Endoplasmic reticulum

Amylyx Pharmaceuticals Announces Formal Intention to Remove RELYVRIO®/ALBRIOZA™ from the Market; Provides Updates on Access to Therapy, Pipeline, Corporate Restructuring, and Strategy

Retrieved on: 
Thursday, April 4, 2024
Health, FDA, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, Cell death, Medication, Survival, Wolfram syndrome, ALS, Partnership, Metabolism, Patient, AMX0035, Research, American Academy, OLE, Tauopathy, Health Canada, Biology, AAN, Clinical trial, Endoplasmic reticulum, Palsy, Workforce, Amylyx Pharmaceuticals, Division, Publication, Food, Vance DeGeneres, Pharmaceutical industry

Amylyx will continue to evaluate and share learnings from PHOENIX to help inform future ALS research.

Key Points: 
  • Amylyx will continue to evaluate and share learnings from PHOENIX to help inform future ALS research.
  • At this time, Amylyx intends to continue to collect available data on survival at the encouragement of ALS specialists.
  • “Our pipeline is supported by compelling clinical and preclinical science demonstrating the potential of AMX0035 and AMX0114 in neurodegenerative diseases.
  • “We are so thankful and grateful to our Amylyx team for their contributions and steadfast dedication,” said Cohen and Klee.

Provectus Biopharmaceuticals Announces Acceptance of Cancer Immunotherapy PV-10 Abstract for HNSCC at AACR 2024 Annual Meeting

Retrieved on: 
Thursday, March 7, 2024
Head, Neck, Chung, Calreticulin, Vaccine, Immunogenic cell death, Endoplasmic reticulum, ICD, HMGB1, Reactive oxygen species, ATP, Poster, Autophagy, Research, Mouse, Abstract

The abstract, which was accepted for a poster presentation, is titled “ PV 10 induces endoplasmic reticulum stress and autophagy, triggering immunogenic cell death and anti-tumor immunity in head and neck squamous cell carcinoma ” (Abstract #6742, Topic Track: Immunology, Session: Vaccines, Antigens, and Antigen Presentation 2).

Key Points: 
  • The abstract, which was accepted for a poster presentation, is titled “ PV 10 induces endoplasmic reticulum stress and autophagy, triggering immunogenic cell death and anti-tumor immunity in head and neck squamous cell carcinoma ” (Abstract #6742, Topic Track: Immunology, Session: Vaccines, Antigens, and Antigen Presentation 2).
  • According to Moffitt’s abstract, “…in vitro findings reveal that PV-10 induces cytotoxicity in both mEER and MTE-RAS cells.
  • Notably, PV-10 promotes a significant increase in [reactive oxygen species], leading to an elevation in late apoptotic cells.
  • At the molecular level, a remarkable activation of endoplasmic reticulum (ER) stress, pro-apoptotic protein, and autophagy markers were observed.

Gain Therapeutics Presents Data at the AD/PD™ 2024 Conference Demonstrating the Mechanism of Action of GT-02287, its Clinical Stage GCase Regulator for the Treatment of Parkinson’s Disease

Retrieved on: 
Tuesday, March 5, 2024
Alzheimer's disease, PD, Research, Treatment, Travel, Conference, Endoplasmic reticulum, Therapy, Poster, DSM-IV codes, Science, GBA1, Pharmaceutical industry

AD/PD™ 2024 is being held March 5th – 9th, 2024 in Lisbon, Portugal.

Key Points: 
  • AD/PD™ 2024 is being held March 5th – 9th, 2024 in Lisbon, Portugal.
  • GCase is consequently able to travel to the lysosome, resulting in enhanced lysosomal activity and efficient processing of the GCase substrate glucosylceramide.
  • Increase in GCase substrate in the lysosome was previously shown to be associated with accumulation of aggregated alpha-synuclein, a pathological hallmark of Parkinson’s disease and related disorders.
  • “These data further confirm our understanding of the mechanism of action of GT-02287, how it impacts cellular health by preventing the downstream consequences of GCase misfolding, including cellular stress and lysosomal dysfunction” commented Dr. Natalia Perez-Carmona, Senior Director of Biology at Gain Therapeutics and presenting author.

