Endoplasmic reticulum

Amylyx Pharmaceuticals Reports First Quarter 2024 Financial Results

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Donnerstag, Mai 9, 2024
Research, FDA, Neurology, Clinical Trials, Biotechnology, Health, Pharmaceutical, Other Science, Science, Investment, Webcast, IND, WFS1, TDP43, AMX0035, Diabetes, Samuel, CSF, Biomarker, Astellas Pharma, Disclosure, Brain, Food, Amylyx Pharmaceuticals, Endoplasmic reticulum, Health Canada, Tauopathy, ALS, Eli Lilly and Company, FCCP, Medication, Merck, Cerebrospinal fluid, Safety, PB, Neurological disorder, Genetic linkage, Week, Wolfram syndrome, C-peptide, DSM-IV codes, Drug development, Clinical trial, Walking, Division, MD, Patient, Disease, Regulation Fair Disclosure, Principal, Regulation, PGIC, Metabolism, Interim, FACP, Pharmaceutical industry

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the first quarter ended March 31, 2024.

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the first quarter ended March 31, 2024.
  • This decision was informed by topline PHOENIX trial results, engagement with regulatory authorities, and discussions with the ALS community.
  • Amylyx anticipates reporting topline data from all 12 participants at Week 24 in the fall of 2024.
  • Amylyx’ management team will host a conference call and webcast today, May 9, 2024, at 8:00 a.m.

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

Retrieved on: 
Donnerstag, April 11, 2024
Autophagy, Endoplasmic reticulum, Calreticulin, Mouse, Cell, Neck, HMGB1, Apoptosis, Research, ATP, Immunogenic cell death, Chung, Head, Vaccine

Titled PV-10 triggers immunogenic cell death and anti-tumor immunity in head and neck squamous cell carcinoma via endoplasmic reticulum stress and autophagy, a copy of Moffitt’s poster presentation is available on Provectus’s website at: https://www.provectusbio.com/media/docs/publications/AACR-poster-3-Apr-2... .

Key Points: 
  • Titled PV-10 triggers immunogenic cell death and anti-tumor immunity in head and neck squamous cell carcinoma via endoplasmic reticulum stress and autophagy, a copy of Moffitt’s poster presentation is available on Provectus’s website at: https://www.provectusbio.com/media/docs/publications/AACR-poster-3-Apr-2... .
  • Moffitt observed that PV-10 induced reactive oxygen species-mediated apoptosis, surface expression of calreticulin, and extracellular ATP and HMGB1 release in HNSCC cells.
  • PV-10-induced immunogenic cell death (“ICD”) led to surface expression of HSP-70 and HSP-90 in these cells.
  • Intralesional (“IL”) injection of PV-10 promoted tumor regression in mice by inducing endoplasmic reticulum stress, triggering autophagy, and initiating apoptosis.

Amylyx Pharmaceuticals Announces Interim Data From Ongoing Phase 2 HELIOS Clinical Trial Demonstrating Improvements in Pancreatic Function and Glycemic Control with AMX0035 in People with Wolfram Syndrome

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Mittwoch, April 10, 2024
Health, Diabetes, Neurology, Genetics, Clinical Trials, Pharmaceutical, FDA, DSM-IV codes, Glycated hemoglobin, Cell, Safety, PB, Blood, Apoptosis, Paratriathlon, C-peptide, Endoplasmic reticulum, Doctor of Philosophy, WFS1, Disease, AMX0035, Week, Wolfram syndrome, Science, Snellen, Patient, Diarrhea, Amylyx Pharmaceuticals, Data analysis, PGIC, Samuel, Metabolism, Aes, Visual acuity, Clinical trial, AUC, Medication, Division, Pharmaceutical industry

“I have been studying Wolfram syndrome and caring for people with the disease for more than 20 years.

Key Points: 
  • “I have been studying Wolfram syndrome and caring for people with the disease for more than 20 years.
  • Outcomes for people with Wolfram syndrome consistently worsen over time, so disease stabilization alone is clinically meaningful for both patients and their doctors.
  • Because of the clear link between WFS1 mutations and ER stress, Wolfram syndrome is considered a prototypical ER stress disorder.
  • Amylyx announced that the FDA granted orphan drug designation to AMX0035 for the treatment of Wolfram syndrome in November 2020.

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

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Donnerstag, 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: 
Donnerstag, März 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: 
Dienstag, März 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

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Freitag, März 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: 
Samstag, 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

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Donnerstag, Oktober 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: 
Dienstag, 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.