Core Insights - The presentation is part of the 44th Annual Healthcare Conference, featuring Jazz's CEO Renée Galá, who emphasizes the company's commitment to advancing its mission and legacy [1][2]. Company Overview - Renée Galá expresses honor in her new role as CEO and aims to build upon the legacy of her predecessor, Bruce Cozadd [2]. - The company is focused on delivering life-changing medicines to patients, enhancing employee experience, and creating shareholder value [2]. Future Strategy - The CEO plans to recap the company's accomplishments for 2025 and discuss future competitive strategies [3].

Jazz Pharmaceuticals FY Conference Summary Company Overview - **Company**: Jazz Pharmaceuticals (NasdaqGS:JAZZ) - **Date of Conference**: January 13, 2026 - **CEO**: Renée Galá Key Industry and Company Insights 2025 Performance Highlights - Jazz Pharmaceuticals had an outstanding year in 2025, achieving record total revenue and significant accomplishments in R&D and commercial activities [4][6] - The company launched Medaso, the first drug approved for H3K27M-mutant DMG, exceeding expectations in its market launch [5] - Epidiolex achieved blockbuster status with $1 billion in sales [5] - The company resolved major litigation issues, including those related to Epidiolex and Xyrem, ensuring no generics are expected before the late 2030s [6] Strategic Focus on Rare Diseases - Jazz is refining its strategy to focus on rare diseases, leveraging its existing capabilities in sleep, epilepsy, and oncology [11][12] - The company aims to deepen its commitment to rare diseases, with plans to expand into new therapeutic areas while enhancing R&D and corporate development [11][12] - The rationale for focusing on rare diseases includes high unmet needs, lower competitive intensity, and favorable regulatory dynamics [12][13] R&D and Product Development - Jazz is confident in the potential of Ziihera (zanidatamab) as a new standard of care for HER2-positive gastroesophageal adenocarcinoma, with unprecedented overall survival data exceeding two years [4][20] - The company plans to submit a supplemental Biologics License Application (sBLA) for zanidatamab in the first half of 2026, with potential for rapid FDA review [20][25] - Upcoming milestones include multiple data readouts across indications, including metastatic breast cancer [21][26] Financial Position and Corporate Development - Jazz ended the third quarter of 2025 with over $2 billion in cash and investments, positioning itself well for future investments and acquisitions [24][25] - The acquisition of Chimerix added Medaso and significant neuro-oncology expertise, contributing to the company's growth strategy [6][23] - The company expects to announce additional corporate development deals in 2026 to optimize future value [29] Market Dynamics and Competitive Landscape - The launch of generics for Xyrem is anticipated, but Jazz believes its differentiation with Xywav (the only low-sodium oxybate) will maintain its market position [37][40] - The company is focused on enhancing profitability while building a strong, fast-growing business in the rare disease space [33] Upcoming Guidance and Expectations - Jazz plans to provide guidance for 2026 during its February earnings call, with expectations for robust growth in non-oxybate revenue [27][39] - The company is optimistic about its ability to navigate the dynamic market environment, particularly with its strong payer contracts and patient support services [41] Additional Important Points - Jazz's strategy emphasizes leveraging its proven capabilities and focusing on areas with significant unmet needs [12][14] - The company is committed to enhancing its customer-centric approach and integrating AI into its operations [17] - Jazz's historical success in rare diseases positions it well to compete for new assets in the market [43] This summary encapsulates the key points from the Jazz Pharmaceuticals FY Conference, highlighting the company's strategic direction, financial performance, and market positioning in the rare disease sector.

January 2026 44th Annual J.P. Morgan Healthcare Conference Redefining Possibilities in Rare Disease Renee Gala, President & CEO Transforming Lives. Redefining Possibilities. Caution Concerning Forward-Looking Statements This presentation contains forward-looking statements and financial targets, including, but not limited to, statements related to: the Company's growth prospects and future financial and operating results, including the ability of the Company's portfolio to drive long-term shareholder value; ...

Core Viewpoint - Needham analyst Ami Fadia has raised the price target for Jazz Pharmaceuticals (JAZZ) to $235 from $210 while maintaining a Buy rating on the shares, indicating positive sentiment towards the company's future performance [1]. Group 1: Study Insights - The company presented the HERIZON-GEA-01 study with additional details on subgroup analysis, showing that overall data favored zani treatment arms across all subgroups [1]. - The study indicated a longer progression-free survival (PFS) for the tislelizumab arm and slightly higher efficacy in TAP under 1% compared to TAP at 1% or higher [1]. Group 2: Expert Opinions - A key opinion leader noted that the benefits demonstrated by zani are "practice-changing," suggesting that zanidatamab plus chemotherapy with tislelizumab is expected to quickly replace the current standard of care [1].

Revenue Guidance - Jazz Pharmaceuticals expects to meet its previously announced total revenue guidance range for the year ended December 31, 2025[4] - The company will provide specific revenue expectations for its products Xywav, Epidiolex, and Modeyso for the year ended December 31, 2025[4] Corporate Events - The corporate overview and financial update will be presented at the J.P. Morgan Healthcare Conference on January 13, 2026[4]

