Here’s an interesting one. ABLYNX N.V. NPV (OTCMKTS:ABLYF) is a relatively under the radar Belgian biotech company with a focus on what are called nanobodies. We will get into these in a little more detail shortly. The company just reported data from a phase 2 trial, and the data sets up some compelling evidence of efficacy and safety. As we have said, Ablynx is relatively under the radar, and so markets are yet to respond to the data. As such, this serves up an opportunity to get in and take advantage of the inefficiency. With this in mind, here is a look at the drug and the technology in question, and a look at what the data tells us about the drug’s chances of approval in Europe.
The drug is called vobarilizumab, and it’s an anti IL-6R nanobody. In order to understand how these sorts of drugs work, it is first important to understand the way antibodies work. Antibodies in our body bind to antigens with the goal of doing one of two things. The first, tagging the antigen for attack by the wider immune system, and the second, directly influencing the antigen’s actions – i.e. stopping it from replicating etc. We have been creating antibodies as therapeutic candidates for a long time now, but they have their limitations. Primarily, the limitations revolve around their size. What we have now discovered, is that llamas and camels have antibodies that are composed of just heavy chain immunoglobulin, as opposed to the combination of heavy chain and light chain immunoglobulin found in human antibodies. From these heavy chain llama antibodies, it is possible to derive nanobodies, which are essentially the active ends of the antibody in question, chopped off the antibody for use on their own. It remains active, but it is something like 1/10 of the size of the antibody, and this has a number of benefits. First, it is able to bind to receptors on antigens that would otherwise be hidden from larger antibodies (these are called deep pocket receptors). Second, it is much more suited for oral administration because the small size makes it far easier for the bloodstream to absorb the drug.
Vobarilizumab is one of these nanobodies, and it targets the receptor IL-6. This receptor is associated with inflammation, and so by stopping it from becoming active, the drug is theoretically able to negate to the inflammation associated with (and the root cause of) rheumatoid arthritis.
So that’s the science out of the way, what did the data tell us?
The trial is looking at the drug compared to placebo, and using an industry standard measurement to assess the difference in response across the patients. The measurement is called ACR, and specifically, ACR 20, ACR 50 and ACR 70. We don’t really need to go into too much detail about how the scores are calculated – all that is really important to understand is that ACR 20 refers to a 20% improvement from baseline in symptoms of rheumatoid arthritis, ACR 50 refers to 50% improvement and ACR 70 refers to a 70% Improvement.
Across the trial, 79% of individuals recorded ACR 20, 51% of individuals recorded ACR 50, 43% of individuals reported ACR 70 at week 24. Coupled with a reported excellent safety profile, these data strongly support an efficacy hypothesis, and are a big step towards getting the drug approved in Europe.
The global rheumatoid arthritis market is crowded with a range of drugs and therapies, a large number of which are the first generation antibody therapies we had discussed in this article, but as yet, an IL-6 receptor target nanobody is yet to reach commercialization. If Ablynx can bring its candidate to market, it will be first in class, and have a considerable advantage from an efficacy and safety standpoint over the currently available treatments.
By 2021, analysts expect the total global market to reach $15.2 billion. If the company picks up a European approval, an FDA campaign will be next, and success across the campaign would open up the potential for Ablynx to go after these double-digit billion dollars. As things stand, the company has a market capitalization of a little over $700 million. One to watch moving forward.