1. PLYMOUTH MEETING, Pa., Sept. 22, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today announced winners of the Patient at the Heart and Progress at the Heart programs in honor of the third annual World Narcolepsy Day.

    The Patients at the Heart program was created in 2019, and awards funds to not-for-profit organizations working to support and address the challenges of people living with sleep disorders and rare diseases who experience excessive daytime sleepiness (EDS). Initiated in 2021, Progress at the Heart supports innovative programs that address disparities, injustice, and inequities in the rare disease and sleep disorder community.

    "We are so inspired by the diverse and inventive programs submitted for this year's Patients and Progress at the Heart programs," said Cate McCanless, SVP, Corporate Affairs and Public Policy. "Our goal is to improve the lives of people living with sleep disorders and their families, and we hope by supporting these cutting-edge programs, we can make a positive difference for these communities."

    The company chose to announce the winners on World Narcolepsy Day, a day dedicated to raising awareness for this sleep disorder globally. The first Patients at the Heart program awarded $65,000 to four organizations, which was increased to $74,000 in 2020. Now, combined with the Progress at the Heart recipients, total funds awarded in 2021 increased to over $180,000 awarded to eight organizations across the United States.

    The following organizations, by category, were chosen as the 2021 Patients at the Heart recipients:

    Narcolepsy

    • ANGEL Aid:
      • Narcolepsy Caregivers Retreat

        The grant will allow the creation of a six-week virtual Caregivers Wellness Retreat with a focus on mental health, self-care, and connection for up to 25 caregivers of people with narcolepsy.
      • Who Am I? Graphic Novel Workshop

        The funds will aid creation of an art-therapy program designed to foster emotional life skills, self-awareness and a sense of connection for people living with narcolepsy and/or EDS.

    Rare Diseases with EDS

    • Prader-Willi Syndrome Association | USA: Disordered Sleep: A Deep Dive for Parents and Practitioners

      Funds to foster creation of free and accessible tools and resources informed by focus groups and PWS sleep best practices, as well as a PWSA | USA Virtual Sleep Summit.

    The following organizations and programs were chosen as the 2021 Progress at the Heart recipients:

    • Community Health Clinic: Enhancing Sleep Health Awareness Among Chicago's Medically Underserved Population

      This project will provide educational materials about sleep health and various sleep disorders to the target population at Community Health Clinic (CHC), which primarily includes patients from Spanish and Polish-speaking communities, whose annual income is at or below 100% of the Federal Poverty Line. Additionally, this project provides the framework and platform upon which to create and disseminate educational materials that are applicable for similar clinics serving low-income communities across Chicago.
    • Duke Department of Neurology: Duke Health Disparities in Neurology Scorecard

      The grant was awarded for the creation of the Duke Racial Disparities in Neurology Scorecard, inspired by the White Coats for Black Lives Racial Justice Scorecard for medical schools. A calculator will be published electronically that will aid other neurology departments across the country to create their own scorecards.
    • Florida Sickle Cell Center: CRiSIS (Circadian Rhythm & Sleep Impact on Sickle Cell)

      An initiative to investigate the sleep quality of patients living with Sickle Cell Disease (SCD). The objective of this program is to better understand the sleep quality of people living with SCD and how their sleep quality may be contributing to or exacerbating their underlying condition.
    • Project Sleep: Focus Groups to Understand Barriers and Improve Sleep Disorders Awareness, Diagnosis, and Treatment in Black American Communities

      This project aims to uncover important insights by conducting focus groups to better understand the first-hand perspectives of Black Americans, healthcare providers, and trusted community leaders. The findings will help to inform future awareness and education campaigns aiming to reduce delays to diagnosis and treatment of sleep disorders in underserved communities.
    • The Hypersomnia Foundation (HF): Diversity, Equity, and Inclusion (DEI) Initiative

      The DEI Initiative is focused on understanding and identifying the challenges people of diverse backgrounds may face when they need healthcare for EDS and other symptoms of sleep disorders. With a five-step program, led by a DEI Task Force, HF plans to hold listening sessions in the community as first steps.
    • The Sleep Doctor: Bio-Rhythm Program

      A behavioral health intervention designed to promote weight loss through using Fitbit technology to monitor circadian rhythm and coach participants to maintain proper sleep, nutrition, and activity habits. This program will help to educate and monitor participants from underserved Hispanic communities in El Paso, TX to address the high prevalence of sleep disorders contributing to obesity.

    About Harmony Biosciences 

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare neurological diseases who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Patti Bank

    415-513-1284

    ir@harmonybiosciences.com 

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  2. PLYMOUTH MEETING, Pa., Sept. 2, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today announced that the American Academy of Sleep Medicine (AASM) has published an updated clinical practice guideline which includes WAKIX® (pitolisant) as a recommended treatment option for adults living with narcolepsy.

    The new clinical practice guideline was published in the Journal of Clinical Sleep Medicine in a special article titled, "Treatment of central disorders of hypersomnolence: an American Academy of Sleep Medicine clinical practice guideline" and was accompanied by another article titled, "Treatment of central disorders of hypersomnolence: an American Academy of Sleep Medicine systematic review, meta-analysis, and GRADE assessment". Together, these papers provide practice recommendations, and the evidence base in support of the recommendations, for the treatment of narcolepsy and other central disorders of hypersomnolence. The new guideline updates and replaces the previous AASM guideline published in 2007, and now includes WAKIX as a strong recommendation for the treatment of narcolepsy in adults based on data that showed clinically significant improvement in excessive daytime sleepiness and cataplexy in patients treated with WAKIX.

    "Harmony is pleased with the inclusion of WAKIX in the updated AASM clinical practice treatment guideline. This evidence-based guideline, which now includes some of the more recent treatment options available, can assist healthcare professionals in therapeutic decision making when managing their patients living with narcolepsy," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness 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. 

    Important Safety Information 

    Contradictions 

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.  

    Warnings and Precautions 

    WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval. 

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD). 

    Adverse Reactions 

    In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and 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 Interactions 

    Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half.    

    Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required (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 Populations 

    WAKIX 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 FDA at 1-800-FDA-1088 or www.fda.gov/medwatch

    About Harmony Biosciences 

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare neurological diseases who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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 Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Patti Bank

    415-513-1284

    ir@harmonybiosciences.com 

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  3. PLYMOUTH MEETING, Pa., Sept. 2, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today announced that Harmony's President and CEO, John C. Jacobs, will participate in fireside chats and host investor meetings at the following upcoming virtual conferences: 

    • Citi's 16th Annual BioPharma Virtual Conference
      • 1x1 Meetings: Wednesday and Thursday, September 8-9, 2021
    • 2021 Virtual Wells Fargo Healthcare Conference
      • Fireside Chat: September 10, 10:40am ET
    • 2021 Cantor Virtual Global Healthcare Conference
      • 1x1 Meetings: Tuesday, September 28, 2021

    The live audio webcast of the fireside chat will be available on the investor page of Harmony's website at https://ir.harmonybiosciences.com/.

    About Harmony Biosciences 

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare neurological diseases who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Investor Contact:

    Patti Bank

    415-513-1284

    ir@harmonybiosciences.com 

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046

    nleone@harmonybiosciences.com  

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/harmony-biosciences-to-participate-in-three-investor-conferences-in-september-301367935.html

    SOURCE Harmony Biosciences

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  4. WAKIX® (pitolisant) Net Revenue of $73.8 Million for Second Quarter 2021 vs. $38 Million for the Same Period in 2020

    Initiated Phase 2 Clinical Trial in Patients with Myotonic Dystrophy

    Acquired Potential First-in-Class Molecule with Novel Mechanism of Action

    Secured Additional Capital Through Strategic Financing Collaboration with Blackstone

    Conference Call and Webcast to be Held Today at 8:30 a.m. ET 

    PLYMOUTH MEETING, Pa., Aug. 10, 2021 (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 second…

    WAKIX® (pitolisant) Net Revenue of $73.8 Million for Second Quarter 2021 vs. $38 Million for the Same Period in 2020

    Initiated Phase 2 Clinical Trial in Patients with Myotonic Dystrophy

    Acquired Potential First-in-Class Molecule with Novel Mechanism of Action

    Secured Additional Capital Through Strategic Financing Collaboration with Blackstone

    Conference Call and Webcast to be Held Today at 8:30 a.m. ET 

    PLYMOUTH MEETING, Pa., Aug. 10, 2021 (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 second quarter ended June 30, 2021.

    "Harmony has made significant progress towards growing the company through our three pillars growth strategy during the first half of 2021," stated John Jacobs, President and Chief Executive Officer of Harmony. "Building on the momentum we saw in the first quarter of this year, we continue to advance the commercialization of WAKIX® with strong second quarter revenues due to both an increase in average number of patients on WAKIX and the number of healthcare professionals prescribing the product.

    With the initiation of our Phase 2 study in patients with Myotonic Dystrophy in the second quarter, we are continuing to advance our clinical development programs, with the goal of broadening the clinical utility of WAKIX beyond narcolepsy. Also, importantly, we took the first step in our plan to build a broad portfolio of products beyond WAKIX, via the acquisition of a potential first-in-class molecule with a novel mechanism of action. In addition, and to further support our expanding clinical programs and business development objectives, we recently consummated a strategic financing collaboration with Blackstone that provides us with additional capital to drive continued long-term growth for Harmony."

         

    Second Quarter 2021 Financial Highlights:

    • Net product revenue of $73.8 million for the quarter ended June 30, 2021, a 94.2% increase versus the prior year quarter revenue of $38.0 million;
    • Strong organic growth in WAKIX sales, supported by an increased average number of patients on WAKIX and number of unique healthcare professionals who have prescribed WAKIX since launch;
    • Positive net income and achieved profitability for the second quarter in a row; and
    • Cash and cash equivalents of $159.7 million.

    Second Quarter 2021 Financial Results

    Net product revenues for the quarter ended June 30, 2021 were $73.8 million, compared to $38.0 million for the same period in 2020. The 94.2% growth versus the prior year quarter can be attributed to strong commercial sales of WAKIX driven by organic demand.

    For the quarter ended June 30, 2021, GAAP net income available to shareholders was $14.1 million, or $0.24 per diluted share, compared to a net loss of $10.5 million, or $1.34 per diluted share, for the same period in 2020. Non-GAAP adjusted net income was $31.9 million, or $0.54 per diluted share, for the quarter ended June 20, 2021, compared to an adjusted net loss of $0.5 million, or a loss of $0.07 per diluted share, for the same period in 2020.

    Reconciliations of applicable GAAP measures to non-GAAP adjusted information are included at the end of the press release.

    The components of Harmony's operating expenses include:

    • Research and Development expenses were $6.5 million in the second quarter of 2021 as compared with $4.2 million for the same quarter in 2020, representing a 55.9% increase;  
    • Sales and Marketing expenses were $17.0 million in the second quarter of 2021 as compared to $12.4 million for the same quarter in 2020, representing a 36.8% increase;
    • General and Administrative expenses were $14.3 million in the second quarter of 2021 as compared to $7.6 million for the same quarter in 2020, representing a 87.5% increase; and
    • Total operating expenses were $37.8 million in the second quarter of 2021 as compared with $24.2 million for the same quarter in 2020, representing a 56.0% increase.

    As of June 30, 2021, Harmony had cash and cash equivalents of $159.7 million.

    Clinical Development and Recent Updates

    • Enrollment continues in Harmony's Phase 2 clinical trial evaluating the safety and efficacy of pitolisant for the treatment of EDS and other symptoms in patients with PWS and is on track to achieve top line data in the first half of 2022.
    • In June 2021, Harmony initiated a Phase 2 clinical trial to evaluate the safety and efficacy of pitolisant for EDS and other non-muscular symptoms in adult patients with type 1 myotonic dystrophy (DM1). Top-line results are anticipated in the second half of 2022.
    • In August 2021, Harmony acquired HBS-102 (formerly CSTI-100), a potential first-in-class molecule with a novel mechanism of action. HBS-102 is a Melanin Concentrating Hormone Receptor 1 (MCHR1) antagonist that has the potential to offer an innovative approach to the treatment of narcolepsy, including the symptoms of Rapid Eye Movement (REM) sleep dysregulation, such as cataplexy, hallucinations and sleep paralysis. HBS-102 targets the generator of REM sleep and its associated behaviors, which is controlled by melanin concentrating hormone (MCH) neurons located in the lateral hypothalamus. Under the terms of the agreement, Harmony will acquire full development and commercialization rights globally, with the exception of Greater China, with financial terms including an upfront payment and potential development and regulatory milestones and royalties.
    • In August 2021, Harmony entered into a strategic financing collaboration with Blackstone to provide Harmony up to $330 million which includes $200 million to refinance its existing debt at a lower interest rate, $100 million for drawdown within the next twelve months, and a $30 million equity investment in Harmony common stock.

    Conference Call Today at 8:30 a.m. ET

    We are hosting our second quarter 2021 financial results conference call and webcast today beginning at 8:30 a.m. Eastern Time. The live and replayed webcast of the call will be available on the investor page of our website at https://ir.harmonybiosciences.com/. To participate in the live call by phone, dial (833) 614-1471 (domestic) or +1 (914) 987-7209 (international), and reference passcode 2955734. A replay will be accessible until August 17, 2021 by dialing (855) 859-2056 (domestic) or +1 (404) 537-3406 (international).

    Non-GAAP Financial Measures

    In addition to our GAAP results, we provide certain non-GAAP metrics including adjusted net income and adjusted net income per share. We believe that the presentation of these measures 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 adjusted net income and 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. Company management uses these non-GAAP measurements as an aid in monitoring our ongoing financial performance from quarter-to-quarter and year-to-year on a regular basis and for benchmarking against comparable companies.

    These non-GAAP financial measures are not meant to 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 its non-GAAP financial measures; and we may in the future cease to exclude items that it has historically excluded for purposes of its non-GAAP financial measures.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness 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 Usage

    WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy.

    Important Safety Information

    Contraindications

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.

    Warnings and Precautions

    WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval. 

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    Adverse Reactions

    In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and 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 Interactions

    Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half. 

    Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required (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 Populations

    WAKIX 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 FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    About HBS-102

    HBS-102 is an investigational compound being developed as a potential treatment for narcolepsy and other rare neurological diseases. HBS-102 is a potential first-in-class molecule with a novel mechanism of action which targets melanin concentrating hormone (MCH) brain cells in the hypothalamus, which make up the control center for REM sleep and related behaviors. In the setting of orexin deficiency (as occurs in patients with type 1 narcolepsy), there is an imbalance between orexin and MCH which could result in the control center for REM sleep going unchecked that could lead to REM sleep intruding into wakefulness. If that occurs, the clinical symptoms are experienced as cataplexy, hallucinations, and/or sleep paralysis. HBS-102, an MCHR1 antagonist, blocks the activity of the MCH neurons, which could potentially reduce REM intrusions into wakefulness and therefore reduce the debilitating symptoms of cataplexy, hallucinations and sleep paralysis.

    About Harmony Biosciences

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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.

    CONSOLIDATED

    STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

    (In thousands except share and per share data)

    (unaudited)

      Three Months Ended

    June 30,
      Six Months Ended

    June 30,
     
      2021  2020  2021  2020 
    Net product revenues $73,821  $38,005  $133,495  $57,845 
    Cost of product sold  12,687   6,456   23,097   9,930 
    Gross profit  61,134   31,549   110,398   47,915 
    Operating expenses:                
    Research and development  6,498   4,169   11,177   7,600 
    Sales and marketing  17,022   12,443   32,529   25,697 
    General and administrative  14,302   7,628   28,848   15,772 
    Total operating expenses  37,822   24,240   72,554   49,069 
    Operating income (loss)  23,312   7,309   37,844   (1,154)
    Loss on debt extinguishment           (22,639)
    Other income (expense), net  4   (400)  (15)  (1,546)
    Interest expense, net  (7,227)  (6,936)  (14,354)  (13,308)
    Income (loss) before income taxes  16,089   (27)  23,475   (38,647)
    Income taxes  (1,972)     (1,972)   
    Net income (loss) and comprehensive income (loss) $14,117  $(27) $21,503  $(38,647)
    Accumulation of dividends on preferred stock     (10,446)     (20,891)
    Net income (loss) available to common stockholders $14,117  $(10,473) $21,503  $(59,538)
    EARNINGS (LOSS) PER SHARE:                
    Basic $0.25  $(1.34) $0.38  $(7.63)
    Diluted $0.24  $(1.34) $0.37  $(7.63)
    Weighted average number of shares of common stock - basic  56,940,840   7,805,848   56,916,282   7,798,928 
    Weighted average number of shares of common stock - diluted  58,592,876   7,805,848   58,635,195   7,798,928 





    HARMONY BIOSCIENCES HOLDINGS, INC.

