Clinical trial data is the lifeblood of the biotechnology space as far as volatility is concerned and, when fresh data hits, it generally dictates the share price direction of the company conducting the study for the near-term future (this is especially true if the company in question is a development stage company with little to no commercial assets).
On Monday this week, a couple of companies put out data from their lead respective clinical studies and things haven’t gone as the companies in question or their shareholders might have hoped. Here’s a look at what happened, what went wrong and where things stand going forward for each.
The two companies that form the basis for today’s discussion are Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN).
Kicking things off then, Zynerba.
This company is trying to develop a drug called ZYN002, which is a sort of cannabidiol gel, and it is designed for the treatment of the condition called osteoarthritis. Many reading will probably already be familiar with this condition, but for those not, it is rooted in the degradation of cartilage and inflammation in the joints (often in the knees) and is associated with chronic pain sufferers. With ZYN002, Zynerba was trying to offer patients a topical treatment that could serve up short-term relief on a regular basis – something that, in this market right now, is severely lacking outside of the standard pain management market (read: opioids).
The drug was under investigation as part of a phase 2 study and the data we just got derived from said investigation. As per the news, ZYN002 failed to hit on its primary endpoint and thus failed the study.
Zynerba is trading down on the news, but all might not be as bad as it seems. The company thinks it can carry forward into a phase 3 study based on promising secondary endpoints and this potential has taken the edge off the negative impact somewhat. Just how much of a chance this drug has of picking up a regulatory approval in the US based on secondary endpoints remains to be seen, but it better than a discontinuation from the perspective of shareholders. The pathway forward is likely going to involve an alteration of the phase 2 protocol that can settle the phase 3 study for a higher chance of success (or in other words, shifting the secondary endpoints into pole position for the investigation) but this is speculative. No doubt we will see more information hit press during the coming weeks as the company absorbs the data and its impact fully.
Moving on, Regeneron.
Regeneron is developing a drug called suptavumab (REGN2222). It is an antibody type drug that is designed to treat what’s called respiratory syncytial virus (RSV), or what is more commonly referred to as the common cold. The current standard of care treatments in the space are relatively strong, but over time, their efficacy reduces, and this has necessitated alternative approaches. Regeneron was part of a handful of companies trying to bring fresh alternatives to market and the latest news relates to a phase 3 study of the drug in question, REGN2222, investigating the asset in the target patient population.
Just as with Zynerba’s asset, REGN2222 failed to hit his primary endpoint. The trial pitched the drug against placebo in newborn infants, with the study investigating its ability to prevent said infants from becoming infected with the virus. As per the data, the population that was treated with the active drug did not see a statistically significant reduction in infection across the study period and – by proxy – failed to hit against the primary endpoint as noted above. In this instance, however, Regeneron is taking a different attitude towards the failure. While the above discussed Zynerba has vowed to push forward with its investigation based on secondary endpoints that seem to imply efficacy, Regeneron has decided to ditch the trial together and ditch the asset in this indication.
This is a $50 billion company so the dropping of one asset isn’t going to have too much of an impact on market capitalization. With that said, don’t be surprised to see a few percentage points wiped off valuation as we head into the middle of the week.