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Use in Specific Populations WAKIX is extensively metabolized by the liver. WAKIX is contraindicated in patients with severe hepatic impairment. Dosage adjustment is recommended in patients with moderate hepatic impairment. To report suspected adverse reactions, contact Harmony Biosciences at 1-800-833-7460 or the FDA at 1-800- FDA-1088 or www.fda.gov/medwatch. This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements con ...

PLYMOUTH MEETING, Pa., June 5, 2024 /PRNewswire/ -- Harmony Biosciences (Nasdaq: HRMY) presented data from its Phase 2 signal detection study showing that pitolisant reduced excessive daytime sleepiness (EDS) and fatigue in adults with Myotonic dystrophy Type 1 (DM1). "More than 80 percent of DM1 patients experience EDS and fatigue, which patient-reported outcomes research has shown to be nearly as debilitating as the primary symptoms of DM1, namely myotonia and muscle weakness,"1 said Kumar Budur, M.D., M. ...

Luis Sanay, CFA 445-235-8386 [email protected] Harmony Biosciences Media Contact: PLYMOUTH MEETING, Pa., May 28, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced that Harmony's management team will participate in a fireside chat at the upcoming Goldman Sachs 45th Annual Global Healthcare Conference in Miami Beach, FL on Monday, June 10, 2024, at 9:20 a.m. ET. A webcast of the fireside chat will be available on the investor page of Harmony's website at https://ir.harmon ...

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Investors interested in Medical - Biomedical and Genetics stocks are likely familiar with Harmony Biosciences Holdings, Inc. (HRMY) and Techne (TECH) . But which of these two stocks offers value investors a better bang for their buck right now? We'll need to take a closer look.We have found that the best way to discover great value opportunities is to pair a strong Zacks Rank with a great grade in the Value category of our Style Scores system. The proven Zacks Rank emphasizes companies with positive estimat ...

Financial Performance - Harmony Biosciences reported Q1 2024 net product revenue of $154.6 million, a 30% increase compared to $119.1 million in Q1 2023[8, 53] - The company's Non-GAAP adjusted net income for Q1 2024 was $50.7 million, a 25% increase from $40.7 million in Q1 2023[62] - Harmony Biosciences has cash, cash equivalents, and investments totaling $453.6 million[53] - The company reiterates its 2024 net revenue guidance of $700-$720 million[68] WAKIX Growth and Potential - WAKIX is considered a potential $1 billion+ net revenue opportunity in adult narcolepsy alone[5, 7] - The average number of patients on WAKIX is approximately 6,300 and growing[11, 39] - WAKIX sales have shown durable growth into year five, with a 30% year-over-year increase[9, 52] Pipeline and Development - The company plans to submit an sNDA for Idiopathic Hypersomnia (IH) in the second half of 2024[5, 15] - A PDUFA date is set in 2026 for Next-Gen Formulation 1 (NG1)[5, 45] - Topline data for ZYN002 in the Phase 3 RECONNECT study for Fragile X syndrome (FXS) is expected in mid-2025[5, 43] - Topline data for clemizole hydrochloride (EPX-100) in a pivotal registrational trial for Dravet syndrome (DS) is expected in 2026[6, 27]

