Rhythm Pharmaceuticals is dedicated to addressing rare neuroendocrine diseases, particularly those related to the melanocortin-4 receptor (MC4R) pathway, which plays a crucial role in regulating hunger and energy expenditure. The company focuses on developing innovative therapies for conditions such as hyperphagia and obesity, which can arise from genetic variants or damage to the hypothalamus. Their lead product, Setmelanotide, is designed to target specific genetic deficiencies associated with obesity, including POMC and LEPR deficiencies, and is currently undergoing various phases of clinical trials to evaluate its safety and efficacy.

In addition to Setmelanotide, Rhythm Pharmaceuticals is exploring a range of investigational therapies aimed at treating acquired hypothalamic obesity and other related conditions. Their pipeline includes Bivamelagon, an oral MC4R-specific agonist, and RM-718, a new weekly MC4R-specific agonist, both of which are designed to provide therapeutic options without causing hyperpigmentation. The company is committed to advancing scientific understanding of these rare diseases through clinical trials and investigator-initiated studies, ultimately striving to improve the quality of life for patients and their families affected by these challenging conditions.

United States

Rhythm is a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity. The company recently announced topline results from pivotal Phase 3 clinical trials in POMC deficiency obesity and LEPR deficiency obesity. Rhythm is also conducting a pivotal Phase 3 trial in Bardet-Biedl syndrome and Alström syndrome. The company is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study in additional rare genetic disorders of obesity, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of these disorders. For healthcare professionals, visit www.UNcommonObesity.com for more information. 

Metabolic Diseases

Pathological Conditions

Rhythm Pharmaceuticals

Obesity

<p class="font_7">Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions.</p>

Rhythm Pharmaceuticals

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