Latest news with #DravetSyndrome
Yahoo
10-07-2025
- Business
- Yahoo
Stoke Therapeutics and Biogen Announce Presentation of Data from Studies of Zorevunersen, an Investigational Medicine for Dravet syndrome, at the 16th European Paediatric Neurology Society (EPNS) Congress
– Data from an analysis designed to evaluate the potential effects of the Phase 3 zorevunersen dosing regimen showed improvements in cognition and behavior at Week 68 – – These findings support the inclusion of key secondary endpoints assessing cognition and behavior in the Phase 3 EMPEROR study and contrast with outcomes observed in natural history data – BEDFORD, Mass., & CAMBRIDGE, Mass., July 10, 2025--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, and Biogen Inc. (Nasdaq: BIIB) today announced the presentation of data from an analysis that informed the design of the Phase 3 EMPEROR study and evaluated the potential effects of the Phase 3 zorevunersen dosing regimen. The data are complementary to previously reported data from a broader cohort of patients treated with zorevunersen in the Phase 1/2a and open label extension (OLE) studies that showed improvements within the first 9 months and continuing improvements through an additional two years. The new analysis is best aligned with the timing and dosing regimen that will be evaluated in the pivotal Phase 3 EMPEROR study and showed improvements in multiple measures of cognition and behavior at Week 68. The results contrasted with findings from a natural history study in which patients with Dravet syndrome were treated with standard of care medicines. Zorevunersen is in development as a first-in-class potential disease modifying treatment for Dravet syndrome. Results were presented as part of the Epilepsy II session at the 16th European Paediatric Neurology Society (EPNS) Congress. "Dravet syndrome is a complex neurodevelopmental disorder that has significant impacts on patients and their families," said Dr. Andreas Brunklaus, Consultant Paediatric Neurologist at the Royal Hospital for Children in Glasgow, Honorary Professor at the University of Glasgow, and a zorevunersen study investigator. "Natural history data shows the limitations of treating this disease with anti-seizure medicines. The zorevunersen data give us early evidence that this new genetically-targeted approach could address the underlying cause of Dravet syndrome, resulting in additional seizure control and offer patients the opportunity to experience improvements in cognition and behavior." Previously presented data from the two Phase 1/2a and OLE studies showed substantial and durable reductions in major motor seizure frequency on top of a background of standard anti-seizure medicines and improvements in multiple measures of cognition and behavior through two years of treatment in the OLE studies. Data indicated responses may be better among patients who were treated with loading doses of 70mg followed by maintenance doses of 45mg. Zorevunersen was generally well-tolerated across these studies. "Effects on behavior and cognition are a key secondary endpoint in our Phase 3 EMPEROR study," said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. "Feedback from caregivers and clinicians, and analyses like this one, give us insight into which assessments have the greatest potential to demonstrate meaningful effects for patients within the year-long treatment period." "Most patients with Dravet syndrome continue to experience seizures despite treatment with the best available anti-seizure medicines, and there are currently no medications approved that address the underlying cognitive and behavioral aspects of the disease," said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. "We look forward to continuing to work together to advance zorevunersen as a potential first-in-class disease modifying medicine for Dravet syndrome." Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) that is characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. There are no approved disease-modifying therapies for people living with Dravet syndrome. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the United States (~16,000), United Kingdom, EU-4 (Germany, France, Italy, Spain) and Japan.1 Description of the Modeled AnalysisA mixed-effects model for repeated measures (MMRM) analysis was used to evaluate the potential effects of the Phase 3 zorevunersen dosing regimen on patient cognition and behavior at Week 68. The model was developed using clinical data from patients in the Phase 1/2a ADMIRAL study and the LONGWING OLE study. An analysis of patients who received a total cumulative dose consistent with the Phase 3 EMPEROR regimen of two loading doses of 70mg followed by two maintenance doses of 45mg, showed improvements in cognition and behavior. The analysis was performed to inform the design of the Phase 3 EMPEROR study. Baseline covariates for patients followed in the BUTTERFLY natural history study were matched to the selected ADMIRAL patient population. Improvements in patients treated with zorevunersen contrasted with findings from BUTTERFLY. These data support the selection of five sub-domains of the Vineland-3 Adaptive Behavior Scales, including Receptive Communication, Expressive Communication, Interpersonal Relationships, Coping Skills, and Personal Skills now under evaluation as key secondary endpoints in the Phase 3 EMPEROR study. Details of the presentation are as follows: Title: Zorevunersen demonstrates potential as a disease modifying therapy in patients with Dravet syndrome through durable seizure reduction and improvements in cognition, behavior, and functioning with up to 24 months of maintenance dosing in open-label