Searchable abstracts of presentations at key conferences in endocrinology

ea0099ep23 | Diabetes, Obesity, Metabolism and Nutrition | ECE2024

3-year setmelanotide weight outcomes in patients with bardet-biedl syndrome and obesity

Yanovski Jack , Angel Martos-Moreno Gabriel , Malhotra Sonali , Yuan Guojun , Chung Wendy , Dollfus Helene , Clement Karine

Background: Bardet-Biedl Syndrome (BBS) is a rare genetic disease in which impaired melanocortin-4 receptor (MC4R) signaling leads to hyperphagia and obesity. In an index Phase 3 trial, broad clinical benefit was observed in patients with BBS based on improvement or stabilization in ≥1 measure of weight, hunger, or quality of life with 1 year of treatment with the MC4R agonist setmelanotide. Reported here are long-term extension (LTE) weight outcomes after 3 years of set...

ea0090p66 | Diabetes, Obesity, Metabolism and Nutrition | ECE2023

Weight Outcomes With Setmelanotide Over 3 Years in Patients With POMC or LEPR Deficiency Obesity

Clement Karine , Wabitsch Martin , van den Akker Erica , Argente Jesus , Navarria Andrea , Srinivasan Madhura , Yuan Guojun , Malhotra Sonali , Kuhnen Peter

Background: Rare variants in the melanocortin-4 receptor (MC4R) pathway are associated with early-onset, severe obesity and hyperphagia. Setmelanotide, an MC4R agonist, reduced body mass index (BMI) and decreased hunger in patients with obesity due to biallelic variants in genes encoding proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) in Phase 3 trials. The current analysis assesses the durability of setmelanotide ef...

ea0090p598 | Diabetes, Obesity, Metabolism and Nutrition | ECE2023

Impact of Setmelanotide Treatment on Weight- and Body Composition–Related Outcomes in Pediatric and Adult Patients With Hypothalamic Obesity

Miller Jennifer , Shoemaker Ashley H. , Jennifer Abuzzahab M. , Gottschalk Michael , Yuan Guojun , Malhotra Sonali , Scimia Cecilia , Roth Christian L.

Background: Hypothalamic obesity (HO) is an acquired form of severe obesity that can occur following surgical resection of or radiotherapy for brain tumors and is often unresponsive to lifestyle modifications or traditional obesity pharmacotherapies. Here, we report weight- and body composition–related findings from a Phase 2 trial of setmelanotide, a melanocortin-4 receptor agonist, in patients with HO.Methods: A Phase 2, open-label, 16-week trial ...

ea0090p613 | Diabetes, Obesity, Metabolism and Nutrition | ECE2023

Long-term Body Composition in Patients With POMC or LEPR Deficiency Obesity Following Setmelanotide

Clement Karine , Wabitsch Martin , van den Akker Erica , Argente Jesus , Navarria Andrea , Srinivasan Madhura , Yuan Guojun , Malhotra Sonali , Kuhnen Peter

Objective: Ideally, treatment strategies designed to reduce weight or body mass index in patients with obesity should reduce fat mass while preserving lean mass because reduced lean mass can negatively impact overall health and energy expenditure, thus favoring weight regain. In Phase 3 trials, 1 year of treatment with the melanocortin-4 receptor agonist setmelanotide led to weight reduction in patients with obesity due to biallelic variants in the genes encoding proopiomelano...

ea0090p680 | Pituitary and Neuroendocrinology | ECE2023

Impact of Setmelanotide Treatment on Reducing Hyperphagia in Pediatric and Adult Patients With Hypothalamic Obesity

Roth Christian L. , Shoemaker Ashley H. , Gottschalk Michael , Miller Jennifer , Yuan Guojun , Malhotra Sonali , Scimia Cecilia , Jennifer Abuzzahab M.

Objective: To report hunger-related results from a Phase 2 trial of setmelanotide in patients with hypothalamic obesity (HO).Methods: A Phase 2, open-label, 16-week trial of setmelanotide in patients aged ≥6 to ≤ 40 years with body mass index (BMI) ≥95th percentile (aged <18 years) or ≥35 kg/m2 (aged ≥18 years) and HO caused by hypothalamic damage secondary to brain tumor, surgical resection, and/or chemothera...

ea0099ep25 | Diabetes, Obesity, Metabolism and Nutrition | ECE2024

Impact of setmelanotide on metabolic syndrome risk in patients with bardet-biedl syndrome

M. Haqq Andrea , Poitou-Bernert Christine , Chung Wendy , Iqbal Anoop , Forsythe Elizabeth , Malhotra Sonali , Touchot Nicolas , Clement Karine , Argente Jesus

Background: Patients with Bardet-Biedl syndrome (BBS), a rare syndrome associated with obesity and hyperphagia, commonly manifest traits of metabolic syndrome, including abdominal obesity, impaired fasting glucose, low high-density lipoprotein cholesterol, hypertriglyceridemia, and hypertension. Setmelanotide treatment results in significant improvement in weight, hunger, and quality of life in these patients and may improve measures associated with metabolic syndrome developm...

ea0099ep56 | Diabetes, Obesity, Metabolism and Nutrition | ECE2024

Weight reduction in patients with hypothalamic obesity treated with setmelanotide for 12 months

L. Roth Christian , Shoemaker Ashley , Gottschalk Michael , Miller Jennifer , Yuan Guojun , Malhotra Sonali , Scimia Cecilia , Jennifer Abuzzahab M.

Background: Hypothalamic obesity (HO) is an acquired form of severe obesity characterized by rapid and excessive weight gain resulting from insult to the hypothalamus—primarily caused by tumor invasion, resection, or radiotherapy—that can impair melanocortin-4 receptor (MC4R) pathway signaling. Treatment with setmelanotide, an MC4R agonist, resulted in weight and hunger reduction at 16 weeks in a Phase 2 trial of patients with HO. Here, we report changes in weight-re...

ea0090p331 | Diabetes, Obesity, Metabolism and Nutrition | ECE2023

Exploration of Clinical Improvements Following Setmelanotide in Patients With Bardet-Biedl Syndrome

Haqq Andrea M. , Chung Wendy , Dollfus Helene , Iqbal Anoop , A. Martos-Moreno Gabriel , Poitou Christine , Yanovski Jack A. , Malhotra Sonali , Miller Paul , Yuan Guojun , Forsythe Elizabeth , Clement Karine , Argente Jesus

Background: In patients with Bardet-Biedl syndrome (BBS), signaling impairments in the melanocortin-4 receptor (MC4R) pathway lead to hyperphagia and severe obesity, which negatively impact quality of life (QOL). We evaluated the impact of setmelanotide, an MC4R agonist, on age-appropriate weight-related parameters, hunger, and QOL in a Phase 3 trial of patients with BBS to further characterize clinical benefit in this patient population.Methods: Patient...