Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q1 2025 Earnings Presentation
27 slides from Rhythm Pharmaceuticals's earnings presentation, May 7, 2025
Slide 1: First Quarter 2025 Financial Results and Business Update
Rhythm Pharmaceuticals First Quarter 2025 Financial Results and Business Update May 7, 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: Executing on Long-term Growth Strategy
Executing on Long-term Growth Strategy Strong results from Ph3 trial of setmelanotide in acquired hypothalamic obesity (HO); On track to complete U.S. and EU regulatory submissions in Q3 2025 Solid patient demand growth for IMCIVREE® (setmelanotide) in Q1 2025 Data readout for Ph2 trial of oral biva
Slide 6: Severe, Life-long Burden for Patients with Acquired HO
Severe, Life-long Burden for Patients with Acquired HO Frequent visits with multiple specialists, a complex regimen of medications, and hospitalization “Treatment of patients with tumor/treatment- 3.7 average hospitalizations during the two years following index; 12 average number of general practit
Slide 7: Setmelanotide Achieved Statistically Significant Reduction in BMI in Phase 3
Setmelanotide Achieved Statistically Significant and Highly Clinically Meaningful Reduction in BMI in Phase 3 Acquired HO Trial Primary analysis cohort (N=120) -16.5% -19.8% +3.3% BMI change from baseline in Placebo-adjusted BMI change from baseline in Setmelanotide arm difference in BMI reduction P
Slide 8: Significant Reductions in BMI Observed in both Adults and Children
Significant Reductions in BMI Observed in both Adults and Children in Phase 3 Trial Evaluating Setmelanotide in Acquired HO ≥18 Years Old (n=49) <18 Years Old (n=71) 10 +2.4% +3.9% % Change from Baseline % Change from Baseline 20 0 10 0 -10 -10 -20 -20 -30 -40 -16.8% -19.2% Placebo-adjusted -30 -40
Slide 9: Mean BMI Reduction Consistent Across Stratified Age Groups
Mean BMI Reduction Consistent Across Stratified Age Groups in Phase 3 Trial Evaluating Setmelanotide in Acquired HO < 12 Yrs 12 – < 18 Yrs ≥ 18 Yrs -19.5% -21.0% -19.2% p <0.0001 p <0.0001 p <0.0001 (n=31: 20 setmelanotide, 11 placebo) (n=40: 28 setmelanotide, 12 placebo) (n=49: 33 setmelanotide, 16
Slide 10: Consistent Response to Setmelanotide Therapy Observed across Majority of Patients
Consistent Response to Setmelanotide Therapy Observed across Majority of Patients in Phase 3 Trial in Acquired HO 90% Setmelanotide 80% 80% Placebo 63% Proportion of patients 70% 60% 51% 50% 43% 40% 30% 20% 10% 5% 3% 10% 0% 0% 5% 10% 15% 20% P<0.0001 P<0.0001 P<0.0001 P<0.0001 Observed Reduction in
Slide 11: Vast Majority of Patients Completed Phase 3 Trial and Transitioned to Extension Study
Vast Majority of Patients with Acquired HO Completed Phase 3 Trial and Transitioned to Extension Study 143 Total Enrolled 120 Remain on Active Therapy 120 from pivotal cohort 108 in open label extension (OLE) 11 supplemental patients 12 in ongoing Japanese cohort 12 from Japanese cohort ® 11
Slide 12: Hypothalamic Obesity: A Potentially Transformative Opportunity
Hypothalamic Obesity: A Potentially Transformative Opportunity 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 13: Jennifer Lee
Jennifer Lee EVP, Head of North America ® 13
Slide 14: Consistent Demand and Steady Growth in U.S. BBS Prescriptions
Consistent Demand and Steady Growth in U.S. BBS Prescriptions Consistent Typical new-year Growth in patients demand for new increase in patients on reimbursed on Bridge resolved prescriptions therapy QoQ by end of Q1 Disease Clinical Decision Rx Education Suspicion Diagnosis to Treat IMCIVREE Access
Slide 15: Growth in both First-time and Repeat IMCIVREE Prescribers in Q1
Growth in both First-time and Repeat IMCIVREE Prescribers in Q1 Consistent growth 13% growth in number in prescriber base of prescribers from Q4’24 to Q1’25 32% of first-time prescribers turn into repeat prescribers (since launch) ® 15
Slide 16: Continued Improvement in Medicaid Access and Reimbursement
Continued Improvement in Medicaid Access and Reimbursement Medicaid Covered Lives ~85 million* >95%** <5%** of covered lives split between of covered lives in states with: states with: • no IMCIVREE prescription • an IMCIVREE policy in place; or received; • or a positive coverage decision • or IMCIV
Slide 17: Endocrinologists Detail Significant Unmet Need in HO
Endocrinologists Detail Significant Unmet Need in HO ~ 80% of patients with acquired HO will remain “We need a treatment that is going to be under endocrinologists’ care in perpetuity* more effective, well tolerated, safe to continue long term because this problem is not going to go away. These pati
Slide 18: Patients and Caregivers Describe Hyperphagia, Weight Gain, and Fatigue
The Majority of Patients and Caregivers Describe Hyperphagia, Weight Gain, and Fatigue as Most Impactful Symptoms on their Daily Lives I was just feeling like a different To not be able to lose a He can’t really get through an person after the tumor and the pound no matter what you do, entire day wi
Slide 19: Yann Mazabraud
Yann Mazabraud EVP, Head of International ® 19
Slide 20: Strong International Growth Driven by BBS, PPL and now HO
Strong International Growth Driven by BBS, PPL and now HO >15 COUNTRIES 6 COUNTRIES Countries with reimbursed access or with reimbursed -early named patient sales for patients with access programs or BBS and POMC or LEPR deficiency named patient sales for obesity patients with HO ® 20
Slide 21: Presentations and Symposia Planned for Key Medical Conferences
10 Presentations (including 5 orals) and 2 Expert-led Symposia Planned for Key Medical Conferences in May Four new data presentations: • ‘Real-world setmelanotide weight outcomes in French patients with acquired or congenital hypothalamic obesity’ (oral) • ‘Patient and caregiver experiences with set
Slide 22: Hunter Smith
Hunter Smith Chief Financial Officer ® 22
Slide 23: Q1 '25: Patient Demand Continued to Grow in U.S. and ex-U.S.
Q1 ’25: Patient Demand Continued to Grow in U.S. and ex-U.S. $41.8M Patients on reimbursed therapy $3.1M $37.7M grew by ~14% globally quarter- $1.1M over-quarter -$8.3M Typical Q4-Q1 inventory swing affected U.S. overall revenue U.S. demand growth driven by increased number of patients on reimbursed
Slide 24: 1Q 2025 Financial Snapshot
1Q 2025 Financial Snapshot ($ in millions, except per share Three months ended Three months ended data and shares outstanding) March 31, 2025 March 31, 2024 Product revenue, net $37.7M $26.0M R&D expenses $37.0M $128.7M SG&A expenses $39.1M $34.4M Net Loss attributable to common stockholders $(50.8)
Slide 25: Q1 2025 Financial Highlights
Q1 2025 Financial Highlights Q1 2025 OpEx $314.5M 65% includes 1Q 2025 cash, cash equivalents and short-term of Q1 2025 revenue from U.S. sales of $12.9M GAAP EPS of investments as of IMCIVREE, compared in stock-based compensation $(0.81) March 31, 2025 to 74% in Q4 2024 expense RYTM expects cash to
Slide 26: 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: $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 Non-GAAP Operatin
Slide 27: Questions
Questions Rhython
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