Accessibility StatementSkip Navigation Modeyso, the first treatment option for this ultra-rare and aggressive brain tumor, is commercially available in the United States For U.S. media and investors only DUBLIN, Sept. 9, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that Modeysoâ"¢ (dordaviprone) is recommended by the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) as a category 2A single-agent treatment option for pediat ...

Question-and-Answer SessionSo Phil, why don't we start with the overview when you were here last year and how do you see the year heading for you and the investment thesis at this point, which investors should hear about?Philip JohnsonExecutive VP & CFO Thanks, Mohit. Thanks for the invite to be here. It's always a pleasure. And there's quite a buzz in the hallways this year as well. So great to have a chance to meet with a number of investors. So it's been a really fun last year. I've been really pleased w ...

Jazz Pharmaceuticals (JAZZ) FY 2025 Conference September 03, 2025 09:30 AM ET Company ParticipantsMohit Bansal - MD & Co-Head of Therapeutics ResearchAmal Melhem-Bertrandt - VP - Oncology Therapy AreaPhilip Johnson - EVP & CFOMohit BansalThank you very much for joining us today. My name is Mohit Bansal. I'm one of the biopharma analysts here at Wells Fargo, and this is my first session, not the conference's first session. I'm happy to have the team from Jazz Pharmaceuticals with us. We have Phil Johnson, th ...

Jazz Pharmaceuticals (JAZZ) FY 2025 Conference September 03, 2025 09:30 AM ET Speaker0Thank you very much for joining us today. My name is Mohit Bansal. I'm one of the biopharma analysts here at Wells Fargo, and this is my first session, not the conference's first session. I'm happy to have the team from Jazz Pharmaceuticals with us. We have Phil Johnson, the CFO of the company, and Amal Bartrand with us. She's the Vice President, Oncology Therapy Area Head, and most importantly, Jenny, Clinical Development ...

Jazz Pharmaceuticals (JAZZ) FY 2025 Conference September 03, 2025 09:30 AM ET Speaker0Thank you very much for joining us today. My name is Mohit Bansal. I'm one of the biopharma analysts here at Wells Fargo, and this is my first session, not the conference's first session. I'm happy to have the team from Jazz Pharmaceuticals with us. We have Phil Johnson, the CFO of the company, and Amal Bartrand with us. She's the Vice President, Oncology Therapy Area Head, and most importantly, Jenny, Clinical Development ...

Jazz Pharmaceuticals (JAZZ) Update / Briefing August 27, 2025 04:30 PM ET Company ParticipantsJack Spinks - Executive Director - IRRenee Gala - President, CEO & DirectorJoshua Allen - CTO & Chief Scientific OfficerTimothy Cloughesy - Director - Neuro-OncologyRobert Iannone - EVP, Chief Medical Officer & Global Head - R&DSamantha Pearce - EVP & Chief Commercial OfficerAnnabel Samimy - Managing DirectorBasma Radwan - Vice PresidentPoorna Kannan - Biotech Equity Research AssociateArsalan Kamran - Biotech Equit ...

Company Participation - Jazz Pharmaceuticals will participate in the 2025 Wells Fargo Healthcare Conference with a fireside chat scheduled for September 3, 2025, at 6:30 a.m. PDT / 9:30 p.m. EDT / 2:30 p.m. IST [1] Webcast Information - An audio webcast of the fireside chat will be available on the Investors section of the Jazz Pharmaceuticals website, with a replay archived for 30 days [2] Company Overview - Jazz Pharmaceuticals is a global biopharma company focused on developing life-changing medicines for serious diseases, including sleep disorders, epilepsy, and cancer treatments [3] - The company is headquartered in Dublin, Ireland, and has a diverse portfolio of marketed medicines and a robust pipeline in oncology and neuroscience [3]