Amylyx Pharmaceuticals Announces Topline Results From Global Phase 3 PHOENIX Trial of AMX0035 in ALS

Retrieved on: 
Friday, March 8, 2024
Health, Neurology, Clinical Trials, General Health, Pharmaceutical, Biotechnology, International, Week, ALS, Wolfram syndrome, PSP, Physician, Edaravone, Palsy, Riluzole, Amylyx Pharmaceuticals, Safety, AMX0035, Webcast, Endoplasmic reticulum, PB, Medication, Trial of the century, Research, Pharmaceutical industry

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced topline results from PHOENIX, a global, 48-week, randomized, placebo-controlled Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]; RELYVRIO® in the U.S., ALBRIOZA™ in Canada) in people living with amyotrophic lateral sclerosis (ALS).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced topline results from PHOENIX, a global, 48-week, randomized, placebo-controlled Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]; RELYVRIO® in the U.S., ALBRIOZA™ in Canada) in people living with amyotrophic lateral sclerosis (ALS).
  • Amylyx intends to share plans for RELYVRIO/ALBRIOZA in ALS, which may include voluntarily withdrawing RELYVRIO/ALBRIOZA from the market.
  • “We are surprised and deeply disappointed by the PHOENIX results following the positive data from the CENTAUR trial.
  • Update on Ongoing AMX0035 Studies:
    The global, randomized, double-blind, placebo-controlled Phase 3 ORION clinical study of AMX0035 in PSP remains ongoing.

Promontory Therapeutics Presents Data on Molecular Mechanisms of PT-112's Immunogenic Effects

Retrieved on: 
Saturday, November 4, 2023
Society for Immunotherapy of Cancer, Mitochondrion, Immunogenic cell death, ICD, Bax, Northern blot, Doctor of Philosophy, Phosphorylation, Bcl-2, SITC, Cancer immunotherapy, Immunotherapy, Reactive oxygen species, ISR, Research and development, Cancer, Flow cytometry, Annual general meeting, Weill Cornell Medicine, Lung cancer, Endoplasmic reticulum, Society, Therapy, Patient, Cytosol, DNA, Vaccine, Platinum, Research

NEW YORK, Nov. 4, 2023 /PRNewswire/ -- Promontory Therapeutics Inc., a Phase 2 stage biotech company advancing immunogenic small molecule approaches in oncology, today presented data demonstrating the molecular mechanism of its lead therapeutic candidate, PT-112, and its ability to induce immunogenic cell death (ICD) in cancer cells. Data suggest that PT-112-induced ICD is mediated by endoplasmic reticulum (ER) and mitochondrial stresses, which are specific intra-cellular events that comprise part of the larger ICD mechanism. The presentation was made at the Society for Immunotherapy of Cancer's (SITC) 38th Annual Meeting, taking place November 1-5, 2023 in San Diego.

Key Points: 
  • Data suggest that PT-112-induced ICD is mediated by endoplasmic reticulum (ER) and mitochondrial stresses, which are specific intra-cellular events that comprise part of the larger ICD mechanism.
  • The presentation was made at the Society for Immunotherapy of Cancer's (SITC) 38th Annual Meeting, taking place November 1-5, 2023 in San Diego.
  • PT-112 effects on mitochondria included increases in mitochondrial mass and reactive oxygen species, changes in membrane polarization, and the release of mitochondrial DNA into the cytosol, a potent immunogenic signal.
  • For more information about Promontory Therapeutics and PT-112, visit www.PromontoryTx.com .

