TLSR: Speaking of neurodegenerative diseases, both Cytokinetics Inc. (CYTK:NASDAQ) and Acadia Pharmaceuticals are operating in that space, and their shares have also appreciated well in the past six months. What's driving the interest in these companies?
GZ: I'll speak first about Cytokinetics. One of its drugs, tirasemtiv, is going into a pivotal phase 2 trial in amyotrophic lateral sclerosis (ALS). I think there's a lot of excitement about the company, given the failure of the Biogen Idec Inc. (BIBB:NASDAQ) product for ALS, dexpramipexole, which had fast-track status but was discontinued by Biogen because it didn't show efficacy in a phase 3 trial. With that failure, Cytokinetics emerged as a leader in the ALS space. We are expecting top-line data from the BENEFIT-ALS trial of tirasemtiv, which is a phase 2b study and potentially registrational. Following the failure of dexpramipexole, tirasemtiv became the only clinical candidate that may slow ALS disease progression.
I'd also like to point out that the FDA's neurology division held an unprecedented full-day hearing on ALS on Feb. 25. Its purpose was to explore ways to accelerate drug development for ALS and to expand patient access to unapproved investigational drugs. Researchers, clinicians and associated nonprofit organizations, as well as ALS patients, urged the FDA to think differently about the risks and benefits to ALS patients because of their short life expectancy. Suggestions included early conditional approval for drugs that are potentially disease-modifying, replacing placebo arms in trials with historical controls so that all ALS patients have access to an investigational drug, expanding access to drugs shown to be safe and then proving efficacy in post-registrational studies, and allowing patients who have participated in trials to continue with a drug after the trial ends, if any benefits were seen. Nothing was decided at this hearing, and it doesn't affect the ongoing BENEFIT-ALS trial, but if the trial delivers positive results it could lead to much faster approval.
In addition to tirasemtiv, we are also expecting the phase 3 development pathway for omecamtiv mecarbil to unfold by year-end. If successful, omecamtiv can provide seamless treatment from the acute to chronic setting of heart failure. Heart failure patients could also begin treatment without having an acute event. This program is partnered and funded by Amgen Inc. (AMGN:NASDAQ) up to phase 2, so Cytokinetics is limited in what it can and cannot say. Recent progress includes accelerating enrollment for the company's 600-patient phase 2b ATOMIC-AHF trial for acute heart failure toward the end of 2012, and Amgen's initiation of a 420-patient phase 2 trial called COSMIC-HF; a dose-escalation trial to find the optimal oral dose of omecamtiv.
TLSR: What is the next share price catalyst for Cytokinetics? Would it be the top-line data from the 400 patients enrolled in the BENEFIT-ALS trial of tirasemtiv?