The BENEFIT-ALS TRIALS: Cytokinetics (CYTK) announced an update on the conduct of BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS). The BENEFIT-ALS is a Phase 2b, multinational, double-blind, randomized, placebo-controlled clinical trial designed to evaluate the safety, tolerability and potential efficacy of tirasemtiv, a fast skeletal muscle troponin activator, in patients with amyotrophic lateral sclerosis (ALS).
BENEFIT-ALS is currently designed to enroll up to 500 patients with ALS. To date, over 450 patients have been enrolled in this study. The primary analysis of BENEFIT-ALS will compare the mean change from baseline in the ALS Functional Rating Scale in its revised form, or ALSFRS-R (a clinically validated instrument designed to measure disease progression and changes in functional status), in patients receiving tirasemtiv versus those receiving placebo.
Cytokinetics was recently informed by its data management vendor that a programming error in the electronic data capture system controlling study drug assignment caused 58 patients initially randomized to and treated with tirasemtiv to receive placebo instead at a certain study visit and for the remainder of the study. No patients randomized to placebo were dispensed incorrect treatment. Cytokinetics and all clinical trial site personnel remain blinded to the specific patients affected by the error.
Since the error was detected, the company has taken steps to ensure that no further incorrect study drug assignments have occurred and the programming error in the electronic data capture system controlling study drug assignment has been corrected.
Safety Data Not Affected: Cytokinetics recently convened an ad hoc meeting of the study`s Data Safety Monitoring Board (DSMB) to assess whether the error in dispensing study drug had impacted the safety of the 58 affected patients. After review of the relevant safety data from BENEFIT-ALS, the DSMB reported no concerns regarding patient safety.
Cytokinetics is in communication with regulatory authorities regarding how best to respond to the error in drug assignment in order to preserve the intended scientific value of BENEFIT-ALS.
The company continues to enroll patients in the study under the current protocol and may amend the protocol to allow increased enrollment. Following further communications with regulatory authorities, Cytokinetics expects to provide updated guidance relating to the conduct of BENEFIT-ALS, which may include revisions to the timing of publicly available results from the study as well as to the projected costs of the study.
Prohost Comments: A mistake occurred in an ALS trial. All we know about this subject now is that the mistake (read above) had no impact on the patients or the trial except for a possible increase the cost of the trials a possible delay in announcing the results. We don’t really understand the reason behind investors’ attempts to sell the stock before even knowing the impact of the error, which seems not to be as grave as, for example, a decision to end the trial for reason of failure.
The fact is that our enthusiasm for this firm does not really stem from this trial because we know how hard it is to treat ALS. We decided to buy CYTK based on Amgen’s enthusiasm towards Cytokinetics oral and intravenous drug omecamtiv mecarbil, which the large biotech firm is actively developing them in Phase 2b based on promising results from previous trials. Our enthusiasm towards the firm’s skeletal muscle activators program also has risen recently based on news that Cytokinetics and Astellas Pharma decided to collaborate in research, development and commercialization of skeletal muscle activators. The collaboration aims at advancing novel therapies for diseases and medical conditions associated with muscle weakness. We would be really concerned at the sight of any setbacks in Amgen heart failure program, or in Astellas/Cytokinetics’ programs, but not about an electronic mistake that harmed no one.
It looks as if traders want to expand their blind rule of thumb: “sell good news” to sell at whatever news biotech companies announce.
We have CYTK in our portfolio
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