BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is among the best stocks with the highest upside potential. According to the announcement on September 8, 2025, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) presented new data from 14 studies at the American Society for Bone and Mineral Research 2025 (ASBMR) Annual Meeting in Seattle. The presentation also outlined the results of treatment with VOXZOGO (vosoritide), highlighting both anatomical improvements in spinal morphology and continued efficacy in children who ...

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]