Tirasemtiv

[nikolab]

Hi,

Is anyone familiar with Tirasemtiv and ALS clinical trials?


Best regards from Serbia !

 

[MaxEidswick]

I have put it on my list to ask about at clinic next week ... Thanks for the heads up!

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Cytokinetics is independently developing tirasemtiv, a fast skeletal muscle activator, as a potential treatment for medical conditions associated with neuromuscular dysfunction. Tirasemtiv is currently involved in a Phase IIb clinical trial, and has been granted orphan drug designation and fast-track status by the U.S. Food and Drug Administration (FDA) for the potential treatment of amyotrophic lateral sclerosis (ALS).

Cytokinetics is also collaborating to develop CK-2127107, a skeletal muscle activator structurally distinct from tirasemtiv, for non-neuromuscular indications. This drug has the opportunity to address needs within diseases and disorders such as Multiple Sclerosis and Parkinson's.

Most Significant Upcoming Catalyst

In the fourth quarter of 2013, Cytokinetics completed the enrollment of its Phase IIb trial for tirasemtiv called BENEFIT-ALS. This trial includes 711 patients, and is designed to document the effects of tirasemtiv in the treatment of ALS.
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[danrising]

....I have put it on my list to ask about at clinic next week ... Thanks for the heads up!

Keep me posted on what you find out..

Thanks

 

[pearshoot]

was in phase II part a.....real thing 14 days....Nov 2012....significant success for me....did not take long to lose gain....often wonder what IF had I been able to continue....think part b lasts a year....50/50 real thing

 

[hjlindley]

I was in the phase 2 part B, but it dropped my blood pressure so dangerously low I had to stop taking it. It also had a serious in the reaction with tizanidine, the muscle relaxant I take.

 

[mbmurray]

Disappointing news , I was in this trial. Another one bites the dust




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.

 

[lgelb]

The necessity of showing statistically significant differences between standard care and treatment groups on the ALSFRS-R over a 12-week period will doom pretty much anything. The heterogeneity of the disease, many failings of the scale and relatively short treatment period, not to mention that you are trying to influence a downstream mechanism (the muscles controlled by the dying nerves), made this one a poor hole-in-one bet from the start. However, more than one neuro drug has returned to life after a failed pivotal trial and this compound is in development for several indications, making it more likely to reach the market ultimately. Cytokinetics, like RPR (Rilutek) before it, may be able to slide through in ALS on the strength of subgroup analyses and a lower bar in this disease.

 

[FSHolbrook]

I just got an email that said tirasemtiv had been approved and may help with respiratory distress.

Rick

 

[MaxEidswick]

>I just got an email that said tirasemtiv had been approved and may help with respiratory distress.

http://campaign.r20.constantcontact...edd6c&ch=98f11920-b6ff-11e3-bb6e-d4ae526edd6c

 

[Nikki J]

If it does slow respiratory decline that would be a huge benefit. I do not believe however it has been "approved" for release to us. It sounds like it might be heading for another clinical trial. Well worth keeping an eye on it!