    CONSOLIDATED BALANCE SHEETS

    (In thousands except share and per share data)

    (unaudited)

      June 30,

    2021
      December 31,

    2020
     
    ASSETS        
    CURRENT ASSETS:        
    Cash and cash equivalents $159,686  $228,631 
    Trade receivables, net  31,196   22,176 
    Inventory, net  4,951   3,823 
    Prepaid expenses  7,322   6,959 
    Other current assets  1,559   1,302 
    Total current assets  204,714   262,891 
    NONCURRENT ASSETS:        
    Property and equipment, net  944   938 
    Restricted cash  750   750 
    Intangible assets, net  153,135   162,343 
    Other noncurrent assets  152   152 
    Total noncurrent assets  154,981   164,183 
    TOTAL ASSETS $359,695  $427,074 
    LIABILITIES AND STOCKHOLDERS' EQUITY        
    CURRENT LIABILITIES:        
    Trade payables $2,006  $2,556 
    Accrued compensation  6,043   8,942 
    Accrued expenses  26,653   122,727 
    Other current liabilities  1,893   314 
    Total current liabilities  36,595   134,539 
    NONCURRENT LIABILITIES:        
    Long term debt, net  195,610   194,250 
    Other noncurrent liabilities  939   1,105 
    Total noncurrent liabilities  196,549   195,355 
    TOTAL LIABILITIES  233,144   329,894 
    COMMITMENTS AND CONTINGENCIES (Note 9)        
    STOCKHOLDERS' EQUITY:        
    Preferred stock - $0.00001 par value; 10,000,000 shares and 0 shares authorized at June 30, 2021 and December 31, 2020, respectively; 0 shares issued and outstanding at June 30, 2021 and December 31, 2020, respectively      
    Common stock—$0.00001 par value; 500,000,000 shares authorized at June 30, 2021 and December 31, 2020, respectively; 57,000,139 shares and 56,890,569 issued and outstanding at June 30, 2021 and December 31, 2020, respectively  1   1 
    Additional paid in capital  593,242   585,374 
    Accumulated deficit  (466,692)  (488,195)
    TOTAL STOCKHOLDERS' EQUITY  126,551   97,180 
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $359,695  $427,074 





    HARMONY BIOSCIENCES HOLDINGS, INC.

    RECONCILIATION OF GAAP TO NON-GAAP RESULTS

    (In thousands except share and per share data)

    (unaudited)

      Three Months Ended

    June 30,
      Six Months Ended

    June 30,
     
      2021  2020  2021  2020 
    Net income (loss) $14,117  $(27) $21,503  $(38,647)
    Non-GAAP Adjustments:                
    Interest expense  7,227   6,936   14,354   13,308 
    Taxes  1,972      1,972    
    Depreciation  100   97   200   194 
    Amortization  4,629   1,907   9,208   3,693 
    EBITDA  28,045   8,913   47,237   (21,452)
                     
    Additional Non-GAAP Adjustments:                
    Stock-based compensation expense  3,827   568   7,078   936 
    Loss on debt extinguishment           22,639 
    Warrant expense     438      1,584 
    Non-GAAP adjusted net income (loss) $31,872  $9,919  $54,315  $3,707 
                     
    Accumulation of yield on preferred stock     (10,446)     (20,891)
                     
    Non-GAAP adjusted net income (loss) available to common stockholders  31,872   (527)  54,315   (17,184)
                     
    GAAP reported net income (loss) per diluted share $0.24  $(1.34) $0.37  $(7.63)
    Non-GAAP adjusted net income (loss) per diluted share $0.54  $(0.07) $0.93  $(2.20)
                     
    Weighted average number of shares of common stock used in non-GAAP diluted per share  58,592,876   7,805,848   58,635,195   7,798,928 

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com



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  5. PLYMOUTH MEETING, Pa. and NEW YORK, Aug. 10, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, and Blackstone (NYSE:BX) today announced that they have entered into a strategic financing collaboration where funds managed by Blackstone have agreed to provide Harmony with up to $330 million of financing and growth capital.

    "We are pleased to partner with Blackstone, one of the world's leading investment firms with strong transactional experience in life sciences and healthcare," said John C. Jacobs, President and Chief Executive Officer of Harmony Biosciences. "This financing provides us with further flexibility to grow our business by providing us with access to capital to expand our portfolio of assets in rare, neurological diseases while also reducing our annual interest expense."

    Commenting on the arrangement, Craig Shepherd, Senior Managing Director with Blackstone Life Sciences, and Brad Marshall, Senior Managing Director with Blackstone Credit, said, "This transaction demonstrates our unique ability to combine deep domain expertise and flexible scale capital in the life sciences, helping to grow companies like Harmony as they optimize their capital structure and fund initiatives that develop important treatments for patients. We are strongly motivated by the Harmony team's track record of success in developing and commercializing its important medicine WAKIX® (pitolisant)."

    The strategic financing collaboration with Blackstone includes up to $300 million in debt capital and a $30 million equity investment in Harmony common stock. The $300 million of debt capital includes a senior secured term loan facility in aggregate original principal amount of $200 million, and a $100 million senior secured delayed draw term loan facility that is available to be drawn within 12 months of closing, subject to the terms of the facility. Substantially all of the proceeds from the initial $200 million term loan and the related sale of Harmony's common stock were used to pay off Harmony's existing debt facility, together with the payment of fees and expenses, resulting in a significantly lower cost of capital.

    Paragon Health Capital, Cantor Fitzgerald & Co., Oppenheimer & Co. Inc., and Raymond James served as financial advisors to Harmony. Hogan Lovells US LLP served as legal advisor to Harmony and Ropes & Gray LLP served as legal advisor to Blackstone.

    About Blackstone Life Sciences

    Blackstone Life Sciences is an industry-leading private investment platform with capabilities to invest across the life cycle of companies and products within the key life science sectors. By combining scale investments and hands-on operational leadership, Blackstone Life Sciences helps bring to market promising new medicines and medical technologies that improve patients' lives. More information is provided at www.blackstone.com/our-businesses/life-sciences/.

    About Blackstone Credit

    Blackstone Credit is one of the world's largest credit-focused asset managers, with $174 billion in AUM. We seek to generate attractive risk-adjusted returns for our clients by investing across the entire corporate credit market, from public debt to private loans. Blackstone Credit capital supports a wide range of companies across sectors and geographies, enabling businesses to expand, invest, and navigate changing market environments. More information is provided at www.blackstone.com/our-businesses/credit/

    Blackstone is the world's largest alternative investment firm with $684 billion of assets under management.

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological diseases who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness 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.

    Cautionary Statement Regarding Forward Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding the future growth of our business, availability of the delayed draw term loan, the use of proceeds, and our product WAKIX. 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. Each of the forward-looking statements is subject to change based on various important factors, many of which are beyond the company's control, 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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 compliance with the terms of our credit facility; our failure to maintain effective internal controls; the impact of government laws and regulations; volatility and fluctuations in the price of our common stock; and 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; and 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 March 25, 2021 and subsequent Forms 10-Q, and in 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. The information in this press release should be read in conjunction with a review of our filings with the SEC including the information in our most recent Annual Report on Form 10-K, and subsequent Forms 10-Q, under the heading MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Blackstone Contact:

    Paula Chirhart

    +1-347-463-5453

    paula.chirhart@blackstone.com

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    SOURCE Harmony Biosciences

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  6. PLYMOUTH MEETING, Pa., Aug. 9, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological diseases, today announced the acquisition of HBS-102 (formerly CSTI-100), a potential first-in-class molecule with a novel mechanism of action, from ConSynance Therapeutics, Inc., a clinical stage biotechnology company focused on rare central nervous system diseases. Under the terms of the agreement, Harmony will acquire full development and commercialization rights globally, with the exception of Greater China, with financial terms including an upfront payment of $3.5 million and potential development…

    PLYMOUTH MEETING, Pa., Aug. 9, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological diseases, today announced the acquisition of HBS-102 (formerly CSTI-100), a potential first-in-class molecule with a novel mechanism of action, from ConSynance Therapeutics, Inc., a clinical stage biotechnology company focused on rare central nervous system diseases. Under the terms of the agreement, Harmony will acquire full development and commercialization rights globally, with the exception of Greater China, with financial terms including an upfront payment of $3.5 million and potential development and regulatory milestone payments and royalties.

    "The acquisition of HBS-102 represents our first addition to the pipeline beyond WAKIX® (pitolisant), and our intention is to continue to pursue additional assets in line with our vision of becoming a leading rare neurological disease company with a robust portfolio of products," said John C. Jacobs, President and Chief Executive Officer of Harmony.

    HBS-102 is a Melanin Concentrating Hormone Receptor 1 (MCHR1) antagonist that has the potential to offer a novel approach to the treatment of narcolepsy including the symptoms of Rapid Eye Movement (REM) sleep dysregulation, such as cataplexy, hallucinations and sleep paralysis. HBS-102 blocks the activity of melanin concentrating hormone (MCH) neurons, which scientific evidence indicates is the generator of REM sleep and its associated behaviors. Therefore, HBS-102 could potentially reduce REM intrusions into wakefulness and reduce the frequency of cataplexy, hallucinations, and sleep paralysis. In a preclinical proof-of-concept study, Dr. Thomas Scammell, Professor, Department of Neurology, Beth Israel Deaconess Medical Center and Division of Sleep Medicine, Harvard Medical School, and his team demonstrated that an MCHR1 antagonist molecule resulted in a significant reduction in cataplexy events in an orexin knockout mouse model of narcolepsy.1 Harmony will complete additional work to prepare and submit an Investigational New Drug (IND) application with the plan to initiate a Phase 2 clinical trial once the IND is open.

    "Similar to WAKIX, this asset offers Harmony another opportunity to lead with the science and potentially bring another first-in-class treatment option to patients living with narcolepsy and other rare neurological diseases," said Jeffrey Dayno, M.D., Chief Medical Officer of Harmony. "The majority of people living with narcolepsy experience symptoms of REM dysregulation that have a significant impact on their lives. The acquisition and successful development of HBS-102 could represent a next-generation therapy for narcolepsy patients by offering a novel approach, directly targeting the control center for REM sleep and its associated behaviors."

    About Narcolepsy   

    Narcolepsy 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 WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX 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. 

    Important Safety Information

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About HBS-102

    HBS-102 is an investigational compound being developed as a potential treatment for narcolepsy and other rare neurological diseases. HBS-102 is a potential first-in-class molecule with a novel mechanism of action which targets melanin concentrating hormone (MCH) neurons in the hypothalamus, which make up the control center for REM sleep and related behaviors. In the setting of orexin deficiency (as occurs in patients with type 1 narcolepsy), there is an imbalance between orexin and MCH which could result in the control center for REM sleep going unchecked that could lead to REM sleep intruding into wakefulness. If that occurs, the clinical symptoms are experienced as cataplexy, hallucinations, and/or sleep paralysis. HBS-102, an MCHR1 antagonist, blocks the activity of the MCH neurons, which could potentially reduce REM intrusions into wakefulness and therefore reduce the debilitating symptoms of cataplexy, hallucinations and sleep paralysis.

    About Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological diseases who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    About ConSynance

    ConSynance Therapeutics is a clinical-stage, biopharmaceutical company targeting rare central nervous system diseases. The Company focuses on innovative therapeutics for rare hypothalamic disorders including narcolepsy, Prader-Willi Syndrome and hypothalamic injury-induced obesity. For more information on ConSynance, please visit the company's website:  www.consynance.com.

    Forward Looking Statement 

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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 Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    1 Naganuma, F., Bandaru, S. S., Absi, G., Mahoney, C. E., Scammell, T. E., & Vetrivelan, R. (2018). Melanin-concentrating hormone neurons contribute to dysregulation of rapid eye movement sleep in narcolepsy. Neurobiology of Disease, 120, 12-20. https://doi.org/10.1016/j.nbd.2018.08.012

     

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    SOURCE Harmony Biosciences

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  7. PLYMOUTH MEEETING, Pa., July 29, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that John C. Jacobs, President and CEO, has been named an EY Entrepreneur Of The Year® 2021 Greater Philadelphia Award Winner.

    Jacobs was selected by a panel of independent judges for his work leading Harmony and joins an esteemed multi-industry community of other entrepreneurs who have driven their companies' success, transformed their industries, and made a positive impact on their employees and communities.

    "I am extremely honored to be selected for this prestigious award and accept it on behalf of the entire Harmony team, who demonstrate their commitment and passion every day through their support of the rare disease community," said John C. Jacobs, President and CEO. "I'm also inspired by the opportunity we have as a company to help patients in need who are living with rare neurological disorders. As Harmony continues to grow and evolve into a leading rare disease company, it is our vision of being able to help an even broader population of patients that drives us forward."

    For 35 years, the EY Entrepreneur Of The Year Award® program has been one of the preeminent competitive awards for entrepreneurs and leaders of high-growth companies. The award recognizes entrepreneurial leaders who are excelling in overcoming adversity, driving financial performance, and committed to building innovative and values-based companies with wider social impact.

    As a Greater Philadelphia award winner, Jacobs is now eligible for consideration for the Entrepreneur Of The Year® 2021 National Awards.

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    About Entrepreneur Of The Year®

    Entrepreneur Of The Year® is the world's most prestigious business awards program for unstoppable entrepreneurs. These visionary leaders deliver innovation, growth and prosperity that transform our world. The program engages entrepreneurs with insights and experiences that foster growth. It connects them with their peers to strengthen entrepreneurship around the world. Entrepreneur Of The Year is the first and only truly global awards program of its kind. It celebrates entrepreneurs through regional and national awards programs in more than 145 cities in over 60 countries. National Overall winners go on to compete for the EY World Entrepreneur Of The Year™.

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com  

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/john-c-jacobs-president-and-ceo-of-harmony-biosciences-named-an-ernst--young-ey-entrepreneur-of-the-year-2021-greater-philadelphia-award-winner-301343799.html

    SOURCE Harmony Biosciences

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  8. PLYMOUTH MEETING, Pa., July 27, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that it will issue a press release on the Company's second quarter 2021 financial results on Tuesday, August 10, 2021 at 8:00 a.m. ET. Following the release, Harmony will conduct a conference call and live webcast on the same day, at 8:30 a.m. ET.

    To participate in the call, please dial (833) 614-1471 (domestic) or +1 (914) 987-7209 (international), and reference passcode 2955734. It is recommended that you dial in at least 10 minutes prior to the call.

    The live and replayed webcast of the call will be available on the investor page of our website at https://ir.harmonybiosciences.com/.

    A replay of the call will be available within 24 hours by dialing (855) 859-2056 (domestic) or +1 (404) 537-3406 (international) and entering conference ID: 2955734. The replay will be available until August 17, 2021.

    About Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/harmony-biosciences-announces-date-of-second-quarter-2021-financial-results-301341458.html

    SOURCE Harmony Biosciences

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  9. PLYMOUTH MEETING, Pa., June 29, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced initiation of a Phase 2 clinical trial to evaluate the safety and efficacy of pitolisant for excessive daytime sleepiness (EDS) and other non-muscular symptoms in adult patients with type 1 myotonic dystrophy (DM1).

    "In addition to the primary symptoms of myotonia and muscle weakness in patients with type 1 myotonic dystrophy, the non-muscular symptoms of excessive daytime sleepiness, fatigue, and cognitive dysfunction are very common in these patients and have a negative impact on daily functioning as much as, or more so, than the primary muscle symptoms of the disease," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "Pitolisant's novel mechanism of action increases histamine transmission in the brain which provides the scientific rationale for its potential clinical utility for the common non-muscular symptoms in patients with DM1. We listened to the needs of patients and caregivers in the myotonic dystrophy patient community and are pleased to have initiated this Phase 2 clinical trial to assess the potential clinical benefit of pitolisant in patients with this rare neurological disease, for which there are currently no approved treatment options."

    Myotonic dystrophy 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, which is the most common form of this disorder. This equates to about 160,000 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.

    The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled study designed to assess the safety and efficacy of pitolisant in patients with DM1 ages 18 – 65 years. Approximately 135 patients will be randomized at baseline to low-dose pitolisant, high-dose pitolisant, or placebo in a 1:1:1 treatment ratio titrated over three weeks, followed by eight weeks of stable dosing. Patients who complete the randomized, controlled phase of the trial will be eligible to participate in an open-label extension phase to assess the long-term safety and effectiveness of pitolisant in patients with DM1.

    The primary objective of the trial is to evaluate the effect of pitolisant compared with placebo on EDS. Secondary objectives include assessments of fatigue, specific measures of cognitive function using validated computer-based assessments, and overall disease burden utilizing a disease-specific, patient-reported outcomes instrument for DM1. Topline results are anticipated from the Phase 2 trial in the second half of 2022.

    Pitolisant is marketed as WAKIX® in the U.S. for the treatment of EDS or cataplexy in adult patients with narcolepsy.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX 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. 

    Important Safety Information

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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 Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/harmony-biosciences-initiates-a-phase-2-clinical-trial-in-myotonic-dystrophy-301321420.html

    SOURCE Harmony Biosciences

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  10. PLYMOUTH MEETING, Pa., June 10, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced results of a new analysis evaluating number needed to treat (NNT) and effect sizes for treatment with WAKIX® (pitolisant) that demonstrates the magnitude of its clinical effectiveness for excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The poster was presented at the 35th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2021," held virtually June 10-13.

