Acorda Therapeutics Inc (NASDAQ:ACOR) just gave an update on its Dalfampridine Trial, and it looks as though the drug and its development could serve as an upside catalyst for the company going forward – assuming everything runs smoothly. Acorda is running a phase IIb in an attempt to expand upon the drug’s current multiple sclerosis indication, to a post stroke victim indication, and data is scheduled for release before the year draws to a close. If the company can demonstrate efficacy, it will be a step closer to what could prove a billion-dollar expansion, so here’s a look at how the drug works, and what to look for going forward as the development timeline progresses.
First then, the science that underpins the drug. As mentioned, Dalfampridine is already approved as a multiple sclerosis treatment for patients with mobility issues. The way it would work in a post stroke indication is very similar, in that it will target patients who have difficulty walking and controlling general movement. So what’s the mechanism of action? The drug is what’s called an isomeric amine of pyridine. We don’t really need to go into detail of what this means, as it is more chemistry than biology, but we can explain the drug’s MOA as follows. It’s all rooted in potassium channels. In patients with multiple sclerosis, and – as Acorda is hoping to demonstrate with the ongoing trial, in post stroke victims – a leading cause of inhibited mobility is rooted in the augmentation of what are called potassium currents. That is, the flow of potassium ions, and the impact this has on competition between synapses, neurons and muscles. Dalfampridine is a potassium channel blocker. By inhibiting the flow of potassium ion’s through potassium channels, the drug is thought to aid conduction, and in turn, mobility.
So where are we with trials? In its latest earnings conference call, Acorda management discussed the progress of the ongoing phase IIb. Initially, the company expected to run the trial through to mid 2017, at which point it would conduct a topline analysis. In line with this, before the close of 2016, we expected an interim futile analysis. As just announced, however, the company has decided to stop the trial while it conducts an analysis across the next few months. This means we should get some insight into both the efficacy and tolerability of the drug in this post stroke indication, and in turn, an idea of whether the drug will be pitched for NDA across the next 24 months. Based on the upcoming analysis, Acorda is going to decide whether to expand into a second and third phase 3 trial during 2017 – the assumption being that these trials would form the basis of any such NDA if efficacy showed.
What are we looking for going forward? First and foremost, we want to see the ongoing phase 2 data. It won’t be quite as extensive as was initially planned, as it only just reached 50% enrollment, but it’s a reasonable scale, and so even with trimmed down numbers we should be able to see pretty clearly whether the drug has a positive impact on patients’ ability to walk after suffering a stroke, or not. Similarly, safety and tolerability should be pretty straightforward. The drug is already approved in its multiple sclerosis indication, and while there are some side effects labeled (nausea, dizziness, some muscle pain and fatigue etc.) these are probably not severe enough to have any impact on the FDA’s eventual decision – that is, assuming we get efficacy.
Looking at its wider pipeline, Acorda also has a number of other trials ongoing, and these look set to generate some volatility (albeit, short-term volatility) as we head into the second half of 2016. CVT-301 and tizanidine for Parkinson’s disease is in a phase III, expected to close out before the end of the year, and Plumiaz for seizure clusters is in an extension trial, again set to close before the end of the year.
If successful in both of the aforementioned trials, the company expects to submit an NDA for both the Parkinson’s disease and seizure clusters indications some time during early 2017. All said, Acorda, and its shareholders, have a busy 12 months ahead of them. One to watch.