Mirum Pharmaceuticals: Transforming Lives in Rare Disease M a y 2 0 2 4 Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management's beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and opportunities, including the future opportunities ...

NTLA-2002 (Hereditary Angioedema - HAE) - NTLA-2002 aims to be the first to offer lifelong freedom from attacks and prophylaxis after a single dose for HAE, with a BLA submission planned in 2026[8] - Phase 2 study results showed 73% of patients were attack-free and off chronic prophylaxis after a single dose of NTLA-2002 at 16 weeks[37, 41] - The global market for HAE is projected to reach approximately $5 billion by 2028[8, 44] Nex-z (Transthyretin Amyloidosis - ATTR) - Nex-z aims to be the first to stabilize or reverse disease progression with a single dose for ATTR, with a BLA submission planned in 2028 for polyneuropathy[8] - Phase 1 data showed deep, rapid, and durable reductions in serum TTR, with an 89% reduction in ATTR-CM and a 90% reduction in ATTRv-PN at day 28[63] - The global market for ATTR is projected to reach approximately $12 billion by 2028[8, 86] Clinical Development and Milestones - Intellia expects three commercial launches starting in 2027[8] - The company plans to complete enrollment in the Phase 3 HAELO trial for NTLA-2002 in the second half of 2025 and the MAGNITUDE-2 trial for ATTRv-PN in 2026[2] - Intellia plans to enroll at least 550 patients in the Phase 3 MAGNITUDE trial for ATTR-CM in 2025 and complete enrollment in early 2027[2]

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 ...

Precision oncology medicines powered by synthetic lethal insights Corporate Presentation May 2025 Disclaimer Statements contained in this presentation regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Words such as "anticipates," "believes," "expects," "intends," "plans," "potential," "projects," "would" and "future" or similar expressions are intended to identify forward-looking statements ...

Zev A. Wainberg, Alisha H. Bent, Victor Moreno, Manuel Pedregal, Rutika Mehta, Eric Chen, Jorge Ramon-Patino, Ryan H. Moy, Brian Madajewski, Adam Petrone, Pooja Adesara-Patel, Yajun Liu, Xizi Sun, Elia Aguado-Fraile, Paul Basciano, Sunantha Sethuraman, Nathan Hawkey, Elisa Fontana DECLARATION OF INTERESTS PHASE 1 STUDY OF THE PKMYT1 INHIBITOR LUNRESERTIB (LUNRE) IN COMBINATION WITH FOLFIRI IN ADVANCED GASTROINTESTINAL CANCERS (MINOTAUR STUDY) Elisa Fontana, MD, PhD Sarah Cannon Research Institute UK, London ...

Introduction Justine Koenigsberg, Vice President, Investor Relations Kymera Therapeutics Fourth Quarter & Full Year 2024 Quarterly Results Call February 27, 2025 Agenda Key Highlights & Business Update Nello Mainolfi, PhD, Founder, President and Chief Executive Officer Clinical Update Jared Gollob, MD, Chief Medical Officer Financial Review Bruce Jacobs, CFA, MBA, Chief Financial Officer Question and Answer Session Forward Looking Statements This presentation contains forward-looking statements within the m ...

January 2025 Revolutionizing Immunology: Oral Drugs with Biologics-Like Efficacy Science-driven clinical stage organization with industry-leading oral immunology pipeline J.P. Morgan Healthcare Conference Nello Mainolfi, Ph.D., Founder, President and CEO Forward Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. These statements include, but are not limited to, implied and expr ...