    PLYMOUTH MEETING, Pa., June 10, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced results of a new analysis evaluating number needed to treat (NNT) and effect sizes for treatment with WAKIX® (pitolisant) that demonstrates the magnitude of its clinical effectiveness for excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The poster was presented at the 35th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2021," held virtually June 10-13.

    "Excessive daytime sleepiness or impaired wakefulness can often result in brain fog, fatigue, changes in mood and other symptoms that have a large physical, emotional and psychological impact on a patient's life," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "We're encouraged to see our data supporting the clinical utility of WAKIX and its value as an effective and safe treatment option to help this patient population." 

    Assessment of the Clinical Benefits of Pitolisant on Excessive Daytime Sleepiness and Cataplexy in Adults With Narcolepsy

    This poster presents results from a new analysis that evaluated the clinical impact of pitolisant in adults with narcolepsy using NNT and effect sizes as response to treatment metrics based on data from the HARMONY 1 and HARMONY CTP randomized, controlled clinical trials. NNT and effect size are important metrics as they measure the number of patients that need to be treated to achieve a specific outcome for one person and the magnitude of drug–placebo difference on outcome measures, respectively.

    At week 8 in the HARMONY 1 trial (pitolisant, n=31; placebo, n=30), pitolisant had a NNT of 5 with 67.7% of patients treated with pitolisant achieving treatment response for EDS compared to placebo (43.3%) and effect size was 0.61 based on change in Epworth Sleepiness Scale scores compared to baseline.

    At week 7 in the HARMONY CTP trial (pitolisant, n=54, placebo, n=51), pitolisant had a NNT of 3 with 68.6% and 66.7% of patients treated with pitolisant achieving treatment response for EDS and for cataplexy respectively compared to placebo (34% and 25.5%). Effect size was 0.86 based on changes in the Epworth Sleepiness Scale score and weekly rate of cataplexy compared to baseline.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX 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. 

    Important Safety Information

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Narcolepsy  

    Narcolepsy is a rare, chronic, debilitating neurological disorder 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, 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, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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 Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-presents-new-analysis-of-clinical-impact-for-wakix-pitolisant-at-the-sleep-2021-annual-meeting-301309651.html

    SOURCE Harmony Biosciences

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  11. PLYMOUTH MEETING, Pa., June 9, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced applications for its Patients at the Heart Grant Program open Wednesday, June 9, 2021 for patient-centric organizations working to support and address the challenges of people living with sleep disorders and rare diseases who experience excessive daytime sleepiness (EDS).

    The Patients at the Heart Grant, a competitive grant opportunity, was developed to underscore Harmony's ongoing commitment to support the comprehensive needs of people affected by sleep disorders. Since its launch in 2019, awards have funded innovative patient-centric programs, with the most recent grants specifically awarded to programs created to support and address the unique challenges of people living with narcolepsy during the COVID-19 pandemic.

    "Our goal is to improve the lives of people living with sleep disorders and their families, and through these innovative programs, we have the opportunity to partner with and make a positive difference for these patient communities," said Cate McCanless, Harmony's Senior Vice President, Corporate Affairs and Public Policy.

    This year's charitable grant applications should focus on one or more of the following categories:

    • Programs that support persons with sleep disorders or rare diseases who experience EDS to live a fuller, more engaged life.
    • Programs that address the psycho-social challenges faced by persons or caregivers of persons with sleep disorders or rare diseases who experience EDS.
    • Programs that improve the overall support of family members of persons living with sleep disorders or rare diseases who experience EDS.
    • Creation of educational materials to increase awareness and reduce time to diagnosis of persons with sleep disorders or rare diseases who experience EDS.

    Applications from eligible organizations are being accepted beginning Wednesday, June 9, 2021 through Friday, August 20, 2021. Announcement of grant recipients will take place in September 2021. For more information, please visit https://www.harmonybiosciences.com/grant-programs/patients-at-the-heart/ for a program overview document and application. Grants can be submitted to patientsattheheart@harmonybiosciences.com.

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-announces-applications-for-2021-patients-at-the-heart-grant-program-open-june-9-301308371.html

    SOURCE Harmony Biosciences

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  12. PLYMOUTH MEETING, Pa., June 7, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that results from two post-hoc analyses from the clinical development program for WAKIX® (pitolisant) will be presented at the 35th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2021." The meeting will be held virtually June 10-13.

    One of the poster presentations is a new analysis that calculated the number needed to treat (NNT) and effect size of WAKIX to demonstrate the magnitude of its clinical effectiveness. The second poster is based on an analysis of the cataplexy data from the clinical development program for WAKIX.

    "The analyses that we are presenting at the SLEEP conference this year highlight the strength of the efficacy data for WAKIX," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "The analyses include both a traditional approach to presenting efficacy data, as well as one that underscores the clinical relevance of the findings, using NNT and effect sizes to capture overall clinical benefit."

    The data being presented by Harmony will be available in the meeting's ePoster gallery beginning June 9 through November 30, and includes:

    • Assessment of the Clinical Benefits of Pitolisant on Excessive Daytime Sleepiness and Cataplexy in Adults With Narcolepsy (Poster 504). Meskill, G, Davis, C, Zarycranski, D, Doliba, M, Schwartz JC, Dayno, J.
      • Analysis evaluating number needed to treat and effect sizes for treatment with pitolisant for excessive daytime sleepiness and cataplexy from the HARMONY-1 and HARMONY-CTP randomized, controlled, 7- or 8-week trials in adults with narcolepsy.
    • Efficacy of Pitolisant in the Treatment of Cataplexy in Adults With Narcolepsy (Poster 505). Davis, C, Zarycranski, D, Doliba, M, Dayno, J, Schwartz, JC.
      • Analysis evaluating the frequency of cataplexy attacks during treatment with pitolisant from the HARMONY-1 and HARMONY-CTP randomized, controlled, 7- or 8-week trials in adults with narcolepsy.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX 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. 

    Important Safety Information

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Narcolepsy  

    Narcolepsy is a rare, chronic, debilitating neurological disorder 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, 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, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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 Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-to-present-new-data-analyses-for-wakix-pitolisant-at-the-sleep-2021-annual-meeting-301306273.html

    SOURCE Harmony Biosciences

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  13. - venBio promoted Richard Gaster M.D., Ph.D. to Managing Partner

    venBio today announced the closing of venBio Global Strategic Fund IV, LP ("venBio Fund IV"), its fourth life sciences venture capital fund, exceeding its target and closing on approximately $550 million in capital commitments in an oversubscribed fundraise. The capital was raised from existing and new investors, including a broad range of institutional investors comprising corporate pensions, financial institutions, university endowments and foundations, family offices and funds-of-funds.

    Led by Managing Partners Corey Goodman, Ph.D., Robert Adelman, M.D., Aaron Royston, M.D., and Richard Gaster, M.D., Ph.D., venBio Fund IV will continue to invest primarily in therapeutics…

    - venBio promoted Richard Gaster M.D., Ph.D. to Managing Partner

    venBio today announced the closing of venBio Global Strategic Fund IV, LP ("venBio Fund IV"), its fourth life sciences venture capital fund, exceeding its target and closing on approximately $550 million in capital commitments in an oversubscribed fundraise. The capital was raised from existing and new investors, including a broad range of institutional investors comprising corporate pensions, financial institutions, university endowments and foundations, family offices and funds-of-funds.

    Led by Managing Partners Corey Goodman, Ph.D., Robert Adelman, M.D., Aaron Royston, M.D., and Richard Gaster, M.D., Ph.D., venBio Fund IV will continue to invest primarily in therapeutics companies that are developing biopharmaceuticals for unmet medical needs. The venBio team takes an active role with each of their portfolio companies, providing strategic guidance on a range of business activities including intellectual property, chemistry, manufacturing and controls (CMC), as well as assisting with clinical trials: from trial design to endpoints to regulatory deliberations.

    "We remain committed to our unique approach and strategy and hope the results speak for themselves – our portfolio companies have delivered four drugs to market for six clinical indications, and another seven drug candidates are demonstrating promising late-stage efficacy," said Dr. Adelman.

    "Our portfolio is directly impacting patient lives and we could not have accomplished that without the ongoing commitment from our limited partners, and we are grateful for their continued support for Fund IV," said Dr. Goodman. "With Fund IV we intend to continue our proven approach of helping to build 12-15 companies per fund while doubling down on winners by providing stronger support for our portfolio companies in crossover rounds and at IPO."

    "We are delighted to announce with the closing of Fund IV, the promotion of Dr. Rich Gaster to Managing Partner," said Dr. Royston. "Our core investment team and investment strategy remain the same as we launch our new fund."

    "Our strategy at venBio has always been to turn exceptional science into impactful medicine," said Dr. Gaster. "Every member of our team is involved in every investment that we make, and we believe this collaborative approach is what helps drive our success."

    Sidley Austin LLP served as legal adviser to venBio.

    About venBio

    Established in 2011, venBio is a life science venture capital firm that focuses on novel therapeutics for unmet medical needs. Since inception in 2011, venBio has raised nearly $1.5 billion in capital commitments and led investment rounds in 34 companies, including: venBio-founded Labrys Biologics (acquired by Teva) and ALX Oncology (NASDAQ:ALXO); Aragon Pharmaceuticals (acquired by Johnson & Johnson); Seragon Pharmaceuticals (acquired by Roche); Aurinia Pharmaceuticals (NASDAQ:AUPH); Apellis Pharmaceuticals (NASDAQ:APLS); Turning Point Therapeutics (NASDAQ:TPTX); Precision Biosciences (NASDAQ:DTIL); Akero Therapeutics (NASDAQ:AKRO); Harmony Biosciences (NASDAQ:HRMY); and Pharvaris (NASDAQ:PHVS). For more information, please visit www.venbio.com.

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  14. PLYMOUTH MEETING, Pa., May 26, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that Harmony's President and CEO, John Jacobs, will participate in fireside chats and host investor meetings at the following upcoming virtual conferences:

    • Jefferies Healthcare Conference

      Fireside Chat: June 2, 2:30pm ET
    • Goldman Sachs 42nd Annual Global Healthcare Conference

      Fireside Chat: June 10, 3:50pm ET
    • Raymond James Human Health Innovation Conference

      Fireside Chat: June 21, 3:20pm ET

    The live audio webcast of the fireside chats will be available on the investor page of Harmony's website at https://ir.harmonybiosciences.com/

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-to-participate-in-three-investor-conferences-in-june-301299307.html

    SOURCE Harmony Biosciences

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  15. PLYMOUTH MEETING, Pa., May 18, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the appointment of John C. Jacobs, President and CEO of Harmony, to the Life Sciences Pennsylvania's (LSPA) Board of Directors, effective May 6, 2021.

    John Jacobs' election to the board is accompanied by other leaders representing the breadth, diversity and geographic reach of LSPA across the medical device, diagnostic, biopharmaceutical, and research communities, bringing important expertise, insights, and partnerships that will help enhance the association's effectiveness. He will provide insight and guidance to the association given his deep experience of almost three decades in the pharmaceutical and life sciences industry.

    "The appointment of John to the Board of Directors is a testament to the work done by Harmony under his leadership to help the local patient community and raise the profile of life science companies in the region. We are grateful that John has agreed to serve on LSPA's Board of Directors and we look forward to working with him to advance the Association's initiatives," said Christopher P. Molineaux, President and CEO of Life Sciences Pennsylvania. 

    "Life Sciences Pennsylvania plays a critical and inspiring role in supporting and elevating the life sciences industry in Pennsylvania," Jacobs said. "I am honored to be joining the Board and helping to continue to make Pennsylvania a global life sciences leader."

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    About Life Sciences Pennsylvania

    Life Sciences Pennsylvania (LSPA) is the trade association for the Commonwealth's life sciences community. Founded in 1989, LSPA works to ensure Pennsylvania has a business and public policy climate that unites and incentivizes all facets of the state's diverse life sciences economy.

    LSPA represents biotechnology, medical device, diagnostic, pharmaceutical, research, and investment entities, along with myriad service providers who support the industry. Together, we unify Pennsylvania's innovators to make the Commonwealth a global life sciences leader.

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/john-c-jacobs-president-and-ceo-of-harmony-biosciences-joins-life-sciences-pennsylvania-board-of-directors-301293009.html

    SOURCE Harmony Biosciences

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  16. PLYMOUTH MEETING, Pa., May 18, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that Harmony's President and CEO, John C. Jacobs and Chief Medical Officer, Dr. Jeffrey Dayno will present a company update and host investor meetings at the Oppenheimer Rare and Orphan Disease Summit on Friday, May 21, 2021.

    A webcast of the presentation will be available on the investor page of Harmony's website at https://ir.harmonybiosciences.com/

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-to-present-at-the-oppenheimer-rare-and-orphan-disease-summit-301293012.html

    SOURCE Harmony Biosciences

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  17. WAKIX® (pitolisant) Total Revenue of $59.7 Million for First Quarter 2021

    Achieves Profitability with $7.4 Million Net Income

    Conference Call and Webcast to be Held Today at 8:30 a.m. ET 

    PLYMOUTH MEETING, Pa., May 11, 2021 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today reported financial results and business updates for the first quarter ended March 31, 2021.

    "We are off to a very strong start in 2021 with the continued demand for WAKIX® driving strong first quarter net revenues," stated John C. Jacobs…

    WAKIX® (pitolisant) Total Revenue of $59.7 Million for First Quarter 2021

    Achieves Profitability with $7.4 Million Net Income

    Conference Call and Webcast to be Held Today at 8:30 a.m. ET 

    PLYMOUTH MEETING, Pa., May 11, 2021 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today reported financial results and business updates for the first quarter ended March 31, 2021.

    "We are off to a very strong start in 2021 with the continued demand for WAKIX® driving strong first quarter net revenues," stated John C. Jacobs, President and Chief Executive Officer of Harmony. "We saw growth in the average number of patients on WAKIX and net revenue, putting us in the position of profitability this quarter, for the first time in our company history, demonstrating our resilience and performance through the lingering pandemic and anticipated seasonal payer dynamics in Q1.

    As a first-in-class medication with a novel mechanism of action and differentiated product profile, WAKIX remains the only FDA approved product for excessive daytime sleepiness or cataplexy in adult patients with narcolepsy that is not scheduled as a controlled substance, filling a significant unmet medical need for patients living with narcolepsy. With a strong cash position and positive cash flow, we are well positioned to continue supporting our commercialization efforts for WAKIX, advance and expand our clinical programs, and to consider the acquisition of complementary assets to build out our product pipeline."

    First Quarter 2021 Highlights:

    • Net product revenue of $59.7 million for the quarter ended March 31, 2021;
    • Significant growth in WAKIX sales, supported by the addition of the cataplexy indication, which strengthens the overall benefit/risk profile;
    • Increased average number of patients on WAKIX and number of unique healthcare professionals who have prescribed WAKIX since launch;
    • Net income positive for the first quarter of 2021; and
    • Cash and cash equivalents of $141.2 million.

    First Quarter 2021 Financial Results

    Net product revenues for the quarter ended March 31, 2021 were $59.7 million, compared to $19.8 million for the same period in 2020. The increase was driven by strong commercial sales of WAKIX since product launch for excessive daytime sleepiness (EDS) in adult patients with narcolepsy coupled with the addition of the cataplexy indication in October 2020, which expanded the label for WAKIX.   

    For the quarter ended March 31, 2021 GAAP net income was $7.4 million, or $0.13 per diluted share, compared to a net loss of $38.6 million or $6.30 per diluted share for the same period in 2020. For the first quarter of 2021, non-GAAP adjusted net income was $22.4 million, or $0.38 per diluted share, compared to an adjusted net loss of $6.2 million or $2.14 per diluted share for the same period in 2020.

    Reconciliations of applicable GAAP measures to non-GAAP adjusted information are included at the end of this press release.

    The components of Harmony's operating expenses include:

    • Research and Development expenses were $4.7 million in Q1 2021 as compared with $3.4 million for the same quarter in 2020, representing a 36.4% increase;
    • Sales and Marketing expenses were $15.5 million in Q1 2021 as compared to $13.3 million for the same quarter in 2020, representing a 17.0% increase;
    • General and Administrative expenses were $14.5 million in Q1 2021 as compared to $9.3 million for the same quarter in 2020, representing a 56.6% increase; and
    • Operating expenses were $34.7 million in the first quarter of 2021 as compared with $26.0 million for the same quarter in 2020, representing a 33.7% increase.

    As of March 31, 2021 Harmony had cash and cash equivalents of $141.2 million compared to cash and cash equivalents of $228.6 million, respectively, at year-end 2020. The decrease in cash is primarily attributed to the $100 million milestone payment owed under our License Agreement with Bioprojet that was paid in connection with the cataplexy indication.

