Rett syndrome

Anavex Life Sciences Provides an Update on Rett Syndrome Program

Retrieved on: 
Tuesday, January 2, 2024
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NEW YORK, Jan. 02, 2024 (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 today reported topline results from the randomized, double-blind, placebo-controlled, Phase 2/3 EXCELLENCE clinical trial, which evaluated the clinical efficacy, safety, and tolerability of 30 mg ANAVEX®2-73 in 92 pediatric patients with Rett syndrome (RTT) between the ages of 5 through 17 years. Participants were randomized 2:1 (ANAVEX®2-73 [62 patients] to placebo [30 patients]) for 12 weeks, followed by a week 16 safety visit. As well, Anavex reported positive Real World Evidence (RWE) feedback from Rett syndrome patients under Compassionate Use Authorization.

Key Points: 
  • As well, Anavex reported positive Real World Evidence (RWE) feedback from Rett syndrome patients under Compassionate Use Authorization.
  • After 12 weeks, the study showed improvement on the key co-primary endpoint Rett Syndrome Behaviour Questionnaire (RSBQ), which is a detailed 45-item questionnaire for assessing multiple Rett syndrome characteristics by the patients’ caregivers.
  • As of today, some patients with Rett syndrome have been on ANAVEX®2-73-treatment for over 4 years, combined OLE and Compassionate Use Program.
  • In addition to Rett syndrome, Anavex is evaluating ANAVEX®2-73 in other neurodevelopmental disorders, including Fragile X syndrome, and in neurodegenerative disorders like Parkinson’s disease.

Anavex Announces First Entire Clinical Gene Pathway Data of ANAVEX®2-73 from AVATAR Study in Patients with Rett Syndrome

Retrieved on: 
Wednesday, December 20, 2023
RNA, DSM-IV codes, RNA-Seq, AVATAR, Rett syndrome, Oxidative phosphorylation, Sequence, DNA, Gene expression, Neurodevelopmental disorder, CNS, Gene, Biomarker, Central nervous system, Patient, WES, Data collection, Therapy, Pharmaceutical industry

A whole genome and exome analysis comparing drug and placebo in patients with Rett syndrome was performed.

Key Points: 
  • A whole genome and exome analysis comparing drug and placebo in patients with Rett syndrome was performed.
  • AVATAR study was a randomized, placebo-controlled clinical trial in 33 patients with Rett syndrome which included prespecified biomarkers of response as well as Whole Exome Sequencing (WES) DNA data and full RNA exome expression (RNA-seq) data collection.
  • ANAVEX®2-73 transcriptomics analysis (RNAseq) identified gene networks that are differentially expressed in Rett syndrome patients treated with ANAVEX®2-73 compared to placebo.
  • Patient samples that were analyzed contained on average over 20 million unique reads in both placebo and ANAVEX®2-73 treated patients.

Stride Autism Centers Announces Expansion to Broaden Access to Premier Autism Therapy in Nebraska

Retrieved on: 
Thursday, November 30, 2023
Suite, Rett syndrome, LCP, Research, BCBA, Applied behavior analysis, Spectrum, ABA, Doctor of Philosophy, Stride, NCSP, LMFT, Nursing

OMAHA, Neb., Nov. 29, 2023 (GLOBE NEWSWIRE) -- Stride Autism Centers ("Stride"), renowned for their evidence-based therapy for children with autism, is proud to announce its expansion across Nebraska.

Key Points: 
  • OMAHA, Neb., Nov. 29, 2023 (GLOBE NEWSWIRE) -- Stride Autism Centers ("Stride"), renowned for their evidence-based therapy for children with autism, is proud to announce its expansion across Nebraska.
  • With three successful centers in Omaha and Lincoln, Stride is set to elevate its mission further with two new centers slated to open by March 2024.
  • Specializing in Applied Behavior Analysis (ABA) therapy , Stride's clinical team offers the “gold standard” of treatment for children with autism, backed by a solid scientific foundation.
  • I founded Stride so individuals like her can get the effective therapy they need as early as possible to live more independent, joyous lives.

