Rett syndrome

Anavex Announces Appointment of Dr. Marwan N Sabbagh, MD as Chairman of the Scientific Advisory Board

Retrieved on: 
Tuesday, September 12, 2023

Prof. Dr. Marwan Sabbagh, MD, is a behavioral neurologist in the Alzheimer’s and Memory Disorders Program at Barrow Neurological Institute.

Key Points: 
  • Prof. Dr. Marwan Sabbagh, MD, is a behavioral neurologist in the Alzheimer’s and Memory Disorders Program at Barrow Neurological Institute.
  • He is also a professor and the Vice Chair of Research in the Institute’s Department of Neurology.
  • He is board certified in neurology by the American Board of Psychiatry and Neurology.
  • “I’m excited to join Anavex’s Scientific Advisory Board at this time of important progress of the Company,” said Professor Dr. Sabbagh.

Taysha Gene Therapies Announces Fast Track Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome  

Retrieved on: 
Thursday, August 24, 2023

DALLAS, Aug. 24, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced the U.S. FDA has granted Fast Track Designation (FTD) to TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome. TSHA-102 utilizes the novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression.

Key Points: 
  • DALLAS, Aug. 24, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced the U.S. FDA has granted Fast Track Designation (FTD) to TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome.
  • FTD is designed to help treatments reach patients faster by facilitating the development and expediting the review of therapies with potential to address unmet medical needs for a serious or life-threatening condition.
  • Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease.
  • The U.S. FDA cleared the IND application for TSHA-102 in pediatric patients with Rett syndrome, and the Company expects to dose the first pediatric patient in the first quarter of 2024.

GordonMD® Invests in Private Placement for Taysha Gene Therapies

Retrieved on: 
Monday, August 21, 2023

GordonMD® Global Investments LP announced today it has participated in a $150 million private placement financing for Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS).

Key Points: 
  • GordonMD® Global Investments LP announced today it has participated in a $150 million private placement financing for Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS).
  • Rett syndrome is a rare genetic neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene.
  • TSHA-102 utilizes a novel miRARE platform designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression.
  • The firm manages a private fund and a public fund, each focused on differentiated investment opportunities in biopharmaceutical companies primarily located in the U.S., Europe and Japan.

Taysha Gene Therapies Announces $150 Million Private Placement Financing

Retrieved on: 
Monday, August 14, 2023

DALLAS, Aug. 14, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), announced today that it has entered into a securities purchase agreement for a private placement financing (the “PIPE”) that is expected to result in gross proceeds of approximately $150 million, before deducting placement agent commissions and offering expenses. The PIPE was led by new investor, RA Capital Management, with participation from a large institutional investor, PBM Capital, RTW Investments, LP, Venrock Healthcare Capital Partners, TCGX, Acuta Capital Partners, Kynam Capital Management, LP, Octagon Capital, Invus, GordonMD® Global Investments LP, and B Group Capital.

Key Points: 
  • Expected net proceeds, along with existing cash and cash equivalents, are expected to extend cash runway into the third quarter of 2025
    DALLAS, Aug. 14, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), announced today that it has entered into a securities purchase agreement for a private placement financing (the “PIPE”) that is expected to result in gross proceeds of approximately $150 million, before deducting placement agent commissions and offering expenses.
  • Each pre-funded warrant has an exercise price of $0.001 per share of common stock and is immediately exercisable and remains exercisable until exercised in full.
  • The PIPE is expected to close by August 16, 2023, subject to customary closing conditions.
  • Jefferies is acting as exclusive placement agent in the private placement.

Anavex Life Sciences Reports Fiscal 2023 Third Quarter Financial Results

Retrieved on: 
Tuesday, August 8, 2023

NEW YORK, Aug. 08, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today reported financial results for its fiscal quarter ended June 30, 2023.

Key Points: 
  • Company expects to announce topline results from this study in the second half of 2023.
  • Alzheimer’s disease: Full data ANAVEX®2-73-AD-004, including newly available preliminary results of surrogate biomarkers of pivotal Phase 2b/3 clinical trial.
  • General and administrative expenses for the quarter of $3.2 million compared to $3.2 million for the comparable quarter of fiscal 2022.
  • Research and development expenses for the quarter of $10.3 million compared to $9.3 million for the comparable quarter of fiscal 2022.