    Clinical Development Update

    • Enrollment continues in Harmony's Phase 2 clinical trial evaluating the safety and efficacy of pitolisant for the treatment of EDS and other symptoms in patients with Prader-Willi Syndrome (PWS) and is on track to achieve top line data in the first half of 2022.
    • Harmony is on-track to initiate a Phase 2 trial in patients with Myotonic Dystrophy Type 1 (DM1) in the first half of 2021.  
    • Harmony's strategic partner, Bioprojet, is evaluating pitolisant in pediatric patients ages 6 to 18 years with narcolepsy in a Phase 3 trial. Bioprojet and Harmony have decided to wait for the data from Bioprojet's trial to read out in order to decide on next steps in pursuit of both a pediatric narcolepsy indication and pediatric exclusivity. Harmony anticipates providing an update on the path forward in the coming months.
    • WAKIX time-to-onset of clinical response and cardiovascular safety data were presented at the 2021 American Academy of Neurology (AAN) annual meeting in April.
    • Data on the efficacy of WAKIX in patients with a high burden of narcolepsy symptoms (both EDS and cataplexy) were recently published in the journal Sleep Medicine.

    Conference Call Today at 8:30 a.m. ET

    We are hosting our first quarter 2021 financial results conference call and webcast today beginning at 8:30 a.m. Eastern Time. The live and replayed webcast of the call will be available on the investor page of our website at https://ir.harmonybiosciences.com/. To participate in the live call by phone, dial (833) 614-1471 (domestic) or +1 (914) 987-7209 (international), and reference passcode 2269379. A replay will be accessible until May 18, 2021 by dialing (855) 859-2056 (domestic) or +1 (404) 537-3406 (international).

    Non-GAAP Financial Measures

    In addition to our GAAP results, we provide certain non-GAAP metrics including adjusted net income and adjusted net income per share. We believe that the presentation of these measures 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 adjusted net income and 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. Company management uses these non-GAAP measurements as an aid in monitoring our ongoing financial performance from quarter-to-quarter and year-to-year on a regular basis and for benchmarking against comparable companies.

    These non-GAAP financial measures are not meant to 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 its non-GAAP financial measures; and we may in the future cease to exclude items that it has historically excluded for purposes of its non-GAAP financial measures.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness 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 Usage

    WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy.

    Important Safety Information

    Contraindications

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.

    Warnings and Precautions

    WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval. 

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    Adverse Reactions

    In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and 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 Interactions

    Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half. 

    Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required (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 Populations

    WAKIX 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 FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    About Harmony Biosciences

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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.

    CONSOLIDATED

    STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

    (In thousands except share and per share data)

    (unaudited)

      Three Months Ended

    March 31,
     
      2021  2020 
    Net product revenues $59,674  $19,840 
    Cost of product sold  10,409   3,474 
    Gross profit  49,265   16,366 
    Operating expenses:        
    Research and development  4,679   3,431 
    Sales and marketing  15,506   13,254 
    General and administrative  14,547   9,290 
    Total operating expenses  34,732   25,975 
    Operating income (loss)  14,533   (9,609)
    Loss on debt extinguishment     (22,639)
    Other expense, net  (20)   
    Interest expense, net  (7,127)  (6,372)
    Income (loss) before income taxes  7,386   (38,620)
    Income taxes      
    Net income (loss) and comprehensive income (loss) $7,386  $(38,620)
    Accumulation of dividends on preferred stock     (10,445)
    Net income (loss) available to common stockholders $7,386  $(49,065)
    EARNINGS (LOSS) PER SHARE:        
    Basic $0.13  $(6.30)
    Diluted $0.13  $(6.30)
    Weighted average number of shares of common stock - basic  56,891,451   7,790,667 
    Weighted average number of shares of common stock -

    diluted
      58,805,285   7,790,667 

    HARMONY BIOSCIENCES HOLDINGS, INC.

    CONSOLIDATED BALANCE SHEETS

    (In thousands except share and per share data)

    (unaudited)

      March 31,

    2021
      December 31,

    2020
     
    ASSETS        
    CURRENT ASSETS:        
    Cash and cash equivalents $141,169  $228,631 
    Trade receivables, net  23,615   22,176 
    Inventory, net  4,405   3,823 
    Prepaid expenses  7,089   6,959 
    Other current assets  1,466   1,302 
    Total current assets  177,744   262,891 
    NONCURRENT ASSETS:        
    Property and equipment, net  842   938 
    Restricted cash  750   750 
    Intangible assets, net  157,764   162,343 
    Other noncurrent assets  152   152 
    Total noncurrent assets  159,508   164,183 
    TOTAL ASSETS $337,252  $427,074 
    LIABILITIES AND STOCKHOLDERS' EQUITY        
    CURRENT LIABILITIES:        
    Trade payables $4,391  $2,556 
    Accrued compensation  4,523   8,942 
    Accrued expenses  24,261   122,727 
    Other current liabilities  262   314 
    Total current liabilities  33,437   134,539 
    NONCURRENT LIABILITIES:        
    Deferred rent  192   212 
    Long term debt, net  194,913   194,250 
    Other noncurrent liabilities  831   893 
    Total noncurrent liabilities  195,936   195,355 
    TOTAL LIABILITIES  229,373   329,894 
    COMMITMENTS AND CONTINGENCIES (Note 9)        
    STOCKHOLDERS' EQUITY:        
    Preferred stock - $0.00001 par value; 10,000,000 shares and 0 shares authorized at March 31, 2021 and December 31, 2020, respectively; 0 shares issued and outstanding at March 31, 2021 and December 31, 2020, respectively      
    Common stock—$0.00001 par value; 500,000,000 shares authorized at March 31, 2021 and December 31, 2020, respectively; 56,892,406 shares and 56,890,569 issued and outstanding at March 31, 2021 and December 31, 2020, respectively  1   1 
    Additional paid in capital  588,687   585,374 
    Accumulated deficit  (480,809)  (488,195)
    TOTAL STOCKHOLDERS' EQUITY  107,879   97,180 
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $337,252  $427,074 

    HARMONY BIOSCIENCES HOLDINGS, INC.

    RECONCILIATION OF GAAP TO NON-GAAP RESULTS

    (In thousands except share and per share data)

    (unaudited)

      For the Three Months Ended

    March 31,
     
      2021  2020 
    Net income (loss) $7,386  $(38,620)
    Non-GAAP Adjustments:        
    Interest expense  7,127   6,372 
    Taxes      
    Depreciation  100   97 
    Amortization  4,579   1,786 
    EBITDA  19,192   (30,365)
             
    Additional Non-GAAP Adjustments:        
    Stock-based compensation expense  3,251   368 
    Loss on debt extinguishment     22,639 
    Warrant expense     1,146 
    Non-GAAP adjusted net income (loss) $22,443  $(6,212)
             
    Accumulation of yield on preferred stock     (10,445)
             
    Non-GAAP adjusted net income (loss) available to common stockholders  22,443   (16,657)
             
    GAAP reported net income (loss) per diluted share $0.13  $(6.30)
    Non-GAAP adjusted net income (loss) per diluted share $0.38  $(2.14)
             
    Weighted average number of shares of common stock used in non-GAAP diluted per share  58,805,285   7,790,667 

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

     



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  18. PLYMOUTH MEETING, Pa., April 27, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("the Company") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that data from three analyses of the clinical development program for WAKIX® (pitolisant), a medication approved for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy, will be presented at the 2021 American Psychiatric Association (APA) Annual Meeting, to be held virtually May 1-3. Two of the three poster presentations feature analyses of efficacy and safety data from the clinical development program…

    PLYMOUTH MEETING, Pa., April 27, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("the Company") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that data from three analyses of the clinical development program for WAKIX® (pitolisant), a medication approved for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy, will be presented at the 2021 American Psychiatric Association (APA) Annual Meeting, to be held virtually May 1-3. Two of the three poster presentations feature analyses of efficacy and safety data from the clinical development program for WAKIX in adult patients with narcolepsy and the third is based on a post-hoc analysis that evaluated the time to onset of clinical response for WAKIX.

    "Both excessive daytime sleepiness and cataplexy can have a significant impact on a person's daily functioning, which may greatly affect their social, emotional and psychological well-being," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "Given this, and the fact that sleep disorders are co-morbid with several different psychiatric disorders, we are pleased to share these data for WAKIX with the psychiatry and mental health professional communities for the first time."

    The data to be presented by Harmony will be available from 11 a.m. to 5:30 p.m. EDT on May 1-3, and includes:

    • Efficacy and Safety of Pitolisant in the Treatment of Excessive Daytime Sleepiness in Adult Patients With Narcolepsy: A Review of Clinical Trials (Poster 4463). L Krahn, C Davis, J-C Schwartz, J Dayno.
      • Analysis of results from HARMONY 1 and HARMONY 1bis, two pivotal randomized, placebo-controlled, 8-week trials that assessed the efficacy and safety of pitolisant for the treatment of excessive daytime sleepiness in adults with narcolepsy.
    • Efficacy of Pitolisant, a Selective Histamine 3 (H3)-Receptor Antagonist/Inverse Agonist, in the Treatment of Cataplexy in Patients With Narcolepsy (Poster 4460). M Thorpy, J-C Schwartz, C Caussé, J Dayno.
      • A review of results from HARMONY 1, HARMONY 1bis, HARMONY CTP and HARMONY 3 Phase 3 clinical trials, which assessed safety and efficacy of pitolisant for the treatment of cataplexy in adults with narcolepsy.
    • Time Course of Improvement in Excessive Daytime Sleepiness and Cataplexy During Treatment with Pitolisant in Patients with Narcolepsy (Poster 4469). C Davis, J Dayno, B Vaughn, Y Dauvilliers, J-C Schwartz.
      • Analysis evaluating the time-to-response to treatment with pitolisant for excessive daytime sleepiness and cataplexy from the HARMONY-1 and HARMONY-CTP randomized, placebo-controlled trials in adults with narcolepsy.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX has been commercially available in the U.S. since Q4 2019. It was granted orphan drug designation for the treatment of narcolepsy in 2010. 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. 

    Important Safety Information

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Narcolepsy  

    Narcolepsy is a rare, chronic, debilitating neurological disorder 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, 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, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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 Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-to-present-wakix-pitolisant-efficacy-and-safety-data-at-upcoming-2021-american-psychiatric-association-annual-meeting-301276982.html

    SOURCE Harmony Biosciences

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  19. PLYMOUTH MEETING, Pa., April 27, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare, neurological disorders who have unmet medical needs, today announced that it will issue a press release on the Company's first quarter 2021 financial results on Tuesday, May 11, 2021 at 8:00 a.m. ET. Following the release, Harmony will conduct a conference call and live webcast on the same day, at 8:30 a.m. ET.

    To participate in the call, please dial (833) 614-1471 (domestic) or +1 (914) 987-7209 (international), and reference passcode 2269379. It is recommended that you dial in at least 10 minutes prior to the call.

    The live and replayed webcast of the call will be available on the investor page of our website at https://ir.harmonybiosciences.com/.

    A replay of the call will be available within 24 hours by dialing (855) 859-2056 (domestic) or +1 (404) 537-3406 (international) and entering conference ID: 2269379. The replay will be available until May 18, 2021.

    About Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website:  www.harmonybiosciences.com.

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-announces-date-of-first-quarter-2021-financial-results-301277200.html

    SOURCE Harmony Biosciences

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  20. PLYMOUTH MEETING, Pa., April 12, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that results from two analyses from the clinical development program for WAKIX® (pitolisant) will be presented at the 2021 American Academy of Neurology (AAN) Annual Meeting, to be held virtually April 17-22. One of the poster presentations is based on a post-hoc analysis that evaluated the time-to-onset of clinical response for WAKIX and the second poster is based on a pooled analysis of the cardiovascular safety data from the clinical development…

    PLYMOUTH MEETING, Pa., April 12, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that results from two analyses from the clinical development program for WAKIX® (pitolisant) will be presented at the 2021 American Academy of Neurology (AAN) Annual Meeting, to be held virtually April 17-22. One of the poster presentations is based on a post-hoc analysis that evaluated the time-to-onset of clinical response for WAKIX and the second poster is based on a pooled analysis of the cardiovascular safety data from the clinical development program for WAKIX in adult patients with narcolepsy.

    "These analyses further characterize the efficacy and safety profile of WAKIX and its broad clinical utility for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "We look forward to sharing these data and the results of these analyses with both the neurology and narcolepsy communities."

    The data that will be presented by Harmony will be available from 7 a.m.-5 p.m. EDT on April 17-22, and include:

    • Time to Onset of Clinical Response During Treatment with Pitolisant (P26.015). C Davis, J Dayno, B Vaughn, Y Dauvilliers.
      • Analysis evaluating the time-to-onset of clinical response during treatment with pitolisant for excessive daytime sleepiness and cataplexy from the HARMONY-1 and HARMONY-CTP randomized, controlled trials in adults with narcolepsy.
    • Cardiac Safety Profile of Pitolisant in Patients with Narcolepsy (P26.016). W Winter, S Wanaski, A Patroneva, J Dayno.
      • Results from pooled analysis of two randomized, placebo-controlled seven-or eight-week studies and from a 12-month, open-label study evaluating the cardiac safety profile of pitolisant in adults with narcolepsy from the clinical development program.

    The abstracts can be viewed on the AAN website here and here.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX has been commercially available in the U.S. since Q4 2019. It was granted orphan drug designation for the treatment of narcolepsy in 2010. 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. 

    Important Safety Information

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Narcolepsy

    Narcolepsy is a rare, chronic, debilitating neurological disorder 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, 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, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Bioscience

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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 Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-to-present-wakix-pitolisant-data-at-the-2021-aan-annual-meeting-301266363.html

    SOURCE Harmony Biosciences

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  21. PLYMOUTH MEETING, Pa., April 7, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the launch of a new grant program, Progress at the Heart, to accelerate programs and solutions that address inequity in access to diagnosis, treatment, and care in the rare disease and sleep disorder community.

    Many people from underrepresented racial, ethnic, or other minority groups often face an even greater disparity in access to care, particularly given underrepresentation in research and clinical trials. In recognition of World Health Day and National Minority Health Month this April, Progress at the Heart seeks community- and education-based solutions and grant applications are currently being accepted. Up to $125,000 in grants will be awarded by September 2021 in an effort to help achieve greater equality of care within the sleep disorder community.

    "Progress at the Heart is a natural extension of our grant programs and supports our mission to address the needs of those living with sleep disorders or rare diseases who experience sleep disruption and problems related to impaired wakefulness," said Cate McCanless, Harmony's Senior Vice President, Corporate Affairs and Policy. "Language barriers, real or perceived discrimination and bias, lack of access to resources and many more factors can contribute to the overall disparities experienced by racial, ethnic and other underrepresented minorities related to sleep health and sleep disorder diagnosis and treatment. We believe that through programs funded by Progress at the Heart grants, we will be able to recognize, realize and replicate effective interventions that address these disparities in the management of patients with sleep disorders, which, in turn, could reduce overall health disparities and improve quality of life for so many people."

    Harmony is accepting two rounds of grant applications in 2021 from April 7May 31 and from June 1August 20 which will be reviewed by a cross-functional team of Harmony employees. Grants will be awarded to applicants by the end of June and September 2021, respectively. For more information, please visit www.harmonybiosciences.com/grant-programs/progress-at-the-heart and submit grant applications to progress@harmonybiosciences.com.

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-launches-grant-program-to-address-inequities-in-the-delivery-of-healthcare-in-patients-with-sleep-disorders-301263519.html

    SOURCE Harmony Biosciences

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  22. WAKIX®(pitolisant) Total Revenue of $160 Million for Full-Year 2020; $56 Million for Fourth Quarter 2020

    Clinical Utility of WAKIX was Expanded with Additional Approval for Treatment of Cataplexy in Adults with Narcolepsy

    Enrollment Continues in Phase 2 Trial of Patients with Prader-Willi Syndrome

    On Track to Initiate Two Additional Clinical Trials with Pitolisant in 2021

    Conference Call and Webcast to be Held Today at 8:30 a.m. ET

    PLYMOUTH MEETING, Pa., March 25, 2021 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today…

    WAKIX® (pitolisant) Total Revenue of $160 Million for Full-Year 2020; $56 Million for Fourth Quarter 2020

    Clinical Utility of WAKIX was Expanded with Additional Approval for Treatment of Cataplexy in Adults with Narcolepsy

    Enrollment Continues in Phase 2 Trial of Patients with Prader-Willi Syndrome

    On Track to Initiate Two Additional Clinical Trials with Pitolisant in 2021

    Conference Call and Webcast to be Held Today at 8:30 a.m. ET

    PLYMOUTH MEETING, Pa., March 25, 2021 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today reported its financial results and business updates for the fourth quarter and full year ended December 31, 2020.

    2020 Financial and Business Highlights:

    • Net product revenue of $56.3 million for the fourth quarter ended December 31, 2020; Full-year net product revenue of $159.7 million;
    • U.S. Food and Drug Administration (FDA) approved expanded use of WAKIX for the treatment of cataplexy in adults with narcolepsy;
    • Enrolled first patient in Phase 2 clinical trial of WAKIX for the treatment of excessive daytime sleepiness (EDS) in patients with Prader-Willi Syndrome (PWS);
    • Completed upsized initial public offering of 6.15 million shares at a price of $24.00 per share on August 21, 2020 for gross proceeds of $147.6 million; and
    • Added to the Russell 2000® Index and Russell 3000® Index.

