What does the drug do?
Tirasemtiv is a muscle activator that increases force (contraction) and power of skeletal muscles and therefore delays muscle fatigue. Instead of aiming to slow down progression of MND, the aim of this drug treatment is to increase quality of life by increasing muscular strength.
Based off some of the results of previous clinical trials (see below), a newer version of drug was made and called Reldesemtiv. This drug is designed to behave in the same way as Tirasemtiv.
Trial outcome
COURAGE-ALS ended earlier than planned. An interim analysis of data from the trial found no differences in ALSFRS-R score or any key secondary endpoints between those on placebo and those on the drug. There was no evidence that Reldesemtiv has a beneficial effect for people with MND based on data collected at the 24-week assessment and, due to this, the COURAGE-ALS trial and open label extension was concluded.
Previous Clinical Trials
Phase 3 (VITALITY-ALS) - Tirasemtiv
A Phase 3 clinical trial (known as VITALITY-ALS) recruited 600 people with MND in the USA, Canada and Europe. The trial tested the effectiveness of different doses of Tirasemtiv on breathing function over a period of 48 weeks. The trial was designed to try and reduce the side effects of the drug experienced in previous studies. Tirasemtiv failed to meet its primary and secondary endpoints in their Phase 3 clinical trial. You can read more about these results here. The development of Tirasemtiv was suspended.
Phase 2 (FORTITUDE-ALS) - Reldesemtiv
A new version of the drug, called Reldesemtiv, was tested in a Phase 2 trial which recruited 458 people with MND worldwide. Treatment with oral reldesemtiv appears to slow the deterioration of function, including breathing capacity and muscle strength in people with MND – regardless of whether they are taking riluzole or edaravone. You can read more about the trial here and the trial results here.
Phase 3 (COURAGE-ALS) - Reldesemtiv
The Phase 3 trial planned to enroll more than 500 people with early MND, to test reldesemtiv to see if it could slow progressive muscle weakness.The trial design built upon the results of the FORTITUDE-ALS Phase 2 trial. The primary goal of COURAGE-ALS was to measure changes in the participants’ ALSFRS-R scores over 24 weeks. You can find out more about the trial here.
The trial ended earlier than planned due too futility. An interim analysis of data from the trial found no differences in ALSFRS-R score or any key secondary endpoints between those on placebo and those on the drug. There was no evidence that Reldesemtiv has a beneficial effect for people with MND based on data collected at the 24-week assessment and, due to this, the COURAGE-ALS trial and open label extension concluded.
Latest News
2023
March 2023- COURAGE-ALS trial and open label exension ended early due to futility. You can read more here.
2022
June 2022 - An Open-Label Extension (OLE) was announced. You can read more here.
2021
Dec 2021 - Most of the patients enrolled are moderate to fast progressors - as discussed at the 32nd International Symposium on ALS/MND. You can read more here.
August 2021 - COURAGE-ALS opened for enrollment.
Resources
Last updated: 21/12/2022