Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Investor Presentation (December 2020)
21 slides from Rhythm Pharmaceuticals's investor presentation, December 2020
Slide 1: Positive Topline Results from Pivotal Phase 3 Trial of Setmelanotide in BBS and Alström Syndromes
Rhythm Pharmaceuticals Positive Topline Results from Pivotal Phase 3 Clinical Trial Evaluating Setmelanotide in Patients with Bardet-Biedl and Alström Syndromes December 2020 ® © 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: Agenda
• David Meeker, MD Chair, Chief Executive Officer and President Agenda • Murray Stewart, MD Chief Medical Officer • Q&A ® 3
Slide 4: David Meeker, M.D.
David Meeker, M.D. Chair, Chief Executive Officer 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 Positive topline Establish proof-of-concept for chronic weight results in in new indications in management for obesity due Bardet-Biedl Phase 2 B
Slide 6: Phase 3 Clinical Trial Met Primary and All Key Secondary Endpoints
Phase 3 Clinical Trial Met Primary and All Key Secondary Endpoints Setmelanotide achieved statistical significance and delivered clinically meaningful weight loss and hunger reduction Phase 3 Topline Data (n=31a) 34.5%b -6.2% -30.8% 60.2% p=0.0024 p<0.0001 p<0.0001 p<0.0001 ≥10% mean mean ≥25% weigh
Slide 7: Murray Stewart, M.D.
Murray Stewart, M.D. Chief Medical Officer ® 7
Slide 8: In Patients With BBS, Cilia Dysfunction May Reduce Downstream Activation of MC4R
In Patients With BBS, Cilia Dysfunction May Reduce Downstream Activation of MC4R, Which Can Lead to Hyperphagia and Obesity1-3 AgRP/NPY AgRP • Loss of functional BBS neuron proteins may disrupt cilia LEPR trafficking to the cell membrane, Leptin MC4R thus impairing its MC4R-expressing Hunger signali
Slide 9: Bardet-Biedl and Alström Syndromes Phase 3 Trial Design
Bardet-Biedl and Alström Syndromes Phase 3 Trial Design Double-blind treatment period Open-label treatment Pivotal cohort Setmelanotide Setmelanotide 3 mg 3 mg • 32 BBS patients Screening and Long-term • 6 Alström syndrome 1:1 randomization Placebo Setmelanotide extension 3 mg patients 14 weeks Week
Slide 10: Baseline Characteristics and Demographics in Evaluable Patients
Baseline Characteristics and Demographics in Evaluable Patients Baseline Characteristics (N=31) Diagnosis, n (%) BBS 28 (90) Alström syndrome 3 (10) Age, mean (range), years 21 (12-44)b Overall age, mean (range) years 14 (45) Adolescents (12-17 years old), n (%) Male, n (%) 14 (45) 117 (62-192) / We
Slide 11: Primary Endpoint Met: Statistically Significant and Clinically Meaningful Weight Reductions at ~1 Year
Primary Endpoint Met: Setmelanotide Achieved Statistically Significant and Clinically Meaningful Weight Reductions at ~1 Year on Therapy 34.5%a of participants (95% CI, 17.5 to 51.6; P=0.0024) achieved the primary endpoint threshold of ≥10% weight loss from baseline at Week 52 ~1 year at target Perc
Slide 12: Statistically Significant Reduction in "Most" Hunger Score at ~1 Year on Therapy
Setmelanotide Achieved Statistically Significant Reduction in “Most” Hunger Score at ~1 Year on Therapy 60.2%a of participants (95% CI, 35.3 to 85.1 P<0.0001) achieved ≥25% reduction from baseline in daily hunger score at Week 52 ~1 year at target Percent change Baseline dose from baseline “Most” hu
Slide 13: A Closer Look at Patients with Alström Syndrome
A Closer Look at Patients with Alström Syndrome 3 Evaluable patients with Alström 2 Patients < 12 years old not syndrome ≥12 years old included in primary endpoint • 1 discontinued • 1 patient lost approximately 8% body weight • 2 gained weight (mean change was +8.9 kg) • 1 patient discontinued Hung
Slide 14: A Closer Look at Patients with Bardet-Biedl Syndrome
A Closer Look at Patients with Bardet-Biedl Syndrome 28 patients with 11 BBS 5 BBS BBS ≥12 years old in patients achieved patients (3 adults, primary analysis primary endpoint of 2 adolescents) • 13 adolescent patients ≥10% weight loss: had 5-9% weight loss < 18 • Mean actual weight loss: reduction
Slide 15: Setmelanotide Was Generally Well Tolerated in Individuals With BBS or Alström Syndrome
Setmelanotide Was Generally Well Tolerated in Individuals With BBS or Alström Syndrome • Treatment-emergent adverse events (TEAEs) Parameter n (%) included mild injection site reactions and nausea with infrequent vomiting Any treatment-emergent adverse 38 (100) • There was one serious TEAE considere
Slide 16: David Meeker, M.D.
David Meeker, M.D. Chair, Chief Executive Officer and President ® 16
Slide 17: Early-onset, Severe Obesity and Hunger Prominent Among Clinical Characteristics of BBS and Alström Syndrome
Early-onset, Severe Obesity and Hunger Prominent Among Clinical Characteristics of BBS and Alström Syndrome Bardet-Biedl syndrome1 Hyperphagia Rare ciliopathy disease resulting from Cognitive impairment genetic variants within BBS family of Progressive vision genes impairment/loss Early-onset, U.S.
Slide 18: Hunger and Obesity Pose a Major Burden to Families and Physicians with No Approved Therapies
Hunger and Obesity Post a Major Burden to Families and Physicians with No Approved Therapies “They are constantly, all day long saying they are hungry “Greatest frustration is lack of efficacious and asking what’s for the next treatment for hyperphagia and the meal and what are we eating obesity…” t
Slide 19: Setmelanotide's Path Forward for Bardet-Biedl and Alström Syndromes
Setmelanotide’s Path Forward for Bardet-Biedl and Alström Syndromes Further analysis of data Analyses on Data from 14-week 1H21 Additional specific to BBS and BMI and BMI-Z placebo-controlled Analyses to Come Alström syndrome scores period Approved for chronic 2H21 Supplemental weight management for
Slide 20: 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 Positive topline Establish proof-of-concept for chronic weight results in in new indications in management for obesity due Bardet-Biedl Phase 2 B
Slide 21: Rhythm Pharmaceuticals
PHARMACEUTICALS
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