extension studiesPresenter: Andreas Brunklaus, M.D., Consultant Paediatric Neurologist at the Royal Hospital for Children, Glasgow, Honorary Professor at the University of GlasgowSession: Epilepsy IIDate/Time: Thursday, July 10, 11:51 AM CESTLocation: Saal 5, International Congress Center München, Germany The presentation will be available for download on the Stoke Therapeutics website under the Investors & News tab. About Dravet SyndromeDravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan.1 About ZorevunersenZorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights. About Stoke TherapeuticsStoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke's proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit About BiogenFounded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at Follow us on social media - Facebook, LinkedIn, X, YouTube. Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; and the design, timing and results of the Phase 3 EMPEROR study. Statements including words such as "anticipate," "could," "expect," "plan," "will," or "may" and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause Stoke's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: Stoke's ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Stoke's collaborators were to breach or terminate their agreements, it would not obtain the anticipated financial or other benefits; the possibility that Stoke and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; Stoke's ability to protect its intellectual property; Stoke's ability to fund development activities and achieve development goals through mid-2028; and the other risks and uncertainties described under the heading "Risk Factors" in Stoke's Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and Stoke undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Biogen Safe HarborThis press release contains forward-looking statements, relating to: our strategy and plans; the potential of, and expectations for, our commercial business and pipeline programs; clinical development programs, clinical trials, and data readouts and presentations; regulatory discussions, submissions, filings, and approvals; the potential benefits, safety, and efficacy of our and our collaboration partners' products and investigational therapies; and actions to improve the risk profile and productivity of R&D pipeline, collaborations, and business development activities. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "assume," "believe," "contemplate," "continue," "could," "estimate," "expect," "forecast," "goal," "guidance," "hope," "intend," "may," "objective," "outlook," "plan," "possible," "potential," "predict," "project," "prospect," "should," "target," "will," "would," and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. This press release includes, among others, forward-looking statements including relating to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome, the design, timing and results of the Phase 3 EMPEROR study and the potential effects of the Phase 3 zorevunersen dosing regimen. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document. These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q , in each case including in the sections thereof captioned "Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise. Reference: Based on Stoke Therapeutics' preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by Wu et al., Pediatrics, 2015. View source version on Contacts Stoke Media & Investor Contacts:Dawn KalmarChief Communications Officerdkalmar@ 781-303-8302Doug SnowDirector, Communications & Investor RelationsIR@ 508-642-6485Biogen Media Contact:Allison + 1 781 464 3260Biogen Investor Contact:Tim PowerIR@ +1 781 464 2442
Yahoo
27-06-2025
- Health
- Yahoo
More people can enjoy Des Moines Arts Festival thanks to Quincy's Sensory Space
DES MOINES, Iowa — Just leaving the house is no small undertaking for Quincy Hostager and his mom, Maria. 'It's a lot,' she says. 'He has a G tube, he has a pacemaker, he wears pull ups, he needs help, and he's heavy.' And that's heavy for their family. 'For years when someone would ask me how I was doing, that was kind of a dangerous question because they would get the real answer. And sometimes I would just start crying.' Quincy has Dravet Syndrome, a severe, rare form of epilepsy that's left him unable to walk or talk. 'I think once I quit resisting it so much it got better,' Maria explains, 'like, okay, how could we have joy given this situation? How could we make the most of this?' They've done it by continuing to do the things the family loves, like biking and listening to live music. 'We try to be our own kind of rebels by living the best life we can possibly live. And we show that no disease is going to get the better of us as a family, too. And so that's our act of rebellion. By having joy every day.' Those joyful outings have often included their friend, Stephen. 'And one of the things as a festival producer that always struck me was, you know, the process of them bringing Quincy to an experience like that and seeing Quincy enjoy it so much and then their time being cut short because there's nothing there for Quincy and or for the caretakers of Quincy if he needs recovery time, or if they just need a space to change or to regroup.' Sensory-friendly game Sunday for Iowa Cubs matchup against Columbus Clippers Seeing that frustration weighed heavily on him, so he decided to do something about it. 'I thought – we need to change this. We need to fundamentally rethink how we approach our festivals and this industry and provide an opportunity for everybody.' And so the idea of 'Quincy's Sensory Space' was born. Jill Wells is carrying the load when it comes to designing the enormous space, working with translucent vinyl to build the ceiling and walls. 