For information on current clinical trials, please go to http://clinicaltrials.gov/ and type in “PEP02″ in the search box.
MM-398 is a novel, stable nanotherapeutic encapsulation, or enclosed sphere carrying an active drug, of the marketed chemotherapy drug irinotecan. MM-398 recently achieved its primary efficacy endpoints in Phase 2 clinical trials in pancreatic and gastric cancer. In the recently completed open label, single arm Phase 2 clinical trial of MM-398 as a monotherapy in 40 metastatic pancreatic cancer patients who had previously failed treatment with gemcitabine, patients treated with MM-398 achieved median overall survival of 22.4 weeks. Additionally, 20% of the patients in this Phase 2 trial survived for more than one year, and Merrimack observed a disease control rate, meaning patients exhibited stable disease or partial or complete response to treatment, of 47.5% at six weeks. There are currently no approved treatments for gemcitabine refractory metastatic pancreatic cancer, nor is there a consensus on standard of care treatment for such patients.
Merrimack plans to initiate a pivotal Phase 3 clinical trial of MM-398 for the treatment of patients with metastatic pancreatic cancer who have previously failed treatment with gemcitabine by the end of 2011. The trial is expected to enroll approximately 250 patients and is designed to compare the efficacy of MM-398 as a monotherapy against the combination of the chemotherapy drugs fluorouracil, or 5-FU, and leucovorin, a regimen often used by physicians to treat this patient population. Merrimack believes that MM-398 has potential uses in a number of other indications, including colorectal cancer, lung cancer, gastric cancer and glioma. There are multiple ongoing Phase 1 and Phase 2 clinical trials of MM-398.
In July 2011, the U.S. Food and Drug Administration, or FDA, granted MM-398 orphan drug designation for the treatment of pancreatic cancer. In the United States, orphan drug designation is granted to a drug intended to treat a rare disease or condition, which is generally defined as a disease or condition that affects fewer than 200,000 individuals in the United States. If MM-398 receives the first FDA approval for the disease for which it has such designation, it is entitled to orphan drug exclusivity, which means that the FDA may not approve any other applications to market the same drug for the same indication, except in very limited circumstances, for seven years.