Inc. Public Company and Not-For-Profit sessions are 40 minutes inclusive of Q&A. There are no separate breakout sessions. Private Company sessions are 25 minutes inclusive of Q&A. APAC sessions are 25 minutes and will not include Q&A. 43rd Annual J.P. Morgan HEALTHCARE CONFERENCE January 13–16, 2025 | The Westin St. Francis • San Francisco, California A legacy Hambrecht & Quist® event since 1983 MONDAY, JANUARY 13 CONFERENCE AGENDA (as of 12.11.2024, subject to change) | Pacific Time | Grand Ballroom | Colo ...

Company and Industry Overview * **Company**: BioMarin Pharmaceutical Inc. * **Industry**: Biotechnology, specializing in treatments for genetically defined conditions. Key Financial Highlights * **Total Revenues**: $2.1 billion (3Q'24 YTD) * **Non-GAAP Operating Margin**: 27.7% * **Non-GAAP Diluted Earnings Per Share**: $2.60 * **Revenue Growth**: +19% YOY * **Operating Margin Growth**: +7.6 ppts YOY Pipeline and Pipeline Highlights * **VOXZOGO**: * Phase 3 data for 5 indications (achondroplasia, hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome, SHOX deficiency) * Expansion into additional countries, including Europe, Japan, Australia, and Canada * Strong growth potential in the U.S. market * **Palynziq**: * Phase 3 data and U.S. & EU sBLA filings * Expansion of label to include 12-17 year-olds * Potential for $1.25B+ in 2027 operating cash flow * **BMN 390**: * First-in-human study start for PKU with novel PEG * **BMN 351**: * Clinical POC data for Duchenne Muscular Dystrophy * **BMN 349**: * Study start for alpha-1 antitrypsin deficiency * **BMN 370**: * First-in-human study start for von Willebrand disease Business Development and External Innovation * Focus on disciplined deal execution with a focus on Skeletal Conditions, Enzyme Therapies, and First in Genetic Conditions * Strong global commercial infrastructure and world-class research, clinical, and regulatory capabilities * Rich biotech arena with many small companies at R&D stage Financial Outlook * Revenue growth targeting mid-teen CAGR from 2023-2034 * Non-GAAP operating margin targeting 40% in 2027 * Non-GAAP operating cash flow targeting $1.25B+ in 2027 Other Important Points * BioMarin is the global leader in treating genetically defined conditions * Strong execution in 2024, with significant growth in revenues and operating margin * Focus on innovation and growth through a robust pipeline and strategic business development initiatives

Summary of BeiGene's Conference Call Company Overview - **Company**: BeiGene - **Industry**: Oncology and Hematology - **Event**: 4th Annual J.P. Morgan Healthcare Conference 2025 Key Points and Arguments Financial Performance - **Q3 2024 Cash Flow**: Generated $188 million in cash flow from operations, driven by improved operating leverage and working capital [10][11][134] - **Revenue Growth**: Product revenues grew by 67% in Q3 2024 compared to Q3 2023 [11] - **Quarterly Revenue**: Achieved $1 billion in quarterly revenue, with significant contributions from BRUKINSA [133] Product Leadership - **BRUKINSA**: - Currently the 1 BTK inhibitor (BTKi) in the U.S. for new Chronic Lymphocytic Leukemia (CLL) patients [10][11] - Holds the broadest label with approvals in 72 countries, covering multiple indications [34] - Expected to launch a new tablet formulation in 2025 [34] - **Pipeline**: - 13 new molecular entities (NMEs) entered the clinic in 2024 [10] - Focus on hematology leadership and advancing internally developed assets [21][72] Market Position and Strategy - **Market Share**: Positioned to capture significant market share in hematologic diseases, particularly in the $12 billion CLL market [10] - **Redomicile to Switzerland**: Anticipated to enhance global presence and operational efficiency, pending shareholder vote in early 2025 [10][126] - **Clinical Team**: A global clinical team of over 3,600 personnel, enabling independence from traditional CRO models [14] Industry Challenges - **R&D Returns**: The industry faces increasing trial costs, with oncology trial costs per patient rising from approximately $100,000 to $250,000-$300,000 [12] - **Regulatory Delays**: Project Optimus has delayed Phase 2 trials by 6-9 months, increasing patient numbers in Phase 1 trials by 50-100% [12] Future Focus - **2025 Goals**: - Solidify hematology leadership - Advance the pipeline of internally developed assets - Drive superior financial performance [21][72] - **Key Catalysts**: Anticipated data readouts and regulatory submissions for various assets, including BRUKINSA and Sonrotoclax [131][132] Competitive Landscape - **Emerging Therapies**: - BTK CDAC (Chimeric Degradation Activation Compound) is being positioned as a potential best-in-class approach, with a Phase 3 head-to-head trial against pirtobrutinib planned for 2025 [55][58] - Combination therapies with Sonrotoclax are expected to enhance treatment efficacy in CLL [52][61] Risk Factors - **Market Risks**: Potential pricing pressures from governmental regulations and competition from other therapies [12][36] - **Clinical Risks**: The efficacy and safety of drug candidates must be demonstrated to achieve marketing approval [4] Additional Important Information - **Clinical Data**: Some data presented are from early-phase trials and should be interpreted with caution [7][8] - **Forward-Looking Statements**: Actual results may differ materially from projections due to various factors, including regulatory actions and market conditions [4][5] This summary encapsulates the critical insights from BeiGene's conference call, highlighting its financial performance, product leadership, strategic focus, and the challenges faced within the oncology industry.

