Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q4 2023 Earnings Presentation
32 slides from Rhythm Pharmaceuticals's earnings presentation, Feb 22, 2024
Slide 1: Fourth Quarter/Year End 2023 Financial Results and Business Update
Rhythm Pharmaceuticals Fourth Quarter/Year End 2023 Financial Results and Business Update February 22, 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 Hunter Sm
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, M Rhython
Slide 5: Continued Execution on Clinical Development, Global Commercialization
Continued Execution on Clinical Development, Global Commercialization Expand opportunity with Multiple development Strong BBS hypothalamic obesity programs advancing commercial execution • Ph 3 enrollment complete with • Acquired license to LB54640, oral • $24.2 million in net revenues from 120 pati
Slide 6: Potential Path to Registration Set based on Feedback from Japan's PMDA
Potential Path to Registration Set based on Feedback from Japan’s PMDA Planned Japanese clinical Regulatory submissions development • No anticipated impact on timing of • Supplemental cohort of Ph3 trial to anticipated FDA and EMA enroll 12 or more Japanese patients regulatory submissions • First pa
Slide 7: Phase 3 Hypothalamic Obesity Trial: Enrollment Complete, Top-line Data Expected in 1H2025
Phase 3 Hypothalamic Obesity Trial: Enrollment Complete, Top-line Data Expected in 1H2025 Pivotal cohort Dose titration Double-blind, placebo-control Setmelanotide QD (n=80) 120 Open-label extension patients randomized Placebo QD (n=40) to setmelanotide: placebo Up to 52 weeks 8 weeks Primary endpoi
Slide 8: Rhythm, LG Chem Agreement Designed to Accelerate Development
Rhythm, LG Chem Agreement Designed to Accelerate Development and Delivery of Additional Therapy Options for Patients Life Sciences Company Highly regarded global LB54640: Oral, highly selective MC4R biopharma company with strong agonist with compelling Phase 1 data; chemistry and translational scien
Slide 9: LB54640 Showed Dose-response Body Weight Loss in Healthy Obese Volunteers
LB54640 Showed Dose-response Body Weight Loss in Healthy Obese Volunteers Favorable safety profile • No serious adverse events • No skin pigmentation, adrenal, or genitourinary adverse events observed • Mild to moderate nausea, diarrhea, vomiting most common As presented by LG Chem at The Obesity So
Slide 10: SIGNAL Trial: 14-week, Phase 2 Open-label Trial Evaluating LB54640
SIGNAL Trial: 14-week, Phase 2 Open-label Trial Evaluating LB54640 in Patients with Hypothalamic Obesity Transfer of sponsorship from LG Chem to Rhythm in process Placebo N=28 Randomized Low dose patients with Open-label extension hypothalamic 1:1:1:1 Middle dose obesity High dose 14 weeks Up to 52
Slide 11: RM-718 has Demonstrated Similar Safety Compared to Setmelanotide Weekly Formulation
RM-718 has Demonstrated Similar Safety Compared to Setmelanotide Weekly Formulation in Pre-clinical Studies In vivo safety results supportive of no off-target cardiovascular effects, like setmelanotide No hyperpigmentation observed in vivo In vivo results suggest potential efficacy for body weight r
Slide 12: Setmelanotide Achieved Clinically Meaningful Reductions in BMI and BMI-Z in 2-<6yo Patients
Setmelanotide Achieved Clinically Meaningful Reductions in BMI and BMI-Z in 2-<6yo Patients with POMC/LEPR Deficiency or BBS Analysis set population (N=12) 83.3% (10 of 12) -18.380% of all patients achieved Mean percent change from baseline ≥0.2 reduction in BMI-Z score from in BMI at Week 52 baseli
Slide 13: Setmelanotide Achieved Consistent Reductions in BMI-Z Score
Setmelanotide Achieved Consistent Reductions in BMI-Z Score POMC (n=3) LEPR (n=4) BBS (n=5) 2 # 1 0 * -3.04 -1 Mean change from baseline in BMI-Z at Week 52 (N=12) -2 -4.3 -1.3 BMI-Z -3 -4 POMC/LEPR BBS -5 (n=7) (n=5) -6 -7 11 patients completed the trial, -8 005-001 006-001 006-002 003-001 004-001
Slide 14: Jennifer Lee: BBS U.S. Launch
Jennifer Lee BBS U.S. Launch ® 14
Slide 15: Fourth Quarter 2023 Prescriptions and Approvals for Reimbursement
Fourth Quarter 2023 Prescriptions and Approvals for Reimbursement >100 >70 Prescriptions Approvals for received during reimbursement received 4Q 2023 during 4Q 2023 30 previously reimbursed patients were transitioned to free drug through bridge program due to a single state Medicaid program requesti
Slide 16: Snapshot of BBS Patients with Prescriptions
Snapshot of BBS Patients with Prescriptions Age Range Since Launch* Adult ~59% (18+) Adolescent ~23% (12-17) Pediatric ~18% (6-11) *As of December 31, 2023 ® 16
