Jazz Pharmaceuticals Announces Second Quarter 2023 Financial Results and Raises 2023 Full Year Financial Guidance

– Strong execution delivered second quarter 2023 total revenues of $957 million –
– Commercial excellence drove continued adoption of low-sodium Xywav^®; net product sales increased 39% year-over-year –
– Continued growth of Epidiolex^®; net product sales increased 15% 2Q23 year-over-year –
– Strong demand for Rylaze^® drove 39% increase in net product sales year-over-year –
– Oncology portfolio expected to reach ~$1 billion in net product sales this year –
– Zanidatamab leads a diversified pipeline with near-term catalysts positioned to deliver as many as four late-stage data readouts by the end of 2024 –

DUBLIN, Aug. 9, 2023 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced financial results for the second quarter of 2023, raised 2023 full year financial guidance and provided business updates.

"Our focused execution delivered strong momentum across all three key growth drivers of our commercial business, with significant demand for Xywav, Epidiolex and Rylaze. We remain confident that our oxybate franchise will reach $2 billion in 2025, underpinned by the durability and growth of low-sodium Xywav. We've achieved another quarter of double-digit, year-over-year growth of Epidiolex, as we unleash its blockbuster potential through in-person engagement, compelling data and ongoing educational efforts. We have achieved impressive diversification with 66% of total revenues stemming from Xywav, Epidiolex and Rylaze, and we expect our Oncology therapeutic area to reach approximately $1 billion in annual revenue this year," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "Our disciplined capital allocation strategy includes investing in our commercial brands to drive top-line growth, investing in our pipeline to drive long-term growth and corporate development, where we remain actively engaged in assessing opportunities and which we continue to believe is an important pillar of growth. Supported by our strong cash flow, we have resumed share repurchases under our existing program. In the second quarter, we completed approximately $100 million of share repurchases of the total $431 million authorized under the program as of March 31, 2023. Our strong operational and financial foundation enables additional investment in growth drivers, and we are well-positioned to execute corporate development and innovate new medicines for patients."

"Since bringing zanidatamab into our pipeline, it has continued to impress, as exemplified by the positive pivotal Phase 2b biliary tract cancers (BTC) data being selected for the 2023 Best of ASCO^® program and published in The Lancet Oncology. We are planning for a potential accelerated approval of zanidatamab in second-line (2L) BTC, and have alignment with FDA on a confirmatory trial in first-line (1L) metastatic BTC. We believe zanidatamab has the potential to address a very large unmet need and raise the standard of care for some of the most difficult-to-treat HER2-expressing cancers. We look forward to the top-line data readout from the zanidatamab Phase 3 gastroesophageal adenocarcinoma (GEA) trial expected in 2024," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "We are on track for multiple near-term pipeline catalysts, including top-line data from a Phase 2 trial of JZP150 in post-traumatic stress disorder (PTSD) and initial proof of concept of JZP441 in healthy volunteers later this year. In the first half of 2024, we expect to have top-line data from a Phase 2b trial of suvecaltamide in essential tremor (ET), as well as top-line progression-free survival (PFS) data from the Zepzelca^® 1L combination trial in extensive-stage (ES) small cell lung cancer (SCLC) as early as the end of 2024. As we advance these promising programs, we remain focused on providing the greatest possible impact for patients and their families."

Key Highlights

• Achieved significant diversification, with nearly 50% of net product sales from Oncology and Epidiolex and more than 50% of oxybate revenues driven by Xywav.
• Xywav continues to be the oxybate of choice and only FDA-approved treatment for idiopathic hypersomnia (IH), achieving blockbuster status and annualizing at well over $1 billion.
• Epidiolex/Epidyolex^® global launch continues to gain momentum further positioning it to achieve blockbuster status.
• Rylaze demand grew for the fourth consecutive quarter with a 39% increase in net product sales to $102 million in 2Q23 compared to 2Q22.
• Raised full year 2023 financial guidance:
  • Raised total and Neuroscience revenue guidance at the mid-points.
  • Raised GAAP earnings per diluted share (EPS) to $6.60-$8.15.
  • Raised non-GAAP adjusted EPS by $1.20 at the mid-point to $18.15-$19.00.
• Zanidatamab Phase 2b HERIZON-BTC-01 results were featured as an oral presentation at ASCO 2023 and concurrently published in The Lancet Oncology.

