Over 50 clinical trials with a single focus:Improve lives
Cytokinetics is committed to translating our advanced understanding of muscle biology into novel treatments that may help people with debilitating diseases in which muscle performance is compromised and/or declining. We recognize that the ALS community is in desperate need of treatment options and interested in the possibility of our making our investigational therapy tirasemtiv available upon request prior to potential regulatory approval.
Any access to tirasemtiv would have to be fair and equitable, safeguard the welfare of patients, and not put at risk the completion of our ongoing research and development. Cytokinetics has sought advice from physicians, ethicists, and patient advocates on these important considerations. Through these interactions, we have concluded that sustainable, long-term availability of tirasemtiv for patients with ALS can best be achieved following demonstration of the investigational drug’s safety, tolerability, and effectiveness through additional controlled clinical trials, which may hopefully address the requirements of regulatory agencies. Offering pre-approval access outside of a controlled clinical trial at this time may jeopardize the conduct of our ongoing clinical trial and may also prevent or delay our broader development program for tirasemtiv, which would likely then result in a setback to the ALS community. Accordingly, we have reached the difficult decision that it would not be appropriate to provide access to tirasemtiv outside of our clinical trials at this time.
We have recently completed enrollment for our Phase 3 multi-national, double-blind, randomized, placebo-controlled clinical trial called VITALITY-ALS (Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices after Treatment for a Year in ALS), which was conducted at clinical sites in North America and Europe. The trial assessed the effects of tirasemtiv versus placebo on measures of respiratory function in patients with ALS. As participants conclude their participation in VITALITY-ALS, they are given the opportunity to enter VIGOR-ALS, an open-label extension clinical trial in which all participants in VITALITY-ALS are able to receive tirasemtiv. More information on VIGOR-ALS is available on clinicaltrials.gov.
We are grateful to the people and families living with ALS who have participated in a clinical trial of tirasemtiv. At this time, we are not offering an additional open-label clinical trial for participants from our previously completed phase 2 clinical trials of tirasemtiv, such as BENEFIT-ALS. However, we do expect to consider options regarding potential pre-approval access to tirasemtiv for these people who participated in prior clinical trials.