Financial Data and Key Metrics Changes - Global revenue for Q2 2025 was $48.5 million, representing a 29% increase quarter over quarter [38] - U.S. revenue accounted for 66% of total revenue, amounting to $32 million, while international revenue was $16.5 million, making up 34% [38] - Year-over-year, net product revenues increased by $19.4 million or 67% compared to Q2 2024 [41] Business Line Data and Key Metrics Changes - The number of reimbursed patients on therapy in the U.S. grew at mid-single-digit percentage rates, with a 12% increase in the global number of patients on therapy [38] - The cumulative number of BBS prescribers grew by 38% from Q2 2024 to Q2 2025, and there was a 9% growth in cumulative prescribers from Q1 2025 to Q2 2025 [22] - The FDA approved label expansion for Encepria down to two years of age, leading to increased prescriptions for younger patients [23] Market Data and Key Metrics Changes - International sales growth was primarily driven by increased sales in France, the UK, and Italy, with early access programs for hypothalamic obesity patients contributing to significant patient growth [40] - The appreciation of the euro and other currencies contributed approximately $1.2 million to the revenue increase [40] - The company is seeing a steady increase in the overall number of patients on therapy in the international region, particularly in countries with early access programs [28] Company Strategy and Development Direction - The company is focused on long-term growth, particularly in the BBS and acquired hypothalamic obesity markets, with expectations of sustained growth over the next 15 years [8] - The company plans to complete U.S. and European regulatory filings for cetaminotide in Q3 2025 and aims to launch Encepria in hypothalamic obesity pending FDA approval [9][26] - The company is building out its team in Japan and focusing on regulatory, medical affairs, marketing, and market access [31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the projected epidemiology for acquired hypothalamic obesity, estimating 5,000 to 10,000 patients in the U.S. and similar numbers in Europe [10] - The management highlighted the importance of the recent oversubscribed equity raise, which strengthens the company's balance sheet and provides a cash runway of at least 24 months [37] - Management noted that the company is well-capitalized and positioned for future growth, with a focus on developing next-generation compounds [9][10] Other Important Information - The company reported a GAAP EPS net loss of $0.75 per share, including $0.02 per share from accrued dividends on convertible preferred stock [44] - R&D expenses for Q2 were $42.3 million, up from $30.2 million in the same quarter last year, primarily due to increased headcount and clinical trial costs [41][42] - The company is preparing for a significant launch in the hypothalamic obesity market, leveraging insights gained from the BBS launch [26] Q&A Session Summary Question: Upcoming pipeline catalyst regarding Prader Willi data - Management characterized the Prader Willi study as exploratory, with a focus on higher dosing and longer duration compared to previous trials [48][49] Question: Expected follow-up for Prader Willi study - Management indicated that patients will continue treatment beyond the six-month trial period for long-term data collection [56] Question: Confidence in higher prevalence estimates for hypothalamic obesity - Management expressed confidence in the higher end of the prevalence range based on claims data and field team insights [72] Question: Growth expectations for international revenue - Management noted that while currency effects contributed to recent growth, they expect continued strong performance in international markets despite potential seasonal fluctuations [75]

Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported global sales revenue of $37.7 million for Omsivri in Q1 2025, with a 14% increase in the number of patients on reimbursed therapy globally during the quarter [34] - Net product revenues increased by $11.7 million, or 45%, compared to Q1 2024 [37] - Cash on hand at the end of the quarter was $314.5 million, sufficient to cover planned operations into 2027 [41] Business Line Data and Key Metrics Changes - The demand for Omsivri, the only treatment addressing hyperphagia and obesity associated with Bardet-Biedl syndrome (BBS), remained strong, with a consistent number of new prescriptions leading to ongoing growth in patients on reimbursed therapy [19] - The inventory swing at specialty pharmacies affected revenue, with an $8.3 million inventory swing impacting Q1 results [35] - The international team is executing a country-by-country launch strategy for BBS, with significant contributions to revenues from France and Italy [10] Market Data and Key Metrics Changes - The U.S. percentage of overall product revenue decreased from 74% in Q4 2024 to 65% in Q1 2025 due to inventory swings [41] - Medicaid access for Emsivri has improved, with positive coverage decisions secured in states accounting for over 95% of covered Medicaid lives [22] Company Strategy and Development Direction - The company is on track for a Q3 filing with the FDA for cetmelanotide, with an in-person Type D meeting scheduled [8] - Rhythm Pharmaceuticals is focused on expanding access for Emsivri and preparing for the launch of INCIVRI