Taysha Gene Therapies Announces Expanded Eligibility in REVEAL Phase 1/2 Adult Trial to Include Adolescent Rett Syndrome Patients

Retrieved on: 
Wednesday, November 29, 2023
MTD, Adolescence, Index, IND, Rett syndrome, FDA, CMC, Ageing, Investigational New Drug, AAV, CNS, Regulatory affairs, Health Canada, Central nervous system, Patient, Safety, Rett, MECP2, Senior, Pharmaceutical industry

DALLAS, Nov. 29, 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 that Health Canada has authorized the protocol amendment to the ongoing REVEAL Phase 1/2 adult trial evaluating TSHA-102 that expands eligibility to include patients aged 12 and older with Rett syndrome.

Key Points: 
  • DALLAS, Nov. 29, 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 that Health Canada has authorized the protocol amendment to the ongoing REVEAL Phase 1/2 adult trial evaluating TSHA-102 that expands eligibility to include patients aged 12 and older with Rett syndrome.
  • “Following review of the initial clinical data from the first two adult patients treated with TSHA-102 and Chemistry, Manufacturing, and Controls (CMC) data, Health Canada has authorized our protocol amendment to include adolescent patients aged 12 years and older in the ongoing REVEAL Phase 1/2 adult trial,” said Sukumar Nagendran, M.D., President, and Head of R&D of Taysha.
  • “Amending our protocol broadens the patient population who can potentially benefit from TSHA-102.
  • The maximum tolerated dose (MTD) or maximum administered dose (MAD) established will then be administered during dose expansion.

Anavex Life Sciences Reports Fiscal 2023 Year End Financial Results and Provides Business Update

Retrieved on: 
Monday, November 27, 2023
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NEW YORK, Nov. 27, 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 year ended September 30, 2023.

Key Points: 
  • “There is a high demand from Alzheimer’s disease patients and families for easy access and scalable treatment options.
  • The Marketing Authorisation would allow direct market access throughout the European Union for oral blarcamesine for the treatment of Alzheimer’s disease.
  • On November 22, 2023, the Company announced that the United States Patent and Trademark Office (USPTO) granted U.S. Patent No.
  • The financial information for the fiscal year ended September 30, 2023, should be read in conjunction with the Company’s consolidated financial statements, which will appear on EDGAR, www.sec.gov and will be available on the Anavex website at www.anavex.com .

Anavex Life Sciences to Announce Fiscal 2023 Year End Financial Results on Monday, November 27, 2023

Retrieved on: 
Friday, November 24, 2023
DSM-IV codes, Conference, Central nervous system, Rett syndrome, Conference call, Therapy, Webcast, CNS

NEW YORK, Nov. 24, 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 fiscal year ended September 30, 2023, on Monday November 27, 2023.

Key Points: 
  • NEW YORK, Nov. 24, 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 fiscal year ended September 30, 2023, on Monday November 27, 2023.
  • Management will host a conference call on Monday November 27, 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.

Anavex Life Sciences Announces Grant of U.S. Patent Covering Blarcamesine (ANAVEX®2-73) for Treatment of Insomnia or Anxiety

Retrieved on: 
Wednesday, November 22, 2023
DSM-IV codes, Rett syndrome, Agitation, Insomnia, Patent, Doctor of Philosophy, Anxiety, Institutionalisation, CNS, Central nervous system, Patient, Therapy, Pharmaceutical industry

11,813,242 entitled “A2-73 AS A THERAPEUTIC FOR INSOMNIA, ANXIETY, AND AGITATION”.

Key Points: 
  • 11,813,242 entitled “A2-73 AS A THERAPEUTIC FOR INSOMNIA, ANXIETY, AND AGITATION”.
  • This patent expands Anavex’s existing patent coverage of ANAVEX®2-73 (blarcamesine), including U.S. Patent No.
  • 11,337,953 to cover Anavex’s leading drug candidate, ANAVEX®2-73 (blarcamesine), ANAVEX®1-41 and ANAVEX®19-144, for treating insomnia, anxiety or agitation.
  • Together with Anavex U.S. Patent No.

