Lighthouse II study terminated after interim analysis
April 5, 2025
TRICALS, in collaboration with King’s College London and Macquarie University, today announced that the global Phase 3 placebo-controlled Lighthouse II study was terminated after the interim analysis did not demonstrate an effect on its primary outcome measure.
The Lighthouse II study is a phase 3 randomized, double-blind, placebo-controlled study testing the drug Triumeq. As part of the trial design, a planned interim analysis was performed. This analysis resulted in the recommendation that the trial be stopped. The results to date showed no benefit of taking the drug Triumeq for people with ALS when compared with placebo on survival, the primary outcome measure.
Due to these disappointing results, the decision has been made to terminate the study. Therefore, all study participants have been contacted about the study results and asked to stop taking the study drug immediately. A full data analysis will be completed and published in due course.
Ammar Al-Chalabi, King’s College London, Co-Director of UK MND Research Institute, UK Coordinating Investigator (CI) of Lighthouse II:
We wish to thank all of the patients, their families and the study teams who participated in the Lighthouse II study. While this result may be disappointing, without the people with ALS who were willing to join the trial, we would not have been able to establish that this treatment is ineffective so quickly. We are grateful for their time, energy and determination to help the research effort to find treatments for this terrible disease.
Leonard van den Berg, chairman of TRICALS and EU CI for Lighthouse II:
This trial was innovative in many ways and a testimony to international collaboration. This study wouldn’t have been possible without the collaborative efforts of TRICALS, King’s College London and Macquarie University, as well as all 29 study sites. The fact that the Lighthouse II study was developed and sponsored by us with support from our funders, is a major accomplishment by itself.
We understand that this news, that this treatment is not helpful, will be disappointing to hear. However, negative trial results are also important, so we can rule out a potential drug candidate. We remain hopeful and will focus our efforts on other potential treatment to ultimately find a treatment for this devastating disease.
About Lighthouse II
The Lighthouse II study was a large-scale phase 3 study, that recruited its first participants in early 2022 and included more than 400 participants in total. The Lighthouse II study is a collaboration with associations from different countries: TRICALS, King’s College London and Macquarie University. Globally, 29 centres participated in the study: seven centres were located in Europe, ten in the UK, and twelve in Australia and New Zealand.
The study investigated whether human endogenous retroviruses (HERVs) play a role in the development of motor neuron disease (MND) and whether targeting HERVs might have therapeutic potential. The investigational drug is called Triumeq. This drug is known to potentially target HERVs, as it is currently being used to treat patients with HIV, a disease where retroviruses also play a role.
Funding
This study has been funded by global ALS patient organizations, foundations and governmental funding. The Lighthouse II study wouldn’t have been possible without the parties below:
Ulla-Carin Lindquist stiftelse för ALS-forskning, Fundación Luzón, Research Motor Neurone, Slovenian Research Agency (ARRS), Dutch ALS Foundation, Viiv Healthcare Macquarie University Kings College London, NIHR, My Name’5 Doddie Foundation, MND Association, MND Scotland, Alan Davidson Foundation, FightMND, MND Australia, MND New Zealand, ALS het licht uit gaat, Miquel Valls Foundation
