) recently provided an update on BENEFIT-ALS (
), the phase IIb study being conducted on its amyotrophic lateral
sclerosis (ALS) candidate, tirasemtiv.
The company reported a programming error in the multinational,
double-blind, randomized, placebo-controlled study which is
evaluating the safety, tolerability and efficacy of
Cytokinetics' data management vendor reported that due to a
programming error in the electronic data capture system
controlling study drug assignment, 58 patients received placebo
at a certain study visit instead of tirasemtiv.
The company said that no incorrect treatment was conducted on
the patients in the placebo arm. Cytokinetics said that the
company as well as trial site personnel remained blinded to the
58 patients affected by the error.
Cytokinetics is taking measures to confirm that no other such
errors have been made and the programming defect has been
corrected. Cytokinetics also conducted an ad hoc meeting of the
study`s Data Safety Monitoring Board (DSMB) to determine that the
safety of the 58 patients affected by the error has not been
impacted. The DSMB reviewed the safety data and reported that
there were no concerns regarding patient safety.
Patient enrolment for the BENEFIT-ALS study is ongoing - the
study, which is designed to enroll up to 500 patients, has
enrolled 450 patients so far. Cytokinetics said that it may
change the current protocol so as to enroll additional
The company is working with regulatory authorities on the most
suitable way to respond to the error so that the intended
scientific value of BENEFIT-ALS may be maintained.
We do not expect the programming error to have a major impact
as the study remains blinded and the error was detected well
before final analysis. Moreover, the safety profile was not
Still, we believe the company will most likely amend the
protocol and enroll additional patients. On its first quarter
2013 conference call, the company had said that results from the
BENEFIT-ALS study would be out by year end. However, enrolment of
additional patients could push out study results by a few months
to early 2014. Study costs will also go up.
An update on the company's plans should be available following
discussions with regulatory authorities.
Cytokinetics currently carries a Zacks Rank #2 (Buy). We
expect investor focus to remain on results from the ATOMIC-HF
study that is evaluating omecamtiv mecarbil in patients with left
ventricular systolic dysfunction who are hospitalized with acute
heart failure. The company intends to present results in late
At present, companies that look well-positioned include
Peregrine Pharmaceuticals, Inc.
Cytori Therapeutics, Inc.
). All three are Zacks Rank #1 (Strong Buy).
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