Slide 1: Fourth Quarter, Full Year 2024 Financial Results and Business Update
Rhythm Pharmaceuticals Fourth Quarter, Full Year 2024 Financial Results and Business Update February 26, 2025 ® © Rhythm® Pharmaceuticals, Inc. All rights reserved.
Slide 2: On Today's Call
On Today’s Call • David Connolly, Executive Director of Investor Relations and Corporate Communications • David Meeker, MD, Chair, President and Chief Executive Officer • Jennifer Lee, Executive Vice President, Head of North America • Yann Mazabraud, Executive Vice President, Head of International •
Slide 3: Forward-looking Statements
Forward-looking Statements This presentation and the accompanying oral presentation contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this presentation that do not relate to matters of historical fact should be
Slide 4: David Meeker, MD
David Meeker, MD Chair, President and CEO ® 4
Slide 5: Focus on Long-term Value Drivers
Focus on Long-term Value Drivers Significant potential expansion opportunity in hypothalamic obesity 1 (both acquired and congenital) 2 Driving life-cycle management with bivamelagon and RM-718 Advancing setmelanotide clinical development in Prader-Willi 3 syndrome and genetically-defined MC4R pathw
Slide 6: 2025: A Year of Important Milestones and Data Readouts
2025: A Year of Important Milestones and Data Readouts Acquired hypothalamic obesity Setmelanotide Bivamelagon RM-718 Pivotal data readout Ph2 enrollment HO enrollment to begin on track for complete; data Q1 2025; aiming Q2 2025 anticipated in for data in H2 2025 H2 2025 ® 6
Slide 7: Phase 3 Hypothalamic Obesity Trial: Enrollment Complete, Top-line Data Expected in Q2 2025
Phase 3 Hypothalamic Obesity Trial: Enrollment Complete, Top-line Data Expected in Q2 2025 Pivotal cohort Dose titration Double-blind, placebo-control Setmelanotide QD (n=80) 120 Open-label extension patients randomized to setmelanotide: Placebo QD (n=40) placebo Up to 52 weeks 8 weeks +12 Japanese
Slide 8: Hypothalamic Obesity: A Transformative Opportunity for Rhythm
Hypothalamic Obesity: A Transformative Opportunity for Rhythm 5,000 – 10,000 Unmet medical need is high estimated U.S. prevalence1 Patients are identified 3,500 – 10,000 Patients engaged with system estimated European prevalence2 5,000 – 8,000 ~500 Estimated incidence in each U.S., Europe and
Slide 9: Multiple Anticipated Milestones
Multiple Anticipated Milestones Begin enrolling patients with acquired hypothalamic obesity in Q1 2025 Part C of Ph1 trial evaluating RM-718 Begin enrolling patients with congenital hypothalamic obesity in substudy of Q1 2025 Ph3 trial evaluating setmelanotide Begin enolling patients with Prader-Wil
Slide 10: Jennifer Lee
Jennifer Lee EVP, Head of North America ® 10
Slide 11: Steady Progress with Prescriptions, Prescribers and Approvals for Reimbursement
Steady Progress with Prescriptions, Prescribers and Approvals for Reimbursement Continued success Multiple process Consistent new with approvals for enhancements prescriptions reimbursement designed to QoQ and maintain patients on re-authorizations therapy Disease Clinical Decision Rx Education Susp
Slide 12: IMCIVREE Now Approved for Children as Young as 2 Years Old
IMCIVREE Now Approved for Children as Young as 2 Years Old Patient report: Ben, now 6, LEPR biallelic patient on therapy for >2.5 years “It has changed his life; his personality is different now “It was a constant that he’s not focused on food, struggle because he was and as a family, we now have al
Slide 13: Planning for Potential Success in Acquired Hypothalamic Obesity
Planning for Potential Success in Acquired Hypothalamic Obesity Ongoing market Engaging research with Field teams actively with physicians, engaging with patient payers, and physicians advocacy patients and families ® 13
Slide 14: Yann Mazabraud
Yann Mazabraud EVP, Head of International ® 14
Slide 15: IMCIVREE available in >15 countries outside the United States
IMCIVREE available in >15 countries outside the United States LAUNCHED EARLY ACCESS OR NAMED PATIENT SALES ® 15
Slide 16: Hunter Smith
Hunter Smith Chief Financial Officer ® 16
Slide 17: Well Capitalized Entering 2025
Well Capitalized Entering 2025 $320.6M $75M cash equivalents and of gross proceeds raised through ATM short-term investments program as of December 31, 2024 $40M raised in 4Q 2024 and $35M raised in January 2025 RYTM expects cash to be sufficient to fund planned operations into 2027 ® 17
Slide 18: Q4, Full-year Financial Snapshot
Q4, Full-year Financial Snapshot ($ in millions, except per share Three months ended Three months ended Year ended Year ended data and shares outstanding) December 31, 2024 December 31, 2023 December 31, 2024 December 31, 2023 Product revenue, net $41.8M $24.2M $130.1M $77.4M R&D expenses $41.2M $29
Slide 19: 4Q, Full-year 2024 Financial Highlights
4Q, Full-year 2024 Financial Highlights $382.3M $41.8M $130.1M 2024 GAAP OpEx includes of Q4 2024 of FY 2024 $39.7M in stock-based revenue from revenue from compensation and $92.4M IMCIVREE sales, IMCIVREE sales, in LGC purchase consideration 76% US 74% US $250.2M* Non-GAAP1 OpEx * Non-GAAP Operatin
Slide 20: 2025 OpEx Guidance
2025 OpEx Guidance $285M to $315M SG&A: $135M to 145M anticipated non-GAAP Operating R&D: $150M to $170M Expenses* for 2025 includes: Does not include $40M LGC in-license milestone payable by July 4 2025 accounted for in 2024 * Non-GAAP Operating Expenses is a non-GAAP financial measure. We define N
Slide 21: Questions
Questions Rhythm