Frontotemporal dementia

AcuraStem Enters into a License Agreement with Takeda to Advance PIKFYVE Therapeutics

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Monday, September 25, 2023
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MONROVIA, Calif., Sept. 25, 2023 /PRNewswire/ -- AcuraStem, a patient-based biotechnology company pioneering how treatments are developed for neurodegenerative diseases, announced today that it has entered into a license agreement with Takeda to develop and commercialize AcuraStem's PIKFYVE targeted therapeutics including AS-202, an innovative antisense oligonucleotide (ASO) for the treatment of Amyotrophic Lateral Sclerosis (ALS).

Key Points: 
  • AcuraStem's therapeutic strategy for PIKFYVE focuses on addressing neurodegeneration by expelling toxic protein aggregates and protecting healthy neuronal function.
  • Under the terms of the agreement, Takeda will receive an exclusive, worldwide license to AcuraStem's PIKFYVE program.
  • "I'm very pleased that AcuraStem is partnering with Takeda around their PIKFYVE program," said Justin Ichida, PhD.
  • Destum Partners acted as transaction advisor to AcuraStem, and Foley Hoag acted as legal counsel to AcuraStem.

AIRAmed Receives FDA 510(k) Clearance for Its AIRAscore Brain Volumetry Software

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Tuesday, September 19, 2023
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German medical technology company AIRAmed has announced U.S. Food and Drug Administration 510(k) clearance of AIRAscore, a medical image management and processing system providing relevant brain volumetry data to assist physicians in early detection of Alzheimer’s and other dementias.

Key Points: 
  • German medical technology company AIRAmed has announced U.S. Food and Drug Administration 510(k) clearance of AIRAscore, a medical image management and processing system providing relevant brain volumetry data to assist physicians in early detection of Alzheimer’s and other dementias.
  • AIRAscore uses deep learning and artificial intelligence (AI) technology to provide quantitative assessment of brain volume with objectively verifiable data using standard MRI brain scans in as little as five minutes.
  • “With AIRAscore, we are now offering physicians a highly precise, quantitative tool for the rapid detection of areas with a brain volume below the normal range.
  • The software is extremely affordable and can be configured by a hospital’s or radiology facility’s IT department in less than an hour.

Therapeutic Solutions International Reports Landmark Advancement in Stem Cell Therapy of Frontotemporal Dementia

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Monday, September 11, 2023
Science, Neurology, Stem Cells, Biotechnology, Research, Pharmaceutical, General Health, Health, Frontotemporal dementia, TSOI, Campbell, Dementia, Patent, Patient, Immune system, Nervous system, B cell, FDA, Memory, Pharmaceutical industry

Therapeutic Solutions International, Inc. (TSOI), announced today the development of a new therapeutic approach for treatment of Frontotemporal Dementia, by combining the activation of endogenous anti-inflammatory cells in the nervous system, together with regenerative cell intervention in the form of stem cell therapy, and molecules capable of activating endogenous stem cells already present in the patient.

Key Points: 
  • Therapeutic Solutions International, Inc. (TSOI), announced today the development of a new therapeutic approach for treatment of Frontotemporal Dementia, by combining the activation of endogenous anti-inflammatory cells in the nervous system, together with regenerative cell intervention in the form of stem cell therapy, and molecules capable of activating endogenous stem cells already present in the patient.
  • The therapy, for which a patent application has been filed, has demonstrated positive preliminary data in an animal model of the condition.
  • The Company plans to make the therapy available to “no option” patients with this condition through the Veltmeyer Institute under the “Right to Try Law”.
  • “Frontotemporal Dementia is a terrifying condition with no disease modifying treatments available.

QurAlis to Present Data Showing Link Between TDP-43 Pathology and Role of UNC13A in Neuronal Biology Related to ALS and Other Neurodegenerative Diseases

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Wednesday, September 6, 2023
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CAMBRIDGE, Mass., Sept. 6, 2023 /PRNewswire/ -- QurAlis Corporation, a clinical-stage biotechnology company developing breakthrough precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets, today announced it will present preclinical data showing that TDP-43 (TAR DNA-binding protein 43) pathology drives loss of synaptic UNC13A function in neurodegenerative diseases including ALS and frontotemporal dementia (FTD). Data also showed that an UNC13A splice-switching antisense oligonucleotide (ASO) prevented cryptic exon inclusion in UNC13A transcripts, increased UNC13A protein levels, and normalized localization of UNC13A protein at the synapse.

