Core Insights - Biogen announced the publication of results from the Phase 2/3 DEVOTE study in Nature Medicine, evaluating a high-dose regimen of nusinersen for spinal muscular atrophy (SMA) [1][2] - The high-dose regimen includes a loading dose of 50 mg/5 mL and a maintenance dose of 28 mg/5 mL, which is more rapid and higher than the previously approved 12 mg regimen [1][6] - The study demonstrated safety and effectiveness across a broad range of SMA patients, showing significant improvements in motor function and other health domains [2][3] Study Details - DEVOTE enrolled 139 participants of various ages and SMA types, with treatment-naïve infants showing statistically significant improvements in motor function compared to a matched sham group [2][3] - The pivotal cohort (Part B) reported a mean difference of 26.19 points in motor function (CHOP-INTEND) between the high-dose regimen and sham group (p<0.0001) [2] - In the open-label Part C, participants transitioning from the 12 mg regimen to the high-dose regimen experienced mean increases of 1.8 points on the Hammersmith Functional Motor Scale – Expanded (HFMSE) [3] Safety Profile - The safety profile of the high-dose regimen was consistent with the known safety profile of the 12 mg regimen, with common adverse events including pneumonia and respiratory failure [4] - In the Part B infantile-onset cohort, the most common adverse events (≥15% of participants) were pneumonia, respiratory failure, pyrexia, COVID-19, and upper respiratory tract infection [4] Regulatory Status - The high-dose regimen of nusinersen is approved in the European Union and Japan, and is currently under review by the U.S. FDA with a PDUFA action date of April 3, 2026 [5][7]

Core Viewpoint - Biogen Inc. has received marketing authorization from the European Commission for a high dose regimen of SPINRAZA (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), which is the most common form of the disease, accounting for approximately 95% of all SMA cases [1][2] Product Authorization and Dosing Regimen - The updated SPINRAZA regimen includes 50 mg/5 mL and 28 mg/5 mL doses, featuring a more rapid loading phase with two 50 mg doses administered 14 days apart, followed by 28 mg maintenance doses every four months [1][11] - Individuals transitioning from the previous 12 mg dose will receive one 50 mg dose instead of their next 12 mg dose, followed by the 28 mg maintenance doses [1] Clinical Study and Efficacy - The European Commission's approval is based on data from the Phase 2/3 DEVOTE study, which demonstrated statistically significant improvements in motor function for treatment-naïve, symptomatic infants using the high dose regimen compared to a matched sham group [2][6] - The pivotal cohort showed a mean difference of 26.19 points on the CHOP-INTEND scale, with a significant p-value of <0.0001 [2][9] - Participants transitioning from the low dose regimen also experienced a mean improvement of 1.8 points on the Hammersmith Functional Motor Scale – Expanded from baseline to Day 302 [2] Safety Profile - The high dose regimen was generally well tolerated, with adverse events consistent with SMA and the known safety profile of nusinersen, with no new safety concerns observed [3][6] - Common adverse events reported in at least 10% of participants included pneumonia, COVID-19, pneumonia aspiration, and malnutrition [3] Community and Regulatory Engagement - Biogen is committed to advancing care for individuals living with SMA and is working with regulatory authorities globally to progress the high dose regimen, which is also approved in Japan and under review by the U.S. FDA [7][11] - The approval highlights the importance of sustained research and investment in SMA care, as noted by community leaders [5]

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 Inc. is considered one of the most undervalued stocks on NASDAQ, with a recent acquisition of Alcyone Therapeutics for an initial cash payment of $85 million and additional milestone payments [1] - The acquisition centers on ThecaFlex DRx, an implantable device aimed at providing chronic medication delivery for patients with neurological illnesses, eliminating the need for repeated lumbar punctures [2] - Following the acquisition, Biogen will oversee the development, production, and marketing of ThecaFlex DRx, with ongoing clinical studies assessing its effectiveness in patients with spinal muscular atrophy using SPINRAZA (nusinersen) [3]