    "Harmony achieved many milestones during 2020 and has entered 2021 well positioned to advance our strategic objectives," stated John C. Jacobs, President and Chief Executive Officer of Harmony Biosciences. "Key to our success was the closing of our initial public offering in August 2020 and the listing of our common stock on the Nasdaq Global Market. These events elevated Harmony's visibility among investors and provided the financial resources that we believe will allow us to continue to support our commercialization efforts for WAKIX, advance our clinical programs, and pursue the acquisition of additional assets that would be complementary to our existing commercial footprint and core areas of expertise."

    Mr. Jacobs added, "With FDA approval of the cataplexy indication for WAKIX and despite the COVID-19 pandemic, WAKIX sales posted double-digit, quarter-over-quarter growth. I am grateful to our employees who met the challenge of achieving these goals during a disruptive global pandemic. Looking ahead, our Phase 2 trial in patients with Prader-Willi Syndrome is actively enrolling patients and our clinical team has made good progress toward the initiation of two additional clinical trials, one in patients with myotonic dystrophy and another in pediatric narcolepsy patients."

    Fourth Quarter 2020 Financial Results

    For the three-month period ended December 31, 2020, Harmony reported net product revenue of $56.3 million, compared to $6.0 million for the same three-month period in 2019. The increase was primarily due to growing sales of WAKIX, which first became commercially available in November 2019, and the label expansion to include cataplexy in patients with narcolepsy that occurred on October 13, 2020.

    For the three-month period ended December 31, 2020, Harmony reported net loss of $0.2 million, or $0.00 per diluted share on a U.S. generally accepted accounting principles (GAAP) basis.

    For the three-month period ended December 31, 2020 and 2019, a comparison of total operating expenses is not meaningful and not included in this commentary as WAKIX did not become commercially available for the treatment of EDS in adult patients with narcolepsy until November 2019.

    Full Year 2020 Financial Results

    For the twelve-month period ended December 31, 2020, net product revenue grew to $159.7 million, compared to $6.0 million during the same twelve-month period in 2019. The increase was primarily due to growing sales of WAKIX following the drug's initial FDA approval in November 2019 as a treatment for EDS in adult patients with narcolepsy and the label expansion in October 2020 to include cataplexy in adult patients with narcolepsy.

    For the twelve-month period ended December 31, 2020, total operating expenses were $115.0 million compared to $150.3 million for the same twelve-month period in 2019. The decrease was primarily driven by a decrease in R&D expenses related to payment of a $50 million milestone to Bioprojet, partially offset by an increase in sales and marketing and general and administrative expenses related to the commercial launch of WAKIX.

    For the twelve-month period ended December 31, 2020, Harmony reported a net loss of $36.9 million, or $2.48 per diluted share on a GAAP basis. For the same period, non-GAAP adjusted net income was $32.5 million, and after deducting $26.9 million of accumulation of yield on preferred stock, non-GAAP adjusted net income available to common stockholders was $5.5 million, or $0.21 per diluted share. Reconciliations of applicable GAAP measures to non-GAAP adjusted information are included at the end of this press release.

    As of December 31, 2020, Harmony had cash and cash equivalents of $228.6 million compared to $24.5 million for the same period in 2019.

    2020 Select Business Highlights

    Initial Public Offering

    On August 21, 2020, Harmony completed an upsized initial public offering of 6.15 million primary shares of common stock at $24.00 per share, which began trading on the Nasdaq Global Market under the ticker symbol "HRMY". The gross proceeds, before deducting any discounts, commissions or expenses, were $147.6 million. Goldman Sachs, Jefferies and Piper Sandler managed the deal.

    FDA Approved New Indication for WAKIX

    On October 13, 2020, the FDA approved WAKIX for the treatment of cataplexy in adult patients with narcolepsy. This approval expands the label for WAKIX and broadens its clinical utility for healthcare professionals managing adult patients living with narcolepsy. In August 2019, the FDA approved WAKIX as the first treatment for EDS associated with narcolepsy that is not scheduled as a controlled substance by the U.S. Drug Enforcement Administration.

    Advances in WAKIX Clinical Programs

    On December 15, 2020 Harmony enrolled the first patient in a Phase 2 randomized, double-blind, placebo-controlled trial for EDS in patients with PWS, a rare genetic condition. The trial is designed to evaluate the safety and efficacy of WAKIX for EDS and other symptoms (behavioral, cognitive dysfunction) in patients with PWS. Harmony expects to report top-line results in first half of 2022.

    In December 2020, Harmony submitted an Investigational New Drug (IND) application to the FDA to evaluate pitolisant in patients with myotonic dystrophy (DM). The IND opened in January 2021 and Harmony is on-track to initiate a Phase 2 trial in patients with DM the first half of 2021.



    Conference Call Today at 8:30 a.m. ET

    We are hosting our fourth quarter and full-year 2020 financial results conference call and webcast today beginning at 8:30 a.m. Eastern Time. The live and replayed webcast of the call will be available on the investor page of our website at https://ir.harmonybiosciences.com/. To participate in the live call by phone, dial (833) 614-1471 (domestic) or +1 (914) 987-7209 (international), and reference passcode 5049024. A replay will be accessible until April 1, 2021 by dialing (855) 859-2056 (domestic) or +1 (404) 537-3406 (international).

    Non-GAAP Financial Measures

    In addition to our GAAP results, we provide certain non-GAAP metrics including adjusted net income and adjusted net income per share. We believe that the presentation of these measures 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 adjusted net income and 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. Company management uses these non-GAAP measurements as an aid in monitoring our on-going financial performance from quarter-to-quarter and year-to-year on a regular basis and for benchmarking against comparable companies.

    These non-GAAP financial measures are not meant to 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 its non-GAAP financial measures; and we may in the future cease to exclude items that it has historically excluded for purposes of its non-GAAP financial measures.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness 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. 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 Société Civile de Recherche (Bioprojet). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.

    Indications and Usage

    WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy.

    Important Safety Information

    Contraindications

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.

    Warnings and Precautions

    WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval. 

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    Adverse Reactions

    In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and 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 Interactions

    Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half. 

    Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required (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 Populations

    WAKIX 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 FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    About Harmony Biosciences

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and 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; 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 March 25, 2021, 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.

    CONSOLIDATED

    STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

    (In thousands except share and per share data)

    (unaudited)

      Three Months Ended

    December 31,
      Twelve Months Ended

    December 31,
     
      2020  2019  2020  2019 
    Net product revenues $56,288  $5,995  $159,742  $5,995 
    Cost of product sold  9,918   1,577   27,738   1,577 
    Gross profit  46,370   4,418   132,004   4,418 
    Operating expenses:                
    Research and development  7,618   5,276   19,448   69,595 
    Sales and marketing  17,526   16,841   55,824   44,318 
    General and administrative  13,466   15,995   39,746   36,409 
    Total operating expenses  38,610   38,112   115,018   150,322 
    Operating income (loss)  7,760   (33,694)  16,986   (145,904)
    Loss on debt extinguishment        (22,639)   
    Other expense, net        (3,071)   
    Interest expense, net  (7,966)  (2,747)  (28,220)  (6,073)
    Loss before income taxes  (206)  (36,441)  (36,944)  (151,977)
    Income taxes            
    Net loss and comprehensive loss $(206) $(36,441) $(36,944) $(151,977)
    Accumulation of dividends on preferred stock     (9,575)  (26,904)  (35,231)
    Net loss available to common stockholders $(206) $(46,016) $(63,848) $(187,208)
    NET LOSS PER SHARE:                
    Basic $(0.00) $(5.92) $(2.48) $(24.07)
    Diluted $(0.00) $(5.92) $(2.48) $(24.07)
    Weighted average number of shares of common stock - basic  56,889,460   7,778,453   25,772,419   7,777,441 
    Weighted average number of shares of common stock - diluted  56,889,460   7,778,453   25,772,419   7,777,441 



    HARMONY BIOSCIENCES HOLDINGS, INC.

    CONSOLIDATED BALANCE SHEETS

    (In thousands except share and per share data)

      December 31,

    2020
      December 31,

    2019
     
    ASSETS        
    CURRENT ASSETS:        
    Cash and cash equivalents $228,631  $24,457 
    Trade receivables, net  22,176   4,255 
    Inventory, net  3,823   1,088 
    Prepaid expenses  6,959   1,436 
    Other current assets  1,302   261 
    Total current assets  262,891   31,497 
    NONCURRENT ASSETS:        
    Property and equipment, net  938   1,330 
    Restricted cash  750   750 
    Intangible asset, net  162,343   72,185 
    Other noncurrent assets  152   941 
    Total noncurrent assets  164,183   75,206 
    TOTAL ASSETS $427,074  $106,703 
    LIABILITIES, CONVERTIBLE PREFERRED STOCK, AND STOCKHOLDERS' EQUITY

    (DEFICIT)
            
    CURRENT LIABILITIES:        
    Trade payables $2,556  $6,360 
    Accrued compensation  8,942   7,917 
    Accrued expenses  122,727   5,500 
    Other current liabilities  314   115 
    Total current liabilities  134,539   19,892 
    NONCURRENT LIABILITIES:        
    Deferred rent  212   287 
    Long term debt, net  194,250   97,946 
    Other noncurrent liabilities  893   163 
    Total noncurrent liabilities  195,355   98,396 
       TOTAL LIABILITIES  329,894   118,288 
    COMMITMENTS AND CONTINGENCIES (Note 9)        
    CONVERTIBLE PREFERRED STOCK        
    Convertible preferred stock, net of placement costs        
       Series A convertible preferred stock - $1.00 stated value; 0 shares

       and 286,000,000 shares authorized at December 31, 2020 and 2019, respectively; 0

       shares and 285,000,000 shares issued and outstanding at December 31, 2020 and

       2019, respectively
         348,203 
       Series B convertible preferred stock - $1.25 stated value; 0 shares and 8,030,000

       shares authorized at December 31, 2020 and 2019, respectively; 0 shares and

       8,000,000 shares issued and outstanding at December 31, 2020 and 2019,

       respectively
         12,023 
       Series C convertible preferred stock - $1.96 stated value; 0 shares and 25,600,000

       shares authorized at December 31, 2020 and 2019, respectively; 0 shares and

       25,510,205 shares issued and outstanding at December 31, 2020 and 2019,

       respectively
         51,051 
    STOCKHOLDERS' EQUITY (DEFICIT):        
    Preferred stock - $0.00001 par value; 10,000,000 shares and 0 shares authorized at

    December 31, 2020 and 2019, respectively; 0 shares issued and outstanding at

    December 31, 2020 and 2019, respectively
          
    Common stock—$0.00001 par value; 500,000,000 shares and 423,630,000 shares

    authorized at December 31, 2020 and 2019, respectively; 56,890,569 shares and

    7,787,470 issued and outstanding at December 31, 2020 and 2019, respectively
      1    
    Additional paid in capital  585,374    
    Accumulated deficit  (488,195)  (422,862)
         TOTAL STOCKHOLDERS' EQUITY (DEFICIT)  97,180   (422,862)
    TOTAL LIABILITIES, CONVERTIBLE PREFERRED STOCK, AND STOCKHOLDERS'

    EQUITY (DEFICIT)
     $427,074  $106,703 



    HARMONY BIOSCIENCES HOLDINGS, INC.

    RECONCILIATION OF GAAP TO NON-GAAP RESULTS

    (In thousands except share and per share data)

    (unaudited)

      Three Months Ended

    December 31,
      Twelve Months Ended

    December 31,
     
      2020  2019  2020  2019 
    GAAP reported loss $(207)  $(36,439)  $(36,944) $(151,977)
    Non-GAAP adjustments:                
    Interest expense  7,967   2,747   28,220   6,073 
    Taxes            
    Depreciation  100   95   394   395 
    Amortization  4,283   1,850   9,843   2,815 
    EBITDA  12,143   (31,747)  1,513   (142,694)
    Additional non-GAAP adjustments:                
    Stock-based compensation expense  2,924   8,805   5,190   9,909 
    Loss on debt extinguishment        22,639    
    Warrant expense        3,109    
    Non-GAAP adjusted net income (loss)  15,067   (22,942)  32,451   (132,785)
    Accumulation of dividends on preferred stock     (9,575)  (26,904)  (35,231)
    Non-GAAP net income (loss) available to common stockholders $15,067  $(32,517) $5,547  $(168,016)
    GAAP reported net loss per diluted share $(0.00) $(5.92) $(2.48) $(24.07)
    Non-GAAP adjusted net income (loss) per diluted share $0.25  $(4.18) $0.21  $(21.60)
    Weighted average number of shares of common stock used in non-

    GAAP diluted per share
      59,128,981   7,778,453   26,982,978   7,777,441 

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com



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  23. PLYMOUTH MEETING, Pa., March 19, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced publication of a white paper sharing outcomes from a Summit on Sleep Disruption the company hosted in December 2020. For the first time, this summit convened a diverse group of people living with rare diseases, caregivers and leaders of advocacy organizations, who represented diseases in which sleep disruption has been recognized, to discuss the role excessive daytime sleepiness (EDS) plays in their respective conditions. 

    PLYMOUTH MEETING, Pa., March 19, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced publication of a white paper sharing outcomes from a Summit on Sleep Disruption the company hosted in December 2020. For the first time, this summit convened a diverse group of people living with rare diseases, caregivers and leaders of advocacy organizations, who represented diseases in which sleep disruption has been recognized, to discuss the role excessive daytime sleepiness (EDS) plays in their respective conditions. 

    In many therapeutic areas, treatment is a balance of – or decision between – managing symptoms, including those not as well known or manifested, and addressing the underlying cause of the disease. Often in rare diseases, one such "hidden" symptom is EDS. The goals of the summit were to describe the physical, emotional, social and cognitive challenges faced by patients with EDS; the challenges they face in having this symptom diagnosed; and its overall impact and burden on daily functioning.

    "On the occasion of World Sleep Day 2021, we want to help raise awareness of how excessive sleepiness is prevalent across the spectrum of rare disease," said Cate McCanless, Senior Vice President, Corporate Affairs & Policy. "EDS is much more closely associated with rare sleep disorders, like narcolepsy, but it can pose significant physical, emotional, social and cognitive challenges for so many others with rare disorders who may not even realize it."

    Participants in the summit discovered common issues related to sleep disruption across different disease states that had not previously been recognized. These issues include behavioral challenges, reduced social and cognitive abilities, increased comorbidities, increased anxiety and depression, and an overall reduced health-related quality of life. The summit also highlighted challenges in sleep measurement data collection in special needs populations and a difficulty with diagnosis and treatment in comparison with symptoms more commonly associated or accepted as a part of their respective rare disease. 

    The white paper can be accessed by clicking here. Harmony Biosciences will continue to work closely with advocacy and patient organizations to share these findings with the broader rare disease community. 

    Summit participants included:

    • Terri Jo Bichell, PhD, MPH, Executive Director, COMBINEDBrain
    • Casey Gorman, Executive Director, Parents and Researchers Interested in Smith-Magenis Syndrome
    • Lindsay Jesteadt, PhD, Director of Development, Wake Up Narcolepsy
    • Jeremy Kelly, Board Chair & Lifetime Trustee, Myotonic Dystrophy Foundation
    • Erica Kelly, person with myotonic dystrophy
    • Rebecca King, Board Member, Hypersomnia Foundation
    • Steve Maier, President, KLS Foundation Board of Directors
    • Sharon O'Shaughnessy, Vice President, Board of Directors, Narcolepsy Network
    • Lara Pullen, PhD, President and Co-Founder, Chion Foundation
    • Paige Rivard, Chief Executive Officer, Prader-Willi Syndrome Association USA
    • Theresa Strong, PhD, Director of Research Programs, Foundation for Prader-Willi Research

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-hosts-summit-which-sheds-light-on-impact-of-sleep-disruption-in-rare-diseases-301250664.html

    SOURCE Harmony Biosciences

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  24. PLYMOUTH MEETING, Pa., March 17, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that results of a post-hoc analysis of data from two Phase 3 clinical trials of pitolisant, a histamine 3 (H3) receptor antagonist/inverse agonist, have been published online in Sleep Medicine. The analysis focused on patients in the trials who suffered from a high burden of narcolepsy symptoms, including both excessive daytime sleepiness (EDS) and cataplexy.

    PLYMOUTH MEETING, Pa., March 17, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that results of a post-hoc analysis of data from two Phase 3 clinical trials of pitolisant, a histamine 3 (H3) receptor antagonist/inverse agonist, have been published online in Sleep Medicine. The analysis focused on patients in the trials who suffered from a high burden of narcolepsy symptoms, including both excessive daytime sleepiness (EDS) and cataplexy.

