Amylyx: no effect after analyzing phase 3 study

The pharmaceutical company Amylyx announced the first results of the PHOENIX study. After analysis, the research drug AMX0035 unfortunately shows no effect in the treatment of ALS.

PHOENIX-study

The PHOENIX-study is a phase 3 trial in which the effectiveness and safety of the drug AMX0035 was tested. Participants were asked to take the drug orally for 48 weeks by dissolving it in water. Globally approximately 664 people with ALS participated in the research.

Response TRICALS

“This is of course not the news we wanted to hear. We are all very disappointed and can not imagine how it must feel for those people living with ALS who had hoped that this drug could have given them some salvation. The negative results are a setback. It will not stop us in finding that much needed cure for ALS. As an international network we believe that together we have the power, knowledge and resources to find an effective treatment. It may not be tomorrow or next week, but we will find it.

We want to express our gratitude to all the participants of the PHOENIX trial, their time and effort is not unnoticed and we would be nowhere without them. We are hopeful and will continue to dedicate our time on ALS research. Subsequently we want to thank Amylyx for the good collaboration and for executing such important research.” – Leonard van den Berg, global PI of the PHOENIX-study on behalf of the TRICALS executive board.

Approval FDA

In September 2022 Amylyx received full and conditional approval in America and Canada based on the results from the phase 2 trial (CENTAUR). The EMA did not give its approval based on these data and wanted to await the results from the current phase 3 PHOENIX trial. Unfortunately, these results turned out negative, there is no effect of the drug. It is unclear how this outcome affects approval in the USA and Canada.

How will this affect people with ALS in Europe?

Amylyx will use the upcoming weeks to investigate the results in more depth. This will not change the negative outcome of the trial but might provide researchers with leads for future studies. Until Amylyx has researched all the data, the open-label extension study will stay open and in effect. As soon as that changes, Amylyx will update TRICALS.

If you have further questions regarding this news, you can always contact us via info@tricals.org or contact your neurologist.

Click here to read the press release from Amylyx.