Core Viewpoint - Biogen Inc. received a Complete Response Letter (CRL) from the FDA regarding its supplemental New Drug Application (sNDA) for a high dose regimen of nusinersen for spinal muscular atrophy (SMA), requesting updates to the technical information in the Chemistry Manufacturing and Controls (CMC) module [1][2] Group 1: FDA Response and Company Actions - The FDA's letter did not indicate any deficiencies in the clinical data for the high dose regimen [2] - Biogen plans to promptly resubmit the application based on readily available information [2] - The company is collaborating with global regulatory authorities to advance the high dose regimen as an additional option for SMA patients [2] Group 2: Product Information - SPINRAZA (nusinersen) is approved in over 71 countries for treating SMA in infants, children, and adults, with more than 14,000 individuals treated worldwide [3] - The drug has demonstrated efficacy across various ages and SMA types, supported by a well-established safety profile from data in patients treated for up to 10 years [4] Group 3: Company Background - Biogen, founded in 1978, is a leading biotechnology company focused on innovative science to develop new medicines and create value for shareholders and communities [6]

Core Insights - Biogen announced new data reinforcing the clinical impact of nusinersen for spinal muscular atrophy (SMA), highlighting findings from the DEVOTE trial and NURTURE trial presented at a recent conference [1][6][8] - The higher dose regimen of nusinersen is under review in multiple global markets, featuring a rapid loading and higher maintenance dosing schedule [1][5] DEVOTE Trial Findings - Part C of the DEVOTE trial demonstrated potential clinical benefits of a higher dose of nusinersen in previously treated patients (n=38) aged 4 to 65, with a median treatment duration of approximately 3.9 years [3][4] - Improvements were observed in motor function across various assessments, with non-ambulatory participants showing an average improvement of +2.5 on the Hammersmith Functional Motor Scale – Expanded (HFMSE) [4][5] NURTURE Study Outcomes - Final data from the NURTURE study indicated that all 25 participants remained alive, with no need for permanent ventilation, and 92% achieved independent walking ability [6][7][8] - Participants with elevated neurofilament light chain (NfL) levels at baseline showed significant reductions in NfL after starting nusinersen, suggesting its potential as a biomarker for treatment response [9] Safety Profile - The safety profile of the higher dose regimen aligns with the known safety profile of the 12 mg SPINRAZA, with most adverse events reported as mild to moderate [5][9] - In the DEVOTE study, 37 out of 40 participants reported adverse events, with serious adverse events occurring in 15% of participants, none related to the treatment [5]

Core Insights - Biogen Inc. announced topline results from the Phase 1 study of salanersen, an antisense oligonucleotide for spinal muscular atrophy (SMA), showing potential for high efficacy and once yearly dosing [1][5] - The Phase 1 study demonstrated substantial slowing of neurodegeneration and clinically meaningful improvements in motor function in children previously treated with gene therapy [2][5] - Biogen is engaging with global health authorities to advance salanersen into registrational studies based on encouraging Phase 1 data [4][5] Study Details - The Phase 1 study included two parts: a randomized placebo-controlled segment in healthy adults and an open-label segment in pediatric SMA participants who had previously received ZOLGENSMA [2][4] - Interim results from the open-label segment (n=24) indicated that both 40 mg and 80 mg doses of salanersen were well-tolerated, with a mean reduction in neurofilament light chain (NfL) of 70% at 6 months [2][4] - Exploratory data showed that half of the participants (4 out of 8) achieved new WHO motor milestones after receiving salanersen [3][4] Safety Profile - The safety profile of salanersen was generally well-tolerated, with most adverse events being mild to moderate, including pyrexia and upper respiratory tract infections [4][5] - The study's findings suggest that salanersen could address critical unmet needs in SMA treatment, building on Biogen's extensive experience in the field [3][5] Industry Context - SMA is a rare genetic neuromuscular disease affecting approximately 1 in 10,000 live births, characterized by progressive muscle atrophy and weakness [7][8] - SPINRAZA, another treatment for SMA, has been approved in over 71 countries and has treated more than 14,000 individuals worldwide, establishing a foundation of care in SMA [9][10]