    The publication, Efficacy of Pitolisant in Patients with High Burden of Narcolepsy Symptoms: Pooled Analysis of Short-Term, Placebo-Controlled Studies reports data pooled from two randomized, placebo-controlled, 7-or 8-week studies in adult patients with narcolepsy, in which pitolisant could be titrated to a maximum dose of 35.6 mg/day. Three independent patient subgroups were analyzed based on the following criteria for high burden of narcolepsy symptoms:

    • High burden of EDS as defined by an Epworth Sleepiness Scale (ESS) baseline score ≥16 (n = 118);
    • High burden of EDS as defined by a sleep latency ≤8 minutes on a Maintenance of Wakefulness Test (MWT) (n = 105); and,
    • High burden of cataplexy as defined by ≥15 cataplexy attacks per week (n = 31).

    Change from baseline to the end of treatment was evaluated for pitolisant compared with placebo in each patient subgroup.

    The publication reports the following results:

    • In the patients with a high burden of EDS as defined by ESS (pitolisant, n=60; placebo, n=58), least-squares mean change from baseline on the ESS was significantly greater for pitolisant (–6.1) compared with placebo (–2.3; p<0.001). In addition, significantly more patients were classified as treatment responders (ESS score reduction ≥3) in the pitolisant group (69 percent) compared with the placebo group (35.1 percent; p=0.001).
    • In the patients with a high burden of EDS as defined by the MWT (pitolisant, n=59; placebo, n=46), increase in mean sleep latency on the MWT was significantly greater for pitolisant (6.9 minutes) compared with placebo (3.4 minutes; p=0.017).
    • In the patients with a high burden of cataplexy (pitolisant, n=20; placebo, n=11), least-squares mean change in the weekly rate of cataplexy was significantly greater for pitolisant (–14.5; baseline, 23.9; final, 9.4) compared with placebo (–0.1; baseline, 23.1; final, 23.0; p=0.004).
    • The adverse event profile from this analysis was consistent with the known safety profile for pitolisant; headache, nausea, and anxiety were the most common adverse events in pitolisant-treated patients.

    "Narcolepsy is a chronic, debilitating neurological disorder characterized by sleep-wake state instability that can disrupt daily functioning, especially in patients who experience a high symptom burden," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "The findings reported in this publication highlight that WAKIX® (pitolisant) was efficacious for reducing the symptom burden for both EDS and cataplexy, even in patients with a high burden of the two most common symptoms in narcolepsy."

    Pitolisant is marketed as WAKIX in the U.S. for the treatment of EDS or cataplexy in adult patients with narcolepsy.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX has been commercially available in the U.S. since Q4 2019. It was granted orphan drug designation for the treatment of narcolepsy in 2010. 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.

    Important Safety Information 

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Narcolepsy   

    Narcolepsy is a rare, chronic, debilitating neurologic disorder 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, 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, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX®. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX®; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and the significant costs and required management time as a result of operating as a public company; the fact that the price of the company's common stock may be volatile and fluctuate substantially; 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 Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on November 12, 2020, 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 Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-highlights-publication-of-wakix-pitolisant-efficacy-data-in-sleep-medicine-301248750.html

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  25. PLYMOUTH MEETING, Pa., March 15, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the appointment of Sandip Kapadia as Chief Financial Officer effective March 29, 2021. Mr. Kapadia is an accomplished executive with more than two decades of operational experience in leading finance operations, supporting commercialization, raising capital and providing strategic guidance within the healthcare sector. 

    "We are very pleased to welcome Sandip to Harmony. His broad and deep financial leadership experience will be an asset as we continue to build upon the momentum of 2020. Sandip's expertise as a strategic and tactical financial executive will contribute significantly to our business as we plan to deliver long-term growth to our company and key stakeholders," said John C. Jacobs, Harmony's President and Chief Executive Officer.

    Mr. Kapadia has over 25 years of industry experience across multiple therapeutic areas and has provided strategic financial oversight and guidance to companies both in the United States and abroad. Most recently, he served as the Chief Financial Officer at Intercept, where he built upon the company's key functional capabilities, developed and executed financial and capital strategies to prepare for commercial launch and raised over $700 million in various financings. Prior to Intercept, Mr. Kapadia spent the majority of his career at Novartis and Novartis affiliates in the United States, Switzerland, the Netherlands and the United Kingdom, where he held positions of varying levels of responsibility, including most recently serving as Chief Financial Officer of North America at Novartis's generic division, Sandoz. He currently serves on the board of directors of Passage Bio, Inc., Molecular Partners AG and VectivBio Holding AG. 

    Mr. Kapadia is a Certified Public Accountant and holds a MBA from Rutgers University and a BBA from Montclair State University.

    "I am delighted to join the team at Harmony and contribute to the company's continued success. I look forward to helping the company create long term value for investors, healthcare providers and patients, true to its mission," said Mr. Kapadia.

    Mr. Kapadia replaces Susan Drexler who will be stepping down as Chief Financial Officer as of March 29, 2021 to pursue other career opportunities.

    About Harmony Biosciences 

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX®. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX®; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and the significant costs and required management time as a result of operating as a public company; the fact that the price of the company's common stock may be volatile and fluctuate substantially; 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 Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on November 12, 2020, 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 Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com 

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-appoints-sandip-kapadia-as-chief-financial-officer-301246924.html

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  26. PLYMOUTH MEETING, Pa., March 4, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that it will issue a press release on the Company's fourth quarter and full year 2020 financial results on Thursday, March 25, 2021 at 8:00 a.m. ET. Following the release, Harmony will conduct a conference call and live webcast on the same day, at 8:30 a.m. ET.

    To participate in the call, please dial (833) 614-1471 (domestic) or +1 (914) 987-7209 (international), and reference passcode 5049024. It is recommended that you dial in at least 10 minutes prior to the call.

    The live and replayed webcast of the call will be available on the investor page of our website at https://ir.harmonybiosciences.com/.

    A replay of the call will be available within 24 hours by dialing (855) 859-2056 (domestic) or +1 (404) 537-3406 (international) and entering conference ID: 5049024. The replay will be available until April 1, 2021.

    About Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-announces-date-of-fourth-quarter-and-full-year-2020-financial-results-301240129.html

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  27. PLYMOUTH MEETING, Pa., March 2, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, announced today that the company has been certified by independent analysts as a Great Place to Work® for the third year in a row. This certification is based on validated feedback from Harmony's employees in an anonymous survey intended to determine exceptional workplace cultures.

    "At Harmony, our team works hard each day to make a difference in the lives of patients and their families who live with rare neurological disorders," said John C. Jacobs, President and CEO. "We are pleased to receive this honor for the third year and grateful that Harmony team members experience the company as a welcoming, caring and collaborative environment that aims to make a positive impact in the patient community."

    Harmony's employees completed survey questions regarding the credibility and competence of management, workplace culture, diversity and inclusion, sense of camaraderie, ethical business practices and available resources. A summary of these ratings is available at: https://www.greatplacetowork.com/certified-company/7011504.

    About Harmony Biosciences 

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA. The company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.  

    About Great Place to Work®

    Great Place to Work® is the global authority on high-trust, high-performance workplace cultures. Through proprietary assessment tools, advisory services, and certification programs, including Best Workplaces lists and workplace reviews, Great Place to Work® provides the benchmarks, framework, and expertise needed to create, sustain, and recognize outstanding workplace cultures. In the United States, Great Place to Work® produces the annual Fortune "100 Best Companies to Work For®" list and a series of Great Place to Work® Best Workplaces lists, including lists for Millennials, Women, Diversity, Small and Medium Companies and over a half dozen different industry lists. 

     

    Harmony Biosciences Media Contact: 

    Nancy Leone 

    215-891-6046 

    nleone@harmonybiosciences.com 

    Harmony Biosciences Investor Contact: 

    Lisa Caperelli 

    484-539-9736 

    lcaperelli@harmonybiosciences.com

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-certified-as-a-great-place-to-work-for-the-third-year-in-a-row-301237928.html

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  28. PLYMOUTH MEETING, Pa. and CHICAGO, Dec. 17, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the Company will be added to the Russell 2000® and Russell 3000® Indexes effective after the U.S. market closes on Friday, December 18.

    "Inclusion in the Russell 2000® and Russell 3000® Indexes is an important milestone for Harmony," said John C. Jacobs, Harmony's President and Chief Executive Officer. "We believe it will enhance Harmony's visibility in the investment community and broaden our shareholder base."

    The Russell Indexes are a leading benchmark for institutional investors to track current and historical market performance. The Russell 3000® Index measures the performance of the largest 3,000 publicly traded companies in the U.S., representing the top 98% of the U.S. equity market. The Russell 2000® Index measures the performance of the 2,000 smallest-cap U.S. companies in the Russell 3000® Index.

    About Harmony Biosciences

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX®. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX®; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and the significant costs and required management time as a result of operating as a public company; the fact that the price of the company's common stock may be volatile and fluctuate substantially; 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 Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on November 12, 2020, 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 Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    484-539-9736

    lcaperelli@harmonybiosciences.com

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-to-be-added-to-russell-2000-and-russell-3000-indexes-301194641.html

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  29. PLYMOUTH MEETING, Pa. and CHICAGO, Dec. 15, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the first patient has been enrolled in a Phase 2 trial evaluating the safety and efficacy of pitolisant for the treatment of excessive daytime sleepiness (EDS) and other key symptoms in patients with Prader-Willi Syndrome (PWS).

    "We view pitolisant as a portfolio in a product opportunity and are taking a mechanism-based approach to drug development in managing its life cycle with the goal of expanding its clinical utility," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "We believe pitolisant could offer an important therapeutic benefit in people living with PWS based on preclinical models of the disorder, pitolisant's demonstrated efficacy in improving EDS in patients with narcolepsy, and anecdotal evidence in patients with PWS. We look forward to continuing our collaboration with the Foundation for Prader-Willi Research, the Prader-Willi Syndrome Association USA, and the patient and family community as we advance our development program in order to help address this unmet medical need."

    PWS is a genetic orphan/rare 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. The hypothalamic dysfunction in patients with PWS results in two of the main symptoms of the disorder: EDS and hyperphagia (an abnormally increased appetite for and consumption of food). 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.

    "We support and encourage research that can potentially help reduce the symptom burden for children and adults living with PWS. We are grateful that Harmony is studying pitolisant in a clinical trial, which may bring us one step closer to finding an important therapeutic advancement," said John Walter, Chief Executive Officer, Foundation for Prader-Willi Research and Paige Rivard, Chief Executive Officer, Prader-Willi Syndrome Association USA in a joint statement.

    The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled study designed to assess the safety and efficacy of pitolisant in patients with PWS ages 6-to-65. An estimated 60-70 patients will be enrolled at approximately 10 sites across the United States. Patients will be randomized to receive one of two doses of pitolisant or placebo for eight weeks of stable dosing following a three-week titration period. The trial's primary objective is to assess improvement in EDS as measured by the Multiple Sleep Latency Test. Topline results are anticipated in the first half of 2022. Patients who complete the trial will be eligible to participate in an open-label extension phase to assess the long-term safety and effectiveness of pitolisant. 

    "I have seen first-hand the debilitating effects this rare disease can have on patients with Prader-Willi Syndrome," said Dr. Daniel Glaze from Baylor College of Medicine and Texas Children's Hospital, and investigator in the Phase 2 clinical trial. "Excessive daytime sleepiness is more common in patients with PWS than previously appreciated and results in significant impact on daily functioning. In addition, EDS has not been studied in clinical trials to date in patients with PWS. I look forward to participating in this trial to assess the effect of pitolisant on EDS and other related symptoms."  

    Pitolisant is marketed as WAKIX® in the U.S. for the treatment of EDS or cataplexy in adult patients with narcolepsy.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX has been commercially available in the U.S. since Q4 2019. It was granted orphan drug designation for the treatment of narcolepsy in 2010. 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.

    Important Safety Information

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Harmony Biosciences

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX®. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX®; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and the significant costs and required management time as a result of operating as a public company; the fact that the price of the company's common stock may be volatile and fluctuate substantially; 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 Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on November 12, 2020, 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 Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    484-539-9736

    lcaperelli@harmonybiosciences.com

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-enrolls-first-patient-in-phase-2-trial-evaluating-pitolisant-for-excessive-daytime-sleepiness-in-patients-with-prader-willi-syndrome-301192466.html

    SOURCE Harmony Biosciences

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  30. WAKIX®(pitolisant) Total Revenue of $45.6 Million for Third Quarter of 2020

    Differentiated Product Profile Aligns with Unmet Medical Need

    On Track to Initiate Phase 2 Trial in Patients with Prader-Willi Syndrome by Year End

    Conference Call and Webcast to be held today at 8:30 a.m. Eastern Time

    PLYMOUTH MEETING, Penn. and CHICAGO, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today reported financial results for the quarter ended September 30, 2020, and provided recent business updates.

    "Harmony experienced another productive…

    WAKIX® (pitolisant) Total Revenue of $45.6 Million for Third Quarter of 2020

    Differentiated Product Profile Aligns with Unmet Medical Need

    On Track to Initiate Phase 2 Trial in Patients with Prader-Willi Syndrome by Year End

    Conference Call and Webcast to be held today at 8:30 a.m. Eastern Time

    PLYMOUTH MEETING, Penn. and CHICAGO, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today reported financial results for the quarter ended September 30, 2020, and provided recent business updates.

    "Harmony experienced another productive quarter with continued WAKIX revenue growth and meaningful advancement of our key clinical programs," commented John C. Jacobs, Harmony's President and Chief Executive Officer. "WAKIX sales continued to increase on a quarterly basis through the COVID-19 pandemic, reflecting the unmet medical need for a first-in-class medication with a novel mechanism of action. The recent approval of the cataplexy indication for WAKIX expanded the label in narcolepsy which, along with WAKIX being the only FDA approved product for narcolepsy that is not scheduled as a controlled substance, provides additional commercial opportunity. With a robust cash position stemming from our recent IPO, we have the financial resources to continue supporting our commercialization efforts for WAKIX, advance our clinical programs, and to pursue the acquisition of additional assets that would be complimentary to our existing commercial footprint and core areas of expertise."

    Third Quarter 2020 Financial Highlights:

    • Net product revenue was $45.6 million for the third quarter ended September 30, 2020.
    • Research and development expenses were $4.2 million for the third quarter of 2020 compared with $4.3 million for the third quarter of 2019.
    • Sales and marketing expenses were $12.6 million for the third quarter of 2020 compared with $12.9 million for the third quarter of 2019.
    • General and administrative expenses were $10.5 million for the third quarter of 2020 compared with $12.6 million for the third quarter of 2019.
    • Net income was $1.9 million for the third quarter of 2020 compared with a net loss of $31.9 million for the third quarter of 2019.
    • Cash and cash equivalents as of September 30, 2020 was $221.7 million.

    Harmony Founder and Chairman, and Paragon Biosciences Chairman and CEO, Jeff Aronin, commented, "From the time we founded Harmony, to its FDA approval of WAKIX, and recent approval of a second indication, the team has worked relentlessly to build a company that has contributed to scientific innovation in neurological disorders. We are pleased with Harmony's continued progress to address unmet medical needs, including the benefits that WAKIX has provided for people living with narcolepsy."  

    Recent Program Highlights and Updates:

    WAKIX® (pitolisant) in Narcolepsy

    • On October 13, 2020, the U.S. Food and Drug Administration (FDA) approved WAKIX for the treatment of cataplexy in adult patients with narcolepsy. This approval expands the label for WAKIX and broadens its clinical utility for healthcare professionals managing adult patients living with narcolepsy. WAKIX is the first and only treatment approved by the FDA for people with excessive daytime sleepiness (EDS) or cataplexy associated with narcolepsy that is not scheduled as a controlled substance by the U.S. Drug Enforcement Administration (DEA).

    Pitolisant in Patients with Prader-Willi Syndrome (PWS) and Myotonic Dystrophy (DM)

    • PWS and DM are rare, genetic multi-system diseases for which there are no approved treatments for many of the symptoms, resulting in significant unmet medical needs.

      • For PWS, clinical sites are being activated to conduct a Phase 2 randomized, double-blind, placebo-controlled trial to assess the safety and efficacy of pitolisant in patients with PWS, with the primary endpoint being EDS. We are on-track to initiate this trial this year.

      • For DM, we are on-track to submit an IND by year end with the plan to initiate a Phase 2 clinical trial in the first half of 2021.

    Recent Business Updates:

    • On August 21, 2020 we successfully completed our upsized IPO of 6,151,162 shares of common stock at a public offering price of $24.00 per share, including an exercise in full of the underwriters' option to purchase additional shares. The gross proceeds from the offering, before deducting underwriting discounts and commissions and other offering expenses, were $147.6 million.
    • In November, we expanded the depth and breadth of our Board of Directors with the addition of Mark Graf and Eric Motley.

    Conference Call Today at 8:30 a.m. ET

    We are hosting our third quarter 2020 financial results conference call and webcast today beginning at 8:30 a.m. Eastern Time. The live and replayed webcast of the call will be available on the investor page of our website at https://ir.harmonybiosciences.com/. To participate in the live call by phone, dial (833) 614-1471 (domestic) or +1 (914) 987-7209 (international), and reference passcode 7489154. A replay will be accessible until November 19, 2020 by dialing (855) 859-2056 (domestic) or +1 (404) 537-3406 (international).

