Company Overview - BioMarin has undergone significant changes in strategy, structure, pipeline, and portfolio since December 2023 [2] - The company has demonstrated strong execution and progress, particularly noted in the last quarter [2] Financial Performance - BioMarin reported a 16% revenue growth in the last quarter, continuing a trend of strong financial and commercial performance [3]

PresentationGood morning, everyone, and welcome to this good morning. Welcome to Morgan Stanley Global Healthcare Conference. I'm Sean Laaman, Head of the SMid-Cap Biotech Equity Research team here at the firm. Before we commence, for important disclosures, please see Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. And if you have any questions, please reach out to your Morgan Stanley sales representative. For this session, we have the pleasure of hosting BioMarin, o ...

Summary of BioMarin Pharmaceutical FY Conference Call Company Overview - **Company**: BioMarin Pharmaceutical Inc. (NasdaqGS: BMRN) - **Date of Conference**: September 09, 2025 - **Speakers**: Alexander Hardy (President and CEO), Brian Mueller (CFO) Key Points Financial Performance - BioMarin reported a **16% revenue growth** in the last quarter, indicating strong financial and commercial performance [6] - The company is on track to meet its **$4 billion revenue guidance for FY27**, with updates expected later this year [28] Pipeline Developments - Positive Phase 1 results for **BMN 333**, a long-acting CNP therapy, have led to plans for a Phase 2/Phase 3 study [7][42] - Progress with **Palynziq** in adolescents, with results presented at a recent meeting in Japan [7] - The acquisition of **InnoSign** for **$270 million** is expected to contribute significantly, with peak sales potential estimated at **$500 million to $600 million** [27] Business Development Strategy - BioMarin is focusing on business development (BD) to supplement internal innovation, with a clear strategy in place [8] - The company is actively monitoring the competitive landscape in China, particularly in genetically defined conditions [10][11] AI Integration - AI is being leveraged across various business functions, including R&D, regulatory documentation, manufacturing, and patient identification [12][14] - The company aims to adopt a "fast follower" strategy in AI until 2027, with ambitions to lead in AI applications for genetically defined conditions thereafter [12][13] Regulatory Environment - Positive interactions with the FDA, particularly regarding rare diseases, have been noted [15][17] - The company is monitoring potential impacts from tariffs and the Most Favored Nation (MFN) policy, with a significant portion of revenue coming from outside the U.S. [18][19] Competitive Positioning - Voxzogo accounts for **one-third of BioMarin's revenue**, with 75% of that revenue generated outside the U.S. [33][36] - The company believes it has a competitive advantage due to its geographic footprint and early treatment indications for achondroplasia [36][37] Future Outlook - The company is optimistic about the growth potential of Voxzogo and its pipeline, with a total addressable patient population of **420,000** across multiple indications [40] - The timeline for the registrational study for BMN 333 is projected for **2030**, with ambitions to expedite this process [44] Market Dynamics - The competitive landscape in the PKU space is evolving, but BioMarin believes Palynziq will continue to grow due to its unique patient population focus [50] - The company is confident in its ability to navigate market changes and maintain growth through both internal and external innovation strategies [54] Investment Strategy - BioMarin is focused on long-term growth, balancing internal innovation with external acquisitions to enhance profitability and cash flow [52][54] - The management emphasizes the importance of investing in future revenue growth over immediate returns to shareholders [55] Additional Insights - The company is committed to innovation in genetically defined conditions, which is seen as the core of its business strategy [58] - Future discussions may include updates on other pipeline assets not covered in this call [56]

On Saturday, BioMarin Pharmaceutical Inc. BMRN released new Phase 3 PEGASUS study results on Saturday.The data show that its therapy Palynziq (pegvaliase-pqpz) significantly reduced blood phenylalanine (Phe) levels in adolescents with phenylketonuria (PKU), a rare metabolic disorder.The trial enrolled 55 patients aged 12–17, with 36 receiving Palynziq and 19 continuing on diet alone. After 72 weeks, 45% of Palynziq patients achieved at least a 50% reduction in Phe from baseline, many reaching guideline-reco ...

