Jazz Pharmaceuticals Announces First Quarter 2024 Financial Results and Affirms 2024 Financial Guidance

On May 1, 2024 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the first quarter of 2024 and affirmed guidance for 2024 (Press release, Jazz Pharmaceuticals, MAY 1, 2024, View Source [SID1234642539]).

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"In the first quarter of 2024, we delivered combined double-digit year-over-year growth from our key growth drivers: Xywav, Epidiolex and Rylaze. We also significantly advanced our zanidatamab program with the completion of the BLA for 2L BTC," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "We believe the robust growth in patients benefitting from Xywav underscores the appreciation physicians and patients have for the long-term health benefits of reducing sodium and expect Xywav to remain the oxybate of choice. We see continued demand for Rylaze as the only non-E. coli asparaginase regimen that provides sustained activity throughout the course of treatment, and we expect continued growth of Epidiolex to be driven by geographic expansion, optimized dosing and data demonstrating its beyond-seizure benefits. Growing and durable revenues from Xywav, Epidiolex and Rylaze, coupled with our pipeline progress, drive our confidence in delivering on our guidance and objectives for 2024."

Key Highlights

Key growth drivers:
Xywav net product sales grew 14% year-over-year.
Epidiolex/Epidyolex net product sales grew 5% year-over-year.
Rylaze/Enrylaze net product sales grew 20% year-over-year.
Zanidatamab:
Completed the zanidatamab BLA submission seeking accelerated approval in 2L BTC.
Updated data with longer follow-up, including overall survival (OS) findings, from the HERIZON-BTC-01 trial will be presented at ASCO (Free ASCO Whitepaper) Annual Meeting 2024.
Plan to initiate Phase 3 EMPOWHER trial in late-line HER2+ breast cancer in 2H24.
Multiple near-term, late-stage pipeline catalysts anticipated:
Suvecaltamide top-line data from Phase 2b trial in ET in late 1H24.
Top-line data from Epidyolex Phase 3 trial in Japan in 2H24.
Top-line PFS data from zanidatamab in Phase 3 1L GEA targeted for late 2024.
Top-line data from Zepzelca 1L SCLC Phase 3 trial at the end of 2024 or early 2025.
Affirmed 2024 total revenue guidance of $4.0 to $4.2 billion.
Business Updates

Key Commercial Products

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

Xywav net product sales increased 14% to $315.3 million in 1Q24 compared to the same period in 2023.
As the only low-sodium oxybate and the only therapy approved to treat idiopathic hypersomnia (IH), expect Xywav to remain the oxybate of choice.
There were approximately 12,950 active Xywav patients exiting 1Q24, with 275 net patient adds in IH.
Data presented at 2024 AAN Annual Meeting demonstrated the real-world impacts of Xywav:
Results from the RHYTHM study demonstrated patients with IH experienced higher odds of comorbid conditions across multiple clinical categories, including cardiovascular conditions.
A review of five clinical studies evaluating the impact of once- and twice-nightly oxybates on sleep quality, sleep architecture and measures of disrupted nighttime sleep in narcolepsy found oxybate was effective in improving these measures regardless of dosing.
Xywav for Narcolepsy:

There were approximately 9,900 narcolepsy patients taking Xywav exiting 1Q24.
Xywav for Idiopathic Hypersomnia (IH):

There were approximately 3,050 IH patients taking Xywav exiting 1Q24.
Xyrem (sodium oxybate) oral solution:

Xyrem net product sales decreased 64% to $64.2 million in 1Q24 compared to the same period in 2023.
High-Sodium Oxybate Authorized Generic (AG) Royalties:

Royalties from high-sodium oxybate AGs were $49.9 million in 1Q24.
The Company expects high-sodium oxybate AG royalty revenue to exceed $200 million in 2024.
Epidiolex/Epidyolex (cannabidiol):

