Rhythm Pharmaceuticals has launched IMCIVREE (setmelanotide) in Japan to treat acquired hypothalamic obesity. It is the first approved therapy for the condition in the country, and it is now on Japan's National Health Insurance drug price list. Japan's Ministry of Health, Labour and Welfare approved the drug in August. The approval rested on the Phase 3 TRANSCEND trial of 142 patients, which met its primary endpoint with an 18.8% placebo-adjusted reduction in body mass index. Rhythm, based in Boston, estimates that 5,000 to 8,000 people in Japan live with the condition. "This launch marks an important advancement for people living with acquired HO in Japan," said Yann Mazabraud, executive vice president and head of international at Rhythm.

Stanley Druckenmiller's Duquesne Family Office disclosed new positions in over 40 stocks in its second quarter 13F filing. Notable healthcare investments included 203,618 shares of Rhythm Pharmaceuticals worth $22.6 million and 356,600 shares of Definium Therapeutics worth $16.8 million. Rhythm Pharmaceuticals develops treatments for hypothalamic obesity, currently the only approved drug for this condition. The company estimates approximately 10,000 US patients have acquired hypothalamic obesity, with similar numbers in Europe and 5,000 to 8,000 in Japan. Second-quarter revenue increased 47% year over year, whilst US revenue jumped 38% sequentially. The company remains unprofitable despite revenue growth and held $330.9 million in cash at quarter end, expected to fund operations for at least 24 months.

Rhythm Pharmaceuticals has received approval from Japan's Ministry of Health, Labour and Welfare for IMCIVREE (setmelanotide) to treat patients with acquired hypothalamic obesity (HO). This marks the first therapy approved in Japan for this condition. The approval was supported by the Phase 3 TRANSCEND trial involving 142 patients, including a Japanese cohort. The study met its primary endpoint with an 18.8% placebo-adjusted reduction in body mass index. Rhythm estimates 5,000 to 8,000 people are living with acquired HO in Japan. The company expects to launch IMCIVREE before the end of 2026, pending final pricing determination. Acquired HO is characterised by accelerated weight gain following hypothalamic injury, often resulting from craniopharyngioma, astrocytoma, or other hypothalamic-pituitary tumours.