Core Insights - The Phase 3 PEGASUS study shows a 49.7% reduction in mean blood phenylalanine (Phe) levels in adolescents aged 12-17 treated with PALYNZIQ compared to diet alone [1][2][3] - BioMarin plans to submit the PEGASUS study data to global health authorities in the second half of 2025 to expand PALYNZIQ's indication to include adolescents [1][4] Study Details - The PEGASUS study involved 55 adolescents, with 36 receiving PALYNZIQ and 19 on diet alone, showing significant efficacy in lowering blood Phe levels [2][8] - At baseline, the mean age of participants was 14.3 years, with a mean blood Phe level of 1026.4 µmol/L; nearly half had levels above 1000 µmol/L [2] - After 72 weeks, 45.2% of participants in the PALYNZIQ group achieved a reduction of 50% or more in blood Phe concentrations [2][3] Efficacy Results - The mean percentage change in blood Phe concentration from baseline for the PALYNZIQ group was -49.7%, while the diet-only group showed a negligible change of -0.3% [3] - Significant achievements in blood Phe levels included 51.6% of PALYNZIQ participants reaching levels below 600 µmol/L, and 38.7% below 360 µmol/L [3] Safety Profile - The safety profile of PALYNZIQ in adolescents was consistent with its known profile in adults, with 5.6% of adverse events being serious [3][4] - The ongoing extension phase of the PEGASUS trial will continue to evaluate long-term safety and efficacy [3][4] Company Commitment - BioMarin has been dedicated to advancing treatments for PKU for over two decades and aims to provide effective solutions for adolescents transitioning to adulthood [4][12] - PALYNZIQ is the first enzyme substitution therapy approved for adults with PKU, and the company seeks to expand its use to younger populations [4][10]

Group 1 - The session features Mohit Bansal, a Senior Equity Analyst at Wells Fargo, who specializes in biotech and pharma [1] - Greg Friberg, the EVP and Chief Research and Development Officer at BioMarin, is present for the discussion [1]

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 - The company has undergone an organizational transformation focusing on operating margins and prioritizing high-impact programs for genetically defined conditions [3][12] - The company has cash and free cash flow, indicating a good position to pursue additional innovations [7] Business Line Data and Key Metrics Changes - The company is advancing several key programs, including BMN333 for long-acting CNP, with pivotal studies expected to begin in the late second half of next year [5][22] - The Voxogo program for achondroplasia is being developed with a focus on health and wellness factors beyond just growth velocity [15][27] Market Data and Key Metrics Changes - The company is actively engaging in business development to assess external science alongside internal innovations, indicating a strategic approach to market opportunities [6][12] - The hypochondroplasia study has recruited faster than anticipated, reflecting strong unmet needs in the market [76] Company Strategy and Development Direction - The company is committed to developing therapies for genetically defined conditions and shaping the way these diseases are defined and treated [12][13] - The strategy includes a combination of internal and external innovations, with a focus on business development to enhance the pipeline [6][7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory landscape, noting that the FDA appears to be more flexible regarding trial endpoints [47] - The company aims to have a registration package ready for approval by 2030, with efforts to expedite the process [46] Other Important Information - The company is preparing for a pivotal study for BMN351, targeting Duchenne muscular dystrophy, with data expected by the end of the year [51][58] - The integration of the Enzyme program (BMN401) is progressing well, with expectations for significant patient benefits [84] 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 [14][15] 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 [10][11] Question: What are the expectations for the upcoming BMN333 trial? - The trial is designed without a placebo arm to facilitate enrollment, with a focus on comparative effectiveness against Voxogo [44][46] Question: What is the anticipated timeline for the BMN351 data release? - Data for BMN351 is expected by the end of the year, with a focus on achieving significant dystrophin levels in patients [56][59] Question: How does the company view the potential for combination therapies? - Management believes that rational combinations of therapies will be essential for future treatments, although growth hormone may not play a major role [36][37]

Core Insights - The conference is being kicked off by a senior biotech analyst from Cantor Fitzgerald, indicating a focus on the biotechnology sector [1] - The presence of Greg Friberg, Chief R&D Officer from BioMarin, suggests that the company is highlighting its research and development efforts [1] Company Highlights - BioMarin is represented by its Chief R&D Officer, indicating the company's commitment to sharing insights on its research initiatives [1] - The event is taking place on the East Coast, which may suggest a strategic location for engaging with investors and industry stakeholders [1]

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]