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Amylyx Pharmaceuticals to Host Virtual Webcast to Discuss Topline Results from Phase 2 HELIOS Study of AMX0035 in Wolfram Syndrome on October 17, 2024

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Amylyx Pharmaceuticals (Nasdaq: AMLX) has announced a virtual webcast on October 17, 2024 at 1:30 p.m. ET to discuss topline results from the HELIOS Phase 2 trial of AMX0035 for the investigational treatment of Wolfram syndrome. The webcast will feature management and Dr. Fumihiko Urano, the principal investigator of the HELIOS trial and a professor at Washington University School of Medicine in St. Louis.

The presentation follows the release of data at the International Society for Pediatric and Adolescent Diabetes 50th Annual Congress in Lisbon, Portugal. The live webcast will be accessible on the company's website and available for replay for 90 days. Dr. Urano, a leading expert in Wolfram syndrome, directs the Wolfram Syndrome Clinic and International Registry & Clinical Study at Washington University.

Amylyx Pharmaceuticals (Nasdaq: AMLX) ha annunciato un webcast virtuale il 17 ottobre 2024 alle 13:30 ET per discutere i risultati preliminari della fase 2 dello studio HELIOS di AMX0035 per il trattamento sperimentale della sindrome di Wolfram. Il webcast ospiterà il management e il Dr. Fumihiko Urano, investigatore principale dello studio HELIOS e professore presso la Washington University School of Medicine di St. Louis.

La presentazione segue il rilascio dei dati durante il 50° Congresso Annuale della Società Internazionale per il Diabete Pediatrico e Adolescenziale a Lisbona, in Portogallo. Il webcast dal vivo sarà accessibile sul sito web dell'azienda e disponibile per la visione per 90 giorni. Il Dr. Urano, un esperto di spicco nella sindrome di Wolfram, dirige la clinica e il registro internazionale per la sindrome di Wolfram presso la Washington University.

Amylyx Pharmaceuticals (Nasdaq: AMLX) ha anunciado un webcast virtual el 17 de octubre de 2024 a la 1:30 p.m. ET para discutir los resultados preliminares del ensayo HELIOS de fase 2 de AMX0035 para el tratamiento experimental del síndrome de Wolfram. El webcast contará con la participación de la dirección y del Dr. Fumihiko Urano, investigador principal del ensayo HELIOS y profesor en la Escuela de Medicina de la Universidad de Washington en St. Louis.

La presentación sigue a la publicación de datos en el 50º Congreso Anual de la Sociedad Internacional de Diabetes Pediátrica y Adolescentes en Lisboa, Portugal. El webcast en vivo estará disponible en el sitio web de la empresa y se podrá reproducir durante 90 días. El Dr. Urano, un experto de renombre en síndrome de Wolfram, dirige la Clínica del Síndrome de Wolfram y el Registro Internacional y Estudio Clínico en la Universidad de Washington.

Amylyx Pharmaceuticals (Nasdaq: AMLX)는 2024년 10월 17일 오후 1:30 ET에 AMX0035HELIOS 2상 시험의 주요 결과를 논의하기 위한 가상 웹캐스트를 발표했습니다. 웹캐스트에는 경영진과 HELIOS 시험의 주요 연구자인 Dr. Fumihiko Urano가 출연하며, 그는 세인트루이스에 있는 워싱턴 대학교 의과대학의 교수입니다.

프레젠테이션은 포르투갈 리스본에서 열린 소아 및 청소년 당뇨병 국제 학회 50주년 연례 회의에서 데이터가 발표된 후 이루어집니다. 이 라이브 웹캐스트는 회사 웹사이트에서 접근 가능하며 90일 동안 다시 볼 수 있습니다. Wolfram 증후군의 저명한 전문가인 Urano 박사는 워싱턴 대학교의 Wolfram 증후군 클리닉 및 국제 등록부 및 임상 연구를 이끌고 있습니다.

Amylyx Pharmaceuticals (Nasdaq: AMLX) a annoncé un webcast virtuel le 17 octobre 2024 à 13h30 ET pour discuter des résultats préliminaires de l'essai HELIOS de phase 2 d'AMX0035 pour le traitement expérimental du syndrome de Wolfram. Le webcast présentera la direction et le Dr Fumihiko Urano, l'investigateur principal de l'essai HELIOS et professeur à l'École de Médecine de l'Université de Washington à St. Louis.

