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Cytokinetics, Inc. (CYTK)
Lazard Capital Markets Healthcare Conference
November 13, 2012 08:00 am ET
Robert Blum - President & Chief Executive Officer
Josh Schimmer -Lazard Capital Markets
Josh Schimmer -Lazard Capital Markets
Previous Statements by CYTK
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» Cytokinetics Inc. Q2 2010 Earnings Call Transcript
So, Robert, thank you for joining us.
Thank you, Josh. Thank you to Lazard for welcoming us back to the conference. We are very pleased to be here and providing this update. I'll be making some forward looking statements. I'll refer you to our SEC filings for caveats relating those statements. And, of course, these forward-looking statements, we recognize that they carry certain risks associated with it and they are explained on this slide.
Cytokinetics, a view from above, we are focused as Josh has said to muscle contractility, in particular the mechanics of how muscles perform. We have advanced portfolio of leading first-in-class, best-in-class small molecule activators of cardiac and skeletal muscle.
One is advancing forward, partnered with Amgen in the potential treatment of heart failure. I'll provide updates to program here today. And, another is undergoing a Phase IIb study, currently in an unpartnered program towards disease indication of ALS, where we have orphan and fast-track designations.
This is a picture of the sarcomere. This is the reconstituted, multi-protein complex that drives muscle contractility. The sarcomere is the fundamental unit of muscle contractility and circled on this graphic are those molecular targets that are the targets of our drug mechanisms that cardiac program is directed to cardiac myosin. It's an engine that drives cardiac muscle mechanics and contractility. Omecamtiv is a small molecule activator of cardiac myosin.
Tirasemtiv is an activator of fast-twitch skeletal troponin. These are the protein components that allow for increased muscle force as I'll now elaborate in each program.
The ALS program is one that came subsequent to the cardiac program, but it's advancing very rapidly now in a Phase IIb study that has registration status implications. We announced a few weeks ago that we opened to enrollment this trial. ALS as you may know is a truly grievous illness. ALS afflicts above 25,000 to 30,000 people in the United States in terms of its prevalence.
But, where the mortality associated with this disease is quite striking ALS patients regrettably have only about three to four years often times following a diagnosis and with about 5,000 to 8,000 patients newly diagnosed each year. One can expect those patients will have about three to four years subsequent to that diagnosis and our goal with tirasemtiv is to substantially enhance the activities of daily living and functional status for those patients.
We've done a very thorough job of characterizing safety and tolerability, potential drug, drug interactions, pharmacokinetics and pharmacodynamics of tirasemtiv, firstly in healthy subjects and more recently in ALS patients and also in other indications. We have announced all of these data at prior meetings, and what we've seen like we have observed pre-clinically is that tirasemtiv in activating troponin is enhancing muscle force and power and endurance. And in ALS patients, that's translated into very encouraging pharmacodynamic effects in three completed Phase IIa clinical trials.
The first one in Evidence of Effect study, CY 4021, demonstrated that patients on the drug compared to those same patients when they receive placebo, so this was a crossover design study. Patients were feeling better and also seeing enhancements in pulmonary function and other measures of muscle strength.
We follow that study with two other Phase IIa trials, one of two-week duration and one of three weeks duration to optimize the dosing schedule and to understand issues of safety and regimen. All of those studies are complete now. The two and three-week studies were presented at the AAN meetings earlier this year and demonstrated for really the first time that there is an opportunity with this mechanism of action to actually help these patients improve their functional status as we saw some trends towards increases in ALSFRS, the functional rating scale that is the pivotal endpoint for clinical trials in ALS.
So, with that as a backdrop, we raised money in June of this year in order to manufacture drug to proceed to a Phase IIb study. That Phase IIb study has been formed by discussions with each of FDA and EMA, and it is now open to enrollment. That study is a 400-patient trial that is being conducted in the U.S. and Canada and Europe and will be evaluating tirasemtiv dosed twice daily versus placebo for duration of three months treatment. And at the end of three months, the primary efficacy analysis will be a measurement of the ALSFRS and will be in particular looking at the change from base line.
The ALSFRS as depicted in this graphic is a 12-domain patient reported outcome tool that measures everything from fine motor skills to speech and swallowing, handwriting and pulmonary function. And in prior studies, we saw across all of these domains signs that tirasemtiv could even increase the ALSFRS albeit as would be deemed a very successful outcome, a change in the rate of decline would also we believe be approvable.