NO.2 GSK收购35Pharma，获得肺动脉高压新药 2月25日，葛兰素史克（GSK）宣布已与35Pharma达成收购协议，将以9.5亿美元现金收购后者的全部股 权。资料显示，35Pharma是一家拥有早期高血压药物资产的生物技术公司，目前的核心管线项目HS235 已在健康受试者中完成I期临床试验，即将在肺动脉高压（PAH）等人群中开展正式的临床研究。 点评：GSK此前在影响心脏和代谢系统疾病方面的药物管线规模较小，公司目前最大的财务压力来自于 其支柱性HIV药物（如Dovato）将在2028年前后遭遇专利到期。本次事件标志着这家制药巨头正向心血 管药物领域扩张。 NO.3 全球首款血友病基因疗法Roctavian退市 丨2026年2月26日星期四丨 NO.1 先声药业2025年归母净利润预增80.1%至93.9% 2月25日，先声药业发布盈利预告，公司2025财政年度将录得收入约人民币77亿元至78亿元，同比增长 约16.0%至17.6%；将录得归属于本公司权益股东的利润约人民币13亿元至14亿元，同比增长约80.1%至 93.9%。 点评：公司的收入及利润增长与创新药收入、对外授权许可收入以及集团持有 ...

Key Takeaways BioMarin reported Q4 EPS of 46 cents, beating estimates while revenues rose 17% Y/Y to $875M.Voxzogo sales jumped 31% to $273M in Q4, while BioMarin's enzyme therapies sales rose 13%.BioMarin will withdraw Roctavian after failing to find a buyer, recording a $119.2M charge in Q4.BioMarin Pharmaceutical (BMRN) reported fourth-quarter 2025 adjusted earnings per share of 46 cents, beating the Zacks Consensus Estimate of 25 cents. However, earnings declined 50% year over year, largely due to a $11 ...

BioMarin Pharmaceutical (NasdaqGS:BMRN) Q4 2025 Earnings call February 23, 2026 04:30 PM ET Company ParticipantsAlexander Hardy - CEOBrian Mueller - CFOCristin Hubbard - Chief Commercial OfficerGena Wang - Managing Director of Biotech Equity ResearchGreg Friberg - Chief Research and Development OfficerJason Gerberry - Managing DirectorMohit Bansal - Managing Director and Co-Head of Therapeutics ResearchPaul Matteis - Managing DirectorTraci McCarty - Head of Investor RelationsConference Call ParticipantsChri ...

BioMarin Pharmaceutical (NasdaqGS:BMRN) Q4 2025 Earnings call February 23, 2026 04:30 PM ET Speaker10Thank you for standing by. My name is Kate, and I will be your conference operator today. At this time, I would like to welcome everyone to the BioMarin Pharmaceutical fourth quarter and full year 2025 conference call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this ti ...

Core Insights - BioMarin Pharmaceutical reported strong third-quarter earnings, leading to a nearly 2% increase in stock price the following day, outperforming the S&P 500 index which rose by 1.2% [1] Financial Performance - The company's revenue for the quarter was $776 million, reflecting a 4% year-over-year increase [2] - Non-GAAP net income fell sharply by 88% to $22 million, equating to $0.12 per share, which was better than analysts' expectations of a non-GAAP net loss of $0.12 per share [2][3] - BioMarin missed revenue expectations, with analysts predicting over $782 million [3] Strategic Decisions - BioMarin announced plans to consider divesting its product Roctavian, which treats severe hemophilia A, aligning with its portfolio strategy to ensure continued patient access to the drug [4] Guidance Revision - The company raised its full-year 2025 revenue guidance to a range of $3.15 billion to $3.2 billion, up from a previous estimate of less than $3.13 billion to $3.2 billion [5] - Adjusted per-share net income guidance was reduced to $3.50 to $3.60, down from a prior forecast of $4.40 to $4.55 [5]