Industry and Company Overview * **Industry**: Biotechnology and pharmaceuticals, focusing on rare and orphan diseases. * **Company**: BridgeBio Pharma, a clinical-stage biopharmaceutical company developing therapies for rare genetic diseases. Key Recent Achievements * **Clinical Impact**: * Publication of positive clinical trial results for Acoramidis (acoramidis) in Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in the New England Journal of Medicine. * Publication of positive clinical trial results for Infigratinib in Achondroplasia in the New England Journal of Medicine. * Publication of positive clinical trial results for Encaleret in Autosomal Dominant Hypocalcemia Type 1 in the New England Journal of Medicine. * **Regulatory Advancement**: * Infigratinib received Breakthrough Therapy Designation (BTD) for Achondroplasia. * BBP-418 received Rare Pediatric Disease Designation (RPDD) for Limb-Girdle Muscular Dystrophy 2I/R9. * BBP-812 received Regenerative Medicine Advanced Therapy Designation (RMAT) for Canavan Disease. * Approval of Acoramidis (acoramidis) for the treatment of adult patients with ATTR-CM. * **Commercial Momentum**: * Acoramidis (acoramidis) scripts have been written to date. * Positive feedback from payers and healthcare providers regarding Acoramidis (acoramidis) market access. Pipeline Programs * **Attruby (acoramidis)**: A treatment for ATTR-CM, with a global annual market sales of over $6.4 billion. * **Infigratinib**: A treatment for Achondroplasia and Hypochondroplasia, with a market opportunity of over $4 billion. * **BBP-418**: A treatment for Limb-Girdle Muscular Dystrophy 2I/R9, with a market opportunity of over $1 billion. * **Encaleret**: A treatment for Autosomal Dominant Hypocalcemia Type 1, with a market opportunity of over $2 billion. * **BBP-812**: A treatment for Canavan Disease, with a potential to change the disease trajectory for affected patients. * **BridgeBio Oncology Therapeutics**: A separate company focused on oncology therapies. * **GondolaBio**: A separate company focused on rare disease therapies. Financials and Future Outlook * BridgeBio Pharma is well-financed and expects to hit numerous milestones in 2025. * The company's vision for 2030 includes de-risking its pipeline, impacting over 100,000 lives, and achieving a market capitalization of over $8 billion.

Striving to Become a Leader in ADCs DAIICHI SANKYO CO., LTD. Sunao Manabe Executive Chairperson and CEO January 13, 2025 Forward-Looking Statements Management strategies and plans, financial forecasts, future projections and policies, and R&D information that Daiichi Sankyo discloses in this material are all classified as Daiichi Sankyo's future prospects. These forward-looking statements were determined by Daiichi Sankyo based on information obtained as of today with certain assumptions, premises and futur ...

