Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q3 2021 Earnings Presentation
38 slides from Rhythm Pharmaceuticals's earnings presentation, Nov 2, 2021
Slide 1: Third Quarter 2021 Financial Results
Rhythm Pharmaceuticals Third Quarter 2021 Financial Results November 2, 2021 ® © Rhythm® Pharmaceuticals, Inc. All rights reserved.
Slide 2: On Today's Call
On Today’s Call David Meeker, MD, Chair, President and Chief Executive Linda Shapiro Manning, MD, PhD, Chief Medical Officer Jennifer Chien, EVP, Head of North America Hunter Smith, Chief Financial Officer ® 2
Slide 3: Forward Looking Statements
Forward Looking Statements 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 witho
Slide 4: David Meeker, MD
David Meeker, M Rhython
Slide 5: Build Strategy Focused on Maximizing Patient Access to Setmelanotide
Build Strategy Focused on Maximizing Patient Access to Setmelanotide U.S. IMCIVREE Strong Market access, Clinical development commercial foundation for reimbursement expansion availability Bardet-Biedl advancing in key meaningfully meeting commercial international increases expectations opportunity
Slide 6: Multiple Highlights and Continued Execution in Third Quarter of 2021
Multiple Highlights and Continued Execution in Third Quarter of 2021 U.S. IMCIVREE commercial Advancing towards BBS U.S. launch availability meeting expectations in mid-2022 • 3Q21 net sales > $1M • Strong regulatory submissions filed in both U.S. and • Continued progress in securing access, EU reim
Slide 7: Community Building Strategy Focuses on Building Referral Networks for Rare Genetic Diseases of Obesity
Community Building Strategy Focuses on Building Referral Networks for Rare Genetic Diseases of Obesity Referral site Referral site Centers of Excellence Trial Sites Referral site Referral site Key Opinion Leaders and HCPs who diagnose and treat ® 7
Slide 8: Uncovering Rare Obesity Shows that 54% of Sequenced Individuals Carry Variants
Uncovering Rare Obesity® Shows that 54% of Sequenced Individuals with Severe Obesity Carry Variants in Genes with Known Relevance to MC4R Pathway Patients with variants potentially supportive of a diagnosis but not eligible for 9.9% setmelanotide treatment Patients with variants who may qualify for
Slide 9: Clinical Programs Designed to Achieve Label Expansion on Track
Clinical Programs Designed to Achieve Label Expansion on Track Phase 2 Phase 3 Registered Bardet-Biedl and U.S. and EU submissions complete Alström syndromes Setmelanotide EMANATE Trial Five independent sub-studies (daily) Pediatrics Trial 2- to 6-years old Exploratory Basket Study MC4R variants Hyp
Slide 10: Linda Shapiro, MD, PhD: Regulatory and Clinical Update
Linda Shapiro, MD, PhD Regulatory and Clinical Update ® 10
Slide 11: Update on Bardet-Biedl syndrome
Update on Bardet-Biedl syndrome ® 11
Slide 12: Setmelanotide is Poised to be First-ever Therapy to Address Unmet Needs of Hyperphagia and Obesity in BBS
Setmelanotide is Poised to be First-ever Therapy to Address Unmet Needs of Hyperphagia and Obesity in Patients with BBS 1 2 3 Severe obesity, Additional hyperphagia from an U.S. and EU compelling data early age affect the regulatory about the impact of health and quality of submissions setmelanotide
Slide 13: Hyperphagia's Severe Impact on Lives of Patients with BBS and their Caregivers
Hyperphagia’s Severe Impact on Lives of Patients with BBS and their Caregivers …I constantly felt like I was failing…. because She couldn’t do as well in school if I did not give her extra food, then I felt because she was thinking about terrible for denying her that when I knew she I was eating pre
Slide 14: U.S. and EU Regulatory Submissions for BBS and Alström Syndrome Recently Completed
U.S. and EU Regulatory Submissions for BBS and Alström Syndrome Recently Completed Pivotal Phase 3 trial met all primary and key secondary endpoints* sNDA Submitted to Type II Variation U.S. FDA Application Submitted to in September EMA in October U.S projected commercial launch mid-2022 *All patien