Product Development and Approvals - As of March 31, 2024, the average number of patients on WAKIX was approximately 6,300, with over 80% of all insured lives in the U.S. having secured formulary access[120] - WAKIX was approved by the FDA for the treatment of cataplexy in adult patients with narcolepsy in October 2020, contributing to favorable access for patients[120] - The FDA granted orphan drug designation for pitolisant in the treatment of idiopathic hypersomnia in September 2023 and fast track designation in November 2023[107] - The Phase 3 registrational trial, TEMPO study, for Prader-Willi Syndrome was initiated in March 2024, with FDA alignment on the protocol received in October 2023[112] - The company is developing two new formulations of pitolisant, NG1 and NG2, with clinical studies initiated in Q4 2023 and data from NG1 expected to support further development[112] - The FDA granted priority review for the pediatric narcolepsy supplemental NDA, with a target action date set for June 21, 2024[110] - The primary endpoint of the INTUNE study for idiopathic hypersomnia did not meet statistical significance, but favorable numerical trends were observed, leading to a planned supplemental NDA submission in H2 2024[107] Financial Performance - Net product revenue increased by $35.5 million, or 29.8%, for the three months ended March 31, 2024, compared to the same period in 2023, primarily due to a 28.3% increase in units shipped and a 7% price increase[143] - Cost of product sales increased by $6.7 million, or 32.3%, for the three months ended March 31, 2024, with cost of product sales as a percentage of net product revenue at 17.8%[144] - Research and development expenses increased by $8.9 million, or 67.0%, for the three months ended March 31, 2024, driven by increased costs for clinical development and regulatory work associated with pitolisant and ZYN002[146] - Sales and marketing expenses increased by $4.7 million, or 20.6%, for the three months ended March 31, 2024, primarily due to increased patient engagement and marketing activities[147] - General and administrative expenses increased by $3.6 million, or 16.4%, for the three months ended March 31, 2024, mainly due to higher stock compensation and legal fees[148] - Net income for the three months ended March 31, 2024, was $38.3 million, compared to $29.5 million for the same period in 2023[142] Cash and Debt Management - As of March 31, 2024, the company had cash, cash equivalents, and investments of $453.6 million, with outstanding debt of $192.5 million[152] - The effective tax rate for the three months ended March 31, 2024, was 26.0%, compared to 22.0% for the same period in 2023, influenced by prior year tax benefits[151] - The company entered into a five-year senior secured term loan agreement totaling $185.0 million in July 2023[154] - An incremental term loan of $15.0 million was added in September 2023, with identical terms to the original loan[155] - The repayment schedule includes quarterly principal payments starting at $3.8 million, increasing to $5.0 million by December 2025, with a final payment of $115.0 million due in July 2028[156] - As of March 31, 2024, the company had $192.5 million in borrowings outstanding, with interest rates based on a senior secured net leverage ratio[177] Strategic Acquisitions and Agreements - The company completed the acquisition of Zynerba Pharmaceuticals in October 2023, focusing on innovative transdermal cannabidiol therapies for orphan neuropsychiatric disorders[113] - Topline data from the pivotal Phase 3 RECONNECT study for Zynerba's ZYN002 is anticipated in mid-2025, with patent protection until 2038[113] - A share repurchase program was approved in October 2023, allowing for up to $200.0 million in repurchases, with $150.0 million remaining as of March 31, 2024[158] - The company completed a tender offer to acquire Zynerba for $60.0 million, plus contingent value rights potentially worth up to $2.5444 per share[160] - An asset purchase agreement for HBS-102 was made for $3.5 million, with additional milestone payments totaling up to $174.0 million[161] - A sublicense agreement for an orexin-2 receptor agonist was established, involving an upfront fee of $25.5 million and potential milestone payments of up to $367.5 million[162] Cash Flow Activities - Net cash provided by operating activities for Q1 2024 was $31.1 million, down from $42.6 million in Q1 2023[165][166] - Net cash used in investing activities increased to $6.2 million in Q1 2024 from $1.8 million in Q1 2023[168][169]