Core Insights - BioMarin Pharmaceutical (BMRN) reported Q3 2025 adjusted EPS of 12 cents, surpassing the Zacks Consensus Estimate of a loss of 15 cents, but reflecting an 87% year-over-year decline due to IPR&D charges of $221 million related to the acquisition of Inozyme Pharma [1][7][14] - Total revenues reached $776 million, a 4% increase year-over-year, but fell short of the Zacks Consensus Estimate of $784 million [1][14] Revenue Breakdown - Product revenues amounted to nearly $761 million, up 4% year-over-year, driven by Voxzogo and Palynziq, although partially offset by lower sales of Aldurazyme and Kuvan [4][10] - Voxzogo sales were $218 million, a 15% increase year-over-year, primarily due to new patient initiations outside the U.S., despite some unfavorable order timing [5][8] - Palynziq injection sales totaled $109 million, up 20% year-over-year, exceeding both the Zacks Consensus Estimate and internal model estimates [10] - Enzyme Therapies sales rose 1% year-over-year to $516 million, with Vimizim sales increasing 3% to $183 million, although it missed estimates [9][10] Financial Guidance and Outlook - BioMarin revised its full-year revenue guidance to $3.15-$3.20 billion, reflecting an increase of $25 million from previous estimates, indicating over 11% growth year-over-year at the midpoint [14][15] - Adjusted EPS guidance was lowered to $3.50-$3.60, down from $4.40-$4.55, implying a 1% growth over last year's level at the midpoint [16] - The company plans to divest Roctavian to focus on strategic growth and partnerships, while maintaining marketing efforts until a final decision is made [17][18] Pipeline Developments - BioMarin is advancing its CANOPY clinical program for Voxzogo, targeting additional indications, with data expected in the first half of 2026 [20] - The company is also pursuing multiple partnership and acquisition opportunities, with an estimated $4-$5 billion available for future growth initiatives [17] - Initial proof-of-concept data for BMN 351, an oral therapeutic for liver disease, is expected by the end of this year [23]

Financial Data and Key Metrics Changes - BioMarin reported an 11% year-over-year increase in total revenue, leading to an updated full-year revenue guidance of $3.15 billion, reflecting double-digit growth [5][11][15] - The company’s cash and investments balance reached approximately $2 billion at the end of Q3 2025, with operating cash flow of $369 million for the quarter and $728 million year-to-date [5][15] - Non-GAAP diluted earnings per share guidance was updated to between $3.50 and $3.60, with non-GAAP operating margin guidance adjusted to between 26% and 27% [15][16] Business Line Data and Key Metrics Changes - The enzyme therapies business unit achieved 8% year-to-date growth, primarily driven by Palynziq, although Q3 revenue was flat compared to Q3 2024 due to prior large orders [11][12] - Voxzogo, the treatment for achondroplasia, is expected to generate over $900 million in revenue for 2025, reflecting a 25% growth at the midpoint of guidance, with a year-to-date increase of 24% [7][11][19] - Palynziq also saw over 20% growth year-to-date, indicating strong demand and adherence among patients [11][18] Market Data and Key Metrics Changes - Approximately 75% of year-to-date Voxzogo revenue was generated outside the U.S., highlighting significant international growth opportunities [21][22] - The company is expanding its global footprint, with Voxzogo now available in 55 countries, and anticipates Q4 to be the highest revenue quarter for Voxzogo due to large contracted orders [19][21] Company Strategy and Development Direction - BioMarin is focusing on strategic investments in enzyme therapies and skeletal conditions, while also pursuing options to divest Roctavian to concentrate on core business units [9][10] - The company is preparing for the launch of Voxzogo's second indication for hypochondroplasia, with pivotal data expected in the first half of 2026 [8][23] - BioMarin aims to enhance its leadership in skeletal conditions by pursuing multiple indications for Voxzogo and advancing its next-generation product, BMN-333 [23][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving record commercial results for the full year, despite challenges from potential competition for Voxzogo [5][10] - The company acknowledged the uncertainty surrounding 2027 revenue projections due to competitive dynamics but remains committed to executing its growth strategy [17][39] - Management emphasized the importance of business development as a priority, with ongoing efforts to identify and acquire under-resourced assets [46][52] Other Important Information - BioMarin announced a $221 million charge for acquired in-process research and development related to the Enzyme Pharma acquisition, impacting both GAAP and non-GAAP financials [12][14] - The company is focused on improving early diagnosis for hypochondroplasia and expanding treatment access through various initiatives [25][26] Q&A Session Summary Question: Can you elaborate on the 2027 guidance and the changes since it was issued? - Management indicated that the original $4 billion 2027 outlook was reassessed due to potential competition for Voxzogo and other market dynamics, leading to a range of scenarios rather than a specific forecast [36][38] Question: Why were Voxzogo sales down quarter-over-quarter? - Management attributed the decline to timing shifts in larger orders and reaffirmed the overall revenue guidance for Voxzogo, emphasizing steady patient additions across all markets [42][44] Question: What is the strategy for business development moving forward? - Management highlighted a strong focus on early-stage collaborations and the pursuit of phase three and commercial assets, aiming to leverage BioMarin's capabilities to enhance value [46][52] Question: What are the expectations for the upcoming BMN-333 data? - Management indicated that they are looking for significant pharmacokinetic improvements over Voxzogo, with a focus on achieving clinical superiority in future trials [56][57]

