8 January 2026 | 5:01AM EST Equity Research Americas Healthcare: Biotechnology: Constructive backdrop for 2026 Following strong performance for the sector exiting 2025 (XBI +35% vs. S&P 500 +16%), driven by the convergence of a number of key factors, notably, broader market dynamics (e.g., macro/AI-trade rotations and rates), easing policy risks (e.g., Most-Favored-Nations (MFN) drug pricing deals), and improving fundamentals (e.g., stable/upward large-cap estimate revisions and SMID catalyst execution), we ...

NEW YORK, Dec. 29, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it has acquired the final portion of PTC Therapeutics’ remaining royalty on Roche’s Evrysdi for $240 million upfront and up to $60 million in sales-based milestones. Evrysdi, marketed by Roche, is an orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy. Evrysdi was approved by the FDA in 2020 and has treated over 21,000 patients worldwide. In 2024, E ...

Hardman Johnston Global Advisors, an investment management company, released its “Hardman Johnston Global Equity Strategy” investor letter for the third quarter of 2025. A copy of the letter can be downloaded here. The portfolio underperformed in the quarter due to stock selection. The composite returned 5.02%, net of fees, compared to 7.62% for the MSCI AC World Net Index. In addition, please check the fund’s top five holdings to know its best picks in 2025. In its third-quarter 2025 investor letter, Ha ...

医药生物 2025 年 06月 09 日 从供需看,中国创新药能从海外分成多少钱? 投资要点: 行情回顾:本周(2025年6月3日-2025年6月6日)中信医药指数 A 上涨 1.2%,跑赢沪深 300指数 0.3 pct,在中信一级行业分类中排名第 16 位;2025年初至今中信医药生物板块指数上涨 8.3%,跑赢沪深 300 指数 9.9pct,在中信行业分类中排名第5位。本周涨幅前五的个股为:易明医药 (+33.09%)、万邦德(+32.59%)、昂利康(+30.28%)、新诺威(+21.36%)、 海辰药业(+20.93%)。 周专题:跨国药企专利悬崖释放超 2400 亿美元市场空间,中国凭借技 A 术平台与研发效率优势成为全球创新药供给的核心力量。截至 2037年,全 球市场 27款 2024年销售额超 40亿美元的重磅药物面临专利失效,MNC 均有迫切寻找重磅创新药的需求。我们认为目前已经进入下一个创新药周 期的投入阶段,中国创新药在细胞疗法、ADC、双抗等技术领域在研管线 数量全球第一,在 716个赛道研发进度第一,有望依托 License-out 模式实 现技术价值全球化兑现,加速填补 MNC ...

Core Viewpoint - Moderna has transitioned from a clinical-stage biotech to a profitable commercial-stage company due to its coronavirus vaccine, but recent declines in vaccine sales and underperformance of its RSV vaccine raise concerns about its growth potential [1][2] Group 1: Moderna's Current Situation - Moderna's stock has seen a decline in vaccine sales, and its RSV vaccine has not met growth expectations [2] - Despite challenges, Moderna has made progress in cost-cutting and is focusing on promising programs, leading to a stock increase of over 30% in the past month [2] - The company aims for cash breakeven by 2028 and the launch of new products, which may not appeal to cautious investors [5] Group 2: Vertex Pharmaceuticals Overview - Vertex Pharmaceuticals is highlighted as a better investment option for both cautious and aggressive investors due to its strong earnings growth in cystic fibrosis treatment [6] - Vertex's CFTR modulators, Trikafta and Alyftrek, treat nearly 95% of cystic fibrosis patients, with ongoing development for remaining patients [7] - Vertex's revenue increased by 11% to over $3 billion in the latest quarter, driven by its CF products [8] Group 3: New Product Approvals and Pipeline - Vertex has expanded its product line with approvals for Casgevy and Journavx, which could significantly boost revenue in the coming years [9] - Journavx addresses a critical need for effective non-opioid pain management, with over 300,000 prescriptions filled since its launch [9] Group 4: Financial Performance and Valuation - Vertex's stock has increased nearly 100% over the past five years and about 15% this year, indicating strong long-term and short-term performance [11] - The current valuation of Vertex is 25 times forward earnings estimates, a reasonable price given its market leadership and expanding product portfolio [11] - Vertex's financial strength and diverse product offerings position it well for future growth, making it a more attractive investment compared to Moderna [12]

