- Building on Statistically Significant Findings from Phase 2 CENTAUR Trial, PHOENIX to Include 600 People with ALS and Broader Inclusion Criteria
- Trial in Europe and U.S. Expected to Begin in Q3 2021 to Support Regulatory Submissions
Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced it will present the trial 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 European Network to Cure ALS (ENCALS) Meeting 2021, 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 that will build upon findings from the CENTAUR trial.
"We are pleased to present the Phase 3 trial design at this year’s ENCALS meeting," said Leonard H. van den Berg, M.D., Ph.D., Professor of Neurology at UMC Utrecht in the Netherlands and Chairman of the Treatment Research Initiative to 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 to people living with ALS. We are excited to evaluate AMX0035 in patients throughout Europe and the U.S. through PHOENIX and look forward to initiating the trial in the coming months."
"The PHOENIX trial builds on the success of the CENTAUR trial, which was designed and conducted at U.S. sites of Northeast ALS Consortium (NEALS), a network of 140 trial-ready research centers primarily based in North America," said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR study, investigator at the Healey & AMG Center for ALS at Mass General and member of the Executive Committee of NEALS. "I strongly believe in global collaboration and PHOENIX will be the first trial to include a global partnership between TRICALS and NEALS. The study design of PHOENIX is a true testament to this collaboration, and we look forward to advancing this research and what it might mean for those living with ALS."
Title: "Design of the International, Randomized, Placebo-Controlled Phase 3 PHOENIX Trial of AMX0035 in Amyotrophic Lateral Sclerosis"
Dates: May 12 – 14
Times: 9:43 a.m. – 10:28 a.m. ET on May 12 and 13; 9:51 a.m. – 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 ALS Functional Rating Scale-Revised (ALSFRS-R). The rates of adverse events were similar between the AMX0035 and placebo arms of the study. However, discontinuations related 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 participants from CENTAUR, those who started on AMX0035 had a 44% lower risk of death compared to those who started on placebo. Median survival duration was 25.0 months (95% CI, 19.0-33.6 months) in the group that started on AMX0035 and 18.5 months (95% CI, 13.5-23.2 months) in the group that started on placebo, a 6.5-month difference.
The primary efficacy outcome of the 48-week, randomized placebo-controlled PHOENIX Phase 3 trial will be a joint assessment of ALSFRS-R total score progression over 48 weeks and survival.
Secondary efficacy outcomes will include change in slow vital capacity (SVC), measured both at home using a self-administered spirometer, to support virtual data collection, and at clinic sites; serial assessments of patient-reported outcomes; ventilation-free survival rates and others.
The Phase 3 trial is planned 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 definite or clinically probable ALS and within 24 months from symptom onset which expands on CENTAUR inclusion criteria.
AMX0035 will be made available to all participants who complete the 48-week PHOENIX study in accordance with each region’s regulatory guidance.
"It is our hope that findings from PHOENIX will build upon compelling results from CENTAUR," said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. "We look forward to continued collaboration with study investigators in Europe and the U.S., advocacy groups, and the ALS community as we seek to advance AMX0035 through the clinical development and regulatory review process." Justin Klee, Co-CEO and Co-Founder of Amylyx added, "For people living with ALS, every moment matters, and we will continue working with regulatory agencies to ensure we meet requirements in the most expeditious way possible."
Amylyx recently announced plans to submit a Marketing Authorization Application (MAA) for AMX0035 to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) 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 on developing a novel therapeutic 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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