Slide 17: Increased Depth and Breadth of BBS IMCIVREE Prescriber Base
Increased Depth and Breadth of BBS IMCIVREE Prescriber Base 11% Endocrinology 34% Pediatric >360* Endocrinology 24% Prescribers Primary Care as of 12/31/2023 15% 28% of prescribers ‘new to Rhythm’ 16% Pediatrics All others 30% of prescribers have written more than one prescription *As of December 31
Slide 18: Incremental Improvement in Medicaid Access and Reimbursement
Incremental Improvement in Medicaid Access and Reimbursement with >85% of Covered Lives in States with Positive Coverage Medicaid Covered Lives ~85 million* >85%** <15%** of covered lives split between of covered lives in states with: states with: • no IMCIVREE prescription • an IMCIVREE policy in p
Slide 19: Vast Majority of IMCIVREE Prescription Re-authorizations Approved
Vast Majority of IMCIVREE Prescription Re-authorizations Approved 110* * approved at initial re-authorization >90% re-authorization approvals (at 3-, 6- or 12- 10 denials: 6 already approved**, working toward months) approval for remaining 4 *As of December 31, 2023; ** As of February 16, 2024 ® 19
Slide 20: Yann Mazabraud, EVP, Head of International
Yann Mazabraud EVP, Head of International ® 20
Slide 21: Significant Opportunity in Japan with Higher Per-capita Incidence of Hypothalamic Obesity
Significant Opportunity in Japan with Higher Per-capita Incidence and Prevalence of Hypothalamic Obesity • Prevalence is 2-3 times higher than in the USA & Europe due to a higher frequency of craniopharyngioma been reported 5,000 – 8,000 • > 100 health care centers treating patients with hypothalami
Slide 22: Rhythm's Initial Hypothalamic Obesity Symposium in Japan Well Attended
Rhythm’s Initial Hypothalamic Obesity Symposium in Japan Well Attended ® 22
Slide 23: Solid Start for IMCIVREE Launch for BBS in Germany
Solid Start for IMCIVREE Launch for BBS in Germany Continued focus on engaging with physicians at care centers across the country Engaging care centers, large hospitals Rhythm@Home ~1,200 leads to prescriptions Estimated German prevalence* ~800 Patients diagnosed with BBS* >250 Patients with BBS ide
Slide 24: Reimbursement Achieved for IMCIVREE for BBS in Two New Markets
Reimbursement Achieved for IMCIVREE for BBS in Two New Markets Spain Italy Launched in 1Q 2024 Launched in 1Q 2024 ~700 ~900 estimated prevalence estimated prevalence ~100 ~200 Patients diagnosed with BBS Patients diagnosed with BBS and identified and identified *Internal company estimates. ® 24
Slide 25: Market Access Achieved for IMCIVREE in 12 Countries outside of North America
Market Access Achieved for IMCIVREE for Biallelic POMC/LEPR and/or BBS in 12 Countries outside of North America United Kingdom Netherlands Launched in Launched in POMC/LEPR; POMC/LEPR; BBS launch BBS launch 2H2024 in 2H2024 Germany Launched in BBS and France POMC/LEPR Reimbursed early- access progra
Slide 26: Hunter Smith: 4Q 2023 Financial Results
Hunter Smith 4Q 2023 Financial Results ® 26
Slide 27: 4Q 2023 Financial Snapshot
4Q 2023 Financial Snapshot $275.8M* cash, cash equivalents and short-term investments expected to be sufficient to fund operations into 2025 ($ in millions except as noted, per share Three months ended Three months ended Year ended Year ended data and shares outstanding) December 31, 2023 December 3
Slide 28: 4Q and Year End 2023 Financial Highlights
4Q and Year End 2023 Financial Highlights $275.8M 76% GAAP OpEx of Non-GAAP1 cash equivalents and of 4Q 2023 revenue $261.8M includes OpEx short-term investments from U.S. sales $32.6M for 2023 of as of December 31, of IMCIVREE in stock-based $219.9M* 2023 vs. 80% in compensation 3Q 2023 in 2023 RYT
Slide 29: Financial Guidance for 2024
Financial Guidance for 2024 $100 million in fixed consideration commitment Anticipated Non-GAAP Operating Expenses1 to acquire LB54640 from LG Chem* for 2024: • $40 million paid in January 2024 $250M to $270M • 432,143 shares of common stock issued to LG • R&D: $145 million to $160 million, Chem, va
Slide 30: David Meeker, MD: Conclusion
David Meeker, MD Conclusion ® 30
Slide 31: Continued Execution: Recent Achievements and Multiple Anticipated Milestones
Continued Execution: Recent Achievements and Multiple Anticipated Milestones Recent achievements Anticipated milestones in 2024 ✓Licensed global rights to oral MC4R agonist LB54640 o 1H2024: Initiate Phase 1 study of RM-718 ✓Completed enrollment in Phase 3 hypothalamic obesity QW trial o 1H2024: Com
Slide 32: Questions
Questions Rhython
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