Sodium Oxybate

-- Generated $50.0 million in net revenue from sales of LUMRYZ™ -- --2,300 patients on LUMRYZ as of September 30th, including 700 patients that initiated therapy in the quarter -- -- Received FDA approval for LUMRYZ for the treatment of cataplexy or EDS in patients 7 years and older with narcolepsy, granted ODE through October 16, 2031 -- -- LUMRYZ approval upheld by court in suit brought by Jazz regarding FDA’s determination that LUMRYZ is clinically superior to twice-nightly oxybate products -- -- Progressed Phase 3 REVITALYZ™ study evaluating efficacy and safety of LUMRYZ in IH -- -- Management to host a conference call today at 8:30 a.m. ET -- DUBLIN, Ireland, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, today provided a corporate update and announced its financial results for the quarter ended September 30, 2024. “Over a year ago, we set out on our mission to transform the lives of those living with narcolepsy, including expanding the oxybate market through the introduction of LUMRYZ. This quarter, we are proud to see that expansion come to fruition and continue to be encouraged by patient uptake and quarter-over-quarter growth of LUMRYZ. In parallel to our launch in adults with narcolepsy, we are expanding into additional patient populations, starting with the recent FDA approval of LUMRYZ for the treatment of cataplexy or EDS in patients 7 years and older with narcolepsy, significantly alleviating the burden on patients and their caregivers of waking up in the middle of the night to administer treatment,” stated Greg Divis, Chief Executive Officer of Avadel Pharmaceuticals. “We are also pleased with the recent court ruling affirming FDA’s determination that LUMRYZ, dosed once at bedtime, demonstrates clinical superiority to twice-nightly oxybates, which mitigates an important legal and business risk for the company. With our Phase 3 pivotal trial in IH ongoing, our continued execution of the LUMRYZ launch including our expansion of LUMRYZ into pediatric narcolepsy, we are closing in our business objectives of being a leader in the sleep field and fulfilling the promise of LUMRYZ for all stakeholders.” Third Quarter and Recent Company Highlights LUMRYZ Commercial Updates: Generated $50.0 million of net product revenue from sales of LUMRYZ in the third quarter of 2024.As of September 30, there were 2,300 patients on LUMRYZ compared to 1,900 patients on LUMRYZ at June 30. In the third quarter, generated consistent patient demand for LUMRYZ with 700 patients initiating therapy.Observed continuing growth in demand from patients who are new to oxybate – these patients represent the fastest growing patient segment for LUMRYZ. Corporate and Pipeline Updates: On October 30, 2024, the U.S. District Court for the District of Columbia ruled in favor of the U.S. Food and Drug Administration (FDA) in a suit brought by Jazz Pharmaceuticals Inc. under the Administrative Procedure Act regarding the FDA’s approval of LUMRYZ. With this ruling, the approval of LUMRYZ is upheld based on the FDA’s determination that LUMRYZ is clinically superior to Jazz’s twice-nightly oxybate products. On October 16, 2024, the FDA approved LUMRYZ for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years or older with narcolepsy. LUMRYZ was granted Orphan Drug Exclusivity (ODE) for this patient population through October 16, 2031. Patient enrollment is ongoing in the REVITALYZ pivotal study, a Phase 3 double-blind, placebo-controlled, randomized withdrawal, multicenter study designed to evaluate the efficacy and safety of LUMRYZ in idiopathic hypersomnia (IH).During the quarter ended September 30, 2024, announced publication of results of: A post-hoc analysis showing consistent efficacy in participants currently taking alerting agents, highlighting the benefit of LUMRYZ to augment response, as well as the 37% who responded positively based upon LUMRYZ monotherapy.Data from the RESTORE open-label study, based upon the largest cohort of switch patients, in which 94% preferred the once-nightly dosing regimen and 93% who would recommend LUMRYZ to a friend or family member with narcolepsy.A post-hoc analysis demonstrating weight loss associated with LUMRYZ compared to placebo, and shifting from obese category into overweight and overweight into normal BMI categories. Overview of Third Quarter Financial Results Recognized $50.0 million in net product revenue for the third quarter 2024 compared to $7.0 million in the same period in 2023. Net product revenue consists of LUMRYZ product sales, which was launched in the U.S. on June 5, 2023. Gross profit for the third quarter 2024 was $43.9 million compared to $6.9 million in the same period in 2023. Total operating expenses were $44.2 million in the quarter ended September 30, 2024 compared to $42.0 million in the same period in 2023. The current quarter operating expenses incudes $6.4 million of non-cash charges comprised of $5.4 million of stock based compensation expense and $1.0 million of depreciation and amortization. Selling, general and administrative (SG&A) expenses were $40.4 million in the quarter ended September 30, 2024, compared to $39.2 million