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Shares of Cytokinetics, Inc. (CYTK - Snapshot Report) tumbled 64.7% after it announced that tirasemtiv failed to meet its primary endpoint in the phase IIb, BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS) study.

Tirasemtiv is Cytokinetics’ lead candidate under its skeletal muscle contractility program.

The BENEFIT-ALS study evaluated the safety, tolerability and efficacy of tirasemtiv in patients suffering from amyotrophic lateral sclerosis (ALS). Enrolment in this study was completed in Dec 2013.

Cytokinetics recently reported top-line results from the BENEFIT-ALS study. The detailed results will be presented during the 66th Annual Meeting of the American Academy of Neurology (AAN) on Apr 29, 2014.

The BENEFIT-ALS study missed the primary endpoint of mean change in the ALS Functional Rating Scale from baseline in its revised form (ALSFRS-R) on tirasemtiv against placebo in the study (-2.98 points (tirasemtiv) versus -2.40 points (placebo). The secondary efficacy endpoints showed mixed results.

Once the complete data from the BENEFIT-ALS study is available, the company will review and evaluate the same. Cytokinetics will then arrive at a decision regarding the development path of the candidate.

The failure of the candidate is disappointing news for Cytokinetics, as it was the lead candidate under its skeletal muscle contractility program.

Cytokinetics currently carries a Zacks Rank #3 (Hold). Investors looking for better-ranked stocks in the biopharma sector may consider companies like Questcor Pharmaceuticals, Inc. , Alexion Pharmaceuticals, Inc. (ALXN - Analyst Report) and Sucampo Pharmaceuticals, Inc. (SCMP - Snapshot Report). Questcor and Sucampo carry a Zacks Rank #1 (Strong Buy) while Alexion carries a Zacks Rank #2 (Buy).

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