Rhythm Pharmaceuticals (RYTM) Q2 2025 Earnings Call August 05, 2025 08:00 AM ET Speaker0Good day, and thank you for standing by. Welcome to the Rhythm Pharmaceuticals Q2 twenty twenty five Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. To ask a question during the session, you will need to press 11 on your telephone.You will then hear an automated message advising that your hand is raised. Ple ...

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]