Financial Performance & Guidance - Rhythm Pharmaceuticals reported Q3 2025 product revenue of $51.3 million, compared to $33.3 million in Q3 2024[51] - The company experienced a 10% quarter-over-quarter growth in patients on reimbursed therapy globally[49] - Q3 2025 operating expenses totaled $98.5 million, including $18.8 million in stock-based compensation expense[52] - Rhythm anticipates non-GAAP operating expenses for 2025 to be between $295 million and $315 million, including R&D expenses of $150 million to $165 million and SG&A expenses of $145 million to $150 million[52] - As of September 30, 2025, Rhythm Pharmaceuticals held $416.1 million in cash, cash equivalents, and short-term investments[51] IMCIVREE & Pipeline Updates - Steady growth in global IMCIVREE sales in Q3 2025 was primarily driven by Bardet-Biedl syndrome (BBS)[10] - The FDA accepted the sNDA for setmelanotide in acquired hypothalamic obesity (HO) for Priority Review, with a PDUFA goal date of December 20, 2025[10] - A German investigator-led study showed that over 80% of BBS patients on setmelanotide exhibited either resolution of MASLD or stabilization at grade S1 after six months of therapy[12] - The same study showed 100% of patients (N=26) with both BBS and metabolic dysfunction-associated steatotic liver disease (MASLD) showed improvement[12] Strategic Initiatives & Market Access - Rhythm Pharmaceuticals estimates the U S prevalence of acquired hypothalamic obesity to be approximately 10,000 patients[15] - IMCIVREE is available in >25 countries outside the United States[36] - Rhythm estimates the European prevalence of acquired hypothalamic obesity to be approximately 10,000 patients[40] - Rhythm estimates the prevalence of acquired hypothalamic obesity in Japan to be approximately 5,000 to 8,000[43]

Core Insights - Rhythm Pharmaceuticals reported continued momentum in the third quarter of 2025, highlighted by FDA acceptance of the supplemental new drug application (sNDA) for acquired hypothalamic obesity and steady growth from Bardet-Biedl syndrome (BBS) commercial efforts [2][5] Financial Performance - Revenue from global sales of IMCIVREE (setmelanotide) was $51.3 million for Q3 2025, a 6% increase from Q2 2025, primarily driven by growth in patients and sales for BBS treatment [3][9] - U.S. revenue accounted for $38.2 million (74% of product revenue), marking a 19% sequential increase, while international revenue was $13.1 million (26% of product revenue), reflecting a 21% decrease [3][9] - The company recorded a one-time charge of $3.2 million in Q3 2025 due to a final reimbursed price agreement for IMCIVREE in France [3][9] - R&D expenses were $46.0 million in Q3 2025, up from $37.9 million in Q3 2024, mainly due to increased headcount and stock-based compensation [10] - SG&A expenses rose to $52.4 million in Q3 2025 from $35.4 million in Q3 2024, driven by higher costs associated with expanding operations and marketing [11] - The net loss attributable to common stockholders was $54.3 million for Q3 2025, compared to a net loss of $45.0 million in Q3 2024 [12] Clinical Developments - Rhythm expects to report preliminary results from the Phase 2 trial of setmelanotide in Prader-Willi syndrome in Q4 2025 [5][13] - A recent observational study indicated that setmelanotide treatment improved measures of metabolic dysfunction-associated steatotic liver disease (MASLD) and kidney function in BBS patients [6][7] Upcoming Milestones - The FDA's PDUFA goal date for the sNDA for setmelanotide in acquired hypothalamic obesity is set for December 20, 2025 [5][7] - The company anticipates completing enrollment in the Phase 1 trial evaluating RM-718 in Q1 2026 and announcing topline data from various ongoing trials in early 2026 [13] Cash Position - As of September 30, 2025, the company had approximately $416.1 million in cash, cash equivalents, and short-term investments, compared to $320.6 million at the end of 2024 [8][20]

Financial Performance & Outlook - Rhythm Pharmaceuticals reported $48.5 million in net product revenue from global sales in Q2 2025, a 29% sequential growth[60] - The company's cash, cash equivalents, and short-term investments totaled $291 million as of June 30, 2025[59,62] - Rhythm anticipates non-GAAP operating expenses for 2025 to be between $285 million and $315 million, including SG&A expenses of $135 million to $145 million and R&D expenses of $150 million to $170 million[63] - A public offering of common stock closed July 11, 2025, resulting in net proceeds of $189.2 million[59] Clinical & Regulatory Updates - Rhythm is on track to complete U S and EU regulatory submissions for setmelanotide in acquired hypothalamic obesity (HO) in Q3 2025[10,33] - Phase 3 trial data showed a -16.5% BMI change from baseline in the setmelanotide arm (n=81) compared to a +3.3% BMI change in the placebo arm (n=39), resulting in a placebo-adjusted difference in BMI reduction of -19.8% (P<0.0001)[21,22,23,24] - Bivamelagon achieved statistically significant BMI reductions at all doses in a 14-week, Phase 2 trial, with reductions of -2.68% (200mg), -7.69% (400mg), and -9.31% (600mg) compared to a +2.18% increase in the placebo group[25] Commercial Performance & Strategy - The company saw a 38% increase in cumulative prescribers from Q2 2024 to Q2 2025 and a 9% increase from Q1 2025 to Q2 2025 for IMCIVREE prescriptions in patients with Bardet-Biedl syndrome (BBS)[40] - IMCIVREE prescriptions for patients aged 2-12 years accounted for 40% of Q2 2025 prescriptions[43]