Stephanie and Tristan Pioneering the Possible in Gene Editing May 2025 1 Forward Looking Statements This presentation contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ''anticipate,'' ''believe,'' ''continue,'' ''could,'' ''estimate,'' ''expect,'' ''intend,'' ''may,'' ''plan,'' ''potential,'' ''predict,'' ''project,'' ''target,'' ''should,'' ''would,'' and similar expressions are intended to identify forward-looking stat ...

Travere Therapeutics Corporate Overview February 2025 Forward-Looking Statements This presentation contains forward-looking statements, including but not limited to statements about: continued progress with the FILSPARI launch; statements regarding our products and products in development as potential foundational treatments and/or treatment standards; additional development and regulatory milestones, including expected data from additional studies and the expected timing thereof; plans and expectations reg ...

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]

Immatics Corporate Presentation May 31, 2025 Delivering the Power of T cells to Cancer Patients © Immatics. Not for further reproduction or distribution. © Immatics. Not for further reproduction or distribution. Forward-Looking Statement This presentation ("Presentation") is provided by Immatics N.V. ("Immatics" or the "Company") for informational purposes only. The information contained herein does not purport to be all- inclusive and none of Immatics, any of its affiliates, any of its or their respective ...

WHEN DO YOU PLAN TO GET YOUR NEXT CHOLESTEROL TEST? ESPERION Q4 & FY 2021 EARNINGS PRESENTATION FEBRUARY 22, 2022 @ 2022 Esperion. All Rights Reserved - For investor audience only. PERIO FORWARD-LOOKNG STATEMENTS & DISCLOSURES 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, restructuring and current and planned operational expenses, future ...

Q4 & FULL YEAR 2020 FINANCIAL RESULTS February 23, 2021 ESPERION ! ESPERION # EVERY PATIENT'S GOALS ARE UNIQUE We are singularly focused on bad cholesterol LIPID MANAGEMENT FOR EVERYBODY 35 Million SAFE HARBOR FORWARD- LOOKING STATEMENTS This presentation contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding the global clinical development and commercialization plans for bempedoic acid tablet and the bempedoic ...

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 ...

WHEN DO YOU PLAN TO GET YOUR NEXT CHOLESTEROL TEST? FORWARD-LOOKNG STATEMENTS & DISCLOSURES 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, restructuring and current and planned operational expenses, future operations, commercial products, clinical development including the timing, designs and plans for the CLEAR Outcomes study and its resu ...

Second Quarter 2024 Business Update August 8, 2024 © 2024 Scholar Rock, Inc. All rights reserved. 1 Forward-Looking Statements Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock Holding Corporation and Scholar Rock, Inc. (collectively, "Scholar Rock"), including without limitation, Scholar Rock's expectations regarding its strategy, its product candidate selection and development timing, including timing for the initiation of and reporting results ...

Dedicated to Dramatically Improving the Lives of Patients with SMA Q1 2025 Business Update May 14, 2025 © 2025 Scholar Rock, Inc. All rights reserved. Q1 Earnings Call | TO P I C | S P E A K E R | | --- | --- | | Scholar Rock Next Phase of Growth | David L. Hallal | | | Chief Executive Officer | | R&D Progress | Akshay Vaishnaw, M.D., Ph.D | | | President of R&D | | Commercial Readiness | Keith Woods | | | Chief Operating Officer | | Company Financials | Vikas Sinha | | | Chief Financial Officer | | Q&A Ses ...