BioMarin Pharmaceutical (BMRN) Conference Call Summary Company Overview - **Company**: BioMarin Pharmaceutical Inc. - **Event**: 2025 Conference on September 03, 2025 - **Key Speaker**: Greg Friberg, Chief R&D Officer Key Priorities and Pipeline Developments - **BMN 333 Program**: Focus on long-acting C-type natriuretic peptide for achondroplasia, considered a top priority for the company [3][4] - **Voxzogo Data**: Anticipation of hypochondroplasia data in the first half of next year, with plans to file in the U.S. and Europe for adolescents [3][4] - **BMN 401**: Acquired from Innozyme Pharma, a first-in-class therapy for ENPP1 deficiency, with the ENERGY3 study results expected in the first half of next year [4][5] Business Development Strategy - **Focus on Quality**: The company prioritizes quality over quantity in business development, seeking both early and late-stage assets [6][7] - **Areas of Interest**: Strong interest in skeletal conditions and enzyme replacement therapies, as well as adjacent opportunities in neuromuscular and pediatric neurologic indications [7][8] BMN 333 Details - **Unique Characteristics**: BMN 333 has a longer half-life compared to existing CNP therapies, allowing for increased dosing without the side effects associated with high Cmax levels [10][12] - **Phase 1 Study Results**: Initial studies show BMN 333 achieving three times the area under the curve (AUC) compared to other long-acting CNPs, suggesting potential for increased linear growth [13][14] Future Study Plans - **Phase 2/3 Study**: Plans to initiate a combined phase 2/3 study in achondroplasia patients, comparing BMN 333 to Voxzogo without a placebo arm [19][20] - **Focus on Health and Wellness**: The goal is to not only measure linear growth but also improve overall health and wellness for patients [21][22] Innozyme Acquisition and BMN 401 - **Acquisition Rationale**: The Innozyme molecule (BMN 401) targets ENPP1 deficiency, a severe genetic disorder with high mortality in infants [33][34] - **Ongoing Studies**: The ENERGY3 study aims to normalize biochemistry and assess functional impacts on bones in children aged 1-12 [34][35] - **Future Studies**: Plans for a pivotal adult study (ENERGY4) and potential studies in infants are being developed [36][43] Conclusion - **Strategic Positioning**: BioMarin is well-positioned with a robust pipeline and financial resources to explore new opportunities in rare diseases, particularly focusing on genetically defined conditions [5][8] - **Commitment to Patients**: The company emphasizes its commitment to improving patient outcomes and wellness through innovative therapies and transparent data sharing [23][24]

Group 1 - The article is authored by Terry Chrisomalis, who operates the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace, offering a two-week free trial for new subscribers [1] - Biotech Analysis Central provides a library of over 600 biotech investing articles, a model portfolio of more than 10 small and mid-cap stocks, and various analysis and news reports to assist healthcare investors [2] Group 2 - The service is priced at $49 per month, with a yearly plan available at a discounted rate of $399, reflecting a 33.50% discount [1] - The analyst has no current stock or derivative positions in any mentioned companies and does not plan to initiate any within the next 72 hours [3]

Core Insights - BioMarin Pharmaceutical (BMRN) reported Q2 2025 adjusted EPS of $1.44, exceeding the Zacks Consensus Estimate of $1.03, with a 50% year-over-year increase driven by higher product sales and lower operating expenses [1][9] - Total revenues reached $825.4 million, reflecting a 16% year-over-year increase, surpassing the Zacks Consensus Estimate of $766.2 million [1][9] Revenue Breakdown - Product revenues totaled $813 million, a 16% year-over-year increase, primarily due to higher sales from Voxzogo, Palynziq, Vimizim, and Aldurazyme, partially offset by lower Kuvan sales [2] - Voxzogo generated $221 million in sales, up 20% year over year, exceeding the Zacks Consensus Estimate of $219 million [3] - Enzyme Therapies sales rose 15% year over year to $555 million, driven by increased patient demand and large government orders [4] - Palynziq injection sales increased 20% year over year to $106 million, surpassing both the Zacks Consensus Estimate and internal model estimates [5] - Vimizim sales rose 21% year over year to $215 million, beating both the Zacks Consensus Estimate and internal model estimates [6] - Aldurazyme sales totaled $56 million, up 44% year over year, attributed to favorable order fulfillment timing [6] Financial Guidance - BioMarin revised its 2025 revenue forecast to $3.13-$3.20 billion, reflecting an 11% year-over-year increase at the midpoint [11] - Adjusted EPS guidance was raised to $4.40-$4.55, indicating a 27% growth over the previous year at the midpoint [14] - The company expects Voxzogo sales to be between $900-$935 million, with higher revenues anticipated in the second half of the year [12] Pipeline Developments - The acquisition of Inozyme added BMN 401, an investigational enzyme replacement therapy for rare disorders, with interim results expected in early 2026 [18][19] - BioMarin is advancing its CANOPY clinical program for Voxzogo, targeting additional indications with data expected in 2026 [22] - BMN 333, a long-acting formulation of CNP, is set to enter a phase II/III study in 2026, aiming for a potential launch in 2030 [23] - The company plans to file for expanded use of Palynziq in adolescents based on positive late-stage study results [21]