Financial Data and Key Metrics Changes - The company raised $219 million in its IPO in February, providing financial flexibility to execute its strategy through 2028 [7] - The company entered Q3 with $325 million in cash, ensuring a cash runway into 2028 [47] Business Line Data and Key Metrics Changes - The company is focused on cystic fibrosis (CF) and is developing two NBD1 stabilizers: SION-719 for an add-on approach and SION-451 for a dual combination strategy [5][6] - The goal is to improve CFTR function in patients, with a target of achieving at least a 10 millimole per liter change in sweat chloride as a primary endpoint in clinical studies [30][69] Market Data and Key Metrics Changes - The CF market is currently valued at $12 billion and is expected to grow to $15 billion [12] - The company aims to create more treatment options for the approximately 85%-90% of CF patients with the F508del mutation, as two-thirds of patients on standard care do not achieve normal CFTR function [16][19] Company Strategy and Development Direction - The company is pursuing a differentiated approach to CF treatment by targeting the NBD1 protein, which has been considered undruggable [4][10] - The strategy includes a dual combination approach to potentially provide meaningful clinical benefits above the current standard of care, which is a triple combination therapy [6][12] - The company is open to exploring new therapeutic areas in the future but is currently focused solely on CF [50][51] Management's Comments on Operating Environment and Future Outlook - Management believes that the current modulators have not fully addressed the unmet needs in CF, as many patients still do not achieve normal CFTR function [16] - The company is confident in its ability to leverage its historical research and partnerships, particularly with the Cystic Fibrosis Foundation, to advance its clinical studies [65][66] - Management emphasized the importance of patient access in pricing strategies, aiming to ensure that new therapies are accessible to patients [60] Other Important Information - The company has licensed three compounds from AbbVie, enhancing its portfolio with clinical data and complementary mechanisms [42][44] - The company is conducting a healthy volunteer phase 1 study for its dual combination strategy, which is expected to inform future development [38][40] Q&A Session Summary Question: What is the company's approach to addressing unmet needs in CF? - The company aims to improve CFTR function and believes that NBD1 stabilization is key to moving more patients to normal CFTR function [16][19] Question: How does the company plan to differentiate its products in the market? - The company is focused on improving efficacy through its dual combination strategy and leveraging historical data to predict clinical outcomes [67][70] Question: What is the expected timeline for clinical data? - The company anticipates data from its phase 2a proof of concept study in mid-2026, which will be crucial for future development decisions [5][30]

Financial Data and Key Metrics Changes - The company raised $219 million in its IPO in February 2025, providing financial flexibility to execute its strategy through 2028 [7] - As of Q3, the company reported having $325 million in cash, ensuring a cash runway into 2028 [46] Business Line Data and Key Metrics Changes - The company is focused on developing two NBD1 stabilizers: SION-719 for an add-on approach to the standard of care and SION-451 for a dual combination strategy [5][6] - The company aims to demonstrate a clinically meaningful improvement of at least 10 millimoles per liter in sweat chloride, which is expected to correlate with a 3-point improvement in FEV1 [34][35] Market Data and Key Metrics Changes - The cystic fibrosis market is currently valued at $12 billion and is projected to grow to $15 billion [12] - The company targets the same patient population as the standard of care, focusing on the F508del mutation, which affects 85%-90% of cystic fibrosis patients [19] Company Strategy and Development Direction - The company aims to transform the standard of care in cystic fibrosis by focusing on the NBD1 target, which has been historically considered undruggable [4][10] - The dual combination strategy is prioritized, with the goal of raising the efficacy bar through innovative combinations of therapies [6][12] Management's Comments on Operating Environment and Future Outlook - Management believes that there is a significant unmet need in the cystic fibrosis community, as two-thirds of patients on standard care do not achieve normal CFTR function [16] - The company is optimistic about its clinical development strategy and the potential to improve patient outcomes significantly [70] Other Important Information - The company has licensed three compounds from AbbVie, enhancing its portfolio with clinical data and complementary mechanisms [41][42] - The Therapeutics Development Network (TDN) of the Cystic Fibrosis Foundation is a key partner, providing access to a well-organized research network [65][66] Q&A Session Summary Question: What is the company's strategy for addressing the unmet needs in cystic fibrosis? - The company aims to improve CFTR function in patients who are currently on standard care but do not achieve normal function, focusing on the NBD1 stabilization approach [16][19] Question: How does the company plan to differentiate its products in the market? - The company plans to leverage its unique dual combination strategy and the historical data from its assays to demonstrate improved efficacy compared to existing therapies [6][70] Question: What are the expected timelines for clinical data? - The company anticipates data from its phase 2a proof of concept study in mid-2026, which will be crucial for informing future development and capital raising decisions [5][29]

