Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q4 2022 Earnings Presentation
29 slides from Rhythm Pharmaceuticals's earnings presentation, Mar 1, 2023
Slide 1: Fourth Quarter / Full Year 2022 Financial Results and Business Update
Rhythm Pharmaceuticals Fourth Quarter / Full Year 2022 Financial Results and Business Update March 1, 2023 ® © 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 Chien, Executive Vice President, Head of North America Yann Mazabraud, Executive Vice President, Head of International Hunter
Slide 3: Important Notice
Important Notice This presentation contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including without limitat
Slide 4: David Meeker, MD
David Meeker, M Rhython
Slide 5: Rare Disease Day, World Obesity Day and Obesity Care Week
Rare Disease Day, World Obesity Day and Obesity Care Week ® 5
Slide 6: Building a Global Company: The Next Chapter
Building a Global Company: The Next Chapter Global commercial Hypothalamic obesity Meaningful new strategy execution leads setmelanotide opportunity with • Strong start to U.S. BBS launch* expansion acquisition of Xinvento • >200 new prescriptions • Actively engaged patient • Congenital hyperinsulin
Slide 7: Clear Unmet Medical Need for Improved Treatment Options for Patients with Congenital Hyperinsulinism
Clear Unmet Medical Need for Improved Treatment Options for Patients with Congenital Hyperinsulinism • Chronic dysregulation of insulin from pancreatic β-cells, resulting in hypoglycemias • Can cause brain damage with neuro-developmental deficits in 1:29,000 - ~50% of patients; without proper and im
Slide 8: Hypothalamic Obesity: Sites Initiated, Patients Being Screened for Phase 3 Trial
Hypothalamic Obesity: Sites Initiated, Patients Being Screened for Phase 3 Double-blind, Randomized Controlled Trial Enrollment Expected to be Complete in 6-12 Months Dose titration Double-blind, placebo-control Screening and Randomization Setmelanotide QD (n=80) Open-label setmelanotide: placebo 2:
Slide 9: Hypothalamic Obesity: A Transformative Opportunity for Rhythm
Hypothalamic Obesity: A Transformative Opportunity for Rhythm ✓ Unmet medical need is high; no approved therapies 5,000 – 10,000* ✓ MC4R pathway deficiency following patients injury to hypothalamic region Estimated U.S. prevalence ✓ Compelling Phase 2 efficacy data with consistent, durable weight lo
Slide 10: Multiple Clinical Trials Ongoing to Expand IMCIVREE Label and Overall Opportunity
Multiple Clinical Trials Ongoing to Expand IMCIVREE Label and Overall Opportunity Pediatrics Trial Weekly Formulation Phase 3 Phase 3 Patients aged 2 to <6 years Switch Trial Hypothalamic obesity Phase 3 Trial initiated Phase 3 Trial Phase 2 Trial ® 10
Slide 11: Bardet-Biedl Community is Established and Patients are Identified
Updated Bardet-Biedl Community is Established and Patients are Identified U.S. prevalence European prevalence estimated to be estimated to be 4,000 to 5,000* 4,000 – 5,000* patients patients More than 600 individuals >1,500 with BBS are enrolled in individuals identified in CRIBBS registry EU4 + UK
Slide 12: Jennifer Chien: BBS U.S. Launch
Jennifer Chien BBS U.S. Launch ® 12
Slide 13: Journey for Patients, Prescribers and Payers
Journey for Patients, Prescribers and Payers Consistent focus since FDA approval for BBS in June 2022 Disease Clinical Decision Rx Education Suspicion Diagnosis to Treat IMCIVREE Access Maintenance ® 13
Slide 14: Results Show Strong Demand and Commercial Execution since FDA Approval for IMCIVREE
Results Show Strong Demand and Commercial Execution since FDA Approval for IMCIVREE through December 31,2022 >200* >100* New BBS prescriptions reimbursement 6/16/22 to 12/31/22 approvals >120 >80 >125* received as received unique of Sept. 30, during prescribers 2022 4Q2022 * As of December 31, 2022.
