Rhythm(US:RYTM)2025-05-07 13:02Financial 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]