American Epilepsy Society

Neurona Therapeutics Raises $120M to Advance Groundbreaking Pipeline of Regenerative Cell Therapy Candidates for Chronic Neurological Disorders

Retrieved on: 
Thursday, February 8, 2024

Proceeds from the financing will be used to advance the company’s pipeline of wholly-owned, off-the-shelf cell therapies for multiple indications, including its lead investigational candidate, NRTX-1001.

Key Points: 
  • Proceeds from the financing will be used to advance the company’s pipeline of wholly-owned, off-the-shelf cell therapies for multiple indications, including its lead investigational candidate, NRTX-1001.
  • “Neurona has pioneered development of a fully-differentiated cell therapy for drug-resistant focal epilepsy that is designed to be disease-modifying, repairing the affected neural network, and is yielding very promising initial clinical data.
  • It signifies the conviction that Neurona’s cell therapies have the potential to transform the treatment of previously refractory, devastating neurological disorders.
  • These subjects received a one-time administration of NRTX-1001 as well as temporary immunosuppression to promote the long-term persistence of the cell therapy.

Praxis Precision Medicines Provides Update on Advancing Clinical Stage Portfolio

Retrieved on: 
Monday, January 8, 2024

BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today provided an update on its portfolio and planned key milestones in 2024:

Key Points: 
  • Praxis made strong progress in developing PRAX-628 for focal epilepsy, which affects over 1 million Americans.
  • Initial data from PRAX-628 show it has a good tolerability profile, a potentially wide therapeutic index and reaching efficacious concentrations within 24 hours.
  • A preliminary analysis of the 15 mg cohort showed that this cohort exceeds the expectations in terms of drug activity.
  • In 2023, Praxis disclosed results from a Phase 1 dose escalation study of PRAX-628 in healthy volunteers:
    PRAX-628 was generally well-tolerated at all tested doses.

Stoke Therapeutics Highlights Strategic Priorities and Anticipated Milestones for 2024

Retrieved on: 
Monday, January 8, 2024

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced strategic priorities and anticipated milestones for 2024.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced strategic priorities and anticipated milestones for 2024.
  • 2024 is all about advancing this potential new medicine toward a registrational study,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics.
  • Stoke ended the third quarter of 2023 with $214.7 million in cash, cash equivalents and marketable securities, anticipated to fund operations to the end of 2025.
  • Stoke’s Presentation at the 42nd Annual J.P. Morgan Healthcare Conference
    Dr. Edward Kaye will discuss Stoke’s strategic priorities and anticipated milestones in a presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, 2024, at 6:45 p.m.

Milestone Scientific Commences Sales of CompuFlo®  Disposables for Use in Neurostimulation Spinal Cord Implantation Procedures

Retrieved on: 
Friday, December 8, 2023

ROSELAND, N.J., Dec. 08, 2023 (GLOBE NEWSWIRE) -- Milestone Scientific Inc. (NYSE:MLSS), a leading developer of computerized drug delivery instruments that provide painless and precise injections, today reported it has commenced commercial sales of CompuFlo® disposables for use in neurostimulation spinal cord stimulator implantation procedures at the University of Texas Medical Branch (UTMB) Health Clear Lake Campus Hospital, expanding use of CompuFlo® within the institution beyond the labor and delivery department and training for residents in epidural needle placement and verification.

Key Points: 
  • A spinal cord stimulator is an implanted device that sends low levels of electricity directly into the spinal cord to relieve pain.
  • An estimated 60,000 spinal cord stimulators are implanted each year.
  • Approval and adoption of the technology for neurostimulation spinal cord stimulator implantation follows a successful evaluation process by Dr. Patrick Karas, MD, a full-time faculty neurosurgeon at UTMB, operating at both the Galveston and Clear Lake campuses.
  • Dr. Karas completed 10 cases using the CompuFlo Epidural system, in which he reported 100% success and no complications.

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

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.

Ovid Therapeutics to Present Five Abstracts Supporting its Epilepsy Programs at the 77th American Epilepsy Society Annual Meeting (2023)

Retrieved on: 
Friday, December 1, 2023

and Chief Medical Officer of Ovid Therapeutics.

Key Points: 
  • and Chief Medical Officer of Ovid Therapeutics.
  • “GABA-AT is a validated target in seizure reduction that has been limited in clinical use to date.
  • Title: Blocking of GABA-AT Activity by OV329 Selectively Alters Tonic and Phasic Inhibition in Dentate Gyrus Granule Cells.
  • Title: Low, Repeat Dosing of OV329 Enhances GABA-AT Inhibition in Rodent Brain: Relationship between Pharmacokinetic (PK) and Pharmacodynamic (PD) Effects.