EQS-News: Defence Therapeutics Inc.: DEFENCE’S NOVEL ACCUTOXTM CONTINUES TO SURPRISE ON RESULTS AGAINST CANCER

Retrieved on: 
Thursday, October 26, 2023
FDA, IND, City of Hope, Nivolumab, Chitosan, Beckman Research Institute, Injection, News, Defense, CDMO, Nanoparticle, BMS, City, Lymphoma, Antibody, City of Hope National Medical Center, Animal, Molecular medicine, Melanoma, Cancer, Lung cancer, Therapy, Relatlimab, Endoplasmic reticulum, Breast, Patient, Peptide, DNA, Pharmaceutical industry, Vaccine

Vancouver, BC, Canada, October 17th, 2023 - Defence Therapeutics Inc. (“Defence” or the “Company”), a Canadian biopharmaceutical company specialized in the development of immune-oncology vaccines and drug delivery technologies, is pleased to announce that the excitement around AccuTOXTM against cancer continues to develop and expand its applications.

Key Points: 
  • Vancouver, BC, Canada, October 17th, 2023 - Defence Therapeutics Inc. (“Defence” or the “Company”), a Canadian biopharmaceutical company specialized in the development of immune-oncology vaccines and drug delivery technologies, is pleased to announce that the excitement around AccuTOXTM against cancer continues to develop and expand its applications.
  • The AccuTOXTM molecule can eradicate cancer cells via different mechanisms including the initiation of immunogenic cells death, endoplasmic reticulum stress and by causing direct damages to DNA.
  • AccuTOXTM triggers cancer tumor regression, Defence also recently demonstrated that encapsulated AccuTOXTM with chitosan nanoparticles triggers complete tumor regression in animals with pre-established solid lymphoma.
  • Further development and expanding applications on Defence’s leading AccuTOXTM therapeutic is ongoing and further news to be released upon results.

Akava Therapeutics, Inc. Announces FDA Clearance of Investigational New Drug Application for the Treatment of Amyotrophic Lateral Sclerosis

Retrieved on: 
Tuesday, August 22, 2023
Research, Neurology, FDA, Clinical Trials, Other Health, Biotechnology, Pharmaceutical, Health, Science, Other Science, Disease, Therapy, Atrophy, Mitochondrion, Neurodegenerative disease, Pharmacokinetics, Brain cell, Paralysis, Endoplasmic reticulum, Walking, Patient, Cancer, Axon, Diagnosis, Death, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, ALS, Food, Safety, Pharmaceutical industry

On July 3rd, the U.S. Food and Drug Administration cleared Akava’s Investigational New Drug (IND) application for the treatment of amyotrophic lateral sclerosis (ALS) to proceed with a Phase I first-in-human study in healthy subjects using Akava’s investigational new drug, AKV9 (formerly NU-9).

Key Points: 
  • On July 3rd, the U.S. Food and Drug Administration cleared Akava’s Investigational New Drug (IND) application for the treatment of amyotrophic lateral sclerosis (ALS) to proceed with a Phase I first-in-human study in healthy subjects using Akava’s investigational new drug, AKV9 (formerly NU-9).
  • The study will evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of AKV9.
  • This pivotal milestone reinforces the company’s commitment to advancing the treatment landscape for ALS and other related neurodegenerative disorders.
  • We are thrilled to take this important step forward in the development of a groundbreaking therapy for ALS patients.

Kanazawa University research: Researchers define a protocol for narrow cantilever fabrication and high-resolution imaging of living cells using AFM

Retrieved on: 
Wednesday, August 9, 2023
Mitochondrion, STAR, Fluorescence, 3D, AC, Cell, Adhesive, 2D, Endoplasmic reticulum, Golgi apparatus, Observation, Lysosome, AFM, Kanazawa University, Microscope, Kotaro Fukuma, Golgi

KANAZAWA, Japan, Aug. 9, 2023 /PRNewswire/ -- Researchers at Kanazawa University report in STAR Protocols procedural details and tips for nanoendoscopy-AFM, for capturing images of nanoscale structures inside living cells.