Core Insights - JAZZ Pharmaceuticals has signed an agreement with Saniona to acquire worldwide rights to the investigational epilepsy drug SAN2355 [1][8] - SAN2355 is designed to selectively activate Kv7.2/Kv7.3 potassium channels, aiming to avoid safety issues associated with previous therapies like GSK's Potiga [2][3] - The deal allows JAZZ to enhance its neuroscience pipeline, complementing its existing epilepsy treatment, Epidiolex, which is expected to achieve blockbuster status [7][9] Financial Terms of the Deal - JAZZ will pay Saniona an upfront cash payment of $42.5 million, with potential milestone payments up to $192.5 million for development and regulatory achievements, and up to $800 million for commercial milestones [4] - Saniona will also receive royalties on future sales of SAN2355 [4] Market Context - JAZZ's stock has seen a decline of 3% year-to-date, contrasting with a 3% growth in the industry [5] - The acquisition of SAN2355 positions JAZZ for future innovation in the epilepsy market, potentially serving as a successor to Epidiolex once it loses exclusivity [9]

Company Overview - Jazz Pharmaceuticals plc is a global biopharma company focused on developing life-changing medicines for patients with serious diseases, particularly those with limited or no therapeutic options [19][20] - The company has a diverse portfolio that includes therapies for sleep disorders, epilepsy, and a growing range of cancer treatments [19] Product Launch - Jazz Pharmaceuticals will host an investor webcast on August 27, 2025, to discuss the clinical data, patient needs, and commercialization strategy for Modeyso™ (dordaviprone) [1][2] - Modeyso received accelerated approval from the U.S. FDA on August 6, 2025, for treating adult and pediatric patients aged 1 year and older with diffuse midline glioma harboring an H3 K27M mutation [1][4] Clinical Data and Efficacy - Modeyso is the first FDA-approved treatment for this ultra-rare and aggressive brain tumor [1] - The approval was based on an integrated efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma from five open-label clinical studies [4] - Continued approval may depend on the ongoing Phase 3 ACTION trial, which is assessing the safety and clinical benefit of Modeyso in newly diagnosed patients [4][22] Administration and Mechanism - Modeyso is an orally administered small molecule taken once weekly, acting as a protease activator of mitochondrial caseinolytic protease P (ClpP) and inhibiting dopamine D2 receptor [4] - The drug activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, which may restore histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma [4] Safety Information - Serious adverse reactions occurred in 33% of patients receiving Modeyso, with common reactions including fatigue (34%), headache (32%), and vomiting (24%) [13][14] - The drug can cause severe hypersensitivity reactions, with Grade 3 hypersensitivity occurring in 0.3% of patients [6][13] - Modeyso is not approved for use outside the U.S. [5]

Core Insights - Jazz Pharmaceuticals has entered into a global license agreement with Saniona to develop SAN2355, a selective Kv7.2/Kv7.3 activator for epilepsy and other indications, with Jazz obtaining exclusive worldwide rights [1][2] - The collaboration aims to leverage Jazz's expertise in epilepsy and neuroscience to advance SAN2355, which is positioned as a promising treatment option [2][3] Transaction Terms - Saniona will receive an upfront payment of US $42.5 million and is eligible for up to US $192.5 million in development and regulatory milestones, including a US $7.5 million payment upon the initiation of the first Phase 1 clinical study [3] - Additionally, Saniona could receive up to US $800 million in commercial milestone payments based on achieving significant annual net sales thresholds, along with tiered royalties ranging from mid-single digits to low-double digits on net sales of products developed from SAN2355 [3] Product Overview - SAN2355 is a preclinical small molecule activator specifically targeting Kv7.2/Kv7.3 potassium channels, which are validated for seizure suppression, and is designed to avoid adverse events associated with non-selective Kv7-targeting compounds [4] - The selectivity of SAN2355 for Kv7.2/Kv7.3 supports its potential as a best-in-class treatment for epilepsy, aiming to optimize dosing for efficacy [4] Company Background - Jazz Pharmaceuticals is a global biopharma company focused on developing life-changing medicines for serious diseases, with a diverse portfolio that includes therapies for sleep disorders and epilepsy [5] - Saniona is a clinical-stage biopharmaceutical company dedicated to innovative treatments for neurological and psychiatric disorders, with a pipeline that includes SAN2219 for epilepsy and SAN2465 for major depressive disorder [6]