Epidiolex/Epidyolex net product sales increased 5% to $198.7 million in 1Q24 compared to the same period in 2023. Epidiolex/Epidyolex growth was negatively affected by inventory drawdown in 1Q24.
Outside of the U.S., Epidyolex is approved in more than 35 countries with additional launches and reimbursements anticipated through the end of 2024.
Long-term and real-world data of treatment-resistant epilepsy were presented at 2024 ANN Annual Meeting:
Data from a long-term Expanded Access Program study demonstrated Epidiolex was associated with a sustained reduction in treatment-resistant, focal-onset seizures through 144 weeks, with an acceptable safety profile.
Updated interim results of seizure and non-seizure outcomes from the BECOME-TSC survey of caregivers of patients with tuberous sclerosis complex (TSC) reported improvements in seizure frequency and severity and in cognition, language and communication in patients.
Rylaze/Enrylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn):

Rylaze/Enrylaze net product sales increased 20% to $102.8 million in 1Q24 compared to the same period in 2023.
Zepzelca (lurbinectedin):

Zepzelca net product sales increased 12% to $75.1 million in 1Q24 compared to the same period in 2023.
Enrollment in the Phase 3 trial evaluating first-line (1L) use of Zepzelca in combination with Tecentriq (atezolizumab) in small cell lung cancer, in partnership with Roche, was completed in 1Q24; expect top-line progression-free survival (PFS) data readout at the end of 2024 or early 2025.
Key Pipeline Highlights

Zanidatamab:

Completed the zanidatamab biologics license application (BLA) seeking accelerated approval from the U.S. FDA for second-line (2L) biliary tract cancer (BTC). If approved, zanidatamab would be the first HER2-targeted treatment specifically approved for BTC in the U.S.
The Company’s plans to submit a marketing authorization application (MAA) to the European Medicines Agency (EMA) are proceeding.
Updated data with longer follow-up, including OS findings, from the HERIZON-BTC-01 trial will be presented at the ASCO (Free ASCO Whitepaper) Annual Meeting 2024.
A confirmatory trial in 1L metastatic BTC is ongoing.
The pivotal HERIZON-GEA-01 trial, evaluating zanidatamab in 1L gastroesophageal adenocarcinoma (GEA), is ongoing and the Company is targeting top-line PFS data in late 2024.
The Company plans to initiate a Phase 3 trial, EMPOWHER, in the second half of 2024 to evaluate zanidatamab plus chemotherapy or trastuzumab plus chemotherapy in patients with HER2-positive breast cancer whose disease has progressed on previous T-DXd treatment.
Suvecaltamide (JZP385):

Enrollment was completed in the Phase 2b essential tremor (ET) trial in 1Q24; top-line data readout is anticipated late 1H24.
A Phase 2 trial in patients with Parkinson’s disease tremor is ongoing.
Financial Highlights

Three Months Ended

March 31,

(In thousands, except per share amounts)

2024

2023

Total revenues

$ 901,983

$ 892,812

GAAP net income (loss)

$ (14,618)

$ 69,420

Non-GAAP adjusted net income

$ 182,215

$ 285,261

GAAP earnings (loss) per share

$ (0.23)

$ 1.04

Non-GAAP adjusted EPS

$ 2.68

$ 3.95

GAAP net loss for 1Q24 was $(14.6) million, or $(0.23) per diluted share, compared to a GAAP net income of $69.4 million, or $1.04 per diluted share, for 1Q23.

Non-GAAP adjusted net income for 1Q24 was $182.2 million, or $2.68 per diluted share, compared to a Non-GAAP adjusted net income of $285.3 million, or $3.95 per diluted share, for 1Q23.

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

Total Revenues

Three Months Ended

March 31,

(In thousands)

