Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported global revenue of $51.3 million for Q3 2025, a sequential increase of 6% from $48.5 million in Q2 2025 [28] - The number of patients on reimbursed therapy increased by 10% globally during the quarter [29] - Year-over-year, net product revenues increased by $18 million, or 54%, compared to Q3 2024 [31] Business Line Data and Key Metrics Changes - InSibiri sales reached $51.3 million, driven predominantly by Bardet-Biedl syndrome (BBS), with a 10% increase in the number of patients on reimbursed therapy [5][29] - The U.S. generated $38.2 million (74% of total revenue), while international revenue accounted for $13.1 million (26% of total revenue) [29] - The proportion of prescriptions for pediatric versus adult patients began to normalize, with 50% of new patients being adults, 22% adolescents, and 28% pediatrics [16] Market Data and Key Metrics Changes - Rhythm's international business saw continued success, with InSibiri now available in over 25 countries outside the U.S. [22] - The company reached an agreement with the French Economic Committee for Health Products on reimbursement pricing for InSibiri for BBS and POMC lipid deficiencies [22] - The estimated prevalence of acquired hypothalamic obesity (HO) in Europe is approximately 10,000 patients, making it a significant market opportunity [25] Company Strategy and Development Direction - The company is preparing for the launch of InSibiri in acquired hypothalamic obesity, pending FDA approval, with a PDUFA date set for December 20, 2025 [11][21] - Rhythm aims to establish InSibiri as a foundational treatment for acquired HO and educate payers to secure long-term access for patients [17][19] - The company plans to initiate a phase three study with Bivamelagon and acquired HO next year, with further timing defined after regulatory feedback [14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming launch of InSibiri for acquired HO, highlighting strong engagement with physicians and payers [11][21] - The regulatory dialogue has been productive, with both the FDA and EMA accepting regulatory filings for HO [6] - Management noted that the company is well-positioned for sustained long-term growth, supported by a strong balance sheet and upcoming data readouts [5][28] Other Important Information - Rhythm raised approximately $189 million in net proceeds from a follow-on equity offering completed in July 2025 [27] - The company ended Q3 2025 with $416.1 million in cash, providing at least 24 months of operational runway [28][33] - R&D expenses for Q3 were $46 million, while SG&A expenses were $52.4 million, reflecting increased costs associated with the upcoming launch [32][33] Q&A Session Summary Question: Can you share your latest thinking on the trial design for your phase three HO study? - Management indicated that the HO trial will be a double-blind randomized controlled trial, with expectations for a full year of data [39] Question: Can you give us more insight into the efficacy endpoints for Prader-Willi syndrome? - Management stated that success will be defined by a BMI % change, with a target of a 5% threshold in BMI decrease over 52 weeks [42][45] Question: Can you discuss the drivers behind the changes to the ongoing Prader-Willi trial? - Management explained that the trial was updated to allow patients to continue beyond the initial six months if they wish, and discussions about adding sites were precautionary [49] Question: Any updates on conversations with payers regarding the HO launch? - Management expressed optimism based on feedback from payers and indicated that they would work through the reimbursement process even if specific policies are not in place at the time of prescription [52][66] Question: How should investors think about the launch curve in hypothalamic obesity? - Management noted that while they are confident in execution, the ramp-up may take time due to the need for patient diagnosis and education [56][59]