Washington Editor

Seizing opportunity from the failure of others, Neumedicines Inc. is breathing new life into Interleukin-12 (IL-12), a biologic once seen as the great hope for cancer.

The Pasadena, Calif.-based company launched a Phase I safety trial Thursday for its lead candidate, HemaMax, a key regulator of the hematopoietic system. Initially, the trial will enroll about 40 subjects, but Neumedicines plans to expand its safety database to cover up to 500 volunteers from diverse populations, said Lena Basile, company president.

The recombinant human IL-12 is being developed as a radiation therapy under a $13.2 million contract with the Biomedical Advanced Research and Development Authority (BARDA). (See BioWorld Today, April 19, 2011.)

The trial is a personal triumph for Basile, who left her job as a patent adviser at the University of Southern California (USC) to develop the promising protein and start up Neumedicines.

While at USC, Basile had seen how the biologic performed in preclinical studies and was impressed. When an angel investor pulled out of a USC spinout that was developing the drug, she stepped up. With no funding and only an exclusive license from the university, the patent attorney/molecular biologist became the president of her own biotech.

Basile's first steps were filing a patent and writing a grant, her first, for funding from the National Cancer Institute. With a grant in hand, Neumedicines became operational at the end of 2004.

Originally, the private company intended to develop HemaMax as an adjuvant to chemotherapy, Basile told BioWorld Today. Although it's further along in its development of the biologic as a radiation therapy, the company is still pursuing cancer indications.

"We're basically just forging ahead," Basile said.

In the early days of biologics, several companies were working with IL-12, seeing it as a promising cancer therapy. But not understanding the power of this new type of drug, researchers used the same kind of dosing regimen they'd use with pharmaceuticals.

"This was a new era for biologics," Basile said, adding that researchers didn't understand then how powerful they were.

Those early IL-12 studies proved that too much of a good biologic can be bad, causing some biotechs to abandon the protein. But it wasn't IL-12 that was wrong; it was the approach. "We all learn from the past," Basile said.

Neumedicines' approach is single, low doses.

Early studies showed that HemaMax, in low doses, can protect the hematopoietic system by mitigating the bone marrow damage caused by acute ionizing radiation and regenerating multilineage hematopoiesis following lethal radiation exposure.

A survival rate of 90 percent was seen in a study in which mice were treated with a single dose of HemaMax 24 hours after they had been exposed to 8 Gy of radiation.

In addition to fighting the effects of radiation, IL-12 has antitumor effects. There's no drug right now that can do both, Basile said.

Neumedicines has big plans for its lead candidate. The company is working on the protocol for a Phase I cancer study with the START Center in San Antonio and hopes to file an investigational new drug application in the fall for pancreatic cancer.

The company also expects to get a second BARDA contract this summer that will take HemaMax through pivotal studies to approval of a biologic license application.

Radiation therapies generally are developed using the animal rule because of ethical concerns about exposing human subjects to radiation. However, Basile is looking into testing the efficacy of HemaMax in cancer patients getting palliative radiation therapy. Such patients are in the last stages of terminal cancer; they turn to radiation to help them deal with the pain.

The therapy involves exposing half the body to radiation doses similar to what could be experienced in a radiation disaster.

Looking back at the short history of her company, Basile said it has moved along rapidly in its development of HemaMax. "We're rather unique, and brave, in doing that," she added.

With its recombinant IL-12 hitting clinical development, Neumedicines is now nursing along its next candidate, a protein targeting glioblastoma.