'I'm constantly thinking; what would work for another person? What wouldn't work? So you get a really interesting experience of how does the material impact other people and I think that's why I've always loved art as a language.' Each area will have different elements — from water and rice for sensory play, to darkness and soothing sounds for relaxation. There will be five in all, stretching nearly 100 feet. 'So you'll have all these choices on how you engage in the festival in a way that meets your needs on that sensory level and that processing, and then go about your day again.' It's the sort of thing Quincy's family has dreamed of and they're thrilled to be part of lightening the load for anyone who refuses to let their needs exclude them from leading a joyful life. 'Is it going to be perfect?' Maria shrugs, 'No. Is it going to be exactly what we need? We don't know. But making the effort and going out of the way to try to include people is what our society is about. And that's what makes the places the best to be is when you all know each other and care about each other.' You'll find 'Quincy's Sensory Space' in the 1400 block of Locust. It's been made possible thanks to the generosity of DLR Group. Metro News: More people can enjoy Des Moines Arts Festival thanks to Quincy's Sensory Space Multiple pedestrians injured in separate Des Moines crashes For first time ever, UFA sends teams to nationals, and it's not just one One injured in shooting on Des Moines' eastside Deaf baseball camp visiting Iowa Cubs for 'Deaf Culture Night' Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Yahoo
17-06-2025
- Business
- Yahoo
Stoke Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
BEDFORD, Mass., June 17, 2025--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today announced that, effective on June 16, 2025, it granted stock options to purchase an aggregate of 37,200 shares of common stock to two new employees, as a material inducement to their employment in accordance with Nasdaq Listing Rule 5635(c)(4). The stock options that were granted have an exercise price of $11.70 per share, which is equal to the closing price of Stoke's common stock on June 16, 2025. Each option will vest over a 4-year period, with 1/4th of the shares underlying the employee's option vesting on the one-year anniversary of the applicable vesting commencement date and the remaining shares thereafter vesting monthly at a rate of 1/48th of the shares underlying each employee's option over the following 36 months, subject to the employee's continued employment with Stoke on such vesting dates. The options have a term of 10 years and are subject to the terms and conditions of the 2023 Inducement Plan and the stock option agreement covering the grant. About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for the Company's proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit View source version on Contacts Stoke Media & Investor Contacts: Dawn KalmarChief Communications Officerdkalmar@ 781-303-8302Doug SnowDirector, Communications & Investor RelationsIR@ 508-642-6485
Yahoo
15-06-2025
- Health
- Yahoo
Cornish charity boss completes month-long 5k-a-day challenge
A Cornish charity boss has completed a month-long challenge to walk or jog 5k every day. Claire Eldred, director at Dravet Syndrome UK, took part in the #EveryDayinMayForDravet challenge to help raise funds and awareness for the rare form of epilepsy. Dravet Syndrome is a severe neurological condition associated with epilepsy and learning disabilities, affecting one in 15,000 live births in the UK. Lucy Murphy, a young woman who has Dravet Syndrome, and Claire Eldred taking part in the challenge. (Image: supplied) Daily, those with Dravet often endure multiple seizures, and face a significantly higher risk of sudden unexpected death in epilepsy (SUDEP). Clare, who lives in Botallack, West Penwith, said: "I'm very proud to be doing #EveryDayinMayForDravet with and for all the families across the UK who are affected by this devastating, life limiting condition. "Sadly, it's all too common for individuals with Dravet Syndrome to have to wait months or even years to receive a diagnosis as sometimes even medics have not heard of Dravet. "I hope that raising awareness across Cornwall and the Southwest will lead to more families getting an earlier diagnosis and access to the treatment and support that they need." She also highlighted the importance of donations for the charity's continued work. Clare added: "As a small charity, all donations received make a huge difference in helping us to continue our vital work supporting and empowering families, educating professionals, and funding research. "A huge thank you to everyone who is sponsoring me or who is taking part or donating for #EveryDayinMayForDravet." The #EveryDayinMayForDravet challenge saw hundreds of participants across the globe, many of whom committed to 5k or 10k daily efforts. The challenge welcomed all ages and fitness levels, with some children participating in a 2k daily task. This family-friendly initiative is designed to not only raise funds but also to bolster community spirit and inclusivity among supporters. Dravet Syndrome UK continues to serve as a lighthouse for families who struggle with the complex challenges posed by Dravet Syndrome. They aim to ensure those affected receive timely diagnosis, treatment, and the essential support required for improved lives. To sponsor Claire or donate to the cause, visit the charity's JustGiving page. For more information, please visit Dravet Syndrome UK's website.
