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 syndrome. Diabetes, 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 deficiency. Nat. 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 2022 – European Society for Paediatric Endocrinology Annual Meeting
The Multifaceted Burden Experienced by Caregivers of Individuals with Bardet-Biedl Syndrome: Findings from the CARE-BBS Study
Elizabeth Forsythe, M.D., Ph.D., UCL Great Ormond Street Institute of Child Health, London.
Slides
Caregiver Burden in Bardet-Biedl Syndrome: a Survey of Obesity and Hyperphagia Impacts
Dr. Forsyth
Poster
Effects of Setmelanotide Treatment in Children and Adolescents with Proopiomelanocortin (POMC) Deficiency, Leptin Receptor (LEPR) Deficiency, and Bardet-Biedl Syndrome (BBS)
Jesús Argente, MD, Professor in the Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid
Slides
VENTURE: Design of a Phase 3 Multicenter, 1-Year, Open-label Trial of Setmelanotide in Pediatric Patients Aged 2 to <6 Years with Rare Genetic Diseases of Obesity
Dr. Argente
Poster
ENDO 2022 – Annual Conference
Long-term Efficacy of Setmelanotide in Patients with Bardet-Biedl Syndrome
Jesús Argente, M.D., Ph.D., Universidad Autónoma de Madrid in Spain
Poster
Long-term Efficacy of Setmelanotide in Patients With POMC or LEPR Deficiency Obesity
Karine Clément, M.D., Ph.D., Professor of Nutrition at Pitié-Salpêtrière Hospital, Sorbonne Université and Head of the INSERM Nutriomics Laboratory in Paris
Poster
Body Mass Index and Weight Reduction in Patients with SH2B1 Deficiency Obesity After 1 Year of Setmelanotide
Sadaf Farooqi, M.D., Ph.D, the Wellcome-MRC Institute of Metabolic Science at the University of Cambridge
Poster
Body Mass Index and Weight Reductions in Patients with SRC1 Deficiency Obesity After 1 Year of Setmelanotide
Jesús Argente, M.D., Ph.D., Universidad Autónoma de Madrid in Spain.
Poster
Body Mass Index and Weight Reductions in Patients with Obesity Due to Heterozygous Variants in POMC, PCSK1, or LEPR After 1 Year of Setmelanotide
Brieana Buckley, PharmD, M.S., Vice President of Medical Affairs at Rhythm
Slides
Setmelanotide in Patients With Obesity Due to Heterozygous Variants in POMC, LEPR, NCOA1, or SH2B1 Genes: Design of EMANATE—a Placebo-Controlled Phase 3 Trial
Sadaf Farooqi, M.D., Ph.D, the Wellcome-MRC Institute of Metabolic Science at the University of Cambridge
Poster
PES 2022 – The Pediatric Endocrine Society Virtual Annual Meeting
Impact of Setmelanotide Treatment on Lipid Parameters and Vital Signs in Patients with Bardet-Biedl Syndrome in a Phase 3 Trial
Andrea Haqq, M.D., M.H.S., Professor, Department of Pediatrics, Division of Pediatric Endocrinology/Metabolism at the University of Alberta
Slides
Setmelanotide Treatment in Pediatric and Adolescent Patients with Bardet-Biedl Syndrome and Severe Obesity
Robert Haws, M.D., Marshfield Clinic Research Institute
Poster
Patient and Caregiver-Reported Experiences of Hyperphagia in Bardet-Biedl Syndrome Before and During Setmelanotide Treatment
Claire Ervin, M.P.H., Senior Director, RTI Health Solutions
Poster
Clinical Safety Summary of Setmelanotide in Healthy Volunteers with Obesity and Patients with Rare Genetic Diseases of Obesity
Jesús Argente, M.D., Ph.D., Professor in the Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid in Spain
Poster
2022 ACMG Annual Clinical Genetics Meeting
Uncovering Rare Obesity® Genetic Testing Program: Overview and Health Care Provider Utilization
Jill Garrison, Ph.D., Director, Medical Affairs, at Rhythm
Poster
Frequency of BBS and ALMS1 Variants in a Cohort with Early-Onset Obesity
Joan Han, MD, Professor of Pediatrics and Chief of the Division of Pediatric Endocrinology and Diabetes in the Jack and Lucy Clark Department of Pediatrics at the Icahn School of Medicine at Mount Sinai and Mount Sinai Kravis Children’s Hospital
Poster
Variants in Obesity-Related Genes in a Population with Early-Onset Obesity
Patrick Kleyn, Ph.D., Senior Vice President, Head of Translational Research and Development
Poster
Frequency of MC4R Pathway Variants in a Large US Cohort of Pediatric and Adult Patients with Severe Obesity
