Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q4 2025 Earnings Presentation
28 slides from Rhythm Pharmaceuticals's earnings presentation, Feb 26, 2026
Slide 1: Fourth Quarter and Year End 2025 Financial Results and Business Update
Rhythm Pharmaceuticals Fourth Quarter and Year End 2025 Financial Results and Business Update February 26, 2026 ® © 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: Well Positioned to Deliver Long-term, Sustained Growth: Business Highlights
Well Positioned to Deliver Long-term, Sustained Growth Business Highlights Steady growth in global IMCIVREE® (setmelanotide) sales in Q4 2025 primarily driven by Bardet-Biedl syndrome U.S. team in place and ready to launch setmelanotide for acquired HO, pending FDA approval on March 20th Early-acces
Slide 6: SIGNAL Trial: Phase 2 Open-label Trial Evaluating Bivamelagon in Hypothalamic Obesity
SIGNAL Trial: 14-week, Phase 2 Open-label Trial Evaluating Bivamelagon in Patients with Hypothalamic Obesity RDZ 1:1:1:1 200 mg 200 mg 400 mg Long-term Screening 200 mg 400 mg 600 mg Extension (LTE) Trial 200 mg 400 mg 600 mg Placebo 2 weeks 12 weeks Open-label Week 0 Week 2 Week 14 Week 15 Week 16
Slide 7: Vast Majority of Patients Transitioned to Open-label Extension and Remained on Bivamelagon
Vast Majority of Patients Transitioned to Open-label Extension and Have Remained on Bivamelagon Therapy ENROLLED 26 of 27 eligible participants (n=28) transitioned to open-label extension (OLE) phase for up to 38 weeks DISCONTINUED 1 discontinuation OLE participants retitrated from due to SAE in Wee
Slide 8: Placebo Patients Titrated to 600mg Achieved Meaningful BMI Reductions
Ph2 Trial of Bivamelagon in hypothalamic obesity: Open-label extension Placebo Patients Titrated to 600mg Achieved Meaningful Reductions in BMI at Week 28 and Deepening Responses at Week 40 Age/Sex: 12/F 13/M 15/M 19/F 21/M 43/M 66/F 0 -2 Percentage Change in BMI, % -4 -4.5 -6 -5.5 -8 -7.6 -8.5 -8.2
Slide 9: Patients Initially Randomized to 200mg and Titrated to 600mg Achieved BMI Reductions
Ph2 Trial of Bivamelagon in hypothalamic obesity: Open-label extension Patients Initially Randomized to 200mg and Titrated to 600mg Achieved Meaningful Reductions in BMI at Weeks 28 and 40 Age/Sex: 12/F 14/M 15/F 21/F 22/M 37/M 10 5.9 5 Percentage Change in BMI, % 0 -5 -3.1 -10 PI suspects -8.2 -8.9
Slide 10: Patients Titrated from 400mg to 600mg Achieved Deepened, Sustained BMI Reduction
Ph2 Trial of Bivamelagon in hypothalamic obesity: Open-label extension Patients* Titrated from 400mg to 600mg Achieved Deepened, Sustained BMI Reduction at Weeks 28 and 40 Age/Sex: 12/F 14/F 16/F 20/M 23/F 28/M 5 1.3 0 Percentage Change in BMI, % -5 -10 PI suspects -10.4 non- -12.0 -12.4 -15 complia
Slide 11: Patients on 600mg Achieved Deepened and Sustained BMI Reduction at Week 40
Ph2 Trial of Bivamelagon in hypothalamic obesity: Open-label extension Patients on 600mg Achieved Deepened and Sustained BMI Reduction at Week 40 Age/Sex: 13/M 14/M 14/F 14/M 29/M 39/F 64/M 68/F 10 7.5 Pt did not consent to OLE 5 Percentage Change in BMI, % 0 -5 -4.9 -10 PI suspects non- -15 complia
Slide 12: Bivamelagon Long-term Extension Data Demonstrated Sustained Efficacy
Bivamelagon Long-term Extension Data Demonstrated Sustained Efficacy Open-label extension data show persistent, deepening BMI reductions at 6- and 9-months Extension safety and tolerability generally consistent with 14- week results End of Phase 2 meeting with FDA completed On track to initiate Phas
Slide 13: Multiple Anticipated Milestones
Multiple Anticipated Milestones PDUFA goal date for setmelanotide in conditions associated with acquired Mar. 20, 2026 hypothalamic obesity Complete enrollment in Part C of Ph1/2 trial evaluating RM-718 in acquired Q1 2026 hypothalamic obesity Topline data from 12-patient Japanese cohort of Ph3 tria
Slide 14: Jennifer Lee
Jennifer Lee EVP, Head of North America ® 14
