The next twelve months promise to be a busy period in the biotechnology sector. A number of companies are about to kick off pivotal trials that – if successful – could bring new and updated treatment options to populations for which there currently exists a large unmet need.
Here are two companies to keep an eye on near term, with a look at the treatments in question and the catalysts that could get each moving.
VBI Vaccines Inc (NASDAQ:VBIV)
VBI Vaccines is a young biotechnology company that's working to bring a host of different treatments to market, most of which are rooted in a technology it calls its envelope virus like particle (eVLP) platform. The technology allows the company to create vaccine candidates (primarily preventative, but not limited to such) that stand to offer patients an improved option over the current standard of care therapies in their respective indications. The company's lead program right now is rooted in hepatitis B and it revolves around an asset called Sci-B-Vac.
Sci-B-Vac is a prophylactic vaccine that VBI hopes can compete against GlaxoSmithKline's (NYSE:GSK) Engerix-B, which is the vaccine that dominates this space right now. The latter has certain limitations (it's been shown to lack efficacy in immunosuppressed patients, for example) and Sci-B-Vac may be able to overcome these limitations and offer patients in this population an improved rate of seroprotection.
VBI has collected a large amount of safety and efficacy data from both real world administrations (the drug is already approved in a number of regions globally, but none of the major markets) and from investigator sponsored trials and the company is about to initiate a global phase III program that will underpin regulatory applications in the US, Canada and Europe. That's the plan, at least.
The program is split into two parts, one of which is further broken down into two separate (but concurrent) primary endpoints. The first part is a lot to lot study that's set up to demonstrate consistent quality. The second is a study that's designed to prove both that Sci-B-Vac is non-inferior to Engerix-B in patients ages 18 years and older (the first co-primary endpoint) and that it is superior to the latter in patients ages 45 years and older (the second co-primary endpoint).
The trial is pretty small for a pivotal in a vaccine indication, with VBI seeking to enroll just 4,800 patients. This is rooted in the above-mentioned legacy data, which the health authorities in the regions for which VBI is targeting approval with this study have suggested they are willing to accept as part of the submission.
The trial should kick off before the end of this year and it's a fifteen-month study, so catalysts derive from initiation, an interim release (which the company hasn’t confirmed, but the protocol allows for at least some safety and early efficacy data after a couple of the pre-planned three doses are administered) and, of course, completion and topline. The latter should hit press during the first or second quarter of 2019.
The second pivotal trial sees us switch from hepatitis to diabetes and, specifically, to a company called Tandem (NASDAQ:TNDM). This one might not be immediately recognizable to some reading but there's a reason we're highlighting this one as a trial to watch – it's heavily tied to diabetes giant, DexCom Inc (NASDAQ:DXCM). The latter developed and had approved its Continuous Glucose Monitor (CGM) a couple of years ago and the product is now widely used among sufferers of both type 1 and type 2 diabetes. For those not familiar with the technology, it's an implant-style asset that – as the name suggests – allows for the continuous monitoring of glucose in a patient and, when combined with certain other technologies (we'll get to one in a little more detail shortly) can provide a sort of closed loop system that helps a diabetes sufferer stay on top of their disease.
Right now, one of the companies that DexCom works with and one a company that provides the secondary component of the above-discussed system is Tandem Diabetes Care Inc. Tandem's technology, what's called the t:slim X-2 insulin pump, combines with DexCom's CGM to administer the insulin patients need as and when they need it. The CGM monitors blood glucose, notifies the t:slim pump when insulin is required and the pump does its job.
So what's under investigation?
Tandem has developed an algorithm that it believes can combine with the DexCom CGM system and its own t:slim pump to improve on the stability and – by proxy – efficacy of the system. It's called the Predictive Low Glucose Suspend (PLGS) algorithm and the way it works is pretty neat. In a nutshell, it is able to predict when blood glucose levels are about to fall and pause insulin delivery accordingly. Conversely, it can predict when blood glucose levels are about to rise and can ramp up insulin delivery in anticipation of said increase.
That's the theory, at least. Right now there's plenty of mid stage data that seems to validate the tech, but this is the US biotechnology industry and, as such, the company needs to prove efficacy and (perhaps more importantly in this instance) safety as part of a large-sample phase III before the algorithm can be built into the on-shelf product.
And it's this pivotal that qualifies the two companies for inclusion here. The trial is actually already underway, having kicked off mid-August, and it's expected to close out at some point during early 2018. It pitches the DexCom/Tandem combination against the same, but with the PLGS algorithm built in, in 90 diabetes patients ages 6 and older. The endpoint on this one is the demonstration of a reduction in the percentage of CGM values below 70mg/dL for patients using the algorithm based tech, as compared to the standard already approved system.
Beyond trial readout (which we can realistically expect as early as first quarter next year) the major catalyst on this one is the submission of a registration application to the FDA, which (assuming the trial hits its endpoint) should come the mid year 2018.
As a final note, it's worth touching on why this algorithm is important and why it could be a game changer for Dexcom and Tandem in this population. Stability of these sorts of automatic systems is tough to achieve when compared to manual administration insulin. A diabetes sufferer knows when he or she is going to eat. Right now, the technology doesn’t. This lack of predictive technology (and by proxy, the reactive nature of the system as opposed to the predictive nature) is one of the hurdles companies like Dexcom and Tandem have to overcome in order to convince patients and physicians this is the right route. If the company can prove its algorithm is effective to the above-stated aim, there's a good chance a large portion of the undecided patient population will make the switch from manual to automatic insulin.