for the same period in 2023. Research and development (R&D) expenses were $3.8 million in the quarter ended September 30, 2024, compared to $2.8 million for the same period in 2023. R&D expenses in the current period include clinical study costs related to the Phase 3 pivotal trial in IH. Operating loss was $0.3 million for the quarter ended September 30, 2024 compared to $35.1 million in the same period in 2023. Net loss for the quarter ended September 30, 2024, was $2.6 million, or ($0.03) per diluted share, compared to a net loss of $36.3 million, or ($0.41) per diluted share, for the same period in 2023. Cash, cash equivalents and marketable securities were $65.8 million as of September 30, 2024. Cash used in the quarter ended September 30, 2024 included the payment of a $2.0 million commitment fee due to the decision to not draw the second financing tranche that was available under the royalty financing agreement entered into in March 2023. Conference Call Details: A live audio webcast of the call can be accessed by visiting the investor relations section of the Company’s website, www.avadel.com. A replay of the webcast will be archived on Avadel’s website for 90 days following the event. To access the conference call, investors are invited to dial (800) 579-2543 (U.S. and International) and reference the conference ID AVADEL. About LUMRYZ™ (sodium oxybate) for extended-release oral suspension LUMRYZ is an extended-release sodium oxybate medication approved by the FDA on May 1, 2023, as the first and only once-at-bedtime treatment for cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. On October 16, 2024, LUMRYZ was additionally approved as a once-at-bedtime treatment for cataplexy or EDS in patients 7 years and older with narcolepsy. The FDA approval of LUMRYZ was supported by results from REST-ON, a randomized, double-blind, placebo-controlled, pivotal Phase 3 trial in adults with narcolepsy. LUMRYZ demonstrated statistically significant and clinically meaningful improvements in the three co-primary endpoints: EDS (MWT), clinicians’ overall assessment of patients’ functioning (CGI-I), and cataplexy attacks, for all three evaluated doses when compared to placebo. With its approval in May 2023 and in October 2024, the FDA also granted 7 years of Orphan Drug Exclusivity to LUMRYZ for the treatment of cataplexy or EDS in adults with narcolepsy and pediatric patients 7 years and older with narcolepsy (respectively) due to a finding of clinical superiority of LUMRYZ relative to currently available oxybate treatments. In particular, the FDA found that LUMRYZ makes a major contribution to patient care over currently available, twice-nightly oxybate products by providing a once-nightly dosing regimen that avoids nocturnal arousal to take a second dose. About Avadel Pharmaceuticals plc Avadel Pharmaceuticals plc (Nasdaq: AVDL) is a biopharmaceutical company focused on transforming medicines to transform lives. Our approach includes applying innovative solutions to the development of medications that address the challenges patients face with current treatment options. Avadel’s commercial product, LUMRYZ, was approved by the U.S. Food & Drug Administration (FDA) as the first and only once-at-bedtime oxybate for the treatment of cataplexy or EDS in patients 7 years and older with narcolepsy. For more information, please visit www.avadel.com Avadel intends to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company’s Investor Relations website, in addition to following the Company’s press releases, SEC filings, public conference calls, presentations, and webcast. IMPORTANT SAFETY INFORMATION WARNING: Taking LUMRYZ™ (sodium oxybate) with other central nervous system (CNS) depressants, such as medicines used to make you fall asleep, including opioid analgesics, benzodiazepines, sedating antidepressants, antipsychotics, sedating anti-epileptic medicines, general anesthetics, muscle relaxants, alcohol or street drugs, may cause serious medical problems, including trouble breathing (respiratory depression), low blood pressure (hypotension), changes in alertness (drowsiness), fainting (syncope) and death.The active ingredient of LUMRYZ (sodium oxybate) is a form of gamma hydroxybutyrate (GHB), a controlled substance. Abuse or misuse of illegal GHB alone or with other CNS depressants (drugs that cause changes in alertness or consciousness) have caused serious side effects. These effects include seizures, trouble breathing (respiratory depression), changes in alertness (drowsiness), coma and death. Call your doctor right away if you have any of these serious side effects.Because of these risks, LUMRYZ is available only by prescription and filled through certified pharmacies in the LUMRYZ REMS. You must be enrolled in the LUMRYZ REMS to receive LUMRYZ. Further information is available at www.LUMRYZREMS.com or by calling 1-877-453-1029. INDICATIONSLUMRYZ (sodium oxybate) for extended-release oral suspension is a prescription medicine used to treat the following symptoms in patients 7 years and older with narcolepsy: sudden onset of weak or paralyzed muscles (cataplexy)excessive daytime sleepiness (EDS) Do