Key Points Industry/Company - **Company**: Biohaven Ltd. - **Industry**: Biotechnology, pharmaceuticals, rare diseases, neuroscience, oncology, immunology, inflammation, and obesity. Core Views and Evidence - **MoDE Platform**: Biohaven's MoDE platform utilizes targeted protein degradation technology to remove disease-causing proteins. This platform has the potential to treat a wide range of diseases, including rare diseases, neurological disorders, oncology, and immunology and inflammation-related conditions. - **Troriluzole**: Troriluzole is a treatment for Spinocerebellar Ataxia (SCA) that has demonstrated significant clinical benefits over a 3-year period in all SCA genotypes. - **Taldefgrobep Alfa**: Taldefgrobep alfa is a treatment for Spinal Muscular Atrophy (SMA) and obesity. It has shown promising results in clinical trials, including significant improvements in motor function and muscle mass in SMA patients. - **BHV-7000**: BHV-7000 is a selective Kv7 activator with potential applications in treating bipolar disorder, major depressive disorder, and epilepsy. - **BHV-2100**: BHV-2100 is a TRPM3 antagonist with potential applications in treating migraine and pain disorders. - **BHV-8000**: BHV-8000 is a brain-penetrant TYK2/JAK1 inhibitor with potential applications in treating Parkinson's disease, anti-amyloid therapy-induced ARIA, Alzheimer's disease, and multiple sclerosis. - **BHV-1400**: BHV-1400 is a Gd-IgA1 degrader with potential applications in treating IgA nephropathy. It has shown rapid, deep, and selective removal of Gd-IgA1 while preserving healthy immune function. - **BHV-1600**: BHV-1600 is a β1AR autoantibody degrader with potential applications in treating peripartum cardiomyopathy (PPCM). It has demonstrated selectivity and deep removal of β1AR autoantibodies while preserving immunity. - **BHV-1300**: BHV-1300 is an IgG degrader with potential applications in treating Graves' disease. It has shown promising results in clinical trials, including selective removal of TSHR-IgG1 autoantibodies. - **BHV-1510**: BHV-1510 is a Trop2 ADC with potential applications in treating advanced or metastatic epithelial tumors. It has demonstrated early clinical activity in phase 1 trials. - **BHV-1530**: BHV-1530 is a FGFR3 ADC with potential applications in treating urothelial cancer. It has shown synergistic activity in vivo with anti-PD-L1 combination. - **BHV-7000**: BHV-7000 is a Kv7.2/7.3 activator with potential applications in treating bipolar disorder, major depressive disorder, and epilepsy. It is nearing completion of pivotal trials with blockbuster potential. Other Important Content - **Market Potential**: Biohaven's degrader platform has a significant market potential, with peak US gross sales potential estimated at $15 billion for degraders and $8 billion for IgG degraders. - **Strategic Collaborations**: Biohaven has entered into strategic collaborations with Merus and GeneQuantum to develop next-generation ADCs and other therapies. - **Financial Updates**: Biohaven has potential royalties from Pfizer's rimegepant and zavegepant sales, with royalty payments expected to be in the low to mid-teens% range. - **Pipeline**: Biohaven's pipeline includes a diverse range of therapies targeting various diseases, with a focus on rare diseases, neurological disorders, oncology, and immunology and inflammation-related conditions.

Ascendis Pharma A/S J.P. Morgan Healthcare Conference San Francisco January 2025 For investor communication only. Not for use in product promotion. Not for further distribution. Cautionary Note on Forward-Looking Statements 1. Approved in the U.S., EU, and other territories, including Norway, Iceland, Liechtenstein, and Great Britain (covering England, Wales, Scotland). 2. Calculated as unaudited preliminary estimate of full year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions re ...

43rd Annual J.P. Morgan Healthcare Conference Christopher Boerner, Ph.D., Board Chair and Chief Executive Officer The non-GAAP information presented herein provides investors with additional useful information but should not be considered in isolation or as a substitute for the related GAAP measures. Moreover, other companies may define non-GAAP measures differently, which limits the usefulness of these measures for comparisons with such other companies. We encourage investors to review our financial statem ...

José (Joe) Almeida 43RD ANNUAL J.P. MORGAN HEALTHCARE CONFERENCE Chair, President & Chief Executive Officer JANUARY 13, 2025 1 SAFE HARBOR STATEMENT This presentation includes forward-looking statements concerning the company's financial results (including the outlook for full-year 2024 and full-year 2025) and business development, regulatory and operational activities (including the anticipated timing and impact of Hurricane Helene recovery efforts, the company's cost containment initiatives and new produc ...

Building a Top-Tier Biotech 43rd Annual J.P. Morgan Healthcare Conference Yvonne Greenstreet, MBChB Chief Executive Officer January 13, 2025 © 2025 Alnylam Pharmaceuticals, Inc. 1 Alnylam Forward Looking Statements This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam's expectations, beliefs, goals, plans or prospect ...