We are midway through the week and we had a whole host of major inputs hit press in the biotechnology space over the last few days. The Labor Day extended weekend muted volume somewhat as the fresh week kicked off, but the downtick and given us plenty to discuss heading into the bell on Thursday.
So, here goes.
The two companies that we will be focusing on for the session today are Verastem, Inc. (NASDAQ:VSTM) and Voyager Therapeutics, Inc. (NASDAQ:VYGR).
First up, then, Verastem.
Both of today’s focus companies are leaving on the back of trial data and in the case of Verastem, the data derives from a phase 3 trial called DUO. The trial was set up to investigate the safety and efficacy of Verastem’s lead development asset, a drug called duvelisib, which the company is trying to get approved in a target indication of patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL).
There aren’t many treatments on the market for this indication right now and, especially in this late stage population, prognosis isn’t great once one or two of the first and second line treatments have failed. Verastem’s aim is to provide an option where that currently isn’t one and – with the recent data having just hit press – looks as though the company may be well on its way to doing just that.
As per the latest release, the trial met its primary endpoint of progression free survival (PFS), achieving a statistically significant improvement in median PFS of 13.3 months, compared to 9.9 months for ofatumumab with a hazard ratio (HR) of 0.52 (p<0.0001), representing a 48% reduction in the risk of progression or death.
In other words, the drug seems to work better than standard of care treatment and, not only that, but as the numbers illustrate, significantly so. That went any immediate safety concerns associated with dosing (at least not outside those that would normally be expected with this sort of treatment) and the forward pathway looks relatively clear for Verastem and duvelisib.
So, on that note, what’s next?
The company expects to submit a New Drug Application (NDA) to the FDA during the first half of 2018. As far as applications go, this one is one of the more low risk from an investor standpoint. Not only because the data was so positive, but because of the lack of options in this space right now and the potential difference a fresh option might lead to patients.
Moving on, let’s look at Voyager.
As noted above, both of today’s focus companies moving on the back of fresh trial data and, in this instance, the numbers derived from a phase 1B study of a drug called VY-AADC01. It is a Parkinson’s disease drug and the trial in question sought to demonstrate that it could be both safe and effective when used as a treatment in patients with an advanced form of the disease. From a mechanism of action point of view, the drug seeks to improve motor function in patients while simultaneously reducing the necessity for said patients to take levodopa, which is the standard of care treatment in this space right now, but is also notorious for bringing about serious and unwanted side effects.
And as per the news, again, it seems that drug dose exactly as both the company and its shareholders (and the target population, of course) were hoping it would. The data showed a mean 42% reduction in Parkinson’s medications at six months compared with baseline in the highest (Cohort 3) of three doses.
Parkinson’s is an incredibly difficult condition to treat very few new treatments have come to market in this space over the past 50 years. If Voyager can get VY-AADC01 green lighted by the FDA in the US, it will join a very exclusive club of biotech companies and markets are recognizing this fact in their response to the news. The company is up close to 25% heading into the session on Thursday and looks set to continue to appreciate as the weekend approaches.
The next step is a phase 2/3 pivotal trial, which Voyager expects to initiate before the end of the year, with first patient dosing slated for the first quarter of next year.