Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — ObesityWeek 2023
9 slides from Rhythm Pharmaceuticals's conference presentation, October 2023
Slide 1: 4-Year Setmelanotide Weight Outcomes in Patients With POMC and LEPR Deficiency Obesity
4-Year Setmelanotide Weight Outcomes in Patients With POMC and LEPR Deficiency Obesity Presenter: Dr James M. Swain Wendy K. Chung,1 James M. Swain,2 Peter Kühnen,3 Martin Wabitsch,4 Erica van den Akker,5 Jill Garrison,6 Guojun Yuan,6 Jesús Argente,7,8 Karine Clément,9,10 Sadaf Farooqi11 1Division o
Slide 2: POMC and LEPR Deficiency Are Rare Forms of MC4R Pathway–Associated Obesity
POMC and LEPR Deficiency Are Rare Forms of MC4R Pathway– Associated Obesity • Under physiologic conditions, the hypothalamic MC4R pathway regulates hunger, satiety, energy expenditure, and, consequently, body weight1-4 • Rare variants in key genes of the MC4R pathway are associated with hyperphagia
Slide 3: Objective
Objective The objective of this analysis was to assess the long-term efficacy of setmelanotide therapy in patients with POMC and LEPR deficiency obesity who had a clinically meaningful weight response at the end of the initial 12 months of therapy and who had long-term, on-treatment outcomes at Year
Slide 4: Clinical Trials Overview
Clinical Trials Overview Index Trial Long-term Extension (NCT02896192,1 NCT03287960,1 or NCT02507492,2,3 NCT03013543) (NCT03651765) Open-label treatment Open-label treatment • Demonstrated efficacy • Acceptable Screening QD setmelanotide Yes c QD setmelanotide safety • Informed consent Baseline End
Slide 5: Patient Disposition and Baseline Characteristics
Patient Disposition and Baseline Characteristics Enrolled in LTE with Total a meaningful Index trial baseline characteristics (n=19) weight response* Not included (n=8) Age, mean (SD; range) 20.2 (6.7; 10-36) (N=27) Age range, n (%) • Transitioned to commercial therapy ≥18 years 11 (57.9) (n=4) <18
Slide 6: Sustained Improvements in Weight and BMI Across Adult Patients Over 4 Years
Sustained Improvements in Weight and BMI Across Adult Patients Over 4 Years Percent change in body weight, adults Percent change in BMI, adults Baseline weight: 148.6 (32.4) kg (n=11) Baseline BMI: 49.2 (11.6) kg/m2 (n=11) 0.0 0.0 -10.0 -10.0 Mean change in weight, % Mean change in BMI, % -20.2% -17
Slide 7: Age-Appropriate Weight Measures Were Reduced in Pediatric Patients Over 4 Years of Treatment
Age-Appropriate Weight Measures Were Reduced in Pediatric Patients Over 4 Years of Treatment Change in BMI Z score, patients <18 years Change in %BMI95, patients <18 years Baseline BMI Z score: 3.2 (0.57; n=8) Baseline %BMI95: 152.7 (16.0; n=8) 0.0 0.0 Mean change in %BMI95, percentage -10.0 -0.5 Me
Slide 8: The Safety Profile of Setmelanotide At Year 3 Was Consistent With Previous Studies
The Safety Profile of Setmelanotide At Year 3 Was Consistent With Previous Studies of POMC or LEPR Deficiency Obesity AEs occurring during the index and LTE trials in the Patients, n (%) overall population (N=24)* Any AE 24 (100.0) Any treatment-related AE 24 (100.0) Serious AEs 11 (45.8) Serious tr
Slide 9: Summary and Conclusions
Summary and Conclusions • Long-term treatment with setmelanotide demonstrated sustained weight-related efficacy in pediatric and adult patients with POMC or LEPR deficiency • The safety profile of setmelanotide was consistent with previous studies • The limitations of this study include lack of a co