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Biogen (BIIB) Expands Neurology Pipeline With Alcyone Buyout, Stock Seen Undervalued
Yahoo Finance· 2025-09-27 04:59
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]
Biogen Provides Regulatory Update on High Dose Regimen of Nusinersen
Globenewswire· 2025-09-23 20:30
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]
New Data for Nusinersen Underscore Biogen's Commitment to Advancing Clinical Research to Improve Outcomes in SMA
Globenewswire· 2025-06-27 14:00
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]
Biogen to Advance Investigational Spinal Muscular Atrophy Asset to Registrational Studies Based on Positive Interim Phase 1 Results
Globenewswire· 2025-06-25 11:30
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]