Top-Line Data of Phase II/III HEALEY ALS Platform Trial Shows Improvements in Function and Mortality
Despite improvements, results from the study of SLS-005 (IV trehalose), a low molecular weight disaccharide, did not meet statistical significance.
Top-line data from the Phase II/III HEALEY amyotrophic lateral sclerosis (ALS) clinical trial showed a 13% improvement in function and mortality with an 88% success probability (versus the pre-specified 98%), according to a press release.1 Performed in collaboration with The Sean M. Healey and AMG Center, the study is evaluating Seelos Therapeutics’ SLS-005 (IV trehalose), a low molecular weight disaccharide. It functions by stabilizing misfolded proteins and activating autophagy.
Seelos received top-line results for regimen E, which includes a full analysis set, efficacy regimen only population and an efficacy RELYVRIO free population. The full analysis set includes shared placebo from all regimens.
In addition to the improvements in function and mortality, data showed a potential signal of efficacy in a pre-specified subgroup. However, the study did not meet statistical significance in the primary and secondary endpoint in the full analysis set.
According to an estimate from the Centers for Disease Control and Prevention, there are over 31,000 patients living with ALS in the United States as of 2017. On average 5,000 new patients are diagnosed with ALS every year.2
"The HEALEY platform is designed to detect signals of efficacy and we believe the observed signal and success probability is competitive to other recently FDA-approved therapies for ALS which also failed to achieve statistical significance when measured for function and mortality on similar primary and efficacy endpoints," Raj Mehra PhD, chairman and chief executive officer of Seelos said in the press release. "We plan to request a meeting with the FDA to discuss potential next steps for the program and will continue our potential partner discussions."
SLS-005 has previously received Orphan Drug Designation for the treatment of ALS from FDA and EMA. In addition to the Phase II/III HEALEY ALS Platform Trial, Seelos announced results from an in vivo study of SLS-005 in an aggressive Alzheimer's disease non-human primate model in September 2023.3
References
1. Seelos Therapeutics Provides Update on Top-Line Results from its Amyotrophic Lateral Sclerosis (ALS) Study with SLS-005 (IV Trehalose). News release. March 19, 2024. Accessed March 20, 2024. https://www.prnewswire.com/news-releases/seelos-therapeutics-provides-update-on-top-line-results-from-its-amyotrophic-lateral-sclerosis-als-study-with-sls-005-iv-trehalose-302092918.html
2. Amyotrophic lateral sclerosis. Centers for Disease Control and Prevention, U.S. Department of Health & Human Services. Accessed March 20, 2024. https://www.cdc.gov/als/WhatisALS.html
3. Seelos Therapeutics Announces Encouraging Data from an In Vivo Study of SLS-005 in an Aggressive Preclinical Model of Alzheimer's Disease. News release. September 27, 2023. Accessed March 20, 2024. https://www.prnewswire.com/news-releases/seelos-therapeutics-announces-encouraging-data-from-an-in-vivo-study-of-sls-005-in-an-aggressive-preclinical-model-of-alzheimers-disease-301939596.html
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