not take LUMRYZ if you take or your child takes other sleep medicines or sedatives (medicines that cause sleepiness), drink alcohol or have a rare problem called succinic semialdehyde dehydrogenase deficiency. Keep LUMRYZ in a safe place to prevent abuse and misuse. Selling or giving away LUMRYZ may harm others and is against the law. Tell your doctor if you or your child have ever abused or been dependent on alcohol, prescription medicines or street drugs. Anyone who takes LUMRYZ should not do anything that requires them to be fully awake or is dangerous, including driving a car, using heavy machinery or flying an airplane, for at least six (6) hours after taking LUMRYZ. Those activities should not be done until you know how LUMRYZ affects you. Falling asleep quickly, including while standing or while getting up from the bed, has led to falls with injuries that have required some people to be hospitalized. LUMRYZ can cause serious side effects, including the following: Breathing problems, including slower breathing, trouble breathing and/or short periods of not breathing while sleeping (e.g., sleep apnea). People who already have breathing or lung problems have a higher chance of having breathing problems when they take LUMRYZ.Mental health problems, including confusion, seeing or hearing things that are not real (hallucinations), unusual or disturbing thoughts (abnormal thinking), feeling anxious or upset, depression, thoughts of killing yourself or trying to kill yourself, increased tiredness, feelings of guilt or worthlessness and difficulty concentrating. Tell your doctor if you or your child have or had depression or have tried to harm yourself. Call your doctor right away if you or your child have symptoms of mental health problems or a change in weight or appetite.Sleepwalking. Sleepwalking can cause injuries. Call your doctor if you or your child start sleepwalking. Tell your doctor if you or your child are on a salt-restricted diet or have high blood pressure, heart failure or kidney problems. LUMRYZ contains a lot of sodium (salt) and may not be right for you.The most common side effects of LUMRYZ in adults include nausea, dizziness, bedwetting, headache and vomiting. Your side effects may increase when you take higher doses of LUMRYZ. The most common side effects in children include nausea, bedwetting, vomiting, headache, decreased weight, decreased appetite, dizziness, and sleepwalking. LUMRYZ can cause physical dependence and craving for the medicine when it is not taken as directed. These are not all the possible side effects of LUMRYZ. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch, or call 1-800-FDA-1088. Please see full Prescribing Information, including BOXED Warning. Cautionary Disclosure Regarding Forward-Looking Statements This press release includes “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. These forward-looking statements relate to our future expectations, beliefs, plans, strategies, objectives, results, conditions, financial performance, prospects or other events. Such forward-looking statements include, but are not limited to, expectations regarding the potential therapeutic benefit of LUMRYZ; the success of the commercialization of LUMRYZ and expansion into additional patient populations; the anticipated market demand and sales opportunity of LUMRYZ; the potential for the Company to be a leader in the market; the Company’s idiopathic hypersomnia clinical study for LUMRYZ, including enrollment and timing related thereto; the Company’s anticipated financial condition, expenses, uses of capital and other future financial results. In some cases, forward-looking statements can be identified by use of words such as “will,” “may,” “could,” “believe,” “expect,” “look forward,” “on track,” “guidance,” “anticipate,” “estimate,” “project,” “next steps” and similar expressions and the negatives thereof (if applicable). The Company’s forward-looking statements are based on estimates and assumptions that are made within the bounds of our knowledge of our business and operations and that we consider reasonable. However, the Company’s business and operations are subject to significant risks, and, as a result, there can be no assurance that actual results and the results of the company’s business and operations will not differ materially from the results contemplated in such forward-looking statements. Factors that could cause actual results to differ from expectations in the Company’s forward-looking statements include the risks and uncertainties described in the “Risk Factors” section of Part I, Item 1A of the Company’s most recent Annual Report on Form 10-K and subsequent filings with the Securities and Exchange Commission. Forward-looking statements speak only as of the date they are made and are not guarantees of future performance. Accordingly, you should not place undue reliance on forward-looking statements. The Company does not undertake any obligation to publicly update or revise our forward-looking statements, except as required by law. Investor Contact:Courtney MogerleyPrecision AQCourtney.Mogerley@precisionAQ.com (212) 698-8687 Media Contact:Lesley