Publications and Presentations

Publications

Clément, K., et al. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. The Lancet Diabetes & Endocrinology. Online first (2020). https://doi.org/10.1016/S2213-8587(20)30364-8

Haws, R., et al.Effect of setmelanotide, a melanocortin-4 receptor agonist, on obesity in Bardet-Biedl syndromeDiabetes, Obesity and Metabolism, 10.1111/dom.14133 (2020). Advance online publication.  https://doi.org/10.1111/dom.14133

Clément, K., et al. MC4R agonism promotes durable weight loss in patients with leptin receptor deficiencyNat. Med. 24, 551–555 (2018). https://doi.org/10.1038/s41591-018-0015-9

Kühnen, P., et al. Proopiomelanocortin Deficiency Treated with a Melanocortin-4 Receptor Agonist. New England Journal of Medicine; 375:240-6. DOI: 10.1056/NEJMoa1512693(2016). https://www.nejm.org/doi/10.1056/NEJMoa1512693

Garfield, AS. et al. Role of central melanocortin pathways in energy homeostasis. Trends in Endocrinology and Metabolism: TEM, 203-215. DOI: 10.1016/j.tem.2009.02.002 (2009).https://doi.org/10.1016/j.tem.2009.02.002

To request a copy of an article, email medinfo@rhythmtx.com.

Presentations

ESPE 2021 – 59th Annual European Society for Paediatric Endocrinology Meeting

“Efficacy and Safety Results of a Phase 2 Trial of Setmelanotide in Obesity Due to SH2B1 Variants and 16p11.2 Deletion Syndrome”
Cecilia Scimia, M.D., Ph.D., Medical Director, Rhythm Pharmaceuticals
Oral presentation slides

“Phase 3 Trial of Setmelanotide in Participants with Bardet-Biedl Syndrome: Placebo-Controlled Results”
Jesús Argente, Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid, University Hospital Niño Jesús, CIBER “Fisiopatología de la obesidad y nutrición” (CIBEROBN), Instituto de Salud Carlos III, IMDEA Institute, Madrid, Spain
Oral presentation slides

“A Phase 2 Trial of the Melanocortin-4 Receptor Agonist Setmelanotide in Obesity Due to SRC1 Insufficiency: Body Weight, Body Mass Index Z Score, and Safety Results”
Sadaf Farooqi, Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, University of Cambridge, Cambridge, UK
Oral presentation slides

“Efficacy and Safety of Setmelanotide in Individuals with Obesity Due to POMC or LEPR Deficiency: Phase 3 Results from Pivotal and Supplemental Cohorts”
Jennifer Miller, Division of Pediatric Endocrinology, University of Florida
Poster

“Design of a Phase 2, Double-Blind, Placebo-Controlled Trial of Setmelanotide in Patients with Genetic Variants in the Melanocortin-4 Receptor Pathway”
Cecilia Scimia, M.D., Ph.D., Medical Director, Rhythm Pharmaceuticals
Poster 

“An Evidence-based Framework to Evaluate Melanocortin-4 Receptor (MC4R) Pathway Relevance for Obesity-associated Genes”
Bhavik Shah, Ph.D., Senior Director Translational Research and Nonclinical Development, Rhythm Pharmaceuticals
Poster

“Frequency of MC4R Pathway Variants in a Large US Cohort of Pediatric and Adult Patients with Severe Obesity”
Ida Moeller, ScD, ScM, MMSc, Director of Biomedical Informatics, Rhythm Pharmaceuticals
Poster

ENDO 2021 – Annual Meeting of the Endocrine Society

“Effects of Setmelanotide in Patients With POMC, PCSK1, or LEPR Heterozygous Deficiency Obesity in a Phase 2 Study,”
an oral presentation by Sadaf Farooqi, M.D., Ph.D., professor at the Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre
 March 20, 2021 oral presentation slides

“A Phase 3 Trial in Participants with Obesity Due to Bardet-Biedl Syndrome or Alström Syndrome: Efficacy and Safety of the Melanocortin 4 Receptor Agonist Setmelanotide,” a poster presentation by Robert Haws, M.D., Clinical Research Center at the Marshfield Clinic Research Institute
March 20, 2021 poster presentation

“Timing of Onset of Adverse Events with Setmelanotide, an MC4R Agonist, in Patients with Severe Obesity Due to LEPR or POMC Deficiency,” a poster presentation by Peter Kühnen, MD, Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin in Berlin
March 20, 2021 poster presentation

The Obesity Society’s ObesityWeek® 2020

“A Randomized Trial of a Once-Weekly Formulation of Setmelanotide in Individuals with Obesity,” an oral presentation by Gregory Gordon, MD, JD, Vice President, Clinical at Rhythm
 Nov. 5, 2020 oral presentation slides

“Long-term Weight and Hunger Reduction With Setmelanotide in Individuals With POMC Deficiency Obesity,” a poster presentation by Karine Clément, MD, PhD, and Professor of Nutrition at Pitié-Salpêtrière Hospital and Sorbonne Université in Paris
Nov. 3, 2020 poster presentation

“Suicidality and Depression in Individuals With Genetic Obesity Treated With Setmelanotide,” a poster presentation by Peter Kühnen, MD, Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin in Berlin
Nov. 3, 2020 poster presentation

The Obesity Society’s ObesityWeek® 2019

“Efficacy and Safety of the MC4R Agonist Setmelanotide in POMC Deficiency Obesity: A Phase 3 Trial,” presented by Peter Kühnen, M.D., Institute for Experimental Pediatric Endocrinology, Charité Universitäts medizin Berlin, Germany
Nov. 4 oral presentation slides
Nov. 7 poster presentation

“Efficacy and Safety of the MC4R Agonist Setmelanotide in LEPR Deficiency Obesity: A Phase 3 Trial,” oral presentation by Erica Van Den Akker, M.D., Ph.D., Erasmus MC-Sophia Children’s Hospital University in Rotterdam, Netherlands
Nov. 4 oral presentation slides
Nov. 7 poster presentation