Slide 15: BBS IMCIVREE Prescribers by Specialty Since Launch
BBS IMCIVREE Prescribers by Specialty Since Launch 36% 11% >125* Prescribers as of 12/31/2022 18% 24% 22% 11% 78% Pediatric Endocrinology Pediatrics All others Primary Care Endocrinology New Physicians Targeted Physicians *As of December 31, 2022 ® 15
Slide 16: Almost 90% of BBS Prescriptions Since Launch Fall Under Commercial and Medicaid Plans
Almost 90% of BBS Prescriptions Since Launch Fall Under Commercial and Medicaid Plans Reimbursement >100 approvals 47% • Typically payer approval process takes between one 40% and three months 9% • Following initial approvals, 3% subsequent approvals are faster for that payer Commercial Medicaid Med
Slide 17: Snapshot of Patient Prescriptions
Snapshot of Patient Prescriptions Age Range Since Launch Adult (18+) ~46% Adolescent (12-17) ~28% Pediatric (6-11) ~26% 95% of BBS prescriptions are written for patients who are consented to InTune *As of December 31, 2022 ® 17
Slide 18: Accelerating Hope: Rhythm North America Kick Off Meeting to Align on 2023 Strategy
Accelerating Hope: Rhythm North America Kick Off Meeting to Align on 2023 Strategy Priorities for 2023 • Solidify the need to treat MC4R pathway driven hyperphagia and obesity • Establish IMCIVREE as the preferred treatment for BBS patients • Cultivate a positive Rhythm and IMCIVREE experience for c
Slide 19: Yann Mazabraud: EVP, Head of International
Yann Mazabraud EVP, Head of International ® 19
Slide 20: International Team in Place in Nine Ex-U.S. Countries
International Team in Place in Nine Ex-U.S. Countries 2023 International Priorities • Best practices in market access, patient identification and omnichannel approach • Execution of integrated, aligned & simple launch plans • Operational excellence ® 20
Slide 21: Market Access for Patients with POMC, PCSK1 and LEPR Achieved in Eight Countries
Market Access for Patients with POMC, PCSK1 and LEPR Achieved in Eight Countries POMC, PCSK1 and LEPR ✓ United Kingdom: Launched Deficiency Obesities ✓ Germany: Launched ✓ France: Paid early access ~100 ✓ Italy: Launched individuals identified in EU4 + UK ✓ The Netherlands: Launched ✓ Austria: Named
Slide 22: Making Significant Progress towards Access for BBS since September 2022 EC Marketing Authorization
Making Significant Progress towards Access for BBS since September 2022 EC Marketing Authorization Bardet-Biedl Syndrome ✓ France: Paid early access Anticipated Launches: >1,500 o Germany: G-BA exemption status progressing and launch in 2Q 2023 individuals identified in EU4 + UK (~20 academic medica
Slide 23: Hunter Smith: 4Q/YE 2022 Financial Results
Hunter Smith 4Q/YE 2022 Financial Results ® 23
Slide 24: 4Q 2022 Financial Snapshot
4Q 2022 Financial Snapshot $333.3M* cash on hand expected to be sufficient to fund operations into 2025 ($ in millions except as noted, per share Three months ended Three months ended Year ended Year ended data and shares outstanding) December 31, 2022 December 31, 2021 December 31, 2022 December 31
Slide 25: Efficient Execution to Maximize Shareholder Value
Efficient Execution to Maximize Shareholder Value Anticipated Non-GAAP Operating Expenses1 Acquisition of Xinvento* for 2023: • $5 million upfront payment; $6 million in $200M to $220M preclinical development milestones • R&D: $120 million to $130 million • Up to additional $50 million upon certain
Slide 26: David Meeker, MD: Conclusion
David Meeker, MD Conclusion ® 26
Slide 27: Plans for Continued Execution: Anticipated Upcoming Milestones and Data Readouts
Plans for Continued Execution: Anticipated Upcoming Milestones and Data Readouts Anticipated clinical milestones ✓ Early 2023: Initiate Ph 3 hypothalamic obesity trial o 2H23: Initiate a Ph 3 weekly “de novo’ study o 2H23: Announce preliminary data from Phase 2 DAYBREAK trial in 2H2023 o 2H23: Annou
Slide 28: Rhythm's Strategic Priorities for 2023
Rhythm’s Strategic Priorities for 2023 Execute on Enroll and execute Expand global commercial Phase 3 trial to IMCIVREE strategy evaluate opportunity with setmelanotide in and clinical BBS launches hypothalamic development obesity pipeline with Xinvento assets ® 28
Slide 29: Questions
Questions Rhython
Rhythm Pharmaceuticals — Q4 2022 Earnings Presentation on 6ix