Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q1 2022 Earnings Presentation
28 slides from Rhythm Pharmaceuticals's earnings presentation, May 3, 2022
Slide 1: First Quarter 2022 Financial Results and Business Update
Rhythm Pharmaceuticals First Quarter 2022 Financial Results and Business Update May 3, 2022 ® © Rhythm® Pharmaceuticals, Inc. All rights reserved.
Slide 2: 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 3: On Today's Call
On Today’s Call David Meeker, MD, Chair, President and Chief Executive Officer Jennifer Chien, Executive Vice President, Head of North America Linda Shapiro Manning, MD, PhD, Chief Medical Officer Hunter Smith, Chief Financial Officer Yann Mazabraud, Executive Vice President, Head of International ®
Slide 4: David Meeker, MD
David Meeker, M Rhythar
Slide 5: MC4R Pathway Biology is Clear and Strong
MC4R Pathway Biology is Clear and Strong: Regulates Hunger, Caloric Intake, Energy Expenditure and, Consequently, Body Weight Setmelanotide can redress MC4R pathway impairment contributing to early-onset, severe obesity POMC NEURON MC4R-EXPRESSING ALMS1 NEURON Leptin BBSx LEPR RAI1 SH2B1 MC4R Appe
Slide 6: On Track for U.S. BBS Launch, International Market Access and Expansion
On Track for U.S. BBS Launch, International Market Access and Expansion of Addressable Patient Populations through Clinical Development U.S. Commercial Achieving Market Access Clinical Development Progress in Key International Advancing • Ready for Bardet-Biedl syndrome Geographies • EMANATE, DAYBRE
Slide 7: First Commercial Sales Achieved in France with Progress in Germany, UK
First Commercial Sales Achieved in France with Progress in Germany, UK and Additional Key Markets United Kingdom • First commercial patients started in • NICE recommendation expected June March under paid early access program Italy • Access achieved approximately one • Final stages of price negotiat
Slide 8: Continued Progress in EMANATE and DAYBREAK Trials
Continued Progress in EMANATE and DAYBREAK Trials to Drive Expansion of Setmelanotide’s Potential Addressable Market Phase 3 EMANATE Trial Four independent sub-studies 6,000† Heterozygous POMC/PCSK1 insufficiency 4,000† Heterozygous LEPR insufficiency 20,000† SRC1 insufficiency 23,000† SH2B1 insuffi
Slide 9: Commercial Readiness for BBS and Alström Syndrome U.S. Launch
Jennifer Chien Commercial Readiness for BBS and Alström Syndrome U.S. Launch ® 9
Slide 10: Roadmap to Identifying Patients with BBS
Roadmap to Identifying Patients with BBS Undiagnosed U.S. prevalence estimated to be >350 1,500 to 2,500 Identified and patients* Suspected diagnosed patients** Diagnosed patients Identified and diagnosed * Company estimate based on published prevalence estimates of one ** As announced on Feb. 16, 2
Slide 11: Pre-launch HCP Focus
Pre-launch HCP Focus ENGAGE SUPPORT DIAGNOSIS Uncovering Rare 2 Obesity genetic tests follow up 1 HCPs with BBS patients and support of the community 3 Machine learning HCP targets Priority targets to prepare for launch ® 11
Slide 12: Supporting the Build of BBS Care Networks
Supporting the Build of BBS Care Networks Pediatric endocrinologist / endocrinologist Retinal Pediatric nephrologist / specialist nephrologist Obesity Medical specialist geneticist ® 12
Slide 13: Rhythm InTune Support Services
Rhythm InTune Support Services Personalized program for patients and health care providers, removes barriers and supports adherence ✓Gaining access and coverage ✓Ongoing support and education ✓Building the community Introduce Set Injection tips Goal Treatment Adherence Discuss therapy IMCIVREE expec
Slide 14: Commercial Team Ready to Launch in BBS on Day One
Commercial Team Ready to Launch in BBS on Day One DAY 1 Direct outreach to priority physicians Rhythm InTune: 1:1 outreach to consented BBS caregivers and patients Marketing outreach to and programs for health care providers, patients and caregivers Outreach with patient advocacy organizations ® 14