Neurona Therapeutics Presents New Clinical Data from First Cohort in Ongoing Phase I/II Open-Label Trial of NRTX-1001 Cell Therapy for Drug-resistant Mesial Temporal Lobe Epilepsy (MTLE)

Retrieved on: 
Friday, December 1, 2023

SAN FRANCISCO, Dec. 01, 2023 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, announced presentation of positive preliminary clinical data from the first cohort of five subjects in its ongoing open-label, single-arm Phase I/II clinical trial of NRTX-1001, an investigational allogeneic cell therapy candidate that is being developed for treatment of drug-resistant MTLE. The data will be presented at the Annual Meeting of the American Epilepsy Society, which is being held December 1-5, 2023 in Orlando, Florida.

Key Points: 
  • The data will be presented at the Annual Meeting of the American Epilepsy Society, which is being held December 1-5, 2023 in Orlando, Florida.
  • “The early data from the first cohort in the trial have been very encouraging, with substantial, durable seizure reduction in the first subjects with drug-resistant MTLE.
  • All subjects who received NRTX-1001 in the ongoing clinical trial entered the study with a history of significant monthly seizure activity that was not controlled by anti-seizure medications.
  • No severe adverse events from the cell therapy, delivery procedure, or immunosuppression regimen have been reported thus far in the ongoing clinical trial.

Avenue Therapeutics to Present at American Epilepsy Society 2023 Annual Meeting

Retrieved on: 
Friday, December 1, 2023

MIAMI, Dec. 01, 2023 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today announced that Amy Chappell, M.D., FAAN, will be presenting preclinical in vivo data evaluating BAER-101 using the SynapCell's Genetic Absence Epilepsy Rat from Strasbourg (“GAERS”) model of absence epilepsy at the American Epilepsy Society (AES) 2023 Annual Meeting in Orlando, FL on December 2, 2023.

Key Points: 
  • MIAMI, Dec. 01, 2023 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today announced that Amy Chappell, M.D., FAAN, will be presenting preclinical in vivo data evaluating BAER-101 using the SynapCell's Genetic Absence Epilepsy Rat from Strasbourg (“GAERS”) model of absence epilepsy at the American Epilepsy Society (AES) 2023 Annual Meeting in Orlando, FL on December 2, 2023.
  • The Company's presentation details are as follows:
    “We are pleased with the progress made with BAER-101, a molecule with unique pharmacology which has demonstrated that it can significantly suppress seizures in a translational animal model of absence epilepsy,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue.
  • In the model, BAER-101 demonstrated full suppression of seizure activity with a minimal effective dose of 0.3 mg/kg, PO.
  • The combination of safety and tolerability in hundreds of patients and the preclinical efficacy data support BAER-101’s continued development in a Phase 2a trial.

GeneDx Presents New Data at the American Epilepsy Society Annual Meeting Demonstrating That Exome Sequencing Outperforms Multi-Gene Panels for Patients with Epilepsy

Retrieved on: 
Friday, December 1, 2023

The gap in diagnosis between exome sequencing and the multi-gene epilepsy panels can be largely attributed to the fact that more than half (57%) of seizure-related genes are not included on many commercially available epilepsy panels.

Key Points: 
  • The gap in diagnosis between exome sequencing and the multi-gene epilepsy panels can be largely attributed to the fact that more than half (57%) of seizure-related genes are not included on many commercially available epilepsy panels.
  • Of those patients with a positive genetic diagnosis from exome sequencing, 11% had a variant in a treatment related gene.
  • GeneDx will also present data at the conference that shows insurance may offer better coverage for exome sequencing than multi-gene panels for patients with epilepsy.
  • At the conference, GeneDx will also be hosting an industry sponsored symposium discussing the ways in which exome sequencing can help epilepsy patients find answers sooner.

Praxis Precision Medicines to Showcase Largest Pipeline of Precision Epilepsy Programs and Breadth of Commitment to Epilepsy Treatments at Upcoming Meetings

Retrieved on: 
Tuesday, November 28, 2023

Presentations at the American Epilepsy Society (AES) Annual Meeting, as well as related scientific and patient advocacy meetings, will cover new patient data, updates on clinical progress and new paradigms for developing epilepsy therapies.

Key Points: 
  • Presentations at the American Epilepsy Society (AES) Annual Meeting, as well as related scientific and patient advocacy meetings, will cover new patient data, updates on clinical progress and new paradigms for developing epilepsy therapies.
  • ET [Platform D | Epilepsy Therapies]
    Summary: Combined preclinical and clinical data demonstrate PRAX-628 is differentiated from standard of care, with the potential to be best-in-class for focal epilepsy.
  • Summary: This study provides novel insights into the clinical validity of commonly used preclinical seizure models across the clinical epilepsy spectrum.
  • Oral presentation highlighting clinical updates from our PRAX-562 small molecule platform at the annual clinician, researcher, and family gathering for SCN8A