StanleyReal Chemistrylestanley@realchemistry.com (609) 273-3162 AVADEL PHARMACEUTICALS PLCCONDENSED CONSOLIDATED STATEMENTS OF LOSS(In thousands, except per share data)(Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Net product revenue $50,025 $7,014 $118,707 $8,510 Cost of products sold 6,155 117 10,465 153 Gross profit 43,870 6,897 108,242 8,357 Operating expenses: Research and development expenses 3,803 2,849 10,922 10,902 Selling, general and administrative expenses 40,394 39,158 136,422 110,404 Total operating expense 44,197 42,007 147,344 121,306 Operating loss (327) (35,110) (39,102) (112,949)Investment and other income, net 610 903 3,114 1,719 Interest expense (2,820) (1,978) (8,128) (7,532)Loss on extinguishment of debt — — — (13,129)Loss before income taxes (2,537) (36,185) (44,116) (131,891)Income tax provision (benefit) 88 89 (327) (401)Net loss $(2,625) $(36,274) $(43,789) $(131,490) Net loss per share - basic $(0.03) $(0.41) $(0.46) $(1.71)Net loss per share - diluted $(0.03) $(0.41) $(0.46) $(1.71) Weighted average number of shares outstanding - basic 96,300 89,380 94,720 76,931 Weighted average number of shares outstanding - diluted 96,300 89,380 94,720 76,931 AVADEL PHARMACEUTICALS PLCCONDENSED CONSOLIDATED BALANCE SHEETS(In thousands, except per share data) September 30, 2024 December 31, 2023 (Unaudited) ASSETS Current assets: Cash and cash equivalents $28,582 $31,167 Marketable securities 37,225 73,944 Accounts receivable, net 37,102 12,103 Inventories 16,097 10,380 Prepaid expenses and other current assets 8,252 6,608 Total current assets 127,258 134,202 Property and equipment, net 469 585 Operating lease right-of-use assets 1,930 2,591 Goodwill 16,836 16,836 Other non-current assets 11,760 10,484 Total assets $158,253 $164,698 LIABILITIES AND SHAREHOLDERS’ EQUITY Current liabilities: Current portion of operating lease liability $725 $934 Accounts payable 7,917 11,433 Accrued expenses 33,907 24,227 Other current liabilities 234 261 Total current liabilities 42,783 36,855 Long-term operating lease liability 1,216 1,690 Royalty financing obligation 34,437 32,760 Other non-current liabilities 5,154 5,654 Total liabilities 83,590 76,959 Shareholders’ equity: Preferred shares, nominal value of $0.01 per share; 50,000 shares authorized; zero issued and outstanding at September 30, 2024 and 5,194 issued and outstanding at December 31, 2023 — 52 Ordinary shares, nominal value of $0.01 per share; 500,000 shares authorized; 96,338 issued and outstanding at September 30, 2024 and 89,825 issued and outstanding at December 31, 2023 963 898 Additional paid-in capital 886,787 855,452 Accumulated deficit (789,285) (745,496)Accumulated other comprehensive loss (23,802) (23,167)Total shareholders’ equity 74,663 87,739 Total liabilities and shareholders’ equity $158,253 $164,698 AVADEL PHARMACEUTICALS PLCCONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS(In thousands)(Unaudited) Nine Months Ended September 30, 2024 2023 Cash flows from operating activities: Net loss $(43,789) $(131,490)Adjustments to reconcile net loss to net cash used in operating activities: Depreciation and amortization 2,013 1,784 Amortization of debt discount and debt issuance costs — 2,796 Share-based compensation expense 16,261 12,293 Loss on extinguishment of debt — 13,129 Other adjustments (1,052) (349)Net changes in assets and liabilities Accounts receivable (24,999) (6,239)Inventories (5,255) (5,286)Prepaid expenses and other current assets (1,615) (3,203)Accounts payable & other current liabilities (3,516) 3,837 Accrued expenses 9,680 10,621 Other assets and liabilities (2,508) 1,625 Net cash used in operating activities (54,780) (100,482) Cash flows from investing activities: Proceeds from sales of marketable securities 298,829 125,498 Purchases of marketable securities (261,962) (203,519)Net cash provided by (used in) investing activities 36,867 (78,021) Cash flows from financing activities: Proceeds from April 2023 public offering, net of issuance costs — 134,149 Payments for February 2023 Notes — (17,500)Payments for debt issuance costs — (4,357)Proceeds from royalty purchase agreement — 30,000 Proceeds from issuance of shares off the at-the-market offering program 9,250 11,913 Proceeds from stock option exercises and employee share purchase plan 5,840 2,241 Net cash provided by financing activities 15,090 156,446 Effect of foreign currency exchange rate changes on cash and cash equivalents 238 (113) Net change in cash and cash equivalents (2,585) (22,170)Cash and cash equivalents at January 1, 31,167 73,981 Cash and cash equivalents at September 30, $28,582 $51,811

