(Bloomberg) — Thousands of ice bucket dumps later, funds from the ALS Association’s blockbuster charity effort that raised $115 million last summer are going to a drug in late-stage trials for the first time.
Cytokinetics Inc. said it received $1.5 million to help finance testing of an experimental drug to improve lung function in patients with the devastating neurodegenerative disorder. The South San Francisco biotechnology company is the first with a drug at such an advanced stage to get money from the viral fundraising effort, which gathered millions of social media views as it gained supporters ranging from actor Matt Damon to billionaire Bill Gates dumping a bucket of ice on their heads as a charity stunt.
Cytokinetics is starting a phase 3 trial for its compound, called tirasemtiv. Although a mid-stage trial of the drug failed to show overall benefit on what is known as the ALS Functional Rating Score, it showed improvement in maintaining patients’ lung function, said Cytokinetics’ Chief Executive Officer Robert Blum. The current study is designed to confirm this effect and test if tirasemtiv can help delay the need for mechanically assisted ventilation.
The grant from the ALS Association is small compared with the estimated trial costs of about $40 million, said Blum. However, it will enable collection of blood plasma samples from patients to be studied by the Northeastern ALS Consortium Repository, which is looking for biological indicators that can be used to measure disease progression and better understand the underlying causes of the disease.