Summary of Sionna Therapeutics FY Conference Call Company Overview - **Company**: Sionna Therapeutics (NasdaqGM:SION) - **Date of Conference**: December 03, 2025 - **Key Speakers**: Mike, Elena, Charlotte, and Corey Casimov (Senior Biotech Analyst at Evercore) Key Accomplishments in 2025 - Successful completion of Phase 1 programs for two NBD1 stabilizers: SION-719 and SION-451 with positive data outcomes [2][4] - Initiation of Phase 2 proof of concept study for SION-719 in combination with Trikafta, aimed at demonstrating improved CFTR function [2][3] - Progression of SION-451 into a Phase 1 study with healthy volunteers, testing its combination with two other modulators [3][4] - Completion of IPO in February 2025, raising over $219 million, providing cash runway into 2028 [4][5] Unmet Needs in Cystic Fibrosis (CF) - Acknowledgment of Vertex's contributions to CF treatment but recognition of ongoing unmet needs, as only about one-third of patients achieve normal CFTR function with current therapies [6][7] - Focus on improving patient outcomes by lowering sweat chloride levels and enhancing overall CFTR function [6][8] Mechanism of Action and Clinical Strategy - Emphasis on the unique mechanism of NBD1 stabilizers, which may provide additional efficacy compared to existing treatments [12][15] - Data from preclinical studies indicating that NBD1 stabilizers can improve CFTR protein half-life to wild-type levels, supporting the rationale for the NBD1 anchored pipeline [13][15] Clinical Trials and Future Directions - Phase 2a precision proof of concept trial for SION-719 aims to show significant improvement in sweat chloride levels compared to Trikafta [22][24] - The dual combination approach is preferred, with expectations of better efficacy than current standard care [25][26] - Plans to evaluate safety and pharmacokinetics in the combination studies of SION-451 with other modulators [30] Assay Confidence and Predictive Power - High confidence in the CFHBE assay used to predict clinical outcomes, validated through comparisons with Vertex's compounds [31][32] - The assay is seen as a tool to support the development of new treatment options for CF patients [34] Strategic Options and Future Pathways - Potential to pursue multiple combinations if data supports it, with a focus on capital availability to fund both add-on and dual combination approaches [35][36] - Anticipation of significant developments in 2026 as data from ongoing studies becomes available [37] This summary encapsulates the key points discussed during the conference call, highlighting Sionna Therapeutics' progress, strategic focus, and future plans in the cystic fibrosis treatment landscape.