– 14% year-over-year revenue increase from combined key growth drivers: Xywav®, Epidiolex® and Rylaze® – – 2024 total revenue guidance affirmed at $4.0 to $4.1 billion – – Zanidatamab 2L BTC PDUFA date of November 29, 2024 – – Plan to submit sNDA for Zepzelca® in 1L ES-SCLC in 1H25 – DUBLIN, Nov. 6, 2024 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced financial results for the third quarter of 2024 and updated guidance for 2024. "Jazz once again delivered record revenues of more than $1.05 billion and a 14% year-over-year increase in revenue from our key growth drivers combined. We continue to see robust patient demand for Xywav with approximately 400 net patient additions in the third quarter, supported by physician and patient appreciation of a low-sodium treatment option. Strong sleep1 performance coupled with continued Epidiolex performance gives us confidence in maintaining our total revenue guidance of $4.0 to $4.1 billion for 2024," said Bruce Cozadd, chairman and chief executive officer, Jazz Pharmaceuticals. "We're preparing for the anticipated launch of zanidatamab in the fourth quarter in 2L BTC, where there remains a high unmet medical need. We expect to provide the first chemotherapy-free dual HER2-targeted bispecific antibody indicated for BTC as well as an opportunity for HCPs to gain important experience ahead of future indications. In addition, results from the Phase 3 IMforte trial were highly encouraging, and we plan to submit an sNDA for Zepzelca in the first half of 2025 to support expansion into the 1L maintenance setting in ES-SCLC." Key Highlights Key growth drivers grew 14% combined year-over-year. Combination of Zepzelca and atezolizumab demonstrated statistically significant and clinically meaningful improvement in OS and PFS primary endpoints, demonstrating the potential of the regimen to delay disease progression in ES-SCLC and extend survival for patients. Zanidatamab: PDUFA date of November 29; expect 2L BTC commercial launch in 4Q24, following approval. Top-line PFS data from zanidatamab in Phase 3 1L GEA estimated to be 2Q25. Initiated a Phase 2 pan-tumor trial to evaluate HER2-positive solid tumors. 2024 Financial Guidance: Affirming 2024 total revenue guidance of $4.0 to $4.1 billion. Affirming neuroscience revenue guidance of $2.825 to $2.925 billion. Lowering oncology revenue guidance to $1.08 to $1.13 billion. Lowering GAAP R&D expense guidance to $862 to $908 million and non-GAAP R&D expense guidance to $790 to $830 million,2 primarily driven by strategic pipeline prioritization. Raising GAAP EPS guidance range to $6.70 to $8.50 and non-GAAP EPS guidance range to $19.50 to $20.60.2 Business Updates Commercial Updates Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution: Xywav net product sales were $388.5 million in 3Q24, an increase of 17% compared to the same period in 2023. There were approximately 400 net patient adds for a total of approximately 13,625 active Xywav patients exiting 3Q24 comprised of: Approximately 10,075 narcolepsy patients. Approximately 3,550 idiopathic hypersomnia (IH) patients, with 250 net patient adds. As the only low-sodium oxybate and the only therapy approved to treat IH, expect Xywav to remain the oxybate of choice. Presented top-line results from the Phase 4 DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) trial at the Psych Congress 2024, which demonstrated efficacy and safety consistent with narcolepsy and IH Phase 3 data. The prospective trial assesses the effect of Xywav treatment on excessive daytime sleepiness, polysomnography parameters and functional outcomes in adults with narcolepsy or IH. Xyrem® (sodium oxybate) oral solution and high-sodium oxybate authorized generic (AG) royalties: Xyrem net product sales were $58.1 million in 3Q24, a decrease of 54% compared to the same period in 2023. Royalties from high-sodium oxybate AGs were $58.2 million in 3Q24, an increase of $29.2 million compared to the same period in 2023. Epidiolex/ Epidyolex (cannabidiol): Epidiolex/Epidyolex net product sales were $251.6 million in 3Q24, an increase of 18% compared to the same period in 2023. Outside of the U.S., Epidyolex is approved in more than 35 countries. Presented data at the European Epilepsy Congress 2024 demonstrating clinically meaningful reductions in drop seizures in patients with Lennox-Gastaut syndrome and subgroup analyses from the BECOME Caregiver Survey showing most caregivers reported patient improvements in seizure and non-seizure outcomes. Ongoing data generation of the seizure and non-seizure benefits of Epidiolex, including from the EpiCom study in tuberous sclerosis complex, to be presented at American Epilepsy Society 2024. Rylaze/ Enrylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn): Rylaze/Enrylaze net product sales were $98.8 million in 3Q24, a decrease of 6% compared to the same period in 2023. There is a temporary impact to Rylaze revenue due to a recent update to pediatric acute lymphoblastic leukemia (ALL) protocols regarding timing of asparaginase administration. The Company does not expect this impact will affect ongoing demand and expects revenue will normalize by early 2025. Zepzelca (lurbinectedin): Zepzelca net product sales were $85.8 million in 3Q24, an increase of 10% compared to the same period in 2023. The Company announced statistically significant and clinically meaningful overall survival (OS) and progression-free survival (PFS) results from the Phase 3 clinical trial, conducted in partnership with Roche, evaluating Zepzelca in combination with Tecentriq ® (atezolizumab) in first-line (1L) extensive-stage (ES) small cell lung cancer (SCLC). Based on positive results from the trial, the Company plans