Business Updates

Key Commercial Products

Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution:

• Xywav net product sales increased 39% to $326.6 million in 2Q23 compared to the same period in 2022.
• There were approximately 11,500 active Xywav patients exiting 2Q23.
• The Company presented robust data sets at two medical meetings in 2Q23, emphasizing leadership in neuroscience and commitment to advancing the understanding of sleep disorders.
  • Six presentations at American Academy of Neurology Annual Meeting, including a study that explored the increased risk of hypertension onset among patients with narcolepsy newly treated with Xyrem^®. The study detected increased risk of new-onset hypertension, within 180 days, among normotensive patients with narcolepsy initiating treatment with high-sodium oxybate, even among patients without a history of cardiovascular disease.
  • Seven presentations at SLEEP 2023, including real-world TENOR study of adults with narcolepsy that demonstrated most patients preferred Xywav over Xyrem due to its lower sodium content.

Xywav for Narcolepsy:

• There were approximately 9,300 narcolepsy patients taking Xywav exiting 2Q23.
• The benefits of reducing sodium intake continue to resonate with patients and prescribers.
• FDA continues to recognize seven years of Orphan Drug Exclusivity (ODE), through July 2027, for Xywav in narcolepsy. FDA published its summary of clinical superiority findings stating that "Xywav is clinically superior to Xyrem by means of greater safety because Xywav provides a greatly reduced chronic sodium burden compared to Xyrem." Further, FDA stated that "the differences in the sodium content of the two products at the recommended doses will be clinically meaningful in reducing cardiovascular morbidity in a substantial proportion of patients for whom the drug is indicated." For clarity, the authorized generics (AGs) of Xyrem contain the exact same drug product as branded Xyrem.
• FDA has also recognized that the difference in sodium content between Xywav and Lumryz is likely to be clinically meaningful in all patients with narcolepsy and that Xywav is safer than high-sodium oxybate products, including Lumryz, in all such patients. Lumryz is a branded, fixed-dose, high-sodium oxybate that has the same sodium content as Xyrem.
• Xywav is the only approved oxybate therapy that does not carry a warning and precaution related to high sodium intake.

Xywav for Idiopathic Hypersomnia (IH):

• There were approximately 2,200 IH patients taking Xywav exiting 2Q23.
• Jazz survey of sleep specialists indicates 70% anticipate increasing their prescribing of Xywav for IH over the next six months, and new prescribers continued to grow in 2Q23.
• Xywav is the first and only treatment approved by FDA to treat the full condition of IH.
• FDA recognized ODE for IH extending regulatory exclusivity to August 2028.

Xyrem (sodium oxybate) oral solution:

• Xyrem net product sales decreased 41% to $159.8 million in 2Q23 compared to the same period in 2022, reflecting the continued adoption of Xywav by patients with narcolepsy and the launch of a high-sodium oxybate AG in January 2023.
• Royalties from high-sodium oxybate AG were $5.5 million in 2Q23. Due to the royalty structures within the AG agreements, we expect the royalties from AGs to be significantly higher in the second half of 2023 relative to the first half.

Oxybate (Xywav and Xyrem):

• Total revenues for the combined oxybate business, including royalties from a high-sodium oxybate AG in 2Q23, decreased 2% to $491.8 million in 2Q23 compared to the same period in 2022.
• Average active Jazz oxybate patients on therapy was approximately 16,200 in 2Q23, a decrease of 5% compared to the same period in 2022.

Epidiolex/Epidyolex (cannabidiol):

• Epidiolex/Epidyolex net product sales increased 15% to $202.2 million in 2Q23 compared to the same period in 2022.
• A pivotal Phase 3 trial of Epidyolex for Dravet syndrome, Lennox-Gastaut syndrome and tuberous sclerosis complex in Japan is enrolling patients.
• Epidiolex/Epidyolex global prescriber base increasing with multiple launches expected outside of the U.S. this year.
• Increased penetration in long-term care setting driven by additional in-person engagement with physicians.
• Additional Epidiolex growth opportunities underscored by BECOME survey's caregiver reported outcomes beyond seizure control, which further differentiates Epidiolex from other anti-seizure medicines, and compelling data for use of Epidiolex in combination with clobazam.
• Epidyolex global launch continues to gain momentum further positioning Epidiolex/Epidyolex to achieve blockbuster status.

Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn):

• Rylaze net product sales increased for the fourth consecutive quarter; 2Q23 net product sales increased 39% to $101.7 million compared to the same period in 2022.
• Continued strong demand for Rylaze reflects the significant unmet patient need for a high-quality, reliable supply of Erwinia asparaginase for patients with acute lymphoblastic leukemia.
• The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted a positive opinion for JZP458 (marketed as Rylaze in the U.S.), recommending the marketing authorization to the European Commission (EC). The Company anticipates EC approval later this year.