2024

2023

Xywav

$ 315,300

$ 277,761

Xyrem

64,232

178,130

Epidiolex/Epidyolex

198,716

188,909

Sativex

2,735

7,098

Total Neuroscience

580,983

651,898

Rylaze/Enrylaze

102,750

85,927

Zepzelca

75,100

67,181

Defitelio/defibrotide

47,676

39,079

Vyxeos

32,023

36,700

Total Oncology

257,549

228,887

Other

3,570

3,434

Product sales, net

842,102

884,219

High-sodium oxybate AG royalty revenue

49,947

2,096

Other royalty and contract revenues

9,934

6,497

Total revenues

$ 901,983

$ 892,812

Total revenues increased 1% in 1Q24 compared to the same period in 2023, driven by higher Oncology product sales of 13%, primarily due to continued growth in Rylaze/Enrylaze, which increased 20% to $102.8 million in 1Q24 compared to the same period in 2023, partially offset by lower neuroscience revenues. Total neuroscience revenue, including high-sodium oxybate AG royalty revenue, of $630.9 million decreased in 1Q24 compared to the same period in 2023, primarily due to decreased Xyrem revenues, reflecting the adoption of Xywav by existing Xyrem patients, high-sodium oxybate competition and changes to formulary coverage, partially offset by increased royalty revenue received on net sales of high-sodium oxybate AG products and increased Xywav and Epidiolex/Epidyolex net product sales.

Operating Expenses and Effective Tax Rate

Three Months Ended

March 31,

(In thousands, except percentages)

2024

2023

GAAP:

Cost of product sales

$ 95,487

$ 128,644

Gross margin

88.7 %

85.5 %

Selling, general and administrative

$ 351,712

$ 297,917

% of total revenues

39.0 %

33.4 %

Research and development

$ 222,847

$ 189,410

% of total revenues

24.7 %

21.2 %

Acquired in-process research and development

$ 10,000

$ 1,000

Income tax expense (benefit)1

$ 11,669

$ (15,324)

Effective tax rate 1

(728.4) %

(27.8) %

_________________________

1.

The GAAP income tax expense for 1Q24 primarily related to tax shortfalls from share-based compensation. The GAAP income tax benefit for 1Q23 related primarily to taxes arising on pre-tax income and losses across tax jurisdictions and deductions on subsidiary equity.

Three Months Ended

March 31,

(In thousands, except percentages)

2024

2023

Non-GAAP adjusted:

Cost of product sales

$ 64,148

$ 64,728

Gross margin

92.4 %

92.7 %

Selling, general and administrative

$ 311,499

$ 260,515

% of total revenues

34.5 %

29.2 %

Research and development

$ 204,015

$ 173,918

% of total revenues

22.6 %

19.5 %

Acquired in-process research and development

$ 10,000

$ 1,000

Income tax expense

$ 65,796

$ 40,197

Effective tax rate1

26.4 %

12.3 %

_________________________

1.

The non-GAAP effective tax rate increased in 1Q24 compared to the same period in 2023, primarily due to the mix of pre-tax income and losses incurred across tax jurisdictions.

Changes in operating expenses in 1Q24 over the prior year period are primarily due to the following:

Cost of product sales, on a GAAP basis, decreased in 1Q24 compared to the same period in 2023, primarily due to lower acquisition accounting inventory fair value step-up expense. Cost of product sales, on a non-GAAP adjusted basis, in 1Q24 was in line with the same period in 2023.
Selling, general and administrative (SG&A) expenses increased in 1Q24 compared to the same period in 2023, on a GAAP and on a non-GAAP adjusted basis, primarily due to increased compensation-related expenses driven by higher headcount in support of our key growth drivers, investment in our priority programs and litigation costs.
Research and development (R&D) expenses increased in 1Q24 compared to the same period in 2023, on a GAAP and on a non-GAAP adjusted basis, primarily due to higher costs related to zanidatamab, as well as our other key pipeline programs, and an increase in compensation-related expenses driven by higher headcount in support of our development programs.
Acquired in-process research and development (IPR&D) expense in 1Q24, on a GAAP and on a non-GAAP adjusted basis, related to an upfront payment made in connection with our asset purchase and collaboration agreement with Redx Pharma plc.
Cash Flow and Balance Sheet

As of March 31, 2024, cash, cash equivalents and investments were $1.8 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 $500.0 million. For the three months ended March 31, 2024, the Company generated $267.2 million of cash from operations reflecting strong business performance and continued financial discipline.

