Staff Writer

As IDM Pharma Inc. prepares to present results of a pivotal Phase III study of mifamurtide (L-MTP-PE) in patients with lung metastases resulting from osteosarcoma, it released data Thursday from a compassionate access program showing evidence of the drug's ability to control the disease and to improve survival.

Of 27 evaluable patients, nine are alive with the disease, nine are living disease-free, and nine have died - data a company spokesman called "quite impressive" for the often fatal bone tumor that affects children and young adults. Fewer than 1,000 new cases are diagnosed in the U.S. each year, and a similar incidence of the disease occurs in Europe. According to the Children's Oncology Group, the survival of children with osteosarcoma has remained at 60 to 65 percent since the mid-1980s.

The standard treatment for osteosarcoma is tumor resection with combination chemotherapy before and after surgery. But because the disease can be resistant to traditional treatments, the company initiated a compassionate access program for patients who have not responded to other therapies. The study was open to patients diagnosed and treated with multiple other therapies but whose cancer has recurred - in other words, patients with no other options.

"There's no approved product for children's osteosarcoma, and we believe in doing the right thing for patients," Tim Walbert, president and CEO of IDM, told BioWorld Today. "We feel the right thing to do is to offer patients with no other options the benefits of this drug."

In March, the company formalized a clinical protocol with the FDA to provide L-MTP-PE to eligible, high-risk osteosarcoma patients through a compassionate access study being conducted at M.D. Anderson Cancer Center in Houston and Memorial-Sloan Kettering Cancer Center in New York.

After submitting results of a single Phase III study of mifamurtide to the FDA in an October 2006 new drug application (NDA), the company received a nonapprovable letter asking for more clinical data in August. (See BioWorld Today, Aug. 28, 2007.) Walbert said IDM plans to submit a revised NDA by the end of this year.

Walbert characterized the agency's concerns about mifamurtide as issues related to follow-up study and manufacturing. "The real issue . . . was that the company only had data from about 80 percent of patients who were in the study. So what we're doing is going back and capturing the remaining 20 percent of patients who happen to be lost to follow-up," he said.

Walbert said the company also will respond to any other issues in the nonapprovable letter, which were primarily related to manufacturing. "We expect to file an amendment by the end of this year," he said.

In the meantime, the company is preparing to present data from its pivotal Phase III study of the drug at the American Society of Clinical Oncology (ASCO) meeting in two weeks in Chicago.

According to Walbert, ASCO marks the first time that IDM will offer mifamurtide data for a "subset of more than 100 patients with metastatic disease." He added that the primary trial enrolled 687 patients who were newly diagnosed patients with nonmetastatic disease, but that the subset with metastatic disease will be the focus of the ASCO data.

Walbert said the results have been astounding for the patients enrolled in the compassionate access program. "We're reporting data from 29 of those patients," he said. "It is quite impressive in patients that have no other options that nine patients are alive with disease and nine are living disease-free." Nine patients have died, and results were not reported for two patients. In addition, L-MTP-PE in combination with other therapies was shown to be safe and well-tolerated in patients.

According to Walbert, the compassionate access study presented more than an opportunity to supplement data supporting mifamurtide; it also offered a chance to reach a desperate segment of the diseased population.

"It was an opportunity to bring a novel therapeutic that improves survival into a very sick population," he said.

And he is optimistic that data from the compassionate access program, combined with the revelations offered at ASCO, will help the company get clearance for this life-changing drug.

"We hope to complete the regulatory process and bring this new therapy to market," Walbert said.