Zepzelca (lurbinectedin):

• Zepzelca net product sales increased 3% to $70.3 million in 2Q23 compared to the same period in 2022.
• Zepzelca is the treatment of choice in 2L SCLC setting, with potential to expand into 1L ES SCLC.
• Zepzelca development program highlights:
  • Phase 3 trial in partnership with F. Hoffmann-La Roche Ltd (Roche) to evaluate 1L use of Zepzelca in combination with Tecentriq^® (atezolizumab), compared to Tecentriq alone, as maintenance therapy in patients with ES SCLC after induction chemotherapy is ongoing. The Company expects top-line data to readout at the end of 2024 or early 2025.
  • The Company's partner, PharmaMar, is conducting the Phase 3 confirmatory trial, LAGOON, in 2L SCLC. If positive, this trial could confirm the benefit of Zepzelca in the treatment of SCLC when patients progress following 1L treatment with a platinum-based regimen.
  • The Company has elected to close the EMERGE-201 Phase 2 basket trial evaluating Zepzelca as monotherapy in select relapsed/refractory solid tumors based on limited response in three solid tumor cohorts. The Company is analyzing findings from that trial and continuing to explore additional tumor types that may benefit from treatment with Zepzelca.

Key Pipeline Highlights

Zanidatamab:

• Zanidatamab is a bispecific antibody with a unique design that results in multiple mechanisms of action and potent effector function leading to encouraging antitumor activity in patients.
• The Company believes zanidatamab has the potential to address a very large unmet need in HER2-positive cancers, with initial focus in BTC and GEA and potential to transform the current standard of care in multiple HER2-positive cancers.
• Positive top-line data from the pivotal HERIZON-BTC-01 clinical trial has the potential to support regulatory submissions for zanidatamab as a monotherapy in patients with previously treated HER2-amplifed and expressing BTC.
• The Company is planning for a potential accelerated approval of zanidatamab in 2L BTC, and has alignment with FDA on a confirmatory trial in 1L metastatic BTC, where there remains unmet patient need.
• Data from the HERIZON-BTC-01 clinical trial were featured as an oral presentation at the 2023 ASCO Annual Meeting and concurrently published in The Lancet Oncology. Results demonstrated meaningful clinical benefit including antitumor activity, confirmed objective response rate (cORR) of 41.3%, median duration of response (DOR) of 12.9 months, and median PFS of 5.5 months (median study follow-up time of 12.4 months).
• The pivotal trial, HERIZON-GEA-01, evaluating zanidatamab in 1L GEA is ongoing and top-line data are expected in 2024.

JZP150:

• JZP150, a selective fatty acid amide hydrolase, or FAAH, inhibitor, is in clinical development for the potential treatment of PTSD.
• Patient enrollment is ongoing in a Phase 2 trial and top-line data readout is anticipated in late 2023.
• The primary endpoint of the Phase 2 trial is clinician-administered PTSD Scale (CAPS-5) total symptom severity score change from baseline to week 12 to establish proof of concept of JZP150 in patients diagnosed with Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) PTSD.
• The Company received Fast Track Designation for JZP150 development in PTSD from FDA, underscoring the significant unmet medical needs of patients.

Suvecaltamide (JZP385):

• Suvecaltamide, a highly selective and state dependent modulator of T-type calcium channels, is in clinical development for the treatment of ET and Parkinson's disease tremor.
• Patient enrollment is ongoing in the Phase 2b ET trial and top-line data readout is anticipated in 1H24.
• A Phase 2 trial in patients with Parkinson's disease tremor is ongoing.

JZP441:

• JZP441, is a potent, highly selective oral orexin-2 receptor agonist designed to activate orexin signaling with the potential to be applicable in the treatment of narcolepsy, IH and other sleep disorders.
• A Phase 1 development program to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of JZP441 in sleep-deprived healthy volunteers is ongoing.
• The Company expects initial proof of concept in healthy volunteers in 2023.

JZP815:

• A Phase 1 trial evaluating JZP815 in patients with advanced or metastatic solid tumors with MAPK pathway alterations is ongoing.
• The pan-RAF inhibitor program is part of a novel class of next-generation precision oncology therapies that has the potential to benefit cancer patients with high unmet needs in multiple different solid tumors.

JZP898:

• JZP898 is an engineered IFNα cytokine pro-drug that is activated specifically within the tumor microenvironment where it can stimulate IFNα receptors on cancer-fighting immune effector cells.
• In July 2023, JZP898 received Investigational New Drug (IND) application clearance. The Company expects to initiate a Phase 1 clinical trial by the end of the year.

