Wolfram syndrome

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 to Host Virtual Webcast to Discuss Interim Data from Phase 2 HELIOS Study of AMX0035 in Wolfram Syndrome on April 10, 2024

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星期一, 四月 8, 2024
Research, Neurology, Diabetes, Clinical Trials, Biotechnology, Health, Pharmaceutical, Optical, Science, Wolfram syndrome, Medication, Metabolism, Patient, AMX0035, Samuel, Immunology, Doctor of Philosophy, MD, Trial of the century, Amylyx Pharmaceuticals, Division, Disorder, Pathology, Pharmaceutical industry

A live webcast of the presentation can be accessed under “Events and Presentations” in the Investor section of the Company’s website, https://investors.amylyx.com/news-events/events , and will be available for replay for 90 days following the event.

Key Points: 
  • A live webcast of the presentation can be accessed under “Events and Presentations” in the Investor section of the Company’s website, https://investors.amylyx.com/news-events/events , and will be available for replay for 90 days following the event.
  • Dr. Fumihiko Urano is a physician and medical researcher specializing in Wolfram syndrome.
  • Dr. Urano is a driving force in the study of Wolfram syndrome and related disorders, including WFS1-related disorders/Wolfram-like disorders.
  • As director of the Wolfram Syndrome Clinic and the Wolfram Syndrome International Registry & Clinical Study at Washington University, Dr. Urano treats patients with Wolfram syndrome and related disorders, and leads basic science, clinical, translational, and interventional studies of Wolfram syndrome and related disorders.

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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星期四, 四月 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.

Amylyx Pharmaceuticals Appoints Bernhardt Zeiher, MD, FCCP, FACP, to its Board of Directors

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星期四, 三月 14, 2024
Biotechnology, Pharmaceutical, Health, Neurology, Medication, Astellas Pharma, Pfizer, ALS, Science, Wolfram syndrome, University, FACP, AMX0035, Research, Doctor of Philosophy, MD, Biology, Critical care, Palsy, Drug development, FCCP, Amylyx Pharmaceuticals, Pharmacovigilance, Eli Lilly and Company, CMO, Wolfram, Biotechnology Innovation Organization, Neurodegenerative disease, Medicine, Pharmaceutical industry

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the addition of Bernhardt G. Zeiher, MD, FCCP, FACP, to its Board of Directors (“Board”).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the addition of Bernhardt G. Zeiher, MD, FCCP, FACP, to its Board of Directors (“Board”).
  • Most recently, he served as Chief Medical Officer (“CMO”) of Astellas Pharma, where he led early- and late-stage drug development, medical and regulatory affairs, pharmacovigilance, and quality assurance.
  • “Dr.
  • Zeiher brings to the Board his distinguished drug development background in overseeing the approval of 15 medicines, and his first-hand experience as a physician.

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

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星期五, 三月 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.

Amylyx Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results

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星期四, 二月 22, 2024
Biotechnology, Neurology, Health, Pharmaceutical, Clinical Trials, Week, Regulation, Research, Investment, University, Regulation Fair Disclosure, Webcast, CRP, Intellectual property, Progressive supranuclear palsy, Medication, Plasma, ALS, Marketing, Disclosure, AMX0035, Neurosurgery, Publication, Psychiatry, C-reactive protein, Safety, Biomarker, Amylyx Pharmaceuticals, Clinical trial, Survival, Journal of Neurology, Mutation, IND, Wolfram syndrome, PSP, Otsuka Pharmaceutical, Brain cell, Pharmaceutical industry

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the fourth quarter and full year ended December 31, 2023.

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the fourth quarter and full year ended December 31, 2023.
  • Net product revenue for the three months ended December 31, 2023 was $108.4 million, compared to net product revenue of $102.7 million for the three months ended September 30, 2023.
  • Amylyx announced the first patient dosed in the Global Phase 3 ORION Study of AMX0035 in Progressive Supranuclear Palsy (PSP).
  • The first participant was dosed in December 2023, and topline results are anticipated in 2025 or 2026.