to submit a supplemental New Drug Application (sNDA) for Zepzelca in 1L ES-SCLC in the first half of 2025. Key Pipeline Highlights Zanidatamab: In 2Q24, the U.S. FDA accepted and granted Priority Review of the Biologics License Application for zanidatamab with a target action date of November 29, 2024. If approved, zanidatamab would be the first HER2-targeted treatment specifically approved for biliary tract cancer (BTC) in the U.S. A confirmatory trial in 1L metastatic BTC is ongoing. The pivotal HERIZON-GEA-01 trial, evaluating zanidatamab in 1L gastroesophageal adenocarcinoma (GEA), is expected to read out in 2Q25. Data presented at ESMO 2024 demonstrated sustained clinical antitumor activity in HER2-positive metastatic GEA. Updated results from the Phase 2 trial included a confirmed objective response rate of 84%, duration of response of 18.7 months, median PFS of 15.2 months and a Kaplan-Meier–estimated OS of 59% at 30 months. The Phase 3 EmpowHER-BC-303 trial to evaluate zanidatamab plus chemotherapy or trastuzumab plus chemotherapy in patients with HER2-positive breast cancer whose disease has progressed on previous trastuzumab deruxtecan (T-DXd) treatment is enrolling patients. The Company initiated a Phase 2 DiscovHER-Pan-206 pan-tumor trial in HER2-positive solid tumors. Senior Notes Offering and Concurrent Share Repurchases In the third quarter of 2024, the Company completed a private placement of $1.0 billion aggregate principal amount of 3.125% exchangeable senior notes due 2030, or 2030 Notes. The Company intends to use a portion of the proceeds from the private placement to make a payment on the Term Loan B following the mid-January 2025 expiration of the 1% prepayment premium period in place after the recent repricing. Concurrently with this transaction, the Company repurchased approximately $150.0 million of its ordinary shares. The Company paid for such repurchases with existing cash on hand, and such share repurchases were effected as part of the Company's share repurchase program announced in July 2024. Financial Highlights GAAP net income for 3Q24 was $215.1 million, or $3.42 per diluted share, compared to $146.8 million, or $2.14 per diluted share, for 3Q23. Non-GAAP adjusted net income for 3Q24 was $416.9 million, or $6.61 per diluted share, compared to $340.1 million, or $4.84 per diluted share, for 3Q23. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release. Total Revenues Total revenues increased 9% in 3Q24 compared to the same period in 2023. Total neuroscience revenue, including high-sodium oxybate AG royalty revenue, was $760.9 million in 3Q24, an increase of 8% compared to $704.0 million in 3Q23, primarily due to increased Xywav and Epidiolex/Epidyolex net product sales and increased high-sodium oxybate AG royalty revenue partially offset by decreased Xyrem revenues. Oncology net product sales were $284.8 million in 3Q24, an increase of 9% compared to the same period in 2023, and included higher net product sales from Defitelio/defibrotide which increased 38% to $65.8 million primarily due to timing of orders and Zepzelca which increased 10% to $85.8 million. In 3Q24, Rylaze net product sales were negatively affected by a recent update to pediatric ALL protocols regarding timing of asparaginase administration. Operating Expenses and Effective Tax Rate _________________________ _________________________ Changes in operating expenses in 3Q24 over the prior year period are primarily due to the following: Cost of product sales, on a GAAP basis, increased in 3Q24 compared to the same period in 2023, primarily due to higher product sales, net and higher acquisition accounting inventory fair value step-up expense. Cost of product sales, on a non-GAAP adjusted basis, increased in 3Q24 compared to the same period in 2023, primarily due to higher product sales, net. Selling, general and administrative (SG&A) expenses, on a GAAP and on a non-GAAP adjusted basis, increased in 3Q24 compared to the same period in 2023, primarily due to increased compensation-related expenses driven by higher headcount in support of our key growth drivers. Research and development (R&D) expenses, on a GAAP and on a non-GAAP adjusted basis, decreased in 3Q24 compared to the same period in 2023, primarily due to lower clinical program costs as a result of JZP150 costs incurred in 3Q23 and lower zanidatamab costs. Cash Flow and Balance Sheet As of September 30, 2024, cash, cash equivalents and investments were $2.6 billion, and the outstanding principal balance of the Company's long-term debt was $6.2 billion. In addition, the Company had undrawn borrowing capacity under a revolving credit facility of $500.0 million. In 3Q24, we repaid the $575.0 million aggregate principal amount of the 1.50% exchangeable senior notes due 2024, or 2024 Notes, and completed the private placement of the 2030 Notes. For the nine months ended September 30, 2024, the Company generated $997.3 million of cash from operations reflecting strong business performance and continued financial discipline. 