Slide 15: Phase 3 Trial to Evaluate Setmelanotide Therapy for 52 Weeks in Patients With BBS
Phase 3 Trial to Evaluate Setmelanotide Therapy for 52 Weeks with 14- week Placebo-controlled Period in Patients With BBS Double-blind Open-label treatment treatment period 52-week treatment periods included in analysisd Setmelanotide Setmelanotideb 3 mga 3 mga Screening and Included 1:1 randomizati
Slide 16: Setmelanotide Led to Significant BMI Reduction in Patients with BBS Versus Placebo at Week 14
Setmelanotide Led to Significant BMI Reduction in Patients with BBS Versus Placebo at Week 14 Percent BMI Change From Baseline 14-week PBO data Setmelanotide Placebo N=16 N=16 1 Patients with BBS treated with -1 setmelanotide achieved an average Average change in BMI, % BMI reduction of −1.5 kg/m2 -
Slide 17: Setmelanotide Achieved Clinically Significant Reduction in BMI at Week 52
Setmelanotide Achieved Clinically Significant Reduction in BMI in Patients with BBS in Phase 3 Trial at Week 52 −9.1% mean % change in BMI in patients ≥18 years old −9.5% mean % change in BMI in patients <18 years old 52 weeks on active Percent change from Baseline treatment start of active treatmen
Slide 18: Setmelanotide Achieved Clinically Meaningful Reduction in Hunger at Week 52
Setmelanotide Achieved Clinically Meaningful Reduction in Hunger in Adults and Children with BBS at Week 52 62.5% Data show separation in hunger reduction during placebo period followed by placebo group reaching treatment levels rapidly after crossover (95% CI, 35.4%–84.8%; P<0.0001) of participants
Slide 19: Setmelanotide Achieved Clinically Meaningful Improvements in Health-related Quality of Life
Setmelanotide Achieved Clinically Meaningful Improvements in Health-related Quality of Life (HRQOL) Impact of Setmelanotide on HRQOL 85% of patients reported Adults (>18 years Children (8-17 old) years old) clinically meaningful Patients, n 11 9 improvements IWQOL-Lite PedsQL or preserved non-impair
Slide 20: Clinical development programs on track to expand patient population
Clinical development programs on track to expand patient population ® 20
Slide 21: Setmelanotide Clinical Development Programs on Track to Expand Patient Populations
Setmelanotide Clinical Development Programs on Track to Expand Patient Populations with Impaired MC4R Pathway 1 2 3 Patients with Growing body of EMANATE and heterozygous variants evidence that supports DAYBREAK trials start- in POMC, PCSK1 and setmelanotide’s up activities underway; LEPR associated
Slide 22: Similar Severe Phenotype Associated with Heterozygous and Homozygous Variants in LEPR, POMC, and PCSK1
Similar Severe Phenotype Associated with both Heterozygous and Homozygous Variants in LEPR, POMC, and PCSK1 In new research, patients with heterozygous variants characterized by severe and early-onset obesity with food impulsivity • Severe phenotype: Comparable BMI between homozygous and heterozygou
Slide 23: Rhythm Delivers Multiple Presentations at Major Medical Conferences
Rhythm Delivers Multiple Presentations at Major Medical Conferences ESPE 2021: New setmelanotide Phase 2 Basket study data show at three months: • 30% (9 of 30) of patients with obesity due to variants of the SRC1 gene achieved clinically meaningful weight loss or BMI-Z reduction 22 oral and poster
Slide 24: EMANATE and DAYBREAK Trials Start-up Activities Underway
EMANATE and DAYBREAK Trials Start-up Activities Underway Trial sites • 75+ sites identified in 14 countries in North America, Europe and the Middle East • Site initiations ongoing; investigator meetings planned in Referral Site Referral Site November and December • First patients in expected in Q4 2
Slide 25: Jennifer Chien: U.S. Commercial Update
Jennifer Chien U.S. Commercial Update ® 25
Slide 26: IMCIVREE Commercial Sales in U.S. Meeting Expectations