La présentation suit la publication de données lors du 50e Congrès Annuel de la Société Internationale de Diabète Pédiatrique et Adolescent à Lisbonne, Portugal. Le webcast en direct sera accessible sur le site web de l'entreprise et disponible en rediffusion pendant 90 jours. Le Dr Urano, un expert de premier plan en syndrome de Wolfram, dirige la Clinique du Syndrome de Wolfram et le Registre International & Étude Clinique à l'Université de Washington.

Amylyx Pharmaceuticals (Nasdaq: AMLX) hat ein virtuelles Webcast für den 17. Oktober 2024 um 13:30 Uhr ET angekündigt, um die vorläufigen Ergebnisse der HELIOS Phase 2 Studie zu AMX0035 zur experimentellen Behandlung des Wolfram-Syndroms zu diskutieren. Das Webcast wird das Management und Dr. Fumihiko Urano, den Hauptuntersucher der HELIOS-Studie und Professor an der Washington University School of Medicine in St. Louis, präsentieren.

Die Präsentation folgt der Veröffentlichung von Daten beim 50. Jahrestagung der Internationalen Gesellschaft für Kinder- und Jugenddiabetes in Lissabon, Portugal. Das Live-Webcast wird auf der Website des Unternehmens zugänglich sein und für 90 Tage zur Verfügung stehen. Dr. Urano, ein führender Experte für das Wolfram-Syndrom, leitet die Wolfram-Syndrom-Klinik und das Internationale Register & Klinische Studie an der Washington University.

Positive
  • Amylyx is presenting topline results from a Phase 2 trial for a potential Wolfram syndrome treatment
  • The company has secured a leading expert, Dr. Fumihiko Urano, to discuss the trial results
  • The results are being presented at a major international diabetes congress
Negative
  • None.

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the Company will host a virtual webcast on October 17, 2024 at 1:30 p.m. ET 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 (WashU Medicine), to discuss topline results from HELIOS, a Phase 2 trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) for the investigational treatment of Wolfram syndrome. The live webcast follows the presentation of these data at the International Society for Pediatric and Adolescent Diabetes 50th Annual Congress in Lisbon, Portugal.

The live webcast 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 leading medical researcher at WashU Medicine in St. Louis specializing 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 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 is committed to the discovery and development of new treatment options for communities with high unmet needs, including people living with serious and fatal diseases. The Company has preclinical or clinical development programs underway in neurodegenerative, neuroendocrine, and endocrine diseases. Since its founding, Amylyx has been guided by science to address unanswered questions, keeping communities at the heart and center of all decisions. Amylyx is headquartered in Cambridge, Massachusetts. For more information, visit amylyx.com and follow us on LinkedIn and X. For investors, please visit investors.amylyx.com.

Media

Amylyx Media Team

+1 (857) 799-7274

amylyxmediateam@amylyx.com

Investors

Lindsey Allen

Amylyx Pharmaceuticals, Inc.

+1 (857) 320-6244

Investors@amylyx.com

Source: Amylyx Pharmaceuticals, Inc.

FAQ

What is the HELIOS Phase 2 trial testing for Wolfram syndrome?

The HELIOS Phase 2 trial is testing AMX0035, a combination of sodium phenylbutyrate and taurursodiol, as an investigational treatment for Wolfram syndrome.

When will Amylyx Pharmaceuticals (AMLX) host the webcast for HELIOS trial results?

Amylyx Pharmaceuticals will host the virtual webcast to discuss HELIOS trial results on October 17, 2024 at 1:30 p.m. ET.

Who is the principal investigator of the HELIOS trial for Amylyx's AMX0035?

Dr. Fumihiko Urano, the Samuel E. Schechter Professor of Medicine at Washington University School of Medicine in St. Louis, is the principal investigator of the HELIOS trial.

Where can investors access Amylyx Pharmaceuticals' (AMLX) webcast on HELIOS trial results?

Investors can access the live webcast under 'Events and Presentations' in the Investor section of Amylyx's website at https://investors.amylyx.com/news-events/events.

Amylyx Pharmaceuticals, Inc.

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