Pipeline and Approvals - Ultragenyx expects Phase 3 data readout for UX143 for Osteogenesis Imperfecta (OI) in 2025[21, 32] - Ultragenyx anticipates completing Phase 3 enrollment for GTX-102 for Angelman Syndrome (AS) in the second half of 2025[46, 49] - The company has a PDUFA date of August 18, 2025, for UX111 for Sanfilippo syndrome (MPS IIIA), with a potential commercial launch in the second half of 2025[63, 67] - Ultragenyx expects to submit a BLA for DTX401 for Glycogen Storage Disease Type Ia (GSDIa) in mid-2025, with a potential launch in 2026[78, 80] Financial Performance and Projections - Ultragenyx projects total revenue to grow by 14-20% in 2025, reaching $640-670 million[93, 95] - The company anticipates Crysvita revenue to be $460-480 million in 2025, representing a 12-17% increase[95] - Dojolvi revenue is projected to be $90-100 million in 2025, a 2-13% increase[95] - Ultragenyx aims to achieve full-year GAAP profitability in 2027, driven by revenue growth, new product launches, and expense management[2, 96, 99] Clinical Trial Results - Phase 2 data for UX143 in OI showed a 67% reduction in annualized fracture rate (AFR) [33] - Phase 3 data for DTX401 in GSDIa demonstrated a statistically significant 41% reduction in daily cornstarch intake at Week 48 (p < 0.0001)[83] - UX111 for MPS IIIA showed >80% of participants reduced CSF HS by 50% in efficacy set[71]

PRAME Franchise & Market Opportunity - Immatics is developing three clinical product candidates (IMA402, IMA203, IMA203CD8) precisely targeting PRAME, an intracellular protein presented on tumor cell surfaces[10, 12] - PRAME expression is prevalent in over 50 cancers[8], with prevalence ranging from 20% in Hepatocellular Carcinoma and Bladder Carcinoma to 95% in Cutaneous Melanoma, Uterine Carcinoma, and Synovial Sarcoma[9] - The addressable PRAME+/HLA-A*02:01+ patient population in the US & EU5 is estimated to be approximately 230,000 per year[20, 24] IMA203 PRAME Cell Therapy (Wave 1) - In a Phase 1 study, IMA203 demonstrated a confirmed objective response rate (cORR) of 56% (18/32) in heavily pretreated patients with metastatic melanoma[34, 45] - The median duration of response (mDOR) was 121 months, with ongoing responses for up to 25+ years[34] - The median progression-free survival (mPFS) was 61 months, and the median overall survival (mOS) was 159 months in all melanoma patients[34, 51] - The SUPRAME Phase 3 trial is actively enrolling patients with unresectable or metastatic melanoma post-ICI, with a planned launch in Q3 2027[35, 59] - Approximately 9,000 PRAME+/HLA-A*02:01+ patients in the US/EU5 with cutaneous and uveal melanoma are addressable[34] IMA203CD8 PRAME Cell Therapy (GEN2) (Wave 2) - IMA203CD8 is a next-generation PRAME cell therapy leveraging CD8 and CD4 T cells, with enhanced pharmacology[24] - In a Phase 1a dose escalation study, IMA203CD8 achieved a cORR of 41% (14/34) at presumably suboptimal doses, with 84% (32/38) of patients experiencing tumor shrinkage[73, 85] - The median duration of response (mDOR) was 92 months, with 3 confirmed responses ongoing at 1+ year[73, 85] - The addressable PRAME+/HLA-A*02:01+ patient opportunity for IMA203CD8 is approximately 75,000 per year, including indications with both high and medium-level PRAME expression[20, 68] IMA402 PRAME Bispecific (Wave 3) - IMA402 is a next-generation half-life extended bispecific primarily targeting first-line settings in combination with ICI and targeted agents[22] - Early signs of clinical activity have been observed, with a melanoma patient achieving a confirmed partial response ongoing at 3 months at DL7[115] - The addressable PRAME+/HLA-A*02:01+ patient population for IMA402 in 1L solid tumors is approximately 145,000 in the US & EU5[20, 100] Beyond PRAME: IMA401 MAGEA4/8 Bispecific - IMA401 is a bispecific targeting MAGEA4/8, with a Phase 1a study demonstrating a 29% (5/17) ORR and 25% (4/16) cORR in patients with MAGEA4/8high expression at relevant doses[132, 149] - The addressable MAGEA4/8+ and HLA-A*02:01+ patient population in the US & EU5 is approximately 62,000 per year[128, 130]

Business Overview - U.S net product revenue of NEXLETOL® and NEXLIZET® grew by 12% from Q3 2021 to $12.2 million in Q4 2021[10] - Sequential prescription growth of 9% from Q3 2021 with over 70,000 cumulative patients[10] - CLEAR Outcomes trial reached 90% MACE Accumulation in February 2022, with topline results expected in Q1 2023[10, 16] Financial Update - Ended Q4 2021 with $309.3 million in cash, cash equivalents, restricted cash and investment securities[10] - FY 2021 Operating Expenses were $305 million[22] - FY 2022 R&D Guidance is $100 - $110 million[22] - FY 2022 SG&A Guidance is $120 - $130 million[22] - Future Ex-U.S Collaboration Milestones from Daiichi Sankyo & Otsuka are >$1.2 billion[22] - Q4 2021 Common Shares Outstanding were 60.9 million[22]

Business Highlights - Approximately 35 million patients are considered statin intolerant in the U S, Japan, and the EU[8] - Statin intolerance impacts up to 20% of individuals treated with a statin[8, 12] - CLEAR Outcomes trial has enrolled over 14,000 patients and is powered for success[12] - The CLEAR Outcomes trial expects a high baseline LDL-C of approximately 140 mg/dL[12] Operational Performance - Over 8,600 doctors are writing at least one prescription for Esperion's medicines[15] - More than 3,300 patients are taking Esperion's medicines every week[15] - Over 420,000 seven-day starter packs have been requested and received[15] - New-to-brand statin prescriptions are down 9% year-to-date 2021 compared to year-to-date 2020 due to COVID-19 impacts[17] Financial Results & Guidance - U S Net Product Sales increased 14 6% quarter over quarter[23] - Royalty revenues from DSE were $8 2 million in the quarter[23] - Ex-U S Collaboration Milestones were $3 3 million[23] - The company anticipates FY 2021 revenues between $120 - $130 million[26]

FORWARD-LOOKNG STATEMENTS & DISCLOSURES MAY 3, 2022 © 2022 Esperion. All Rights Reserved – For investor audience only WHEN DO YOU PLAN TO GET YOUR NEXT CHOLESTEROL TEST? This presentation contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding marketing strategy and commercialization plans, current and planned operational expenses, future operations, commercial products, clinical development, including the timing ...

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