Zorevunersen
- Generic Name
- Zorevunersen
- Drug Type
- Biotech
- CAS Number
- 2415330-04-8
- Unique Ingredient Identifier
- U4YV46D2DT
Breakthrough Drug Trial Shows "Life-Changing" Results for Children with Dravet Syndrome
• A clinical trial of zorevunersen at Sheffield Children's Hospital has dramatically improved the lives of children with Dravet syndrome, a rare form of epilepsy affecting approximately one in 15,000 babies.
• Seven-year-olds Albie Kelly and Freddie Truelove, who were previously non-verbal and experienced frequent seizures, have shown remarkable improvements in speech, mobility, and independence after receiving the trial medication.
• The pioneering treatment has reduced seizure frequency significantly, with some participants going up to 10 days without seizures, while also enabling children to develop new skills like swimming, skiing, and forming friendships.
Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas
• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics.
• February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million.
• Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.
Biogen Secures Global Rights to Novel Dravet Syndrome Treatment in $550M Deal
• Biogen has entered a strategic partnership with Stoke Therapeutics, investing up to $550 million for ex-North American rights to zorevunersen, a promising treatment for Dravet syndrome.
• Early-stage trials of zorevunersen have demonstrated encouraging results, showing reductions in seizure frequency and improvements in cognition and behavior in Dravet syndrome patients.
• The late-stage study of zorevunersen is scheduled to commence in Q2 2024, with results expected in 2027, targeting approximately 38,000 patients across major markets.
Rapport Therapeutics' RAP-219 Shows Promise in Phase 1 Trials for CNS Disorders
• Rapport Therapeutics' RAP-219 achieved target receptor occupancy within five days, supporting its potential for treating CNS disorders.
• The Phase 1 trials demonstrated a favorable tolerability profile for RAP-219, with no serious adverse events reported in healthy volunteers.
• RAP-219 selectively targets TARPγ8-associated AMPA receptors, potentially enhancing the therapeutic index for AMPA receptor modulation.
• A Phase 2a trial of RAP-219 in focal epilepsy is ongoing, with topline data expected in mid-2025, according to Rapport Therapeutics.
J&J's Alzheimer's Therapies, Posdinemab and JNJ-2056, Receive FDA Fast Track Designation
• Johnson & Johnson's posdinemab, a monoclonal antibody targeting phosphorylated tau, has received FDA Fast Track designation for early Alzheimer's treatment.
• JNJ-2056, an anti-tau active immunotherapy, also received Fast Track status, marking the second such designation for J&J's Alzheimer's portfolio in 2024.
• Both therapies are currently in Phase 2b trials, with posdinemab in the "AuTonomy" study and JNJ-2056 in the "ReTain" trial, aimed at slowing disease progression.
• The FDA's Fast Track designation is designed to expedite the review of drugs addressing serious conditions with unmet medical needs, potentially accelerating patient access.
Stoke Therapeutics Receives Buy Rating Based on Strong Phase 3 Trial Design and Early Results
Stoke Therapeutics has been given a Buy rating by analyst Andrew Fein from H.C. Wainwright, supported by the promising design of its Phase 3 trial for zorevunersen and positive early results in reducing seizure frequency in Dravet syndrome patients.
Biogen and Stoke Therapeutics Collaborate to Advance Zorevunersen for Dravet Syndrome
• Biogen and Stoke Therapeutics will collaborate to develop and commercialize zorevunersen for Dravet syndrome outside the U.S., Canada, and Mexico.
• Stoke's Phase 3 EMPEROR study of zorevunersen, expected to begin in mid-2025, aims to reduce seizure frequency and improve cognition and behavior in children with Dravet syndrome.
• Zorevunersen, an antisense oligonucleotide targeting the SCN1A gene, has shown promising results in earlier trials, including an 87% median reduction in convulsive seizure frequency.
• Biogen will provide Stoke with an upfront payment of $165 million, potential milestone payments up to $385 million, and tiered royalties on net sales in Biogen's territory.
FDA Roundup: Approvals, Breakthrough Designations, and Clinical Holds in Neurology and Rare Diseases
• The FDA granted breakthrough therapy designation to STK-001 for Dravet syndrome, highlighting its potential to improve upon current treatments by restoring NaV1.1 protein levels.
• Tolebrutinib received breakthrough therapy designation for non-relapsing secondary progressive multiple sclerosis based on phase 3 trial results showing delayed disability progression.
• The FDA placed a clinical hold on PepGen’s PGN-EDO51 phase 2 study for Duchenne muscular dystrophy, pending further clarification from the agency.