Slide 15: Anticipated US Launch of IMCIVREE in Acquired Hypothalamic Obesity
Anticipated US Launch of IMCIVREE in Acquired Hypothalamic Obesity PDUFA goal date of March 20, 2026 Significant Estimated US unmet need Prevalence of Team in place without an ~10,000 and ready to approved patients1 launch therapy 1. U.S. estimates based on reported incidence of hypothalamic obesity
Slide 16: Pituitary Centers Use Multiple Care Models for HO
Pituitary Centers Use Multiple Care Models for HO Tumor Neurosurgery Radiation Neuro-oncology treatment Endocrinopathy Endocrinology management Weight management / Clinical nutrition/ Community Providers Treatment Endocrinology (PCP, ABOM) Metabolic medicine Supportive care of HO ® 16
Slide 17: Ready to Launch Upon FDA Approval
Ready to Launch Upon FDA Approval Establish IMCIVREE as Patient services team Access team the foundational engaging with engaging with payers treatment for aHO patients, families ® 17
Slide 18: Yann Mazabraud
Yann Mazabraud EVP, Head of International ® 18
Slide 19: IMCIVREE available in >25 countries outside the United States
IMCIVREE available in >25 countries outside the United States Ongoing BBS, POMC/LEPR sales and Early-access aHO programs in France and Italy Continued execution >100 employees in 13 countries Eight countries added in 2025 64 abstracts at 12 international scientific congresses ® 19
Slide 20: Early-onset Obesity Model published in Obesity Facts Journal
Early-onset Obesity Model published in Obesity Facts Journal Obesity Facts. 2025 Nov 14:1-15. doi: 10.1159/000549499 ® 20
Slide 21: Preparing to Launch Setmelanotide for Acquired Hypothalamic Obesity in Japan and Europe
Preparing to Launch Setmelanotide for Acquired Hypothalamic Obesity in Japan and Europe 5,000 – 8,000 ~10,000 estimated Japanese prevalence1 estimated European prevalence2 Top-line Phase 3 Data EMA Authorization expected in March 2026 expected in H2 2026 1. Rhythm estimates the prevalence of acquire
Slide 22: Hunter Smith
Hunter Smith Chief Financial Officer ® 22
Slide 23: Q4 2025: Continued Growth in IMCIVREE Global Sales
Q4 2025: Continued Growth in IMCIVREE Global Sales $57.3M * QoQ increase 12% from Q3 2025** Q4 2025 of Q4 2025 revenue ~10% 68% from U.S. increase in number of patients globally on reimbursed therapy $194.8M 69% of FY 2025 revenue from U.S. FY 2025 * Q4 2025 revenue from vials shipped to U.S. specia
Slide 24: Q3 to Q4 '25: Consistent Growth in Global Patient Demand Continues
Q3 to Q4 ’25: Consistent Growth in Global Patient Demand Continues $5.2M $57.3M $51.3M $2.1M Q3 2025 -$1.3M Revenue growth from Ex-US revenue in Q3 2025 Q4 2025 * product dispensed to included a one-time Product Q3-Q4 inventory $3.2M charge due to Product US patients Revenue effect at U.S. French re
Slide 25: Q4, Full-year 2025 Financial Snapshot
Q4, Full-year 2025 Financial Snapshot ($ in millions, except per share Three months ended Three months ended Year ended Year ended data and shares outstanding) December 31, 2025 December 31, 2024 December 31, 2025 December 31, 2024 Product revenue, net $57.3M $41.8M $194.8M $130.1M R&D expenses $42.
Slide 26: 4Q, Full-year 2025 Financial Highlights
4Q, Full-year 2025 Financial Highlights $362.3M 68,285,039 2025 GAAP OpEx, which includes Common shares outstanding2 $66.8M in stock-based including compensation 729,164 $295.5M* shares of common stock converted from preferred shares Non-GAAP1 OpEx * Non-GAAP Operating Expenses is a non-GAAP financi
Slide 27: 2026 OpEx Guidance
2026 OpEx Guidance $385M to $415M R&D: $197M to $213M anticipated non-GAAP Operating Expenses* for 2026 includes: SG&A: $188M to $202M * Non-GAAP Operating Expenses is a non-GAAP financial measure. We define Non-GAAP Operating Expenses as GAAP operating expenses excluding stock-based compensation an
Slide 28: Questions
Questions Rhythm
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