– Building on statistically significant results from the CENTAUR phase 2 trial, PHOENIX will include 600 people with ALS and broader inclusion criteria
– The trial in Europe and the United States is expected to begin in the third quarter of 2021 to support regulatory submissions
Amylyx Pharmaceuticals, Inc., a pharmaceutical company specializing in the development of new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that it will present the design of its phase 3 study (A35-004 PHOENIX) of AMX0035 (sodium phenylbutyrate (PB) -taurursodiol (TURSO)) for the treatment of people with ALS at the 2021 meeting of the European Network to Cure ALS (ENCALS), to be held virtually May 12-14, 2021. The study will assess the safety and efficacy of AMX0035 in an international population of people with ALS based on the results of the CENTAUR trial.
“We are pleased to present the design of the Phase 3 trial at this year’s ENCALS meeting,” said Leonard H. van den Berg, MD, Ph.D., professor of neurology at UMC Utrecht in Pays -Bas and Chairman of the Treatment Research Initiative at Cure ALS (TRICALS), a large European trial network dedicated to finding a treatment for ALS. “Based on data from CENTAUR, we believe that AMX0035 may have the potential to provide much-needed hope for people with ALS. We are delighted to evaluate AMX0035 in patients in Europe and the United States through PHOENIX and we look forward to launching the trial in the coming months. “
“The PHOENIX trial builds on the success of the CENTAUR trial, which was designed and conducted at the US sites of the Northeast ALS Consortium (NEALS), a network of 140 trial-ready research centers primarily based in North America, ”said Sabrina Paganoni, MD, Ph.D., principal investigator of the CENTAUR study, investigator at the Healey & AMG Center for ALS in Mass General and member of the NEALS Executive Committee. “I strongly believe in a global collaboration and PHOENIX will be the first trial to include a global partnership between TRICALS and NEALS. The PHOENIX study design is a true testament to this collaboration, and we look forward to advancing this research and what it could mean for those living with ALS. “
Title: “Design of the PHOENIX Phase 3 International, Randomized, Placebo-Controlled Trial of AMX0035 in Amyotrophic Lateral Sclerosis”
Appointment: May 12 to 14
Time: 9:43 a.m. to 10:28 a.m. ET on May 12 and 13; 9:51 a.m. to 10:29 a.m. ET on May 14
As previously reported, the CENTAUR study met its primary efficacy endpoint of slowing ALS, as measured by the revised ALS Functional Rating Scale (ALSFRS-R). Adverse event rates were similar between the AMX0035 and placebo arms of the study. However, discontinuations due to adverse events occurred more frequently in the AMX0035 arm than in the placebo arm. In a nearly three-year overall survival analysis of all CENTAUR participants, those who started on AMX0035 had a 44% lower risk of death compared to those who started on placebo. The median survival time was 25.0 months (95% CI, 19.0 to 33.6 months) in the group started on AMX0035 and 18.5 months (95% CI, 13.5 to 23 , 2 months) in the group that started on placebo, i.e. a difference of 6.5 months.
The primary efficacy outcome of the 48-week, placebo-controlled, randomized phase 3 PHOENIX trial will be a joint assessment of progression of the ALSFRS-R total score over 48 weeks and survival.
Secondary efficacy endpoints will include change in slow vital capacity (SVC), measured both at home using a self-administered spirometer, to support virtual data collection, and at clinical sites. ; serial evaluations of patient-reported outcomes; ventilation-free survival rate and others.
The Phase 3 trial is expected to be conducted at approximately 55 TRICALS and NEALS sites in Europe and the U.S. It is expected to enroll approximately 600 participants with definitive or clinically probable ALS and within 24 months of symptom onset. which expands CENTAUR’s inclusion criteria.
AMX0035 will be made available to all participants who complete the 48-week PHOENIX study according to regulatory guidelines for each region.
“We hope that the conclusions of PHOENIX will build on the convincing results of CENTAUR,” said Joshua Cohen, co-CEO, president and co-founder of Amylyx. “We look forward to continued collaboration with study researchers in Europe and the United States, advocacy groups and the ALS community as we seek to advance AMX0035 through the process of clinical development and regulatory review. Justin Klee, Co-CEO and Co-Founder of Amylyx added, “For people with ALS every moment counts, and we will continue to work with regulatory agencies to ensure we meet requirements in the most appropriate way. fast possible. “
Amylyx recently announced its intention to submit a Marketing Authorization Application (MAA) for AMX0035 to the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) by the end of 2021. The company continues to discuss AMX0035 with health authorities around the world and will keep the global ALS community updated.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working to develop a new treatment for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.
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