Cytokinetics Incorporated (NASDAQ:CYTK) is one of the bigger movers in the biotechnology space this week. The company puts out data from one of its lead development trials and – as per the numbers – has failed to impress markets, inducing a decline in share price of around 27%.

The trial in question was called VITALITY-ALS and, as its name suggests, it was set up to investigate the safety and efficacy of a drug targeting the treatment of patients with amyotrophic lateral sclerosis (ALS).

This is a relatively high profile condition these days but, as little as three or four years ago, it was practically unknown. Subsequent to the ice bucket challenge that swept the globe and was put in place initially to raise money for research rooted in ALS, the prevalence of both awareness and programs set up to investigate potential treatments into the disease has dramatically increased.

It’s also known as being the condition from which renowned physicist Stephen Hawking’s suffers.

Anyway, as mentioned, the trial was looking to prove that a drug that Cytokinetics has developed could be effective in treating these patients and, specifically, was a phase 3 study investigating a drug called tirasemtiv.

The primary endpoint of the study looked at what’s called slow vital capacity (SVC), which is essentially a measurement of airway obstruction through the use of various lung capacity and forced expiratory volume measurements.

For the patient to have been deemed improved from baseline, the SVC would have had to have either increased, remain the same or – as compares to placebo – reduced to a smaller degree.

In this instance, however, things didn’t turn out that way.

The decline in SVC from baseline to 24 weeks was smaller in patients who received any dose of tirasemtiv in the study when compared to the decline in patients receiving placebo. In other words, the placebo arm actually performed better than did the active arm of the study.

That’s never what you want to see when investigating this sort of asset and especially in an indication like this.

and to make matters worse, while no new safety or tolerability findings related to tirasemtiv were identified in the investigation and the rate of serious adverse events was similar between patients who received tirasemtiv and those who received placebo, more patients discontinued double-blind treatment on tirasemtiv than on placebo – reportedly primarily due to non-serious adverse events related to tolerability.

So, to put all this another way, not only did the drug not outperform placebo in the phase 3 trial in this target population, it also induced a higher degree of non-serious adverse events related to tolerability and, in turn, caused more patients to discontinue the study based on this tolerability profile.

So what comes next?

Well, first of all, it’s worth noting that this is a pretty devastating result for the target population. There are basically no effective therapies on the market right now for treating ALS and the hope was that, with tirasemtiv, Cytokinetics could bring a much-needed treatment to market.

As such, nobody wanted to see a drug fail – especially in the manner in which it has failed on the back of this study.

With this failure in mind, there’s no immediately obvious path forward for the drug in this indication and management has supported this statement with the suggestion that it intends to discontinue (or at least suspend) development immediately subsequent to the data release.

However, all is not lost.

While Cytokinetics hasn’t managed to make the grade with tirasemtiv, the company has a secondary asset that it is developing in the same indication, ALS, called CK-2127107. This one works in a different way (it’s what’s called a fast skeletal muscle activator) but, based on early-stage data, it could be more tolerable than tirasemtiv and potentially more effective.

As such, market attention now turns to the development pathway for this drug and the preplanned data drops associated with the program that underpin its advance along said pathway. Phase 1 clinical studies pointed to a degree of efficacy and there are some phase II results slated to hit press during early to mid-2018 that – shareholders hope – will build on the early-stage numbers and support advance into a late-stage investigation.

Management is set to present the results of the VITALITY-ALS on December 8 in Boston but don’t expect markets to respond to any major degree to the news, given that the asset is already suspended.