STV News
15-06-2025
- Health
- STV News
Rare form of epilepsy causing caregivers to develop PTSD
New research has shed light on the enormous psychological impact on families caring for a child or adult living with Dravet Syndrome. It's a devastating and complex form of epilepsy that affects around 1 in 15,000 babies – the mother of one woman living with the condition has compared the emotional impact to a form of PTSD. Marie McLeish's daughter, Amanda, was 18 months old when she suffered a series of debilitating seizures – a hundred in one day. She was eventually diagnosed with Dravet Syndrome – it's left her needing round-the-clock care for the last 36 years. 'We never slept properly for those first five years,' Marie told STV News. STV News Amanda McLeish Dravet Syndrome 'My husband and I would take shifts every night to make sure Amanda was safe. It has a major impact on the whole family, we're always on high alert. I know every sound in the house, it's 24/7.' She added: 'I don't actually think about it now, second nature to me, but it's had a huge impact on me. 'Anxiety levels, stress levels, I'm sure I've had PTSD throughout this time. It's very, very isolating as well.' The study, which was conducted by researchers at the University of Glasgow, provides new insight into the ways in which parents cope and adjust to caring for a child with Dravet Syndrome – with a focus on adapting to trauma. STV News Amanda McLeish Dravet Syndrome It highlights the challenges families face and reveals an urgent need for better informed professional practice, and the development of packages of support. 'We have done 35 years of caring and there is no end – there is no suitable accommodation or respite that would meet the actual needs of our loved ones,' Marie said. 'You just continue to do it through love. You continue to do it. You just hope and pray that something better will come.' Like her, parents taking part in the study described symptoms of psychological distress and PTSD. They also told how the lack of support needed to care for those affected presents a continual struggle for them with costs to their mental health. The condition can also cause intellectual disability and a range of difficulties with mobility, speech, behaviour, eating and sleeping. STV News Amanda McLeish Dravet Syndrome Additionally, those living with Dravet Syndrome are also at risk of Sudden Unexpected Death in Epilepsy – almost one in 5 children don't make it into adulthood. Marie said: 'You're putting your loved one to bed and it's the last thing that enters your head…hopefully we see you tomorrow. Galia Wilson, chair of Trustees for Dravet Syndrome UK, says: 'Dravet Syndrome is a devastating, life-long and life-changing condition that affects the emotional well-being of all the family, but all too often this is overlooked. 'Someone with Dravet Syndrome needs 24/7 care for life. Severe and life-threatening seizures mean parents must constantly be on alert, while the developmental, behavioural and mobility aspects of the condition require constant support and care. 'This research serves as a call to action for greater awareness among all professionals involved in the care of families affected by Dravet Syndrome – to be mindful of signs of psychological distress and trauma, and importance of offering therapeutic interventions, such as talking therapies. 'By empowering parents like Marie to share their experiences, Dravet Syndrome UK hopes to bridge the gaps in understanding and help drive meaningful change.' Get all the latest news from around the country Follow STV News Scan the QR code on your mobile device for all the latest news from around the country
