Cytokinetics Inc. (NASDAQ: CYTK) shares took a step back on Monday after the firm announced that its midstage trial for amyotrophic lateral sclerosis (ALS) failed to meet expectations. Specifically, the Fortitude ALS trial did not achieve statistical significance for a pre-specified dose-response relationship in its primary endpoint of change from baseline in slow vital capacity after 12 weeks of dosing.
While the dose-response analyses for the primary and secondary endpoints did not achieve statistical significance, patients who received reldesemtiv in the Fortitude ALS trial declined less than patients who received placebo.
The trial showed effects favoring reldesemtiv across dose levels and timepoints with clinically meaningful magnitudes of effect observed at 12 weeks for the primary and secondary endpoints. The differences between reldesemtiv and placebo in slow vital capacity and ALSFRS-R total score observed after 12 weeks of treatment were still evident at follow-up, four weeks after the last dose of study drug.
The incidence of early treatment discontinuations, serious adverse events and clinical adverse events in Fortitude-ALS were similar between placebo and active treatment arms.
Dr. Jeremy Shefner, Professor and Chair of Neurology at Barrow Neurological Institute, and Professor and Executive Chair of Neurology at the University of Arizona, Phoenix, commented:
Results from FORTITUDE-ALS are among the most impressive we have seen in a Phase 2 clinical trial in ALS. … Especially noteworthy are the consistency and durability of effects observed across treatment arms on clinically meaningful endpoints.
Shares of Cytokinetics closed Friday at $8.85, in a 52-week range of $5.75 to $10.44. The consensus price target is $13.50. Following the announcement, the stock was down about 19% at $7.15 in early trading indications Monday.