An email has been sent to your address with instructions for changing your password.
There is no user registered with this email.
Sign Up
To create a free account, please fill out the form below.
Thank you for signing up!
A confirmation email has been sent to your email address. Please check your email and follow the instructions in the message to complete the registration process. If you do not receive the email, please check your spam folder or contact us for assistance.
Welcome to our platform!
Oops!
Something went wrong while trying to create your new account. Please try again and if the problem persist, Email Us to receive support.
Amylyx Pharmaceuticals, Inc. announces a virtual webcast to discuss interim data from the Phase 2 trial of AMX0035 for Wolfram syndrome treatment. Dr. Fumihiko Urano to present findings on April 10, 2024.
Positive
None.
Negative
None.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the Company will host a virtual webcast with management and Fumihiko Urano, MD, PhD, Principal Investigator of the HELIOS clinical trial and the Samuel E. Schechter Professor of Medicine in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine in St. Louis to discuss interim data from HELIOS, a Phase 2 trial of AMX0035 for the treatment of Wolfram syndrome on April 10, 2024 at 1:30pm ET.
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 serves as a Professor of Medicine and of Pathology & Immunology and is the Samuel E. Schechter Professor of Medicine at Washington University School of Medicine in St. Louis. 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.
About the HELIOS Trial
The HELIOS trial (NCT05676034) is a 12-participant, open-label, proof of biology, Phase 2 trial designed to study the effect of AMX0035 on safety and tolerability, and various measures of endocrinological, neurological, and ophthalmologic function in adult participants living with Wolfram syndrome.
About Wolfram Syndrome
Wolfram syndrome is an autosomal recessive neurodegenerative disease characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration. Common manifestations of Wolfram syndrome include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorineural deafness, neurogenic bladder, and progressive neurologic difficulties. Genetic and experimental evidence suggests that endoplasmic reticulum (ER) dysfunction is a critical pathogenic component of Wolfram syndrome. The prognosis of Wolfram syndrome is poor, and many people with the disease die prematurely with severe neurological disabilities.
About AMX0035
AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the U.S.). AMX0035 was designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum (ER) stress and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration. We believe that our proprietary combination of PB and TURSO and their complementary mechanisms of action will allow us to synergistically target abnormal cell death to better prevent neurodegeneration than treatment targeted at either mechanism of action alone. AMX0035 is being studied as a potential treatment for Wolfram syndrome and progressive supranuclear palsy, two neurodegenerative diseases.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative disease community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts. For more information, visit amylyx.com and follow us on LinkedIn and X, (formerly Twitter). For investors, please visit investors.amylyx.com.
Investors
Lindsey Allen
Amylyx Pharmaceuticals, Inc.
+1 (857) 320-6244
Investors@amylyx.com
Source: Amylyx Pharmaceuticals, Inc.
The virtual webcast is scheduled for April 10, 2024.
Dr. Fumihiko Urano is the Principal Investigator of the HELIOS clinical trial.
The ticker symbol of Amylyx Pharmaceuticals, Inc. is AMLX.
The live webcast can be accessed under 'Events and Presentations' in the Investor section of the Company's website.
Dr. Fumihiko Urano is a physician and medical researcher specializing in Wolfram syndrome. He is a driving force in the study of Wolfram syndrome and related disorders.
Amylyx Pharmaceuticals, Inc is a biopharmaceutical company headquartered in Cambridge, Massachusetts. Amylyx is best known for AMX0035, an experimental therapy for amyotrophic lateral sclerosis.
