Calidi Biotherapeutics Announces Upcoming Presentations at the 2024 ASCO Annual Meeting

On May 1, 2024 Calidi Biotherapeutics Inc. (NYSE American: CLDI) ("Calidi"), a clinical-stage biotechnology company developing a new generation of targeted immunotherapies, reported the acceptance of three abstracts that will be presented in a poster session at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place from May 31-June 4, 2024, in Chicago, Illinois (Press release, Calidi Biotherapeutics, MAY 1, 2024, View Source [SID1234642543]).

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The posters will include an update from a City of Hope-led Phase 1 study of Calidi’s CLD-101 program, focusing on the treatment of recurrent high-grade glioma, now advancing into its fourth cohort of participants. CLD-101 is a cutting-edge therapeutic candidate in Calidi’s NeuroNova program, comprising tumor-tropic neural stem cells (NSCs) that deliver an oncolytic adenovirus – CRAd-S-pk7 – selectively to tumor sites.

Calidi will also present preclinical data highlighting its RTNova delivery platform, a novel systemic enveloped oncolytic virotherapy program designed to target all tumor sites, and its CLD-201 program, which is expected to dose its first patient in a Phase 1 trial in the second half of 2024 subject to Calidi’s ability to raise additional capital.

Details on the posters and corresponding abstracts are shown below.

Poster Title: Phase 1 study of multiple intracerebral doses of a neural stem cell-based oncolytic virotherapy for treatment of recurrent high-grade gliomas
Abstract Number: TPS2102
Session Title: Central Nervous System Tumors
Session Date and Time: June 1, 2024, from 9:00 AM to 12:00 PM CDT

Poster Title: Transforming tumor immune microenvironments with a novel systemic enveloped oncolytic virotherapy targeting all tumor sites
Abstract Number: 2559
Session Title: Developmental Therapeutics – Immunotherapy
Session Date and Time: June 1, 2024, from 9:00 AM to 12:00 PM CDT

Poster Title: Non-clinical evidence supporting the upcoming CLD-201 clinical trial: Cell-based oncolytic virotherapy for multiple solid tumors
Abstract Number: 2553
Session Title: Developmental Therapeutics – Immunotherapy
Session Date and Time: June 1, 2024, from 9:00 AM to 12:00 PM CDT

Copies of the posters will be available on the Publications section of Calidi’s website following presentation at the meeting.

Affini-T Therapeutics to Present Data From Its Preclinical Programs Targeting KRAS G12D and TP53 R175H at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting

On May 1, 2024 Affini-T Therapeutics, Inc., a precision immunotherapy company unlocking the power of T cells against oncogenic driver mutations, reported that data from its preclinical gene edited HLA-A*11:01 KRAS G12D (AFNT-212) and HLA-A*02:01 TP53 R175H T Cell Receptor (TCR) T cell therapy products for the treatment of solid cancers will be presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting held in Baltimore, MD May 7-11 (Press release, Affini-T Therapeutics, MAY 1, 2024, View Source [SID1234642542]).

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"Our mission is to address the significant unmet needs of patients with hard-to-treat solid tumors by advancing precision immunotherapies that target oncogenic driver mutations," said Loïc Vincent, Ph.D., Chief Scientific Officer, Affini-T Therapeutics. "We believe that our non-viral targeted knock-in platform, THRIVETM, will enable us to cost-effectively engineer safe and effective T cell therapy products. We look forward to presenting new data showing that THRIVETM-engineered A11 KRAS G12D-targeting TCR T cells (AFNT-212) and TP53 R175H-targeting TCR T cells demonstrated superior cytotoxicity against tumor cells relative to LVV-engineered cells both in vitro and in vivo, at ASGCT (Free ASGCT Whitepaper) this year."

Affini-T also announced that data from a genomic profiling dataset illustrating the low frequency of HLA loss of heterozygosity in cancer patients will be presented at ASGCT (Free ASGCT Whitepaper).

"TCR based therapies targeting cancer driver mutations recognize the human leukocyte antigen (HLA)/peptide complex on tumor cells," said Dirk Nagorsen, M.D., Chief Medical Officer at Affini-T Therapeutics. "In this context, loss of heterozygosity (LOH) of HLA may be of interest but only in cases when the specific HLA allele that is presenting the targeted peptide is involved. Conversely, historical allele-agnostic HLA-LOH analyses, which assessed the loss of any MHC class I allele, would only be relevant in the setting of HLA-agnostic therapies, e.g., immune checkpoint inhibitors. We look forward to presenting data showing that the allele-specific HLA-LOH frequency is low, which suggests that allele-specific HLA-LOH frequency is unlikely to contribute significantly to clinical data readouts of TCR-based therapies."

Poster presentation details are as follows:

Title: THRIVE TM Non-Viral Targeted Transgene Knock-In Platform Generates Specific and Potent TCR T Cell Products for the Treatment of Solid Cancers
Abstract #836, Session: G2 – Immune Targeting and Approaches with Genetically-Modified Cells and Cell Therapies
Session Date/Time: Wednesday, May 8, 2024, 12:00 PM – 7:00 PM
Presenting Author: Ankit Gupta, Ph.D., Senior Director, Gene Editing, Discovery, Affini-T Therapeutics

Title: Loss of Heterozygosity is Low for Specific HLA Alleles in Cancer Patients with Driver Mutations
Abstract #785, Session: F3 – Cancer – Targeted Gene and Cell Therapy
Session Date/Time: Wednesday, May 8, 2024, 12:00 PM – 7:00 PM
Presenting Author: Christian Roy, Ph.D., Associate Director, Precision and Translational Medicine, Affini-T Therapeutics

IDEAYA Biosciences to Participate in Upcoming May 2024 Investor Relations Event

On May 1, 2024 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported its participation in an upcoming investor relations event (Press release, Ideaya Biosciences, MAY 1, 2024, View Source [SID1234642541]).

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RBC Capital Markets Global Healthcare Conference
Tuesday, May 14th, 2024 at 3:05 PM ET

Fireside chat with Yujiro S. Hata, Chief Executive Officer, hosted by Gregory Renza, Director and Senior Biotechnology Research Analyst
A live audio webcast of the conference event, as permitted by the conference host, will be available at the "Investors/Events" section of the IDEAYA website at View Source and/or through the conference host. A replay of available webcasts will be accessible for 30 days following the live event.

Genprex to Present and Participate at Upcoming May Investor and Industry Conferences

On May 1, 2024 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported the Company’s participation in the following upcoming investor and industry conferences to be held in May 2024 (Press release, Genprex, MAY 1, 2024, View Source [SID1234642540]).

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Event: Sidoti Microcap Conference
Conference Dates: May 8-9, 2024
Presentation Date: Wednesday, May 8, 2024
Presentation Time: 3:15 p.m. ET
Venue: Virtual
Presenter: Ryan Confer, Genprex’s Chief Financial Officer
Presentation link: https://bit.ly/3UlTsgl

Mr. Confer will deliver an overview of the Company’s pioneering gene therapies for cancer and diabetes and will be available for virtual meetings with investors through the conference platform.

An archive of Mr. Confer’s presentation will be available in the Investor Relations section of the Company’s website.

Event: American Society of Gene & Cell Therapy Annual Meeting
Dates: May 7-11, 2024
Location: Baltimore Convention Center
Company Participant: Thomas Gallagher, Senior Vice President of Intellectual Property and Licensing

Mr. Gallagher will be available for in-person meetings with conference attendees.

For those interested in meeting Genprex management during these conferences, please request a meeting through the conference portal or reach out to Investor Relations at [email protected].

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.