[Q1 2024 Business and Financial Highlights](index=1&type=section&id=Q1%202024%20Business%20and%20Financial%20Highlights) Harmony Biosciences reported strong Q1 2024 results, with WAKIX® net revenue up 30% to $154.6 million, and expanded its portfolio Q1 2024 Key Financial Metrics | Metric | Q1 2024 | YoY Growth | Source | | :--- | :--- | :--- | :--- | | WAKIX® Net Revenue | $154.6 million | ~30% | WAKIX® (pitolisant) Net Revenue of $154.6 Million for First Quarter 2024; ~30% Growth Year-over-Year | | GAAP Net Income | $38.3 million | 30% | GAAP net income for the quarter ended March 31, 2024, was $38.3 million... compared to GAAP net income of $29.5 million... for the same period in 2023. | | GAAP Diluted EPS | $0.67 | 39.6% | $0.67 per diluted share, compared to... $0.48 per diluted share, for the same period in 2023. | | Non-GAAP Adj. Net Income | $50.7 million | 26.4% | Non-GAAP adjusted net income was $50.7 million... compared to Non-GAAP adjusted net income of $40.1 million... for the same period in 2023. | | Non-GAAP Adj. Diluted EPS | $0.88 | 33.3% | $0.88 per diluted share... compared to... $0.66 per diluted share, for the same period in 2023. | - The company has accelerated its growth strategy by adding a rare epilepsy franchise, now possessing three late-stage orphan/rare CNS franchises, each with potential peak sales opportunities of **$1 billion to $2 billion**[3](index=3&type=chunk)[4](index=4&type=chunk) - Harmony expects its pipeline to deliver at least one new product or indication launch **every year for the next five years**, with revenue potential extending **beyond 2040**[4](index=4&type=chunk) [Key Franchise Updates](index=3&type=section&id=Key%20Franchise%20Updates) Harmony updated its three CNS franchises, noting WAKIX growth, ZYN002 progress, and a new Rare Epilepsy franchise [Sleep/Wake Franchise](index=3&type=section&id=Sleep%2FWake%20Franchise) The Sleep/Wake franchise saw WAKIX® revenue up 30%, advancing its pipeline with an IH sNDA and new assets - WAKIX® net revenue was **$154.6 million** in Q1 2024, a **30% increase YoY**, with the average number of patients growing to approximately **6,300**[5](index=5&type=chunk) - A supplemental New Drug Application (sNDA) for pitolisant in Idiopathic Hypersomnia (IH) is planned for the **second half of 2024**[1](index=1&type=chunk)[5](index=5&type=chunk) - Positive pharmacokinetic data was reported for the next-generation pitolisant formulation (NG1), with a PDUFA date expected in **2026** and potential patent protection out to **2044**[1](index=1&type=chunk)[5](index=5&type=chunk) - Licensed TPM-1116, a highly potent oral orexin-2 receptor agonist, to strengthen the sleep/wake portfolio. An IND is expected to be filed by **mid-2025**[6](index=6&type=chunk) [Neurobehavioral Franchise](index=4&type=section&id=Neurobehavioral%20Franchise) The Neurobehavioral franchise is advancing ZYN002, with FXS Phase 3 trial enrollment completing Q1 2025 - Patient enrollment in the Phase 3 RECONNECT trial for ZYN002 in Fragile X syndrome (FXS) is expected to be completed in **Q1 2025**, with topline data expected **mid-2025**[10](index=10&type=chunk) - Intellectual property protection for ZYN002 in FXS is secured out to **2040**[10](index=10&type=chunk) [Rare Epilepsy Franchise](index=4&type=section&id=Rare%20Epilepsy%20Franchise) Harmony established a new Rare Epilepsy franchise via Epygenix acquisition, advancing EPX-100 for Dravet and LGS - Acquired Epygenix Therapeutics, establishing a new rare epilepsy franchise[1](index=1&type=chunk)[10](index=10&type=chunk) - Lead