michael J. Fox Foundation. Heralded as "the study that's changing everything" about how Parkinson's is diagnosed, managed and treated, PPMI and its data are made possible by the more than 2,000 in-clinic and 40,000 online research volunteers with and without Parkinson's disease. "It's still early, but this framework will have an immediate impact in terms of how we're designing clinical protocols and optimizing research that can lead to better treatments that patients are waiting for," said Peter DiBiaso, MHSA, a coauthor on the paper, a drug development professional, and member of MJFF's Patient Council who was diagnosed with PD at 49. "We know there's a lot of work to be done, but this is the most important first step the field can take together to rapidly advance breakthroughs for patients and families." About The Michael J. Fox Foundation for Parkinson's Research (MJFF) As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding $1.75 billion in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; creates a robust open-access data set and biosample library to speed scientific breakthroughs and treatment with its landmark clinical study, PPMI; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit us at , Facebook or Twitter. SOURCE The Michael J. Fox Foundation for Parkinson's Research

PLEASANTON, Calif., Jan. 16, 2024 /PRNewswire/ -- Noctrix Health, Inc. announced today that it has closed a $40M Series C financing round led by Sectoral Asset Management with participation from other new investors Angelini Ventures, ResMed, and Asahi Kasei Corporation with strong support from insiders including OrbiMed and Treo Ventures. This infusion of capital will propel the advancement of Nidra™ Tonic Motor Activation (TOMAC) therapy towards its eagerly anticipated launch in the US market. Nidra™, a revolutionary new device, stands as the first and only approved treatment for drug-refractory Restless Legs Syndrome (RLS), a chronic condition that has debilitating impacts on sleep and quality of life of millions of adults worldwide. Continue Reading Nidra TOMAC therapy The Nidra™ Tonic Motor Activation therapy is a clinically validated treatment indicated for reducing the symptoms of RLS and improving sleep quality in adults refractory to medications and was granted a De Novo authorization by the US Food and Drug Administration (FDA) in April 2023 after receiving a Breakthrough Device Designation from the agency in 2020. Noctrix Health Founder and CEO, Shri Raghunathan, expressed his excitement at the successful funding round, stating, "We are thrilled to welcome Sectoral Asset Management and other new investors to this esteemed group of partners. This significant milestone, coupled with unwavering support from our existing investors, reflects our shared commitment to establishing a new standard of care for RLS. I extend my gratitude to our dedicated team for their hard work and am humbled by the overwhelming support received." Michael Sjöström, co-founder, and Partner at Sectoral Asset Management, shared his enthusiasm, stating, "I am honored to join Noctrix Health on its journey in redefining RLS treatment. We believe in the transformative potential of TOMAC therapy and look forward to contributing to the company's continued success." "The strength of Noctrix Health's clinical data combined with the tremendous unmet need underscore the importance of bringing TOMAC therapy to patients with RLS," said Evan Caplan, Principal at OrbiMed. "We have seen how well the team has executed and are excited to continue partnering with them as the company initiates commercialization." "Treo Ventures is thrilled to continue supporting Noctrix Health. This significant financing will allow the company to reach millions of patients with RLS. TOMAC therapy is a patient-friendly, highly effective, non-pharmacologic innovation for patients unhappy with current solutions who need better treatment options," commented Tracy Pappas, General Partner, Treo Ventures. Noctrix Health has experienced a banner year in 2023, marked by the FDA marketing authorization of TOMAC therapy, followed by several key publications including a recent study demonstrating TOMAC therapy's efficacy in reducing opioid use in patients with RLS, further reinforcing its potential impact on patient outcomes. About Nidra™ TOMAC Therapy The Nidra™ Tonic Motor Activation (TOMAC) system is the first and only non-pharmacologic, wearable treatment intended to reduce symptoms of primary moderate-severe Restless Legs Syndrome (RLS) and improve sleep quality in adults refractory to medications. It is available in the US by prescription only. For more information on availability or clinical and important safety information, please visit . About Noctrix Health, Inc. Noctrix Health is a medical device company committed to developing clinically validated breakthrough medical technologies that improve the quality of life for patients with chronic conditions. For enquiries contact [email protected]. SOURCE Noctrix Health, Inc