ALS TDI acknowledges the more than 1500 people with ALS in a dozen different countries whom participated in clinical trials of tirasemtiv over the last 10 years, leading to this disappointing yet definitive read out.
Cytokinetics announced today results from their phase 3 clinical trial of tirasemtiv in people with ALS. According to the company’s press release, there were no statistically significant impacts on disease progression measured in the yearlong study in more than 700 people with ALS. The company used a measure of respiratory function (slow vital capacity) as its primary endpoint and several others, including ALSFRS-R, as secondary endpoints. It reported that analysis showed that none of the endpoints were met. The company is halting development of tirasemtiv and turning their attention to a second-generation form of fast twitch skeletal muscle activator called CK-107. It is already in phase 2 clinical trial in people with ALS.
Robert I. Blum, Cytokinetics’ President and CEO, said in the press release this morning “While we are deeply disappointed by the results of VITALITY-ALS, we remain committed to people with ALS who are fighting this devastating disease and who need new therapies to slow the decline of respiratory function and muscle strength that are key hallmarks of disease progression. We have decided to suspend the development of tirasemtiv. While we believe that VITALITY-ALS demonstrated pharmacologic activity for the mechanism of action, we also believe that limitations of tirasemtiv may be addressed with our next-generation fast skeletal muscle activator, CK-2127107. Based on previous Phase 1 clinical studies, we believe CK-2127107 will be better tolerated and potentially more effective than tirasemtiv in patients with ALS and look forward to Phase 2 trial results in 2018. We are grateful to the trial investigators, site personnel, patients and caregivers who participated in VITALITY-ALS.”
Cytokinetics is holding teleconferences with investors, media, investigators and ALS community members over the next couple of days. It will also present data at the International Symposium on ALS/MND being co-hosted locally in Boston by the ALS Therapy Development Institute (ALS TDI) this December. ALS TDI will comment further on this important phase 3 clinical trial read out as additional data are expected to be made available over the coming days and weeks. This post will be updated with more information following review of trial data.