Slide 1: Weight Outcomes With Setmelanotide Over 3 Years in Patients With POMC or LEPR Deficiency Obesity
Weight Outcomes With Setmelanotide Over 3 Years in Patients With POMC or LEPR Deficiency Obesity Presenter: Dr. Sonali Malhotra Oral presentation 037 Clément, K1,2; Wabitsch, M3; Van den Akker, E4; Argente, J5,6; Navarria, A7; Srinivasan, M7; Yuan, G7; Malhotra, S7-9; Kühnen, P10 1Assistance Publiqu
Slide 2: Energy Expenditure Is Regulated by the Hypothalamic MC4R Pathway
Energy Expenditure Is Regulated by the Hypothalamic MC4R Pathway • Under physiologic conditions, the hypothalamic melanocortin-4 receptor (MC4R) pathway regulates hunger, satiety, energy expenditure, and, consequently, body weight1-4 • Rare variants in the MC4R pathway are associated with hyperphagi
Slide 3: Methods
Methods Objective: This analysis of a long-term extension trial (NCT03651765) assessed weight-related measures following setmelanotide treatment over 3 years among patients with POMC or LEPR deficiency obesity who achieved clinically beneficial weight loss in the index Phase 2 and 3 trials after 1 y
Slide 4: Patient Disposition and Baseline Characteristics
Patient Disposition and Baseline Characteristics 24 patients demonstrated meaningful responsea to setmelanotide after 1 year of treatment and were included in this analysis Total Index trial baseline characteristics (N=24) Age, years Mean (SD) 18.6 (7.4) Enrolled in LTE Age categories, n (%) <18 13
Slide 5: Reduced BMI Sustained Over 3 Years Across Patients
Reduced BMI Sustained Over 3 Years Across Patients Mean BMI change was −10.6 kg/m2 after 3 years of setmelanotide treatment (SD, 8.0 kg/m2 [n=15]) Mean BMI was 32.5 kg/m2 after 3 years of setmelanotide treatment (SD, 9.8 kg/m2 [n=15]) 5 Baseline BMI: 41.7 kg/m2 Mean (SD) BMI change, % -5 Month Month
Slide 6: Sustained Body Weight Reductions in Patients Aged ≥18 Years
Sustained Body Weight Reductions in Patients Aged ≥18 Years Mean weight change was −35.4 kg in adult patients after 3 years of setmelanotide treatment (SD, 23.6 kg [n=8]) Mean weight was 105.0 kg in adult patients after 3 years of setmelanotide treatment (SD, 25.7 kg [n=8]) 5 Baseline body weight: 1
Slide 7: Reduced BMI Z Score in Patients Aged <18 Years With LEPR or POMC Deficiency Obesity
Reduced BMI Z Score in Patients Aged <18 Years With LEPR or POMC Deficiency Obesity Mean BMI Z score change was −1.01 points in pediatric patients after 3 years of setmelanotide treatment (SD, 1.22 [n=4]) Mean BMI Z score was 2.42 points in pediatric patients after 3 years of setmelanotide treatment
Slide 8: The Safety Profile of Setmelanotide Was Consistent With Previous Studies
The Safety Profilea of Setmelanotide Was Consistent With Previous Studies of Patients With LEPR or POMC Deficiency Obesity n (%) AEs reported in ≥15% of population n (%) Any AE 24 (100.0) Injection site reactionsb,c 23 (95.8) Other disordersb,d 22 (91.7) Any treatment-related AEs 24 (100.0) Skin hyp
Slide 9: Summary and Conclusions
Summary and Conclusions • Patients with a meaningful clinical response in weight-related measures after 1 year of setmelanotide therapy continued to demonstrate sustained clinical benefit at 3 years of setmelanotide treatment • The safety profile of setmelanotide was consistent with previous reports