2024 Financial Guidance

The Company is affirming its full year 2024 financial guidance as follows:

(In millions)

Guidance

Revenues

$4,000 – $4,200

–Neuroscience (includes royalties from high-sodium oxybate AG)

$2,800 – $2,950

–Oncology

$1,120 – $1,220

(In millions, except per share amounts and percentages)

GAAP

Non-GAAP

Gross margin %

89 %

93%1,6

SG&A expenses

$1,346 – $1,426

$1,170 – $1,2302,6

SG&A expenses as % of total revenues

32% – 36%

28% – 31%

R&D expenses

$877 – $935

$800 – $8503,6

R&D expenses as % of total revenues

21% – 23%

19% – 21%

Effective tax rate

(22)% – (3)%

10% – 13%4,6

Net income

$385 – $530

$1,275 – $1,3506

Net income per diluted share5

$5.80 – $7.70

$18.15 – $19.356

Weighted-average ordinary shares used in per share calculations5

71

71

___________________________

1.

Excludes $125-$145 million of amortization of acquisition-related inventory fair value step-up and $17-$19 million of share-based compensation expense.

2.

Excludes $176-$196 million of share-based compensation expense.

3.

Excludes $77-$85 million of share-based compensation expense.

4.

Excludes 32%-16% from the GAAP effective tax rate of (22)%-(3)% relating to the income tax effect of adjustments between GAAP net income and non-GAAP adjusted net income, resulting in a non-GAAP adjusted effective tax rate of 10%-13%.

5.

Diluted EPS calculations for 2024 include an estimated 6.4 million shares related to the assumed conversion of the 2.00% exchangeable senior notes due 2026, or the 2026 Notes, and the associated interest expense, net of tax, add-back to net income of $20 million and $18 million, on a GAAP and on a non-GAAP adjusted basis, respectively, under the "if converted" method.

6.

See "Non-GAAP Financial Measures" below. Reconciliations of non-GAAP adjusted guidance measures are included above and in the table titled "Reconciliation of GAAP to non-GAAP Adjusted 2024 Net Income Guidance" at the end of this press release.

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. IST) to provide a business and financial update and discuss its 2024 first 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: 8991966

Interested parties may access the live audio webcast via the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. 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 www.jazzpharmaceuticals.com.

Jazz Pharmaceuticals to Participate in the BofA Securities 2024 Health Care Conference

On May 1, 2024 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that Company management will participate in the BofA Securities 2024 Global Health Care Conference on Wednesday, May 15, 2024, in Las Vegas. The fireside chat will be held at 10:00 a.m. PT / 1:00 p.m. ET / 6:00 p.m. IST (Press release, Jazz Pharmaceuticals, MAY 1, 2024, View Source [SID1234642538]).

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An audio webcast of the fireside chat will be available via the Investors section of the Jazz Pharmaceuticals website at View Source A replay of the webcast will be archived on the website for 30 days.

Beyond Cancer Selected to Present on the Clinical Development of Ultra-High Concentration Nitric Oxide (UNO) Therapy at the 2024 American Society of Clinical Oncology Annual Meeting

On May 01, 2024 Beyond Cancer, Ltd., a clinical stage biotechnology company developing ultra-high concentration nitric oxide (UNO) as an immunotherapeutic for solid tumors, reported being selected to present on the clinical development program of its Ultra-High Concentration Nitric Oxide (UNO) at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place May 31 – June 4, 2024 at the McCormick Place Convention Center in Chicago, Illinois (Press release, Beyond Air, MAY 1, 2024, View Source [SID1234642535]). In addition, the Company will host a KOL event that will highlight the ongoing clinical development of UNO (additional details below).

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Company Sponsored Event:
Beyond Cancer will host an event on May 31, 2024 featuring Key Opinion Leaders who will discuss the clinical development program for UNO, including new data from the Phase 1 trial. The event will take place at the Hyatt Regency McCormick Place, from 6:00 p.m. – 8:00 p.m. Central Time.

To learn more and register for the company sponsored event please contact Matt Johnson at [email protected].