product EPX-100 (clemizole) is in a pivotal trial for Dravet syndrome (DS) with data expected in **2026**. A Phase 3 trial for Lennox-Gastaut syndrome (LGS) is planned for **H2 2024**[10](index=10&type=chunk) - EPX-100 has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for both DS and LGS[10](index=10&type=chunk) [Q1 2024 Financial Performance](index=4&type=section&id=Q1%202024%20Financial%20Performance) Harmony Biosciences reported Q1 2024 net product revenue of $154.6 million, GAAP net income of $38.3 million [Key Financial Metrics](index=4&type=section&id=Key%20Financial%20Metrics) Q1 2024 net product revenue was $154.6 million, GAAP net income $38.3 million, and cash $453.6 million Q1 2024 vs Q1 2023 Financial Summary | Metric | Q1 2024 | Q1 2023 | | :--- | :--- | :--- | | Net Product Revenue | $154.6 million | $119.1 million | | GAAP Net Income | $38.3 million | $29.5 million | | GAAP Diluted EPS | $0.67 | $0.48 | | Non-GAAP Adj. Net Income | $50.7 million | $40.1 million | | Non-GAAP Adj. Diluted EPS | $0.88 | $0.66 | Q1 2024 Operating Expenses (YoY) | Expense Category | Q1 2024 | Q1 2023 | % Change | | :--- | :--- | :--- | :--- | | Research & Development | $22.2 million | $13.3 million | +67% | | Sales & Marketing | $27.2 million | $22.6 million | +21% | | General & Administrative | $25.7 million | $22.1 million | +16% | | **Total Operating Expenses** | **$75.1 million** | **$57.9 million** | **+30%** | - As of March 31, 2024, cash, cash equivalents, and investments totaled **$453.6 million**, an increase from **$425.6 million** at the end of 2023[12](index=12&type=chunk) [Consolidated Statements of Operations](index=13&type=section&id=Consolidated%20Statements%20of%20Operations) Q1 2024 consolidated operations show net product revenue of $154.6 million, gross profit of $127.1 million Q1 2024 Statement of Operations (in thousands) | Line Item | Q1 2024 | Q1 2023 | | :--- | :--- | :--- | | Net product revenue | $154,615 | $119,126 | | Gross profit | $127,131 | $98,346 | | Total operating expenses | $75,098 | $57,923 | | Operating income | $52,033 | $40,423 | | Net income | $38,334 | $29,485 | | Diluted EPS | $0.67 | $0.48 | [Consolidated Balance Sheets](index=14&type=section&id=Consolidated%20Balance%20Sheets) As of March 31, 2024, total assets were $847.0 million, liabilities $331.6 million, and equity $515.4 million Balance Sheet Summary (in thousands) | Account | March 31, 2024 | Dec 31, 2023 | | :--- | :--- | :--- | | Cash and cash equivalents | $332,981 | $311,660 | | Total current assets | $479,503 | $451,070 | | **Total Assets** | **$846,985** | **$811,448** | | Total current liabilities | $154,253 | $163,781 | | **Total Liabilities** | **$331,591** | **$344,456** | | **Total Stockholders' Equity** | **$515,394** | **$466,992** | [Reconciliation of GAAP to Non-GAAP Financial Results](index=15&type=section&id=Reconciliation%20of%20GAAP%20to%20Non-GAAP%20Financial%20Results) Q1 2024 GAAP net income was $38.3 million, adjusted to non-GAAP net income of $50.7 million Q1 2024 GAAP to Non-GAAP Reconciliation (in thousands) | Line Item | Q1 2024 | Q1 2023 | | :--- | :--- | :--- | | **GAAP net income** | **$38,334** | **$29,485** | | Amortization | $5,961 | $5,961 | | Stock-based compensation | $10,434 | $6,561 | | Other adjustments (net of tax) | ($4,027) | ($1,270) | | **Non-GAAP adjusted net income** | **$50,722** | **$40,738** | | GAAP diluted EPS | $0.67 | $0.48 | | Non-GAAP adjusted diluted EPS | $0.88 | $0.67 | [2024 Outlook and Corporate Updates](index=5&type=section&id=2024%20Outlook%20and%20Corporate%20Updates) Harmony reiterated 2024 net product revenue guidance of $700-720 million and $150 million for share repurchase - The company reiterates its full-year 2024 net product revenue guidance of **$700 