– 3-year data from open-label extension (OLE) studies demonstrate the potential for disease modification with durable seizure reductions and improvements in cognition and behavior on top of standard anti-seizure medicines – – Substantial improvement in overall seizure burden and seizure free days observed with ongoing treatment – CAMBRIDGE, Mass. and BEDFORD, Mass., Aug. 25, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to ...

Zorevunersen for Dravet Syndrome - Stoke Therapeutics is developing zorevunersen as a potential disease-modifying medicine for Dravet syndrome [16] - Initial 70mg doses of zorevunersen demonstrated substantial and sustained reductions in convulsive seizure frequency [18] - Ongoing zorevunersen treatment showed substantial and durable reductions in major motor seizure frequency in OLE studies [21] - 36-month OLE data shows continuing improvements in cognition and behavior with zorevunersen treatment [56] - The EMPEROR Phase 3 study is designed to assess disease modification in Dravet syndrome with a dosing regimen of 2x70mg followed by 2x45mg over a 52-week treatment period [31, 32] - The EMPEROR Phase 3 study has dosed its first patient in August 2025 and anticipates data in 2H 2027, randomizing approximately 170 patients across ~70 global sites [35] STK-002 for Autosomal Dominant Optic Atrophy (ADOA) - Approximately 65-90% of ADOA cases are caused by mutations in one allele of the OPA1 gene, leading to haploinsufficiency [64] - Up to 46% of ADOA patients are registered as legally blind [65] - Preclinical findings support further development of STK-002, showing increased OPA1 protein and ATP-linked respiration [70] - A Phase 1 study of STK-002 in ADOA patients is underway in the UK, involving 21 patients aged ≥18 to <55 years receiving single ascending doses (0.1, 0.3, 0.5, & 0.7 mg/eye) [76]

– Global, pivotal Phase 3 study will evaluate efficacy and safety of zorevunersen compared to sham over a 52- week treatment period – – Dravet syndrome is a rare genetic disease characterized by refractory seizures and neurodevelopmental impairments, with no currently approved medicines that address the underlying cause of the disease – CAMBRIDGE, Mass. and BEDFORD, Mass., Aug. 11, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedica ...

Core Insights - The analysis of the Phase 3 zorevunersen dosing regimen indicates improvements in cognition and behavior at Week 68, supporting its potential as a disease-modifying treatment for Dravet syndrome [1][2][4] - Zorevunersen has shown substantial and durable reductions in major motor seizure frequency and improvements in cognitive and behavioral measures over two years of treatment [2][4] - The Phase 3 EMPEROR study will assess key secondary endpoints related to cognition and behavior, which are critical for evaluating the treatment's effectiveness [3][5] Company Overview - Biogen Inc. and Stoke Therapeutics are collaborating on the development of zorevunersen, an investigational therapy aimed at addressing the underlying causes of Dravet syndrome [1][8] - Stoke Therapeutics focuses on restoring protein expression through RNA medicine, with zorevunersen being their first medicine in development [9] - Biogen is a leading biotechnology company that aims to deliver innovative treatments and create value for shareholders [10] Disease Context - Dravet syndrome is a severe neurodevelopmental disorder characterized by recurrent seizures and significant cognitive and behavioral impairments, affecting an estimated 38,000 individuals in the U.S., UK, EU-4, and Japan [3][7] - The condition is primarily caused by mutations in the SCN1A gene, leading to insufficient NaV1.1 protein levels in neuronal cells [7][8] - Current treatments primarily focus on seizure control, with no approved therapies addressing the cognitive and behavioral aspects of the disease [3][7] Clinical Study Insights - The Phase 3 EMPEROR study is designed to evaluate the effects of zorevunersen on cognition and behavior, with a focus on five sub-domains of the Vineland-3 Adaptive Behavior Scales [5][6] - The dosing regimen includes two loading doses of 70mg followed by maintenance doses of 45mg, which has shown promising results in previous studies [4][5] - The analysis utilized a mixed-effects model for repeated measures to assess the potential effects of the dosing regimen on patient outcomes [4]

