Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q1 2023 Earnings Presentation
29 slides from Rhythm Pharmaceuticals's earnings presentation, May 9, 2023
Slide 1: First Quarter 2023 Financial Results and Business Update
Rhythm Pharmaceuticals First Quarter 2023 Financial Results and Business Update May 2, 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: Building a Global Company Focused on Rare MC4R Pathway Diseases
Building a Global Company Focused on Rare MC4R Pathway Diseases Strong BBS Phase 3 trial in Meaningful expansion commercial execution hypothalamic obesity opportunity • Strong start to U.S. BBS launch underway • Executing on six active clinical trials continues • First patients enrolled and dosed in
Slide 6: MC4R Pathway Regulates Hyperphagia, Energy Expenditure and Body Weight
MC4R Pathway Regulates Hyperphagia, Energy Expenditure and Body Weight Hyperphagia: Insatiable, pathological hunger that leads to abnormal food-seeking behaviors Hypothalamus Upstream Downstream Downstream AgRP neuron MC4R activity Low Normal LEPR AgRP Adipose Hyperphagia tissue PCSK1 MC4R Obesity M
Slide 7: Rare MC4R Pathway Diseases Combine for Significant Opportunity
Rare MC4R Pathway Diseases Combine for Significant Opportunity Approved in U.S. and Europe Bardet-Biedl syndrome and POMC, PCSK1 and LEPR deficiencies 4,600 – 7,500 patients in the U.S.* Phase 3 Trial Initiated Hypothalamic obesity 5,000 – 10,000 patients in the U.S.* Phase 3 EMANATE Trial Heterozyg
Slide 8: Hypothalamic Obesity: First Patients Dosed in Phase 3 Trial
Hypothalamic Obesity: First Patients Dosed in Phase 3 Trial Trial designed with FDA feedback under Breakthrough Designation; Enrollment expected to be complete in 1Q 2024 Screening and Dose titration Double-blind, placebo-control Randomization Setmelanotide QD (n=80) 120 patients Open-label randomiz
Slide 9: Clinical Programs Designed to Achieve Label and Pipeline Expansion
Clinical Programs Designed to Achieve Label and Pipeline Expansion Product/Program Patient Population Phase 2 Phase 3 Commercially Approved POMC, PCSK1 or LEPR (PPL) deficiency Bardet-Biedl syndrome (BBS) Hypothalamic Obesity Setmelanotide daily formulation Pediatrics (age 2 to <6 years, POMC or LEP
Slide 10: Jennifer Chien: BBS U.S. Launch
Jennifer Chien BBS U.S. Launch ® 10
Slide 11: Strong Demand for IMCIVREE for BBS Continues
Strong Demand for IMCIVREE for BBS Continues * >160* >300 reimbursement New BBS prescriptions approvals >100 >175* received during unique 1Q 2023 prescribers * As of March 31, 2023. IMCIVREE was approved by the U.S. FDA on June 16, 2022. ® 11
Slide 12: BBS IMCIVREE Prescribers by Specialty Since Launch
BBS IMCIVREE Prescribers by Specialty Since Launch 10% >175* 34% 20% Prescribers as of 3/31/2023 27% 16% 73% 20% Pediatric Endocrinology Pediatrics All Others Primary Care Endocrinology TM Led Non-TM Led *As of March 31, 2023 ® 12
Slide 13: Almost 90% of BBS Prescriptions Fall Under Commercial and Medicaid Plans
Almost 90% of BBS Prescriptions Since Launch Fall Under Commercial and Medicaid Plans >160 Reimbursement approvals Commercial Medicaid Federal Medicare *As of March 31, 2023 ® 13
Slide 14: ~75% of Medicaid Covered Lives in States with Positive Coverage
~75% of Medicaid Covered Lives in States with Positive Coverage Policies or Positive Coverage Decisions for IMCIVREE Medicaid Covered Lives = ~85 million ~75% ~25% of covered lives split between states with: of covered lives in states with: • an IMCIVREE policy in place; • no IMCIVREE prescription o