Neurogene Announces Closing of Merger with Neoleukin Therapeutics and Concurrent Private Placement of $95 Million

Retrieved on: 
Tuesday, December 19, 2023
Oncology, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Janus, DSM-IV codes, Food, Cooley LLP, Rett syndrome, FDA, Stifel, Trial of the century, Orphan, SVB Securities, Investment, MECP2, Toxicity, Patient, CLN5, Batten disease, Exercise, Pharmaceutical industry

Neurogene Inc. (NASDAQ: NGNE) (“Neurogene”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the closing of its merger with Neoleukin Therapeutics, Inc. (“Neoleukin”).

Key Points: 
  • Neurogene Inc. (NASDAQ: NGNE) (“Neurogene”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the closing of its merger with Neoleukin Therapeutics, Inc. (“Neoleukin”).
  • Neurogene shares are expected to begin trading on the NASDAQ Global Market under the ticker “NGNE” beginning today at the market open.
  • In the reverse stock split, every four shares of Neoleukin common stock outstanding were combined and reclassified into one share of Neoleukin common stock.
  • Gibson Dunn & Crutcher LLP served as legal counsel to Neurogene and Cooley LLP served as legal counsel to the placement agents.

Trinity Capital Inc. Provides $40 Million Term Loan to Taysha Gene Therapies

Retrieved on: 
Tuesday, December 19, 2023
Central nervous system, Patient, Neurodevelopmental disorder, Creditor, Rett syndrome, Disease, AAV, Security, Pharmaceutical industry

PHOENIX, Dec. 19, 2023 /PRNewswire/ -- Trinity Capital Inc. (NASDAQ: TRIN) ("Trinity"), a leading provider of diversified financial solutions to growth-stage companies, today announced the commitment of $40 million in term loans to Taysha Gene Therapies, Inc. (NASDAQ: TSHA) ("Taysha"), a clinical-stage gene therapy company, pursuant to a Loan and Security Agreement dated November 13, 2023, by and among Taysha, the lenders party thereto from time to time (the "Lenders"), and Trinity, as administrative agent and collateral agent for the Lenders.

Key Points: 
  • PHOENIX, Dec. 19, 2023 /PRNewswire/ -- Trinity Capital Inc. (NASDAQ: TRIN) ("Trinity"), a leading provider of diversified financial solutions to growth-stage companies, today announced the commitment of $40 million in term loans to Taysha Gene Therapies, Inc. (NASDAQ: TSHA) ("Taysha"), a clinical-stage gene therapy company, pursuant to a Loan and Security Agreement dated November 13, 2023, by and among Taysha, the lenders party thereto from time to time (the "Lenders"), and Trinity, as administrative agent and collateral agent for the Lenders.
  • Taysha is focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system ("CNS").
  • Its lead clinical program TSHA-102 is in evaluation for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that treat the root cause of the disease.
  • With the term loan, Taysha believes it will be able to fund its operating expenses and capital requirements into 2026 to support the clinical development of its TSHA-102 program in Rett syndrome.

Taysha Gene Therapies Reports Third Quarter 2023 Financial Results and Provides Corporate and Clinical Updates

Retrieved on: 
Tuesday, November 14, 2023
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Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic function, socialization, and gross and fine motor skills.

Key Points: 
  • Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic function, socialization, and gross and fine motor skills.
  • Additional information on available clinical data is available in the Company’s quarterly report on Form 10-Q for the quarter ended September 30, 2023, to be filed with the SEC.
  • Cash and cash equivalents: As of September 30, 2023, the Company had cash and cash equivalents of $164.3 million.
  • Taysha management will hold a conference call and webcast today at 4:30 pm ET to review its financial and operating results and to provide corporate and clinical updates.