IMCIVREE Commercial Sales in U.S. Meeting Expectations First two quarters of IMCIVREE commercial availability: • Positive coverage decisions, reimbursement and access • Continued focus on HCP engagement • Patient Services and Corporate Accounts teams in place and making a difference INITIAL PATIENT
Slide 27: Bardet-Biedl Community is Established and Identified
Bardet-Biedl Community is Established and Identified U.S. prevalence Estimated European estimated to be prevalence estimated to be 1,500 to 2,500 ~2,500 patients patients More than 600 individuals living More than 1,500 individuals with BBS are enrolled in CRIBBS identified in EU4 + UK at ~20 academ
Slide 28: Experience with POMC, PCSK1 and LEPR Commercial Availability is Foundational to BBS Launch Strategy
Experience with POMC, PCSK1 and LEPR Commercial Availability is Foundational to BBS Launch Strategy S Establish access Treat obesity and hyperphagia Accelerate diagnosis Ensure diagnosed BBS patients Facilitate BBS diagnosis and Establish market access and will be treated for hyperphagia initiate fu
Slide 29: Patient Support Team in Place to Empower Patients through Personalized Engagement
Patient Support Team in Place to Empower Patients through Personalized Engagement Education Treatment Initiation Access Adherence For all patients and families, For patients and providers: Support for patients with Robust, individualized regardless of treatment • Comprehensive access barriers: adher
Slide 30: BBS Territory Managers Now in the Field and Focused on Targeted Physician Engagement
BBS Territory Managers Now in the Field and Focused on Targeted Physician Engagement TERRITORY MANAGER PROFILE: 20 6 100% 6 Average years Average years rare have launch average number of pharma/biotech disease experience experience launches per sales experience territory manager ® 30
Slide 31: New Market Research Highlights Positive Impressions of IMCIVREE Profile
New Market Research Highlights Positive Impressions of IMCIVREE Profile HCPs: • Obesity was universally viewed as a serious concern in individuals w/ BBS, and cited significant challenges with current options for management • Strongest attributes and reasons to prescribe: wide age 75% of respondents
Slide 32: Area Development Managers and Targeted URO Strategy Drives Trial Enrollment and Patient ID
Area Development Managers and Targeted URO Strategy Drives Trial Enrollment and Commercial Patient ID Trial enrollment • Identifying patients for EMANATE and DAYBREAK trials Commercial patients • Genetic identification and confirmation for biallelic POMC, PCSK1 and LEPR Patients with BBS gene varian
Slide 33: Hunter Smith: 3Q 2021 Financial Results
Hunter Smith 3Q 2021 Financial Results ® 33
Slide 34: Q3 2021 Financial Highlights
Q3 2021 Financial Highlights ($ in millions except as noted, per share data and shares Three months ended outstanding) September 30, 2021 Product revenue, net $1.0M R&D expenses $27.5M SG & A expenses $17.5M Net loss $(35.1)M Shares outstanding (basic and diluted share count) 50,246,303 Net loss per
Slide 35: David Meeker, MD: Conclusion
David Meeker, MD Conclusion ® 35
Slide 36: Transformational Progress Expected in 2021 and 2022
Transformational Progress Expected in 2021 and 2022 1H 2021 ✓ Proof-of-concept data in HET patients, SRC1 and SH2B1 deficiency obesities ✓ Update on genetic sequencing and epidemiology data ✓ IMCIVREE commercially available in U.S. for POMC, PCSK1 and LEPR deficiency obesities ✓ Initiate Phase 2 tri
Slide 37: Clinical Development Programs Designed to Expand the Setmelanotide Opportunity
Clinical Development Programs Designed to Expand the Setmelanotide Opportunity Clinical development expansion Phase 3 EMANATE Trial€ Five independent substudies 45,000† Heterozygous POMC/PCSK1 deficiency Potential U.S. launch 25,000† Heterozygous LEPR deficiency in mid-2022£ Now approved 20,000† SRC
Slide 38: Questions
Questions Rhython
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