Slide 15: Regulatory, Clinical & Medical Update
Linda Shapiro, MD, PhD Regulatory, Clinical & Medical Update ® 15
Slide 16: BBS Regulatory Reviews Progressing in US and Europe
BBS Regulatory Reviews Progressing in US and Europe EU EMA FDA PDUFA CHMP opinion in 2H2022 June 16, 2022 CHMP Positive Opinion for patients with renal impairment announced April 22, 2022* *The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended the European
Slide 17: Multiple Ongoing Clinical Trials Evaluating Setmelanotide
Multiple Ongoing Clinical Trials Evaluating Setmelanotide Hypothalamic Obesity Phase 2 Trial Phase 3 Trial Phase 2 Trial Weekly Formulation Pediatrics Trial Phase 3 Phase 3 Switch Trial Patients aged 2 to <6 years ® 17
Slide 18: Phase 3 EMANATE Trial to Evaluate Setmelanotide Across Four Rare Genetic Subtypes
Phase 3 EMANATE Trial to Evaluate Setmelanotide Across Four Rare Genetic Subtypes with Highest Likelihood for Success Targeted patient populations: Patients with pathogenic, likely pathogenic or suspected pathogenic variants • Genetic variants with highest likelihood of impairing MC4R pathway functi
Slide 19: Phase 3 EMANATE Trial Comprised of Four Independent Sub-studies
Phase 3 EMANATE Trial Comprised of Four Independent Sub-studies Design allows for independent data readouts in each sub-study and potential registration for each gene • Pathogenic POMC/ 86 • Likely pathogenic a. • VUS*-Suspected Each sub-study: Patients randomized 1:1 PCSK1* patients pathogenic • Pa
Slide 20: Phase 2 DAYBREAK Trial to Evaluate Setmelanotide Across 10 Genes
Phase 2 DAYBREAK Trial to Evaluate Setmelanotide Across 10 Genes Relevance to MC4R Pathway: Rhythm’s ClinGen- based framework suggests all 10 genes have very strong relevance to MC4R pathway Efficient, two-stage trial design • Open-label run-in allows for fast signal-seeking in individual gene cohor
Slide 21: Hypothalamic Obesity: Acute Onset of Hyperphagia, Weight Gain
Hypothalamic Obesity: Acute Onset of Hyperphagia, Weight Gain and Other Endocrine-related Sequelae from Injury to Hypothalamus Rapid onset of acute, severe obesity and hyperphagia post treatment FDA Patient Listening Session on Hypothalamic Obesity Most commonly caused by craniopharyngioma, a type o
Slide 22: Phase 2 Open-label Trial in Patients with Hypothalamic Obesity
Phase 2 Open-label Trial Designed to Evaluate Setmelanotide’s Therapeutic Effect in Patients with Hypothalamic Obesity Enrollment completed in February 2022; Interim data expected mid-year PATIENTS 6-16 YEARS OLD 1 mg 2 mg 3 mg setmelanotide Primary Screening PATIENTS 16- 40 YEARS OLD efficacy Open-
Slide 23: Rhythm Delivers Multiple Presentations at Major Medical Meetings
Rhythm Delivers Multiple Presentations at Major Medical Meetings Pediatric Endocrine Society (PES) Annual Meeting 2022, April 28-May 1: Presentation Title Presenting Author Patient- and Caregiver-Reported Experiences of Hyperphagia in Bardet-Biedl Syndrome Before and During Claire Ervin 17 Setmelano
Slide 24: Hunter Smith 1Q 2022 Financial Results
Hunter Smith 1Q 2022 Financial Results ® 24
Slide 25: 1Q2022 Financial Snapshot
1Q2022 Financial Snapshot ($ in millions except as noted, per share data Three months ended Three months ended and shares outstanding) March 31, 2022 March 31, 2021 Product revenue, net $1.5M $0.035M R&D expenses $32.5M $19.9M SG & A expenses $21.4M $15.5M Net income / (loss) $(52.8)M $43.8M* Shares
Slide 26: Conclusion
David Meeker, MD Conclusion ® 26
Slide 27: Continued Transformational Progress in 2022
Continued Transformational Progress in 2022 Recently achieved milestones Anticipated milestones in 2022 ✓ Initiated Phase 2 DAYBREAK trial 1H: PDUFA for BBS and Alström June 16, 2022 ✓ Initiated Phase 3 “switch study” of 1H: Long-term data in BBS; SRC1; SH2B1, heterozygous weekly formulation and bia
Slide 28: Questions
Questions Rhythar
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