– Despite treatment with standard-of-care anti-seizure medicines, children with Dravet syndrome experienced high seizure burden and plateaued in neurodevelopment, resulting in a widening gap relative to children with typical development – – Findings underscore the urgent need for new medicines that target the underlying genetic cause of Dravet syndrome to improve long-term outcomes – CAMBRIDGE, Mass. and BEDFORD, Mass., Nov. 17, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Stoke Therapeutics, Inc ...

Core Viewpoint - Biogen Inc. announced a positive opinion from the CHMP of the EMA recommending the approval of a high dose regimen of nusinersen for treating 5q spinal muscular atrophy (SMA), with a final decision expected in January 2026 [1][7]. Company Developments - The high dose regimen of nusinersen includes a loading dose of two 50 mg doses 14 days apart, followed by a maintenance dose of 28 mg every four months for treatment-naïve patients [3][10]. - Nusinersen, marketed as SPINRAZA, is currently approved in over 71 countries at a lower dose of 12 mg [2][13]. - The high dose regimen has shown promising results in clinical studies, with significant improvements in motor function and a reduction in the risk of death or permanent ventilation [4][5]. Clinical Study Insights - The positive CHMP opinion is based on data from the Phase 2/3 DEVOTE study, which evaluated the efficacy and safety of the high dose regimen in both treatment-naïve and previously treated patients [3][11]. - In the pivotal Part B cohort of the DEVOTE study, treatment-naïve infants showed a statistically significant improvement in motor function compared to a matched sham group, with a mean difference of 26.19 points [4][11]. - The high dose regimen was generally well tolerated, with adverse events consistent with SMA and no new safety concerns identified [6][10]. Regulatory Landscape - The high dose regimen is under review by the U.S. FDA, with a decision expected by April 3, 2026 [9]. - Biogen is actively working with regulatory authorities worldwide to advance the high dose regimen as an additional option for SMA patients [9].

Core Viewpoint - Biogen Inc. has successfully acquired Alcyone Therapeutics, enhancing its capabilities in innovative CNS therapy delivery solutions, particularly through the ThecaFlex DRx™ drug delivery system [1][4]. Company Overview - Biogen is a leading biotechnology company founded in 1978, focused on pioneering innovative science to deliver new medicines and create value for shareholders and communities [10]. - The company emphasizes a deep understanding of human biology and leverages various modalities to advance first-in-class treatments [10]. Acquisition Details - The acquisition of Alcyone Therapeutics is aimed at strengthening Biogen's portfolio and expanding its expertise in drug delivery methods [4]. - The ThecaFlex DRx™ system is designed to provide an alternative to repeated lumbar punctures for chronic intrathecal administration of medicines, potentially improving patient experience and accessibility [2][5]. ThecaFlex DRx™ System - The ThecaFlex DRx™ is an investigational implantable device that allows access to cerebrospinal fluid for therapy infusion, representing a significant advancement over the current standard of care [5]. - The system has received a CE Mark in Europe and an Investigational Device Exemption from the FDA, although it is not yet approved for commercial use in the U.S. [5]. Clinical Development - ThecaFlex DRx™ has been in development since 2019, with ongoing clinical studies for nusinersen, marketed as SPINRAZA, which treats spinal muscular atrophy (SMA) [3]. - Biogen plans to introduce the new drug delivery system for SPINRAZA in early 2028, pending successful clinical trials and regulatory approval [3]. SPINRAZA Overview - SPINRAZA (nusinersen) is approved in over 71 countries for treating SMA, with more than 14,000 individuals treated globally [6]. - The drug has demonstrated efficacy across various ages and SMA types, supported by a well-established safety profile [7]. Regulatory Updates - A high dose regimen of SPINRAZA has recently been approved in Japan and is under review by the European Medicines Agency and the FDA, with a decision expected by April 3, 2026 [9].

Group 1 - Biogen has reinvigorated its Immunology and Inflammation (I&I) efforts, indicating a strategic shift beyond its historical focus on neuroscience [1] - The company's expertise in neuro-immunology, particularly in the central nervous system (CNS), underpins its strategy in I&I and kidney areas [1] - Biogen has been involved in lupus research for over a decade, highlighting its long-term commitment to immunological pathways [2] Group 2 - The development of drugs targeting immunological pathways is aimed at supporting a diverse product portfolio [2]

