Shares of Celldex Therapeutics (CLDX) were up as much as 16% yesterday after the company announced that it had received breakthrough therapy designation for its cancer drug Rintega (rindopepimut). Rindopepimut is a drug that is used to treat patients with Glioblastoma Multiforme -- GBM. GBM is a cancer of the brain and it produces devastating problems for the patient's body. Such problems include: Seizures, nausea, vomiting, and severe headaches. The patients to be treated with rindopepimut are those 30% of GBM patients who have a mutation variant known as EFGRVIII. This EFGRVIII mutation variant protein is detected in glioblastoma cancer patients but is not detectable at all in healthy cells. Therefore this protein target is ideal to treat these patients by using immunotherapy. Rindopepimut activates the patient's immune system and then calls out to target the EFGRVIII mutation variant.

Celldex has already seen great success with rindopepimut. The company achieved its FDA breakthrough status with the positive results seen in its phase 2 study. Three phase 2 studies have been done to date and all three clinical trials showed significant improvement in overall median survival and progression-free survival. The drug is even safe, as the only adverse effects seen by the drug are fatigue and a rash. This is not bad considering that standard of care treatments have unbearable side effects. Celldex makes note that there have only been three other drugs approved for Glioblastoma over the last 20 years. Therefore there is a demonstrable need for drug like this that will not only help improve patients' quality of life but may even extend their lives for longer periods of time.

As the name suggests, FDA Breakthrough Status is only given to biotech companies that prove that theirs is an improvement over other therapeutic drugs approved in the market and that they treat a large unmet medical need. Rindopepimut achieved Breakthrough Status despite so many other biotech companies failing. Since the FDA breatkthrough status designation program there have been 275 applications. About 77 requests were granted for Breakthrough status while the other 144 requests were denied. We will have to wait and see how the phase 3 trials pan out but at the moment Celldex is on track with rindopepimut in addition to its other pipeline drug products. The positive results seen thus far makes Celldex a very attractive long-term cancer biotech investment.