2024 Financial Guidance The Company is updating its full year 2024 financial guidance as follows: GAAP: Non-GAAP: ___________________________ Conference Call Details Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. ET (9:30 p.m. GMT) to provide a business and financial update and discuss its 2024 third quarter results. Audio webcast/conference call: U.S. Dial-In Number: +1 800 715 9871 Ireland Dial-In Number: +353 1800 943 926 Additional global dial-in numbers are available here. Passcode: 5080203 Interested parties may access the live audio webcast via the Investors section of the Jazz Pharmaceuticals website at . To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. A replay of the webcast will be available via the Investors section of the Jazz Pharmaceuticals website at . About Jazz Pharmaceuticals Jazz Pharmaceuticals plc (NASDAQ: JAZZ) is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit for more information. Non-GAAP Financial Measures To supplement Jazz Pharmaceuticals' financial results and guidance presented in accordance with U.S. generally accepted accounting principles (GAAP), the Company uses certain non-GAAP (also referred to as adjusted or non-GAAP adjusted) financial measures in this press release and the accompanying tables. In particular, the Company presents non-GAAP adjusted net income (and the related per share measure) and its line-item components, as well as certain non-GAAP adjusted financial measures derived therefrom, including non-GAAP adjusted gross margin percentage and non-GAAP adjusted effective tax rate. Non-GAAP adjusted net income (and the related per share measure) and its line-item components exclude from GAAP reported net income (and the related per share measure) and its line-item components certain items, as detailed in the reconciliation tables that follow, and in the case of non-GAAP adjusted net income (and the related per share measure), adjust for the income tax effect of the non-GAAP adjustments. In this regard, the components of non-GAAP adjusted net income, including non-GAAP adjusted cost of product sales, SG&A expenses and R&D expenses, are income statement line items prepared on the same basis as, and therefore components of, the overall non-GAAP adjusted net income measure. The Company believes that each of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors and analysts and that each of these non-GAAP financial measures, when considered together with the Company's financial information prepared in accordance with GAAP, can enhance investors' and analysts' ability to meaningfully compare the Company's results from period to period, to its forward-looking guidance, and to identify operating trends in the Company's business. In addition, these non-GAAP financial measures are regularly used by investors and analysts to model and track the Company's financial performance. Jazz Pharmaceuticals' management also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate the Company's business and to make operating decisions, and compensation of executives is based in part on certain of these non-GAAP financial measures. Because these non-GAAP financial measures are important internal measurements for Jazz Pharmaceuticals' management, the Company also believes that these non-GAAP financial measures are useful to investors and analysts since these measures allow for greater transparency with respect to key financial metrics the Company uses in assessing its own operating performance and making operating decisions. 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 the Company's 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 the reconciliation tables that follow. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its non-GAAP financial measures; and the Company has ceased, and may in the future cease, to exclude items that it has historically excluded for purposes of its non-GAAP financial measures. Likewise, the Company may determine to modify the nature of its adjustments to arrive at its non-GAAP financial measures. Because of the non-standardized definitions of non-GAAP financial measures, the non-GAAP financial measures as used by Jazz Pharmaceuticals in this press release and the accompanying tables have limits in their usefulness to investors and may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Caution Concerning Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to: the Company's growth prospects and future financial and operating results, including the Company's 2024 financial guidance and the Company's expectations related thereto and anticipated catalysts; expectations that Xywav will remain the oxybate of choice; expectations of high-sodium oxybate AG royalty revenue in 2024; the ability to generate long-term sustainable growth and value; the Company's advancement of pipeline programs and the timing of development activities, regulatory activities and submissions related thereto, including plans to submit a sNDA for Zepzelca in 1L ES-SCLC in the first half of 2025 and the anticipated launch of zanidatamab in 2L BTC; planned or anticipated clinical trial events, including with respect to initiations, enrollment and data read-outs, and the anticipated timing thereof, including: top-line PFS data from a Phase 3 trial of zanidatamab in 