Core Insights - Boehringer Ingelheim and Jazz Pharmaceuticals have announced a strategic clinical collaboration to explore a novel combination therapy for HER2-positive breast cancer patients, initiating a Phase 1b cohort within Boehringer's ongoing Beamion-BCGC1 trial [1][2] Group 1: Collaboration and Objectives - The collaboration aims to improve patient outcomes by combining zongertinib and zanidatamab, potentially offering a new therapeutic option for patients with HER2-positive breast cancer [2][3] - The research collaboration is focused on transforming treatment paradigms in HER2-expressing breast cancer through the investigation of this novel HER2-targeted combination therapy [3] Group 2: Product Information - Zongertinib (HERNEXEOS®) has received FDA accelerated approval in the U.S., conditional approval in China, and marketing authorization in Japan for previously treated patients with HER2-mutant advanced non-small cell lung cancer (NSCLC), demonstrating an objective response rate (ORR) of 71% in pre-treated patients [3][4] - Zanidatamab (ZIIHERA®), a bispecific HER2-directed antibody, has received accelerated approval from the U.S. FDA for the treatment of adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer and is currently in Phase III trials for various cancers [4] Group 3: Future Directions - The companies anticipate that the study will generate critical insights into the safety and efficacy of dual HER2 blockade, informing the development of a potential future combination therapy [5]

Core Viewpoint - Jazz Pharmaceuticals presented groundbreaking data from the Phase III HERIZON-GEA-01 clinical trial for zanidatamab in combination with chemotherapy for first-line HER2-positive GEA patients at a recent symposium [2]. Group 1: Clinical Trial Insights - The HERIZON-GEA-01 trial assesses zanidatamab in combination with chemotherapy, with or without tislelizumab, targeting first-line HER2-positive GEA patients [2][3]. - The presentation of the trial data occurred at the American Society of Clinical Oncology Gastrointestinal Cancer Symposium, highlighting the significance of the findings [2]. Group 2: Leadership and Expertise - Dr. Geoff Ku, an associate attending physician at Memorial Sloan Kettering Cancer Center, participated in the call to discuss the HERIZON data, indicating the involvement of leading experts in the field [3].

Core Insights - The article highlights the background and expertise of Brendan, who has a strong foundation in organic synthesis and experience in both pharmaceutical and biotech sectors [1] Group 1: Background and Experience - Brendan completed a Ph.D. at Stanford University in organic synthesis in 2009 [1] - He worked for Merck, a major pharmaceutical company, from 2009 to 2013 [1] - Brendan has experience in biotech startups, including Theravance and Aspira, before joining Caltech [1] Group 2: Entrepreneurial Ventures - He was the first employee and co-founder of 1200 Pharma, which spun out of Caltech and secured significant investment in the 8 figures [1] - Brendan remains an avid investor, focusing on market trends, particularly in biotechnology stocks [1]

Core Insights - The article highlights the professional background of Brendan, who has extensive experience in the pharmaceutical and biotechnology sectors, including a Ph.D. from Stanford and roles at major companies like Merck and start-ups [1] Group 1: Professional Background - Brendan completed a Ph.D. in organic synthesis at Stanford University in 2009 [1] - He worked for Merck from 2009 to 2013, gaining significant experience in the pharmaceutical industry [1] - Brendan has been involved in biotech start-ups, including Theravance and Aspira, before joining Caltech [1] Group 2: Entrepreneurial Ventures - He was the first employee and co-founder of 1200 Pharma, which spun out of Caltech and secured major investments in the eight-figure range [1] - Brendan remains an active investor, focusing on market trends, particularly in biotechnology stocks [1]

Summary of Jazz Pharmaceuticals Investor Call on Zanidatamab Horizon-GEA-01 Company and Industry - **Company**: Jazz Pharmaceuticals (NasdaqGS:JAZZ) - **Industry**: Oncology, specifically focusing on HER2-positive gastroesophageal adenocarcinoma (GEA) Core Points and Arguments 1. **Clinical Trial Presentation**: Jazz Pharmaceuticals presented data from the phase III Horizon-GEA-01 Clinical Trial at the American Society of Clinical Oncology Gastrointestinal Cancer Symposium, focusing on zanidatamab in combination with chemotherapy for HER2-positive GEA patients [2][4] 2. **Efficacy of Zanidatamab**: The trial demonstrated a 35% reduction in the risk of disease progression or death, translating to over four months of improvement in median progression-free survival (PFS) compared to trastuzumab plus chemotherapy [7][24] 3. **Overall Survival Benefits**: There was a statistically significant 28% reduction in the risk of death for zanidatamab plus tislelizumab plus chemotherapy versus trastuzumab, leading to a more than seven-month improvement in median overall survival (OS) [8][24] 4. **Mechanism of Action**: Zanidatamab is a bispecific antibody targeting two extracellular domains of HER2, which differentiates it from traditional therapies and enhances its efficacy [22][23] 5. **Safety Profile**: The safety profile of zanidatamab was consistent with known profiles of the individual treatments, although there was an increase in grade three or more toxicities in zanidatamab-containing arms [18][19] 6. **Regulatory Plans**: Jazz Pharmaceuticals plans to submit a supplemental biologics license application in the first half of the year and seeks inclusion in NCCN guidelines based on the trial results [21][33] Additional Important Content 1. **Patient Demographics**: The trial included 914 patients, with approximately 50% enrolled from Asia and 80%-85% of tumors being HER2 IHC3+ [12][13] 2. **Adverse Events**: Infusion-related reactions occurred in 25% of patients receiving zanidatamab, compared to 13.2% for trastuzumab, indicating a need for careful monitoring [18] 3. **Market Opportunity**: GEA is the fifth most common cancer globally, with a significant unmet need for effective HER2-targeted therapies, as the last new option was introduced in 2010 [30][31] 4. **Commercial Strategy**: Jazz Pharmaceuticals is positioned to leverage existing infrastructure for a rapid launch of zanidatamab if approved, with a focus on the strong data supporting its efficacy [31][32] 5. **Future Development**: The company is exploring zanidatamab in various HER2-positive solid tumors, indicating a robust pipeline and potential for broader applications [29][28] This summary encapsulates the key points discussed during the investor call, highlighting the promising data for zanidatamab and its potential impact on the treatment landscape for HER2-positive GEA.