Resumption of Repurchases under Previously Announced $1.5 Billion Share Repurchase Program

The Company resumed repurchases of its ordinary shares on the open market in the second quarter of 2023 as part of the Company's previously authorized and announced share repurchase program. Under the share repurchase program, the Company may repurchase its ordinary shares for up to an aggregate purchase price of $1.5 billion, exclusive of any brokerage commissions. As of June 30, 2023, approximately $336 million remained available and authorized for share repurchases under the program, reflecting the purchase of approximately $100 million shares during the second quarter of 2023. The timing and amount of repurchases under the program will depend on a variety of factors, including the price of the Company's ordinary shares, alternative investment opportunities, restrictions under the Company's credit agreement, corporate and regulatory requirements and market conditions.

Irrevocable Election of Settlement Method of $575 Million for the 1.50% Exchangeable Senior Notes due 2024

Jazz Investments I Limited, a wholly-owned subsidiary of the Company, announced that it provided written notice today to the exchange agent, the trustee and the noteholders of its 1.50% exchangeable senior notes due 2024 (2024 Notes) that it has irrevocably elected to fix the settlement method for exchanges of the 2024 Notes to a combination of cash and ordinary shares of the Company with a specified cash amount per $1,000 principal amount of 2024 Notes of $1,000. As a result, for 2024 Notes exchanged subsequent to such notice, an exchanging noteholder will receive (i) up to $1,000 in cash per $1,000 principal amount of 2024 Notes and (ii) ordinary shares of the Company, together with cash in lieu of any fractional shares, for any exchange consideration in excess of $1,000 per $1,000 principal amount of 2024 Notes.

Financial Highlights

GAAP net income for 2Q23 was $104.4 million, or $1.52 per diluted share, compared to $34.7 million, or $0.55 per diluted share, for 2Q22.

Non-GAAP adjusted net income for 2Q23 was $325.1 million, or $4.51 per diluted share, compared to $305.5 million, or $4.30 per diluted share, for 2Q22.

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

Total Revenues

Total revenues increased 3% in 2Q23 compared to the same period in 2022.

• Total neuroscience revenue, including high-sodium oxybate AG royalty revenue, was $696.9 million in 2Q23 compared to $696.8 million in 2Q22. Neuroscience net product sales in 2Q23 decreased 1% to $691.4 million compared to the same period in 2022 primarily driven by decreased Xyrem revenues, reflecting the continued strong adoption of Xywav by patients with narcolepsy and the launch of a high-sodium oxybate AG in January 2023, offset by increased Xywav and Epidiolex/Epidyolex net product sales. High-sodium oxybate AG royalty revenue relates to royalty revenue received from Hikma Pharmaceuticals plc on net sales of a high-sodium oxybate AG product.
• Oncology net product sales in 2Q23 increased 10% to $252.2 million compared to the same period in 2022 primarily driven by the continued growth in Rylaze product sales, which increased 39% to $101.7 million.

Operating Expenses and Effective Tax Rate

Changes in operating expenses in 2Q23 over the prior year period are primarily due to the following:

• Cost of product sales decreased in 2Q23 compared to the same period in 2022, on a GAAP basis, primarily due to lower acquisition accounting inventory fair value step-up expense, partially offset by changes in product mix. Cost of product sales increased in 2Q23 compared to the same period in 2022, on a non-GAAP adjusted basis, primarily due to changes in product mix.
• Selling, general and administrative (SG&A) expenses decreased in 2Q23 compared to the same period in 2022, on a GAAP basis, primarily due to the loss on disposal of Sunosi and transaction and integration expenses related to the acquisition of GW Pharmaceuticals plc incurred in 2Q22, partially offset by costs related to program terminations. SG&A expenses, on a GAAP and on a non-GAAP adjusted basis, included decreased compensation-related expenses primarily driven by lower headcount, increased litigation costs and increased investment in our priority programs.
• Research and development (R&D) expenses increased in 2Q23 compared to the same period in 2022, on a GAAP and on a non-GAAP adjusted basis, primarily due to the inclusion of costs related to zanidatamab, as well as our other key pipeline programs.

Cash Flow and Balance Sheet
As of June 30, 2023, cash, cash equivalents and investments were $1.4 billion, and the outstanding principal balance of the Company's long-term debt was $5.8 billion. In addition, the Company had undrawn borrowing capacity under a revolving credit facility of $500 million. For the six months ended June 30, 2023, the Company generated $617.5 million of cash from operations, which is an increase of $105.5 million as compared to the same period in 2022, reflecting strong business performance and continued financial discipline.

2023 Financial Guidance
The Company is updating its full year 2023 financial guidance as follows:

GAAP:

Non-GAAP: