SCN1A

Xenon Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, February 29, 2024
Research, FOS, FDA, Pain, Food, Drug discovery, SCN8A, Epilepsy, Safety, SCN1A, Neurocrine Biosciences, Nav1.7, Webcast, Depression, Patient, OLE, KV7, DSM-IV codes, MDD, IND, NDA, Ageing, Neurology, Pharmaceutical industry

VANCOUVER, British Columbia, Feb. 29, 2024 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today reported financial results for the fourth quarter and full year ended December 31, 2023 and provided a corporate update.

Key Points: 
  • We also continue to evaluate other neurological indications that present a potential fit for XEN1101’s novel mechanism and product profile.
  • Other income was $8.7 million for the fourth quarter of 2023, and $31.4 million for the year ended 2023, compared to $7.1 million and $3.9 million for the same periods in 2022.
  • Net loss was $44.7 million for the fourth quarter of 2023, and $182.4 million for the year ended 2023, compared to $37.4 million and $125.4 million for the same periods in 2022, respectively.
  • Xenon will host a conference call and webcast today at 4:30 pm Eastern Time (1:30 pm Pacific Time) to discuss its fourth quarter and full year 2023 results.

Encoded Therapeutics Announces US IND Clearance and Australian CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101

Retrieved on: 
Tuesday, February 6, 2024
Children, Biotechnology, FDA, Health, General Health, Pharmaceutical, Neurology, Health Technology, Genetics, Consumer, Clinical Trials, CTA, Patient, IND, Central nervous system, GABAergic, Dravet syndrome, CNS, Food, SCN1A, Disease, Therapy, Pharmaceutical industry

Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome.

Key Points: 
  • Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome.
  • Dravet syndrome is primarily caused by loss-of-function variants in the SCN1A gene, accounting for over 85% of cases.
  • ETX101 is an AAV9-mediated candidate gene regulation therapy designed to selectively upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons and potentially address the underlying cause of the disease.
  • Encoded has received clearance for its Investigational New Drug (IND) application from the US Food and Drug Administration and approval under the Clinical Trial Approval (CTA) scheme from the Australia Therapeutic Goods Administration to initiate clinical trials of its gene therapy candidate, ETX101.

Orphan designation: Adeno-associated virus serotype 9 expressing a transcription factor for the SCN1A gene Treatment of Dravet syndrome, 22/05/2023 Positive

Retrieved on: 
Wednesday, January 24, 2024
COMP, Dravet syndrome, Committee, European Commission, IRIS, Patient, SCN1A, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Adeno-associated virus serotype 9 expressing a transcription factor for the SCN1A gene
    - Intended use
    - Treatment of Dravet syndrome
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2780
    - Date of designation
    - Sponsor
    Pharma Gateway AB
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Xenon Pharmaceuticals Outlines Key Milestone Opportunities for 2024

Retrieved on: 
Monday, January 8, 2024
SCN1A, Drug discovery, DSM-IV codes, IND, Nav1.7, Depression, Epilepsy, FOS, Pain, SCN8A, Ageing, KV7, Food, Neurocrine Biosciences, MDD, Patient, Safety, OLE, Channel modulator, Pharmaceutical industry

VANCOUVER, British Columbia, Jan. 08, 2024 (GLOBE NEWSWIRE) --  Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today outlined progress within its pipeline programs and key milestones for 2024.

Key Points: 
  • VANCOUVER, British Columbia, Jan. 08, 2024 (GLOBE NEWSWIRE) --  Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today outlined progress within its pipeline programs and key milestones for 2024.
  • Xenon anticipates that patient enrollment in X-TOLE2 will be completed in the second half of 2024.
  • Xenon is actively assessing various clinical and regulatory pathways to support late-stage clinical development of XEN1101 in MDD and expects to initiate the Phase 3 clinical program in 2024.
  • The near-term focus is on development candidates targeting Kv7, Nav1.1 and Nav1.7 where Xenon expects multiple candidates will enter IND-enabling studies in 2024 and 2025.

Xenon Pharmaceuticals Provides Updates on Neurology Pipeline Programs at the Annual Meeting of the American Epilepsy Society (AES 2023)

Retrieved on: 
Saturday, December 2, 2023
Xenon, Quality of life, Skill, SCN1A, Convention, Aes, Efficacy, Depression, Epilepsy, AES, Poster, American Epilepsy Society, ASMS, DSM-IV codes, SFG, Seizure, MPC, Dravet syndrome, Anxiety, Annual general meeting, Neurology, Food, Safety, MDD, Patient, Orange County Convention Center, OLE, Pharmaceutical industry

VANCOUVER, British Columbia, Dec. 02, 2023 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today announced it will provide updates on its neurology programs at the Annual Meeting of the American Epilepsy Society (AES 2023).

Key Points: 
  • VANCOUVER, British Columbia, Dec. 02, 2023 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today announced it will provide updates on its neurology programs at the Annual Meeting of the American Epilepsy Society (AES 2023).
  • Importantly, we are looking forward to presenting new interim data from our ongoing open-label extension study from our Phase 2b X-TOLE trial.
  • The exhibit will also feature the topline results from the Phase 2 proof-of-concept X-NOVA clinical trial in major depressive disorder.
  • Posters will be added to the Xenon website consistent with AES 2023 conference guidelines.

Catalyst Pharmaceuticals Announces a Recent Third-Party Publication in Peer-Reviewed Journal Epilepsia Showing Perampanel (FYCOMPA®) Demonstrated High Efficacy in Patients with Rare Genetic Epilepsies

Retrieved on: 
Wednesday, March 8, 2023
Perampanel, Catalyst Pharmaceuticals, PIGA, NEU1, PCDH19, Patient, Network, Seizure, POLG2, SYNGAP1, Rare, Neurology, CORAL, GNAO1, Pharmaceutical industry, Catalysis, Epilepsy, SCN1A

The case review from a group of European neurologists treating rare epilepsies working under the framework of NETRE (Network for Therapy in Rare Epilepsies) reports how perampanel (FYCOMPA®) is capable of significantly reducing seizures in patients with epilepsies that result from specific genetic mutations.

Key Points: 
  • The case review from a group of European neurologists treating rare epilepsies working under the framework of NETRE (Network for Therapy in Rare Epilepsies) reports how perampanel (FYCOMPA®) is capable of significantly reducing seizures in patients with epilepsies that result from specific genetic mutations.
  • Out of 137 patients treated with perampanel, 60 patients (43.5%) sustained over 75% reduction in seizure frequency, including 38 (27.5%) with greater than 90% reduction in seizure frequency.
  • High efficacy was consistently observed in certain rare genetic variants, including SCN1A, GNAO1, PIGA, PCDH19, SYNGAP1, POLG1, POLG2, and NEU1.
  • “We look forward to continuing to bring FYCOMPA® to all patients that may benefit from it.”

Stoke Therapeutics Presents Data from a Combined Interim Analysis of the Phase 1/2a MONARCH and ADMIRAL Studies of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2022 Annual Meeting

Retrieved on: 
Friday, December 2, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Linda, Safety, International League Against Epilepsy, Intellectual disability, Pharmacokinetics, Fenfluramine, DS, Patient, Adolescence, Language, Hospital, AES, Phase, Caregiver, Quality of life, Associate, Movement, Therapy, EMA, Cognition, SUDEP, Development of the nervous system, Lurie Children's Hospital, Dravet syndrome, Seizure, Even and odd functions, Syndrome, Autonomic nervous system, Helen Cross, Disease, Stoke, Annual general meeting, Diagnosis, Death, Ann, MB, Sleep, Medicine, SCN1A, Government budget balance, ASO, DMPK, OLE, Plasma, Prognosis, American Epilepsy Society, Observational study, FDA, Senior, Feinberg School of Medicine, Neurology, European Journal of Paediatric Neurology, Risk, Cerebrospinal fluid, Pharmaceutical industry, Vaccine, Medical imaging, Pediatric nursing, BioAnalyt, Epilepsy

A total of seven posters will be presented at the American Epilepsy Society (AES) 2022 Annual Meeting, December 2-6.

Key Points: 
  • A total of seven posters will be presented at the American Epilepsy Society (AES) 2022 Annual Meeting, December 2-6.
  • Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures beginning within the first year of life.
  • STK-001 represents an entirely new approach to treatment, one that aims to treat the syndrome, not just the seizures.
  • Topline data from a combined interim analysis of the Phase 1/2a MONARCH and ADMIRAL studies showed single and multiple doses of STK-001 up to 45mg were well-tolerated.

Stoke Therapeutics to Present Data from the Company’s Dravet Syndrome Program at the American Epilepsy Society 2022 Annual Meeting

Retrieved on: 
Tuesday, November 29, 2022
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Linda, Development of the nervous system, American Epilepsy Society, Dravet syndrome, Risk, Cognition, Therapy, International League Against Epilepsy, Ann, Government budget balance, OLE, Stoke, Neurology, Cerebrospinal fluid, Associate, SUDEP, Patient, Intellectual disability, Hospital, Autonomic nervous system, Caregiver, Movement, Medicine, European Journal of Paediatric Neurology, Prognosis, ASO, Seizure, Plasma, FDA, Diagnosis, EMA, Disease, Safety, Sleep, Adolescence, Lurie Children's Hospital, 2021 Essex County Council election, DMPK, AES, MB, Language, Annual general meeting, Senior, SCN1A, Death, Quality of life, Feinberg School of Medicine, Pharmaceutical industry, Paint, Vaccine, Epilepsy, Pharmacokinetics, Pediatric nursing, BioAnalyt

The company is advancing STK-001 as potentially the first medicine to treat the underlying cause of Dravet syndrome.

Key Points: 
  • The company is advancing STK-001 as potentially the first medicine to treat the underlying cause of Dravet syndrome.
  • Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP.
  • STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials.
  • Stokes first compound, STK-001, is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy.

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates

Retrieved on: 
Monday, November 14, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Even and odd functions, Brain, Epilepsy, Sleep, Autonomic nervous system, Safety, Fenfluramine, Disease, Risk, License, Cognition, Plasma, SCN1A, Targeted molecular therapy for neuroblastoma, Dravet syndrome, OLE, Death, Research, Government budget balance, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Seizure, CSF, Diagnosis, TANGO, EMA, Patient, FDA, Language, United Kingdom, Intellectual disability, Prognosis, Therapy, Caregiver, Quality of life, Pharmacokinetics, Collaboration, ID, Asos, Protein, RNA, Food, American Epilepsy Society, Cerebrospinal fluid, ASO, Medicine, Haploinsufficiency, SUDEP, Movement, Pharmaceutical industry, Medical imaging, Coffee, Reinsurance, Animal, Vaccine, Stoke

MONARCH and ADMIRAL are multi-center, Phase 1/2a studies of children and adolescents who have an established diagnosis of Dravet syndrome.

Key Points: 
  • MONARCH and ADMIRAL are multi-center, Phase 1/2a studies of children and adolescents who have an established diagnosis of Dravet syndrome.
  • Today, the Company provided topline results from the ongoing Phase 1/2a MONARCH and ADMIRAL studies of STK-001.
  • Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
  • Using Stokes proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels.

Bright Minds Biosciences Initiates Dosing in Phase I Clinical Trial of BMB-101 for Dravet Syndrome

Retrieved on: 
Wednesday, August 31, 2022
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The Phase I trial is being conducted in Adelaide, Australia, by CMAX Clinical Research, a clinical trial center specializing in a range of early-phase trials and first-time in-human studies.

Key Points: 
  • The Phase I trial is being conducted in Adelaide, Australia, by CMAX Clinical Research, a clinical trial center specializing in a range of early-phase trials and first-time in-human studies.
  • The trial aims to evaluate the safety, tolerability and other pharmacokinetics of BMB-101 for use in Phase II clinical trials.
  • Dosing the initial subject in the first-in-human trial of BMB-101 is an exciting milestone for Bright Minds.
  • Forward looking information in this news release contains information related to the Phase I clinical trial and the future therapeutic potential of BMB-101.