Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q2 2023 Earnings Presentation
29 slides from Rhythm Pharmaceuticals's earnings presentation, Aug 1, 2023
Slide 1: Second Quarter 2023 Financial Results and Business Update
Rhythm Pharmaceuticals Second Quarter 2023 Financial Results and Business Update August 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 Rhythm
Slide 5: High-level Execution on Global Commercialization and Market Access for BBS
High-level Execution on Global Commercialization and Market Access for BBS, Expansion of Overall Opportunity through Clinical Development Strong BBS Hypothalamic obesity Multiple development commercial execution programs progressing programs advancing • Strong growth with $19.2M in • Based on rapid
Slide 6: RM-718: More Selective MC4R Agonist Extending Patent Protection
RM-718: More Selective MC4R Agonist Extending Patent Protection Investigational New Drug (IND) application on track to be filed by end of 2023 MC4R-specific, MC1R-sparing, Weekly formulation potentially designed to with composition more potent eliminate of matter hyperpigmentation patent protection
Slide 7: Rare MC4R Pathway Diseases Combine for Significant Opportunity
Rare MC4R Pathway Diseases Combine for Significant Opportunity Approved for:€ Bardet-Biedl POMC, PCSK1 and LEPR syndrome deficiencies 4,000 - 5,000 600 – 2,500 patients in the U.S.* patients in the U.S.* Phase 3 Trial Initiated Hypothalamic obesity 5,000 – 10,000 patients in the U.S.* Phase 3 EMANAT
Slide 8: Accelerating Guidance: Enrollment Completion by End of 2023 in Ph3 Hypothalamic Obesity Trial
Accelerating Guidance: Now Expect to Complete Enrollment by the end of 2023 in Ph3 Hypothalamic Obesity Trial 25% patients screened as of June 30 1/3 Starting dose for all patients is 0.5mg QD; Maximum dose for patients<6yo is between 1.5mg QD and 3.0mg QD based on body of planned sites weight; maxi
Slide 9: Hypothalamic Obesity: LTE Data Demonstrate Sustained, Deepening BMI Reduction
Hypothalamic Obesity: LTE Data Demonstrate Sustained, Deepening BMI Reduction over Six Months * 5 0 BMI % change from index trial baseline -5 -10 -15 -20 -25 -16.8% -21% mean percent reduction mean percent reduction -30 in BMI at week 16 in BMI at month 6 from baseline from baseline -35 Week 16 Mont
Slide 10: Progression in BMI Reduction Consistent Across All Patients in LTE
Progression in BMI Reduction Consistent Across All Patients in LTE Mean BMI reduction of 21.0% from baseline observed in 13 patients at six months, showing progression from 16.8% mean BMI reduction at 16 weeks Error bars are the standard deviation. *One pediatric patient did not have a Month-6 visit
Slide 11: Ongoing Programs Designed to Achieve Label and Pipeline Expansion
Ongoing Programs Designed to Achieve Label and Pipeline Expansion Patient Population Pre-clinical Phase 1/2 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 LEPR
Slide 12: Jennifer Chien: BBS U.S. Launch
Jennifer Chien BBS U.S. Launch ® 12
Slide 13: Hyperphagia and Early-onset Obesity Have a Significant Impact on Patients
Hyperphagia and Early-onset Obesity Have a Significant Impact on Patients with MC4R Pathway Diseases and their Families IMCIVREE Patient Ambassador program launched with 8 patient/caregiver speakers “I was hungry all day long. I even started sneaking food in the middle of the night because my mind w
Slide 14: Strong U.S. Demand Continues throughout First Full Year of BBS Launch
Strong U.S. Demand Continues throughout First Full Year of BBS Launch Cumulative New BBS Prescriptions since Launch >425* > 425 New BBS prescriptions launch to date > 300 * >125 2Q 2023 received during > 200 > 120 >250 * reimbursement approvals > 50 >250* unique 2Q 2022 3Q 2022 4Q 2022 1Q 2023 2Q 20