million to $720 million**[2](index=2&type=chunk)[13](index=13&type=chunk) - As of March 31, 2024, **$150 million** remains authorized for the company's share repurchase program[14](index=14&type=chunk) [Product and Pipeline Overview](index=6&type=section&id=Product%20and%20Pipeline%20Overview) Harmony's key products and pipeline include WAKIX® for narcolepsy, ZYN002 for FXS/22q, and EPX-100 for epilepsy [WAKIX® (pitolisant) and Target Indications](index=6&type=section&id=WAKIX%C2%AE%20%28pitolisant%29%20and%20Target%20Indications) WAKIX® (pitolisant) is a first-in-class H₃ receptor antagonist for narcolepsy, also studied for IH and PWS - WAKIX is a first-in-class selective histamine 3 (H₃) receptor antagonist/inverse agonist approved for excessive daytime sleepiness or cataplexy in adult narcolepsy patients[19](index=19&type=chunk) - Narcolepsy is a rare neurological disease affecting approximately **170,000 Americans**, primarily characterized by excessive daytime sleepiness (EDS) and cataplexy[32](index=32&type=chunk) - The pipeline includes expanding WAKIX's use to Idiopathic Hypersomnia (IH) and Prader-Willi Syndrome (PWS), both rare disorders involving EDS[33](index=33&type=chunk)[34](index=34&type=chunk) [ZYN002 and Target Indications](index=9&type=section&id=ZYN002%20and%20Target%20Indications) ZYN002 is a synthetic, THC-free cannabidiol gel developed for Fragile X and 22q syndromes - ZYN002 is a synthetic, THC-free cannabidiol in a transdermal gel formulation[35](index=35&type=chunk) - It is being developed for Fragile X syndrome (FXS), which affects up to **80,000 patients** in the U.S., and 22q11.2 deletion syndrome (22q), affecting a similar number[36](index=36&type=chunk)[39](index=39&type=chunk) [EPX-100 (clemizole) and Target Indications](index=10&type=section&id=EPX-100%20%28clemizole%29%20and%20Target%20Indications) EPX-100 (clemizole) is an oral drug targeting serotonin receptors for Dravet and LGS - EPX-100 (clemizole hydrochloride) is an oral drug candidate that targets central 5-hydroxytryptamine (serotonin) receptors[40](index=40&type=chunk) - It is under development for Dravet syndrome (DS), which has an incidence of **~1:15,700**, and Lennox-Gastaut syndrome (LGS), which affects approximately **48,000 patients** in the U.S[41](index=41&type=chunk)[42](index=42&type=chunk) [WAKIX® Important Safety Information](index=6&type=section&id=WAKIX%C2%AE%20Important%20Safety%20Information) WAKIX® safety notes contraindications for hypersensitivity or hepatic impairment, QT prolongation, and common adverse reactions - WAKIX is contraindicated in patients with severe hepatic impairment or known hypersensitivity to pitolisant[21](index=21&type=chunk) - A primary warning is that WAKIX prolongs the QT interval; it should be avoided in patients with known QT prolongation or with other risk factors for torsade de pointes[22](index=22&type=chunk)[23](index=23&type=chunk) - The most common adverse reactions (≥5% and twice placebo) in clinical trials were **insomnia (6%)**, **nausea (6%)**, and **anxiety (5%)**[25](index=25&type=chunk) - WAKIX may reduce the effectiveness of hormonal contraceptives; an alternative non-hormonal method is recommended during and for 21 days after treatment[27](index=27&type=chunk)[28](index=28&type=chunk)

WAKIX® (pitolisant) Net Revenue of $154.6 Million for First Quarter 2024; ~30% Growth Year-over-Year Supplemental New Drug Application for Pitolisant in Idiopathic Hypersomnia Planned for Second Half 2024 On Track Toward Pediatric Exclusivity to Extend WAKIX Exclusivity to September 2030: Pediatric Narcolepsy sNDA PDUFA Date of June 21, 2024; PWS Phase 3 TEMPO Study Initiated in March 2024 Pitolisant Franchise Revenue Potential Extended Beyond 2040 With Next-Generation Formulations; Reports Positive Pharma ...