Rhythm Pharmaceuticals

Rhythm is developing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic disorders.

 
 
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PIPELINE > PUBLISHED RESEARCH

PUBLISHED RESEARCH

Han JC, Smith WA, Burton ET, Joshi VM, Onder AM, Wright TS, Hylan M, Folster C, Geberhiwot T, Fiedorek FT. Effect of Setmelanotide (MC4R Agonist) on Obesity and Hunger in Individuals With Alström Syndrome. ObesityWeek 2018, November 15, 2018.

Shoemaker AH, Yanovski J, Muniyappa R, Argente J, Kuhnen P, Clement K, Bemert CP, Oubem B, Ayers K, Kiel M, Chittenden TW, Martos-Moreno GA, Reitman M, Moeller IH, Fiedorek FT, Connors H, Miller JL, Garfield A, Van der Ploeg L. Identification of MC4R Pathway Variants in Individuals With Extreme Obesity. ObesityWeek 2018, November 15, 2018.

Haws RM, Fletty KL, McIntee TJ, Green C, Pomeroy J, Hylan M, Folster C, Davis EK, Brady SM, Fiedorek FT, Yanovski JA. Effect of the Melanocortin-4 Receptor Agonist Setmelanotide on Obesity and Hyperphagia in Individuals Affected by Bardet-Biedl Syndrome. ESPE 2018, September 28, 2018.

Han JC, Fiedorek FT, Hylan M, Folster C, Geberhiwot T. Effect of a Melanocortin-4 Receptor Agonist, Setmelanotide, on Obesity and Hyperphagia in Individuals Affected by Alström Syndrome. ESPE 2018, September 28, 2018.

Eneli I, Xu J, Fiedorek F, Webster M, McCagg A, Ayers K, Van der Ploeg L, Garfield A, Estrada E. Tracing the Effect of the Melanocortin-4 Receptor Pathway in Obesity: Study Design and Methodology of the TEMPO Registry. ESPE 2018, September 28, 2018.

Clément K, Biebermann H, Farooqi IS, Van der Ploeg L, Wolters B, Poitou C, Puder L, Fiedorek F, Gottesdiener K, Kleinau G, Heyder N, Scheerer P, Blume-Peytavi U, Jahnke I, Sharma S, Mokrosinski J, Wiegand S, Müller A, Weiß K, Mai K, Spranger J, Grüters A, Blankenstein O, Krude H, Kühnen P. MC4R Agonism Promotes Durable Weight Loss in Patients with Leptin Receptor Deficiency. Nat Med. 2018 May 7. doi: 10.1038/s41591-018-0015-9. [Epub ahead of print]

Ayers KL, Glicksberg BS, Garfield AS, Longerich S, White JA, Yang P, Du L, Chittenden TW, Gulcher JR, Roy S, Fiedorek F, Gottesdiener K, Cohen S, North KE, Schadt EE, Li SD, Chen R, Van der Ploeg LHT. Melanocortin-4 Receptor Pathway Dysfunction in Obesity: Patient Stratification Aimed at MC4R Agonist Treatment. J Clin Endocrinol Metab. 2018 May 2. doi: 10.1210/jc.2018-00258. [Epub ahead of print]

Ayers KL, Glicksberg BS, Garfield AS, Longerich S, White JA, Yang P, Du L, Chittenden TW, Gulcher JR, Moeller IH, Roy S, Fiedorek F, Gottesdiener K, Cohen S, North KE, Schadt EE, Li SD, Chen R, Van der Ploeg LHT. Melanocortin 4 Receptor Pathway Dysfunction in Obese Patients: Prevalence Estimates of LEPR, POMC, and PCSK1 Variants. ENDO 2018, March 19, 2018.

Haws RM, Fletty KL, McIntee TJ, Green C, Pomeroy J, Hylan M, Folster C, Fiedorek FT. Obesity and Hyperphagia Therapy in Bardet-Biedl Syndrome With a Melanocortin-4 Receptor Agonist. ObesityWeek 2017, October 31, 2017.

Kühnen P, Handke D, Waterland RA, Hennig BJ, Silver M4, Fulford AJ, Dominguez-Salas P, Moore SE, Prentice AM4, Spranger J, Hinney A8, Hebebrand J, Heppner FL, Walzer L2, Grötzinger C, Gromoll J, Wiegand S, Grüters A, Krude H. Interindividual Variation in DNA Methylation at a Putative POMC Metastable Epiallele Is Associated with Obesity. Cell Metab. 2016 Sep 13; 24(3):502-9. doi: 10.1016/j.cmet.2016.08.001.

