WFS1

Amylyx Pharmaceuticals Reports First Quarter 2024 Financial Results

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星期四, 五月 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.

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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星期三, 四月 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 First Participant Dosed in Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome

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星期四, 四月 13, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, PB, Cell death, Mitophagy, Diabetes, Metabolism, Doctor of Philosophy, Person, Wolfram syndrome, Trial of the century, Medication, Endoplasmic reticulum, MD, Pathology, Division, Washington University School of Medicine, Apoptosis, WS, AMX0035, Immunology, FDA, Phenotype, WFS1, Disease, Safety, Pharmaceutical industry, Vaccine, Medicine, Amylyx Pharmaceuticals, Endocrinology, Wolfram

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the first participant has been dosed in the HELIOS study, a Phase 2 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the first participant has been dosed in the HELIOS study, a Phase 2 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS).
  • HELIOS is an exploratory open-label proof of biology study assessing the effect of AMX0035 safety and tolerability, and various measures of endocrinological, neurological and ophthalmologic function.
  • Gene enrichment analysis revealed that treatment with AMX0035 ameliorated organelle dysfunction, mitophagy, endoplasmic reticulum (ER) stress, and apoptosis.
  • Furthermore, AMX0035 delayed the onset of the diabetic phenotype in vivo in the Wfs1-knockout mouse model of Wolfram syndrome.