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 ...

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]

PresentationMohit BansalSenior Equity Analyst All right. Thank you very much for joining us today. My name is Mohit Bansal. I am one of the biotech pharma analysts here at Wells Fargo. And this is my first session of the morning. And I'm very happy to have, from BioMarin, Greg Friberg with us. Greg is the EVP and Chief Research and Development Officer at BioMarin. Thank you very much for being here. ...

BioMarin Pharmaceutical (BMRN) FY 2025 Conference September 04, 2025 08:45 AM ET Company ParticipantsMohit Bansal - MD & Co-Head of Therapeutics ResearchGreg Friberg - EVP, Chief Research & Development OfficerMohit BansalThank you very much for joining us today. My name is Mohit Bansal. I'm one of the biotech pharma analysts here at Wells Fargo. And this is my first session of the morning, and I'm very happy to have from BioMarin, Greg Fryberg with us.Greg is the EVP and Chief Research and Development Offic ...

BioMarin Pharmaceutical (BMRN) FY 2025 Conference September 04, 2025 08:45 AM ET Speaker0Thank you very much for joining us today. My name is Mohit Bansal. I'm one of the biotech pharma analysts here at Wells Fargo. And this is my first session of the morning, and I'm very happy to have from BioMarin, Greg Fryberg with us.Greg is the EVP and Chief Research and Development Officer at BioMarin. Thank you very much for being here.Speaker1Yes. Thanks for the opportunity to join you. Great. SoSpeaker0I mean, bef ...

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) 2025 Conference September 03, 2025 08:00 AM ET Company ParticipantsGreg Friberg - EVP, Chief Research & Development OfficerConference Call ParticipantsOlivia Brayer - Director & Senior Biotech AnalystOlivia BrayerOkay, great. Hey, good morning, everyone. My name is Olivia Breyer. I'm one of the Senior Biotech Analysts here at Cantor Fitzgerald. Thank you so much for being up early with us this morning to kick off our conference. We're really excited to start the day with someo ...