Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — ESPE 2021
14 slides from Rhythm Pharmaceuticals's conference presentation, September 2021
Slide 1: Phase 3 Trial of Setmelanotide in Participants With Bardet-Biedl Syndrome: Placebo-Controlled Results
Phase 3 Trial of Setmelanotide in Participants With Bardet-Biedl Syndrome: Placebo-Controlled Results Jesús Argente,1,2 Karine Clément,3,4 Hélène Dollfus,5 Joan C. Han,6 Andrea M. Haqq,7 Gabriel Á. Martos-Moreno,1 Robert S. Mittleman,8 Murray Stewart,8 Matt Webster,8 Jack A. Yanovski,9 Guojun Yuan,8
Slide 2: ESPE 2021 Online Conflict of Interest
ESPE 2021 Online Conflict of Interest Name: Jesús Argente I have the following potential conflicts of interest to report: Research contracts Consulting Employment in the industry Stockholder of a healthcare company Owner of a healthcare company X Other(s) – Payment for lectures and parti
Slide 3: Disclosures
Disclosures This study was sponsored by Rhythm Pharmaceuticals, Inc. ESPE 2021 ● September 22-26, 2021 3
Slide 4: Energy Regulation in Bardet-Biedl Syndrome
Energy Regulation in Bardet-Biedl Syndrome • Bardet-Biedl syndrome (BBS) is a rare autosomal Mechanism of energy balance regulation by different pleiotropic and multigenic syndrome involving POMC neuron populations6 primary cilia dysfunction1-3 • Individuals with variants in BBS-associated genes POM
Slide 5: Setmelanotide Treatment Demonstrates Weight and Hunger Benefits After 1 Year in Patients With BBS
Setmelanotide Treatment Demonstrates Weight and Hunger Benefits After 1 Year in Patients With BBS Body Weight and BMI Z Score Change From Baseline at Week 52 With Setmelanotide: Phase 3 Trial of Individuals With BBS1 • In a preliminary analysis of a Phase 3 trial in Age group Metric Absolute (percen
Slide 6: Phase 3 Trial to Evaluate Setmelanotide in Individuals With BBS
Phase 3 Trial to Evaluate Setmelanotide in Individuals With BBS Double-blind treatment period Open-label treatment Primary analysis End of study Treatment periods included in Setmelanotide Setmelanotideb primary analysis 3 mga 3 mga Screening and Included 1:1 randomization Placebo Setmelanotidec Not
Slide 7: Phase 3 Trial Endpoints
Phase 3 Trial Endpoints Primary endpoint Key secondary endpoints Additional secondary endpoints Week 52 Week 52 Week 14a • Proportion of all participants achieving ≥10% weight loss • Mean percent change from baseline • Mean percent change in body in body weight in participants ≥12 weight with setmel
Slide 8: Disposition and Baseline Demographics of Patients With BBS
Disposition and Baseline Demographics of Patients With BBS Enrolled N=32 Setmelanotide Placebo Total Baseline characteristics (N=16) (N=16) (N=32) Age, years Mean (SD) 19.1 (9.8) 21.2 (10.7) 20.2 (10.2) Range 7–42 10–44 7–44 Treated Age group, n (%) N=32 ≥12 years 14 (87.5) 15 (93.8) 29 (90.6) Disco
Slide 9: Setmelanotide Led to Significant Weight Loss Compared With Placebo in Participants Age ≥12 Years With BBS
Setmelanotide Led to Significant Weight Loss Compared With Placebo in Participants Age ≥12 Years With BBS Percent Weight Change From Baseline At Week 14, participants with Setmelanotide Placebo BBS age ≥12 years treated N=14 N=15 with setmelanotide lost an 0 Average change in body weight, % average
Slide 10: BMI at Week 14 Was Significantly Reduced With Setmelanotide Treatment Versus Placebo
BMI at Week 14 Was Significantly Reduced With Setmelanotide Treatment Versus Placebo in Patients With BBS Percent BMI Change From Baseline Setmelanotide Placebo In all patients with BBS, those N=16 N=16 treated with setmelanotide 1 achieved an average BMI reduction -1 of −1.5 kg/m2 (−3.8%) more at A
Slide 11: Setmelanotide Treatment Led to Reduced Hunger Compared With Placebo
Setmelanotide Treatment Led to Reduced Hunger With 14 Weeks of Treatment in Participants ≥12 Years With BBS Compared With Placebo Weekly Average “Most Hunger” Score Weekly Average “Most Percent Change at Week 14 Hunger” Score Percent Change Setmelanotide Placebo From Baseline to Week 14 N=14 N=15 0
Slide 12: Rates of Common AEs Were Generally Similar Between Setmelanotide and Placebo
Rates of Common AEs Were Generally Similar Between Setmelanotide and Placebo During the Double-Blind Period AEs occurring in the double-blind period, n Setmelanotide Placebo Total (%) N=16 N=16 N=32 Treatment-emergent AEs occurring in ≥15% of participants Injection site erythema 8 (50.0) 7 (43.8) 15
Slide 13: Setmelanotide Was Generally Well Tolerated in Patients With BBS
Setmelanotide Was Generally Well Tolerated in Patients With BBS During the 14-Week Double-Blind Period Setmelanotide Placebo Total AEs, n (%) N=16 N=16 N=32 Treatment-related AEs 14 (87.5) 14 (87.5) 28 (87.5) Serious AEs 0 2 (12.5) 2 (6.3) Serious treatment-related AEs 0 1 (6.3) 1 (3.1) AEs leading
Slide 14: Summary and Conclusions
Summary and Conclusions • Overall, participants with BBS lost on average 3.2% of their body weight from baseline at Week 14, compared with negligible weight loss with placebo • Setmelanotide significantly reduced “most hunger” compared with placebo at Week 14 in participants with BBS • Setmelanotide