Key Points: 
  • Data also showed that an UNC13A splice-switching antisense oligonucleotide (ASO) prevented cryptic exon inclusion in UNC13A transcripts, increased UNC13A protein levels, and normalized localization of UNC13A protein at the synapse.
  • "UNC13A is a genetically validated target in ALS and FTD and our results showed that TDP-43 pathology drives loss of synaptic UNC13A function.
  • "We recently launched our newest program that targets UNC13A mis-splicing and look forward to advancing this program along with our other programs targeting neurodegenerative diseases so that we can make a real difference in patients' lives."
  • QurAlis will present these data in a poster presentation at the 1st Biennial Conference on TDP-43 Function and Dysfunction in Disease in Trieste, Italy on Thursday, September 7, 2023.

Athira Pharma Reports Second Quarter 2023 Financial Results and Pipeline and Business Updates

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Thursday, August 10, 2023
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BOTHELL, Wash., Aug. 10, 2023 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today reported financial results for the quarter ended June 30, 2023, and provided pipeline and business updates.

Key Points: 
  • Based on FDA interactions, the Company believes that all registrational pathways remain viable and contingent on LIFT-AD results.
  • The Company appointed seasoned finance and corporate strategy executive Andrew Gengos as Chief Financial Officer and Chief Business Officer.
  • R&D expenses were $21.6 million for the quarter ended June 30, 2023, compared with $14.8 million for the quarter ended June 30, 2022.
  • G&A expenses were $10.0 million for the quarter ended June 30, 2023, compared with $8.8 million for the quarter ended June 30, 2022.

Oligomerix Publishes Preclinical Data on Novel Small Molecule Therapeutic in Development for Alzheimer’s Disease and Rare Neurodegenerative Diseases

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Wednesday, August 9, 2023
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Earlier this year, Oligomerix announced first-in-human dosing of OLX-07010 in a Phase 1a clinical trial.

Key Points: 
  • Earlier this year, Oligomerix announced first-in-human dosing of OLX-07010 in a Phase 1a clinical trial.
  • The self-association of tau protein into progressively larger aggregates and tau tangles is a common pathological process in AD and multiple rare inherited neurodegenerative diseases such as PSP and FTD.
  • Since tau tangles and amyloid plaques are hallmarks of AD, treating both could be more beneficial to patients than targeting a single pathology.
  • Compared to controls, oral treatment with the drug over four months significantly prevented the development of tau aggregates in this mouse model.

MITSUBISHI TANABE PHARMA CANADA ANNOUNCES COLLABORATION WITH THE CNDR ON FIRST CANADIAN REAL-WORLD EVIDENCE STUDY OF RADICAVA® IV (EDARAVONE) SURVIVAL BENEFITS IN ALS

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Wednesday, August 9, 2023
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"The objective of this study is to describe real-world survival effectiveness over a longer timeframe."

Key Points: 
  • "The objective of this study is to describe real-world survival effectiveness over a longer timeframe."
  • "Access to innovative therapies is an urgent issue for people living with ALS," said Tammy Moore, President, ALS Canada.
  • The retrospective cohort study includes patients with ALS across Canada with a minimum six-month RADICAVA® IV exposure between 2017 and 2022.
  • The impact of edaravone on survival in patients with ALS has not been established and is the subject of the real-world evidence collaboration with CNDR.

Anavex Life Sciences Reports Fiscal 2023 Third Quarter Financial Results

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Tuesday, August 8, 2023
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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

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Monday, August 7, 2023
Frontotemporal dementia, Single, Central nervous system, Therapy, Disease, ECG, Rett syndrome, Doctor of Philosophy, CNS, Trial of the century, Alzheimer's disease, DSM-IV codes, Drug development, Pharmacology, Safety, Treatment, Pharmaceutical industry, Schizophrenia

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.

Passage Bio Reports Second Quarter 2023 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Monday, August 7, 2023
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“We continued to make steady progress advancing our lead clinical programs in the second quarter and further improved our financial strength, extending our cash runway into the fourth quarter of 2025.

Key Points: 
  • “We continued to make steady progress advancing our lead clinical programs in the second quarter and further improved our financial strength, extending our cash runway into the fourth quarter of 2025.
  • Additionally, we are thrilled to have already dosed the first patient at Dose 3, demonstrating effective execution across the program.
  • In FTD, we are excited by emerging data from initial treated patients, which indicates translation from preclinical models into the clinic.
  • General and Administrative (G&A) Expenses: G&A expenses were $8.1 million for the quarter ended June 30, 2023, as compared to $13.0 million as of June 30, 2022.