Financial Data and Key Metrics Changes - The company has undergone an organizational transformation focusing on operating margins and prioritizing high-impact programs for genetically defined conditions [6][15] - The company has reported positive cash flow and is in a good position to seek additional innovations [11] Business Line Data and Key Metrics Changes - The company is advancing several key programs, including BMN 333 for long-acting CNP, with pivotal studies expected to begin in the second half of next year [8][25] - The Palynziq project for PKU is set for adolescent expansion filing, indicating progress in their pipeline [7] Market Data and Key Metrics Changes - The company is actively exploring external innovation opportunities alongside internal R&D efforts, indicating a dual approach to growth [10][11] - The company is focusing on the unmet needs in various markets, particularly in rare diseases, which is reflected in their strategic direction [21][88] Company Strategy and Development Direction - The company is committed to developing therapies for genetically defined conditions and shaping the medical landscape for these diseases [15] - There is a strong emphasis on leveraging both internal and external scientific advancements to enhance their product offerings [9][10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of their long-acting CNP program, predicting significant growth benefits based on animal model data [22][24] - The regulatory landscape is perceived to be more flexible, allowing for a one-year endpoint for pivotal studies, which could expedite the approval process [55][56] Other Important Information - The company is preparing for a registration study for BMN 333, which will not include a placebo arm to facilitate patient enrollment [52][60] - The company is also advancing its DMD asset, BMN 351, with data expected by the end of the year, aiming for a significant dystrophin level increase [62][68] Q&A Session Summary Question: Can you discuss the significance of the recent data generated for Voxogo? - Voxogo is seen as a safe and effective therapy for achondroplasia, with ongoing efforts to communicate its value to patients beyond just growth metrics [17][18] Question: How does the company prioritize assets in R&D? - The company applies rigorous scientific standards and market research to prioritize programs, ensuring they align with patient needs and market demands [12][13] Question: What are the expectations for the upcoming BMN 351 data? - The company aims for a 10% dystrophin level in muscle tissue, with initial results expected to show a 3% to 5% range at the six-month mark [67][68] Question: How does the company view the combination of CNP with growth hormones? - Management expressed skepticism about the efficacy of growth hormones in achondroplasia, emphasizing the need for targeted therapies instead [43][44] Question: What is the bar for success in the hypochondroplasia study? - The study is powered to measure growth velocity acceleration similar to Voxogo, with expectations for potentially larger effects based on prior data [91][92]

Financial Data and Key Metrics Changes - Total revenues grew 16% in Q2 2025 and 15% year-over-year compared to the same periods in 2024, driven by strong global demand and new patient starts [13][19] - Non-GAAP diluted earnings per share increased to $1.44, reflecting a growth rate more than three times that of revenue growth [18] - Operating cash flow reached $185 million in Q2, a 55% increase compared to the same period in 2024 [18] Business Line Data and Key Metrics Changes - VOXZOGO revenue increased 20% year-over-year to $221 million, supported by global expansion and new patient starts [13][20] - Enzyme Therapies revenue rose 15% year-over-year to $555 million, with Palynziq and Vimizim contributing significantly to growth [15][26] - Rocadian revenue was $9 million in Q2, primarily from the U.S. and Italy [15] Market Data and Key Metrics Changes - The company expects second half VOXZOGO revenue to be higher than the first half, with full-year revenue guidance adjusted to between $900 million and $935 million [14][15] - The company anticipates continued strong growth in the second half of 2025, leading to an increase in full-year guidance for total revenues [11][19] Company Strategy and Development Direction - The company plans to advance BMN 333, a long-acting therapy for achondroplasia, to a registrational Phase 2/3 study in the first half of next year [9][28] - The acquisition of Inozyme broadens the enzyme therapies portfolio, with a focus on BMN 401 for ENPP1 deficiency [10][31] - The company aims to augment its portfolio with strategic business development transactions to diversify growth strategies [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to deliver strong growth and value creation for stakeholders through the remainder of 2025 and beyond [11][19] - The management highlighted the essential nature of their medicines and strong patient support programs as key factors in maintaining availability for patients [26] Other Important Information - The company expects to account for the Inozyme acquisition as an asset purchase, impacting financial results in 2025 [19] - The company is on track to submit applications for age extension for Palynziq to include adolescents in the U.S. and EU in the second half of this year [30] Q&A Session Summary Question: Clarification on BMN 333 and safety profile - Management confirmed that the agent referred to is Trans Con CNP and stated that no unexpected safety issues have been observed in the healthy volunteer study [36][38] Question: Thoughts on competitive landscape for achondroplasia - Management noted that while early data showed added growth with growth hormone, long-term benefits remain uncertain, and further data is needed to assess the impact of combinations [42][45] Question: Contributions to VOXZOGO guidance - Management explained that the adjustment in guidance is primarily due to shifting large orders and a better understanding of revenue trends as the year progresses [50][52] Question: Update on ITC proceedings - Management expects the initial determination from the ITC on June 8, 2026, with a completion date targeted for October 8, 2026 [56][58] Question: Details on BMN 333 trial design - Management refrained from providing specific details on powering but expressed confidence in the biological hypothesis being tested [58][60] Question: Update on citizen petition with the FDA - Management stated that the FDA is reviewing the citizen petition and no updates are expected until the PDUFA date of November 30 [88][90]