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 based on data from multiple biobanks, indicating a consistent finding across different cohorts [18][19] - The company is comparing the patient identification and market development strategies for ADH-1 to those used in the ATTR field, which has seen significant improvements in diagnosis rates [22] 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 for encaleret includes differentiating the product through urine calcium reduction and long-term safety, aiming to establish it as a standard of care [27][28] 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][48] - The company is optimistic about the upcoming data presentations that may address skepticism regarding the efficacy of encaleret in chronic hypoparathyroidism [25][26] 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 compelling clinical data and new treatment guidelines in driving diagnosis rates for ADH-1 [13][14] 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][59] Question: What is the confidence level regarding the safety profile of infigratinib? - The phase two data provided confidence in a clean safety profile, and the company expects to continue monitoring safety closely in phase three [65][66]