Zorevunersen Receives FDA Breakthrough Therapy Designation for Dravet Syndrome
• Stoke Therapeutics' zorevunersen has received FDA Breakthrough Therapy Designation for Dravet syndrome with confirmed SCN1A gene mutation.
• Clinical data from Phase 1/2a and open-label extension studies demonstrated substantial seizure reduction and improvements in cognition and behavior.
• Zorevunersen is a novel antisense oligonucleotide designed to upregulate NaV1.1 protein expression, addressing the underlying cause of Dravet syndrome.
• Stoke plans to provide an update on its global Phase 3 registrational study plans by the end of the year.
Stoke Therapeutics Advances RNA-Based Therapy Zorevunersen for Dravet Syndrome
• Stoke Therapeutics is advancing zorevunersen, an RNA-based drug, into a Phase 3 trial for Dravet syndrome after promising mid-stage study results.
• Zorevunersen aims to increase protein production in cells, offering a novel approach compared to gene silencing therapies for genetic diseases.
• The company is also focusing on manufacturing and patient access to ensure smooth commercialization, drawing on past experiences to avoid supply issues.
• Stoke is collaborating with Acadia Pharmaceuticals to develop treatments for rare genetic neurodevelopmental diseases, expanding its pipeline.
Stoke Therapeutics Advances Dravet Syndrome Treatment with Zorevunersen
• Stoke Therapeutics is progressing zorevunersen (STK-001) for Dravet syndrome, with ongoing discussions with regulatory agencies for a global Phase 3 study.
• Recent data from clinical studies of zorevunersen demonstrate substantial and sustained effects across multiple measures of Dravet syndrome.
• The FDA has removed the partial clinical hold on higher doses of zorevunersen, allowing for continued chronic dosing in ongoing trials.
• Stoke Therapeutics reported $269.2 million in cash, cash equivalents, and marketable securities as of September 30, 2024, ensuring operations are funded through at least the end of 2025.
Stoke Therapeutics Advances Zorevunersen for Dravet Syndrome to Phase III After FDA Lifts Clinical Hold
• Stoke Therapeutics is set to initiate Phase III trials for zorevunersen in Dravet syndrome following the FDA's decision to lift its clinical hold.
• Zorevunersen, an SCN1A activator, has demonstrated positive efficacy in earlier trials, reducing convulsive seizure frequency in children and adolescents with Dravet syndrome.
• The company plans to discuss Phase III trial designs with the FDA and other global regulatory agencies in the second half of 2024, aiming for a unified global study.
• Dravet syndrome, a severe genetic epilepsy with limited treatment options, highlights the need for new therapies like zorevunersen to improve seizure control and overall prognosis.
Stoke Therapeutics Announces Alignment with Global Regulatory Agencies
Stoke Therapeutics has achieved alignment with the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the EMPEROR study, focusing on zorevunersen's effects on major motor seizure frequency, behavior, and cognition in children and adolescents.
Stoke Therapeutics' STK-001 Shows Promising Results in Dravet Syndrome Trials
• Stoke Therapeutics' STK-001 demonstrated substantial reductions in convulsive seizure frequency in children and adolescents with Dravet syndrome.
• The treatment also showed improvements in cognition and behavior, suggesting potential disease modification in this patient population.
• The FDA has allowed Stoke to proceed with a dosing regimen of 70mg followed by 45mg, paving the way for registrational studies.
• Stoke Therapeutics is preparing to meet with regulatory agencies to discuss a registrational study based on the encouraging data.
Stoke Therapeutics Plans Phase 3 Trial of STK-001 for Dravet Syndrome Following Promising Phase 1/2a Data
• Stoke Therapeutics is advancing STK-001, a novel treatment for Dravet syndrome, into a Phase 3 trial in 2024 based on encouraging Phase 1/2a results.
• Interim data from the MONARCH and ADMIRAL trials showed STK-001 safely and effectively reduced seizure frequency in children and adolescents with Dravet syndrome.
• STK-001 aims to restore normal levels of NaV1.1 protein in nerve cells by increasing its production from the healthy SCN1A gene copy.
• Additional data expected in 2023 will inform dose level and frequency for the Phase 3 program, potentially shifting Dravet syndrome treatment from seizure management to comprehensive syndrome management.
Advancements in Neurology: From Hunter Syndrome to Alzheimer's Disease
Recent studies and trials in neurology have shown promising results across a range of conditions, including Hunter syndrome, Alzheimer's disease, and Dravet syndrome. Innovations in treatment and diagnostic tools are paving the way for more effective management of these complex conditions.
Drug Development Updates
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