Rhythm Pharmaceuticals Inc

Billionaire Stanley Druckenmiller's Duquesne Family Office initiated a position in Rhythm Pharmaceuticals during the second quarter. The biotech, which focuses on rare forms of obesity, saw its revenue rise 47% year over year to $71.3 million in the second quarter. Its only approved drug, Imcivree, received a label expansion in March for acquired hypothalamic obesity, a condition affecting 25,000 to 28,000 patients in the U.S., Europe, and Japan. The company also reported positive phase 2 results for Imcivree in Prader-Willi syndrome, a rare genetic condition with an estimated 20,000 patients in the U.S. and 400,000 worldwide. Rhythm Pharmaceuticals' stock accounts for less than 1% of Duquesne Family Office's portfolio.

Rhythm Pharmaceuticals reported second-quarter 2026 net product revenue from global sales of IMCIVREE of $71.3 million, a 19% sequential increase, and announced that more than 400 patient start forms had been received for the drug’s newly approved acquired hypothalamic obesity indication as of June 30, 2026. U.S. revenue accounted for $51.0 million of the total, up 38% from the first quarter, driven primarily by demand in acquired HO and continued growth in Bardet-Biedl syndrome, while ex-U.S. revenue was $20.3 million, down 13% sequentially due in part to a $3.8 million retrospective charge in France. The company also disclosed preliminary data from Part C of its Phase 1/2 study of the weekly injectable MC4R agonist RM-718, showing a mean BMI reduction of 11.6% in seven acquired HO patients after 16 weeks of treatment. Net loss attributable to common stockholders widened to $50.4 million, or $0.73 per share, compared with $48.0 million, or $0.75 per share, in the prior-year period, while cash, cash equivalents and short-term investments stood at approximately $330.9 million as of June 30, 2026. Rhythm expects its existing cash to fund operations for at least 24 months and provided full-year 2026 non-GAAP operating expense guidance of approximately $363 million to $397 million.

Rhythm Pharmaceuticals announced that results from its pivotal Phase 3 TRANSCEND trial of Setmelanotide in patients with acquired hypothalamic obesity have been published in The New England Journal of Medicine. The study, the largest and longest placebo-controlled trial in this population, showed that over 52 weeks, patients treated with Setmelanotide achieved a placebo-adjusted BMI reduction of 19.8%, with a mean BMI decrease of 16.5% compared to a 3.3% increase in the placebo group. 80% of treated patients achieved at least a 5% BMI reduction, alongside clinically meaningful improvements in hunger. In March 2026, the U.S. FDA approved Setmelanotide, marketed as IMCIVREE, as the first and only therapy for acquired hypothalamic obesity in adults and children aged four years and older, and the European Medicines Agency also recommended marketing authorization for this indication.

Canaccord raised its price target on Rhythm Pharmaceuticals to $151 from $143 and maintained a Buy rating after a positive interim review of the Phase 2 study of setmelanotide in Prader-Willi syndrome. The firm highlighted continued BMI improvements with longer treatment exposure, with reductions reaching 3.06% at six months compared with 1.84% at three months. It also noted that 8 of 10 patients with moderate to severe baseline hyperphagia achieved a clinically meaningful reduction of at least 7 points on the HQ-CT assessment. Rhythm Pharmaceuticals also presented new data from its MC4R agonist programs in acquired hypothalamic obesity, Bardet-Biedl syndrome, and Prader-Willi syndrome at ENDO 2026, showing improvements in weight-related outcomes and hyperphagia across multiple patient populations.

Rhythm Pharmaceuticals shared preliminary Phase 2 trial results showing setmelanotide improved weight, body composition, hunger, and behavioral outcomes in patients with Prader-Willi syndrome. Patients saw an average BMI reduction of just over 3% at six months, with consistent results across adults and children, and 8 out of 10 patients with moderate to severe hyperphagia experienced significant improvements in hunger scores. The safety profile was consistent with prior studies, giving the company confidence to advance into a Phase 3 trial. Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare genetic diseases of obesity.