Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Corporate Update (November 2020)
20 slides from Rhythm Pharmaceuticals's corporate update, Nov 27, 2020
Slide 1: Rhythm Pharmaceuticals
Rhythm Pharmaceuticals Targeting MC4 receptor pathway and transforming the care of patients with rare genetic diseases of obesity November 27, 2020 US-SET-2000084 11/2020 ® © 2020, Rhythm Pharmaceuticals, Inc. All Rights Reserved. Rhythm, IMCIVREE and their corresponding logos are trademarks of Rhyt
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: Agenda
• David Meeker, MD Chairman, CEO and President • Murray Stewart, MD Agenda Chief Medical Officer • Jennifer Chien EVP, Head of North America • Q&A ® 3
Slide 4: David Meeker, MD
David Meeker, MD Chairman, CEO and President ® 4
Slide 5: Working Toward Changing the Paradigm for the Treatment of Rare Genetic Diseases of Obesity
Working Toward Changing the Paradigm for the Treatment of Rare Genetic Diseases of Obesity Validation Meaningful Growth Opportunity Potential FDA approved Deliver Bardet-Biedl and Establish proof-of-concept for chronic weight Alström syndromes in new indications in management for obesity due pivotal
Slide 6: Now Approved in the United States
Now Approved in the United States ® 6
Slide 7: Murray Stewart, MD
Murray Stewart, MD Chief Medical Officer ® 7
Slide 8: MC4R Pathway Regulates Hunger, Caloric Intake, and Energy Expenditure, and, Consequently, Body Weight
MC4R Pathway Regulates Hunger, Caloric Intake, and Energy Expenditure, and, Consequently, Body Weight When signaling cascade is impaired, setmelanotide may restore function by replacing MSH stimulating hormone POMC NEURON MC4R-EXPRESSING NEURON Leptin LEPR MC4R Appetite Satiety Signals ACTIVATION
Slide 9: Approval of IMCIVREE Based on Phase 3 Data from Largest Studies Conducted in Obesity
Approval of IMCIVREE Based on Phase 3 Data from Largest Studies Conducted in Obesity due to POMC, PCSK1 or LEPR Deficiency POMC/PCSK1 LEPR 0% 80% of participants achieved ≥10% weight loss (95% CI, 44.39% to 97.48%); P=0.0002; n=10 PRIMARY ENDPOINT 0% 45.5% of participants achieved ≥10% weight loss (
Slide 10: IMCIVREE Reduced Hunger Over 1 Year in Phase 3 Trials
IMCIVREE Reduced Hunger Over 1 Year in Phase 3 Trials Study 1 Hunger scores generally Worst hunger in last 24 hours* (POMC/PCSK1) Study 2 (LEPR) (N=8) (N=11**) worsened during the 7.9 7.0 double-blind, placebo Baseline Median withdrawal period among Min, Max 7, 9 5, 8 those patients who had 1 year M
Slide 11: Overview of U.S. Prescribing Information for IMCIVREE
Overview of U.S. Prescribing Information for IMCIVREE Indications and Usage IMCIVREE is a melanocortin 4 (MC4) receptor agonist indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subti
Slide 12: Overview of U.S. Prescribing Information for IMCIVREE
Overview of U.S. Prescribing Information for IMCIVREE Dosage in Adults and Pediatric Patients 12 Years of Age and Older • The starting dose of IMCIVREE is 2 mg (0.2 mL) injected subcutaneously once daily for 2 weeks. Monitor patients for gastrointestinal (GI) adverse reactions. • If the starting dos
Slide 13: Adverse Events Reported in Open-Label Trials of Setmelanotide for Obesity due to POMC/PCSK1 or LEPR Deficiency
Adverse Events Reported in Open-Label Trials of Setmelanotide for Obesity due to POMC/PCSK1 or LEPR Deficiency Adverse events occurring ≥20% of patients treated with setmelanotide in open-label clinical studies of 52-week duration Adverse event Setmelanotide-treated patients (N=27) Injection site re
Slide 14: Jennifer Chien
Jennifer Chien EVP, Head of North America ® 14
Slide 15: Unmet Needs may be Addressed with Effective Therapy
Unmet Needs may be Addressed with Effective Therapy Patient, caregiver interviews on challenges of obesity due to POMC or LEPR deficiency without treatment It became overwhelming “We’ve worked so hard .… planning meals, at trying to manage this limiting access to food, condition. We’ve done instilli
Slide 16: Strategic Imperatives for IMCIVREE's Successful Debut in U.S. Market
Strategic Imperatives for IMCIVREE’s Successful Debut in U.S. Market Ensure positive experience for patients, Establish efficient and scalable model to meet caregivers and prescribing physicians the needs of identified and future patients Education and Genetic testing Access, reimbursement and Posit
Slide 17: David Meeker, MD
David Meeker, MD Chairman, CEO and President ® 17
Slide 18: Rhythm Milestones and Next Steps
Rhythm Milestones and Next Steps Updated data in HET patients FDA approval Initial data in for obesity SRC1 and SH2B1 POMC/ due to deficiency PCSK1 and POMC/ BBS and PCSK1 and AS obesities LEPR Phase Initiation of 3 trials LEPR topline Initiation of meet deficiency Phase 3 obesity due to obesity due
Slide 19: Pharmaceuticals
PHARMACEUTICALS
Slide 20: Rhythm Pipeline Focused on MC4R Pathway Diseases
Rhythm Pipeline Focused on MC4R Pathway Diseases Early-stage Regulatory Disorder development Phase 2 Phase 3 Submission Approved Obesity due to POMC/PCSK1 deficiency** EU U.S. Obesity due to LEPR deficiency** EU U.S. Pivotal Bardet-Biedl syndrome Studies Alström syndrome POMC/PCSK1 or LEPR heterozyg
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