What: After management reported impressive late stage trial results for its treatment for acute myeloid leukemia (AML), shares of Celator Pharmaceuticals (UNKNOWN:CPXX.DL) were sent sky-rocketing 483.6% in March, according to data from S&P Global Market Intelligence.
So what: The clinical stage drugmaker's Vyxeos delivered impressive efficacy and similar efficacy in a phase 3 study evaluating its use against the current standard of care in AML patients.
Specifically, in a phase 3 trial involving 309 people, the overall survival of secondary AML patients receiving Vyxeos was 9.56 months, which was much better than the 5.95 months survival for patients receiving the standard of care 7+3 chemotherapy regimen.
Those trial results are impressive, especially considering that Vyxeos is an optimized 5:1 synergistic ratio of the two drugs that are used in the 7+3 regimen: cytarabine and daunorubicin.
Also encouraging is the fact that Celator Pharmaceuticals formulation of this two drug cocktail delivered similar safety to the 7 + 3 approach, suggesting that doctors will have little objection to replacing the 7 + 3 regimen with this new therapy, if it notches regulatory approval.
Now what: Vyxeos could be the most significant advance in this tough-to-treat patient population in thirty years and as such, investors are probably right to assume that a rapid shift away from 7 + 3 to it could occur if the FDA gives it a green light.
Celator Pharmaceuticals management plans to file for U.S. approval of Vyxeos in the third quarter and then file for its approval in the EU early next year. Although there's no guarantee that regulators will approve Vyxeos, I think its efficacy and safety profile is good enough for it to happen. If I'm right, then Celator Pharmaceuticals could begin reporting meaningful sales of Vyxeos by as early as late next year.