BridgeBio(BBIO) - 2025 FY - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company reported a significant increase in the number of treatment-naive patients on acoramidis, indicating a positive trend in commercial execution [47][53] - The gross-to-net ratio is expected to stabilize in the range of 30% to 40%, reflecting a more representative payer mix as of Q2 [55][56] Business Line Data and Key Metrics Changes - The ADH-1 program is transitioning from a single-center phase two study to a global phase three study with over 25 sites, which may lead to a more heterogeneous patient population [3][4] - In the phase two study of ADH-1, approximately 70% of patients responded to encaleret, and the company aims to replicate this success in phase three [16][38] Market Data and Key Metrics Changes - The prevalence of ADH-1 has been estimated at about 1 in 25,000, consistent across multiple biobanks, indicating a stable understanding of the patient population [18][19] - The company is comparing its market potential to that of ATTR and X-linked hypophosphatemia, suggesting a similar trajectory in patient identification and market development [22][23] Company Strategy and Development Direction - The company is focused on driving disease education and the utilization of genetic testing to improve diagnosis rates for ADH-1 [19] - The strategy includes leveraging compelling clinical data and new treatment guidelines to enhance awareness and diagnosis of the condition [13][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the launch of acoramidis, noting that the share of treatment-naive patients continues to increase, which is a positive indicator for long-term success [47][52] - The company is optimistic about the upcoming data presentations that may address skepticism regarding the efficacy of encaleret in chronic hypoparathyroidism [25][66] Other Important Information - The company is preparing for a webinar to provide more details on the ADH-1 program, indicating a commitment to transparency and stakeholder engagement [3] - Management highlighted the importance of long-term safety and efficacy data for encaleret, particularly in relation to urine calcium reduction, which could differentiate it in the market [27][66] Q&A Session Summary Question: What are the risks in translating phase two findings to phase three for ADH-1? - The main difference is the number of sites and patients, with phase three being a global study involving over 25 sites [3] Question: Why is PTH replacement therapy not optimal for ADH-1 patients? - PTH replacement does not address the root cause of the condition, and urine calcium levels remain high even with treatment [10][11] Question: How is the company addressing patient identification for ADH-1? - The company is focusing on compelling clinical data, new treatment guidelines recommending genetic testing, and increased awareness through ICD-10 coding [13][14][18] Question: What is the expected impact of competition on acoramidis? - The company has not seen significant pressure from competitors and maintains a strategy of parity access without needing to offer larger rebates [57][58] Question: How does the company view the potential for generic competition for tafamidis? - The company believes that the existing patents provide strong protection against generics, and the discontinuation of Vyndaqel complicates entry for potential competitors [60][61]