Verastem (NASDAQ:VSTM) is having its annual research and development day in a few weeks, on July 10.
Call it a catalyst to announce catalysts. We might even get some new data from the cancer-stem-cell expert.
Verastem's lead drug, VS-6063, targets a protein called focal adhesion kinase, or FAK, which is involved in cell survival, among other things. The biotech also has a backup compound, VS-4718 that targets FAK and a third drug that targets the PI3K/mTOR signaling pathway, which is also involved cell survival. Both pathways are up regulated in cancer cells, making them good potential targets.
Verastem's drugs preferentially target cancer stem cells, which are believed to be the root of reoccurrence of cancer. Cancer treatments kill a majority of the cancerous cells, shrinking the tumors, but the cancer stem cells often survive, thereby regenerating the primary tumor. The formation of metastases -- tumors at secondary sites -- may be initiated by these cancer stem cells.
When in 2015?
Without a doubt, Verastem's biggest catalyst is a clinical trial called Command, testing VS-6063 in patients with mesothelioma, a form of lung cancer. The 350 to 400 patients expected to enroll in the trial have already responded to a combination of Eli Lilly's (NYSE:LLY) Alimta and a platinum-based chemotherapy but haven't yet progressed following treatment.
Coming in as a closer -- if I can use a baseball analogy -- to finish off the remaining cells in the tumor is a smart move. Verastem doesn't have to compete directly with Eli Lilly and avoids the really hard-to-treat patients who might not respond to its treatment but doesn't have to wait for patients to progress like a typical second-line treatment.
While Command is only a phase 2 trial, it's designed to be sufficient to gain FDA approval given the unmet need in patients with mesothelioma.
Interim results for Command are expected in the middle of 2015. Management promised an update on the trial's enrollment at the research and development day, so perhaps that date could move up if enrollment is going well or maybe later if patients are responding better than expected. Investors should be careful interpreting a delay as good news, because interim data can also be delayed if the placebo group is living longer than the initial estimate or if enrollment is slower than expected.
Verastem is in a race with GlaxoSmithKline (NYSE:GSK), which also has a FAK inhibitor dubbed GSK2256098, so data that's quick and/or produces longer survival is welcome. GlaxoSmithKline's drug is still in phase 1 trials.
While useful for potential investors who know little about the company, research and development days can be relatively boring for current investors and analysts who follow the company intently.
Companies know this, and if they want to keep investors interested, they sometimes throw them a bone, saving data for the research and development day.
Verastem said previously to expect data from a few trials in the second half of the year, so we could theoretically get that data at the meeting, although it's admittedly very early in the second half of the year.
The most interesting of the three is an interim analysis for a phase 2 trial of VS-6063 in patients with Kras-mutated lung cancer. Lung tumors with Kras mutations are hard to treat, but Verastem hopes that tumors that also contain a mutation in checkpoint proteins p16 and p53 will make the tumors more susceptible to treatment with a FAK inhibitor. The trial will report separate results for Kras alone, in combination with either p16 or p53, and a combination of the three, although the latter is pretty rare.
It's a bit of a high-risk, high-reward proposition, but showing that VS-6063 can work in other tumor types would give investors confidence that sales won't be limited to mesothelioma.
Verastem has also guided for interim data from a phase 1 trial of VS-4718 and another testing VS-5584 in the second half of 2014, but being phase 1 trials, investors won't be able to read much into the data they produce.