for acquired hypothalamic obesity [22] - The company is also looking forward to the Bivomelagon Phase II readout in Q3 and ongoing studies in Prader-Willi syndrome and hypothalamic obesity [10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of cetmelanotide to transform the lives of patients with hypothalamic obesity, highlighting the medical complexity and unmet needs in this patient population [11] - The management team noted that the interactions with the FDA have been normal and responsive, indicating a positive outlook for the upcoming filing [8] - The company remains well-capitalized with a projected cash runway into 2027, allowing for continued investment in research and development [41] Other Important Information - The company reported a gross-to-net ratio for U.S. sales of 84.2%, consistent with previous quarters [37] - R&D expenses for Q1 2025 were $37 million, significantly lower than the previous year due to decreased costs associated with the acquisition of Bivomelagon [37] Q&A Session Summary Question: Can you talk about the non-responders highlighted? - Management noted that non-responders included patients who dropped out for various reasons, including inability to keep up with the trial and adverse reactions [46] Question: What is the real-world experience regarding patient compliance? - Management indicated that compliance rates are generally around 30% for patients, with expectations of better compliance in the hypothalamic obesity population [48] Question: Can you discuss the FDA meeting scheduled? - The meeting is to clarify what will be presented in the filing and ensure alignment with the FDA [69] Question: What is the payer mix for BBS patients? - Approximately 80% of the BBS patient population is covered by commercial and Medicaid, with Medicare being a smaller portion [71] Question: What is the expected revenue ramp for hypothalamic obesity? - Consensus estimates HO revenue ramping from around $115 million in 2026 to over $1 billion by 2030, with management expressing confidence in the launch [95]

Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported global sales revenue of $37.7 million for Q1 2025, with a 14% increase in the number of patients on reimbursed therapy globally during the quarter [32] - Net product revenues increased by $11.7 million, or 45%, compared to Q1 2024 [36] - Cash on hand at the end of the quarter was $314.5 million, projected to cover operations into 2027 [39] Business Line Data and Key Metrics Changes - Demand for Omsivri, the treatment for Bardet-Biedl syndrome (BBS), remained strong, with a consistent number of new prescriptions leading to ongoing growth in patients on reimbursed therapy [17][18] - The inventory swing at specialty pharmacies resulted in a net revenue decrease of $4.1 million compared to Q4 2024, despite strong underlying growth [35] Market Data and Key Metrics Changes - The international team is executing a country-by-country launch strategy for BBS, with revenue contributions from France and Italy continuing to grow [8] - Medicaid access for Emsivri has improved, with positive coverage decisions secured in states accounting for over 95% of covered Medicaid lives [21] Company Strategy and Development Direction - The company is on track for a Q3 filing with the FDA for cetmelanotide, with an in-person Type D meeting scheduled [6] - Rhythm Pharmaceuticals is focused on expanding its commercial efforts globally, particularly in the endocrinology specialty, which is critical for patients with hypothalamic obesity [22][86] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of cetmelanotide to transform the lives of patients with hypothalamic obesity, highlighting the medical complexity and unmet needs in this patient population [10][16] - The company anticipates a solid launch trajectory for acquired hypothalamic obesity, with a higher percentage of diagnosed and actively managed patients compared to BBS [94] Other Important Information - The company is well-capitalized with a projected cash runway into 2027, allowing for continued investment in R&D and commercial activities [39] - The company plans to present real-world data at upcoming medical conferences, which is expected to enhance understanding and interest in their therapies [28][31] Q&A Session Summary Question: Can you elaborate on the non-responders in the trial? - Management noted that several non-responders had good responses before dropping out, with reasons including inability to keep up with the trial and adverse reactions [44] Question: What is the real-world experience regarding patient compliance? - Compliance rates are generally around 30%, with expectations that the hypothalamic obesity population may see lower discontinuation rates due to the relief provided by treatment [46][47] Question: Can you discuss the FDA meeting scheduled? - The meeting is to clarify what will be presented in the filing and ensure alignment with the FDA [65] Question: What is the payer mix for BBS and expectations for HO? - Approximately 80% of the payer mix consists of commercial and Medicaid, with ongoing evaluations for the hypothalamic obesity opportunity [68] Question: What is the expected revenue ramp for HO? - Management expressed confidence in the launch potential, noting a higher percentage of diagnosed patients compared to BBS, but refrained from providing specific revenue guidance [94]