Financial Data and Key Metrics Changes - Vertex Pharmaceuticals reported nearly $500 million in revenue from the launch of vanzacaftor/tezacaftor/deutivacaftor in the first nine months, indicating a strong start for the product [20][21] - The company expects revenue for Casgevy to exceed $100 million in 2025, showing a steady ramp in patient initiations and infusions [78] Business Line Data and Key Metrics Changes - The launch of the acute pain therapy has resulted in over 300,000 prescriptions written, with a significant increase from 10,000 in Q1 to 190,000 in Q3 [7][6] - The company has achieved formulary placement in several large hospitals, exceeding initial expectations [6] Market Data and Key Metrics Changes - Vertex has over 170 million covered lives in the US for its new pain medication, with a goal of reaching approximately 300 million [5] - The company is making progress in discussions with Medicare and Medicaid, although there is still work to be done [9] Company Strategy and Development Direction - Vertex is focusing on expanding its portfolio in renal medicine, with multiple potential medicines in development for conditions like IgA nephropathy and APOL1-mediated kidney disease [70][69] - The company aims to leverage synergies between its renal products to enhance market presence and access [75] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the momentum in prescription numbers and the overall patient experience with the new pain medication [7] - The company is excited about the potential of POVI and its differentiated profile in treating B-cell mediated diseases, indicating a strong pipeline for future growth [53][52] Other Important Information - Vertex is actively engaged in share buyback programs, having repurchased approximately $2 billion worth of shares this year [87] - The company is open to various types of transactions to enhance its portfolio, maintaining high standards for scientific and medical quality [87] Q&A Session Summary Question: What is the outlook for the new pain medication? - Management highlighted the significant ramp in prescriptions and positive feedback from physicians and payers, indicating strong market acceptance [6][7] Question: How is the company addressing access and reimbursement for Casgevy? - Management noted that access and reimbursement are strong in key markets, including the US and Europe, and are not limiting factors for Casgevy [84] Question: What are the company's plans for future acquisitions or investments? - Management stated that they are open to adding new verticals or enhancing existing ones, with a focus on maintaining flexibility in capital allocation [87]

Financial Data and Key Metrics Changes - Vertex Pharmaceuticals reported nearly $500 million in revenue for the first nine months of the launch of vanzacaftor/tezacaftor/deutivacaftor, indicating a strong start for the product [20][21][22] - The company expects revenue for Casgevy to exceed $100 million in 2025, showing a steady ramp in patient infusions [79] Business Line Data and Key Metrics Changes - The launch of the acute pain product has seen over 300,000 prescriptions written, with a significant increase from 10,000 in Q1 to 190,000 in Q3 [7][21] - The company has achieved formulary placement in several large hospitals and healthcare systems, exceeding initial expectations [6] Market Data and Key Metrics Changes - Vertex has over 170 million covered lives in the US for its new pain product, with a goal of reaching approximately 300 million [5][6] - The company is making progress in discussions with payers, particularly on the Medicaid side, while still working on Medicare access [8][9] Company Strategy and Development Direction - Vertex is focusing on diabetic peripheral neuropathy (DPN) as a key area for development, with ongoing Phase 3 studies expected to complete enrollment in 2026 [12][19] - The company is exploring opportunities in B-cell mediated diseases, particularly through the POVI program, which targets IgA nephropathy and membranous nephropathy [52][54] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the momentum of the acute pain product launch and the positive feedback from physicians and payers [6][7] - The company is optimistic about the potential of POVI and its differentiated profile in treating renal diseases, anticipating significant market opportunities [54][71] Other Important Information - Vertex is actively engaged in share buyback programs, having repurchased approximately $2 billion worth of shares this year [88] - The company is in late preclinical development for novel conditioning regimens to improve patient outcomes in gene therapies [82] Q&A Session Summary Question: What is the outlook for the new pain product? - Management highlighted the significant progress in prescriptions and payer access, indicating a strong trajectory for the product [5][6] Question: How is the company addressing the challenges in chronic pain? - The focus is on DPN due to its well-defined clinical path and the lack of high-quality treatments, with ongoing studies expected to yield positive results [12][19] Question: What are the expectations for Casgevy's market access? - The company reported strong access and reimbursement across major markets, with expectations for significant revenue growth in 2025 [85] Question: What is the strategy for future acquisitions or investments? - Vertex aims to maintain flexibility for internal and external investments, with a focus on high-quality science and potential new therapeutic areas [88]