Since it was founded nearly five years ago, based on research from John Hopkins University, Erimos Pharmaceuticals LLC has been advancing its lead oncology product.

The Raleigh, N.C.-based company has an ongoing Phase II trial of its small-molecule compound, EM-1421, in cervical intraepithelial neoplasia (CIN), and recently filed an investigational new drug application for an intravenous formulation of EM-1421 in solid refractory tumors. Preclinical work is being completed on an oral formulation.

"We feel we are using absolutely breakthrough technology," said Neil Frazer, chief medical officer of the privately held firm, which also has an HIV focus.

Erimos was established in 2000 as Biocure Medical, a joint venture between a private investor and John Hopkins University in Baltimore, where professor Ru Chih Huang had been evaluating the possible use of nordihydroguaiaretic acid (NDGA) to treat diseases such as HIV.

"She initially had been creating a number of molecules that were antiviral, but later discovered they also exhibited anticancer activity," Frazer said, adding that a number of derivates of NDGA were tested before researchers settled on EM-1421, or tetra-O-methyl NDGA, as the safest and most selective drug candidate.

The company exclusively licensed Huang's technology, and later changed its name to Erimos, partly to avoid confusion with a similarly named firm and partly to better reflect its overall development goals. Frazer said "erimos" is a Greek word meaning "desert," the home of a plant called Larrea tridentate, the source from which NDGA originally was derived.

After licensing the technology, Erimos proceeded with a clinical trial of an intratumoral injection of EM-1421 in head and neck cancer patients. The Phase I study demonstrated safety and tolerance, and also showed visible tumor necrosis in five of the six patients.

"We began focusing on other formulations of the drug, knowing we had an efficacy of about 80 percent," Frazer told BioWorld Today. "Most of the [cancer] drugs tend to cause some kind of toxic damage, because they can't differentiate between healthy cells and cancer cells, so we've also got something that is non-toxic."

EM-1421 has three different components. First, it is designed to stop the production and activation of survivin, which is found in most cancer cells and inhibits apoptosis.

"These cancer cells have given themselves armor plating," he said, "and we want to knock out the production of survivin so the tumor cell will kill itself."

Second, EM-1421 aims at preventing cell replication, for both healthy and tumor cells, by inhibiting the protein Cdc2. The effect on healthy cells is only temporary and they will start to replicate again as soon as the drug has left the patient's system. But the tumor cells will have been stopped, because of the drug's attack on survivin, Frazer said.

EM-1421 also is designed to "interfere with the formation of multidrug resistance" pathways, he added, "which may allow it a synergy" with other complementary cancer treatments.

Erimos, which has 25 employees, is about halfway through patient recruitment in the Phase II trial in CIN, using a topical formulation of the drug, and expects to hear from the FDA by the end of May regarding its IND for intravenous administration in solid refractory tumors. The company completed a Phase I study of an intratumoral administration of EM-1421 in solid tumors, but Frazer said, "we're more interested in a systemic approach" that will affect not just the tumor, but also metastasis.

"We may seek fast-track status in refractory tumors," he added, to address the most pressing needs.

There also are tentative plans to evaluate EM-1421 in hematological cancers.

In addition to EM-1421, Erimos has the sole license opportunity for other products coming out of Huang's lab at John Hopkins, focusing on the inhibition of viral replication in HIV, human papillomavirus and herpes simplex virus, and "we'll be focusing on those in due course," Frazer said.

He said Erimos might look at partnering or licensing opportunities for intravenous EM-1421 outside of the U.S., but the company hopes to handle U.S. marketing on its own. Erimos likely will seek a partner for commercialization of the topical CIN version.

Since its inception, the company has spent about $10 million.

"We have $15 million in the bank," Frazer said, which he expects to sustain the company into the third quarter of 2006.