While the company completes a confirmatory Phase III trial of Ceplene, Maxim Pharmaceuticals Inc. received a vote of confidence from the FDA this week when the agency gave permission to provide the drug as a combination therapy for the most critically ill malignant melanoma patients.
The FDA allows companies to distribute their drugs before approval in cases in which a disease is serious and life threatening, and the patients have no satisfactory treatment alternatives. The drug to be used also must be in a controlled clinical trial and the company must be actively seeking marketing approval.
"These programs are also referred to often as expanded-access programs and in some cases compassionate-use programs," said Larry Stambaugh, Maxim's CEO, president and chairman, in a conference call Wednesday. "This is a program that initiated with the agency in the late 1980s, and there have been only 11 cancer drugs that have been under the treatment protocol since that time. And 10 of those have been approved subsequently."
On the news, Maxim's stock (NASDAQ:MAXM) rose 27 cents on Wednesday, to close at $8.80.
Ceplene is in a confirmatory Phase III trial being evaluated in combination with interleukin-2. The company will file a new drug application amendment in early 2005 following the trial's results, expected later this year.
Maxim initially filed the NDA in 2000 based on its first Phase III study in advanced malignant melanoma. The FDA determined in January 2001 that the company needed to do the second Phase III trial, which Maxim began in 2002.
Maxim completed enrollment in September of its confirmatory Phase III trial of Ceplene in combination with IL-2 to treat advanced malignant melanoma patients with liver metastases. The 230-patient trial is comparing patient survival of the combination treatment vs. IL-2 alone.
The FDA allowed Maxim to provide the expanded access based on clinical findings indicating that the Ceplene combination therapy might provide a survival benefit to critically ill patients who face limited treatment options.
"Of course the goal for any cancer research company, including Maxim, is to break through the long-term survival and we are very determined about that goal," Stambaugh said.
The agency also approved reimbursement for study medication, allowing the company to recover some of the $1.5 million in costs of providing qualified patients access to the therapy over this fiscal year.
The treatment will be available to eligible patients in the U.S. initially in 10 geographic locations. There will be no control group as part of the expanded-access protocol - all patients will receive treatment. The protocol will be expanded beyond the 10 sites to additional qualified treatment centers in subsequent months. And the National Organization of Rare Diseases is working with Maxim to provide treatment for indigent patients.
About 200,000 people in the U.S. and 150,000 in Europe have advanced malignant melanoma - the most deadly form of skin cancer. The cases of the disease have more than doubled since 1980. Currently, there are two approved therapies for the disease - DTIC chemotherapy, and immunotherapy with bolus high-dose IL-2, which is highly toxic.
Ceplene (histamine dihydrochloride) has orphan drug status in the U.S. It has been tested in seven clinical trials for malignant melanoma and in more than 1,000 patients. Ceplene is based on the naturally occurring molecule histamine, and has been shown to reduce oxidative stress. Treatment with Ceplene may prevent the production and release of oxygen free radicals, protecting NK cells and T cells, as well as other critical cells and tissues.
The company filed a marketing authorization application in Europe last November.