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]

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 [12][18] - Non-GAAP diluted earnings per share increased to $1.44, reflecting a growth rate more than three times that of revenue growth [17] - 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 [12][20] - Enzyme Therapies revenue rose 15% year over year to $555 million, with Palynziq and Vimizim contributing significantly to growth [14][28] - Rocadian revenue was $9 million in Q2, primarily from the U.S. and Italy [14] Market Data and Key Metrics Changes - The company expects second half VOXZOGO revenue to be higher than the first half, with a full year target of $900 million to $935 million [13][14] - The company anticipates continued strong growth throughout the remainder of 2025, leading to an increase in full year guidance for total revenues [9][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 2026 [7][31] - The acquisition of Inozyme broadens the enzyme therapies portfolio, with BMN 401 expected to address ENPP1 deficiency [8][35] - The company aims to diversify its growth strategy through strategic business development transactions [9][10] 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 2025 and beyond [10][19] - The management highlighted the importance of patient adherence and support programs to maintain high compliance rates [66][68] 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 Palynziq to include adolescents in the U.S. and EU in the second half of 2025 [32] Q&A Session Summary Question: Clarification on BMN 333 and safety profile - Management confirmed that the agent referred to is TransCon CNP and stated that no unexpected safety issues have been observed in the healthy volunteer study [39][40] Question: Thoughts on competitive landscape for achondroplasia - Management noted that while early data shows growth hormone combinations may provide short-term increases, long-term benefits remain uncertain [44][45] Question: Contributions to VOXZOGO guidance - Management explained that the adjustment in guidance is due to order timing and a better understanding of trends as the year progresses [48][50] Question: Update on ITC proceedings - Management expects initial determination from the ITC on June 8, 2026, with a completion date targeted for October 8, 2026 [53][54] Question: Details on BMN 333 superiority trial design - Management refrained from providing specific details on powering but expressed confidence in the biological hypothesis being tested [56][58] Question: Update on hypochondroplasia and patient enrollment - Management emphasized the importance of early diagnosis and treatment for hypochondroplasia, leveraging existing relationships to raise awareness [100][101]

Financial Performance - Total Revenues for Q2 2025 reached $825 million, a 16% year-over-year increase[17, 22] - VOXZOGO revenue in Q2 2025 was $221 million, representing a 20% year-over-year growth[17, 22] - Enzyme Therapies revenue in Q2 2025 was $555 million, a 15% year-over-year increase[17, 22] - Non-GAAP Operating Margin for Q2 2025 was 39.9%, an increase of 8.7 percentage points year-over-year[25] - Non-GAAP Diluted EPS for Q2 2025 was $1.44, a 50% increase year-over-year[17, 30] - Operating Cash Flow for Q2 2025 was $185 million, a 55% increase year-over-year[30] - The company increased FY'25 Total Revenues guidance to $3,125 million to $3,200 million[24, 34] - The company increased FY'25 Non-GAAP Diluted EPS guidance to $4.40 to $4.55[33, 34] Product and Pipeline Updates - VOXZOGO's contribution to full-year 2025 Total Revenues is expected to be in the range of $900 million to $935 million[24, 34] - BMN 333 Phase 1 data showed >3x AUC PK free CNP levels vs other long-acting CNP[17, 45] - Submissions for PALYNZIQ age eligibility expansion for adolescents (12-17 y/o) in the U S and Europe are planned for 2H 2025[3, 48, 51, 56]

Financial Performance - Total Revenues for Q2 2025 were $825 million, representing a 16% increase year-over-year, driven by a 20% growth in VOXZOGO revenue from new patients [5][12][13] - GAAP Net Income increased by $134 million to $241 million, a 125% increase compared to Q2 2024, primarily due to higher gross profit and lower SG&A expenses [5][13][14] - Non-GAAP Income rose by $93 million to $282 million, reflecting a 49% growth year-over-year [5][13][14] Earnings Per Share - GAAP Diluted EPS for Q2 2025 was $1.23, a 124% increase year-over-year [5][13][14] - Non-GAAP Diluted EPS was $1.44, representing a 50% increase compared to the same period in 2024 [5][13][14] Business Development - The acquisition of Inozyme was completed on July 1, 2025, enhancing the company's portfolio with BMN 401 for ENPP1 Deficiency treatment [4][5][13] - BMN 333, a potential treatment for achondroplasia, showed promising early data and is expected to enter pivotal studies in 2026 [3][4][9] Pipeline Progress - VOXZOGO revenue increased by 20% year-over-year, with treatment available for children with achondroplasia in 51 countries [9][10][12] - The company is advancing its CANOPY clinical program for VOXZOGO in additional indications, with pivotal study data expected in 2026 [9][10][12] Operational Efficiency - GAAP Operating Margin expanded to 33.5%, a 16.6 percentage point increase year-over-year, while Non-GAAP Operating Margin reached 39.9%, an 8.7 percentage point increase [13][14] - R&D expenses decreased year-over-year due to focused investment following a strategic portfolio review [13][14] Cash Flow and Guidance - Operating cash flows totaled $185 million in Q2 2025, a 55% increase compared to the same period in 2024 [13][14] - The company raised its full-year 2025 guidance for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS, reflecting continued growth in patient demand [13][17]