Anavex Life Sciences Reports Publication of ANAVEX®3-71 in Clinical Journal Confirming ANAVEX®3-71 Clinical Cardiovascular Safety

Retrieved on: 
Monday, August 7, 2023

The publication is entitled, ‘Concentration-QTc Relationship from a Single Ascending Dose Study of ANAVEX3-71, a Novel Sigma-1 Receptor and Allosteric M1 Muscarinic Receptor Agonist in Development for the Treatment of Frontotemporal Dementia, Schizophrenia, and Alzheimer's Disease’.

Key Points: 
  • The publication is entitled, ‘Concentration-QTc Relationship from a Single Ascending Dose Study of ANAVEX3-71, a Novel Sigma-1 Receptor and Allosteric M1 Muscarinic Receptor Agonist in Development for the Treatment of Frontotemporal Dementia, Schizophrenia, and Alzheimer's Disease’.
  • 1
    The publication reports the cardiodynamic evaluation part of the single ascending dose study in healthy participants with the primary objective of assessing the effect of ANAVEX®3-71 on ECG (electrocardiogram) parameters.
  • These data also expand the safety objectives met in this first-in-human study of ANAVEX®3-71, further supporting its drug development program.
  • “This published clinical study demonstrates Anavex’s commitment to advance Anavex’s clinical pipeline including ANAVEX®3-71 for Schizophrenia, Frontotemporal Dementia, and Alzheimer's Disease,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex.

Acadia Pharmaceuticals Reports Second Quarter 2023 Financial Results and Operating Overview

Retrieved on: 
Wednesday, August 2, 2023

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced its financial results for the second quarter ended June 30, 2023.

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced its financial results for the second quarter ended June 30, 2023.
  • “Our second quarter 2023 results reflect strong performances from both commercial franchises.
  • The Company recently aligned on plans with the FDA to initiate a Phase 3 study in the fourth quarter of 2023.
  • Acadia met with the FDA and aligned on dosing and plans to initiate a Phase 2/3 program in the fourth quarter of 2023.

Anavex Life Sciences to Present at the BTIG Virtual Biotechnology Conference 2023

Retrieved on: 
Wednesday, August 2, 2023

NEW YORK, Aug. 02, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders, today announced that Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex, will participate in a fireside chat at the BTIG Virtual Biotechnology Conference 2023, taking place on August 7-8, 2023.

Key Points: 
  • NEW YORK, Aug. 02, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders, today announced that Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex, will participate in a fireside chat at the BTIG Virtual Biotechnology Conference 2023, taking place on August 7-8, 2023.
  • The Fireside chat will take place on August 7, 2023, at 12:30 pm ET.
  • A replay will also be available through the BTIG Virtual Biotechnology Conference platform.

Anavex Life Sciences to Announce Fiscal 2023 Third Quarter Financial Results on Tuesday August 8, 2023

Retrieved on: 
Tuesday, August 1, 2023

NEW YORK, Aug. 01, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that it will issue financial results for its quarter ended June 30, 2023, on Tuesday August 8, 2023.

Key Points: 
  • NEW YORK, Aug. 01, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that it will issue financial results for its quarter ended June 30, 2023, on Tuesday August 8, 2023.
  • Management will host a conference call on Tuesday August 8, 2023, at 8:30 am ET to review financial results and provide an update on the execution of the Company’s growth strategy.
  • Following management’s remarks, there will be a question-and-answer session.
  • A replay of the conference call will also be available on Anavex’s website for up to 30 days.

Taysha Gene Therapies Announces Positive Recommendation from Independent Data Monitoring Committee of REVEAL Phase 1/2 Trial in Rett Syndrome

Retrieved on: 
Monday, July 31, 2023

DALLAS, July 31, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), announced today that the Independent Data Monitoring Committee (IDMC) recommended the continuation of the REVEAL Phase 1/2 trial and that dosing of the second patient in the first cohort can proceed. The decision follows a pre-specified IDMC review of initial clinical data from the first patient dosed with TSHA-102 following the 42-day evaluation period.

Key Points: 
  • The decision follows a pre-specified IDMC review of initial clinical data from the first patient dosed with TSHA-102 following the 42-day evaluation period.
  • “We thank the IDMC members for their guidance and are pleased with their recommendation to continue the REVEAL Phase 1/2 trial,” said Sukumar Nagendran, M.D., President and Head of R&D of Taysha.
  • “This recommendation was based on the analysis of initial clinical data from the first adult patient with Rett syndrome to receive TSHA-102.
  • The REVEAL Phase 1/2 trial is a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adult females with Rett syndrome due to MECP2 loss-of-function mutation.