Verastem Inc (NASDAQ:VSTM) Has Rewarded Our Readers So Far This Year

On April 19, 2017, Verastem Inc (NASDAQ:VSTM) was our biotech stock of the day. We pointed out that the company was trading at a discount to its potential market value based on a number of near-term catalysts, primarily rooted in the outcome of some ongoing clinical programs investigating the efficacy of its lead development asset – a drug called IPI – 145, or Duvelisib. At the time, the company went $1.93 a share. At last close, Verastem broke $4 to trade at $4.01 a piece – a 107% run.Here is a look at the science that underpins the mechanism of action (MOA) of the drug in question, what the data that drove this action tells us about efficacy and what we are looking for going forward as supportive of an argument that there is potential for further upside revaluation in this company.First, then, the drug.Duvelisib is what's called a dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma. That seems a bit jargony but it's not all that complicated. These are two enzymes that play a key role in the growth and survival of malignant B cells and T cells, two of the cells responsible for (and that serve as the root of) various types of lymphoma.Through the inhibition of these enzymes, the idea is that the drug can curb the proliferation of these types of malignant cells and – in turn – alter the macroenvironment that serves as key to supporting the advance of the cancers in question.It is a pretty neat MOA, but as with any fresh approach, plenty needs to be done in terms of the gathering of supportive evidence before it can gain any real traction in the market. And that is what company is doing with the trials that are currently underway. The one that we highlighted in our previous coverage, and the one that drove the recent run, was a phase II investigation looking at the safety and efficacy of the drug in a target population of patients with follicular lymphoma (FL) or small lymphocytic lymphoma (SLL). Specifically, the data related to a long-term follow-up of the patients enrolled as part of the initial study and sought to build on the efficacy first established for this early last year. And, as the numbers showed, the drug seems to work almost as well longer-term as it did short term.Notably, 83% of evaluable patients with FL treated with duvelisib had a reduction in the size of their target lymph nodes. Median duration of response was 7.9 months, median progression-free survival was 8.3 months and median overall survival was 27.8 months. In this population, that's pretty darn good.In the SLL indication, 19 of the 28 patients enrolled were responders, all of which were PRs, for an ORR of 68% as determined by an independent review committee. Responses generally occurred shortly after the start of treatment (median 2 months). Further, 100% of evaluable patients with double-refractory SLL treated with duvelisib had a reduction in the size of their target lymph nodes ( a key biomarker for severity).That's strong data and – for us - definitely,supports the drug's advance into a pivotal program in 2017. Alongside this catalyst, there are also a couple of other phase 3 trials underway investigating the asset in similar indications but through various combo and timeline doses. One is a phase III in SLL and CLL, investigating safety and efficacy in patients that experienced disease progression after treatment with either IPI-145 or Ofatumumab in a previous investigation. This one isn’t set to wrap up before the end of next year, but we should see some degree of interim analysis before the end of 2017.The second compares IPI-145 directly to Ofatumumab monotherapy in SLL/CLL patients. Again, this is a relatively long-term study, with estimated study completion date not slated until November 2021. However, primary completion should hit press in November that shape, meaning we should get some indication of efficacy during the final quarter of 2017.Cash on hand was pretty strong at the end of March this year, reported at $28 million. With no short or current debt and long-term debt of a little over $2 million, the balance sheet looks pretty good.We will be updating our subscribers as soon as we know more. For the latest updates on VSTM, sign up below!Image courtesy of Andrew Mason via FlickrDisclosure: We have no position in VSTM and have not been compensated for this article.