Kühnen P, Clément K, Poitou-Bernert C, Fiedorek F, van der Ploeg L, Connors H, Gottesdiener K, Farooqi S, Wiegand S, Grüters A, Krude H. T-P-LB-3656: Proof of Concept for Treatment of a Second Rare Genetic Disorder of the Leptin-Melanocortin Pathway: Successful Therapy of Extreme Obesity in a Leptin-Receptor (LepR) Deficient Patient with Setmelanotide. ObesityWeek 2016, November 4, 2016.

Low, MJ. Neuroendocrinology: New Hormone Treatment for Obesity Caused by POMC-Deficiency. Nat Rev Endocrinol. 23 September 2016. doi:10.1038/nrendo.2016.156

Kühnen P, Clémen K, Wiegand S, Blankenstein O, Gottesdiener L, Martini LL, Mai K, Blume-Peytavi U, Grüters A, Krude H. Proopiomelanocortin Deficiency Treated with a Melanocortin-4 Receptor Agonist. N Engl J Med. July 2016; 375:240-246.

Gottesdiener K, Conners H, van der Ploeg L, Collet T, Farooqi S, Dubern B, Clement K, Lewis W. A Pilot Study with the Synthetic Peptide Setmelanotide (RM-493), a Melanocortin-4 Receptor Agonist, for the Treatment of Heterozygous MC4R Deficiency Obesity. Obesity Society, November 5, 2015.

Gottesdiener K, Conners H, Van der Ploeg L, Fiedorek F, Hylan M, Louis W, Lasseter K. T-P-3134: Analysis of the Synthetic Peptide Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, on Cardiovascular Parameters in Three Phase1b/2a Studies. ObesityWeek 2015, November 5, 2015.

Chen KY, Muniyappa R, Abel BS, Mullins KP, Staker P, Brychta RJ, Zhao X, Ring M, Psota TL, Cone RD, Panaro BL, Gottesdiener KM, Van der Ploeg LHT, Reitman ML, Skarulis MC. RM-493, a Melanocortin-4 Receptor (MC4R) Agonist, Increases Resting Energy Expenditure in Obese Individuals. J. Clin. Endocrinol. Metab. 2015 Apr, 100(4):1639-45. doi: 10.1210/jc.2014-4024.

Clemmensen C, Finan B, Fischer K, Tom RZ, Legutko B, Sehrer L, Heine D, Grassl N, Meyer CW, Henderson B, Hofmann SM, Tschöp MH, Van der Ploeg L, Müller TD. Dual Melanocortin-4 Receptor and GLP-1 Receptor Agonism Amplifies Metabolic Benefits in Diet-Induced Obese Mice. EMBO Mol Med. 2015 Feb 4, doi: 10.15252/emmm.201404508.

Muniyappa R, Chen K, Brychta R, Abel B, Mullins K, Staker P, Van der Ploeg L, Connors H, Gottesdiener K, Reitman ML, Skarulis MC. A Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Effect of a Melanocortin Receptor 4 (MC4R) Agonist, RM-493, on Resting Energy Expenditure (REE) in Obese Subjects. International Society of Endocrinology and the Endocrine Society (ICE/ENDO 2014), June 22, 2014.

Schaaf CP, Gonzalez-Garay ML, Xia F, Potocki L, Gripp KW, Zhang B, Peters BA, McElwain MA, Drmanac R, Beaudet AL, Caskey CT, Yang Y. Truncating Mutations of MAGEL2 Cause Prader-Willi Phenotypes and Autism. Nat.Genet. (November 2013) 45, 1405–1408.

Mercer RE, Michaelson SD, Chee MJS, Atallah TA, Wevrick1 R, Colmers WF. Magel2 is Required for Leptin-Mediated Depolarization of POMC Neurons in the Hypothalamic Arcuate Nucleus in Mice. PLOS Genet.. 2013;9(1):e1003207. doi: 10.1371/journal.pgen.1003207. Epub 2013 Jan 17.

Kievit P, Halem H, Marks DL, Dong JZ, Glavas MM, Sinnayah P, Pranger L, Cowley MA, Grove KL, Culler MD. Chronic Treatment With a Melanocortin 4 Receptor Agonist Causes Weight Loss, Reduces Insulin Resistance, and Improves Cardiovascular Function in Diet-Induced Obese Rhesus Macaques. Diabetes. 2012 Oct 9 [Epub ahead of print]. doi:10.2337/db12-0598.

Roubert P, Dubern B, Plas P, Lubrano-Berthelier C, Alihi R, Auger F, Deoliveira D, Dong J, Basdevant A, Thurieau C, Clément K. Novel Pharmacological MC4R Agonists can Efficiently Activate Mutated MC4R from Obese Patient with Impaired Endogenous Agonist Response. J Endocrinol. (2010) 207, 177-183.

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