Dive Brief:
- The ALS Foundation's Ice Bucket Challenge is still funding research to bring new drugs for the devastating neurodegenerative disease to market, even two years after the viral fundraising campaign first began.
- One of the latest contributions—approximately $1.5 million— are going to fund Cytokinetics' Phase 3 VITALITY-ALS study, which will test tirasemtiv compared with placebo in affecting slow vital capacity.
- The South San Francisco biotech said the late-stage study is fully enrolled with 700 patients at 81 centers in 11 countries.
Dive Insight:
The ALS Foundation made a splash two years ago when it used an "Ice Bucket Challenge" to help fundraise for the fight against the disease. The challenge required participants to make videos of themselves getting drenched in ice water to raise awareness of amyotrophic lateral sclerosis (ALS). The challenge went viral and helped the foundation raise more than $220 million for the disease worldwide.
Despite the major funding push from the ALS Foundation, the Centers for Disease Control says that diagnosis of ALS is increasing with more than five in every 100,000 people having the disease in 2013, the most recent year that prevalence was assessed.
There are currently no treatments for ALS, which affects nerve cells in the brain and spinal cord resulting in skeletal weakness and, ultimately, respiratory failure. The disease affects about 30,000 people in the U.S. and an equivalent number in Europe.
Tirasemtiv, a fast skeletal muscle troponin activator, is Cytokinetics' lead drug candidate that is partnered with Astellas Pharma, which has the option to commercialize the drug based on the results of the VITALITY-ALS study. Astellas recently contributed $65 million in funding to Cytokinetics in conjunction with the deal.
Cytokinetics expects the VITALITY-ALS study to report out in the second half of 2017 and plans to begin an open-label extension of the trial in the fourth quarter.