Eric Bend, Ph.D., FACMG, Prevention Genetics
Poster
Biochemical Characterization of Single Minded-1 Missense Variants Associated with Severe Obesity
Megan Vogel, Ph.D., Scientist, Pre-Clinical Biology at Rhythm
Poster
Design of a Phase 2, Double-Blind, Placebo-Controlled Trial of Setmelanotide in Patients with Genetic Variants in the Melanocortin-4 Receptor Pathway
Sadaf Farooqi, PhD, Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, University of Cambridge, Cambridge, UK
Poster
An Evidence-based Framework to Evaluate Melanocortin-4 Receptor (MC4R) Pathway Relevance for Obesity-associated Genes
Megan Vogel, Ph.D., Scientist, Pre-Clinical Biology at Rhythm
Poster
Quality of Life in Patients with Bardet-Biedl Syndrome in a Setmelanotide Phase 3 Trial
Elizabeth Forsythe, Ph.D., Great Ormond Street Institute of Child Health, Faculty of Population Health Sciences, University College London
Poster
The Obesity Society’s ObesityWeek® 2021
“Quality of Life in Patients with Bardet-Biedl Syndrome in a Setmelanotide Phase 3 Trial“
Elizabeth Forsythe, Ph.D., Great Ormond Street Institute of Child Health, Faculty of Population Health Sciences, University College London
Poster
“Efficacy and Safety of Open-Label Setmelanotide in Bardet-Biedl Syndrome: A Phase 3 Trial“
Robert Haws, M.D., Clinical Research Center at the Marshfield Clinic Research Institute
Oral presentation slides
“Effects of Setmelanotide on Obesity, Hunger, and Safety in SRC1 Insufficiency“
Sadaf Farooqi, PhD, Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, University of Cambridge, Cambridge, UK
Poster
“Effects of Setmelanotide on Obesity, Hunger, and Safety in SH2B1 Deficiency: A Phase 2 Trial“
Jesús Argente, M.D., Ph.D., Professor in the Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid in Spain
Poster
“Frequency of MC4R pathway variants in a large US cohort of patients with severe obesity“
Ida Moeller, ScD, ScM, MMSc, Director of Biomedical Informatics at Rhythm
Poster
“Uncovering Rare Obesity Genetic Testing Program: Overview and Health Care Provider Utilization“
Jill Garrison, Ph.D., Associate Director, Medical Affairs, at Rhythm
Poster
“Biochemical Characterization of Single Minded-1 Missense Variants Associated with Severe Obesity“
Megan Vogel, Ph.D., Scientist, Pre-Clinical Biology at Rhythm
Poster
Obesity Medicine Association’s Overcoming Obesity 2021 Conference and the Digital Experience
“Efficacy and Safety of the Melanocortin-4 Receptor Agonist Setmelanotide in Obesity Due to Bardet-Biedl Syndrome: a Phase 3 Trial Phase 3 Trial of Setmelanotide in Bardet-Biedl Syndrome”
Robert Haws, M.D., Clinical Research Center at the Marshfield Clinic Research Institute
Poster
“Setmelanotide in POMC, PCSK1, or LEPR Heterozygous Deficiency Obesity (Phase 2)”
Sadaf Farooqi, PhD, Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, University of Cambridge, Cambridge, UK
Poster
“Variants in Obesity-related Genes in a Population with Early-onset Obesity”
Patrick Kleyn, Ph.D., Vice President, Translational R&D, Rhythm Pharmaceuticals
Poster
“Trial of a Once-weekly Setmelanotide Formulation in Patients with Obesity”
Annette Valles-Sukkar, MHP, CCRA, MT(ASCP), Director Clinical Operations, Rhythm Pharmaceuticals
Poster
“Quality of Life in POMC or LEPR Deficiency: Setmelanotide Phase 3 Trials”
Peter Kühnen, MD, Charité – Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin und Humboldt-Universität zu Berlin, Institute for Experimental Pediatric Endocrinology, Berlin, Germany
Poster
“Patient Experience of Hunger in POMC or LEPR Deficiency”
Martin Wabitsch, Division of Pediatric Endocrinology and Diabetes, Center for Rare Endocrine Diseases, Department of Pediatrics and Adolescent Medicine, University of Ulm, Ulm, Germany
Poster
“Frequency of BBS and AS Gene Variants in a Cohort with Early-onset Obesity”
Joan Han, MD, Director of the Pediatric Obesity Program at Le Bonheur Children’s Hospital, Associate Professor, Division of Pediatric Endocrinology at the University of Tennessee Health Science Center
Poster
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
