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Cytokinetics: What's Next?


Amyotrophic lateral sclerosis , also referred to as Lou Gehrig's disease, is a fatal neurological disease having no known cause or cure, and a majority of the patients die 3-5 years after diagnosis. The only treatment option for amyotrophic lateral sclerosis, or ALS, is the FDA-approved Rilutek (riluzole) of Sanofi-Aventis. Although this drug prolongs the life of patients by 2-3 months, it does not improve the disease symptoms or quality of life.

Developing a potential drug for ALS is South San Francisco-based biotechnology company Cytokinetics Inc. (CYTK).

For readers who are new to Cytokinetics, here's a brief overview of its pipeline and what to expect in the coming days...

There are two phase II clinical trials in patients with ALS with CK-2017357, the lead drug candidate from the company's skeletal muscle contractility program.

One of the phase II trials is evaluating multiple doses of CK-2017357 in ALS patients with and without the concurrent administration of FDA-approved Rilutek. The Part A of the ongoing phase II study enrolled 24 ALS patients who were not concurrently taking Riluzole and they were randomized to receive daily oral doses of either placebo or 125 mg, 250 mg, or 375 mg of CK-2017357, respectively, for two weeks.

The results of the first cohort, or Part A, of the study were reported last December. According to the results, CK-2017357 was well-tolerated at all dose levels and no serious adverse events were reported. The incidence of dizziness, the most common adverse event, appeared dose-related but was mild in severity in all patients who completed study drug treatment.

The second cohort, or Part B, of the study is identical in design to Part A, except that it has enrolled ALS patients who are concurrently taking Riluzole. The results from this cohort - Part B, are scheduled to be announced at the American Academy of Neurology 64th Annual Meeting in New Orleans, on April 25, 2012.

The second phase II clinical trial in ALS is a dose titration study to evaluate multiple ascending doses of CK-2017357. In this study, patients were randomized to one of two dosing groups and given twice daily oral ascending doses of CK-2017357 or placebo. The primary objective of the trial is to assess the safety and tolerability of this alternative dosing regimen of CK-2017357 in patients with ALS. The results from the study are also scheduled to be announced on April 25.

In the U.S., ALS afflicts 20,000 to 30,000 people, and roughly 5,600 new cases are diagnosed each year. CK-2017357 has orphan drug designation for the potential treatment of ALS in both the U.S. and Europe.

Biogen Idec Inc., Mitsubishi Tanabe Pharma Corp., Eisai Inc., Trophos SA, Neuraltus Pharmaceuticals Inc., Isis Pharmaceuticals Inc. and GlaxoSmithKline plc. are some of the other companies developing potential new therapies for ALS. Biogen Idec's ALS drug candidate Dexpramipexole is under phase III trial and data from the trial are expected in the second half of this year.

Rilutek, the only FDA approved drug for ALS patients, is reportedly said to have logged U.S. sales of about $50 million in 2011.

A phase IIa "Evidence of Effect" clinical trial of CK-2017357 in patients with claudication associated with peripheral artery disease was successfully completed last June.

Another phase IIa "Evidence of Effect" clinical trial of CK-2107357 in patients with generalized myasthenia gravis is also underway. Myasthenia gravis is chronic, autoimmune, neuromuscular disease. Data from this trial are anticipated in the first half of this year. The development of CK-2107357 as a potential treatment for myasthenia gravis is supported by the National Institute of Neurological Disorders and Stroke, or NINDS. A grant in the amount of about $2.8 million was awarded by the NINDS in July 2010 for a three-year research.

Omecamtiv Mecarbil is the lead drug candidate from Cytokinetics' cardiac muscle contractility program. Amgen holds an exclusive license to develop and commercialize Omecamtiv Mecarbil worldwide, except Japan.

An intravenous formulation of Omecamtiv Mecarbil is under phase IIb testing to evaluate its safety and efficacy in patients with left ventricular systolic dysfunction who are hospitalized with acute heart failure, known as ATOMIC-AHF (Acute Treatment with Omecamtiv Mecarbil to Increase Contractility in Acute Heart Failure).

A phase I study with oral formulations of Omecamtiv Mecarbil was initiated in healthy volunteers in February of this year. The study is designed to enroll approximately 60 subjects.

A quick look at the company's balance sheet...

Since inception in 1997, Cytokinetics has incurred operating losses every year, and had an accumulated deficit of $408.5 million at the end of 2011. The company has no marketed products, and currently recognizes revenue from grant funding for its myasthenia gravis preclinical and clinical activities, and from collaboration agreement with Amgen.

The company is scheduled to announce first quarter results on April 26.

Cytokinetics went public in April, 2004, offering its shares at $13 each. CYTK gained 1.74% on Friday to close the day's trading at $1.17. Over the last 52 weeks, the stock traded in the range of $0.89 - $1.59. CYTK has jumped over 14% in the last five days and more than 20% year-to-date.

by RTTNews Staff Writer

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