Core Insights - Eisai Co., Ltd. and Biogen Inc. announced the approval of LEQEMBI® (lecanemab) for once every four weeks intravenous maintenance dosing in the UK, following its earlier approval for treating mild cognitive impairment and mild dementia due to Alzheimer's disease [1][2]. Group 1: Product Approval and Usage - LEQEMBI was initially approved in August 2024 for treating mild cognitive impairment and mild dementia in adult patients who are either apolipoprotein E ε4 heterozygotes or non-carriers [2]. - The new maintenance dosing regimen allows patients to transition from an 18-month treatment of 10 mg/kg every two weeks to 10 mg/kg every four weeks, or to continue the bi-weekly regimen [2]. Group 2: Alzheimer's Disease Context - Alzheimer's disease (AD) is characterized by amyloid-beta plaques and tau protein tangles in the brain, leading to neurodegeneration [3]. - LEQEMBI targets both amyloid plaques and protofibrils, which are believed to contribute to cognitive decline, making ongoing maintenance treatment crucial for slowing AD progression [3][5]. Group 3: Market and Demographics - In the UK, approximately 982,000 individuals are living with dementia, with AD being the cause in 60-70% of these cases, and these numbers are expected to rise with an aging population [4]. Group 4: Development and Collaboration - Eisai leads the global development and regulatory submissions for lecanemab, with both Eisai and Biogen co-commercializing and promoting the product [4][10]. - Lecanemab has been approved in 51 countries and is under regulatory review in 9 additional countries, indicating a broad international interest and potential market [7].

Biogen Conference Call Summary Company Overview - **Company**: Biogen (NasdaqGS:BIIB) - **Date**: November 13, 2025 - **Event**: TD Cowen's 2025 I&I Summit Key Industry and Company Insights Immunology and Kidney Strategy - Biogen has reinvigorated its immunology and kidney efforts, leveraging its expertise in neuroimmunology from its historical focus on neuroscience [2][3] - The strategy aims to support a diverse product portfolio by targeting immunological pathways applicable to multiple disease indications [2][3] R&D Pipeline Structure - The ideal R&D pipeline in immunology is driven by scientific understanding, allowing for efficient early development trials and multi-indication strategies [4][5] - Biogen's approach includes starting with multiple indications simultaneously to build confidence for further expansion [5] Investor Perception - Investors currently undervalue Biogen's immunology and kidney pipeline, potentially missing the strategic rationale behind the development of drugs like felzartamab [6] - Felzartamab is positioned to address antibody-mediated rejection in kidney transplants, supported by robust clinical data [6][7] Lupus Pipeline Confidence - Biogen has two late-stage candidates for lupus: dapirolizumab pegol and litifilimab, with confidence stemming from data-driven insights and successful proof of concept studies [9][10] - The company emphasizes the importance of clinical trial execution and understanding disease management in lupus, which has historically been challenging [10][11] Ongoing Trials - The Topaz 1 and 2 trials for systemic lupus erythematosus (SLE) have completed enrollment, with data expected by the end of 2026 [14][15] - These trials are designed to test different dosing paradigms and measure various endpoints related to disease control [15][16] Key Risks and Considerations - Risks to trial success include the complexity of lupus management and the need for precise clinical trial execution [17][18] - Biogen aims to meet statistical significance and meaningful patient outcomes in its trials, focusing on reducing flares and steroid use [19][20] New Programs and Future Directions - Biogen recently introduced BIIB142, an IRAK4 degrader, which is an oral molecule with potential applications across various autoimmune diseases [32][33] - The company is exploring multi-indication strategies for this new asset and continues to build its early-stage pipeline through collaborations and acquisitions [36] Additional Insights - Biogen is committed to reducing steroid dependency in lupus patients, aligning with evolving treatment guidelines [20] - The company is optimistic about the potential of its lupus candidates to provide meaningful improvements in patient quality of life [10][11] This summary encapsulates the key points discussed during the conference call, highlighting Biogen's strategic focus on immunology and kidney disease, ongoing clinical trials, and future directions in research and development.

Core Insights - November is National Alzheimer's Disease Awareness Month in the U.S., highlighting the significance of the disease and the growing relevance of disease-modifying treatments [1] - The healthcare sector, particularly companies focused on Alzheimer's, presents a compelling investment opportunity through Healthcare exchange-traded funds (ETFs) [2] Group 1: Alzheimer's Drug Innovation - Eli Lilly's drug Kisunla (donanemab) received FDA approval in July 2024, showing a 22% slowing of clinical progression in treated patients, equating to a 1.4-month delay in disease progression [5] - Biogen, in collaboration with Eisai, launched Leqembi in 2023, which also gained full FDA approval [5] - Other significant players include Johnson & Johnson, developing two Alzheimer's medicines, and AbbVie, with its investigational drug ALIA-1758 [6] Group 2: Impact on Healthcare ETFs - The success of Alzheimer's therapies is crucial for the revenue and stock performance of companies like Eli Lilly and Biogen, making them key components in many healthcare ETFs [7] - The Dow Jones U.S. Health Care Index has increased by over 9% year to date, reflecting positive momentum from stocks like Eli Lilly and Biogen [8] Group 3: Government Support and Funding - The U.S. Senate Appropriations Committee proposed a $100 million increase for Alzheimer's and dementia research at the NIH for fiscal year 2026, indicating sustained government commitment to the biopharma sector [9] Group 4: Recommended Healthcare ETFs - Vanguard Health Care ETF (VHT) has net assets of $16.2 billion, with top holdings including Eli Lilly (10.33%) and AbbVie (5.76%), and has surged 13.4% year to date [10][11] - First Trust NASDAQ Pharmaceuticals ETF (FTXH) has net assets of $18.9 million, with top holdings including AbbVie (7.14%) and Johnson & Johnson (7.07%), rising 18.2% year to date [12] - Health Care Select Sector SPDR ETF (XLV) has assets worth $38.79 billion, with Eli Lilly (14.36%) and Johnson & Johnson (8.53%) as top holdings, increasing by 12.6% year to date [13] - iShares Neuroscience and Healthcare ETF (IBRN) has net assets of $4.38 million, with Biogen (4.04%) among its top holdings, and has surged 15.1% year to date [14]