Core Viewpoint - Biogen reported first-quarter 2025 adjusted earnings per share (EPS) of $3.02, missing the Zacks Consensus Estimate of $3.32, with an 18% year-over-year decline in earnings due to a $165 million upfront payment to Stoke Therapeutics for a collaboration agreement [1][2][19] Financial Performance - Total revenues for Q1 2025 were $2.43 billion, reflecting a 6% year-over-year increase on a reported basis and an 8% increase on a constant-currency basis, surpassing the Zacks Consensus Estimate of $2.23 billion [2] - Total product sales reached $1.73 billion, up 1% year over year on a reported basis and 3% on a constant-currency basis [4] - Contract manufacturing and royalty revenues surged 61% year over year to $293 million, while Alzheimer's collaboration revenues increased to $33 million from $3 million in the prior year [5][6] Product Sales Breakdown - Multiple sclerosis (MS) revenues totaled $953 million, down 11% on a reported basis due to generic competition for Tecfidera [9] - Sales of Spinraza rose 24.2% to $423.9 million, exceeding the Zacks Consensus Estimate of $365 million [12] - New drug Qalsody for ALS recorded sales of $15.5 million, while Zurzuvae for postpartum depression generated $28 million in sales, reflecting a 21.7% sequential increase [13][14] Cost and Guidance - Adjusted R&D expenses decreased 3% year over year to $427 million, while adjusted SG&A expenses rose 1% to $572 million [16] - The company reaffirmed its total revenue guidance for 2025, expecting a mid-single-digit percentage decline in constant currency terms compared to 2024, and lowered its adjusted EPS guidance to a range of $14.50 to $15.50 [18][19] Market Performance - Year to date, Biogen's shares have declined 20.9%, compared to a 3.3% decrease in the industry [3]

Core Viewpoint - Biogen is expected to report first-quarter 2025 results on May 1, with sales and earnings estimates at $2.24 billion and $3.34 per share, respectively. The company faces challenges from declining sales of multiple sclerosis (MS) drugs, which may be offset by revenues from new drugs [1][2][5]. Sales Performance of MS Drugs - Sales of Biogen's MS drugs, including Tecfidera and Tysabri, are anticipated to decline due to increased competition and the introduction of generic versions in various markets [2][5]. - The Zacks Consensus Estimate for Tecfidera sales is $199.0 million, while the internal estimate is $185.7 million. For Tysabri, the estimates are $363.0 million and $348.6 million, respectively [3][4]. - Vumerity's sales rose in the fourth quarter due to higher demand, with estimates for the first quarter at $141.0 million (Zacks) and $139.8 million (internal) [4]. Impact of Biosimilars and Generics - The decline in MS revenues is expected to be more pronounced in 2025 due to the anticipated entry of biosimilars for Tysabri and generics for Tecfidera in certain European markets [5]. Other Drug Sales - Spinraza's sales improved in the fourth quarter, but demand may have decreased in the first quarter, with estimates at $364.0 million (Zacks) and $338.1 million (internal) [6]. - Sales of Skyclarys for Friedreich's ataxia are expected to rise year-over-year, with estimates at $110.0 million (Zacks) and $111.4 million (internal) [7]. - Zurzuvae's launch has exceeded expectations, with strong patient demand likely contributing to improved sales in the first quarter [8][9]. Alzheimer's Collaboration and Revenues - Revenues from contract manufacturing, royalties, and Alzheimer's collaboration are expected to rise, particularly from the drug Leqembi, which has shown strong sales growth in recent quarters [10][11]. - Leqembi's sales have improved sequentially, with strong growth noted in China and Japan, and it was recently approved in the European Union [11][12]. Overall Revenue Expectations - Overall revenues for the first quarter of 2025 are expected to be negatively impacted by seasonality, higher discounts, and channel dynamics in the U.S., along with foreign exchange headwinds [13]. Key Events - Biogen announced a collaboration with Stoke Therapeutics to develop zorevunersen for Dravet syndrome, with a pivotal study expected to begin in the first quarter of 2025 [14]. Earnings Surprise History - Biogen has consistently beaten earnings estimates in the past four quarters, with an average earnings surprise of 11.8%. However, the current model does not predict a beat for the upcoming quarter, with an Earnings ESP of -15.8% [15][16][17].