Slide 15: Snapshot of Patient Prescriptions
Snapshot of Patient Prescriptions Age Range Since Launch Adult (18+) ~50% Adolescent (12-17) ~27% Pediatric (6-11) ~23% 97% of BBS prescriptions are written for patients who are consented to InTune *As of March 31, 2023 ® 15
Slide 16: Results of Patient Identification Efforts Support Opportunity for Continued Growth
Results of Patient Identification Efforts Support Opportunity for Continued Growth Successful Patient Identification Efforts Opportunities Ahead • Genetic testing with Uncovering Rare Obesity • Prescribers of IMCIVREE with additional BBS patients • Machine learning using ICD-10 codes • Physicians wh
Slide 17: Yann Mazabraud, EVP, Head of International
Yann Mazabraud EVP, Head of International ® 17
Slide 18: IMCIVREE Approved for BBS in Germany and Eligible for Full Reimbursement
IMCIVREE Approved for BBS in Germany and Made Eligible for Full Reimbursement by Federal Joint Committee (G-BA) ® 18
Slide 19: IMCIVREE Launched for BBS in Germany with Experienced Team
IMCIVREE Launched for BBS in Germany with Experienced Team Setmelanotide has strong legacy in Germany Strong team in place engaging with physicians and centers of excellence • German General Manager has led multiple launches • 6 people dedicated to IMCIVREE launch • Genetic confirmation • ~50% of pa
Slide 20: Significant Opportunity for IMCIVREE and BBS in Germany
Significant Opportunity for IMCIVREE and BBS in Germany Team engaging with BBS sites and focused on patient identification 18 ~1,200 Estimated German prevalence* BBS sites Patients diagnosed Aachen ~800 with BBS* Ulm >250 Patients with BBS identified* *Internal company estimates. ® 20
Slide 21: Europe is an Important Market for BBS, POMC and LEPR Deficiencies
Europe is an Important Market for BBS, POMC and LEPR Deficiencies IMCIVREE available in eight ex-U.S. countries through full reimbursement, named patients sales or early access programs POMC, PCSK1 and LEPR Bardet-Biedl Deficiency Obesities Syndrome ~100 >1,500 individuals identified individuals ide
Slide 22: First International Webinar on Genetic Causes of Obesity
First International Webinar on Genetic Causes of Obesity Draws >125 Healthcare Providers from 17 Countries Angela Scudder Mother of a person living with BBS; Staff member at BBS UK and Patient Liaison Officer at BBS UK Clinics Philip Beales, M.D., Ph.D. University College London, UCL Great Ormond St
Slide 23: Hunter Smith: 1Q 2023 Financial Results
Hunter Smith 1Q 2023 Financial Results ® 23
Slide 24: 1Q 2023 Financial Snapshot
1Q 2023 Financial Snapshot ($ in millions, except per share data and shares Three months ended Three months ended outstanding) March 31, 2023 March 31, 2022 Product revenue, net $11.5M $1.5M R&D expenses $37.9M $32.5M SG&A expenses $24.6M $21.4M Net Loss $(52.2)M $(52.8)M Shares outstanding (basic a
Slide 25: 1Q 2023 Financial Highlights
1Q 2023 Financial Highlights ~83% 1Q 2023 1Q 2023 Non-GAAP of 1Q 23023 revenue R&D costs include OpEx includes OpEx Guidance from U.S. sales $5.4M $6.4M for 2023: of IMCIVREE associated with in stock-based $200M to vs. 85% in Xinvento acquisition compensation $220M 4Q 2022 vs. $5.3M in 4Q 2022 $294.
Slide 26: David Meeker, MD: Conclusion
David Meeker, MD Conclusion ® 26
Slide 27: 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 2H2023: Present data analyses from Ph2 and LTE trials in hypothalamic obesity at medical conferences o 2H23: Initiate a Ph 3 weekl
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
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