Core Insights - The Chinese innovative drug market is experiencing strong growth, with product licensing totaling nearly $66 billion in the first half of 2025, particularly in the rare disease treatment sector [1] - The industry anticipates that entering markets with more established payment systems, such as Europe and the U.S., will help distribute R&D costs and balance commercial and social value [1] Company Overview - Huayi Lekan, founded by biologist Rao Yi in 2019, focuses on gene therapy drug development, with its leading product being a gene therapy for Hemophilia A [1][2] - GS1191 is the first domestically approved clinical gene therapy drug for Hemophilia A, with Phase III clinical trials expected to complete patient enrollment within the year [2] Market Strategy - The initial overseas market chosen by the company is Saudi Arabia, due to its significant number of hemophilia patients and favorable economic conditions for high-priced new drugs [2] - The company is exploring registration advantages in Saudi Arabia, where it can use Chinese clinical data for international multi-center studies [2] Clinical Development - Chinese companies may complete clinical trials faster due to a larger patient base, potentially reducing the time to market compared to international competitors [3] - The recruitment of patients for rare disease drugs can be challenging, but the hemophilia patient organization network is well-developed, facilitating information dissemination [3][4] Patient Demographics - Most recruited patients are under 40 years old, showing a higher willingness to engage with gene therapy and a strong desire for improved treatment options [4] - The company has reported no spontaneous bleeding issues in patients tracked for up to four years under effective dosing [4] Cost Advantages - The lower clinical development and production costs in China provide a competitive pricing advantage for domestic drugs, with GS1191 priced significantly lower than existing therapies [4] - The high cost of rare disease drugs is a common issue globally, creating opportunities for Chinese companies to expand into international markets [4] Regulatory Engagement - The company is in close communication with Saudi regulatory authorities to expedite the registration process for GS1191 and is encouraged to apply for local innovation funds [5] - In addition to rare diseases, the company is also developing products for common neurological diseases and is preparing for a new round of financing [5]

Core Viewpoint - The AAV (Adeno-Associated Virus) gene therapy sector is facing unprecedented challenges, including safety issues, high costs, and a significant withdrawal of major pharmaceutical companies from AAV projects [1][2][3][4]. Industry Challenges - AAV has transitioned from a highly sought-after delivery system to one facing skepticism and abandonment by major pharmaceutical companies [2][3]. - The industry is experiencing a crisis of confidence due to multiple safety incidents, including patient deaths linked to AAV therapies [13][14]. - High costs associated with AAV therapies, often exceeding $1 million, limit accessibility and create financial burdens for companies [10][12]. Technical Limitations - AAV's small capacity (approximately 4.7 kb) restricts its ability to deliver larger genes, necessitating complex strategies that may compromise efficacy [6]. - Immune responses triggered by AAV can lead to severe complications, including inflammation and organ damage, complicating treatment outcomes [7][8][9]. - The presence of neutralizing antibodies in the population poses significant barriers to the effectiveness of AAV therapies, limiting patient eligibility and treatment options [9]. Market Dynamics - Major companies like Pfizer, Roche, and Takeda have withdrawn from AAV research, reallocating resources to more promising areas [14][15]. - The capital market's enthusiasm for AAV has shifted to a more cautious approach, leading to financing difficulties for biotech firms focused on AAV therapies [15][16]. Future Directions - Despite the challenges, some companies are exploring new delivery systems, such as lipid nanoparticles (LNPs) and polymer nanoparticles, which may offer advantages over AAV [18]. - Companies like uniQure are focusing on optimizing AAV vectors and targeting specific diseases, indicating that AAV may still have a role in certain niches [19][21]. - The industry consensus suggests that while gene therapy remains promising, AAV is no longer the sole solution, and innovation in delivery methods is essential for future success [21].

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]