Amylyx Pharmaceuticals Announces Completion of Enrollment in HELIOS, a Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome

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星期四, 二月 8, 2024
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, AMX0035, Death, FDA, Medication, Trial of the century, Amylyx Pharmaceuticals, Safety, Caregiver, Division, Clinical trial, Wolfram syndrome, PB, Person, Metabolism, Disease, Doctor of Philosophy, DSM-IV codes, Pharmaceutical industry

The trial has enrolled 12 adult participants living with WS, and preliminary results are anticipated in the second half of 2024.

Key Points: 
  • The trial has enrolled 12 adult participants living with WS, and preliminary results are anticipated in the second half of 2024.
  • Data from this initial study will inform potential future Amylyx trials for individuals with WS.
  • The data explored the potential of AMX0035 as a novel therapeutic approach for WS and provided initial proof-of-concept for its therapeutic development in WS.
  • “These preclinical findings suggest the possibilities of AMX0035 for individuals with WS that we continue to explore, including in the HELIOS trial.

Amylyx Pharmaceuticals Reports Second Quarter 2023 Financial Results

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星期四, 八月 10, 2023
Biotechnology, General Health, Other Health, Health, Pharmaceutical, Disease, Regulation Fair Disclosure, Ludwig Maximilian University of Munich, Disclosure, European Directive on Traditional Herbal Medicinal Products, Pan-Canadian Pharmaceutical Alliance, Brain, Research, Department of Neurology, Xuanwu hospital, Wolfram syndrome, Medication, EMA, Committee, Walking, Investment, Committee for Medicinal Products for Human Use, PEGASUS, Webcast, Neurology, AMX0035, Neurological disorder, ORION, MAA, Marketing Authorisation Application, European Medicines Agency, Palsy, ALS, Regulation, PSP, CHMP, Cryptocurrency, Pharmaceutical industry, Amylyx Pharmaceuticals, Wolfram

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the second quarter ended June 30, 2023.

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the second quarter ended June 30, 2023.
  • Net product revenue for the three months ended June 30, 2023 was $98.2 million, compared to net product revenue of $71.4 million for the three months ended March 31, 2023.
  • Financial Results for the Second Quarter Ended June 30, 2023
    For the three months ended June 30, 2023, net product revenue was $98.2 million, and cost of sales were $5.6 million.
  • Cash, cash equivalents, and short-term investments were $357.3 million at June 30, 2023, compared to $345.7 million at March 31, 2023.

Amylyx Pharmaceuticals Reports First Quarter 2023 Financial Results

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星期四, 五月 11, 2023
Biotechnology, General Health, Neurology, Health, Pharmaceutical, Disease, National Defense Authorization Act, Regulation Fair Disclosure, European Medicines Agency, Policy, Disclosure, European Directive on Traditional Herbal Medicinal Products, Brain, Research, Marketing, Wolfram syndrome, Medication, Committee, EMA, Globalization, Walking, Investment, Physician, PEGASUS, Committee for Medicinal Products for Human Use, Webcast, AMX0035, Growth, Neurological disorder, MAA, Palsy, ALS, Regulation, PSP, Safety, CHMP, Pharmaceutical industry, Cryptocurrency, PHOENIX, Amylyx Pharmaceuticals, Wolfram

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the first quarter ended March 31, 2023.

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the first quarter ended March 31, 2023.
  • The Company continues to expect an opinion from the CHMP mid-year and a decision in the third quarter of 2023 at the earliest.
  • Financial Results for the First Quarter Ended March 31, 2023
    For the three months ended March 31, 2023, net product revenue was $71.4 million and cost of sales were $5.3 million.
  • Amylyx’ management team will host a conference call and webcast today, May 11, 2023, at 4:30 p.m.