Slide 1: Third Quarter 2024 Financial Results and Business Update
Rhythm Pharmaceuticals Third Quarter 2024 Financial Results and Business Update November 5, 2024 ® © 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 contains 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 considered forward-looking statements,
Slide 4: David Meeker, MD
David Meeker, MD Chair, CEO and President ® 4
Slide 5: 2024: Continued Execution to Achieve Rhythm's Value Drivers
2024: Continued Execution to Achieve Rhythm’s Value Drivers 1 BBS global commercial execution Acquired hypothalamic obesity offers 2 a significant expansion opportunity Advancing clinical development pipeline 3 with new MC4R agonists, disease states ® 5
Slide 6: DAYBREAK 2-Stage Design: 16-Week Run-in Followed by 24-week Randomized Withdrawal
DAYBREAK 2-Stage Design: 16-Week Run-in Followed by 24-week Randomized Withdrawal and Double-blind, Placebo-controlled Stage 1: Open-label Run-in Stage 2: Double-blind, Placebo-controlled Open-label dose titration Open-label run-in Randomized, double-blind ≥12 years starting dose Setmelanotide Scree
Slide 7: DAYBREAK Baseline Demographics
DAYBREAK Baseline Demographics Sex, N=49 All <18 yrs ≥18 yrs Randomized into DAYBREAK Stage 2, n=49 Male n (%) 22 (44.9) 10 (45.5) 12 (54.5) Female n (%) 27 (55.1) 14 (51.9) 13 (48.1) Discontinued from stage 2, n=4* N=49 n Mean (SD) Range % of S1 starters Moved to early bridging due to weight regain
Slide 8: Data Highlights from Stage 2 of DAYBREAK Phase 2 Trial
Data Highlights from Stage 2 of DAYBREAK Phase 2 Trial -12.4% 84% 29% Mean BMI change from baseline or 27 of 32 VS. or 5 of 17 (SD: 8.0%; range 1.2%-35.0%) patients on patients on n=32 setmelanotide placebo patients on continuous setmelanotide therapy* achieved or maintained >5% BMI reduction from b
Slide 9: Variable Responses Observed in DAYBREAK Stage 2 in Different Genetic Cohorts
Variable Responses Observed in DAYBREAK Stage 2 in Different Genetic Cohorts Several genetically defined subgroups may merit further study with next-generation MC4R agonists PHIP Adult, n=5 Pediatric, n=4 SIM1 Adult, n=2 Pediatric, n=3 Early bridging to 0.0 0.0 Percentage BMI change Percentage BMI c
Slide 10: Positive Real-world Setmelanotide Data from French Early-access Program
Positive Real-world Setmelanotide Data Reported from French Early- access Program in Adult Patients with Acquired Hypothalamic Obesity -5.6% -12.8% -21.3% N=8* Mean BMI reduction Mean BMI reduction Mean BMI reduction patients ◼ Month 1 ◼ Month 3 ◼ Month 6 19.3 years #1 #2 #3 #4 #5 #6 #7 #8 Mean age
Slide 11: Multiple Anticipated Milestones
Multiple Anticipated Milestones Dec. 26, 2024 FDA PDUFA goal date for IMCIVREE label expansion to treat 2- to <6 year olds Complete enrollment in supplemental, 12-patient Japanese cohort 4Q 2024 in Ph3 acquired hypothalamic obesity trial 4Q 2024 Complete enrollment in 2 cohorts in Ph3 EMANATE trial
Slide 12: Jennifer Lee
Jennifer Lee EVP, Head of North America ® 12
Slide 13: 3Q 2024: Steady Progress with Prescriptions, Prescribers and Approvals for Reimbursement
3Q 2024: Steady Progress with Prescriptions, Prescribers and Approvals for Reimbursement Consistent Continued increase Continued success demand with new in both depth with approvals for prescriptions QoQ and breadth reimbursement of prescribers and re-authorizations ® 13
Slide 14: PDUFA Date for IMCIVREE Label Expansion to 2 Year Olds: Dec. 26, 2024
PDUFA Date for IMCIVREE Label Expansion to 2 Year Olds: Dec. 26, 2024 Significant unmet Treating severe Regulatory approval need for clearly obesity early can lead for patients as young differentiated MC4R to better health as 2yo would reinforce pathway diseases outcomes, improved IMCIVREE’s unique
Slide 15: Planning for Anticipated Success in Acquired Hypothalamic Obesity
Planning for Anticipated Success in Acquired Hypothalamic Obesity Conducting Engaging Preparing for market research actively with team with physicians, patient expansion in payers, and advocacy 2025 patients and families ® 15
Slide 16: Yann Mazabraud
Yann Mazabraud EVP, Head of International ® 16
Slide 17: Continued Progress in Securing Market Access for IMCIVREE
Continued Progress in Securing Market Access for IMCIVREE IMCIVREE is available in >15 countries outside the United States LAUNCHED EARLY ACCESS OR NAMED PATIENT SALES ® 17
Slide 18: Reimbursed Early-access Programs in France and Italy
Setmelanotide Benefiting Patients with Hypothalamic Obesity through Reimbursed Early-access Programs in France and Italy France Italy • Steady uptake since first • Eligibility: patients between available in early 2024 6-24 yo with HO caused by • Access granted by joint craniopharyngioma federal mult
Slide 19: BBS Commercial Launch Underway in England and Wales in Q4 24
BBS Commercial Launch Underway in England and Wales in Q4 2024 England and Wales • NICE recommended NHS reimbursement for patients younger than 18yo with BBS • 4 NHS BBS specialized clinics: 2 for adult patients, 2 for pediatric patients • Patients to be trained in hospital setting with at-home nurs
Slide 20: Continued Engagement with European MC4R Pathway Experts
Continued Engagement with European MC4R Pathway Experts ® 20
Slide 21: Hunter Smith
Hunter Smith Chief Financial Officer ® 21
Slide 22: 3Q 2024 Financial Snapshot
3Q 2024 Financial Snapshot ($ in millions, except per share Three months ended Three months ended data and shares outstanding) September 30, 2024 September 30, 2023 Product revenue, net $33.3M $22.5M R&D expenses $37.9M $33.6M SG&A expenses $35.4M $30.5M Net Loss attributable to common stockholders
Slide 23: 3Q 2024 Financial Highlights
3Q 2024 Financial Highlights 3Q 2024 OpEx $298.4M 70% includes 3Q 2024 cash, cash equivalents and short-term of 3Q 2024 revenue from U.S. sales of $11.0 GAAP EPS of investments as of IMCIVREE, compared in stock-based compensation $(0.73) September 30, 2024 to 74% in 2Q 2024 expense ® 23
Slide 24: 2024 OpEx Guidance
2024 OpEx Guidance $245M to $255M SG&A: $113M anticipated non-GAAP Operating Expenses* R&D: $137M** for 2024 includes: RYTM expects cash to be sufficient to fund planned operations into 2026 * Non-GAAP Operating Expenses is a non-GAAP financial measure. We define Non-GAAP Operating Expenses as GAAP
Slide 25: Questions
Questions Rhythm