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]