1L GEA, plans to initiate a Phase 1b trial of JZP441 in type 1 narcolepsy patients; and the Company's development, regulatory and commercialization strategy, including the Company's expectations to executing multiple Epidyolex launches through 2024; the Company's expectations with respect to its products and product candidates and the potential of the Company's products and product candidates and the potential regulatory path related thereto; the Company's capital allocation and corporate development strategy; the potential successful future development, manufacturing, regulatory and commercialization activities; the Company's ability to realize the commercial potential of its products; the Company's net product sales and goals for net product sales from new and acquired products; the Company's views and expectations relating to its patent portfolio, including with respect to expected patent protection, as well as expectations with respect to exclusivity; the Company's clinical trials confirming clinical benefit or enabling regulatory submissions; planned or anticipated regulatory submissions and filings, and the anticipated timing thereof; potential regulatory approvals; and other statements that are not historical facts. These forward-looking statements are based on the Company's current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward- looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with: maintaining or increasing sales of, and revenue from, Xywav, Rylaze and Epidiolex/Epidyolex and other marketed products; the introduction of new products into the U.S. market that compete with, or otherwise disrupt the market for the Company's products and product candidates; effectively launching and commercializing the Company's other products and product candidates; the successful completion of development and regulatory activities with respect to the Company's product candidates, obtaining and maintaining adequate coverage and reimbursement for the Company's products; the time-consuming and uncertain regulatory approval process, including the risk that the Company's current and/or planned regulatory submissions may not be submitted, accepted or approved by applicable regulatory authorities in a timely manner or at all; the costly and time-consuming pharmaceutical product development and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to the Company's business operations and financial results; geopolitical events, including the conflict between Russia and Ukraine and related sanctions; macroeconomic conditions, including global financial markets, rising interest rates and inflation and recent and potential banking disruptions; regulatory initiatives and changes in tax laws; market volatility; protecting and enhancing the Company's intellectual property rights and the Company's commercial success being dependent upon the Company obtaining, maintaining and defending intellectual property protection and exclusivity for its products and product candidates; delays or problems in the supply or manufacture of the Company's products and product candidates; complying with applicable U.S. and non-U.S. regulatory requirements, including those governing the research, development, manufacturing and distribution of controlled substances; government investigations, legal proceedings and other actions; identifying and consummating corporate development transactions, financing these transactions and successfully integrating acquired product candidates, products and businesses; the Company's ability to realize the anticipated benefits of its corporate development transactions and its collaborations and license agreements with third parties; the sufficiency of the Company's cash flows and capital resources; the Company's ability to achieve targeted or expected future financial performance and results and the uncertainty of future tax, accounting and other provisions and estimates; the Company's ability to meet its projected long-term goals and objectives, in the time periods that the Company anticipates, or at all, and the inherent uncertainty and significant judgments and assumptions underlying the Company's long-term goals and objectives; fluctuations in the market price and trading volume of the Company's ordinary shares; the timing and availability of alternative investment opportunities; and other risks and uncertainties affecting the Company, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals' Securities and Exchange Commission filings and reports, including the Company's Annual Report on Form 10-K for the year ended December 31, 2023, as supplemented by our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, and future filings and reports by the Company. Other risks and uncertainties of which the Company is not currently aware may also affect the Company's forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. ________________________________________________ Contacts: Investors: Andrea N. Flynn, Ph.D. Vice President, Head, Investor Relations Jazz Pharmaceuticals plc [email protected] Ireland +353 1 634 3211 U.S. +1 650 496 2717 Media: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED