Expert Financial Analysis and Reporting

Cytokinetics has announced that the VITALITY-ALS trial of tirasemtiv has completed enrollment. The study includes three phases. The first is a two week open label phase; those patients who can tolerate tirasemtiv are then randomized into the second phase. This is a 48 week double-blind, placebo-controlled trial in which patients are randomized 3:2:2:2 to placebo and three different dose levels of tirasemtiv. The third phase is four weeks and is double-blind, placebo-controlled in which tirasemtiv is withdrawn from patients who were receiving it.

This indicates that the last patient enrolled can be evaluated about 52 weeks from now or sometime after mid-August of 2017. Then gathering and analysis of the data could take two to three months which suggests that we could see topline data in October or November of 2017.

The primary endpoint of the trial is change from baseline to week 24 of slow vital capacity (SVC).

Secondary outcome measures are:

• Time to the first occurrence of a decline from baseline in percent predicted SVC ≥ 20 percentage points or the onset of respiratory insufficiency or death during double-blind, placebo-controlled treatment (time frame: 48 weeks)
• Time to the first occurrence of a decline in SVC to ≤ 50% predicted or the onset of respiratory insufficiency or death during double-blind, placebo-controlled treatment (time frame: 48 weeks)
• Time to the first occurrence of the first use of mechanical ventilatory assistance or death during double-blind, placebo-controlled treatment (time frame: 48 weeks)
• Time to the first occurrence of a decline in the respiratory components of the ALSFRS-R (i.e., items 10, 11, and 12) or death during double-blind, placebo-controlled treatment (time frame: 48 weeks )
• Slope in muscle strength mega-score change from baseline during the randomized, double-blind, placebo-controlled phase (time frame: 24 weeks )

Other reports on Cytokinetics can be found in the reports section of my website.