What: Celator Pharmaceuticals (NASDAQ: CPXX) -- an under-the-radar cancer drugmaker -- saw its shares climb by nearly 450% in pre-market trading today on exceptionally high volume. This monster surge was triggered by the announcement that the company's experimental treatment for secondary acute myeloid leukemia (AML), Vyxeos, demonstrated a significant improvement in overall survival in a pivotal late-stage study (when compared to patients receiving the current standard of care for this indication).
Vyxeos is a novel combination of two drugs (cytarabine and daunorubicin) encased in a nano-scale liposome designed to maintain the desired formulation following administration.
So what: The outlook for AML hasn't improved in decades, despite the extremely poor prognosis for patients. In fact, 95% of AML patients aged 65 or older pass away within five years after being diagnosed, according to Cancer Research UK. In brief, Celator may have just developed the new standard of care for this devastating blood-based cancer.
Now what: Celator's management said that it plans on presenting the full data set at this year's American Society of Clinical Oncology meeting. In the meantime, the company's goal is to start putting together regulatory applications for the drug in both the EU and the U.S. In light of the deadly nature of AML and the dearth of effective treatments, my quick take is that Celator probably stands a very good chance at getting a green light from regulators later this year.
Having said that, the recent emergence of immunotherapy companies like Juno Therapeutics (NASDAQ: JUNO) may ultimately limit Vyxeos' commercial potential. After all, Juno, as well as some of its competitors, have signaled out AML as one of the first major blood-based disorders that could benefit from genetically modified T cell therapies.
While Juno and its peers are still perhaps two years away from presenting pivotal-stage data for their experimental treatments, big pharma and blue-chip biotechs alike have put their collective weight behind this approach in the hopes of moving away from chemotherapy-based treatments like Vyxeos. Until then, however, Celator should enjoy a significant first-mover advantage, assuming that its drug makes it to the market later this year or early next year.