Slide 15: BBS IMCIVREE Prescribers by Specialty Since Launch
BBS IMCIVREE Prescribers by Specialty Since Launch 11% Endocrinology, 34% >250* Pediatric Prescribers 20% Endocrinology as of 6/30/2023 Primary Care 26% Of prescribers ‘new to Rhythm’ 16% 18% of prescribers have All others Pediatrics >25% written more than one prescription *As of June 30, 2023 ® 15
Slide 16: Improved Medicaid Reimbursement with ~80% of Covered Lives in Positive Coverage States
Improved Medicaid Reimbursement now with ~80% of Covered Lives in States with Positive Coverage Policies, Decisions for IMCIVREE Medicaid Covered Lives ~85 million* ~80%** ~20% of covered lives split between of covered lives in states with: states with: • no IMCIVREE prescription • an IMCIVREE polic
Slide 17: Snapshot of BBS Patients with Prescriptions, Duration on Therapy
Snapshot of BBS Patients with Prescriptions, Duration on Therapy Since Re-authorizations Age Range Launch Adult (18+) ~54% 50 re-authorization Adolescent ~25% approvals to date (12-17) (at 3-, 6- or 12-months) Pediatric ~21% (6-11) Majority of plans have 12-month re-authorization timeframes *As of J
Slide 18: Patient Identification, Physician Engagement and Community Building
Patient Identification, Physician Engagement and Community Building Patient Identification Physician Engagement Community Building • Uncovering Rare Obesity • Increased depth and • Rhythm InTune genetic testing breadth of prescribers • Patient advocacy • Non-personal promotion with direct engagement
Slide 19: Yann Mazabraud: EVP, Head of International
Yann Mazabraud EVP, Head of International ® 19
Slide 20: Important Market for IMCIVREE for BBS, POMC and LEPR Deficiencies
Important Market for IMCIVREE for BBS, POMC and LEPR Deficiencies POMC, PCSK1 and LEPR Bardet-Biedl Deficiency Obesities Syndrome Estimated European prevalence Estimated European prevalence 600 - 2,500 4,000 – 5,000 ~100 >1,500 individuals identified individuals identified in EU4 + UK in EU4 + UK (~
Slide 21: IMCIVREE to be Available in More than Ten Countries Outside United States in 2023
IMCIVREE for Biallelic POMC/LEPR and/or BBS to be Available in More than Ten Countries Outside United States in 2023 United Kingdom Italy Netherlands Spain France BBS pricing Reimbursed early- NICE BBS discussions ‘Innovation’ rating Ongoing PPL & BBS from AIFA; ongoing negotiations ongoing, access
Slide 22: Strong Start for IMCIVREE Launch for BBS in Germany
Strong Start for IMCIVREE Launch for BBS in Germany Leveraging KOL engagement, BBS patient advocacy and patient support programs Engaging care centers, large hospitals Rhythm@Home ~1,200 leads to prescriptions Estimated German prevalence* ~800 Patients diagnosed with BBS* Aach en >250 Patient suppor
Slide 23: Hunter Smith: 2Q 2023 Financial Results
Hunter Smith 2Q 2023 Financial Results ® 23
Slide 24: 2Q 2023 Financial Snapshot
2Q 2023 Financial Snapshot ($ in millions, except per share data and shares Three months ended Three months ended outstanding) June 30, 2023 June 30, 2022 Product revenue, net $19.2M $2.3M R&D expenses $33.5M $31.5M SG&A expenses $30.0M $22.3M Net Loss $(46.7)M $(45.0)M Shares outstanding (basic and
Slide 25: 2Q 2023 Financial Highlights
2Q 2023 Financial Highlights $278.0M ~86% 2Q 2023 Non-GAAP pro forma cash, cash OpEx includes OpEx Guidance of 2Q 2023 revenue equivalents and short- $8.9M for 2023: from U.S. sales term investments as of of IMCIVREE in stock-based $200M to June 30, 2023, together vs. 83% in compensation $220M* with
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: Announce preliminary
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 new product BBS launches hypothalamic with development obesity assets ® 28
Slide 29: Questions
Questions Rhythm
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