Key Points: 
  • KANAZAWA, Japan, Aug. 9, 2023 /PRNewswire/ -- Researchers at Kanazawa University report in STAR Protocols procedural details and tips for nanoendoscopy-AFM, for capturing images of nanoscale structures inside living cells.
  • Images of nanoscale structures inside living cells are in increasing demand for insights into cellular structure and function they can reveal.
  • So far, the tools for capturing such images have been limited in various ways, but researchers led by Takeshi Fukuma and Takehiko Ichikawa at Kanazawa University have now devised and reported a full protocol for using atomic force microscopy (AFM) to image inside living cells.
  • Protocol for live imaging of intracellular nanoscale structures using atomic force microscopy with nanoneedle probes, STAR Protocols September 15, 2023.

Kanazawa University research: Researchers define a protocol for narrow cantilever fabrication and high-resolution imaging of living cells using AFM

Retrieved on: 
Wednesday, August 9, 2023
Mitochondrion, STAR, Fluorescence, 3D, AC, Cell, Adhesive, 2D, Endoplasmic reticulum, Golgi apparatus, Observation, Lysosome, AFM, Kanazawa University, Microscope, Kotaro Fukuma, Golgi

KANAZAWA, Japan, Aug. 9, 2023 /PRNewswire/ -- Researchers at Kanazawa University report in STAR Protocols procedural details and tips for nanoendoscopy-AFM, for capturing images of nanoscale structures inside living cells.

Key Points: 
  • KANAZAWA, Japan, Aug. 9, 2023 /PRNewswire/ -- Researchers at Kanazawa University report in STAR Protocols procedural details and tips for nanoendoscopy-AFM, for capturing images of nanoscale structures inside living cells.
  • Images of nanoscale structures inside living cells are in increasing demand for insights into cellular structure and function they can reveal.
  • So far, the tools for capturing such images have been limited in various ways, but researchers led by Takeshi Fukuma and Takehiko Ichikawa at Kanazawa University have now devised and reported a full protocol for using atomic force microscopy (AFM) to image inside living cells.
  • Protocol for live imaging of intracellular nanoscale structures using atomic force microscopy with nanoneedle probes, STAR Protocols September 15, 2023.

Imvax Announces Publication in Journal for ImmunoTherapy of Cancer Showing IGV-001 Can Induce Clinically Relevant Anti-Cancer Immune Responses in Glioblastoma via Immunogenic Cell Death

Retrieved on: 
Tuesday, August 8, 2023
Research, Publishing, Communications, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Oncology, Radiation, Society, Immunogenic cell death, GBM, Progression-free survival, E pluribus unum, Neoplasm, OS, SITC, Immunotherapy, SOC, Radiation therapy, PFS, Glioblastoma, Assistant, Society for Immunotherapy of Cancer, Cell biology, Patient, Weill Cornell Medicine, Endoplasmic reticulum, Vaccine, Medical imaging, Cancer, Journal

Imvax, Inc. , a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies, today announced the publication of preclinical research with IGV-001 in Journal for ImmunoTherapy of Cancer, the official journal of the Society for Immunotherapy of Cancer (SITC).

Key Points: 
  • Imvax, Inc. , a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies, today announced the publication of preclinical research with IGV-001 in Journal for ImmunoTherapy of Cancer, the official journal of the Society for Immunotherapy of Cancer (SITC).
  • The article — “A biologic-device combination product delivering tumor-derived antigens elicits immunogenic cell death-associated immune responses against glioblastoma” — is available online .
  • Imvax’s Goldspire immuno-oncology platform for solid tumors involves a unique approach to inducing a broad and durable immune response against tumors.
  • In March 2023, Imvax initiated a Phase 2b clinical trial of IGV-001 in ndGBM patients ( NCT04485949 ).