ASCO Annual Meeting Presentations:

Title: A phase 1, multi-center, safety, feasibility, and preliminary efficacy study evaluating a single dose of UNO101 in relapsed or refractory, unresectable, primary, or metastatic cutaneous and subcutaneous malignancies
Session Date and Time: Saturday, June 1, 2024 at 9:00 a.m. – 12:00 p.m. Central Time
Session Type and Title: Poster Presentation/Developmental Therapeutics – Immunotherapy
Abstract Number: TPS2686
Authors: Frederick M. Dirbas, Amichay Meirovitz, David Greenberg, Edith Dekel, Gavin Choy, Jedidiah M. Monson

Title: Effect of UNO101 on tumor microenvironment in relapsed or refractory, unresectable, primary, or metastatic cutaneous and subcutaneous malignancies
Session Type and Title: Publication Only; New Targets and New Technologies
Abstract Number: e14575
Authors: Amichay Meirovitz, David Greenberg, Mark Pegram, Jedidiah M. Monson

A copy of the ePublication can be accessed on the Science and Technology page of the Company’s website after May 23, 2024 at 5:00 p.m. ET when made available by ASCO (Free ASCO Whitepaper).

About Nitric Oxide

Nitric Oxide (NO) is a potent molecule, naturally synthesized in the human body, proven to play a critical role in a broad array of biological functions. In the airways, NO targets the vascular smooth muscle cells that surround the small resistance arteries in the lungs. Currently, exogenous inhaled NO is used in adult respiratory distress syndrome, post certain cardiac surgeries and persistent pulmonary hypertension of the newborn to treat hypoxemia. Additionally, NO is believed to play a key role in the innate immune system and in vitro studies suggest that NO possesses anti-microbial activity not only against common bacteria, including both gram-positive and gram-negative, but also against other diverse pathogens.

About UNO Therapy for Solid Tumors

Cancer is the second leading cause of death globally, with tumor metastases responsible for approximately 90% of all cancer-related deaths. Current cancer treatment modalities generally include chemotherapy, immunotherapy, radiation, and/or surgery. Ultra-high concentration Nitric Oxide (UNO) therapy is a completely new approach to preventing relapse or metastatic disease. In vitro murine data show that local tumor ablation with UNO stimulates an anti-tumor immune response in solid tumor cancer models. Beyond Cancer, Ltd. believes that UNO has the potential to prevent relapse or metastatic disease with as little as a single 5-minute treatment and with limited toxicity or off-target effects.

OmniAb to Participate in Five Investor Conferences in May

On May 1, 2024 OmniAb, Inc. (NASDAQ: OABI) reported that management will be participating in five investor conferences during the month of May (Press release, OmniAb, MAY 1, 2024, View Source [SID1234642533]).

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RBC Capital Markets Global Healthcare Conference, May 14-15 at the InterContinental Barclay Hotel in New York City. Management will participate in a fireside chat on Wednesday, May 15th at 11:30 a.m. Eastern time and will hold one-on-one meetings with investors. A live and archived webcast of the fireside chat will be available in the Investors section of OmniAb’s website.

H.C. Wainwright 2nd Annual BioConnect Investor Conference at Nasdaq, May 20 in New York City. Management will participate in a fireside chat on Monday, May 20th at 9:30 a.m. Eastern time and will hold one-on-one meetings with investors.

Benchmark 4th Annual Virtual Healthcare House Call 1×1 Investor Conference, May 21-22. Management will participate in a virtual call on Wednesday, May 22nd at 3:00 p.m. Eastern time and will hold one-on-one virtual meetings with investors.

Craig-Hallum 21st Annual Institutional Investor Conference, May 29 at The Depot Renaissance Minneapolis Hotel. Management will be holding one-on-one and small group meetings on Wednesday, May 29th with investors.

2024 Leerink Partners Healthcare Crossroads Conference, May 28-30 at the Omni Barton Creek Resort & Spa in Austin, Texas. Management will participate in a fireside chat on Thursday, May 30th at 8:00 a.m. Central time and will hold one-on-one meetings with investors.

Quarterly report which provides a continuing view of a company’s financial position

On May 1, 2024 Alkermes reported first quarter 2024 results (Press release, Alkermes, MAY 1, 2024, View Source [SID1234642532]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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