Tiny Celator surges on promising cancer data and preps an FDA submission

Celator Pharmaceuticals ($CPXX) saw its shares roughly quintuple in value on Monday afternoon after the biotech posted positive Phase III results for its nano-powered blood cancer therapy, putting the tiny company in line for an FDA filing this year.

The Ewing, NJ, company's lead drug, CPX-351, is a 5:1 formulation of the standard cancer treatments cytarabine and daunorubicin, packaged in a nano-scale delivery platform that Celator says can boost efficacy while limiting the risks of administering each drug on its own.

Now the company has Phase III data to back up that claim. In a trial on 309 patients with high-risk acute myeloid leukemia, Celator tested CPX-351 against standard cytarabine and daunorubicin therapy, finding that its combo treatment notched a statistically significant effect on overall survival, meeting the study's primary endpoint.

Patients in the treatment arm posted a median survival rate of 9.56 months, besting the 5.95 months observed in the other group, Celator said. CPX-351 patients also demonstrated a 31% reduction in risk of death compared with the old drugs, according to the company, and Celator's therapy came through with a statistically significant improvement in response rate.

On the safety side, Celator said the rates of serious side effects were comparable across the two study arms, and CPX-351 charted a lower rate of all-cause mortality than cytarabine and daunorubicin after 60 days. Celator said it's holding off on disclosing detailed safety and efficacy data until it can present its findings at the American Society of Clinical Oncology meeting in Chicago this summer.

The data are particularly impressive considering that acute myeloid leukemia, or AML, has a dismal survival rate, and fewer than 10% of patients with high-risk forms of the disease live more than 5 years after diagnosis, according to Celator. Furthermore, if CPX-351's top-line results survive future scrutiny, Celator will have done something no small-cap biotech has ever done: successfully execute a Phase III trial and break the Feuerstein-Ratain Rule.

The data sent Celator's shares, trading at $1.68 as of Monday's close, up as much as 500% postmarket before they settled at around 3.5 times their earlier value. The company, which reported having just 20 employees in its last annual report, is now planning to file CPX-351 for FDA approval in AML this year.

- read the results (PDF)

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