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Rhythm Pharmaceuticals

Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company developing precision medicines for rare neuroendocrine diseases linked to the MC4R pathway, including severe obesity.

Rhythm Pharmaceuticals is a global commercial-stage biopharmaceutical company founded in 2008 and headquartered in Boston. It develops precision medicines for rare neuroendocrine diseases, focusing on severe obesity and hyperphagia caused by genetic impairments in the melanocortin-4 receptor (MC4R) pathway. The company listed on Nasdaq in 2017 and employs more than 400 people across 19 countries in North America, Europe and beyond.

Its lead product, IMCIVREE (setmelanotide), was the first precision medicine approved by the FDA, in 2020, for severe obesity caused by deficiencies in the MC4R pathway - including POMC deficiency, LEPR deficiency and Bardet-Biedl syndrome - and received European Commission authorisation in 2021. The pipeline has since widened: RM-718 has entered clinical testing with first patients dosed, and LB54640, an oral MC4R agonist, was acquired from LG Chem Life Sciences in 2024.

Technical work at the company spans rare disease drug development, MC4R pathway biology, melanocortin receptor agonism, peptide therapeutics, clinical trial development and orphan drug commercialisation. Phase 2 results have been published in the New England Journal of Medicine and Nature Medicine. The organisation describes itself as science-led and mission-driven, with an expanding clinical pipeline and a stated focus on unmet needs in rare disease communities worldwide.

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