Forced to remedy an error in a study for a potential ALS drug, Cytokinetics ($CYTK) has revealed plans to amend the protocol of the midstage trial with expanded enrollment. The change boosts the number of patients in the study from 500 to 680 people, and at least one analyst thinks the larger data set could bolster the case for an expedited route to market for the drug.
South San Francisco-based Cytokinetics tackled fixes for the so-called Benefit-ALS study after discovering a programming error in its vendor's electronic data capture system for the trial, which caused 58 patients who were supposed to receive the company's experimental drug tirasemtiv to get placebo. The company is increasing enrollment and changing the statistical methods section of the study, expecting $5 million in added costs and a three-month delay in the trial, which is expected to report results in early 2014.
However, there could be a silver lining to the changes, especially if the data from the Phase IIb study are positive. Leerink Swann analyst Joseph Schwartz commented on the potential for Cytokinetics to use the larger data set in a case for accelerated approval of tirasemtiv, as the FDA sometimes allows companies to commercialize their therapies for patients with serious unmet medical needs after successful midstage studies.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, certainly fits the bill of a disease without ample treatment options. The incurable disease attacks the central nervous system, leading to eventual paralysis and in most cases death after patients lose their ability to control muscles needed to sustain life. Biogen Idec ($BIIB), Regeneron Pharmaceuticals ($REGN) and other companies have left a trail of trial failures in the ALS field with various treatment approaches that never panned out.
Cytokinetics could reverse the trend with positive data on tirasemtiv, a fast skeletal muscle troponin activator. The experimental drug follows the company's lead candidate, omecamtiv mecarbil, a potential med for heart failure under development with partner Amgen ($AMGN). Key data from the lead program are expected in September.
- here's the release
- check out Bloomberg's article