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness 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. WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. Although, the mechanism of action of WAKIX is unclear, 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 Société Civile de Recherche (Bioprojet). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.

    Indications and Usage

    WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy.

    Important Safety Information

    Contraindications

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.

    Warnings and Precautions

    WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval. 

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    Adverse Reactions

    In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and 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 Interactions

    Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half. 

    Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required (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 Populations

    WAKIX 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 FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    About Harmony Biosciences

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX®. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX®; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and the significant costs and required management time as a result of operating as a public company; the fact that the price of the company's common stock may be volatile and fluctuate substantially; 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 Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on November 12, 2020, 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.

    UNAUDITED CONDENSED CONSOLIDATED

    STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)

    (In thousands except share and per share data)

     Three Months Ended September 30,Nine Months Ended September 30,
     2020201920202019
    Net product revenues$45,609 $ $103,454 $ 
    Cost of product sold 7,890    17,820   
    Gross profit 37,719    85,634   
    Operating expenses:    
    Research and development 4,230  4,336  11,829  62,319 
    Sales and marketing 12,601  12,908  38,297  27,477 
    General and administrative 10,508  12,560  26,280  22,415 
    Total operating expenses 27,339  29,804  76,406  112,211 
    Operating income (loss) 10,380  (29,804) 9,228  (112,211)
    Loss on debt extinguishment     (22,639)  
    Other expense, net (1,525)   (3,071)  
    Interest expense, net (6,946) (2,095) (20,254) (3,326)
    Income (loss) before income taxes 1,909  (31,899) (36,736) (115,537)
    Income taxes        
    Net income (loss) and comprehensive loss$1,909 $(31,899)$(36,736)$(115,537)
    Accumulation of dividends on preferred stock (6,013) (9,027) (26,904) (25,656)
    Net loss available to common stockholders$(4,104)$(40,926)$(63,640)$(141,193)
    NET LOSS PER SHARE:    
    Basic$(0.14)$(5.26)$(4.15)$(18.15)
    Diluted$(0.14)$(5.26)$(4.15)$(18.15)
    Weighted average number of shares of common stock - basic 30,212,959  7,777,100  15,324,362  7,777,100 
    Weighted average number of shares of common stock - diluted 30,212,959  7,777,100  15,324,362  7,777,100 
         

    HARMONY BIOSCIENCES HOLDINGS, INC.

    UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands except share and per share data)

     September 30, 2020December 31, 2019
    ASSETS  
    CURRENT ASSETS:  
    Cash and cash equivalents$221,740 $24,457 
    Trade receivables, net 16,326  4,255 
    Inventory, net 2,311  1,088 
    Prepaid expenses 4,240  1,436 
    Other current assets 5,625  261 
    Total current assets 250,242  31,497 
    NONCURRENT ASSETS:  
    Property and equipment, net 1,038  1,330 
    Restricted cash 750  750 
    Intangible asset, net 66,625  72,185 
    Other noncurrent assets 1,418  941 
    Total noncurrent assets 69,831  75,206 
    TOTAL ASSETS$320,073 $106,703 
    LIABILITIES, CONVERTIBLE PREFERRED STOCK, AND STOCKHOLDERS' EQUITY (DEFICIT)  
    CURRENT LIABILITIES:  
    Trade payables$9,347 $6,360 
    Accrued compensation 5,243  7,917 
    Accrued expenses 17,200  5,500 
    Other current liabilities   115 
    Total current liabilities 31,790  19,892 
    NONCURRENT LIABILITIES:  
    Deferred rent 305  287 
    Long term debt, net 192,858  97,946 
    Other noncurrent liabilities 571  163 
    Total noncurrent liabilities 193,734  98,396 
    TOTAL LIABILITIES 225,524  118,288 
    COMMITMENTS AND CONTINGENCIES (Note 9)  
    CONVERTIBLE PREFERRED STOCK  
    Convertible preferred stock, net of placement costs  
    Series A convertible preferred stock - $1.00 stated value; 0 shares and 286,000,000 shares authorized at September 30, 2020 and December 31, 2019, respectively; 0 shares and 285,000,000 shares issued and outstanding at September 30, 2020 and December 31, 2019, respectively   348,203 
    Series B convertible preferred stock - $1.25 stated value; 0 shares and 8,030,000 shares authorized at September 30, 2020 and December 31, 2019, respectively; 0 shares and 8,000,000 shares issued and outstanding at September 30, 2020 and December 31, 2019, respectively   12,023 
    Series C convertible preferred stock - $1.96 stated value; 0 shares and 25,600,000 shares authorized at September 30, 2020 and December 31, 2019, respectively; 0 shares and 25,510,205 shares issued and outstanding at September 30, 2020 and December 31, 2019, respectively   51,051 
    STOCKHOLDERS' EQUITY (DEFICIT):    
    Preferred stock - $0.00001 par value; 10,000,000 shares and 0 shares authorized at September 30, 2020 and December 31, 2019, respectively; 0 shares issued and outstanding at September 30, 2020 and December 31, 2019    
    Common stock - $0.00001 par value; 500,000,000 shares and 423,630,000 shares authorized at September 30, 2020 and December 31, 2019, respectively; 56,888,625 shares and 7,787,470 issued and outstanding at September 30, 2020 and December 31, 2019, respectively 1   
    Additional paid in capital 582,535   
    Accumulated deficit (487,987) (422,862)
    TOTAL STOCKHOLDERS' EQUITY (DEFICIT) 94,549  (422,862)
    TOTAL LIABILITIES, CONVERTIBLE PREFERRED STOCK, AND STOCKHOLDERS' EQUITY (DEFICIT)$320,073 $106,703 
       

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Primary Logo

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  31. PLYMOUTH MEETING, Pa. and CHICAGO, Nov. 11, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the appointment of Mark Graf and Eric Motley to the Company's Board of Directors. Mark Graf is an executive with deep financial expertise as well as an experienced board member with a strong background in corporate governance. Eric Motley is an expert in human capital development who has advised previous government, not-for-profit and private sector organizations as an organizational development executive, in addition to his governance…

    PLYMOUTH MEETING, Pa. and CHICAGO, Nov. 11, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the appointment of Mark Graf and Eric Motley to the Company's Board of Directors. Mark Graf is an executive with deep financial expertise as well as an experienced board member with a strong background in corporate governance. Eric Motley is an expert in human capital development who has advised previous government, not-for-profit and private sector organizations as an organizational development executive, in addition to his governance experience on corporate and philanthropic boards.

    "The wealth of financial, management and governance expertise Mark and Eric bring adds to the depth and breadth of Harmony's board," said Jeff Aronin, Harmony Founder and Chairman, and Paragon Biosciences Chairman and CEO. "Their insights will be invaluable to help oversee the strategic growth of Harmony as a public company. We are honored to have them join our Board."

    Before retiring in 2019, Mark Graf spent over eight years as the Chief Financial Officer of Discover Financial Services and was a core member of the firm's Executive Committee. In addition to all aspects of financial management, he was responsible for managing the company's $110 billion balance sheet, corporate development and investor relations. He was named the #1 Chief Financial Officer in the consumer financial services sector by the readers of Institutional Investor magazine every year from 2014 through 2019. 

    Prior to joining Discover, Mark spent five years in the private equity realm. Most recently, he was an Investment Advisor at Aquiline Capital Partners, a private equity firm specializing in investments in financial services and financial technology enterprises. Prior to Aquiline, he briefly served as a Partner at Barrett Ellman Stoddard Capital Partners and he served as Chief Financial Officer of Fifth Third Bancorp from 2004 to 2006 and was its Corporate Treasurer from 2001 to 2004. He also served in various roles at AmSouth Bancorporation from 1994 to 2001.

    Mark brings a strong background in corporate governance and qualifies as an audit committee financial expert. Mark holds a Bachelor of Science in Economics from the University of Pennsylvania's Wharton School.

    Eric Motley is a senior management, talent, and organizational development executive with 20 years of experience assisting government, not-for-profit, and private sector organizations with building high-performing teams. Currently, Eric is an Executive Vice President and the Corporate Secretary at the Aspen Institute, a global, non-partisan public policy organization, based in Washington, DC. He previously served as Director of the Henry Crown Fellowship Program, recognized by many as one of the most prestigious leadership development programs in the United States. 

    Eric served at the White House for over four years, eventually as a Special Assistant to President George W. Bush, working in the Office of Presidential Personnel, where he managed the appointment process for over 1,200 presidentially appointed advisory board and commission positions.

    Eric also has strong governance experience through his work with corporate and philanthropic boards. He earned his undergraduate degree from Samford University, and received his master and doctorate degrees at the University of St. Andrews in Scotland.

    About Harmony Biosciences

    Harmony Biosciences is a commercial stage pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established by Paragon Biosciences, LLC, and is focused on providing novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    484-539-9736

    lcaperelli@harmonybiosciences.com

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-appoints-mark-graf-and-eric-motley-to-its-board-of-directors-301171325.html

    SOURCE Harmony Biosciences

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  32. PLYMOUTH MEETING, Pa. and CHICAGO, Oct. 28, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced that it will issue a press release on the Company's third quarter financial results on Thursday, November 12, 2020 at 8:00 a.m. ET. Following the release, Harmony will conduct a conference call and live webcast on the same day, at 8:30 a.m. ET.

    To participate in the call, please dial (833) 614-1471 (domestic) or +1 (914) 987-7209 (international), and reference passcode 7489154. It is recommended that you dial in at least 10 minutes prior to the call.

    The live and replayed webcast of the call will be available on the investor page of our website at https://ir.harmonybiosciences.com/.

    A replay of the call will be available within 24 hours by dialing (855) 859-2056 (domestic) or +1 (404) 537-3406 (international) and entering conference ID: 7489154. The replay will be available until November 19, 2020.

    About Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, please visit the company's website: www.harmonybiosciences.com.

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    610-608-0215

    lcaperelli@harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/harmony-biosciences-announces-date-of-third-quarter-2020-financial-results-301161163.html

    SOURCE Harmony Biosciences

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  33. PLYMOUTH MEETING, Pa. and CHICAGO, Oct. 14, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the U.S. Food and Drug Administration (FDA) has approved WAKIX® (pitolisant) for the treatment of cataplexy in adult patients with narcolepsy. WAKIX is the first and only treatment approved by the FDA for people with excessive daytime sleepiness or cataplexy associated with narcolepsy that is not scheduled as a controlled substance by the U.S. Drug Enforcement Administration. WAKIX received FDA approval for the treatment of excessive…

    PLYMOUTH MEETING, Pa. and CHICAGO, Oct. 14, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the U.S. Food and Drug Administration (FDA) has approved WAKIX® (pitolisant) for the treatment of cataplexy in adult patients with narcolepsy. WAKIX is the first and only treatment approved by the FDA for people with excessive daytime sleepiness or cataplexy associated with narcolepsy that is not scheduled as a controlled substance by the U.S. Drug Enforcement Administration. WAKIX received FDA approval for the treatment of excessive daytime sleepiness in adult patients with narcolepsy in August 2019.

    "All people living with narcolepsy have excessive daytime sleepiness and up to two-thirds of them also experience cataplexy, which is one of the most debilitating symptoms of this chronic, rare neurological disorder," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "Today's FDA approval of the cataplexy indication for WAKIX, coupled with it being the first and only non-scheduled treatment option approved for adult patients with narcolepsy to treat both excessive daytime sleepiness or cataplexy, provides an opportunity for WAKIX to offer broad clinical utility to healthcare professionals managing adult patients living with narcolepsy."

    "This approval underscores our ongoing commitment to support people who are living with narcolepsy," said John C. Jacobs, Harmony's President and Chief Executive Officer. "At Harmony, we always keep patients at the heart of all we do and with this approval, we are inspired to continue our mission to develop novel treatment options for those living with rare, neurological disorders who have unmet medical needs."

    "From the very beginning, our passion at Bioprojet has been to bring WAKIX to people living with the daily challenges that are associated with impaired wakefulness and the risk of cataplexy attacks. This approval highlights the recognition by the FDA of a new therapeutic option in the treatment of the two major symptoms of narcolepsy by a drug with a novel mechanism of action," said Professor Jean-Charles Schwartz, PhD. Professor Schwartz is the discoverer of histaminergic neurotransmission in the brain and the histamine-3 receptor, which is the target receptor of WAKIX. He is also the co-founder, with Jeanne-Marie Lecomte, of Bioprojet.

    WAKIX, a first-in-class medication, is a selective histamine 3 (H₃) receptor antagonist/inverse agonist that works through a novel mechanism of action to increase the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain. WAKIX is administered orally, once daily in the morning upon wakening.

    FDA approval of WAKIX for the treatment of cataplexy in adult patients with narcolepsy is based on the results from two randomized, controlled trials (HARMONY CTP and HARMONY 1) from the clinical development program for WAKIX. After a complete response letter (CRL) for the cataplexy indication was issued by the FDA in August 2019, Harmony met with the Agency in December 2019 to discuss the deficiencies cited in the CRL. After that meeting, the FDA agreed to review the reanalysis of the HARMONY 1 data that were submitted during the NDA review, after which the Agency acknowledged that those analyses confirmed that a statistically significant reduction in the rate of cataplexy for WAKIX compared to placebo was demonstrated, which supported the positive results from the HARMONY CTP trial. Subsequently, the FDA recommended that Harmony submit a resubmission to the CRL, which Harmony submitted in August 2020 and has led to today's FDA approval of the cataplexy indication for WAKIX.

    Cataplexy is characterized by sudden, temporary loss of muscle tone and is often triggered by strong emotions, such as excitement or laughter. Cataplexy can be subtle, such as drooping of eyelids, or severe, such as knee buckling or total body collapse. Cataplexy may often go unrecognized because of the subtle nature of the symptoms in some patients, variability of how cataplexy is expressed, and/or lack of patient complaints or physician recognition of the symptoms as manifestations of cataplexy. Up to two-thirds of all patients with narcolepsy have cataplexy, which can have a significant impact on a person's daily functioning.

    About WAKIX® (pitolisant) Tablets

    WAKIX, a first-in-class medication, was approved by the U.S. Food and Drug Administration in August 2019 for the treatment of excessive daytime sleepiness 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. 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.

    Indications and Usage

    WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy.

    Important Safety Information

    Contraindications

    WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.

    Warnings and Precautions

    WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval. 

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD).

    Adverse Reactions

    In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and 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 Interactions

    Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half. 

    Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required (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 Populations

    WAKIX 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 FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    About Narcolepsy

    Narcolepsy is a rare, chronic, debilitating neurological disorder of sleep-wake state instability that impacts up to 165,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy along with other manifestations of REM sleep dysregulation, 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. Cataplexy is characterized by sudden temporary loss of muscle tone and is often triggered by strong emotions, such as excitement or laughter. In most patients, narcolepsy is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. 

    Forward Looking Statement

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX®. 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 plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX®; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and 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; and the significant costs and required management time as a result of operating as a public company; the fact that the price of the company's common stock may be volatile and fluctuate substantially; 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 final prospectus in connection with our initial public offering dated August 18, 2020 filed with the Securities and Exchange Commission (the "SEC") on August 19, 2020 pursuant to Rule 424(b) under the Securities Act of 1933, as amended, 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 Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    484-539-9736

    lcaperelli@harmonybiosciences.com

     

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  34. PLYMOUTH MEETING, Pa. and CHICAGO, Sept. 22, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced recipients of the second-annual Patients at the Heart Grant in recognition of World Narcolepsy Day. The Patients at the Heart Grant was originally designed as an annual competitive grant opportunity dedicated to funding innovative patient-centric programs; however, this year's grants were specifically awarded to programs created to support and address the unique challenges of people living with narcolepsy during the COVID-19 pandemic…

    PLYMOUTH MEETING, Pa. and CHICAGO, Sept. 22, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced recipients of the second-annual Patients at the Heart Grant in recognition of World Narcolepsy Day. The Patients at the Heart Grant was originally designed as an annual competitive grant opportunity dedicated to funding innovative patient-centric programs; however, this year's grants were specifically awarded to programs created to support and address the unique challenges of people living with narcolepsy during the COVID-19 pandemic.

    "We are proud to recognize the second-annual World Narcolepsy Day by continuing to support the narcolepsy community, especially during a year when all of us need support more than ever," said Cate McCanless, Harmony's Vice President, External Affairs. "Being able to pivot our Patients at the Heart Grant opportunity to fund programs that support and address the challenges of people with narcolepsy during the COVID-19 pandemic underlines our commitment to keeping patients at the heart of everything we do."

    World Narcolepsy Day, which takes place on September 22, is a day dedicated to raising awareness of narcolepsy on a global scale. In 2020, the day is co-led by 26 patient advocacy organizations across six continents.