Core Viewpoint - The appointment of Richard Pazdur as the new director of the FDA's Center for Drug Evaluation and Research has boosted investor confidence in the pharmaceutical sector, leading to record-high stock prices for major pharmaceutical companies [1][2]. Group 1: Appointment Details - Richard Pazdur, with 26 years of experience at the FDA, has been appointed as the new director, succeeding George Tidmarsh, who resigned following an investigation [1]. - Pazdur will continue to serve as the director of the FDA's Oncology Center of Excellence until a successor is determined [1]. Group 2: Market Reaction - The VanEck Vectors Pharmaceutical ETF, covering 25 global pharmaceutical companies, has seen a three-day rise, reaching an all-time high, with notable gains from companies like Novo Nordisk, Bristol-Myers Squibb, and Gilead [1]. - The SPDR S&P Biotech ETF also recorded a three-day increase, hitting its highest level since January 2022, with strong performances from Regeneron and BioNTech [1]. Group 3: Analyst Sentiment - Analysts generally view Pazdur's appointment positively, with Raymond James analyst Chris Mkins stating he could be the best choice for patients and the industry [2]. - The appointment may signal a significant shift in regulatory strategy from FDA leadership, according to industry experts [2]. - Medical media outlet Stat News welcomed the appointment, highlighting it as a positive development for the FDA during a turbulent period [2].

Core Insights - The ASN meeting highlighted a significant shift towards innovative therapies in nephrology, emphasizing the potential for cures in the field [3]. Group 1: ASN Meeting Highlights - The opening plenary by ASN President Prabir Roy-Chaudhury set a tone focused on ushering a new era in nephrology, with a clear emphasis on the possibility of cures [3]. - There was a notable increase in enthusiasm among attendees regarding new therapies and the direction of nephrology [3].

Summary of Biogen's Conference Call on Felzartamab and Nephrology Developments Company and Industry Overview - **Company**: Biogen - **Industry**: Biopharmaceuticals, specifically focusing on nephrology and autoimmune diseases Key Takeaways from ASN Meeting - The ASN meeting highlighted a significant shift towards innovative therapies in nephrology, emphasizing the potential for cures rather than just managing complications of kidney diseases [3][4] - There is a growing enthusiasm for new therapies that could transform treatment paradigms in nephrology [3] Felzartamab (Felza) Overview - Felzartamab is a targeted therapy developed for severe immune diseases, particularly focusing on conditions like IgA nephropathy and antibody-mediated rejection (AMR) [5][6] - The therapy targets CD38, which is expressed on antibody-producing cells, offering a novel approach in treating autoimmune diseases [9][10] Indications and Clinical Studies - Initial indications for Felza include: - **IgA Nephropathy**: Demonstrated durable treatment effects extending beyond dosing intervals, with significant efficacy observed in early studies [12][14] - **AMR**: A small phase two study showed an 80% resolution rate in microvascular inflammation, indicating transformative efficacy [12][24] - **Primary Membranous Nephropathy (PMN)**: Targeting high-risk patients who are refractory to existing therapies [33] Competitive Landscape and Market Positioning - The treatment landscape for IgA nephropathy is evolving, with multiple therapies expected to enter the market soon, including foundational therapies and targeted therapies [15][16] - Felza is positioned as a disease-modifying therapy that may provide a durable response without the need for continuous dosing, unlike other therapies that require ongoing administration [17][18] Regulatory and Development Strategy - The primary endpoint for the phase three AMR study is the resolution of AMR by histology, with a focus on durability and stabilization of kidney function [29][30] - The anticipated timeline for AMR data is 2027, with a filing expected in 2028, followed by studies for isolated microvascular inflammation and PMN [36][37] Safety and Efficacy Considerations - Long-term safety data is still needed, as current studies have been relatively small and of short duration [18] - The potential for combination therapies and patient preferences will play a crucial role in treatment decisions [18][22] Future Directions and Insights - Ongoing research aims to deepen the understanding of disease mechanisms, particularly the role of NK cells in AMR and the complexities of antibody-mediated diseases [40][41] - Biogen is exploring subcutaneous dosing options for Felza to improve patient access and tolerability [38] Conclusion - Biogen is at the forefront of advancing Felzartamab in nephrology, with promising data and a strategic approach to addressing significant unmet needs in autoimmune kidney diseases [42][43]