This week was a good one for a few biotechs, but not so great for others. Two in particular have just put out news relating to trials for their lead investigative assets, and both have taken a hit on the back of the release. Here’s a look at the companies in question, what happened, and where we think they go next.
The two companies we are looking at are Cyclacel Pharmaceuticals Inc (NASDAQ:CYCC) and Nivalis Therapeutics Inc (NASDAQ:NVLS).
Normally we’d start things out with the good news and ease readers into the bad. Unfortunately, this time around, there’s no real good news, so we’ll get right in to it and kick things off with Cyclacel.
The company just put out a release detailing the progress of a trial investigating a drug called sapacitabine, which is Cyclacel’s lead blood cancer asset. The trial was set up to investigate efficacy and safety in an indication of acute myeloid leukemia, and specifically in an elderly patient population. There’s a real unmet need at the moment in elderly cancer patients. To put it plainly, chemo is often too risky for these patients because they are, for the most part, not physically up to withstanding the toxic impact that standard of care chemo treatment has on their bodies. Sapacitabine is a chemotherapy drug in composition and MOA, but it’s what’s called a prodrug, meaning it goes through a delayed activation process. The delay means it doesn’t have to go through liver pass activation, and this reduces the toxic volume requirement (and by proxy, reduces the toxic impact of the chemotherapy treatment as a whole.
Anyway, none of this really matters anymore.
The news that Cyclacel just put out revealed that the drug doesn’t seem to work – at least not to the degree that the company and its shareholders (and of course, patients) had hoped.
The primary endpoint of the study was overall survival, and the drug did not meet this endpoint. There is some degree of silver lining, however. Against a secondary endpoint, an improved rate of complete remission, the drug hit. This doesn’t guarantee its future development, but given that this one is targeting a sub group of patients that have very little in the way of safe options on the table, then it may be a path forward.
It’s not enough to keep the company from taking a hit, however. Cyclacel currently trades for a little over $4 a share, at a 25% discount to its pre-announcement market cap.
Now let’s move on to Nivalis.
This one is a young biotech with a focus on developing therapies for patients with cystic fibrosis (CF), and just as with Cyclacel, Nivalis just put out data relating to one of its lead trials. The drug under investigation is called Cavosonstat, and the company was looking at it in combination with an already approved therapy called Ivacaftor (which many reading will likely be more familiar with if we call it by its commercial name, Kalydeco).
The theory was that by combining the mechanisms of action of the two drugs, Nivalis could improve upon the efficacy of the drug on its own. It was a big ask, but one with a large potential reward if it played out as an accurate thesis.
The trial set up a a double blind study, controlled using an arm that received placebo and Kalydeco, as opposed to active compound and Kalydeco. The primary endpoint of the trial was change in absolute percent predicted FEV1 from baseline to week 8. The drug failed to hit against this endpoint, with no stat sig improvement seen in the active arm over the control arm at the end of the 8-week period. To add insult to injury, a secondary endpoint, which was looking at sweat chloride reduction at 8 weeks (which as it sounds, is a measurement of chloride in patient’s sweat) also failed to hit.
Unlike Cyclacel, it doesn’t look as though Nivalis has a path forward with this one. Management has seemingly written off the drug with those infamous words – we hope that the data can contribute to the future understanding of the condition – and we take this as a sign of near term discontinuation of the drug in this indication.