    The grant program awarded $65,000 to four organizations in 2019, a figure which this year rose to $74,000 awarded to three organizations. The following organizations were selected as grant recipients:

    Wake Up Narcolepsy (WUN)

    Program: Unique Digital Resource Collection

    These funds enabled the development of the 'Doing Life During COVID' page of the Wake Up Narcolepsy website. This page includes links to free and affordable resources on healthy eating, working from home, homeschooling, exercise apps, meditation and mental health resources, access to key areas of support and relevant organizations and more.

    Program: Digital Support Groups

    WUN offers free, targeted online support groups to individuals with narcolepsy and their families, world-wide. Programs are designed to bring together individuals who have been affected by the diagnosis of narcolepsy and provide the support and community necessary to thrive. These funds were used to aid the expansion of digital support groups due to the demand for online programming during COVID-19.

    Project Sleep

    Program: Rising Voices of Narcolepsy (RVN)

    Rising Voices of Narcolepsy, Project Sleep's leadership training program, helps people with narcolepsy share their story more effectively through writing and speaking. This online program empowers participants while fostering a new generation of narcolepsy advocates spreading awareness. In the wake of COVID-19, this program saw a significant increase in interested participants. Given its existing online format, this grant will enhance the program by providing more frequent opportunities for trained speakers and writers to share their story and offering additional support tools to help them adapt to the demands of a virtual presentation environment.

    Program: Narcolepsy Awareness Program (NAP)

    The NAP aims to reduce both self-stigma and societal stigma by providing people with narcolepsy with the leadership, role modeling, and information to feel hope, inspiration, connection and empowerment to succeed with narcolepsy. The grant was used to produce a new article and video series featuring health care professionals from the narcolepsy community and additional live broadcast interviews with key narcolepsy and sleep experts.

    Narcolepsy Network

    Program: Children's Outreach by Narcolepsy Network Educate, Connect, Thrive (CONNECT)

    Narcolepsy Network established the CONNECT program for youth that aims to address the needs and impact of COVID-19 on children and their parents affected by narcolepsy within the ages of 8-13 years old. The program will also aim to connect the community virtually to minimize the sense of seclusion fostered by COVID-19, as well as promote advocacy within the community by inviting a diverse audience and guest speakers.

    Program: Cooking Connects

    This webinar series will promote healthy eating for people with narcolepsy and their families and foster a sense of community during virtual times.

    Program: Narcolepsy Access Project (NAP)

    Due to COVID-19, Narcolepsy Network suspended their dozens of in-person support groups and utilized this grant to host virtual support group meetings.

    About Narcolepsy

    Narcolepsy is a rare, chronic, debilitating neurologic disorder of sleep-wake state instability that impacts up to 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, 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, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established in October 2017 by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. 

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    484-539-9736

    lcaperelli@harmonybiosciences.com

     

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  35. PLYMOUTH MEETING, Pa. and CHICAGO, Aug. 28, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, announced today results from post-hoc analyses of the efficacy and safety data for WAKIX® (pitolisant) presented at the 34th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2020." The meeting is being held virtually from August 27—30. Harmony is presenting a total of four posters in the Virtual SLEEP 2020 Exhibit Hall, three of which are post-hoc analyses of the efficacy and safety data from the clinical development…

    PLYMOUTH MEETING, Pa. and CHICAGO, Aug. 28, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, announced today results from post-hoc analyses of the efficacy and safety data for WAKIX® (pitolisant) presented at the 34th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2020." The meeting is being held virtually from August 27—30. Harmony is presenting a total of four posters in the Virtual SLEEP 2020 Exhibit Hall, three of which are post-hoc analyses of the efficacy and safety data from the clinical development program for WAKIX.

    "We are pleased to participate in this year's SLEEP 2020 virtual meeting and share clinically relevant data supporting WAKIX with healthcare professionals and the sleep medicine community," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D.  "The results presented from post-hoc analyses of the clinical data help further characterize the efficacy and safety profile of WAKIX to assist healthcare professionals in making treatment decisions for their patients living with narcolepsy."

    Time-to-Response in Excessive Daytime Sleepiness and Cataplexy During Treatment with Pitolisant in Patients with Narcolepsy

    This poster presents results from a post-hoc analysis that examined the time-to-response to pitolisant on improvement in both excessive daytime sleepiness (EDS) and reduction in cataplexy based on pooled data from the HARMONY 1 and HARMONY CTP randomized, controlled clinical trials. Pitolisant was titrated to a maximum dose of 35.6 mg/day and the change from baseline in mean Epworth Sleepiness Scale (ESS) scores (in both studies) and mean weekly rate of cataplexy (WRC; in HARMONY CTP) was compared for pitolisant versus placebo.

    In the HARMONY 1 (pitolisant, n=31; placebo, n=30) and HARMONY CTP (pitolisant, n=54; placebo, n=51) trials, the ESS score improvement was significantly greater with pitolisant versus placebo beginning at Week 2 (LS mean difference, -2.8; p=0.015) and Week 3 (LS mean difference, -2.0; p=0.005), respectively. In HARMONY CTP, the LS mean WRC with pitolisant was 11.7 at baseline, 4.6 at end-of treatment, and 5.1 after a one-week, placebo-washout period. Improvement in the WRC was significantly greater with pitolisant versus placebo beginning at Week 2 (LS mean difference, -5.3; p=0.004) and continued through end-of-treatment (LS mean difference, -6.2; p<0.001 ); there was no evidence of rebound cataplexy after placebo-washout (LS mean difference, -4.9; p=0.027).

    Efficacy of Pitolisant in Patients with High Burden of Narcolepsy Symptoms

    This post-hoc analysis, based on pooled data from the HARMONY 1 and HARMONY CTP clinical trials, examined the efficacy of pitolisant in patients with narcolepsy who had a high symptom burden of EDS and cataplexy at baseline. The analyses included three independent patient subgroups: baseline score of >16 on the ESS (pitolisant, n=54; placebo, n=54), sleep latency of ≤8 minutes on the Maintenance of Wakefulness Test (MWT) (pitolisant, n=59; placebo, n=46), and ≥15 cataplexy attacks per week (pitolisant, n=20; placebo, n=11).  

    Mean change in ESS from baseline was significantly greater for pitolisant (-6.1) compared with placebo (-2.6; p=0.0002). A significantly greater percentage of pitolisant-treated patients were classified as treatment responders: for ESS score reduction ≥3, 68.5 percent in the pitolisant group versus 35.2 percent in the placebo group (p=0.0006); for final ESS score ≤10, 35.2 percent versus 9.3 percent, respectively (p=0.0026). Mean increase in sleep latency on the MWT was significantly greater for pitolisant (7.0 minutes) compared with placebo (3.4 minutes; p=0.0089). Decrease in mean WRC was significantly greater for pitolisant (baseline, 21.8; final, 3.9) compared with placebo (baseline, 20.9; final, 18.2); the rate ratio was 0.35 (95% CI, 0.26–0.47; p<0.001). Adverse events in the analysis populations were consistent with the known safety profile of pitolisant; headache was the most common adverse event in pitolisant-treated patients (10.0–20.4 percent).

    Cardiac Safety Profile of Pitolisant in Patients with Narcolepsy

    The third post-hoc analysis examined the cardiac safety events associated with pitolisant based on a pooled analysis of the HARMONY 1 (8-week) and HARMONY CTP (7-week) clinical trials, and from the 12-month, open-label HARMONY 3 trial. Cardiovascular adverse effects are of concern with narcolepsy medications, as cardiovascular diseases are comorbid conditions in some patients with narcolepsy, and these patients usually require lifelong pharmacotherapy for both narcolepsy and cardiovascular disorders.

    The pooled analysis included 166 patients (pitolisant, n=85; placebo, n=81). Mean change in heart rate from baseline to end-of-treatment was -0.5 beats/min with pitolisant and -0.2 beats/min with placebo (LS mean difference, -0.4; p=0.744). Mean change was also similar for pitolisant versus placebo in systolic (LS mean difference, 0.0; p=0.983) and diastolic (LS mean difference, -0.6; p=0.552) blood pressure, as was mean change in QTc interval (LS mean difference, 0.4; p=0.911). Cardiac adverse events with pitolisant included heart rate increase (n=4), right bundle branch block (n=1), sinus tachycardia (n=1), and palpitations (n=1), and with placebo included blood pressure increase (n=1). In the long-term study, mean change from baseline in QTc interval was 3.1 msec at Month 6 (n=70) and 6.1 msec at Month 12 (n=67); three patients had a post-baseline increase >60 msec but none had an increase in QTc >500 msec.

    About WAKIX® (pitolisant) Tablets

    WAKIX is a first-in-class medication approved by the U.S. Food and Drug Administration in August 2019 for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. It was granted orphan drug designation for the treatment of narcolepsy in 2010. 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 Societe Civile de Recherche (Bioprojet), who has marketed the product in Europe since its approval by the European Medicines Agency in 2016. Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.

    Important Safety Information

    WAKIX is contraindicated in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).  

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Narcolepsy

    Narcolepsy is a rare, chronic, debilitating neurologic disorder of sleep-wake state instability that impacts up to 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, 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, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established in October 2017 by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. For more information on Harmony Biosciences, visit www.harmonybiosciences.com

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

     

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  36. PLYMOUTH MEETING, Pa. and CHICAGO, Aug. 24, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, announced today it will present new data on the safety and efficacy of WAKIX® (pitolisant), including four poster presentations at the upcoming 34th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2020." The meeting is being held virtually from August 27-30. Abstracts of the WAKIX data will also be published in the journal SLEEP as an online supplement.

    PLYMOUTH MEETING, Pa. and CHICAGO, Aug. 24, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ:HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, announced today it will present new data on the safety and efficacy of WAKIX® (pitolisant), including four poster presentations at the upcoming 34th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2020." The meeting is being held virtually from August 27-30. Abstracts of the WAKIX data will also be published in the journal SLEEP as an online supplement.

    The data include a final analysis of the Pitolisant Expanded Access Clinical Evaluation Program (PEACE), two post-hoc analyses on clinically relevant outcomes from the Phase 3 clinical trials, and analyses that further characterize the cardiac safety profile of WAKIX from the clinical development program.  

    "We continue to analyze the extensive database from the clinical development program for WAKIX and are pleased to have the opportunity to highlight new analyses that demonstrate clinically relevant outcomes for WAKIX in the treatment of adult patients with narcolepsy," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "These analyses add to the growing body of scientific evidence supporting WAKIX and validate our commitment to continuing to provide patients and their healthcare providers with important information about this treatment option."

    The data being presented by Harmony include:

    • Safety and Tolerability of Pitolisant in the Treatment of Adult Patients With Narcolepsy: Final Analysis of An Open-Label, Expanded Access Program in the United States (Poster 0766) E Bauer, C Davis, A Patroneva, J Dayno, M Thorpy.
      • Results from a final analysis of the Pitolisant Expanded Access Clinical Evaluation (PEACE) Program, which provided adult patients in the U.S. living with narcolepsy access to treatment with pitolisant before its approval by the U.S. Food and Drug Administration (FDA).
    • Time Course of Improvement in Excessive Daytime Sleepiness and Cataplexy During Treatment with Pitolisant in Patients with Narcolepsy (Poster 0768) A Roy, C Davis, B Vaughn, J Dayno, Y Dauvilliers, J-C Schwartz.
      • Analysis evaluating the time-to-response of pitolisant for both excessive daytime sleepiness and cataplexy from three randomized, placebo-controlled studies in adult patients with narcolepsy.
    • Efficacy of Pitolisant in Patients with High Burden of Narcolepsy Symptoms (Poster 0762) C Davis, U Kallweit, L Krahn, B Vaughn, M Thorpy.
      • Results from a post-hoc analysis evaluating the efficacy of pitolisant in patients who had a high burden of the main narcolepsy symptoms of excessive daytime sleepiness and cataplexy at baseline. Data were pooled from two randomized, placebo-controlled studies of pitolisant in adult patients with narcolepsy.
    • Cardiac Safety Profile of Pitolisant in Patients with Narcolepsy (Poster 0744) W Winter, S Wanaski, A Patroneva, J Dayno.
      • Analysis of the cardiac safety profile of pitolisant in adults with narcolepsy from the clinical development program.

    About WAKIX® (pitolisant) Tablets

    WAKIX is a first-in-class medication approved by the U.S. Food and Drug Administration in August 2019 for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. It was granted orphan drug designation for the treatment of narcolepsy in 2010. 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 Societe Civile de Recherche (Bioprojet), who has marketed the product in Europe since its approval by the European Medicines Agency in 2016. Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.

    Important Safety Information

    WAKIX is contraindicated in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

    WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

    The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).  

    In 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.

    Please see the Full Prescribing Information for WAKIX for more information.

    About Narcolepsy

    Narcolepsy is a rare, chronic, debilitating neurologic disorder of sleep-wake state instability that impacts up to 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, 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, a neuropeptide in the brain that supports sleep-wake state stability. This disorder 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 Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established in October 2017 by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs.

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

     

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  37. PLYMOUTH MEETING, Pa. and CHICAGO, Aug. 21, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony"), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced it has closed its upsized initial public offering of 6,151,162 shares of common stock, including 802,325 shares sold pursuant to the exercise in full of the underwriters' option to purchase additional shares of common stock, at the public offering price of $24.00 per share. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Harmony, are expected to be $147.6 million

    PLYMOUTH MEETING, Pa. and CHICAGO, Aug. 21, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony"), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced it has closed its upsized initial public offering of 6,151,162 shares of common stock, including 802,325 shares sold pursuant to the exercise in full of the underwriters' option to purchase additional shares of common stock, at the public offering price of $24.00 per share. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Harmony, are expected to be $147.6 million. All of the shares were offered by Harmony.

    Harmony shares began trading on the Nasdaq Global Market under the ticker symbol "HRMY" on August 19, 2020. 

    Goldman Sachs & Co. LLC, Jefferies and Piper Sandler acted as lead book-running managers for the offering.

    A registration statement on Form S-1 relating to these securities has been filed with the Securities and Exchange Commission and was declared effective on August 18, 2020. The offering was made only by means of a prospectus. Copies of the final prospectus may be obtained by contacting: Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 866-471-2526, facsimile: 212-902-9316 or by emailing Prospectus-ny@ny.email.gs.com; or Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone: (877) 547–6340 or by email at Prospectus_Department@Jefferies.com; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by email at prospectus@psc.com or by phone: 1-800-747-3924.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established in October 2017 by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. 

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    484-539-9736

    lcaperelli@harmonybiosciences.com

     

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  38. PLYMOUTH MEETING, Pa. and CHICAGO, Aug. 18, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc., a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, announced today the pricing of its upsized initial public offering of 5,348,837 shares of common stock at a price to the public of $24.00 per share. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Harmony, are expected to be $128.4 million. In addition, Harmony has granted the underwriters a 30-day option to purchase up to an additional 802,325 shares of common stock at the initial public offering…

    PLYMOUTH MEETING, Pa. and CHICAGO, Aug. 18, 2020 /PRNewswire/ -- Harmony Biosciences Holdings, Inc., a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, announced today the pricing of its upsized initial public offering of 5,348,837 shares of common stock at a price to the public of $24.00 per share. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Harmony, are expected to be $128.4 million. In addition, Harmony has granted the underwriters a 30-day option to purchase up to an additional 802,325 shares of common stock at the initial public offering price, less underwriting discounts and commissions. All of the shares are being offered by Harmony.

    The shares are expected to begin trading on the Nasdaq Global Market under the ticker symbol "HRMY" on August 19, 2020. The offering is expected to close on August 21, 2020, pending customary closing conditions.

    Goldman Sachs & Co. LLC, Jefferies and Piper Sandler are acting as joint book-running managers for the offering.

    A registration statement on Form S-1 relating to these securities has been filed with the Securities and Exchange Commission and was declared effective on August 18, 2020. The offering will be made only by means of a prospectus. Copies of the final prospectus, when available, may be obtained by contacting: Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 866-471-2526, facsimile: 212-902-9316 or by emailing Prospectus-ny@ny.email.gs.com; or Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone: (877) 547–6340 or by email at Prospectus_Department@Jefferies.com; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by email at prospectus@psc.com or by phone: 1-800-747-3924.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Harmony Biosciences

    Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA and Chicago, IL. The company was established in October 2017 by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare, neurological disorders who have unmet medical needs. 

    Forward-Looking Statements

    This press release includes certain disclosures that contain "forward-looking statements," including, without limitation, statements regarding Harmony's expectations regarding the commencement of trading of its shares on the Nasdaq Global Market, the completion and timing of the closing of offering and the anticipated use of net proceeds from the offering. Forward-looking statements are based on Harmony's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to the satisfaction of customary closing conditions and the completion of the offering, and the risks inherent in commercial sales, product development and clinical trials. These and other risks and uncertainties are described more fully in the section titled "Risk Factors" in the final prospectus related to the offering to be filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of this date, and Harmony undertakes no duty to update such information except as required under applicable law.

    Harmony Biosciences Media Contact:

    Nancy Leone

    215-891-6046

    nleone@harmonybiosciences.com

    Harmony Biosciences Investor Contact:

    Lisa Caperelli

    484-539-9736

    lcaperelli@harmonybiosciences.com

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