Rhythm(US:RYTM)2025-11-04 14:00Financial 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 [28] - Year-over-year, net product revenues increased by $18 million, or 54%, compared to Q3 2024 [32] 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 [4][28] - 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 - In the U.S., $38.2 million or 74% of Q3 net revenue was generated, while $13.1 million or 26% came from international markets [28] - International revenue decreased by $3.4 million quarter-over-quarter, but patients on reimbursed therapy increased at a low double-digit percentage [30] Company Strategy and Development Direction - The company is preparing for the launch of InSibiri for hypothalamic obesity, pending FDA approval, with a PDUFA date set for December 20, 2025 [5][11] - Rhythm is focused on engaging with physicians and payers to establish InSibiri as a foundational treatment for acquired hypothalamic obesity [20] - The company aims to complete enrollment of the RM-718 weekly phase 2 study in HO patients during Q1 2026 and initiate a phase 3 study with Bivamelagon next year [12][13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming launch of InSibiri for hypothalamic obesity, highlighting strong growth in BBS and promising regulatory dialogues [5][11] - The company anticipates that the upcoming data readouts and regulatory approvals will position it for sustained long-term growth [4][5] Other Important Information - Rhythm raised approximately $189 million in net proceeds from a follow-on equity offering completed in July 2025, ending Q3 with $416.1 million in cash [28] - The company expects to have sufficient cash to fund planned operations for at least 24 months [34] Q&A Session Summary Question: Can you share your latest thinking on the trial design for your phase 3 HO study? - The HO trial will be a double-blind randomized controlled trial, with expectations for a full year of data [39] Question: Can you provide more details on the efficacy endpoints for Prader-Willi syndrome? - Success will be defined by a BMI percent change, with a target of a 5% threshold in BMI decrease in 52 weeks [45] Question: What are the drivers behind the changes to the ongoing Prader-Willi trial? - The trial was extended to allow patients to continue beyond the initial six months if they wish, and there is a possibility of adding additional sites [51] Question: How should investors think about the launch curve in hypothalamic obesity? - The company has a solid foundation based on learnings from the BBS launch, but the process of getting patients diagnosed may take time [60][62] Question: What is the anticipated gross to net in HO relative to BBS? - The gross to net mix is uncertain, but the company expects to work through reimbursement processes similar to those for BBS [66]